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Pharma R&D

Annual Review 2024

Look back at key pipeline trends from the last
12 months so you can forecast success in 2024

Contents
Welcome03

Introduction: Total Pipeline Size
Pleasant outlook for pharma as things continue to heat up 

05

Developing Systems: The 2024 Pipeline by Phase
Clinical development advances still face a stiff headwind 

08

Top Companies			Some enjoy a moment in the sun, but for others the future looks more hazy 

11Therapies and Diseases 											 19Cancer still taking R&D by storm 
Regional Variations												 31Where the outlook seems to be brightest
Mechanisms and Targets
Immuno-oncology makes hay while the sun shines

54

Types of Pipeline Drugs
Is the continued move into biotech starting to meet a frosty reception?

59

What’s the Forecast for Pharma?		
Plenty of rays of sunshine should hold off any storm clouds on the horizon 

62

About the Author 

65

Welcome
Welcome to Pharmaprojects’ 2024 review of trends inpharmaceutical R&D
Welcome to Pharmaprojects’ 2024 reviewof trends in pharmaceutical R&D. For over
30 years now, I’ve been taking an annual
look at the evolution of pharma R&D, and in
this article I’ll take the temperature of theindustry at the start of 2024. We’ll assess
industry trends by examining the pipeline bycompany, therapeutic area, disease, target,and drug type, using data primarily fromPharmaprojects, part of the Citeline suiteof products, which has been tracking global
drug development since 1980. This report will
be followed up by our annual supplement
reviewing the New Active Substance (NAS)launches for the year just passed. But here,
we’ll examine how the pharma R&D climate ischanging, look at where the pressure is risingand falling, and try to determine where theoutlook is sunny and where storm clouds aregathering. Hopefully, it will help you to takethe industry’s temperature and forecast
its outlook.
Regular readers of this report (which has beenrunning since 1993, so is presented here in its
32nd edition) will know that, in recent years, I’ve
threaded a different theme through each edition,
to highlight points, to draw analogies, and to
add a little character into what could otherwise
be a rather lengthy narrative through a paradeof statistics, charts, and tables — duller than a
December day in Doncaster. Themes selected so
far have included astronomy, movies, the natural
world, music, food and drink, science fiction,
travel, and, last year, literature. This year, as you
may have guessed, I decided to hang my (rain)
hat on something that affects us all every day:the weather, and the allied most important issueof our times, climate change.

3

Pharma R&D Annual Review 2024

Since our early cave-dwelling days, the activitiesof humans have been somewhat governedby the vagaries of the weather. We needed toknow when conditions were best for hunting,
and whether our crops would flourish or fail. The
earliest attempts to predict the weather werelargely unscientific and based on prophecy,
astrology, or prayers to invisible weather gods.
As with so many things, the ancient Greeks were
the first to turn superstition into science, andin fact the term “meteorology” comes from theGreek meaning “the study of things high in theair.” The acknowledged father of the scienceis Aristotle, who published a treatise on thesubject as early as 350 BC. Of course, accurate
instruments to measure weather-related
phenomena only really came along with the
Enlightenment, but weather forecasts were still
notoriously unreliable, especially in countries likethe UK where I am based, as little as 50 years
ago. It was only with the growth of computing
power, and with it, computer modelingalgorithms, that forecasters were able to provide
predictions with any measure of certainty. The
key, as always, is data — and lots of it. My hopeis that this report will fulfill the same function:
it will provide enough information for you todetermine which parts of the industry arelikely to be basking in unbroken sunshine,
and which are preparing for a period of
unsettled conditions.

Copyright © 2024 Citeline

While our primary focus is naturally on the
short term; i.e., whether we need to put on an
extra jumper or to bring our umbrellas, thelong-term trends are what exercise the mindsof both climate scientists and pharma industry
followers. In the past few decades, the topic of
the weather has grown from being a parochialconsideration to something threatening our very
existence. But, in fact, the idea that our impact
on the planet is changing it irrevocably has beenaround longer than you might think. It was at
the end of the 19th century when scientists firstpredicted that changes in atmospheric carbon
dioxide levels could substantially alter a planet’s
surface temperature through the greenhouseeffect, and just prior to World War II when Guy
Callendar connected carbon dioxide increases
in Earth’s own atmosphere to global warming.
Particularly since the turn of the millennium, theevidence seems irrefutable that, unless action istaken and taken fast, we are building towards a
threat to our continued existence. The pharma
industry has of course weathered a more acuteexistential threat recently — the COVID-19
pandemic, which it helped to deliver us from —and has emerged to enjoy a moment in the sun.
But both climate change and the coronavirus
crisis are evidence of the fact that we need to
identify — and act on — emerging trends very
quickly. We have the data; it’s what we do withit that counts.
Of course, changes in climate patterns predate
humans and the transformational effect of theindustrial revolution. Within the past million
years, there have been at least eight cycles of
ice ages and warmer periods, along with shorter
cycles such as the Little Ice Age in the North
Atlantic between the 16th and 19th centuries,
which famously led to London’s River Thames
freezing over and the Victorian “Frost Fairs” held
there. While climate is bound to have inherent
variability, this change has been variously
attributed to cyclical lows in solar radiation,heightened volcanic activity, changes in ocean
circulation, and variations in Earth’s orbit andaxial tilt, so the human race hasn’t always
historically been behind climate change. It’s
clear now, however, that our effect extends far
beyond such natural cycles, and is potentiallysending our planet towards irreversible disaster,at least as far as life is concerned. It’s theorizedthat other planets such as Mars have alreadybeen through this process, and that the deadplanet we see today is potentially a spooky view
into our own future.

4

Pharma R&D Annual Review 2024

The pharma industry, as we shall see, has been
heating up for some time now, despite the miniice age which COVID presented. Within that
overall trend, though, there are ever-shifting
patterns, more complex than weather pressure
charts. Drug development has its own areas ofhigh and low pressure, its own fronts; in someplaces, it is experiencing headwinds, in others,
the air is calm; and some therapeutics are mostdefinitely hotter than others. In this report, we
will present to you our meteorological map ofpharmaceutical R&D as it is now, and comparethat to our charts from a year ago, to get thebest view of where things are warming up andwhere a deep depression may be setting in.
We will also occasionally look further back todefine longer-term trends; after all, you don’t
always just need to know whether it will rain
tomorrow, you need to know whether to packyour sunscreen or your Wellington boots for yourforthcoming holiday. In that sense, our pharma
industry review will try to provide both a weather
forecast and an insight into broader changes in
the climate.
I hope this introduction has broken the ice andset you up to ride along the trade winds of data
we have on today’s pharma industry. So, takea rain check from whatever you’re doing, find aplace in the sun, and read on to find out where
the industry is basking in unbroken sunshine,
and where the outlook is more overcast. We
won’t try to boil the ocean, but we will provide
a clear view to the horizon so that you can see
which way the wind is blowing in drug R&D.
We’ve cooked up a veritable storm of statistics
and will help you to navigate any choppy waters
they stir up. Hopefully, after reading this report,
understanding drug R&D trends will be a breeze.

Copyright © 2024 CitelineIntroduction: Total Pipeline Size
Pleasant outlook for pharma as things continue to heat up
As a foreword to this year’s almanac, let’s
define the climate system in which ourchanging weather patterns analyses willtake place, by looking at the total numberof drugs currently in the R&D pipeline. All
the analyses in this report will be focusing
on this set of drugs, so it’s worth starting
off with a definition of what exactly wemean by the term “pipeline.” Here, weare counting all drugs in development bypharmaceutical companies, from those at
the preclinical stage, through the various
stages of clinical testing and regulatory
approval, and up to and including launch.
Launched drugs are still counted, but
only if they are still in development for
additional indications or markets. Drugs
whose development has been terminated,
or is complete, are not included. All data
were collected on Jan. 2, 2024.

We’ve also consistently seen increases in thenumber of drugs in development year-onyear — is “pharma warming” still occurring in
2024? Well, Figure 1 confirms that the overall
pipeline size has again hit a new high, andthe rate of expansion is on the up again.
2024’s headline figure is 22,825 drugs, an
expansion of 7.20% from last year’s figureof 21,292. The rate of growth represents anincrease from the previous year’s 5.89%,
which places the 2024 rate nicely in the
middle of that rate and the year before(8.22%). It also places this year’s rate prettymuch dead on the average of the past five
years, which is 7.14%. So it would seem that
this year is at least right on trend.

Are things still heating up in pharma R&D,
as well as the climate? Records for extremesof high temperature have repeatedly beenbroken in recent years. In the UK, thesummer of 2022 saw the mercury peakat 40.3°C (104.5°F), while Australia hit
its own record just a few months earlier,hitting a blistering 50.7°C (123.3°F).
Perhaps surprisingly, the highest verifiabletemperature humans have recorded globallythus far dates back over a century to
July 1913, when the appropriately named
Furnace Creek, California, recorded a facemelting 56.7°C (134°F). Globally, all 10 of
the warmest years on record have occurred
post-2000, with 2023 recently confirmed
to be the warmest yet. It’s now well
documented that the overall trend for global
temperatures is climbing at an alarmingly
accelerating rate, with average global
temperatures already having increased byabout 1°C (1.7°F) since 1880, and projected
to warm by 1.5°C (2.7°F) by 2050, andpotentially by 2–4°C (3.6–7.2°F) by 2100.

5Pharma R&D Annual Review 2024

Copyright © 2024 CitelineFigure1: Total R&D pipeline size by year, 2001­­—­24
25,00022,825
21,292
20,10920,00018,582
17,737DRUG COUNT

16,181
14,872

15,00015,267

13,71812,30011,307
9,605 9,737 9,713

10,00010,452 10,479

9,217

6,416
5,995 6,198

6,994

7,360 7,406

7,737

5,000

0
24
20

23
20

22
20

21
20

20
20

19
20

18
20

17
20

16
20

15
20

14
20

13
20

12
20

In real terms, the size of the pipeline has thusincreased by 1,533 drugs, representing a bigger
increase than the 1,183 seen during 2022–23,
but almost exactly the same as the 1,527 uptick
reported over 2021–22. But this doesn’t tell thewhole story. During 2023, a total of 5,428 drugs
were added to the Pharmaprojects database,considerably more than the 5,082 added during
the previous year. So how come the total
pipeline size only grew by 1,533? The answer isthat a total of 3,895 candidates left the active
pipeline during the same period, a figure which
again almost exactly matches the one from theprevious year. This indicates that, once again,
there has been considerable turnover within
the pipeline in the course of a single year,
but the amount of change has at least
remained constant.

11
20

10
20

09
20

08
20

07
20

06
20

05
20

04
20

03
20

02
20

01
20

Source: Pharmaprojects®, January 2024

YEAR

the big hitters point to a slightly broader spreadof candidates by therapeutic area joining drug
companies’ portfolios last year; as with weather,a mixed, balanced picture is healthier, rather
than too much of one feature causing a flood
or a drought somewhere else.

Let’s look a little deeper into the types of drugs
that entered the pipeline during 2023. By far
the largest chunk of drugs fall into the oncology
bucket, which provided 38.0% of all new
candidates — interestingly, a little down from2022’s 40.7%. Neurologicals came in second,with 12.7%, also down from 13.5% the previous
year. Taken together, these slight declines for

6

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineThere might be some subtle shifts in winddirection this year, which are exemplified bya look at which companies added the most
new drugs to the pipeline. This year, Pfizer
tops the list with 32 new candidates, pushing
Jiangsu Hengrui Pharmaceuticals into second
place with 30. The leading Chinese firm topped
the equivalent ranking last year, somewhat
eclipsing the more traditional Western big
pharmas. However, in terms of the location ofdevelopment of 2023’s new intake of drugs,
there seems further evidence of China’s
continued advance. The US is still the preeminent territory this year, with 1,856 new
candidates (up from 1,840), but China is closing
the gap, with 1,627 neophyte drugs, up from
1,457 in 2022. The varying strengths of Easternand Western trade winds is a subject we’ll be
returning to later in this report.
However, just as ever-warming temperatures
might lead to nicer summers but have somepotential downsides (droughts, forest fires,the total extinction of the human race, etc.),

7

Pharma R&D Annual Review 2024

continuously increasing drug R&D pipeline sizes
are not wholly a good thing. As we’ll see shortly,
the vast majority of the drugs in this number
are not yet — and, in the majority of cases, will
never be — launched. Thus, they are a cost to theindustry, and a risk. Might the bloated pipeline
reach a tipping point where it no longer becomessustainable, in the same way that global warming
might? Just as switching to renewable energy
sources might save the planet, the way in which
the industry renews itself and keeps progressing
is by successfully launching new drugs, whichearn revenue. We are still gathering and finessing
data on NAS launches for 2023 as I write, but
early indications are it’s likely that, while it wasa less successful year than 2021, which produced
a record haul on 97 NAS launches, it surpassed
2022’s 74 to become the second best year
ever. Be sure to check out the forthcoming NAS
Supplement for the final tally, and confirmationof whether the pharma industry is continuing
to reap a sizable harvest, or whether drought
conditions might be setting in.

Copyright © 2024 CitelineDeveloping Systems: The 2024 Pipeline by Phase
Clinical development advances still face a stiff headwind
Weather, like new drugs, doesn’t justappear fully formed — it develops. And,
like a healthy human, it relies on many
interrelated pieces working together tofunction. The large-scale structure of the
Earth’s atmospheric circulation systems,
like the drug development process,remains fairly constant, but smaller-scale
systems such as mid-latitude depressions
or tropical convective cells occur
chaotically and are impossible to predict
accurately beyond around 10 days ahead.
Chaos theory plays a large part in climate
modeling algorithms and unpredictability
is innate, in much the same way as wecannot predict accurately whether a drug in
preclinical development will make it throughthe development process. There are as
many variables along the path to produce a
successfully marketed drug versus a failed
one as there are between a weather systemdeveloping into a hurricane versus a spellof light drizzle. In the pursuit of maximizing
success in both fields, increases in computer
power to better model such variables are an
important step forward.

8

Pharma R&D Annual Review 2024

Drug development of course follows anorderly pattern, as candidates move frompreclinical development, through the various
phases of clinical trials, into registration, and(hopefully) finally onto the market. But justas the sun rising into a cloudless sky doesn’tmean it will be fine all day, it might stayclear, or conditions might deteriorate; a drug
can have a clear run to market but, more
often than not, it will get lost in the fog and,before you know it, it’s rain stopped play.
Let’s take a look at how the pipeline breaks
down by phase of development, and try to
determine whether the outlook is improving.

Copyright © 2024 CitelineFigure 2: Pipeline by development phase, 2024 vs. 2023
14,00012,48512,00011,835DRUG COUNT

10,000

8,000

6,000

3,703

4,000

3,263

3,131

3,374

2,000

1,425
1,297

1,229 1,2480PreclinPhase I

Phase II

Phase III

254 270

135 174

Pre-reg

Registered2023

63 120

Launched

Suspended

85

85

N/A

2024

(N/A = not applicable and is applied to companion diagnostics prelaunch)
Source: Pharmaprojects®, January 2024

Figure 2 breaks down the 2024 pipeline by the
drugs’ current global statuses. Global statusis the most advanced stage of developmenta drug has reached in any country, for any
disease, and by any company, so each drug iscounted only once here. You can immediately
see that a considerable amount of the increasein pipeline size growth occurred by adding
drugs at the preclinical stage, which posted a
total up by 650. However, in percentage terms,
this is a 5.5% increase; lower than the overall
rate of increase in the pipeline as a whole, buthigher than 2023’s equivalent number of 4.3%.
In percentage terms, it is the number of drugs
in Phase I which has increased the most, risingby an impressive 13.5%, accelerating from the
10.7% increase reported last year. Rises at Phase
II also showed a positive trend, with the numberof drugs at that phase up by 7.8%, compared to
7.2% last year. Note that while the numbers inPhase I being higher than those in Phase II may
at first seem counterintuitive, this is a functionof these data being a snapshot in time. The factthat there are roughly the same numbers of
drugs in Phase II as there are in Phase I in noway means that virtually all drugs undergoing
9

Pharma R&D Annual Review 2024

Phase I then progress serenely to Phase II.
There is considerable attrition between the twophases, but as Phase II development generally
takes much longer, drugs pile up at the Phase IIstage, so that at any one time, there are moredrugs in Phase I than there are in Phase II.

Copyright © 2024 CitelineBut there is cause for concern when we move to
the Phase III number, which seems in contrast
somewhat becalmed. It’s wiser here to takea deeper dive into our historical records, as
Figure 3 pulls out the numbers of drugs in theclinical development stages across a longer time
period, going back to 2007. You can see here
how the increases in Phase III numbers during

the 2017–21 period failed to keep pace withequivalent rises seen in Phase I and II, indicating
worsening attrition. But the sun had appeared
to be breaking through the clouds here during2022 and 2023, with Phase III numbers on the up
again. This year’s data indicate darkening skies
again, which could be a cause for concern for
the industry.

Figure 3: Clinical phase trends, 2007–24
4,000
3,703

2007

3,500

2008

3,374

3,263

2009

3,131

2010

3,000

DRUG COUNT

2011
2012

2,500

2013
2014

2,000

2015
2016

1,500

1,248
1,229

1,000

2017
2018
2019
2020
2021

500

2022
0

2023
Phase I

Source: Pharmaprojects®, January 2024

Phase II

Phase III

2024

CLINICAL STAGE OF DEVELOPMENT

It’s undoubtedly true that if you are spending
more on pushing more drugs through Phase
I and Phase II trials, you want more drugs
to make it into Phase III. However, there isa caveat to this. Phase III trials are by somemeasure the most expensive part of drug
development, so it’s only a good thing tohave more drugs in Phase III if the industry isconsequently able to bring more drugs to themarket. Indeed, data from industry analysts
such as Deloitte suggest that the year-on-year
cost of developing drugs continues to rise,
while return on investment is falling. This, likethe ongoing rise in global temperatures, clearlycannot be sustainable.

10

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

Top Companies
Some enjoy a moment in the sun, but
for others the future looks more hazy

Which companies in 2024 are enjoying extensive sunny periods, and which are undergoinga sudden cold snap? In this section, we’ll take a look at the leading companies enjoying fairweather, plus the industry as a whole, which is composed not just of big fluffy clouds, but many
tiny raindrops. Like the atmosphere, the pharma industry is composed of a number of distinct
layers, and we’ll be peering through the fog at each.
Table 1: Top 25 pharma companies by size of pipeline
POSITION
2024(2023)COMPANYNO. OF DRUGS IN
PIPELINE 2024 (2023)

NO. OF ORIGINATEDDRUGS 2024 (2023)

TREND

1 (1)

Roche

218 (194)

125

↔

2 (5)

Pfizer205 (171)

127

↑

3 (7)

AstraZeneca

166 (155)103↔

4 (10)Eli Lilly

159 (135)

90

↑

5 (4)

Bristol-Myers Squibb

158 (175)

90

↓

6 (2)

Novartis154 (191)

96

↓

7 (6)

Johnson & Johnson

150 (156)81↓

8 (13)

Jiangsu Hengrui
Pharmaceuticals

147 (106)

138

↑↑

9 (8)

Merck & Co.

145 (151)69↓

10 (9)

Sanofi142 (145)

79↓

11 (11)

GlaxoSmithKline

138 (123)70↔

12 (3)

Takeda130 (178)50↓

13 (12)

AbbVie111 (122)

46↓

14 (14)

Boehringer Ingelheim

111 (99)80↔

15 (27)

Sino Biopharmaceutical

103 (60)84↑↑

16 (15)Bayer

91 (93)61↔

17 (24)

Shanghai Fosun
Pharmaceutical (Group)

90 (64)62↑

18 (16)

Gilead Sciences

86 (86)46↔

19 (17)

Otsuka Holdings

85 (85)45↔

20 (20)Eisai

81 (74)46↔

21 (18)Amgen

79 (79)47↔

22 (26)

Astellas Pharma

78 (63)40↑

23 (19)Novo Nordisk

76 (77)53↔

24 (23)CSPC Pharmaceutical73 (68)60↔

25 (21)

Regeneron71 (73)53↔

Source: Pharmaprojects®, January 2024
11

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

The upper level of the atmosphere is the
exosphere. At this rarified level, there’s noweather as such, but the lower parts canhost that most spectacular of atmosphericphenomena, the aurora. Shimmering on theupper echelons of the pharma industry are theTop 25 companies by pipeline size, which arelisted in Table 1. Shining brightest for the second
year in a row is the Swiss-based multinational
Roche, which deposed its Basel-based neighbor
Novartis last year. The latter slips notably thisyear with a pipeline which has considerably
slimmed down. Taking the runner-up position
this year is Pfizer, whose portfolio is about 20%
bigger than it was this time last year, a factwhich can largely be attributed to its completionof its acquisition of Seagen (the former Seattle
Genetics) in December 2023. In a year which
saw an unusual degree of turbulence in theupper atmosphere, it’s just one of the significant
changes in a top 10 which, in recent years, has
exhibited a high level of stability. AstraZeneca
hops up to number 3, having itself acquired acouple of small firms, Neogene Therapeutics
and CinCor Pharma, while Eli Lilly shoots up
from number 10 to number 4, having taken over
Versanis Bio, DICE Therapeutics, and Sigilon
Therapeutics during a particularly acquisitive
year (it also subsumed POINT Biopharma,
although that completed too late in the year
to be included in the Jan. 2, 2024 data cut).
Away from this year’s top four, though, therest of last year’s top 10 all report pipelines ofsmaller size this year. While Bristol Myers Squibb
and Johnson & Johnson steered clear of M&A
activity during 2023, Novartis, Merck & Co.,
Sanofi, and most dramatically Takeda report
smaller pipelines despite having acquired some(admittedly small) companies last year.

Most manufactured satellites orbit in theexosphere, and some of the larger and morereflective ones are sometimes visible fromthe Earth’s surface as they catch the sunlight.
Undoubtedly, one of the brightest “stars” inthe pharma exosphere this year has to be
Jiangsu Hengrui Pharmaceuticals. The company
becomes the first ever Chinese firm to makeit into the top 10 in terms of pipeline size,recording a 38.7% increase in its pipeline to burst
into the upper firmament at number 8. It also
has by some measure the biggest proportionof “homemade” drugs, with almost 94% of its
drugs being self-originated. In fact, by this lattermeasure it is at number 1, having more drugs
in the pipeline which it discovered itself thanany other company, period. This is a landmark
moment for the Chinese pharmaceuticalindustry, which, 20 years ago, had little to nohomegrown pharma R&D focused on originalcompounds.
The next level of the atmosphere is thethermosphere, another rarified layer devoid ofclouds and water vapor, but where you will findthe International Space Station. Our next level,
the companies numbering 11 to 25 by pipelinesize, also has some interesting features in it, andagain it’s China that is lighting up the sky here.
Sino Biopharmaceutical leapfrogs its compatriot
Shanghai Fosun to become the country’s
second biggest R&D powerhouse, bursting into
the upper echelons this year with a massive
72% increase in its pipeline size, all via organic
growth. Similarly, Shanghai Fosun also reported
decent growth with a 41% bigger R&D portfolio.
These were by far the most significant changes
in companies at this level; most other firms had
pipelines of pretty similar sizes to last year.
With the possible exception of Pfizer’s Seagen
acquisition, our Top 25 is not really affectedsignificantly by M&A activity through 2023.
Overall, M&A activity across the industry
appears to have slightly risen from last year’s
nadir, with 88 mergers and acquisitions reportedby Pharmaprojects to have closed during 2023,
slightly up from the 81 noted in 2022. There has
now been a long period of calm weather after
the stormy period of the late 1990s and early
2000s when tempestuous megamergers ofpharmaceutical giants were commonplace.
Like storms, these big mergers may havecleared the air, but many ultimately may
have inflicted significant damage.

12

Pharma R&D Annual Review 2024

Copyright © 2024 Citelineminnows comprise a large part of the industry.
Figure 4 shows that these minute, emerging
companies produce just over 18% of the drugs
in development, a slightly bigger slice than lastyear, but roughly the same percentage as 10
years ago. This is a larger tranche than either theTop 10 or the Top 25 companies. It’s interesting
to note, however, that this year seems to havewitnessed a small reversal to the long-term
trend whereby the biggest pharmaceutical firms
were contributing smaller and smaller shares ofdrugs to the overall pipeline. The Top 10 are now
providing 4.4% of drugs, up from 4.1%, while theTop 25 are delivering 8.2%, rising from 7.8%.

Most of the Earth’s weather occurs in the lowest
level of the atmosphere, the troposphere. Almost
all clouds that are generated by weather arefound here, with the exception of cumulonimbus
thunder clouds, whose tops can rise into thelowest parts of the neighboring stratosphere.
It’s perhaps surprising how much activity occurs
at the lowest level of the pharma industry too­­— at those companies that are only developing
one or two drugs. This year, we are reporting 931
companies with only two drugs in their pipelines(up from 825 last year), and a huge 2,249 (upfrom 2,083) with just a single candidate. Just
as it takes many tiny water droplets to makeup a large cloud, taken together, these pharma

Figure 4: Share of the pipeline contributed by top 10 companies,
top 25 companies, and companies with just one or two drugs, 2011—24
Top 10Top 25Companies with 1 or 2 drugs% OF TOTAL PIPELINE

24

19

14

9

4
2011

2012 2013 2014 2015 2016 2017 2018 2019 2020 2021

Source: Pharmaprojects®, January 2024

13

Pharma R&D Annual Review 2024

2022 2023 2024

YEAR

Copyright © 2024 Citeline

The Top 10 companies are continuing to display
interests across a wide variety of therapeuticareas, as illustrated in Figure 5. In fact, thetop five firms have drugs in development inevery single one of the 14 therapeutic areas,
while numbers 6 to 10 only miss out on oneof the smaller areas each (antiparasitics
for one, hormonal drugs for the other four).
This indicates that, despite received wisdom
suggesting that companies are refining
their efforts to specialize in just a handful of
disciplines, for the very largest companies atleast, this simply isn’t the case. There are someinteresting diversifications within that, however,with Eli Lilly once again bucking the trend by
being the only Top 10 company that doesn’t
have anticancers as its largest therapeutic focus(it instead prefers alimentary/metabolic drugs).
Meanwhile, Bristol Myers Squibb is the company
with the most skewed pipeline, with nearly 50%
of its drugs having an oncology focus, whereasinterest in other areas languishes at single-digit
percentages. Sanofi almost matches its cancer
portfolio size with that of its anti-infectives,with Pfizer and Merck & Co. also having sizable
portfolios in that therapeutic area.
Figure 5: Disease focus areas of the Top 10 pharma companies

2024
100%
90%
80%
70%
60%
50%
40%
30%
20%
10%
0%
Pﬁzer

Roche

AstraZeneca

Novartis

Bristol-Myers
Squibb

Sanoﬁ

Eli Lilly

TakedaJohnson& Johnson

Merck
& Co.

Alimentary/Metabolic

Anticancer

Anti-infective

Antiparasitic

Blood and Clotting

Cardiovascular

Dermatological

Genitourinary (including sex hormones)

Hormonal (excluding sex hormones)

Immunological

Musculoskeletal

Neurological

Respiratory

Sensory

Source: Pharmaprojects®, January 2024

14

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineRare diseases also continue to excite thoseat the helms of the biggest pharma beasts,
with all the Top 10 companies featuring in ourtable of the 20 companies with the most drugs
against rare diseases in their pipelines (Table
2). Novartis comes out on top by this measure,pushing Pfizer back into second place. Novartis
has the second-highest proportion of its pipeline
focused on rare diseases, its 53.6% being justedged out by Biogen’s 53.7%. Elsewhere, only
Bristol Myers Squibb also breaks the 50% barrier.
At the other end of the scale, the company
with the lowest percentage is the previously
highlighted Chinese company Jiangsu Hengrui,with just 24.8%. Eli Lilly comes pretty close tothat figure, with just 25.4% of its pipeline hittingrare diseases, further cementing the view thatthe company is something of an anomaly
among the big hitters.
Table 2: Top 20 pharma companies with a rare disease focus
COMPANYNUMBER OF DRUGSFOR RARE DISEASES% OF PIPELINE

Novartis

118

53.6

Pfizer112

40.1

Bristol-Myers Squibb

110

50.9

Roche

102

36.8

Sanofi85

40.5

AstraZeneca

81

36.2

Takeda80

39.8

Johnson & Johnson

71

37.0

GlaxoSmithKline

68

36.0

Merck & Co.

59

30.9

AbbVie

58

37.2

Amgen

52

46.8

Eli Lilly

51

25.4

Eisai

42

45.7

Bayer

40

34.2

Astellas Pharma

39

37.1

Jiangsu Hengrui Pharmaceuticals

37

24.8

Biogen36

53.7

Otsuka Holdings

36

34.6

Sino Biopharmaceutical

36

34.3

Source: Pharmaprojects®, January 2024

15

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

The total number of companies involved in thepharma R&D ecosystem has now hit 6,124,
as evidenced by Figure 6, continuing anotherupward trend. Unfortunately, a calculation
error means that the equivalent figure for 2023
published last year was incorrect, so we have
replaced this with an estimated total of 5,700.
If accurate, this would give us a growth in the
total number of companies developing drugs of
7.4%. Either way, it’s clear that the sun continues
to shine
7,000 on the industry, with no shortage of newcompanies bubbling up in its sky. We can say that

there were definitely more companies entering
the fray during 2023, with 921 being added toour database, considerably more than the 809
discovered the previous year. This would suggestthat around 500 companies were removed fromour database over the past 12 months, either
having been acquired, having folded, or, in the
majority of cases, seemingly having become
inactive. It’s possible that, for some of thesecompanies, while their current status is somewhat
hazy, they will yet emerge from the mist andrejoin the active set for next year.

Figure 6: Total number of companies with active pipelines, 2001—24
6,124
6,000
5,700
5,416
5,099
5,000

4,816

COMPANY COUNT

4,323
4,134
4,003

4,000
3,687

3,286
2,984

3,000
2,705

2,745

2,387
2,207
1,965

2,000

2,084

1,769
1,503
1,198

1,633
1,576 1,621

1,313

1,000

0
2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023 2024

*Estimated figure
Source: Pharmaprojects®, January 2024
16

Pharma R&D Annual Review 2024

YEAR

Copyright © 2024 Citeline

Which way is the wind blowing in terms ofgeographies for drug development? Figure
7 suggests that the pharma pressure map
indicates that the breeze is continuing to flowfrom west to east. We can see that, while 43%of all companies involved in pharma R&D a
year ago were headquartered in the US, thisfigure has taken a significant dip in 2024 to 39%.
Meanwhile, the proportion of China-based firms

has increased once again, up from 13% to 16%.
This represents an acceleration in the latter’s
growth, with 1,008 Chinese-headquarteredcompanies reported this year, up from 808 lastyear and 792 the year before. Whereas lastyear we had suggested that growth in Chinamight be slowing, in 2024, there appears to be astrengthening of the air currents in this direction
from gentle to gusty.

Figure 7: Distribution of R&D companies by HQ country/region, 2023 and 2024
1%
4%
13%

3%
2%
3%

2023

43%

USA
UK

11%

Rest of Europe
Rest of Asia-Paciﬁc
15%

5%

Japan

1%

Germany

3%France16%

China
Canada

2024

3%

39%

2%

C and S America/Africa

3%

13%
5%
15%
Source: Pharmaprojects®, January 2024

17

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

Similar trends can be observed in Table 3, which
examines the locations where drug development
is actually occurring. While the US still dominates,
its presence is in decline. In all, 49.1% of all drugs
in development have some US R&D activity; thisis down from 51.1% last year, and falls below the
50% mark for the first time. Again, this groundhas been ceded largely to China, which increases
its share from 23.6% to 26.7%. Another Asian
country, South Korea, has snuck into third place,pushing the UK into fourth. Note that the table
only lists countries where 5% or more of drugs
are in development. In total, our databasereports active R&D in 162 countries this year.

The data for 2024 reinforce trends seen overa number of years in that R&D is experiencing
an easterly airflow. This year, we have our first
Chinese Top 10 pharma company, and the US
losing its majority as the favored place forpharma R&D. Our forecast here is that suchclimactic changes are likely to continue forseveral years to come.

Table 3: Where is R&D actually occurring?
DRUG DEVELOPMENT COUNTRYNUMBER OF DRUGS% OF PIPELINEUSA

11,200

49.1

China

6,098

26.7

South Korea

3,233

14.2

UK

3,156

13.8

Germany

2,479

10.9

Canada

2,387

10.5

Australia

2,372

10.4

France

2,363

10.4

Spain

2,259

9.9

Japan

2,041

8.9

Netherlands

1,850

8.1

Italy

1,823

8.0

Belgium

1,783

7.8

Poland

1,724

7.6

Sweden1,562

6.8

Denmark

1,536

6.7

Switzerland

1,504

6.6

Czech Republic

1,406

6.2

Hungary

1,398

6.1

Austria

1,369

6.0

Taiwan, China

1,362

6.0

Bulgaria

1,244

5.5

Finland

1,178

5.2

Portugal

1,174

5.1

Greece1,171

5.1

Ireland

1,158

5.1

Romania

1,153

5.1

Israel

1,147

5.0

Norway1,146

5.0

Source: Pharmaprojects®, January 2024

18

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineTherapies and Diseases
Cancer still taking R&D by storm

However, over the years we have added both tothe broad therapeutic areas and to the individual
250+ therapeutic categories to more closely reflectmodern drug development.

How many different types of weather are there?
There doesn’t seem to be an internationallyagreed answer, and even in the various weather
apps you probably have on your phone, there
will probably be some discord. Try this for abasic list: sunny, partly cloudy, cloudy, drizzle,
rain, stormy, foggy, windy, snowy. But, ofcourse, many of these are not mutually
exclusive. Similarly, drug development for
disease types can cross multiple therapeuticareas, and we likewise assign them into a
number of big buckets.

We start our analysis in this area by looking at how
numbers have changed year-on-year across those
14 therapeutic areas, along with the two additionalclasses that we created, biotechnology products
and reformulations. This shows anticancer drugs
reaching stratospheric levels again, with 9,142
drugs in development for oncology, up from 8,480
last year, an expansion of 7.8%, slightly ahead of
the global pipeline expansion rate we reported
earlier in this review. Neurologicals also marginallybeat the average, with a 7.5% growth rate, but
alimentary/metabolics put in a really impressive
performance, swelling the pipeline by 13.1%.
Meanwhile, growth in anti-infective R&D, which
saw a boost during the COVID-19 pandemic but
had since fallen to a below-average growth rate,
has now almost stalled completely, increasing bya paltry 0.8% this year.

In this section, we move to looking at trends
across the 14 major therapeutic area types forwhich drugs are under development. Just likewith weather, there is no internationally agreed
classification for therapeutic areas, with several
ontologies such as ICD-10 and MeSH coexisting.
Our own system has its origins in the EphMRA
classification system, which itself is closelyaligned with the well-known ATC code taxonomy.

Figure 8: R&D pipeline by therapeutic area, 2023 and 2024
10,000
9,142

9,100

9,000
8,480

8,394

8,000

7,000

6,000

5,000

4,000

3,713
3,319
2,933

3,000

2,951
2,927

2,066

2,000

2,210

2,074
1,914
1,453
1,262

1,000

1,219
1,130

1,215
1,143

1,126

1,157
988

1,087
783

820
750

741

Source: Pharmaprojects®, January 2024
19

Pharma R&D Annual Review 2024

2023

275

H
or
m
on
al

ar
G
en
ito
ur
in
ar
y
Bl
oo
d
&
Cl
ot
tin
g

Ca
rd
io
va
sc
ul

Re
sp
ira
to
ry

Se
ns
or
y

An
tic
an
ce
r

Bi
ot
ec
hn
ol
og
y
N
eu
ro
lo
Al
gi
im
ca
en
l
ta
ry
/M
et
ab
ol
ic
An
tiIn
fe
ct
iv
e
Re
fo
ru
m
ul
at
io
ns
M
us
cu
lo
sk
el
et
al
Im
m
un
ol
og
ic
al
D
er
m
at
ol
og
ic
al

247

0

110

112

An
tip
ar
as
iti
c

3,454

2024

Copyright © 2024 Citeline

Oncology’s above-average result has meant afurther grab of its overall share of the pipeline,
which Figure 9 shows. However, the rate at which
its advance has slowed might be some comfort
for the other therapeutic areas, which must beconcerned to see their metaphorical polar ice
caps continuing to recede.

Figure 9: Proportion of the pipeline in development for cancer, 2010—24% OF PIPELINE WHICH IS ONCOLOGY

45.0

40.0

39
36.7

39.8

40.1

2023

2024

37.5

35.2
34.1

35.0
32.6

29.5

30.0

30.1

30.4
29.3

29.3

2014

2015

28
26.8
25.0

20.0
2010

2011

2012

2013

Source: Pharmaprojects®, January 2024

20

Pharma R&D Annual Review 2024

2016

2017

2018

2019

2020

2021

2022

YEAR

Copyright © 2024 Citeline

Table 4 zooms in on this analysis further,
providing the Top 25 of the 243 individual
therapeutic categories used to classify drugs
in Pharmaprojects and the Citeline suite ofproducts. Unsurprisingly, there are big boosts forthe top two anticancer categories: anticancer,
immunological (+8.2%); and anticancer, other(+8.6%). Gene therapy again comes in third,

but there is further evidence of interest here
peaking, with a growth rate of just 3.3% thisyear, down from 6.3% last year and 23.3% theyear before. The Top 10 of this chart is once
again pretty stable, as if an anticyclone has
settled over therapeutic category changes,
leading to a long period of calm weather.

Table 4: Top therapeutic categories
POSITION 2024
(2023)

THERAPY

NUMBER OF ACTIVECOMPOUNDS 2024 (2023)

TREND

1 (1)

Anticancer, immunological

4,860 (4,492)

↑↑

2 (2)

Anticancer, other

3,935 (3,622)

↑↑

3 (3)

Gene therapy

2,151 (2,083)

↔

4 (4)

Monoclonal antibody, other

1,595 (1,395)

↑

5 (6)

Neurological

1,125 (1,045)

↔

6 (5)

Prophylactic vaccine, anti-infective

1,115 (1,064)

↔

7 (7)

Ophthalmological, other

1,054 (984)

↔

8 (8)

Antiviral, other935 (983)

↓

9 (9)

Immunosuppressant899 (797)↑

10 (10)

Cellular therapy, chimaeric antigen
receptor

795 (792)↔

11 (11)

Antidiabetic

772 (747)↔

12 (14)

Musculoskeletal

767 (677)↑

13 (12)Anti-inflammatory

747 (722)↔

14 (13)

GI inflammatory/bowel disorders

740 (705)↔

15 (15)

Cognition enhancer

702 (641)↑

16 (17)

Monoclonal antibody, humanized

672 (624)↑

17 (16)

Respiratory

654 (632)↔

18 (18)

Cardiovascular

645 (599)↑

19 (19)Neuroprotective

632 (595)↔

20 (20)Hepatoprotective

618 (594)↔

21 (21)

Dermatological589 (559)↔

22 (22)

Urological578 (519)↑

23 (24)

Antiparkinsonian

570 (516)↑

24 (32)

Antibody-drug conjugate

551 (405)↑↑

25 (23)

Analgesic, other547 (517)↔

Source: Pharmaprojects®, January 2024

21

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineDespite the fact that gene therapy and celltherapy growth appears from this table tobe slowing, they both remain major driversof drug development. Figure 10 shows this,but also reinforces the fact that their rates of
expansion are slowing down from the frankly
remarkable snowballing of R&D seen in the last
decade. Of course, these two categorizations
are not mutually exclusive; indeed, 1,192 drugs
are classified as both — these are generally
therapeutics whereby cells are removed fromthe body, genetically altered ex vivo, and thenreintroduced to the patient (such as with CAR-T
therapies). This means there are 959 genetherapies which are not also cell therapies;
these will be primarily in vivo gene therapies,
where genes are delivered in vivo to cells via a
vector, or where genes are edited using newer,
cutting-edge technologies (of which more later).
This figure has grown from 886 last year. Celltherapies without a gene therapy component
now stand at 916, up from 852.
Figure 10: The ongoing rise of gene and cell therapies

2,151
2,108

1,589
1,140

1,273

1,427

1,500

722
610

22

564

479

Source: Pharmaprojects®, January 2024

Pharma R&D Annual Review 2024

2024

2023

2022

2021

2020

2018

2017

2016

238

2014

327

220
276

2013

302

232
242

2012

2015

206
209

2011

217

2010

168

239
193

2009

223
194

2008

191
150

2007

204
117

2006

196
93

2005

208
106

2004

275
122

2003

255
115

2002

249
64

2001

221
54

2000

191
54

1999

158
39

1998

142
32

1997

19

1996

114

16

1995

99

416

500

0

687

867

Cell
862

Gene

1,000

2019

NUMBER OF DRUGS

1,836

2,000

2,083
2,048

1,960
1,936

2,500

YEAR

Copyright © 2024 Citeline

Within the umbrella term of gene therapy, amultitude of different technologies are covered,with one major variation being how the genetic
change is introduced into cells. Whereas
sometimes this is achieved via chemical
methods or using plasmids, by far the mostcommon method is to use a viral vector. The
breakdown of the viral vector types is shown in
Figure 11, which further breaks each down by
global phase of development. You can see here
that, by some measure, adeno-associated viral(AAV) vectors are both the most popular andthe most mature. They account for just over 500

projects, including two of the six launched genetherapies using viral vectors. They also havesignificant numbers of drugs at Phase I, II, andIII, although much of the work here is still at the
preclinical stage of development. Lentiviruses
come in second; this type of vector is morecommonly used for ex vivo transfection of cells.
Whereas the number of projects using AAV
vectors has increased this year, lentivirus use isin decline, with 185 drugs this year, down from
241. The use of adenovirus took an even bigger
tumble, falling from 109 to 43. AAV seems to be
the weapon of choice.

Figure 11: Viral vectors used in gene therapies
600

500

400

300

200

100

0
Adeno-associatedvirusPreclinical

Lentivirus

Phase I Clinical Trial

Adenovirus

Retrovirus

Phase II Clinical Trial

Herpes simplex
virus

Phase III Clinical Trial

Poxvirus

Pre-registration

Polyomavirus

Registered

Rhabdovirus

Launched

Suspended

Source: Pharmaprojects®, January 2024

23

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

The types of cells used for cell therapy are
considerably more diverse, with in total 24
different kinds of cells currently being employed.
T cells remain the most popular, due to theirpredominance in the popular CAR-T-based
therapeutics, although there are somewhat
fewer this year. In contrast, the temperatureis rising for stem cells, which have posted
a modest increase, along with blood cells
and antigen-presenting cells. Figure 12 has
the details.
Figure 12: Cell types used in cell therapies
900
800
700
600
500
400
300
200
100

Phase II Clinical Trial

Phase III Clinical Trial

ce
ll
sp
ec
iﬁ
ed

ce
ll
ow
m
ar
r

Pa
nc
r

ea
tic

ce
ll

ce
ll
Pr
og
en
ito
r

Pre-registration

O
th
er

Phase I Clinical Trial

Bo
ne

Preclinical

Ex
os
om
e

An
ce
ll
tig
en
-p
re
se
nt
in
g
ce
Co
ll
nn
ec
tiv
e
tis
su
e
ce
ll

Bl
oo
d

ille
rc
el
l
al
k

ce
ll
N
at
ur

St
em

T

ce
ll

0

Registered

Launched

Suspended

Source: Pharmaprojects®, January 2024

24

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

We now move to what is always one of the mostinteresting Top 25s — the individual diseases on
which the pharmaceutical industry is focusing its
attention most. These are listed in Table 5, whereit should be noted that only specific diseases
are listed; non-specific indications such as“Cancer, unspecified” have been removed
from this analysis.

Table 5: Top 25 diseases/indications
POSITION 2024
(2023)

THERAPY

NUMBER OF ACTIVECOMPOUNDS 2024 (2023)

TREND

1 (1)

Cancer, breast

1,031 (965)

↑

2 (2)

Cancer, lung, non-small cell

1,010 (925)

↑↑

3 (3)

Cancer, colorectal

825 (741)

↑↑

4 (4)

Cancer, pancreatic

740 (675)

↑

5 (6)

Cancer, ovarian

625 (587)

↔

6 (5)

Cancer, brain

580 (539)

↑

7 (7)

Infection, coronavirus, novel
coronavirus

580 (653)

↓

8 (8)

Cancer, prostate

576 (523)

↑

9 (9)

Alzheimer's disease

563 (529)↔

10 (10)Cancer, liver

501 (444)↑

11 (11)Diabetes, Type 2

498 (475)↔

12 (14)

Cancer, leukaemia, acute
myelogenous

497 (484)↔

13 (12)

Cancer, melanoma

496 (476)↔

14 (13)Cancer, gastrointestinal, stomach

473 (449)↔

15 (15)Cancer, myeloma

459 (445)↔

16 (17)

Parkinson's disease457 (407)↑

17 (16)Arthritis, rheumatoid

449 (431)↔

18 (18)Cancer, head and neck432 (408)↔

19 (19)Infection, coronavirus, novel
coronavirus prophylaxis

425 (444)↓

20 (20)Cancer, lymphoma, non-Hodgkin's

419 (399)↔

21 (21)

Non-alcoholic steatohepatitis

383 (369)↔

22 (22)

Psoriasis

348 (337)↔

23 (24)

Obesity300 (230)↑↑

24 (32)

Cancer, renal

299 (281)↔

25 (23)

COVID-19 complications

285 (332)↓

Source: Pharmaprojects®, January 2024

Breast cancer remains the hottest place on
the temperature chart, with a pipeline that has
warmed up by a further 6.8%, slightly below theoverall expansion rate. As a result, it is in dangerof being overtaken by second-placed non-small
cell lung cancer, which itself advanced by 9.2%.
Even more impressively, at number 3, the size

25

Pharma R&D Annual Review 2024of the colorectal cancer pipeline has risen by
11.3%. Tumors of the pancreas, the ovary, andthe brain round off an all cancer top six this year.
The incursion made into the top five by the novel
coronavirus has now ended, with this indication
dropping to number 7 with a pipeline thatshrank by 11.2%. Nonetheless, with 580 drugs in

Copyright © 2024 Citelinedevelopment against it, there is still significant
effort being put into the ongoing battle with thevirus, which thankfully has now become endemic
rather than pandemic.
Elsewhere, that eternal tough nut to crack,
Alzheimer’s disease, remains as the only othernon-oncology disease in the Top 10, which alsosees a new entrant in the form of liver cancer,
up a significant 12.8%. Further down in the Top
25, we see declines for two further COVID-19related categories, those covering vaccinesand complication therapies; but again, there
is still considerable activity in both. Perhaps
the most striking change in an otherwise fairly
stable table is the 30% increase in the R&D ofdrugs to treat obesity. This is no doubt related
to the success of Novo Nordisk’s Wegovy(semaglutide), which was first launched for thisadditional indication in mid-2021, setting off
a stampede for access that has led to supply
issues. Note that this global picture masks someregional variations in the most popular diseasetargets for drug R&D, which we will look at laterin this report.
While sunshine, cloudy skies, or rain showers
are commonplace for most of us, there aresome weather events which are thankfully
rarer. 2023 saw a number of freak weather

events across the globe: temperatures plunging
to -28°C (-18.4°F) in Afghanistan, floodingin California and South Korea, heat-induced
wildfires in Canada and Hawaii, and a recordbreaking cyclone in Brazil. Climate change
appears to increase the frequency of extreme
weather, a concern for us all. For most of us,
such catastrophic weather events can still beconsidered rare. But they nevertheless quite
rightly grab our attention.
There is also a big focus on rare diseases inpharma R&D. Such syndromes may not affectmany of us, but for those unfortunate enoughto suffer from them, they can be similarly
devastating. This, and the big paydays pharma
can command for successfully developing drugs
for orphan indications, means that rare diseases
are a major focus for the pharmaceuticalindustry too. In Pharmaprojects, a rare diseaseis defined as one with a prevalence of 1 in
2,000 people in the EU, or affecting fewer than
200,000 people in the US (equivalent to around
1 in 1,600 people). Figure 13 demonstrates thatthe number of individual rare diseases being
targeted by drug development continues togrow, hitting 761 by the end of 2023, with slightly
more new additions to this pot over the courseof that year than during the previous one.

Figure 13: Number of rare diseases being targeted by pharma
2023

761

2022

718

2021

677

YEAR

2020

648

2019

608

2018

580

2017

546

2016

509

2015

476

2014

440

2013

389
0

100

Source: Pharmaprojects®, January 2024

26

Pharma R&D Annual Review 2024

200

300

400

500

600

700

800

NUMBER OF NEWLY IDENTIFIED TARGETS

Copyright © 2024 Citeline

In total, the number of drugs under development
for rare diseases at the start of 2024 hit 7,191,
up 7.6% from last year’s 6,682, and representing
31.5% of all drugs in the pipeline, up a shadefrom 30.2% in 2023. As Figure 14 shows, thelargest proportion of rare diseases, 19%, sit in
the alimentary/metabolic area — this is not
surprising, since there are many rare inherited
genetic conditions that cause disorders of themetabolism. In contrast, cancer is only the fourthmost common therapeutic area for rare diseases.
Despite this, it still commands the most attention
in terms of drugs under development, with 39%
of all drugs in R&D for rare diseases being fora rare cancer. By this measure, alimentary/
metabolic is pushed to joint third place, witha paltry 7% of the effort sitting there.

Figure 14: Rare diseases by therapeutic area, by number of diseases and number of drugs
Rare Diseases by TA

Sensory Products

4%

3%

Alimentary/Metabolic Products

19%

Respiratory Products

Anticancer Products
16%

Anti-infective Products

Neurological Products

Antiparasitic Products

Blood and Clotting Products
12%

Musculoskeletal Products

Cardiovascular Products
9%

Dermatological Products

Immunological Products

Genitourinary Products (including sex hormones)
6%

Hormonal Products (excluding sex hormones)

13%
2%

2%

Hormonal Products (excluding

Immunological Products
3%

4%

2%

5%

Musculoskeletal Products

Genitourinary Products (includ

Neurological Products

Drugs for Rare Diseases by Project4%

Respiratory Products

Dermatological
Products
Sensory
Products

Sensory Products

7%

7%

Cardiovascular
Products
Respiratory
Products
Blood and Clotting
Products
Neurological
Products

12%

39%
5%

Anti-infective Products
Immunological
Products

7%

1%
2%
1%

Antiparasitic Products
Musculoskeletal
Products

AnticancerProducts
Products(excluding s
Hormonal
2%

5%

6%
2%

Alimentary/Metabolic
Genitourinary
ProductsProducts(includi

Source: Pharmaprojects®, January 2024

27

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

Like all drug R&D, most of the rare disease
work is at the preclinical stage, some of it at
the cutting edge of development techniques.
But there is considerable clinical activity too.
Data from Trialtrove, another product in the
Citeline suite that tracks individual clinical trials,
shows that there were 2,430 clinical trial starts
associated with rare disease-focused drugs
during 2023 — up from 2022’s figure, but bya somewhat lower percentage than in theprevious 12 months (Figure 15).
Figure 15: Industry-sponsored rare disease trials by start date, 2010—present

NUMBER OF TRIALS
3,000

2430

2,500

2335

2,000
1704
1490

1,500
1236

1298

1202

1261

1807

1845

2018

2019

1747

1862

1540

1325

1,000
sex hormones)

ng sex hormones)
500

0
2010

2011

2012

2013

2014

2015

2016

2017

2020

2021

2022

2023

Source: Trialtrove®, January 2024

28

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

While we’re on the subject of clinical trial
statistics rather than drugs, let’s take a look at
all of the ongoing (open, closed, or temporarilyclosed) clinical trials reported by Trialtrove
during 2023. Figure 16 breaks this down by
trial phase and by therapeutic area (note that
Trialtrove uses slightly different therapeuticareas from Pharmaprojects). The dominanceof cancer is far more pronounced here, skewed
by the fact that oncological candidates may
often be tested in multiple clinical trials acrossdifferent cancer types. There are 18,966 trials
under way in cancer at the start of 2024,

a 7.7% rise on 2023’s figure, representing aslight slowdown in the growth rate from lastyear’s 8.7%. CNS in second posted a similar
growth rate of 7.6%. But infectious disease inthird reported a decline of 3.1%, the strongest
evidence yet that the spike caused by the
COVID crisis is well and truly over (note that
infectious disease vaccines was the only othertherapeutic area to show a drop). Indeed, thenumber of novel coronavirus-related ongoingtrials took a tumble this year, standing at 2,016,
down from 2,384 last year.

Figure 16: Ongoing clinical trials, by therapeutic area

2024
20,00018,00016,00014,00012,00010,000
8,000
6,000
4,000
2,000

I/II

II

II/III

III

III/IV

IV

O
ph
th
al
m
ol
og
y

(I
D
)
Va
cc
in
es

I

G
en
ito
ur
in
ar
y

M
D
et
ise
ab
as
ol
e
ic
/E
nd
Au
oc
rin
to
im
ol
og
m
un
y
e/
In
ﬂa
m
m
at
io
n
Ca
rd
io
va
sc
ul
ar

CN
S

In
fe
ct
io
us

O
nc
ol

og
y

0

Source: Trialtrove®, January 2024

29

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

The number of approvals of emergency
authorization — a strategy which came to thefore during the pandemic — fell back too lastyear. Figures for this metric, shown in Figure
17, would seem to exactly track the course of
the crisis. The amount of orphan drug and
expedited review designations also dropped
slightly in 2023.

Figure 17: Numbers of drugs receiving Orphan Drug status, Expedited Review designation*,and Emergency Authorization**, 2013—23
500
434

450
394

NUMBER OF DRUGS

400
350
300

299

369

348

326
250

250
200

157

182

425

269

397

381

378

367

338

341

298

316

302

188

150
100
50

50
1

0
2013

2014

2015

2016

2017

2018

2019

24
2020

39

2021

22
2022

2023

YEAR
Orphan Drug Status Granted

Expedited Review Status Granted

Emergency Authorization Granted*Data for 2013 not complete as we only began systematically recording the dates of these events mid-year.
**Emergency Authorizations only tracked from 2019.
Source: Pharmaprojects®, January 2024

So, we seem to be entering a sunshine-after-therain period of drug development, as the cloudsof COVID, if not exactly dissipating, appear tobe scooting out to sea towards the horizon. And
the horizons for drug R&D are still expanding;there are 1,514 individual diseases being
targeted by active drug development this year,
up from 1,452 a year ago. The pandemic actually
accelerated progress in drug development ina number of areas, such as the use of mRNAbased vaccine technology. In that sense, thecoronavirus-shaped cloud really does seem tohave had a silver lining.

30

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

Regional
Variations
Where the outlook
seems to be brightest

While understanding changes in
global climate is undoubtedly important,such data are not likely to be of much
use in helping you to decide whether
you can wear shorts tomorrow orwhether you need to bring a woolly
hat. So, new for this year’s report, weare introducing several regional subanalyses, looking at pipeline size growth
rates, leading companies, and top
diseases across a range of some of thelargest pharmaceutical markets acrossthe world. Is it sunny in China, buttipping it down across Europe?
Let’s find out…

Source: Pharmaprojects®, January 2024
Pharma R&D Annual Review 2024
31

Copyright © 2024 CitelineUnited States
It seems prudent to start our regional analyses in the largest of the world’s pharma markets, theUS. The number of drugs in active development in the US has consistently grown since 1995, withan average pipeline increase of 284.8 drugs per year, corresponding to 4.8% year-on-year (YoY)
growth. While the number of active pipeline drugs continues to increase in the US, the rateof growth has decelerated.
From 2023 to 2024, there was an increase of 393 active drugs in the US pipeline, equivalent to 3.6%
YoY growth. This is around half the overall global pipeline growth rate of 7.3%. The US has historically
been a powerhouse of drug development, but, as we have seen, has been ceding some ground inrecent years. Since 2021, the US’s annual pipeline growth rate has been well below the worldwide
growth rate (2.9 percentage points [pp] lower in 2021–22, 4.4pp lower in 2022–23, and 3.7pp lower
in 2023–24).

11,197

10,804

9,477

2023

8,535

10,679

6,305
2014

8,370

7,907
6,144
2013

7,034

6,162

5,751
2011

2012

5,648
2010

4,497
2007

5,586

4,342
2006

3,781
2002

4,265

3,699
2001

2005

3,590
2000

4,354

3,476
1999

2004

3,350
1998

4,134

3,396
1997

2,989
1996

4,000

2,937

6,000

2009

5,312

8,000

1995

DRUG COUNT

10,0009,140

10,271

12,0002022

Figure 18: Total US R&D pipeline size, 1995—2024

2,000

Source: Pharmaprojects®, January 2024

32

Pharma R&D Annual Review 2024

2024

2021

2020

2019

2018

2017

2016

2015

2008

2003

0

YEAR

Copyright © 2024 CitelineAs of January 2024, there were 2,609 companies
developing drugs that are headquartered in the
US. Of these, 39.9% were developing one drug,
83.5% were developing ≤5 drugs, and 96.6%
were developing ≤10 drugs. The Top 10 companies
are listed in Table 6, alongside the size of
their pipelines.
(Note: data in this chapter were pulled at a slightly later
date than the data used for Table 1, hence there may beminor differences in numbers.)

Table 6: Top 10 US—headquarteredcompanies by size of pipeline
PIPELINEDRUGSCOMPANYPfizer204

Johnson & Johnson

149

Eli Lilly

144

Merck & Co.

139

AbbVie109

Bristol-Myers Squibb

106

Gilead Sciences

85

Amgen

76

Regeneron

74

Moderna69

Source: Pharmaprojects®, January 2024

The Top 10 diseases in active development in the US are of course dominated by cancer indications,but here, non-small cell lung cancer tops the table, pushing breast cancer into second. Two noncancer indications appear in the Top 10 disease list for the US: Alzheimer’s disease (ranked sixth with
236 drugs in development, higher than globally) and the novel coronavirus (ranked ninth with 224
drugs in development, lower than globally). The Top 10 diseases in development in the US are nearly
identical to the Top 10 worldwide list, the only exception being that acute myelogenous leukemia
appears in the US Top 10, replacing liver cancer from the worldwide list.
Table 7: The US’s Top 10 diseases for pipeline drugs
POSITIONDRUG DISEASEPIPELINE
DRUGS

1Cancer, lung, non-small cell414

2Cancer, breast

356

3

Cancer, colorectal

287

4Cancer, pancreatic

249

5Cancer, ovarian237

6Alzheimer's disease236

7

Cancer, brain

235

8

Cancer, prostate

225

9Infection, coronavirus, novel coronavirus

224

10

Cancer, leukaemia, acute myelogenous

223

Source: Pharmaprojects®, January 2024

33

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineJapanThe size of Japan’s R&D pipeline has steadily increased since 2007, following an overall decrease inpipeline size from 1995. The pipeline has increased by an average of 75.6 drugs per year since 2007,
with a mean 6.1% YoY growth rate. From 2023 to 2024, the number of drugs in active R&D in Japan
increased from 1,981 to 2,044, corresponding to a 3.2% growth rate. Based on these data, while thenumber of pipeline drugs continues to increase in Japan, the rate of growth has begun to decelerate.
That said, since 2007, growth of Japan’s pipeline has largely kept pace with the rest of the world. The
average worldwide annual rate of pipeline growth since 2007 is 6.9%, only 0.8pp higher than in Japan.
Figure 19: Total Japan R&D pipeline size, 1995—2024

1,981

2,044
2024

1,835
2022

2023

1,796

1,567
2018

2021

1,583
2017

1,732

1,579
2016

1,413

1,350

1,248

1,173

1,028
2011

2007

977

759

2006

2010

785

2005

840

767

2004

814

859

811

886
2002

2003

883
2001

1,000

981

1,084
1999

1,237
1997

1,147

1,207
1996

1998

1,181

1,500

1995

DRUG COUNT

2,000

1,632

2,500

500

2020

2019

2015

2014

2013

2012

2009

2008

2000

0

YEAR

Source: Pharmaprojects®, January 2024

As of January 2024, there were 184 companies developing drugs that are headquartered inJapan. Of these, 35.3% were developing one drug, 63.6% were developing ≤5 drugs, and 83.7%
were developing ≤10 drugs. The Top 10 Japanese companies are listed in Table 8, alongside thesize of their pipelines.
Table 8: Top 10 Japan-headquarteredcompanies by size of pipeline
COMPANYPIPELINE DRUGS

Takeda130

Astellas Pharma

74

Eisai

73

Daiichi Sankyo

55

Ono Pharmaceutical

50

Shionogi

48

Otsuka Pharmaceutical

34

Sosei Heptares

32

Taiho

32

Mitsubishi Tanabe Pharma

31

Source: Pharmaprojects®, January 2024

34

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineCancer indications dominate Japan’s Top 10 diseases in active development, taking seven outof the 10 spots. However, while all the cancer indications are solid forms in the worldwide Top 10
diseases (breast, non-small cell lung, colorectal, pancreatic, ovarian, brain, prostate, and liver),Japan’s Top 10 list also includes the hematological cancers of non-Hodgkin’s lymphoma andmyeloma. The non-cancer indications included in Japan’s Top 10 differ from both the worldwide
list and that of the US, being type 2 diabetes, ulcerative colitis, and rheumatoid arthritis.
Table 9: Japan’s Top 10 diseases for pipeline drugs
POSITIONDRUG DISEASEPIPELINE DRUGS

1

Cancer, lung, non-small cell

97

2Cancer, breast

56

3

Cancer, colorectal

53

4Cancer, gastrointestinal, stomach

43

5Diabetes, Type 2

40

6Cancer, lymphoma, non-Hodgkin's

38

7

Cancer, pancreatic

34

8Colitis, ulcerative

33

9Arthritis, rheumatoid

32

10

Cancer, myeloma

31

Source: Pharmaprojects®, January 2024

35

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

ChinaAlthough Chinese R&D companies only emerged into the pharmaceutical market in 1997, with one
active drug in the market, they are currently responsible for developing 26.7% of globally active
drugs, as we’ve seen, making China the second-largest developer of new drugs. This reflects thestaggering growth China has gone through. The last five years have seen a boom for Chinese drug
development with an increase of 200.9% since 2019, as shown in Figure 20, partly due to the surge ofgovernment investment in the pharmaceutical sector.

6,119

7,000

4,203

5,136

6,000

2024

2012

2023

2011

2022

2010

2021

2009

2,590
2020

2008

1,452

2007

2019

2006

2018

2005

1,269

2004

2017

2003

1,131

2002

851

199

2001

2015

187

2000

701

171

1999

Source: Pharmaprojects®, January 2024

2014

160

1998

0

570

167

1

1997

1,000

2013

0

155

483

0

170

1

142

392

0

123

276

0

1996

2,000

2016

3,000

2,033

4,000

3,295

5,000

1995

NUMBER OF ACTIVE PIPELINE DRUGS

Figure 20: Total China R&D pipeline size, 1995—2024

YEAR

There are a total of 1,270 pharmaceutical companies headquartered in China, which as previously
noted is now a total that is second only to the US. The Top 10 companies in China can be seen in
Table 10. From 2023 to 2024, the size of the active pipelines of nine of the Top 10 Chinese companies
increased, reflecting the growth seen across the Chinese pharmaceutical sector.
Table 10: Top 10 China-headquartered companies by size of pipeline
COMPANYPIPELINE DRUGS 2024

Jiangsu Hengrui Pharmaceuticals

147

Sino Biopharmaceutical

103

Shanghai Fosun Pharmaceutical (Group)

90

CSPC Pharmaceutical73

BeiGene58

Shanghai Junshi Biosciences

57

Shanghai Pharmaceuticals Holding

51

Innovent Biologics

50

Qilu Pharmaceutical50

Huadong Medicine

44

Source: Pharmaprojects®, January 2024

36

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineAs shown in Table 11, nine out of the Top 10 diseases in China are forms of cancer, with the top
three being non-small cell lung cancer, breast cancer, and gastrointestinal stomach cancer.
Table 11: China’s Top 10 diseases for pipeline drugs
POSITIONDRUG DISEASENUMBER OF DRUGS

1

Cancer, lung, non-small cell383

2Cancer, breast

325

3

Cancer, gastrointestinal, stomach

227

4

Cancer, colorectal

218

5Cancer, liver

207

6

Diabetes, Type 2

187

7

Cancer, pancreatic

169

8

Cancer, lymphoma, non-Hodgkin's

168

9Cancer, myeloma

159

10

Cancer, leukaemia, acute
myelogenous

141

Source: Pharmaprojects®, January 2024

37

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

IndiaIn India, active pipeline growth saw its steepest change from 2007 to 2013, across which there wasa 309.9% increase in total active pipeline drugs. After 2013, the number of active drugs plateauedat around 800; however, since 2021, a steady decrease has been observed. There was an 18.9%
decrease in the total number of active pipeline drugs observed from 2021 to 2023, as shown in
Figure 21. But this year, that decline was almost completely reversed.

810

2020

631

694

714

778

816

2019

747

2015

812

803

567

600

285

400

191

148

2006

2024

2023

2022

2021

2018

2017

2016

2013

2012

2011

2010

2009

2008

2007

0

2000

153

0

1999

147

0

1998

2003

0

1997

Source: Pharmaprojects®, January 2024

2002

0

1996

2001

0

1995

62

100

90

132

200

2005

300

323

428

500

0

794

648

700

2014

783

800

2004

NO. OF ACTIVE PIPELINE DRUG

900

832

Figure 21: Total India R&D pipeline size, 1995—2024

YEAR

In India, there are 120 pharmaceutical companies with their headquarters in that country, less than
10% of the total number of companies in China. Out of the Top 10 pharmaceutical companies inIndia shown in Table 12, seven saw a decrease in their total pipeline size this year.
Table 12: Top 10 India-headquartered companies by size of pipeline
COMPANYNUMBER OF DRUGS

Dr. Reddy’s Laboratories

36

Lupin

29

Sun Pharmaceutical Industries

21

Serum Institute of India

20

Zydus Lifesciences

19

Biocon

18

Cipla

13

Intas Pharmaceuticals

12

Suven Life Sciences

12

Bharat Biotech

11

Source: Pharmaprojects®, January 2024

38

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

Table 13 shows that India’s top disease targets for R&D confound trends seen in other territories,
with type 2 diabetes coming out on top. Breast cancer and non-small cell lung cancer are relegatedto second and third place, respectively, here. They are two of only three cancer indications inIndia’s Top 10, which has a wider variety of diseases across many therapeutic areas. These include
metabolic and autoimmune indications, such as rheumatoid arthritis and psoriasis, as well as
vaccines for diphtheria and tetanus.
Table 13: India’s Top 10 diseases for pipeline drugs
POSITIONDRUG DISEASENUMBER OF DRUGS

1Diabetes, Type 2

1752

Cancer, breast

143

3

Cancer, lung, non-small cell

105

4Arthritis, rheumatoid

99

5

Cancer, colorectal

73

6Infection, tetanus prophylaxis

61

7

Psoriasis618

Infection, coronavirus, novel coronavirus

55

9Infection, diphtheria prophylaxis

53

10

Pain, nociceptive, general

52

Source: Pharmaprojects®, January 2024

39

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineUnited Kingdom
In the UK at the start of 2024, there were 3,156 pipeline drugs, up from 3,015 in 2023. This 4.5%
increase is less than the 7.8% growth rate seen from 2022 to 2023. There was a slight dip from 2019
to 2020; however, since 2020, drug development in the UK has been steadily increasing.

2,637

2,690

2020

2021

2,779

2,704

2,590

2018

2019

2,564

2017

2,275

2,147

1,966

1,815

1,648

2010

1,701

1,581

2009

1,394

2007

1,570

1,371

2006

2008

1,431

2005

1,354

2000

1,466

1,329

1999

2004

1,288

1998

1,416

1,284

1997

1,297

1,186

1,500

1,115

2,000

1,519

2,500

1996

DRUG COUNT

3,000

2,483

3,156

3,500

3,015

Figure 22: Total UK R&D pipeline size, 1995—2024

1,000
500

2024

2023

2022

2016

2015

2014

2013

2012

2011

2003

2002

2001

1995

0

YEAR

Source: Pharmaprojects®, January 2024

There are 321 companies developing drugs headquartered in the UK. The Top 10 UK companies arelisted in Table 14, alongside the size of their pipelines. AstraZeneca and GSK dominate the UK pharma
industry, with their closest competitors having pipeline sizes that are an order of magnitude less.
Table 14: Top 10 UK-headquarteredcompanies by size of pipeline
COMPANYNUMBER OF DRUGS

AstraZeneca

163

GlaxoSmithKline

128

Mundipharma
International

20

Hikma Pharmaceuticals

19Albumedix

17

Healx

15

ViiV Healthcare

14

Bicycle Therapeutics

13

PhoreMost

13

Oxford BioTherapeutics

12

Source: Pharmaprojects®, January 2024

40

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineWe have seen how the global pipeline is dominated by drugs in development for various cancer
indications, and the data from early 2024 in the UK certainly reflect this, with the top seven of theTop 10 diseases falling into the oncology space. This pattern broadly falls in line with the global
rankings; however, non-small cell lung cancer is the number 1 disease being targeted in the UK,
whereas breast cancer is number 1 globally. Three non-cancer indications appear in the Top 10
disease list for the UK: type 2 diabetes (ranked eighth), Alzheimer’s disease (ranked ninth), andnovel coronavirus vaccines (ranked 10th).
Table 15: The UK’s Top 10 diseases for pipeline drugs
POSITIONDRUG DISEASEPIPELINE DRUGS

1

Cancer, lung, non-small cell

113

2Cancer, breast

87

3

Cancer, colorectal

82

4Cancer, prostate

63

5Cancer, ovarian

57

6

Cancer, melanoma

52

7

Cancer, lymphoma, non-Hodgkin’s

51

8

Diabetes, Type 251

9Alzheimer’s disease49

10

Infection, coronavirus, novel coronavirus prophylaxis

48

Source: Pharmaprojects®, January 2024

41

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineIrelandSince 1995, there has been an overall increasing trend in the total pipeline size in Ireland. In terms ofthe most recent changes in pipeline size in Ireland, there are currently 1,158 pipeline drugs, which isup from 1,099 in 2023. However, this 5.4% increase is smaller than the 6.9% growth from 2022 to 2023.
Figure 23: Total Ireland R&D pipeline size, 1995—2024

1,158

1,028

1,037

989

1,030

975

932

860

800

760
652

597

563

512

491

462

468

466

490

507

Anti-infective Products
Anticancer Products

313

440

429

396
1997

423

356
1996

600

460

800

333

DRUG COUNT

1,000

950

Antiparasitic Products

1,200

400

1,099

Blood and Clotting Pro

1,400

Alimentary/Metabolic

200

2024

2023

2022

2021

2020

2019

2018

2017

2016

2015

2014

2013

2012

2011

2010

2009

2008

2007

2006

2005

2004

2003

2002

2001

2000

1999

1998

1995

0

YEAR

Source: Pharmaprojects®, January 2024

There are 40 companies developing drugs headquartered in Ireland. The top 10 Irish companies arelisted in Table 16, alongside the size of their pipelines.
Table 16: Top 10 Ireland-headquarteredcompanies by size of pipeline
COMPANYPIPELINE DRUGS 2024

Jazz Pharmaceuticals

20

Alkermes

10

Mallinckrodt

9

Prothena

8

Perrigo5

ONK Therapeutics

5

Shorla Pharma

4

GH Research

3

Inflection Biosciences

3

Orbsen Therapeutics

2

Source: Pharmaprojects®, January 2024

42

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineAs we’ve seen, globally, the Top 10 diseases against which most drugs are in development areconcentrated among cancer indications, but the data from early 2024 for Ireland show that thiscountry is another that bucks the trend, with only four of the Top 10 diseases falling into the oncology
space. While the top two diseases for Ireland are non-small cell lung cancer and breast cancer,
the Top 10 indications in Ireland include a relatively higher number of metabolic and autoimmune
indications, including type 2 diabetes, rheumatoid arthritis, juvenile arthritis, psoriatic arthritis,
psoriasis, and ulcerative colitis.
Table 17: Ireland’s Top 10 diseases for pipeline drugs
POSITIONDRUG DISEASEPIPELINE DRUGS

1

Cancer, lung, non-small cell51

2Cancer, breast

44

3

Arthritis, rheumatoid

34

4

Diabetes, Type 2

325Arthritis, psoriatic

31

6Psoriasis

31

7

Cancer, colorectal

288Colitis, ulcerative27

9Arthritis, juvenile

25

10

Cancer, renal

24

Source: Pharmaprojects®, January 2024

43

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineDenmark
In Denmark, there are currently 1,536 active drugs, which is up from 1,444 in 2023. This 6.4% increaseis substantially less than the 11.9% YoY growth rate recorded from 2022 to 2023. The growth over the
past year is slightly above the mean YoY growth rate in the country since 1996 (5.1%). In comparison,
the progression of development in Denmark over the past year was slightly lower than the global
growth rate of 7.2%. The last two years have stood out in the recent history of Denmark’s drug
development landscape as the country has had its two best years of growth since 2016.

1,321

1,300

2020

2021

1,444

1,320

1,234
2018

2019

1,210
2017

1,080

1,029

1,199

867
2012

944

809

739

674

605
2007

2009

599
2006

680

583
2005

2008

594
2004

503
2000

607

516

557

490

1999

405

468
1997

1998

412
1996

600

399

800

2003

1,000

2011

1,200

1995

DRUG COUNT

1,400

2016

1,600

1,290

1,800

1,536

Figure 24: Total Denmark R&D pipeline size, 1995—2024

400
200

2024

2023

2022

2015

2014

2013

2010

2002

2001

0

YEAR

Source: Pharmaprojects®, January 2024

Continued pharmaceutical development growth in Denmark is likely dependent on the 70 companies
currently headquartered in the country, led by the Top 10 companies listed in Table 18. Novo Nordisk
is head and shoulders above the rest, but there are a few other well-known companies based in the
country, including Genmab, Lundbeck, and Leo Pharma.
Table 18: Top 10 Denmark-headquarteredcompanies by size of pipeline
COMPANYPIPELINE DRUGS

Novo Nordisk

68

Genmab25

Lundbeck

16

Gubra

15

Bavarian Nordic

10

Leo Pharma

10

Zealand Pharma

10

Evaxion Biotech

8

ALK-Abello

7

Xellia Pharmaceuticals

7

Source: Pharmaprojects®, January 2024

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Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

One would expect the vast majority of developmental therapies to be indicated for various formsof cancer or alimentary/metabolic diseases, and the data from early 2024 in Denmark confirm
this, with seven of the Top 10 diseases falling into these spaces. With only four cancer indications,
Denmark differs from the global rankings, where eight of the top 10 indications are cancer. The
other two diseases of focus in the global landscape are Alzheimer’s disease and novel coronavirus
infection; however, out of the 589 indications under study in Denmark, Alzheimer’s disease and novel
coronavirus infection rank 34th and 55th, respectively.
Table 19: Denmark’s Top 10 diseases for pipeline drugs
POSITIONDRUG DISEASENUMBER OF DRUGS

1

Cancer, lung, non-small cell

63

2Cancer, breast

49

3

Cancer, colorectal

39

4Diabetes, Type 2

38

5Arthritis, rheumatoid

37

6

Psoriasis32

7Arthritis, psoriatic

31

8Cancer, myeloma

30

9Colitis, ulcerative

29

10

Crohn's disease28

Source: Pharmaprojects®, January 2024

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Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

SwedenFrom one Scandinavian country to another, we turn our focus to Sweden, which has similar development
metrics to Denmark. In Sweden, there are currently 1,563 active drugs, which is up from 1,467 in 2023.
This represents 6.5% YoY growth, which pales in comparison to the rate seen from 2022 to 2023, which
was 9.2%. However, the past year’s growth is still above the mean growth rate in Sweden since 1996
(5.2%), so drug development is clearly still growing in this market. Much like Denmark, the YoY growth
rates seen in the past two years in Sweden are the country’s highest since 2016.

1,353

1,351

2021

2022

1,467

1,389

2020

1,263

1,309

1,112

663

903

2018

2006

1,301

528

2005

2017

548

2004

563

581

579

524

2000

2003

511

1999

529

481

1998

416

454

1997

422

1996

600

393

800

618

769

1,000

831

1,024

1,200

1995

DRUG COUNT

1,400

1,172

1,600

1,309

1,800

1,563

Figure 25: Total Sweden R&D pipeline size, 1995—2024

400
200

2024

2023

2019

2016

2015

2014

2013

2012

2011

2010

2009

2008

2007

2002

2001

0

YEAR

Source: Pharmaprojects®, January 2024

Currently there are 136 companies headquartered in Sweden that are actively developing drugs.
Table 20 shows the Top 10 of these companies and the number of drugs they have in their current
pipeline. For Sweden to continue growing its pharmaceutical industry and remain productive in the
global development landscape, the 10 companies below will need to continue their recent progress.
Table 20: Top 10 Sweden-headquarteredcompanies by size of pipeline
COMPANYPIPELINE DRUGS

Ribocure
Pharmaceuticals

16

Anocca

15

Sobi

15

BioArctic

13

Medivir

9

Alligator Bioscience8

Salipro Biotech

7

AlzeCure Pharma

6

BioInvent

6

Camurus6

Source: Pharmaprojects®, January 2024

46

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

Sweden also lags in oncology development compared to globally, with only five cancer diseases in
its Top 10. Interestingly, comparing the top pipeline indications between Denmark and Sweden showsthat both countries heavily contribute to the development of therapies for arthritis and psoriasis,
diseases that are at least 10 places lower in the global landscape. The Scandinavian countries appear
to focus more on immunological diseases than many other countries around the world.
Table 21: Sweden’s Top 10 diseases for pipeline drugs
POSITIONDRUG DISEASEPIPELINE DRUGS

1

Cancer, lung, non-small cell

62

2Cancer, breast

50

3

Cancer, colorectal

40

4Diabetes, Type 2

40

5Arthritis, rheumatoid

38

6

Cancer, myeloma

35

7

Psoriasis

35

8Arthritis, psoriatic

32

9Alzheimer's disease

31

10

Cancer, prostate30

Source: Pharmaprojects®, January 2024

47

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

France
We next move south to France, which dwarfs both Denmark and Sweden in the total number ofdrugs, while also besting both countries in YoY growth, with a rate of 8.1%. The most recent growthrate is down from 2022—23 (12.2%), but is also still above the global 2023—24 growth rate of 7.2%.
The past year’s growth is almost double the mean YoY growth rate since 1996 (4.3%), and is its
second largest YoY metric since 2016.

1,911

1,955
2021

1,951

1,923

2020

1,869

2019

1,891

1,870

2017

1,589

1,492

1,336

1,185

2018

949
2009

1,016

956

953
2007

2008

859

838
2006

858

2005

878

794
2000

2004

785
1999

799

791
1998

2003

798
1997

688

767
1996

1,000

735

1,500

1995

DRUG COUNT

2,000

1,706

2,188

2,500

2,365

Figure 26: France’s R&D pipeline size, 1995—2024

2024

2023

2022

2016

2015

2014

2013

2012

2011

2010

2002

0

2001

500

YEAR

Source: Pharmaprojects®, January 2024

With a large number of active drugs in the country, one would expect the companies headquartered in
France to have large current pipelines, and that is what we can see in Table 22. With big companies like
Sanofi and Ipsen leading the way, the development landscape in France looks to be in good hands for
future years of continued growth.
Table 22: Top 10 France-headquarteredcompanies by size of pipeline
COMPANYPIPELINE DRUGSSanofi

130

Ipsen

38

Servier

32

TheraVectys

22

Pierre Fabre

18

Valneva

11

Innate Pharma

10

OSE
Immunotherapeutics

10

Vectans Pharma

9

Xenothera

9

Source: Pharmaprojects®, January 2024

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Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

France’s active pipeline is more comparable to the Top 10 targeted indications across the globe thanthose of many other countries, reporting nine out of the Top 10 diseases from the oncology space.
Of the nine cancer indications, four are in both the French and global Top 10s: non-small cell lungcancer, breast cancer, colorectal cancer, and prostate cancer. As for the diseases that round out
the global Top 10, Alzheimer’s disease and novel coronavirus infection developmental drugs are notnearly as prevalent in France, with Alzheimer’s disease placing 31st and novel coronavirus infection
placing 37th.
Table 23: France’s Top 10 diseases for pipeline drugs
POSITIONDRUG DISEASEPIPELINE DRUGS

1

Cancer, lung, non-small cell134

2Cancer, breast

89

3

Cancer, colorectal

69

4Cancer, melanoma

58

5

Cancer, lymphoma, non-Hodgkin's

55

6

Cancer, leukaemia, acute myelogenous

54

7

Cancer, myeloma

46

8

Cancer, prostate

44

9Cancer, renal

40

10Colitis, ulcerative

38

Source: Pharmaprojects®, January 2024

49

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineGermany

Since 1995, there has been an overall increasing trend in the total pipeline size in Germany. In
terms of the most recent changes in pipeline size, between 2023 and 2024 the total pipeline size inGermany rose, with a 5.0% increase in the number of active drugs.

2,246

2,231

2,175

2018

2020

2021

2022

2,479

2,151
2,207

2017

2,360

2,188

1,982

1,870

1,703

1,423

2,187

1,078
2009

1,249

1,048
2008

912
2006

876

923
2005

958
2004

842
2000

944

845
1999

2003

815
1998

883

811

2002

793

1997

748

793

1,000

1996

1,500

1995

DRUG COUNT

2,000

1,570

2,500

2016

Figure 27: Germany’s R&D pipeline size, 1995—2024

500

2024

2023

2019

2015

2014

2013

2012

2011

2010

2007

2001

0

YEAR

Source: Pharmaprojects®, January 2024

There are 143 companies headquartered in Germany. The top 10 companies are listed in Table 24,
alongside the size of their pipelines.
Table 24: Top 10 Germany-headquarteredcompanies by size of pipeline
COMPANYPIPELINE DRUGS

Boehringer Ingelheim

103

Bayer

91

BioNTech

53

Evotec49

Merck KGaA

36

CureVac22

Grunenthal

12

Stada

11

Biotest10

Medigene

10

Source: Pharmaprojects®, January 2024

50

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

The top three diseases in Germany are non-small cell lung cancer, breast cancer, and colorectal
cancer, which are also the top three drug diseases globally. The Top 10 German indications also
include a number of metabolic, dermatological, and autoimmune indications, such as type 2
diabetes, psoriasis, and rheumatoid arthritis.
Table 25: Germany’s Top 10 diseases for pipeline drugs
POSITIONDRUG DISEASEPIPELINE DRUGS

1

Cancer, lung, non-small cell

109

2Cancer, breast

71

3

Cancer, colorectal

60

4Diabetes, Type 2

59

5

Psoriasis

46

6

Cancer, myeloma

44

7

Cancer, leukaemia, acute myelogenous

43

8

Arthritis, rheumatoid

41

9Cancer, lymphoma, non-Hodgkin's

41

10

Cancer, melanoma

40

Source: Pharmaprojects®, January 2024

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Pharma R&D Annual Review 2024

Copyright © 2024 CitelineSwitzerland
Since 1995, there has been an almost tripling of the total pipeline size in Switzerland. Between 2023
and 2024, the total pipeline size in Switzerland rose, with a 6.1% increase in the number of active
drugs this year.

1,071

1,113

1,127
1,165

2019

2020

1,120

1,154

1,089

2018

872
774
531

537

519
536

564

582

2004

2005

2006

2008

2009

673

523
2003

457

425

464
2000

540
1998

494

591
1997

1999

577
1996

600

558

800

1995

DRUG COUNT

1,000

955

1,200

1,035

1,400

1,232

1,419

1,600

1,505

Figure 28: Switzerland’s R&D pipeline size, 1995—2024

400

2024

2023

2022

2021

2017

2016

2015

2014

2013

2012

2011

2010

2007

2002

0

2001

200

YEAR

Source: Pharmaprojects®, January 2024

There are 188 companies headquartered in Switzerland. The Top 10 companies are listed in Table 26,
alongside the size of their pipelines.
Table 26: Top 10 Switzerland-headquarteredcompanies by size of pipeline
COMPANYPIPELINE DRUGS

Roche30: Top 10 Switzerland-headquartered
217
Table
companies
by size of pipeline
Novartis
153
Medicines for Malaria
Venture

32

DNDi

29

CRISPR Therapeutics

23

Debiopharm18

AC Immune

17

Sandoz17

Light Chain Bioscience

14

Idorsia Pharmaceuticals

14

Source: Pharmaprojects®, January 2024

52

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

The top three diseases for Switzerland are non-small cell lung cancer, breast cancer, and colorectal
cancer, which are also the top three drug diseases globally. As with the global pipeline, the Swisspipeline is dominated by drugs in development for different cancer indications.
Table 27: Switzerland’s Top 10 diseases for pipeline drugs
POSITIONDRUG DISEASENUMBER OF DRUGS

1

Cancer, lung, non-small cell46

2Cancer, breast

43

3

Cancer, colorectal

33

4Cancer, melanoma

32

5

Cancer, brain

27

6

Alzheimer's disease

26

7

Cancer, myeloma

26

8Diabetes, Type 2

23

9Infection, malaria

21

10

Cancer, leukaemia, acute myelogenous

19

Source: Pharmaprojects®, January 2024

That concludes our whistle-stop
review of the R&D climates acrossa range of countries with significant
pharma industries. It suggeststhat while some territories areexperiencing fair weather, for others
the skies are darkening somewhat.
It also demonstrates how different
countries have different emphases
in terms of their disease focuses, as
every company continues to search
for the pot of gold at the end of
the rainbow.

53

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineMechanisms and Targets
Immuno-oncology makes hay while the sun shines
What makes weather work? The Earth’s climate system is complex, but a small change
to temperature, atmospheric pressure, cloud formation, wind, humidity, or rain patterns
can have a significant effect on the development of weather systems. It is the interactionof all these different components, in an environment where chaos theory also plays
its part, that produces our weather in all its infinite variety. Everything is very finely
balanced, hence how an external force, for example human interventions such as
the release of greenhouses gases into the atmosphere, can perturb the status quo,potentially irrevocably.
What makes drugs work is the subject of
this section of our report, which looks at
drugs’ mechanisms of action and theirtargets. It’s a similarly complex ecosystem.
Just as we can model the weather to
get predictions of the outcome of what’s
happening now, we can predict from drugs’
mechanisms and targets what will happen
when they are administered, but both arestill incredibly inexact sciences. The whole
point of the drug R&D process is to test
hypotheses and gather evidence, so that
we can have confidence in the outcome oftaking a drug, just like we want to be able
to trust our weather forecasts.

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Pharma R&D Annual Review 2024

Beginning with mechanisms, Table 28
lists the current Top 25 mechanisms in
Citeline’s proprietary mechanism of action
classification. It’s important to note that
this is hierarchical, one effect of which is tomake the broader terms at the top of thehierarchy crop up most commonly. This isbecause, with over half of the pipeline stillat the preclinical phase, where often fullmechanistic information is yet unknown
or undisclosed, there tend to be a lot ofdrugs where only a broad mechanisticclass can be ascribed. As drugs move up
through development stages, these generalcategorizations are often replaced bysomething more precise. There are also
a number of “umbrella” terms, created topermit searching across mechanisms incertain broader categories. The immunooncology (IO) category is one such of theseand is applied to drugs in the IO class even
if more specific mechanistic informationis also known, so that all IO drugs can befound in a single search. This is one of thereasons that it is applied to so many drugs.
These caveats should be borne in mind
when examining the Top 25 terms.

Copyright © 2024 Citeline

Table 28: Top 25 mechanisms of action (pharmacologies)
POSITION
2024(2023)MECHANISM OF ACTION

NUMBER OF DRUGS 2024
(2023)% AT PR, R
OR L

TREND

1 (1)

Immuno-oncology therapy

3,932 (3,393)

2.7

↑↑

2 (2)

Immunostimulant

1,812 (1,472)

10.3

↑

3 (3)

T cell stimulant

1,148 (1,091)

1.6

↔

4 (4)

Immune checkpoint inhibitor

986 (618)

4.0

↑↑

5 (5)

Gene expression inhibitor

354 (283)

1.7↑

6 (15)

Immune checkpoint stimulant

303 (150)

1.3

↑↑

7 (7)

Protein degrader

292 (221)

1.0

↑

8 (6)

Genome editing

290 (274)

0.3

↔

9 (9)

Radiopharmaceutical

277 (192)

7.2↑↑

10 (11)Natural killer cell stimulant

223 (186)0↑

11 (10)

Angiogenesis inhibitor

192 (192)

28.1↔

12 (8)

CD3 agonist

190 (198)

5.3↓

13 (12)

PD-L1 antagonist

178 (181)

6.2↓

14 (13)

Immunosuppressant174 (179)

38.5↓

15 (17)Microbiome modulator, live
microorganisms

167 (128)

1.2↑

16 (14)PD-1 antagonist

165 (152)12.7↔

17 (18)Glucagon-like peptide 1 receptor
agonist156 (111)

5.8↑

18 (16)

Vascular endothelial growth factor
receptor antagonist

152 (142)26.3↔

19 (352)

Ubiquitin ligase E3 stimulant

125 (11)0↑↑20 (21)

K-Ras inhibitor

108 (87)

2.8↑

21 (31)

DNA topoisomerase I inhibitor

98 (67)

6.1↑

22 (19)Apoptosis stimulant

90 (103)

14.4↓

23 (34)

Tubulin inhibitor

89 (62)

7.9↑

24 (24)

DNA inhibitor

87 (82)32.2↔

25 (20)

ErbB-2 antagonist

87 (97)

20.7↓

Abbreviations used in table: PR = pre-registration R = registered L = launched
Source: Pharmaprojects®, January 2024

55

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineThis certainly helps to explain the table’s number
1 category being IO therapy. There are now 3,932
drugs that fall into that bucket, meaning morethan one in six of all drugs in development canbe categorized in this way — a striking statistic
considering how relatively new this technique,whereby the body’s own immune system
is trained to attack cancer cells, is. Further
evidence of how the pharmaceutical industry isboldly placing a lot of faith in this strategy canbe seen by looking at the column to the rightof the number of drugs column in this table: thepercentage of drugs using this strategy whichhave made it to the later stages (pre-registration,
registered, or launched) of development. Only
2.7% of drugs in this category have made it thisfar, so the overwhelming majority of candidates
are still in clinical or preclinical studies. As moreproducts do progress, though, this is looking
less like an enormous leap of faith and more likean exciting new front. With a remarkable 15.9%
increase in candidates, this looks to be one area
where it never rains but it pours.
One of the subgroups of IO therapy, immune
checkpoint inhibitors, reports a nothing lessthan stupendous 59.5% growth in pipeline size.
This category embraces a plethora of different
individual categories, including some whichappear under their own right in the top 25, suchas PD-L1 and PD-1 antagonists. Even more
dramatically, the size of the pipeline of immune
checkpoint stimulants has more than doubled.
This can be partly accounted for by the fact
that, during the course of last year, we createda new Target Family to encompass targets inthis area, which led to a cleanup of the data andconsequently more drugs being assigned thismechanism of action.

But there is also an internal reason for the
stratospheric increase, as a review of suchdrugs led to us adding this mechanism to quite
a few existing PROTAC drugs which didn’t havethis assigned before. But despite the ongoinginterest in protein degradation and E3 ligase
signaling, development remains firmly rooted
at the preclinical to early clinical stage. To
date, there are only six protein degrader drugs
approved worldwide, with just two new launches
in 2023. Another big increase within the Top 10
was in radiopharmaceuticals, which recorded a
44% rise, with imaging agents making up overhalf of this increase.
There was stagnation in the developmentof some of the more traditional anticancer
cytotoxic drugs, such as DNA intercalators,
tubulin binding agents, and angiogenesis
inhibitors. This is perhaps indicative of thecontinued push towards targeted therapy as amainstay, with little movement in this area sincelast year’s report. One of the elders of targeted
therapy, ErbB-2 antagonists, also took a hit,with the number of development candidates
actually falling. Elsewhere, the continued riseof antibody-drug conjugates produced anincrease in DNA topoisomerase I-targeting drugcandidates, with TOP1 representing an attractive
target for antibody-drug conjugate warheads.

Two other broad IO categories involved in celltherapy, T cell stimulants and natural killer cellstimulants, feature, although their progress has
somewhat stalled this year. Overall, the field of
immuno-oncology is keenly represented, as it
continues to have its moment in the sun.
The development of targeted protein degraders
also saw a noticeable increase in 2023, withubiquitin E3 ligase being the most populartarget for these novel therapeutic agents. Keenobservers will note an extraordinary rise in thenumber of ubiquitin ligase E3 stimulants inthis year’s Top 25, the explanation for whichis twofold. First, there were a huge numberof protein degraders (around 60) using thismechanism entering development over the
past 12 months.
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Pharma R&D Annual Review 2024

Copyright © 2024 CitelineImmuno-oncology makes its presence similarly felt in Table 29, where we look at the Top 25
individual protein targets which today’s pipeline drugs are hitting. Here, there has been a change
at the top, with CD3e, a target heavily used by CAR-T therapeutics, returning to the pole position.
It deposes another IO-based target, PD-L1, down to number 3, while a more traditional anticancer
target, Her2, moves up to number 2, so IO is not having everything its own way. Another IO-basedtarget involved in CAR-T, CD19, also drops down the top five.
Table 29: Top 25 drug protein targets
POSITION
2024(2023)TARGET

NUMBER OF DRUGS
2024 (2023)

TREND

1 (2)

CD3 epsilon subunit of T-cell receptor complex

223 (207)

↔

2 (4)

erb-b2 receptor tyrosine kinase 2 [Her2]

217 (187)

↔

3 (1)

CD274 molecule [PD-L1]

211 (210)

↔

4 (5)

epidermal growth factor receptor

205 (178)

↑

5 (3)

CD19 molecule

199 (194)

↔

6 (6)

programmed cell death 1 [PD-1]

194 (165)

↑

7 (8)

glucagon like peptide 1 receptor

169 (126)

↑

8 (7)

vascular endothelial growth factor A

168 (160)

↔

9 (9)

KRAS proto-oncogene, GTPase [K-Ras]

133 (118)↔

10 (10)5-hydroxytryptamine receptor 2A

127 (117)↔

11 (11)

opioid receptor mu 1

104 (107)↓

12 (12)

insulin receptor

97 (87)↔

13 (14)cannabinoid receptor 1

94 (86)↔

14 (13)

TNF receptor superfamily member 17 [BCMA]

94 (87)↔

15 (16)

nuclear receptor subfamily 3 group C member 1[glucocorticoid receptor]

91 (84)↔

16 (18)

membrane spanning 4-domains A1 [CD20]

90 (82)↔

17 (16)

TNF receptor superfamily member 9 [CD137]

88 (84)↔

18 (21)

claudin 18

87 (75)↔

19 (15)tumor necrosis factor

87 (86)↔

20 (22)

androgen receptor

76 (75)↔

21 (23)transforming growth factor beta 1

76 (74)↔

22 (19)

opioid receptor kappa 1

75 (80)↓23 (24)

CD47 molecule74 (73)↔

24 (26)kinase insert domain receptor

73 (69)↔

25 (25)prostaglandin-endoperoxide synthase 2

72 (73)↓

Note: NCBI names are used, except for additions in italics made by us for clarity.
Source: Pharmaprojects®, January 2024

57

Pharma R&D Annual Review 2024

Copyright © 2024 Citelinedevelopment does continue to expand, however,with the total for 2024 hitting 2,035, up from 1,974
the year before, with a bigger increase than thatseen from 2022 to 2023. We are clearly a longway still from there being nothing new under
the sun.

Outside of the Top 10, the air is stiller this year,with few dramatic changes, aside from thecontinued decline of the opioid targets mu1 andkappa1. Conspicuous by its absence this year is
the COVID-19 spike protein, which drops out of
the upper echelons to number 31. It might haveappeared in 2021’s chart like a bolt from the blue,
but its decline has been similarly lightning fast.
The lifeblood of the industry, though, has to be
the continued identification of novel targets for
drug development, showers of which are needed
every year to prevent the drug development
landscape rapidly becoming an arid desert.
Figure 29 records that the number of newly
identified drug targets in 2023 was very similar
to that seen in 2022, at 93. This paints the year
as another one very slightly below the par of 100
per year. The universe of targets for active drug

Figure 29: Number of new drug protein targets identified by Pharmaprojects, by year
200
179

180

NUMBER OF NEW TARGETS

160
139

140
131

120

116
100

100
93

113

97

97

94

92

89

80

107

102

77

75

68

72

67

60

40

20

0

2023

2022

2021

2020

2019

Source: Pharmaprojects®, January 2024

2018

2017

2016

2015

2014

2013

2012

2011

2010

2009

2008

2007

2006

2005

YEAR

Famously, no two snowflakes are alike. While ways to treat diseases and targets for drug development
will presumably not be similarly infinite, it seems likely that we may have barely scratched the surfaceof the myriad ways in which science may yet treat the burden of disease. Let’s hope that the flurry of
new targets identified, and new drugs produced, can turn into a blizzard.

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Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

Types of Pipeline Drugs
Is the continued move into biotech
starting to meet a frosty reception?

Most experts believe that climate change is rapidly approaching an inflection point. If action
is not taken now, we might reach the point of no return, whereby we lose the ability to control
or reverse the changes happening to our world’s weather. Last year, scientists from the British
Antarctic Survey reported that ice melt in West Antarctica has accelerated and is now inevitable
for the rest of the century, no matter by how much carbon emissions are cut. If this ice sheet is lostcompletely, it would raise global sea levels by five meters, leading to many coastal cities having to
be abandoned. However, it is believed that if we act now, we can at least save the East Antarctic
ice sheet, which has the potential to cause sea levels to rise by as much as 10 times that amount.
A change in pharma that also has seemed to behitting a tipping point — although by no meansa similarly negative trend — is the ongoing moveaway from traditional small molecule drug R&D
to that based on biotechnological techniques.
There were signs last year that this too might behitting an inflection, and that the disappearanceof large chunks of chemistry-based drug
discovery might be slowing. This is one data
point we will address in this last section of thisyear’s report, which looks at the types ofdrugs under development.

Figure 30 therefore makes interesting reading.
It now looks like there is clear evidence thatthe ongoing march into biotech has indeed
plateaued. The overall proportion of biotech
drugs this year stands at 44.2%, up just 0.2pp
on last year, and still 0.5pp below where it wasin 2022. It does appear that, after many yearsof biotechnology drugs taking an increasingly
large slice of the pie, a set point might havebeen reached.

Figure 30: Biological vs. non-biological drugs as a percentage of the pipeline, 1995—2024
100%
90%

% OF PIPELINE

80%
70%
60%

Non-Biotech

50%

Biotech

40%
30%
20%
10%

Source: Pharmaprojects®, January 2024

59

Pharma R&D Annual Review 2024

2023

2024

2021

2022

2019

2020

2018

2017

2016

2015

2013

2014

2011

2012

2010

2009

2007

2008

2006

2005

2004

2003

2001

2002

1999

2000

1997

1998

1996

1995

0%

YEAR

Copyright © 2024 Citeline

We can break this down further in Table 30, which gives data on the number of active drugs assigned
to each of Citeline’s individual drug type categories, referred to as origins as they indicate how the
drug was originally produced. Here is further confirmation that predictions of the demise of the small
molecule may well have been premature. Synthetic chemicals still top this chart, and in fact post
a 5.1% increase on last year, although admittedly this is a smaller percentage increase than 2023’s
7.8%. The next five categories provide some insight into the most common types of biotechnologybased candidates. This kicks off with antibodies at number 2, posting a heftier 11.1% rise in numbers,
followed by four categories whose numbers report little change: recombinant proteins, autologous
cell therapies, virally delivered nucleic acids, and heterologous cell therapies. Note that the origin
classification is another hierarchical one, hence the category for cell therapies whose type isunknown appearing at number 7.
Table 30: Top 25 origins of pipeline drugs
POSITION
2024(2023)ORIGINNO. OF DRUGS IN PIPELINE 2024 (2023)

TREND

1 (1)

Chemical, synthetic

10,831 (10,307)

↑

2 (2)

Biological, protein, antibody

3,038 (2,734)

↑

3 (3)

Biological, protein, recombinant

957 (932)

↔

4 (4)

Biological, cellular, autologous

722 (758)

↓

5 (6)

Biological, nucleic acid, viral vector

716 (677)

↔

6 (5)

Biological, cellular, heterologous

695 (687)

↔

7 (7)

Biological, cellular

688 (583)

↑↑

8 (8)

Chemical, synthetic, nucleic acid612 (536)↑

9 (10)

Biological, protein

558 (523)↔

10 (9)

Biological, virus particles

535 (534)↔

11 (11)

Biological, nucleic acid

493 (477)↔

12 (12)

Chemical, synthetic, peptide

491 (461)↔

13 (13)

Biological, other392 (358)↔

14 (14)

Biological, bacterial cells

339 (330)↔

15 (15)Biological, peptide

272 (254)↔

16 (17)

Biological, nucleic acid, non-viral vector

266 (185)↑

17 (19)Biological251 (164)↑

18 (16)Natural product, plant

218 (215)↔

19 (18)Biological, peptide, recombinant

176 (171)↔

20 (21)Natural product, fungal

58 (51)↔

21 (20)Chemical, semisynthetic

53 (57)↔

22 (22)Natural product, bacterial

46 (50)↔

23 (23)Natural product38 (38)↔

24 (25)Chemical, synthetic, isomeric21 (21)↔

25 (24)Natural product, animal

20 (22)↔

Source: Pharmaprojects®, January 2024

60

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineIn a table that, as usual, doesn’t exhibit much structural change, there are a couple of increases
for individual categories that are worth calling out. Nucleic acids produced by synthetic chemistryrise by 14.2% at number 8, while biologically produced nucleic acids delivered via non-viral means
are up by an impressive 43.8% at number 16. It’s also interesting to note how little natural productdevelopment contributes to the R&D pipeline now, with just 1.7% of current candidates being sourced
this way.
Despite the stasis of this table, there is a bit of movement in the related metrics on drug delivery
route, as evidenced in Figure 31. The proportion of drugs administered by injection is up a bit thisyear, with a concomitant fall in those delivered orally. Taken together, these delivery types cover 90%
of all pipeline drugs, with topical and inhaled preparations constituting 3% each, and other routes
even smaller percentages.
Figure 31: Pipeline by delivery route, 2023 and 2024
0%

1%

0%

0%

Vaginal3%

Transdermal
Topical2023

29%

Rectal61%

Otic
Injectable2%

3%

Implant

1%

InhaledOral

Ophthalmological

Ophthalmological

Oral
0%

1%

0%

0%

Otic

3%

VaginalInhaled

RectalTopicalTransdermal
Implant

Transdermal
VaginalTopicalInjectable

26%

2024

64%

Rectal
Otic

2%
3%
1%

Oral
Ophthalmological

Source: Pharmaprojects®, January 2024

Inhaled61

Pharma R&D Annual Review 2024

Copyright © 2024 CitelineWhat’s the Forecast for Pharma?
Plenty of rays of sunshine should hold off any storm
clouds on the horizon
Since I began compiling this report at
the beginning of January 2024, it’s been
officially confirmed that 2023 was thehottest year on record for planet Earth.
The average land and ocean surface
temperature during the year was 1.18°C(2.12°F) above the mean for the 20th
century, making it the highest global
temperature among all years in the US
National Oceanographic and Atmospheric
Administration’s (NOAA’s) climate records,
which began in 1850. In doing so, itbeat the next warmest year, 2016. Morealarmingly, the 10 warmest years since1850 have all occurred in the past decade.
The NOAA estimates that the chances of2024 being in the top five warmest years
ever are 99%.

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Pharma R&D Annual Review 2024

2023 was the “hottest” year for pharma
R&D in terms of the number of drugs indevelopment too, and I think we can makea similar prediction that 2024 is extremely
likely to be even hotter still. The trend here,
like the temperature, is heading inexorably
upwards. But, just as climate change mightmean a nice warm summer and a mild
winter for some, but wildfires and floods forothers, bigger drug pipelines are similarly
a mixed blessing. They are signs that theindustry is continuing to grow and that
there is money available for investment,
but as the vast majority of drugs will still
fail, it could be said that ever-increasing
amounts of capital are being spent for no
return. While the last few years have seensome very encouraging signs in terms ofthe numbers of new drugs making it onto
the market, 2023’s data on this are still
pending at the time of writing, and whether
there is an overall upward trend is at thispoint moot. Certainly, the fact that a lower
percentage of drugs in Phase II seem to be
making it into Phase III must be a worrying
statistic. Drug development will always be
an inherently risky undertaking, but the
chances of success ought to be getting
better, not worse.

Copyright © 2024 Citeline

on further, it has been somewhat eclipsed by
the ghastly events of the Israel-Gaza conflictthat erupted in early October. At the time ofwriting, no resolution is in sight, with both sides
entrenched in a whirlwind of horror. Thankfully,
as yet, this hasn’t expanded into a hurricaneof regional conflict, although recent events in
Yemen and across the Iran-Pakistan border
are a chilling reminder that this remains apossibility. With the Middle East a major sourceof the world’s oil, there is the potential thatrising oil prices could again put the kibosh ona recovering economic picture. Maybe climatechange isn’t the only reason why we should be
weaning ourselves off fossil fuels.

I reflected last year on how well the industry had
done to not only just continue doing what it does
during the COVID-19 pandemic, but also how it
significantly contributed to society moving pastit, and to use it as a springboard to superchargeadvances. The use of mRNA vaccine technology
comes to mind particularly here. To mix a range
of weather-based idioms, the industry had beencooking up a storm of innovation for some time,
and when the clouds of COVID gathered, andwe were suddenly all snowed in, this definitely
enabled us to see the sunshine after the rain
sooner. Many countries around the world are
now in the post-match analysis phase, holding
scientists, administrators, and politicians aliketo account for what we can learn so that we
can handle the (inevitable) next pandemic
better, as many parts of the world were woefully
underprepared. We should all agree that pharma
was a rare ray of sunshine here. It also emergedwith its reputation enhanced, even if it is still notconsidered to be as pure as the driven snow.

And as we go into another US election year,
we can expect more instability in the politicalatmosphere, which is likely to be exacerbatedby a bruising increase in the temperature of
the rhetoric of the culture wars. The result ofNovember’s poll could seriously affect thedirection of travel of the US in multiple fields, notleast in respect of its relationship with China. We
have seen in this report just how much China has
emerged as a global player in pharma R&D, andit’s not done yet. However, “business in China isgoing through a period of uncertainty,” pointed
out Helen Chen, Greater China managing
partner and Asia healthcare sector head at
L.E.K. Consulting, in an article of our sister
publication Scrip. “Distractions are many,” shecontinued, “a relatively low 5.5% GDP growth,
political tensions with the US, pricing pressuresand the hospital anti-corruption campaigns.”
Meanwhile, there is still the potential that out
to sea, a storm could brew over control of the
disputed territory of Taiwan.

Forecasting the fortunes of pharma can be as
tricky as predicting next week’s weather, as a
COVID-style tempest can always wind up outof nowhere. However, we can certainly look at
how external factors have the potential to blow
things off course. I always make note of the
global geopolitical outlook in my conclusion, as,just like how El Niño affects weather thousandsof miles away, the pharma industry is always
buffeted by world events, no matter how well it
is insulated from their worst effects, like donninga duffel coat on a blustery day. Unfortunately,
despite the sun peeping out as the clouds fromCOVID disperse, the outlook became grimmer
during 2023. While the war in Ukraine grinds

63

Pharma R&D Annual Review 2024

Copyright © 2024 Citeline

All the above feeds into a funding environment
for pharma that remains problematic. While thisaffects big pharma to a lesser degree, it could
be a major source of disquiet for smaller biotech
or start-up companies, which, as we haveseen, are legion. “The funding environment forbiopharma start-ups in 2024 is anticipated to begenerally challenging,” noted Koji Tamada, CEOof Japanese start-up Noile-Immune Biotech,also in Scrip. Michael Salako, investment director
at UK life science venture capital company Start
Codon, concurred: “Signs are that the funding
environment for 2024 is generally going to be adifficult one,” he said. “If the US Federal Reserve
starts to reduce interest rates, this would enable
capital to be moved out of risk-free money
market funds and into potentially higheryielding, riskier asset classes such as venture
capital. However, this kind of change takes timeto trickle down to those seeking funding. I’d
expect any benefit from a change like this to
only be felt by companies towards the end of2024 or early in 2025,” he stated.
What about the environment for deals? Anotherof our sister publications, Biomedtracker,
recorded 2,218 pharma deals in 2023, including
1,237 financing deals. These numbers were
down on their 2022 equivalents of 2,600 and
1,340, respectively, and continued an ongoing
downward trend. But here, the mood fromindustry commentators was notably more
optimistic. “I’m convinced that 2024 is theturning point for the biotech sector,” said Cary
Claiborne, CEO of Adial Pharmaceuticals, in
Scrip. “Low valuations may initiate a flurry ofacquisition activity, becoming an opportunity
during the harsh conditions.” Issei Tsukamoto,senior vice president, head of businessdevelopment at Astellas Pharma, concurred,noting, “While many faced a challenging year indeal-making, there is good cause for optimism
in 2024. M&A and financing activities in biotech
sector are returning, and we can hope for amore ‘normal’ if not quite a ‘boom’ in activity.”
One way to get a better sense of which way thewind is blowing is to look at industry output interms of new drug launches. This is a topic we’ll
be diving into in more detail in the forthcoming
companion volume to this report, the NAS
Supplement, which reports on the full list of newactive substances that made it onto the sunlit
uplands of commercialization during the year
just past. At the time of writing, mid-January2024, there is still some work to do to finalize
2023’s list (while drug approvals are usually64

Pharma R&D Annual Review 2024

widely reported, actual launches are less so,
so our team undertakes major research topresent you with a cast-iron list of products thatgenuinely received their actual market debut
during the year). Looking at the figures it looks
like 2023 has surpassed 2022’s number. But
check back in April to grab a copy of thisreport to be sure and to incorporate thismissing piece of the jigsaw.
So, the pharmaceutical industry has entered2024 snowed under by a plethora of new
drug candidates but seems to continue toblow hot and cold during the clinical stagesof development. Thus, it can’t be said to be
on cloud nine just yet. A continuing rising tideof new drug delivery to the market is vital if
occasional sunny periods are to give way tothere being not a cloud in the sky. As withclimate change, whether it is pleasant tomorrowis largely immaterial — it’s the longer-term
trends that matter, and will be something wewill continue to analyze. Come rain or shine,
the Citeline Pharma R&D Review will be backnext year to take the industry’s temperature
once again.

“The funding environment
for biopharma start-ups
in 2024 is anticipated tobe generally challenging…”
Koji Tamada,CEO of Japanese start-up
Noile-Immune Biotech

Copyright © 2024 CitelineAboutthe Author
Ian Lloyd
Senior Director, Pharmaprojects& Data Integration

Ian Lloyd is the Senior Director of Pharmaprojects
and Data Integration, overseeing the content andanalyst services for our drug development solution.
He supports clients in their drug pipeline datarequirements and inquiries, providing insight into
the best search strategies to answer their drugrelated business questions and also identifying
and analyzing trends in pharma R&D. For the past
32 years, he has authored the “Pharma Annual
R&D Review” and its new active substances (NAS)launches supplement. This has become a musthave industry report for those seeking to identify
the changing fortunes of drug R&D. Ian joined
Pharmaprojects in 1987, when it was part of PJB
Publications. It was acquired by Informa in 2003,
and spun out to form Citeline, now part of the
Norstella group, in 2022. He previously worked inmolecular biology as a research assistant at the
University of Bristol.
This reports also contains contributions from thefollowing analysts in the Pharmaprojects team:Jonathan Stephens, Sydney Enokawa, Tollan Bell,
Abdirazak Mohamed, JT Toebbe, and Arbesa Bela.

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Pharma R&D Annual Review 2024

Copyright © 2024 Citeline