Suffering in silence: Assessing rare diseaseawareness and management in China

Suffering in silence: Assessing rare diseaseawareness and management in China
In November-December 2019, The Economist
Intelligence Unit surveyed 100 practising
healthcare professionals from China; as part ofan Asia-Pacific analysis of understanding andchallenges faced in managing rare diseases.
Our survey respondents from China
comprised general practitioners or primarycare physicians (44%), specialist physicians(36%), nurses (6%) and pharmacists (14%). The
majority of respondents reported working
in public hospitals (74%) with 13% working ina teaching hospital, and 8% employed in a
private hospital or outpatient clinic.

Results in context: China’s
health system• China’s population was estimated tobe 1.43bn in 2019.1 The average life
expectancy was estimated to be 76
years in 2018, and infant mortality
was an estimated 11.8 deaths per
1,000 births—significantly higher thanthe OECD average of 3.8. China’s
rapidly ageing population, coupledwith its low birth rate, represents animpending healthcare challenge forthe government.2 Non-communicablediseases account for 85% of deaths in
China, with ischaemic heart disease,chronic obstructive pulmonary disease,lung cancer and Alzheimer’s the leading
drivers of mortality.
• Around 95% of the population has somebasic level of health insurance, although
36% of total health expenditure is still

attributable to out-of-pocket spending.2
Three public health insurance schemes
exist, including the mandatory
employment-based Urban Employee
Basic Medical Insurance. Voluntary
private health insurance remains rare,accounting for just 4% of healthcare
spending in 2016 according to the WHO.2• China has around 3.6m doctors and 4mnurses, with an estimated 2.3 doctors
per 1,000 population. There are anestimated 32,000 hospital beds in China,
or 3.9 beds per 1,000 population. While
60% of China’s hospitals are private, themajority of inpatient care (over 80%) isprovided by public hospitals. Top-tierpublic hospitals provide around 50% of
all care, despite representing only 5% of
the total number of hospitals.2

Prioritising rare disease: A lack ofdata to inform policy in China• China’s Centre for Disease Control
estimates—given the size of thecountry’s population—that China has
the world’s largest number of people
with rare diseases (16.8m in 2014).3• China has no formal definition for raredisease, although local medical experts
generally accept a cut-off of one in 10,000
population for neonatal diseases, andone in 500,000 for others. Any numericalboundary enshrined in policy would
be largely academic due to the lack ofepidemiological data. 4

1 United Nations Population Division, World Population Prospects 2019. Estimates 1950–2020.
2 The Economist Intelligence Unit. Industry Report Healthcare China. 3rd Quarter 2019.
3 Peipei Song et al., “Innovative measures to combat rare diseases in China,” Intractable & Rare Diseases Research, 2017.
4 Jiangjiang He et al., “China has officially released its first national list of rare diseases,” Intractable and Rare
Diseases Research, 2018.
© The Economist Intelligence Unit Limited 20201

Suffering in silence: Assessing rare diseaseawareness and management in China

Our survey found that 31% ofrespondents from China did not know
if there was a unified rare diseases
definition, while 59% reported that one
did exist.
• Beyond a low prevalence, the mostfrequently cited considerations fora unified rare disease definition byhealthcare professionals in China were:heredity or genetic basis of the disease;severity or chronic nature; difficulty indiagnosis and financial burden of
the disease.
• China’s National Health Commission
created an Expert Committee of Rare
Disease Treatment and Support in2016 and published the first national
list of rare diseases two years later.
However, national figures on incidence
and prevalence exist for only 14 of the121 rare diseases on the list which China
recently issued.
• In China, policymakers look morenarrowly at the possible population
benefits from existing medicalinterventions. Its list of rare diseases
therefore focuses on relatively common
ones for which some treatment
is available.5• An increasing number of orphan drugs
are receiving approval and, since
October 2019, the large Chinese medical
insurance schemes appear to be looking
at ways to improve coverage.
• Health authorities are working on aNational Rare Diseases Registry System
which is projected to cover 50 conditions
by 2020.6

Healthcare professionals seem
more confident in their knowledgedespite seeing patients infrequently• Respondents in our survey rated raredisease knowledge in China with anaverage of 3.69 out of 5 for their own
knowledge, and 3.62 out of 5 for theirpeers’ knowledge.
• More than half (55%) of respondents
in China reported seeing a new raredisease patient at least every 6 months,
with 16% reporting a new patientpresenting on a monthly basis.

Multiple challenges to deliveringhigh-quality care in China• Respondents from China, on average,
give their health system the highest
grades for speed and quality of anyin the survey. While these healthcare
professionals clearly have confidence
in the system, this may not reflect
the reality for rare disease patients.
Respondents reported that only 24.4%
of patients receive the best-availablecare, the lowest proportion in ourstudy (Figure 1).
• In terms of difficulty in diagnosing andmanaging rare diseases, all categories
were considered a challenge more
often than not. The median category
was 4 (on a scale where 1 is never and
5 is always) for the following elements:
availability of evidence; reaching thecorrect diagnosis; access to medicines;
speed of medicine approval; funding fordiagnosis or treatment; defined referral
pathways; communicating with patientsand general populations’ knowledge.

5 Jiangjiang He et al., “China has officially released its first national list of rare diseases,” Intractable and Rare Diseases Research, 2018.
6 Peipei Song et al., “Innovative measures to combat rare diseases in China,” Intractable & Rare Diseases Research, 2017.

© The Economist Intelligence Unit Limited 20202

Suffering in silence: Assessing rare diseaseawareness and management in China• Notably, availability of specialist staff
was reported as ‘always a challenge’ by
37.0% of respondents.

• There are several well-establishedpatient groups working in China, notably
the Chinese Organisation for Rare
Disorders (CORD) and Illness Challenge
Foundation—representatives of whom
contributed to our research.

• When asked what one action should betaken to improve the management ofrare diseases in China, responses weresplit between improving the financial
support available, improved government
support, and better defining diagnosis
and treatment pathways.

• Broadly, healthcare professionals in
China felt that patient organisations
should contribute more frequently in allareas (between 61.5% and 69.2%
of responses).

Low awareness of the potential
contributions by patient groups

o Notably, a small but significant
proportion of respondents felt thatpatient groups should not be as
involved in matters of designing carepathways (17.9%) and identifying
outcomes needing research (15.4%).
This finding was not seen in the othermarkets of study.

• 41.0% of healthcare professionals
responding to our survey did not know
if rare disease patient organisations areactive in China, and 20.0% incorrectly
reported they are not active.

Figure 1

Survey responses reporting average proportion of patients managed with the optimal to sub-optimal carein five Asia-Pacific markets.
Managed with the best evidence-based care
Not managed with the best evidence-based care due to lack of clinical practice guidelines
Not managed with the best evidence-based care due to lack of regulatory approval of medicine
Not managed with the best evidence-based care due to lack of funding for testing/treatment
Not managed with the best evidence-based care for other reasonsTaiwan
38.2%

19.1%

14.3%

14.1%

14.3%

South Korea
28.5%

24.0%

14.8%

16.0%

16.7%

18.3%

14.1%

Japan
24.8%

23.8%

23.7%

22.4%

19.0%

China
17.4%

19.6%

16.9%

Australia
42.6%
0%

10%

20%

19.9%
30%

40%

50%

12.9%
60%

70%

15.7%
80%

While every effort has been taken to verify the accuracy of this information, The Economist
Intelligence Unit Ltd. cannot accept any responsibility or liability for reliance by any person on
this report or any of the information, opinions or conclusions set out in this report. The findingsand views expressed in the report do not necessarily reflect the views of the sponsor.

8.8%
90%

100%

© The Economist Intelligence Unit Limited 20203