EFPIA Viewpoint: Health Technology Assessment (HTA)
Application in Select Markets and Implications for Japan“Putting the patient at the center of healthcare decisions”

Copyright © 2015 by EFPIA JapanFor any inquires, please contact at info@efpia.jp
www.efpia.jp

Contents
1. Introduction		4

2. What is Health Technology Assessment (HTA)?

5

3. Healthcare in Japan Today

6

4. Lessons from HTA Abroad

9

5. Impact of HTA on Cancer Treatment

12

6. EFPIA’s Position on HTA in Japan13

7. Executive Summary		

16

1

I nt ro d u c t i o n

Introduction

National Health Insurance,1 given that aspects of HTA are alreadyembedded in the current pricing and reimbursement system.

In 2012, the Ministry of Health, Labor and Welfare (MHLW)
asked the advisory body, Chuikyo, to conduct a study on CostEffectiveness Analysis (CEA), an aspect of Health Technology
Assessment (HTA), and to make recommendations regarding
its introduction. Initially planned for launch in 2014, the greaterthan expected complexity of the task led to postponement of

Looking at the overseas experience, it is clearthat the introduction of an inappropriate HTA
system in Japan risks creating barriers to accessand undoing the progress Japan has made in
reducing its drug lag.

any introduction until April 2016.

EFPIA Japan believes it is important that decision-makers

Since 2012, a project team led by MHLW officials and a small

finalizing a proposal regarding the trial introduction of Cost-

group of health economists has focused its studies primarily on

Effectiveness Analysis (CEA) in April 2016. Specifically, EFPIA

CEA and HTA methodologies, drawing from experiences in thesuggests the following five principles guide the evolution and

UK and other countries. In June 2014, as part of specific measures

discussion of HTA in Japan:

collaborate in a transparent process with all stakeholders before

for healthcare in the Japan Revitalization Strategy, the Cabinet
requested the testing of CEA in the assessment for insurance
coverage of innovative medical technologies. Subsequently,
a pilot project was initiated involving five pharmaceutical andthree medical device companies with the intention of identifying
specific challenges that would result from the introduction of an
HTA system.

1. Involve all stakeholders in meaningful discussion(including patients, healthcare providers, andindustry) at all stages of the process
2. Set priorities for the initial, trial introduction of HTA
3. Focus on achieving better health outcomes, notsolely on costs
4. Ensure no negative impact on patient access or

The MHLW-led study has not yet solicited the views of a broadgroup of key stakeholders, such as patient advocates, clinicians,
epidemiologists, health policy specialists, and the pharmaceuticalphysician’s freedom to prescribe
5. Reward innovation and minimize burden to bothgovernment and industry

industry. These stakeholders each have a unique perspective,knowledge and experience of dealing with HTA systems in othermarkets, which are critical to informing the discussion in Japan.
The MHLW study has also not yet addressed Chuikyo’s initialconcerns regarding the purpose of introducing CEA and what
benefits could reasonably be anticipated. Indeed, it is unclear
where value will be added with respect to improved costeffectiveness within the context of Japan’s healthcare system and1 MHLW. 23 May 2012. Meeting Minutes. 1st meeting of Chuikyo HTA sub-committee. Retrieved fromhttp://www.mhlw.go.jp/stf/shingi/2r9852000002a7t6.html

About EFPIA Japan
EFPIA Japan represents 25 R&D-based pharmaceutical companies operating in Japan. Our Mission is to contribute to healthcare
and patients in Japan through the early introduction of innovative medicines and vaccines. To do this we encourage access tothe most innovative therapies in the shortest possible time; support Japan in becoming a more dynamic and attractive place inwhich to invest; and aim to be seen as a trusted healthcare partner.
The combined sales of our member companies account for about one quarter of the pharmaceutical market in Japan, and
EFPIA members account for around one-third of all new drugs approved in Japan. EFPIA member companies therefore deliversignificant health benefits to Japanese patients and are an important part of the Japanese pharmaceutical industry.

4

HTA Application in Select Markets and Implications for Japan

What is Healt h Tec hnology Assessment (H TA)?

What is Health Technology
Assessment (HTA)?

2• More collaborative: engaging a wider range ofstakeholders.

Micro-HTA vs. Macro-HTA?

The International Society for Pharmacoeconomics and HTA is just one of many tools available to improve health system
Outcomes Research (ISPOR) defines Health Technology
Assessment (HTA) as: “the process that uses evidence to evaluate
the clinical efficacy, cost-effectiveness and broader impact of ahealth technology on patients and the health care system.” 1

Additionally, HTA International (HTAi) stresses that the assessment
goes beyond clinical and economic aspects:2
HTA is a multidisciplinary field that addresses the clinical,
economic, organizational, social, legal, and ethicalimpacts of a health technology, considering its specifichealthcare context as well as available alternatives.
The scope and methods of HTA may be adapted to theneeds of a particular health system, but HTA processes
and methods should be transparent, systematic, andrigorous.
HTA can take many forms, and be used to address such healthcare
topics as:•

Drugs, biologics, devices, procedures•

Support systems, organizational, delivery andmanagement systems (e.g., disease management
programs, health care payment systems).

In contrast to this robust range of possible applications, CEA,
which is only one of several approaches within HTA, employs
a single outcome metric to compare costs and health effects
across different interventions. Furthermore, it is importantthat HTA be used appropriately. For example, according to theEuropean Network of HTA organizations (EUnetHTA), HTA is not a
replacement for proper price setting methodologies.

The definition of HTA continues to evolve
The definition of HTA continues to evolve, as does its application
and use. Across all markets where HTA is used, it is continuallyin a state of change: constantly adapting to incorporate newmeasures of value, reflect changing societal priorities, andaddress access challenges and patient needs. Although HTA is bynature contextual and takes different forms in different settings,
HTA is in general becoming:

performance. As with any methodology, it has both strengths andlimitations. It is critical that policymakers fully understand thesestrengths and limitations if they are to apply HTA appropriately.
HTA can be applied at either or both of two levels: the “MicroLevel” and the “Macro- Level.” Micro-HTA analyzes data to assess
the perceived value of a unique technology, e.g., pharmaceutical,
device, or medical procedure, to foster or limit its utilization, oras an input to therapeutic guidelines. As Micro-HTA looks only
at individual technologies, this alone will not likely result in theiroptimal use due to complexities and inefficiencies elsewhere in
the healthcare system.

In contrast, Macro-HTA focuses on policy, infrastructure andorganization, thus helping to guide or assess the policy-making
process in such areas as: organizational structure, public healthintervention programs, and the efficient allocation of resources.3
Given its more comprehensive approach, Macro-HTA is likely
a better choice to support policy decisions that achieve better
patient outcomes and more efficient healthcare systems.

Health	  Care	  Policy	
  Ques2on	“Macro-­‐HTA”	

Informed	  Policy-­‐Making	  	
  • More comprehensive: focusing on diseases and carepathways rather than individual technologies, andinforming broader policy decisions beyond pricing andreimbursement.
• More adaptive: continuously collecting / assessing dataand informing decisions across the life cycle.
1 International Society for Pharmacoeconomics and Outcomes. (2014). Health care cost, quality, andoutcomes: ISPOR book of terms.
2 Health Technology Assessment International. 2015. Health Technology Assessment. Retrieved fromhttp://www.htai.org/htai/health-technology-assessment.html• Priori%ze	
  public	
  health	
  interven%ons	
  • Op%mize	
  organiza%onal	
  structure	
  • Choose	
  appropriate	
  architecture	
  for	  healthcare	
  systems	  • Understand	
  the	
  economics	
  of	
  a	
  policy	  decision	  3 Towse, Adrian. 6 September 2014. Office of Health Economics. ISPOR 6th Asia-Pacific Conference. Broader
approaches to HTA stregthening health systems to improve patient care: introduction of the concept of‘Macro’ HTA.

HTA Application in Select Markets and Implications for Japan5

3

H eal t h c are i n Ja pa n To day

The Context—Healthcare in
Japan Today
Japan has one of the most successful national healthcare
systems in the world

Foreign governments, nonprofit organizations, and otherauthorities have recognized the Japanese healthcare systemrepeatedly for delivering unparalleled access to medicines andhigh-quality healthcare. Japanese authorities have succeeded indeveloping a healthcare model that:•

Provides universal access without restriction based onresidence, income or financial resources•

Provides high-quality treatments by properly trained
medical specialists in private medical practice or employedby public or private medical institutions•

Controls healthcare expenditures through adequate andwell-established mechanisms

Japan scores highly on health outcomes such as child mortality
and longevity, and yet historically, Japan has had one of thelowest healthcare costs to GDP ratio of any major developed
country. It is only in the past couple of years that Japan’s ratio hasrisen slightly above the OECD average, as a result of care for the
aging population and slower economic growth—not as a resultof pharmaceutical expenditure, which has grown only modestly.

on the Japanese health care system, which showed that thecurrent pricing system is a key part of its overall success.

“Japan is a country that achieves good health atrelatively low cost. As well as long life expectancy,
some indicators of healthcare are amongst the bestin the world”—2014 OECD Report.
“Low cost is achieved through nationally binding
prices based on a fee-schedule that is revised every
other year”—2014 OECD Report.
Despite this praise, for the future betterment of Japanese
healthcare, the OECD report also highlights key challenges theJapanese healthcare system will need to address in the coming
years:
Weak Primary Care: Japan has an opportunity to improve
many practices at the community physician level, adjusting
infrastructure to better fit Japan’s changing demographics.
This not only benefits patients, but will continue to improve
Japan’s healthcare quality and efficiency.
Weak Information Structure: Japan faces some challenges
due to an information structure that is underdeveloped andtherefore under-utilizes quality outcomes—highlighting theneed to improve infrastructure for evidence such as the RealWorld Evidence [that can be] used in HTA.

In 2014, the OECD and the World Bank issued separate reports
1

2

1 OECD. 5 November 2014. OECD Reviews of Health Care Quality: Japan. Retrieved from http://www.oecd.
org/els/health-systems/ReviewofHealthCareQualityJAPAN_ExecutiveSummary.pdf
2 Ikegami, Naoki (World Bank). 25 September 2014. Universal health coverage for inclusive and sustainable development: lessons from Japan. Retrieved from http://documents.worldbank.org/curated/
en/2014/09/20278271/universal-health-coverage-inclusive-sustainable-development-lessons-japan

Health	  Spending	
  in	
  OECD	
  Countries	
  as	
  Percent	
  of	
  GDP	  Japan	
  falls	
  in	
  line	  with	  OECD	
  na-ons	
  and	  performs	
  beAer	
  than	  many	  markets	
  where	
  HTA	  is	
  used	6

Figure	  1.	
  	
  Expenditure	
  on	
  healthcare	
  in	
  Japan	  rela2ve	
  to	
  other	
  OECD	
  countries.	
  Source:	
  Organiza2on	
  for	
  Economic	
  Coopera2on	
  and	  Development	  (OECD).	
  (2014).	
  OECD	
  Health	
  Sta-s-cs	
  2014—How	
  does	  Japan	  compare?	
  Retrieved	
  from:	
  hEp://www.oecd.org/els/health-­‐systems/Brieﬁng-­‐Note-­‐JAPAN-­‐2014.pdf	
  	
  	
HTA Application in Select Markets and Implications for Japan

Healt hc are in Jap an To day
Japan’s reduction of its drug lag has
been one of the great public policy45	
  successes of recent years

Over the past five years, the government

3

Increased	
  EFPIA	
  Member	  Company	
  Drug	
  
R&D	
  in	
  Japan	  Improved	
  PMDA	  Review	
  Times	  PMDA	
  has	
  nearly	
  eliminated	
  Japan’s	
  ‘drug	
  lag’	Innova7on	
  premium	  has	  contributed	
  to	
  boos7ng	
  drug	  R&D	

Number	  of	
  NME	  40	
  Average	
  Review	
  
Time	
  

35	
  

Change	  vs.	
  2009	Project	
  #	
  of	
  
RfUD	
  

10.1	
  Months	

+40%	
  	
  	
  	
  	
  	
  	
  	
  	
  +56%	
  	
  	
  	
  	
  	
  	
  	
  	
  	
  	
  +64%	
  	
  	
  	
  	
  	
  	
  	
  	
  	
  +71%	
  	
360	
  

Project	
  #	
  exluding	
  RfUD	
  

344	
  
327	
  

29
25

20
00
20 	
  
01
20 	
  
02
20 	
  
03
20 	
  
04
20 	
  
05
20 	
  
06
20 	
  
07
20 	
  
08
20 	
  
09
20 	
  
10
20 	
  
11
20 	
  
12
20 	
  
13
	
  

22
of Japan has taken major steps towards the
293	
  
30	
   28.3*RfUD	  =	
  Requested	
  
f
or	
  
elimination of the “drug lag”—the period
37
unapproved	
  drugs	
22.8
from first global launch to launch in Japan. 25	
  
21.5
211	
  
210	
  
20
19.1
19 19.1
5
9
18.3
182	
  
This has been achieved partly by doubling 20	
  
17.7
16.8
2
14.8
331
the number of staff at the regulatory agency, 15	
  
319
305
256
10.1 9.5 10.1
the Pharmaceutical and Medical Devices
10	
  
206
201
179
Agency (PMDA), and streamlining approval
processes. This has significantly reduced the 5	
  time for the regulatory review of new drugs 0	
  
2007	
  
2008	
  
2009	
  
2010	
  
2011	
  
2012	
  
2013	
  
(Figure 2). Another significant factor has been
Figure	
  
2
.	
  
	
  
Source:	
  医薬産業政策研究所.	
  2014.	
  日本における新薬のFigure	
  3.	
  Source:	
  Press	
  release	
  on	
  R&D	
  projects	
  of	
  15	
  companies	
  in	
  the introduction in 2010 of the “innovation 臨床開発と認審査の実積	
  (No.	
  63,	  p.	
  21).	
  	
  Japan	  (n	  =	  15).	
  *RfUD	
  =	
  Requested	
  for	
  unapproved	
  drugs.	
	premium”, which largely protects patented
direct control of the Chief Cabinet Secretary. Pharmaceuticals
products from price cuts. This has led to a sharp increase in theand medical devices have been identified as “globally promisingnumber of new drug development projects in Japan (Figure 3).
industries” that will play a central role in the Abe administration’s
Taken together, these policy changes amount to a significant
economic revitalization and growth strategy.
improvement in the environment for innovation, and as aresult many more innovative medicines are now available to Access in Japan
Japanese patients. Notwithstanding the success in reducingroad, rapid and stable access to medicines is a hallmark of thethe drug lag, Japan has not stopped there. The revision of thePharmaceutical Affairs Law, the creation of the Japan Agency for Japanese healthcare system; patients and physicians have theMedical Research and Development (AMED1) and the Sakigake treatment options they need. Based on EFPIA surveys of waiting
registration package, all indicate a desire to further boost the times, Japan tops the list, with reimbursement usually following
within 60 - 90 days of regulatory approval and immediately for
pro-innovation environment in Japan.
indication extensions. If HTA is applied to these new, innovative
The Japanese government also recognizes the strategic economic products, it has the potential to delay or disrupt access, as it
importance of the pharmaceutical industry. In February 2013, has done in many European markets. Chuikyo estimates that
the Office of Health and Medical Strategy was moved under the reassessment for CEA would add 3 to 6 months in Japan.2

B

1 AMED: Established in April 2015 to pool the public funding to healthcare research from three ministries:
MEXT, METI and MHLW.

2 Professor Fukuda at Chuikyo meeting of May 27th, 2015.

Europe	  –	
  PaMents	
  W.A.I.T.	
  Indicator	
  	
  Pa#ents	
  Wai#ng	
  to	
  Access	
  Innova#ve	
  Therapies	
Percent	
  Available	
  (2013)	
  Elapsed	  Mme	
  aNer	  EMA*	  approval	
  (days)	
  75

Japan	
  Denmark	
  102

Switzerland	
  

107

90

Switzerland	
  Switzerland	

83

Austria	
  89Finland	
  177

Finland	
  100

Denmark	
  

2013	
  
2010	

131

Austria	  Japan	  Japan:	  60-­‐90	
  days	

71

Sweden	  364

Sweden	  Belgium	  368

Belgium	
  Norway	  378

Norway	  Spain	  384

Spain	
  74

Italy	
  71

Italy	
  432

France	
  

443
602

Greece	
  

611
100	
  

200	
  

300	
  

400	
  

500	
  

*Elapsed	  Mme	
  between	  MarkeMng	
  AuthorizaMon	
  and	
  Accessibility	
  	

63
90

France	  Portugal	
  

0	
  

83

600	
  

63

Portugal	
  29

Greece	
  
700	
  

55
0	
  

20	
  

40	
  

60	
  

Japan:	
  100%	
80	
  

100	
  

**In-­‐country	
  availability.	
  In	
  %	
  of	
  medicines	
  with	  an	
  EU	
  central	
  authorizaMon	
  in	
  2011-­‐12,	
  e.g,	
  70	
  at	
  the	
  Mme	  of	
  survey.	

Figure	  4.	
  	
  Source:	
  EFPIA	
  annual	
  survey	
  2010	
  &	
  2013.	
  NB:	  UK	
  and	
  Germany	
  allow	  access	
  immediately	
  upon	
  markeMng	
  authorizaMon,	
  but	  HTA	
  hurdles—not	
  within	
  the	
  scope	  of	
  this	
  W.A.I.T.	
  analysis—apply	
  and	
  delay	
  actual	
  access.	
  *EMA:	
  European	
  Medicines	
  Agency.	

HTA Application in Select Markets and Implications for Japan7

3

H e al t h c are i n Ja pa n To day

Pharmaceutical Expenditure Not Driving
Healthcare Spending Increases

market through 2025. The study, recognizing pricing changes
that took place in 2014, including scheduled loss of exclusivity
after patent expiry and generic use incentives, shows slightmarket growth over the next 2 or 3 years, leveling off towards
2019 and receding as 2025 approaches.

While healthcare costs overall have increased in recent years,

this has been driven mostly by aging and long hospital stays,
which have a major impact on the public budget. Conversely,
pharmaceutical expenditure in Japan is projected to remain
stable over the next decade. A recent study completed by IMS in
partnership with EFPIA Japan, modeled Japan’s pharmaceutical

In 2014, the pharmaceutical market grew by only 1.4% even
including the consumption tax increase, and once the impactof the tax change is stripped out, the market actually shrank by
0.9%.

Costs	
  Well	  Controlled,	
  Market	
  Broadly	
  Flat	  (￥Tn)*1,2	
  Forecast	
  market	
  growth	
  by	
  segment	Innova:on	
  premium	
  scheme	  and	
  biennial	
  pricing	  con:nue	

0.13%	
0.14%	

1.77%	
9.9	

4.8	

10.2	

10.2	

4.8	

4.8	

10.5	

4.8	

10.4	

4.8	

-1.48%	
10.4	

10.6	

0.3	

4.6	

5.1	

5.0	

4.5	
0.8	

10.6	

4.8	
 4.8	

10.3	

10.4	

4.4	

4.3	

10.0	

10.0	

4.3	
4.4	
4.8	
 4.5	
 4.5	
 4.4	
 4.6	
 4.6	
 4.2	
 4.0	
 3.9	
3.9	
 3.9	

1.7	

2.0	

1.9	

1.0	

1.3	

1.3	

1.3	

1.2	

2.2	

2.0	

1.9	

1.8	

1.8	
1.7	
0.8	
 1.0	
 1.2	
 1.3	
 1.2	
1.6	
1.9	
1.8	
1.1	
1.7	
 1.7	
1.7	
1.5	
1.4	
1.6	
1.3	

1.6	

1.7	

1.8	

1.8	

1.9	

0.6	
’17
	

0.6	
’18	

0.6	
’19	

0.6	
’20	

0.6	
	
0.6	
’21

3.1	

2.7	

2.4	

1.1	

1.3	

1.4	

0.6	
’14	

0.6	
’15
	

0.6	
’16	

Original drugs	

LLPs (b)	

LLPs (a)	

2.0	

1.9	

2.0	

1.9	

0.6	
0.6	
0.6	
0.6	
’25	
 0.6	
’23
	
 0.6	
 0.6	
’24
	
0.6	
0.6	
 0.6	
0.6	
’22	
0.6	
 0.6	
 0.6	

Generics	

Others	0.6	

*1	
  LLPs	  (a)	  are	
  long-­‐listed	
  products	
  (LLPs)	
  whose	  ﬁrst	  generic	
  alterna:ve	
  was	
  launched	
  before	
  2013.	
  LLPs	  (b)	  are	  the	
  other	  LLPs,	
  whose	  ﬁrst	  generic	
  compe:tor	
  is	
  launched	
  aDer	  2013.	
  *2	
  Sales	  are	
  calculated	
  assuming	  that	  the	  5%	  consump:on	
  tax	
  rate	  that	  existed	
  on	
  1	
  January	  2014	
  con:nues	
  throughout	
  the	  period.	
  This	  is	  in	
  order	
  to	
  look	  at	
  the	
  underlying	
  growth	
  in	
  the	
  market,	  stripping	
  out	  the	  consump:on	
  tax	
  eﬀect.	
  Figure	  5.	
  	
  Source:	
  IMS	
  Consul:ng	
  Group.	

Generic promotion providing significant cost savings

The governments’ efforts to increase generic uptake in Japan

have greatly boosted the generic market share over the past
year, and projections based on current policies demonstrate anincreased market share through 2025. The expense increase asa result of the innovation premium is more than offset by theincreased market share of generics.

Therefore, it is very important that any policy decision, such as
the introduction of HTA, is made with a full understanding of
the impact it will have on the healthcare system. HTA has caused
access delays in every market where it has been introduced—
Japan should avoid undoing all the progress made in shortening
the drug lag by implementing an inappropriate system.

Savings	
  from	  Generic	
  Promo0on	
  Greater	
  than	  Cost	  of	
  “Innova0on	
  Premium”	
  Improve the current system as
it provides timely access and70%
controls costs well

Japan has created a healthcare

60%
50%

Innova&on	
  premium	
  encourages	
  drug	  R&D	
  
59%

54%	

48%
48%	
44%
40%

63% 65%
51%

53%

66%
55%

70%

1,000	

500	*2	

Expense increase by the premium	
Expense reduction by Gx promotion measures 1)	

59%

Balance of payment	

190	
 141	

242	
 248	

309	
 325	

390	
 407	

456	
 469	
 500	

503	
 489	

438	

-7	
33%
system that both controls costs 40%
0	
-136	
40%
-176	
26%
-195	
 -228	
-183	-247	 -244	
37%-265	and provides quick and broad 30% 25% 29% 33%-304	
 -313	
-197	
-258	Actual Gx penetration	
-275	
-316	
access to patients. The efforts of 20%
-363	
-379	
-500	
Forecasted Gx penetration
-494	
-552	
 -583	
-585	(based on this project results)	
the government to address the 10%
-639	
-636	
-734	
-775	
Forecasted Gx penetration
-802	
-800	
(using trend before 2010)-807	
drug lag, promote innovation, and-1,000	
0%
	
 ’19
	
 ’25
	
 ’21
	
 ’22
	
 ’14	
 ’15
	
 ’20
	
 ’23
	
 ’24
	
’12	
 ’13
	
 ’16	
 ’17
	
 ’18
’03
	
 ’05
	
 ’07
	
 ’09
	
 ’11
	
 ’13
	
 ’15
	
 ’17
	
 ’19
	
 ’21
	
 ’23
	
 ’25
	
increase the generic market share
Average impact
Sum of Impact
per year	(2012 – 2025)	
have provided savings and also
Without	
  addi0onal	
  measures	
  for	
  generic	
  penetra0on,	
  Expense	  Increase	
￥365	
  Bn	
  
￥5,105	
  Bn	
  Generic	
  penetra0on	
  
r
ate	
  
w
ould	
  
g
row	
  
a
ccording	
  
t
o	
  
t
he	
  
helped to create an environment
trend	  before	
  2010	
  and	
  reached	
  at	
  59%	  in	
  2025	
Expense	  Reduc0on	
￥593	
  Bn	
  
￥8,298	  Bn	
  conducive to innovation and
Figure	
  6.	
  *1	
  	
  We	
  used	
  Gx	
  penetra0on	
  trend	
  from	  2006	
  to	
  2009	
  	
  *2	
  	
  We	
  assumed	
  Gx	
  penetra0on	
  rate	  will	  not	
  grow	
  in	
  2021	
  and	
  2022,	  same	  as	
  this	  research.
project	
  calcula0on	
  result.	
  Source:	
  EFPIA	
  Japan	
  and	  IMS	
  Joint	
  Simula0on	
  Project.	
  
*1	

8

	
  

HTA Application in Select Markets and Implications for Japan

Lessons from HTA Ab ro a d

4

How HTA is Applied Around theWorldclosed environment of integrated payer-provider of the US

Unlike Japan, where the price of new drugs and price revisions istheir usage, at the expense of choice for prescribers.

set according to a detailed set of rules, some EU countries decide

Broadly, there are two main approaches to the application of

on reimbursement coverage and prices in consideration of Cost-

HTA: [1] countries like the UK or Australia (with a centralized

Utility Analysis (CUA or cost per QALY, CPQ) or Cost-Effectivenessnational health system) decide reimbursement coverage based

Analysis (CEA). The most visible consequences are additionalon modeling and an economic, monetary threshold across all

delays and, frequently, the denial of access to many innovativeinterventions and indications, while [2] countries like France andproducts, an additional level of medical review after regulatory

Germany (mix of private and public medical practice, similar to

approval, and the creation of a large bureaucracy.

Japan) assess and rate the relative medical benefits (Relative

Since its introduction in countries like Australia (1993), Canada(1994) or the UK (1999), HTA has become different from its original

managed care organizations, which track and analyze their own
outcomes, HTA is used to support product selection and force

Effectiveness Assessments, REA) as a basis for a negotiation of
the price.

intent, e.g., to solve regional differences in reimbursement in
the UK and Canada. Recent trends point to HTA being used tosupport pricing decisions or to restrict access to innovative
medicines deemed too expensive for public budgets. In the

1. Reimbursement	
  based	  on	
  set	  Cost	  Per	  QALY	
  (CPQ)	
  	
  Impact	
  of	
  	
  
HTA	
  on	
  
Reimbursement	
  Decision	
  Making	

Decide	  reimbursement	
  coverage	
  based	  on	
  economic	
  threshold	
  across	  all	
  interven4ons	
  and	  indica4ons,	
  broadly	
  impac4ng	
  pa4ent	  access	
  (e.g.,	
  UK	
  &	
  Australia).	
  2. Price	
  negoDaDon	
  based	  on	
  assessment	
  (Non-­‐CPQ)	
  	
  Price	
  nego4a4on	
  	
  based	  on	
  assessment	
  demonstra4ng	
  rela4ve	
  medical	  beneﬁt,	
  s4ll	  impac4ng	
  access,	
  but	
  less	  so	
  than	  CPQ	
  (e.g.,	
  France	
  &	
  Germany).	

No country has found the ideal systembeen set up in March 2015 to begin review of the current HTA
system.

No country has found the ideal system and CEA itself has not Generally, because all countries experience delays in access,prevented increases to healthcare costs as a result of aging.
Currently, countries are reconsidering their HTA principles andmethodologies with renewed emphasis on improving patient
access, as there are great inequalities across European countries.
One example is the creation of the Cancer Drug Fund in England

the trend is for Coverage with Evidence Development (CED), a
convergence of pharmacovigilance and real-life observation foreconomic assessment, and for adjustment of price and coverageover time.

in 2010.1 In Scotland, after a review of the HTA process, patientsare now specifically asked for their opinion when end-of-life ororphan drugs are rejected by the Scottish Medical Council (SMC),
the HTA agency. Additionally, in France, a working group has

1 NHS England. 2015. The Cancer Drugs Fund. Retrieved from: http://www.england.nhs.uk/ourwork/pe/cdf/

HTA Application in Select Markets and Implications for Japan9

4

Les s o n s fro m HTA Abro a d

HTA in the NewsHTA often makes headlines, but not for the reasons it should.

valuable information to policymakers, it is critical that patientsand physicians are part of the decision-making process and thatmaintaining access continues to be a key government priority.

HTA agencies in the UK (NICE), Australia and Canada have been
the target of numerous protests by patients and physicians
where HTA was applied by governments to limit patients’
access to new and innovative therapies. While HTA can provide“Patients protest after kidney drugs rejected [by NICE]”—The Guardian (England)1

“NHS set for record £1.75bn surplus as patients protest
over [access to] cancer drugs”—The Telegraph (England)2“Pancreatic cancer patients to pay $15,000 or miss out”
“A drug company will stop supplying cut-price treatment for pancreatic cancer
after the PBAC rejected a deal to list the medication for subsidy”—Herald Sun (Australia) 3“Alzheimer’s drugs [access] court challenge”“The first time a judicial review has been sought on a NICE decision”—BBC News (England)4

1 Patients protest after kidney cancer drugs rejected. (2008 August 27). The Guardian. Retrieved from http://www.theguardian.com/society/2008/aug/27/health.cancer
2 Smith, R. NHS set for record £1.75bn surplus as patients protest over cancer drugs. (2008 August 27). The Telegraph. Retrieved from http://www.telegraph.co.uk/
news/health/2633064/NHS-set-for-record-1.75bn-surplus-as-patients-protest-over-cancer-drugs.html
3 Mcarthur, G. Pancreatic cancer patients to pay $15,000 or miss out. (2014 March 26). Herald Sun. Retrieved from http://www.heraldsun.com.au/news/pancreaticcancer-patients-to-pay-15000-or-miss-out/story-fni0fiyv-1226865659707
4 Alzheimer’s drugs court challenge. (2007 June 25). BBC News. Retrieved from http://news.bbc.co.uk/2/hi/health/6230530.stm

10

HTA Application in Select Markets and Implications for Japan

Lessons from HTA Ab ro a d

4negative impact on patient access, limited patient involvement,
Global Overview of HTA Systems
the creation of a large, expensive bureaucracy, and in some, a lacktransparency. This comparison highlights the need to develop
Where HTA has been introduced, it has taken on a number ofa systemcontextualized for the Japanese market—addressingof forms. The chart below provides a brief overview of the HTA
systems in five selected markets: France, Germany, UK, Sweden,
and South Korea. Commonalities among all markets are aFrance	Authority	  	

the specific needs of patients and the healthcare system.

Germany	UK	

Sweden	South	
  Korea	

Japan	

Organiza2on	
  	

HAS	

G-­‐BA/IQWiG	NICE	  (+CHTE)	
  	

TLV	

HIRA	

N/A	

Staﬀ	
  
Number	350	

300/160	

560	

125	

2,121	

-­‐	

54	
  M	
  EUR	
  
7.53	
  B	
  Yen*	

18	
  M	
  EUR	
  	
  

2.51	
  B	
  Yen*	

£60.8	  M	
  11.64	  B	  Yen*	  	

155.74	
  M	
  SEK	
  
2.33	
  B	
  Yen*	

324	
  B	
  KRW	
  36.25	  B	  Yen*	

-­‐	

2011	
  AMNOG	

1999	

2002	

2006	

-­‐	

Budget	
  	

Year	
  of	
  Introduc2on	

2013	
  UPDATE	Some	
  Impact	
  on	
  Access	  Impact	  on	
  Access	

France	  &	
  Germany	
  use	  HTA	  before	
  price	  nego2a2ons	
  leading	  to	
  limited	
  direct	  impact	  on	
  access.	
  S2ll	  some	  examples	
  of	
  impact	  or	
  limited	
  access.	
  	
  Signiﬁcant	  Impact	
  on	
  Access		
  Fixed	  Cost	  Per	  QALY	
   Long	
  2me	  to	
  create	
  requested	
  evidence	  (CPQ)	
  threshold	
  delays	  launch.	
  nega2vely	
  impacts	  access.	
  Extensive	  value	  dossier	  requirements,	
  and	
  strict	
  CPQ	
  leads	
  to	  very	  limited	
  access.	
  Ini2a2on	
  of	
  HTA	
  triggered	  by	
  growing	
  drug	
  costs	

Products	
  Chosen	
  for	
  Assessment	-­‐	
Medical	  Economic	
  Evalua2on	
  (MEE)	
  is	
  undertaken	
  for	  selected	  products	  based	  on	
  healthcare	
  priori2es.	
  	
  Selected	  products,	
  All	
  new	  priori2es	
  pharmaceu2cals	
  except	  those	
  that	  are	
   determined	
  by	
  an	
  
academic	
  group	
  reference	
  priced.	
  through	
  “Horizon	  (*Hospital	
  products	  Scanning.”	
  outside	
  scope	
  of	
  HTA).	
  All	
  new	
  pharmaceu2cals	
  	

-­‐	

(*Hospital	
  products	  outside	
  scope	
  of	
  HTA).	
  -­‐	

Decisions	
  	
  	
  	
  	
  	
  	
  	
  	
  	
  	
  	
  CPQ	
Mainly	  (Clinical	
  Driven	  By	
  	
   Non-­‐CPQ	
  Eﬀec2veness)	

Pa2ent	  Input	
  in	
  HTA	
  Process	  	
  Transparency	  	
  	

Currently	
  broad	
  access	
  is	
  supported.	

-­‐	Li?le/No	  Involvement	  	
  Some	  Involvement	  Pa2ents	
  do	
  not	  have	  a	
   Limited	
  input	  by	
  regular	
  role	  in	
  the	
  pa2ents	
  (e.g.,	
  evalua2on	
  process.	
  pa2ent	  org.	
  &	
  socie2es).	
  

Some	
  Involvement	  Limited,	
  but	
  formally	
  well-­‐structured.	
  Scotland’s	
  SMC	  allows	  for	
  signiﬁcant	
  pa5ent	
  involvement.	
  Li?le/No	  Involvement	  	
Pa2ents	
  do	
  not	  have	  a	
  regular	
  role	  in	
  the	  evalua2on	
  process,	
  
nor	
  a	
  vote	  in	
  the	  decision.	
  

-­‐	

Clear	
  criteria	
  and	  The	
  process	
  of	
  
All	
  guidance	
  is	
  to	
  be	
   The	  process	
  is	
  very	   Lack	
  of	
  transparency	
   Transparency	
  and	  conduc2ng	
  MEE	  is	
   methodologies	
  are	
   published	
  and	  the	
  unpredictable	
  and	
   in	  HTA	
  process	
  and	
   collabora2on	
  have	  communicated	
  to	
  clear,	
  but	
  lack	
  of	
  
its	
  impact	
  on	
  pricing.	
   been	  very	  limited.	
  not	
  transparent.	
  evidence	
  on	
  which	  There	
  has	  not	  been	  transparency	
  of	
  how	
   stakeholders	
  before	
   decision	
  was	
  made	  is	
   Only	  a	
  shorter	
  extensive	  dialogue	
  MEE	
  impacts	  pricing	
   an	  HTA	  is	  conducted.	
   available	
  to	
  the	  summary	
  of	
  the	
  ﬁnal	  Subsequent	
  price	  with	  industry,	  decisions.	
report	  is	
  published.	
  company.	
  nego2a2on	
  lacks	  pa2ents,	
  or	
  other	  transparency.	
  stakeholders.	
MEEs	
  undertaken	
  for	
  new	
  drugs	
  are	  reassessed	
  amer	  5	
  years.	
  Notes	
  Product	
  enters	
  the	
   The	  UK	
  has	  High	
  costs	  to	
  market	  with	  free	  introduced	
  pa2ent	
   develop	
  Health	
  pricing	  un2l	
  HTA	  is	
   access	
  schemes,	  Economic	
  models	
  carried	
  out.	
  Process	
   such	  as	
  Cancer	
  Drug	
   which	
  take	  a	
  takes	
  about	
  12	
  signiﬁcant	
  amount	  Fund,	  months	  un2l	  pricing.	
   acknowledging	
  the	
   of	
  2me.	
  nega2ve	
  impact	
  HTA	   	
  *Methodology	
  o<en	  Societal	
  aspects	  and	
  applied	  so	
  strictly	
  that	   has	  on	
  access.	
  	
costs,	
  e.g.,	  indirect	
  many	
  assessments	  	
  result	  in	
  “no	  costs	  are	
  usually	
  conclusion.”	
  considered.	
  	

-­‐	

Japan	
  has	
  yet	  to	
  iden2fy	
  how	  HTA/CEA	
  adds	  value	  to	  Japan’s	
  pricing	  system	  and	
  biennial	
  pricing	  revisions.	
  	*Lean	
  dossier	
  for	  orphan	  drugs	  *Based	  on	
  June	  2015	
  exchange	
  rates.	
  

HTA Application in Select Markets and Implications for Japan11

4

I mp ac t o f H TA o n Ca n cer Treatment

Impact of HTA on Cancer
Treatment•

Some data show lower rates of both cancer drug spending
and cancer survival in countries using CPQ methodologies,
particularly the U.K.

HTA can, and has, negatively impacted access within particular•

CPQ countries do not necessarily spend less overall on
cancer, but they may achieve less for patients.

therapeutic areas—particularly oncology. Patients in countries
that apply a strict cost-per QALY (CPQ) measure have restricted
access to innovative medicines and lower overall cancer survival
rates than patients from countries that are non-CPQ. In 2014, IMS
published a study that looked at the impact of cost-per-QALY
reimbursement criteria on access to cancer drugs.*
The study made the following primary findings:1•

In the five CPQ countries examined (England, Scotland,
Sweden, Canada, & Australia), patients have less accessto new cancer drugs than patients in the five non-CPQ
countries (U.S., France, Germany, Italy, & Spain).

•In these five CPQ countries, fewer new cancer drugs are
reimbursed; reimbursement decisions take longer; andnew cancer drugs have historically been adopted more
slowly and, in the longer term, at lower rates.

•

CPQ analyses are subject to many uncertainties andinconsistencies due to the nature of the variables used andtheir interpretation.

1 IMS Institute for Healthcare Informatics. December 2014. Impact of cost-per-QALY reimbursement criteria
on access to cancer drugs.

*Nine Products Reviewed: Afinitor, Halaven (eribulin), Votrient (pazopanib), Jevtana(cabazitaxel), Zytiga (abiraterone acetate), Yervoy (ipilumab), Zelboraf (vemurafinib),
Azerra (ofatumumab), and Xgeva (denosumab).

Fewer new cancer drugs are reimbursed
While both CPQ and non-CPQ countries reimburse most noncancer products, CPQ countries reimburse fewer cancer therapies
resulting in limited access for their patients. Specifically, four ofthe five CPQ countries surveyed deny reimbursement of three ofthe nine cancer drugs reviewed—markedly worse than in nonCPQ countries.
In particular, recent reports from the NHS of the U.K., have foundthat overall survival rates of cancer patients in the U.K. are lagging
10 years behind the rest of Europe.2 3 While there are a numberof factors that contribute to this disparity, including diseaseawareness and early diagnosis, uneven and low access has also
been identified as a challenge patients face.

2 NHS Choices. 5 December 2013. UK cancer survival rates below European average. Retrieved from http://
www.nhs.uk/news/2013/12December/Pages/UK-cancer-survival-rates-below-European-average.aspx
3 Foot, Harrison, Tony. June 2011. How to improve cancer survival: explaining England’s relatively poor
rates. The King:s Fund.

NaDonal	
  Reimbursement	
  Status	
  Reimbursed	

Fewer	  new	
  cancer	
  drugs	
  are	  reimbursed	
  in	
  CPQ	
  countries	
  Cancer Products	

CPQ Countries	

Non-Reimbursed

Non-launched/Non-marketing approved	

Non-CPQ Countries	
11%	
  

33%

44%

56%

44%

100%	
  
67%	
  

56%	
  

44%	
  

England	U.K.	
  Scotland	
  Sweden	  Canada	
  

100%

100%

100%

U.S.	
  

France	  Germany	  Italy	  Spain	
  Non-CPQ Countries	
5%5%Australia	
  

5%

95%	
  

95%	
  

100%	
  

England	U.K.	
  Scotland	
  Sweden	
  

89%

56%	
  

Non-Cancer Products	

CPQ Countries	
5%

100%

21%

21%

79%	
  

79%	
  

Canada	  Australia	
  

16%

100%

100%

95%

95%

Germany	  Italy	  Spain	
  

84%

U.S.	
  

France	  Figure	  7.	
  	
  Source:	  	
  IMS	
  InsDtute	
  for	
  Healthcare	  InformaDcs.	
  December	
  2014.	
  Impact	
  of	
  cost-­‐per-­‐QALY	
  reimbursement	
  criteria	
  on	
  access	
  to	
  cancer	
  drugs.	
  *In	
  Sweden,	
  reimbursement	
  at	
  the	
  county	
  level	  was	  considered,	
  as	
  naDonal-­‐level	
  reimbursement	
  decisions	  are	  not	
  made	  for	
  hospital	
  drugs.	
  Fig.	
  19	
  lists	
  consideraDons	
  for	
  other	
  markets.	
  Sources:	
  NaDonal	
  InsDtute	
  for	
  Health	
  and	  Care	  Excellence	
  (NICE)	
  (England),	
  ScoXsh	  Medicines	
  ConsorDum	  (SMC)	
  (Scotland),	
  The	
  Dental	
  and	
  PharmaceuDcal	
  Beneﬁts	
  Agency	  (TLV)	
  (Sweden),	
  Reimbursement	
  status	
  listed	
  for	
  Ontario,	
  BriDsh-­‐Columbia	
  and	  Alberta	
  (Canada),	
  PharmaceuDcal	
  Beneﬁts	
  Scheme	  (PBS)	
  (Australia),	
  Base	  des	
  Médicaments	
  et	  InformaDons	
  Tarifeires,	
  eVidal	  (France),	
  Federal	
  Joint	
  Commibee	
  (GBA)	
  (Germany),	
  Italian	
  Drug	  Agency	
  (AIFA),	
  paginesanitaria	
  (Italy),	
  BotPLUS	
  (Spain)	
  	
  

12

HTA Application in Select Markets and Implications for Japan

EFPIA’s Posit ion on HTA in Ja p a n

6always requires making assumptions and modeling
EFPIA’s Position on HTA in Japan drugs
outcomes due to the lack of real-life usage in the clinical setting.
often results in increased uncertainty and may adversely
Japan’s healthcare system delivers excellent outcomes and an This
impact decision-making. As such, EFPIA recommends that any
outstanding level of access while simultaneously controlling
costs. As such, EFPIA recommends that the first priority of MHLW
should be the progressive adaptation and implementation ofpolicy changes within Japan’s current healthcare system, e.g.,
NHI pricing, technical fees, PMS and increasingly on prevention
and care continuum through referrals, medical guidelines,
integrated care, etc. EFPIA believes that placing a priority on
further improvements to the current system is most likely toresult in long-term cost containment together with continued
broad access to innovative medical treatments in Japan.

HTA system adopted by Japan be applied solely to marketed
products at the time of re-examination or re-pricing as real-life
drug usage and performance post-launch can be combined, as
appropriate, with clinical data and modeling to provide a morecomprehensive and accurate assessment of a therapy’s actual
clinical usage and value.
Experience in other countries demonstrates that HTA systems
often impose significant burdens on industry and governments.
MHLW must keep in mind that an excessively burdensome HTA
system will negatively impact industry’s ability to invest in R&D
in Japan. EFPIA therefore recommends that prior to any decision
to adopt HTA that MHLW establish a dialogue with industry
and other stakeholders to assess data collection requirements,
assessment measures, costs and other factors with the goalof developing pragmatic and creative solutions that minimize
burden.

Analysis across multiple markets where HTA has been applied to
new drugs reveals that its inappropriate use has had a significant
negative impact on access, particularly to cancer drugs, forpatients and physicians. It is critical, therefore, that Japan notsimply adopt methodologies developed for use within foreign
healthcare systems that differ drastically from Japan’s. EFPIA
strongly recommends that in considering the possible adoptionof HTA that MHLW harness the intellectual resources of healtheconomists, industry, physicians, and patients to assist policymakers in the task of identifying methodologies specificallytailored to Japan’s unique needs. For example, assessing newSpecifically, EFPIA suggests the following five principles
guide the discussion and evolution of HTA in Japan:

Five Principles to Guide HTA in Japan1	
Collabora'on	  “Pa$ent	
  Centered”

2	

stakeholders	
  in	
  

Limited	  Introduc'on	
  meaningful	  Set	
  priori<es	
  for	
  Involve	
  all	  discussion	
  (including	
  pa<ents,	
  healthcare	
  

	
  

ini<al,	  trial	
  introduc<on	
  of	
  HTA	  3	Outcomes	
  &	
  Real	  World	
  Evidence	  	
  Focus	  on	
  achieving	  providers,	
  and	  

beFer	
  outcomes,	
  not	  industry)	
  at	
  all	
  stages	
  solely	  on	
  costs	
  of	
  the	
  process	
  	
  	
  

4	
No	
  Nega've	  Impact	  on	
  Access	
  Ensure	  no	
  nega<ve	
  5	
Minimize	
  Burden	  impact	  on	
  pa<ent	  Reward	  innova<on	
  access	  or	  	
  physician's	
  and	
  minimize	
  the	
  freedom	
  to	
  prescribe	
  burden	  to	
  both	  government	
  and	  industry	  	
  HTA Application in Select Markets and Implications for Japan

13

4

E FP I A’s Po s it i o n o n HTA i n Ja pa n

Five Principles to Guide HTA in Japan1	
 Collabora'on	
  •EFPIA	
   Japan	
   believes	
   that	   the	   understanding	
   and	   involvement	
   of	
  pa:ents	
   in	
   determining	
   the	   impact	
   of	
   therapy	   on	
   their	
   health	
   and	  quality	
   of	
   life	
   is	
   cri:cal	
   for	   sound	
   policy-­‐making.	
   Involving	
   pa:ents	
  can	
  lead	  to	
  more	  subjects	
  involved,	
  beCer	
  data	  and	
  beCer	  outcomes,	
  and	
   also	
   give	
   pa:ents	
   a	
   beCer	
   understanding	
   of	
   their	
   own	  responsibility	
   towards	
   care,	
   preven:on	
   and	   the	   use	   of	
   scarce	  resources.1	
  

•

As	
   HTA	   can	
   nega:vely	
   impact	
   access,	
   healthcare	
   providers	
   and	  medical	
  socie:es	
  should	
  be	
  a	
  key	  part	  of	
  the	  discussion.	
  •Ensure	   transparency	
   in	
   the	   HTA	   process,	
   including	   the	   selec:on	
  criteria	
  for	  reviewed	
  products,	
  the	
  analysis	
  methodology,	
  and	  the	
  use	  of	
  the	
  analysis	
  in	
  any	
  pricing	
  decisions.	
  	
  

•

EFPIA	
   Japan	   recommends	
   that	
   MHLW	
   and	   Chuikyo	
   involve	
   the	  industry	
   much	   more	   in	
   the	   debate,	
   given	
   industry’s	
   extensive	  experience	
  of	
  HTA	  in	
  other	
  markets.	
  Limited	  2	
 Introduc.on	
  

•

The	
   eﬀort	
   and	   resources	
   required	
   by	
   the	   introduc3on	
   of	
   HTA,	
   even	
   if	  limited	   to	
   CEA,	
   are	   considerable.	
   The	   cost	   and	   3me	   required	
   to	
   build	
  capabili3es	
  and	
  infrastructure	
  should	  not	  be	
  underes3mated.	
  Set	
  priori*es	
  for	
  ini*al,	
  •Exis3ng	
   data	
   collec3on	
   systems,	
   such	   as	   post-­‐marke3ng	
   surveillance	
  Involve	
  all	
  stakeholders	  in	
  meaningful	  discussion	
  (including	
  pa8ents,	  healthcare	
  providers,	
  and	
  industry)	
  at	
  all	
  stages	
  of	
  the	
  process	
  	
  	
  

and	
   pharmacovigilance	
   or	   disease	
   registries,	
   should	
   be	
   customized	
   to	
  generate	  real-­‐life	
  data	  sets	  during	
  the	
  life	  cycle	  of	
  drugs.	
  trial	
  introduc*on	
  of	
  HTA	
  
	
  
•

HTA	
   is	
   beDer	   suited	
   to	
   products	
   already	
   on	
   the	   market,	
   not	   at	
   new	  products	
   about	
   to	
   be	
   launched	
   for	   which	
   no	
   experience	
   and	   only	  limited	
  data	  are	  available.	
  	
  

•

The	
  applica3on	
  of	
  HTA	
  should	
  remain	
  ﬂexible,	
  recognizing	
  therapeu3c	  area	
   diﬀerences	
   and	   exemp3ng	
   speciﬁc	
   classes	
   (e.g.,	
   orphan	
   drugs,	
  oncology,	
  unmet	  medical	
  need,	
  etc.)１ HTAi. (2015). HTA Resources for Involving Patients & Citizens. Retrieved from http://www.htai.org/interest-sub-groups/patient-and-citizen-involvement/pcisg-resources/hta-resources.html

14

HTA Application in Select Markets and Implications for Japan

EFPIA’s Posit ion on HTA in Ja p a n•HTA	
   could	
   be	
   used	   to	
   assess	
   the	   most	
   eﬀec2ve	
   treatment	
   paths	
   for	   a	
  given	   disease,	
   looking	
   at	   all	   the	   treatments	
   required	
   and	   not	   only	
   the	  medicines.	
   In	
   doing	
   so,	
   such	   a	
   “Macro-­‐HTA”	
   approach	
   could	
   deliver	
  beDer	
  outcomes	
  for	
  pa2ents.	
  

•

To	
  improve	
  outcomes,	
  it	
  makes	  more	  sense	  to	
  combine	
  results	
  from	  randomized	
   control	
   trials	
   (RCT)	
   with	   other	
   data	
   sources	
   and	   real	
  world	
   evidence	
   (RWE),	
   capturing	
   how	
   diseases	
   are	   actually	
   treated	
   in	  real	
  clinical	
  prac2ce,	
  rather	
  than	  limi2ng	
  the	
  analysis	
  to	
  the	
  ar2ﬁcial	
  environment	
  of	
  a	
  regulatory	
  clinical	
  trial.	
  

•

•

•

Any	
  decision	
  to	
  implement	
  HTA	  on	  a	
  trial	
  basis	  in	
  April	
  2016	
  must	  not	  reverse	  the	
  great	  progress	
  Japan	
  has	
  made	  in	
  reducing	
  the	
  drug	  lag,	
  shortening	
   the	   regulatory	
   review	
   period	
   for	   new	
   drugs	
   and	   new	
  indica@ons,	
  and	
  ensuring	
  access	
  to	
  innova@on.	
  HTA	
   should	
   not	   restrict	
   physician	   choice.	
   Physicians	
   should	
   retain	
   the	  authority	
  to	
  select	
  the	  best	  therapy	
  for	  their	
  pa@ents.	
  Any	
   trial	
   introduc.on	
   of	
   HTA	   should	
   impose	
   the	   minimal	
   burden	
  necessary	
  on	
  both	  government	
  and	  industry	
  by	
  avoiding	
  unnecessary	  costs	
  and	
  crea.on	
  of	
  bureaucracy.	
  These	  resources	
  would	
  be	
  be<er	  spent	  on	  research	
  and	
  improving	
  outcomes	
  for	
  pa.ents.	
  

	
  	
  
• To	
  con.nue	
  to	
  a<ract	
  innova.on,	
  Japan	
  needs	
  to	
  oﬀer	  companies	
  a	
  predictable	
   and	   a<rac.ve	
   environment.	
   To	
   do	
   this,	
   ﬂexibility	
   will	
  likely	   be	
   required,	
   together	
   with	
   a	
   recogni.on	
   of	
   the	   challenges	
   of	  data	
  collec.on.	
  Pragma.c	
  and	
  crea.ve	
  solu.ons	
  may	  be	
  necessary.	
  	
  • The	  types	  of	
  data	  required	
  and	  the	
  assessment	
  measures	
  used	  should	  be	
  agreed	
  through	
  industry	
  and	
  government	
  dialogue.	
  Further Considerations: Use HTA to guidetreatment and policy-making, not set prices or restrict
accessRather than using HTA to determine pricing and reimbursement
decisions for individual technologies, where Japan alreadyhas an effective NHI price system, resources could be spent

63	

Outcomes	
  &	
  
Real	
  World	
  Evidence	
  Focus	  on	
  achieving	  be0er	
  outcomes,	
  not	
  solely	  on	
  costs	
  No	
  Impact	
  on	
  Access	  4	

Ensure	  no	
  nega,ve	
  impact	
  on	
  pa,ent	
  access	
  or	
  	
  
physician's	
  freedom	
  to	
  prescribe	
  Minimize	
  Burden	  5	

Reward	  innova-on	
  and	  minimize	
  the	
  burden	
  to	
  
both	
  government	
  and	  industry	  	
  more meaningfully to address broader issues such as disease
prevention, development of disease management programs,
and healthcare delivery system planning. It is in these areas thatthe greatest opportunities for improvements lie, as suggested in
the 2014 OECD and World Bank Reports on Healthcare in Japan(see page 6).
HTA Application in Select Markets and Implications for Japan

15

Executive Summary
EFPIA Viewpoint: HTA Application in Select Markets and Implications for Japan
EFPIA member companies have significant experience in theapplication of HTA in European markets, and are familiar withthe challenges that arise as a result of such systems.

Specifically, EFPIA suggests the following five principles guide theevolution and discussion of HTA in Japan:

1. Involve	
  all	
  stakeholders	
  in	
  meaningful	  discussion	
  (including	
  pa9ents,	
  healthcare	
  providers,	
  and	  industry)	
  at	
  all	
  stages	
  of	
  the	
  process	
  	
  	
  
2. Set	  priori9es	
  for	
  ini9al,	
  trial	
  introduc9on	
  of	
  HTA	
  3. Focus	
  on	
  achieving	  beEer	  outcomes,	
  not	
  solely	
  on	
  costs	  4. Ensure	
  no	
  nega9ve	
  impact	
  on	
  pa9ent	
  access	  or	  	
  physician's	
  freedom	
  to	  prescribe	  5. Reward	
  innova9on	
  and	  minimize	
  the	
  burden	  to	
  both	  government	
  and	  industry	  	
  HTA delays and may
prevent access toinnovative therapies
Japanese citizens have broad
access to medicines, as new
therapies progress relatively
quickly from regulatory
approval to reimbursement.
Based on EFPIA surveys,
Japan tops the list, withreimbursement usuallyfollowing within 60-90 daysof regulatory approval.
The decision to implement
CEA on a trial basis in April
2016 must not reverse theprogress made in reducing
the drug lag.

Europe	  –	
  PaMents	
  W.A.I.T.	
  Indicator	
  	
  Pa#ents	
  Wai#ng	
  to	
  Access	
  Innova#ve	
  Therapies	
Percent	
  Available	
  (2013)	
  Elapsed	  Mme	
  aNer	  EMA*	  approval	
  (days)	
  75

Japan	
  Denmark	
  102

Switzerland	
  107

Sweden	  Belgium	  368

Belgium	
  Norway	  378

Norway	  Spain	  384

Spain	  France	  443

Greece	
  

611
200	
  

300	
  

400	
  

500	
  

83
63
90
74

Italy	
  

602

100	
  

71

71

France	  Portugal	
  

0	
  

89

Finland	
  

364

432

83

Austria	  Sweden	  Italy	
  90

Switzerland	
  Switzerland	

177

Finland	
  100

Denmark	
  

2013	
  
2010	

131

Austria	  Japan	  Japan:	  60-­‐90	
  days	

600	
  

63

Portugal	
  29

Greece	
  

55
0	
  

700	
  

20	
  

40	
  

60	
  

Japan:	
  100%	
80	
  

100	
  

**In-­‐country	
  availability.	
  In	
  %	
  of	
  medicines	
  with	  an	
  EU	
  central	
  authorizaMon	
  in	
  2011-­‐12,	
  e.g,	
  70	
  at	
  the	
  Mme	  of	
  survey.	

*Elapsed	  Mme	
  between	  MarkeMng	
  AuthorizaMon	
  and	
  Accessibility	  	

Figure	  4.	
  	
  Source:	
  EFPIA	
  annual	
  survey	
  2010	
  &	
  2013.	
  NB:	  UK	
  and	
  Germany	
  allow	  access	
  immediately	
  upon	
  markeMng	
  authorizaMon,	
  but	  HTA	
  hurdles—not	
  within	
  the	
  scope	  of	
  this	
  W.A.I.T.	
  analysis—apply	
  and	
  delay	
  actual	
  access.	
  *EMA:	
  European	
  Medicines	
  Agency.	

Costs	
  Well	  Controlled,	
  Market	
  Broadly	
  Flat	  (￥Tn)*1,2	
   Innova:on	
  premium	
  Costs of pharmaceuticals
are already wellcontrolled by the current
pricing scheme andgeneric use incentives

Forecast	
  market	
  growth	
  by	
  segment	

scheme	  and	
  biennial	
  pricing	  con:nue	

0.13%	
0.14%	

1.77%	
9.9	

4.8	

10.2	

10.2	

4.8	

4.8	

10.5	

4.8	

10.4	

4.8	

-1.48%	
10.6	

5.0	

10.4	

5.1	

10.6	

4.6	

10.3	

10.4	

4.4	

4.3	

10.0	

10.0	

4.4	
4.2	

4.3	

4.8	
 4.5	
 4.5	
 4.4	
 4.6	
 4.6	
4.0	
 3.9	
3.9	
 3.9	
A recent IMS study (Fig. 5),
4.8	
 4.8	
4.5	
0.8	
1.7	
2.0	
which recognizes pricing
1.3	
1.9	
1.7	
1.3	
1.2	
1.0	
1.3	
1.8	
0.3	
0.8	
 1.0	
 1.2	
 1.3	
 1.2	
changes that took place in
1.1	
 1.6	
 1.8	
 1.9	
 1.7	
 1.7	
2.7	
1.8	
1.7	
1.5	
2.0	
1.9	
1.6	
2.4	
2.2	
1.4	
1.3	
3.1	
2014, including scheduled
1.9	
1.9	
1.6	
1.7	
1.8	
1.8	
1.9	
2.0	
2.0	
loss of exclusivity after patent
1.1	
1.3	
1.4	
0.6	
0.6	
0.6	
0.6	
0.6	
0.6	
0.6	
0.6	
0.6	
0.6	
0.6	
0.6	
expiry and generic use
’20	
’14	
’15
	
’16	
’17	
’18	
’19	
’23	
 0.6	
 0.6	
’24
	
’25	
 0.6	
0.6	
’21	
 0.6	
’22	
0.6	
0.6	
 0.6	
0.6	
 0.6	
 0.6	
incentives, shows broadly
Original drugs	
LLPs (b)	
LLPs (a)	
Generics	
Others	0.6	
flat pharmaceutical market*1	
  LLPs	  (a)	  are	
  long-­‐listed	
  products	
  (LLPs)	
  whose	  ﬁrst	  generic	
  alterna:ve	
  was	
  launched	
  before	
  2013.	
  LLPs	  (b)	  are	  the	
  other	  LLPs,	
  whose	  ﬁrst	  generic	
  growth over the next decade.
compe:tor	
  is	
  launched	
  aDer	  2013.	
  *2	
  Sales	  are	
  calculated	
  assuming	  that	  the	  5%	  consump:on	
  tax	
  rate	  that	  existed	
  on	
  1	
  January	  2014	
  con:nues	
  throughout	
  the	  period.	
  This	  is	  in	
  order	
  to	
  look	  at	
  the	
  underlying	
  growth	
  in	
  the	
  market,	
  stripping	
  out	  the	  consump:on	
  tax	
  eﬀect.	
  16

Figure	  5.	
  	
  Source:	
  IMS	
  Consul:ng	
  Group.