Suffering in silence: Assessing rare diseaseawareness and management in Taiwan

Suffering in silence: Assessing rare diseaseawareness and management in Taiwan
In November-December 2019, The Economist
Intelligence Unit surveyed 100 practising
healthcare professionals from Taiwan, as partof an Asia-Pacific analysis of understanding
and challenges faced in managing
rare diseases.
Our survey respondents from Taiwan
comprised general practitioners or primarycare physicians (46%), specialist physicians(34%), nurses (10%) and pharmacists (10%).
Respondents were split between private(54.0%) and public (21%) or teachinghealthcare institutions (25%).

Results in context: Taiwan’s
health system• Taiwan’s population was estimatedto be 23.7m in 2019.1 The average life
expectancy in 2018 was 80.4 years.
Infant mortality is 4.3 per 1,000 live
births. Taiwan has stagnant population
growth, and its population is ageing at
a rapid pace. Cancer, heart disease andpneumonia were the leading causes ofdeath in 2017.2• Total health expenditure wasUS$35.6bn in 2017. The mandatory
National Health Insurance (NHI)
system—covering all registered Taiwan
residents with a comprehensive
package of inpatient, outpatient andpreventive care—accounts for aroundhalf of all health spending. Co-payment
is required for all services, and out-ofpocket spending accounts for 33.6% of
all health expenditure.2• There are shortages of key healthcare
professionals in Taiwan—many haveleft for higher-paid positions overseas—with over 46,000 doctors and 159,000
registered nurses in 2017. There areover 450 hospitals and 10,400 clinics
in Taiwan; the majority areprivately operated.2

Prioritising rare disease: Healthpolicy goes from strength tostrength in Taiwan• Taiwan has made highly-regarded, longstanding efforts to address rare diseases.
The first Rare Disease Control andOrphan Drug Act was passed in 2000.
In late 2017, the Rare Diseases and Rare
Genetic Disorders Care and Services
Plan was introduced.
o These policy responses are notable
for their focus on not only provision
for drug development and medicalcare, but a broader consideration forpatients’ rights, legal protection andsocial care.
• Taiwan does not have a defined numberof cases for a disease to be classified
as rare. Instead, its Rare Diseases
and Medicine Review Committee isauthorised to set and regularly review
the target prevalence for its policies.
Since 2000, any disease with less than
one person per 10,000 population isdeemed rare.
• Healthcare professionals responding
to our survey from Taiwan had thehighest awareness of the existence of a
unified rare disease definition in any ofour study markets, with 83% correctly
reporting that one exists (Figure 1).
Sponsored by:

1 United Nations Population Division, World Population Prospects 2019. Estimates 1950–2020.
2 The Economist Intelligence Unit. Industry Report Healthcare Taiwan. 3rd Quarter 2019.
© The Economist Intelligence Unit Limited 20201

Suffering in silence: Assessing rare diseaseawareness and management in Taiwan

Additionally, 75.9% correctly identified
the prevalence cut-off in the definition.
Among the small number of
respondents who reported there was nounified rare disease definition, the mostvalued attributes of any definition werereported to be prevalence and difficultyin diagnosis.

Figure 1

Knowledge of rare disease definitions among physiciansin Taiwan; Responses to the question “Is there a unified
definition of rare diseases in your market?”

100%

80%

o This is in contrast to the highproportion of respondents whoreported awareness of various
aspects of rare disease policy andcare. Whether this lower confidence
is due to lack of exposure to suchpatients or reflects a better graspof the scope of missing knowledge
among a more informed community
in Taiwan is unclear.

Prioritising social care forrare disease patients is an
important challenge• Taiwanese respondents report that on
average 40.8% of rare disease cases aremanaged with the best evidence-basedcare, the second highest in our research(Figure 2).

83.0%

60%

• Multiple areas were cited by our survey
respondents as a challenge indiagnosing and managing rare diseases,
most notably:

40%

20%

15.0%
2.0%

0%
Yes

No

Don’t know

Less confidence in knowledge andexposure to rare diseases among
healthcare professionals in Taiwan• Healthcare professionals from Taiwan
reported seeing a new rare disease
patient presenting for care lessfrequently than any other market in ourstudy. Only 15% reported seeing a newpatient more frequently than once every
six months, and 24% reported neverseeing such patients.
• Healthcare professionals in our survey
rated rare disease knowledge in Taiwan
among the lowest of our study markets(an average of 2.69 for themselves and2.71 for their peers on a scale of 1 to 5).

o Reaching the correct diagnosis (56.6%
report this is nearly always or isalways a challenge).
o Access to medicines (59.3% report
this is nearly always or is always
a challenge).
o Funding for diagnosis (48.7% report
this is nearly always or is always
a challenge).
o Funding for treatment (51.3% report
this is nearly always or is alwaysa challenge).
o Availability of specialist staff (51.3%
report this is nearly always or isalways a challenge).
o Access to ongoing professional
training (47.3% report this is nearly
always or is always a challenge).

© The Economist Intelligence Unit Limited 20202

Suffering in silence: Assessing rare diseaseawareness and management in Taiwan

Patient organisations in Taiwan
are more visible and activities arevalued by healthcare professionals• Among the elements of Taiwan’s healthsystem that survey respondents felt
were most effective, arriving at a correct
diagnosis, initiation of care and qualityof care scored highest. At the opposite
end, speed of diagnosis, cost of care andproviding social care were reported as
least effective.

• Awareness of patient organisations
among healthcare professionals in
Taiwan was the highest in our research;
59% reported correctly that such groups
exist, and only 7% reported incorrectlythat they did not.

• When our survey respondents wereasked about the one action that should
be taken to improve the lives of raredisease patients in Taiwan, the mostcommon responses were:• Among healthcare professionals
responding to our survey from Taiwan,
87% and 74% felt that patients andpatient organisations, respectively,
were important or the most important
stakeholders in designing care pathways.

o Increased coverage through healthinsurance schemes.
o Provision of social welfare assistanceand rehabilitative services.

Figure 2

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%

8.8%
90%

100%

© The Economist Intelligence Unit Limited 20203

Suffering in silence: Assessing rare diseaseawareness and management in Taiwan• When considering the activities ofrare disease patient organisations, ourrespondents from Taiwan wanted to see
more participation across the board.
Areas respondents were particularly
keen for development and growth in theareas of:o Disease awareness activities (69.5%).
o Design of care pathways andprovision of patient education (62.7%and 50.8% respectively).
o Identifying outcomes for research or
designing research (66.1%).

o Influencing policy and budget
allocation (55.9% and57.6% respectively).
• One notable patient group, the Taiwan
Foundation for Rare Disorders (TFRD),
has been active in advocating for raredisease patients with government
stakeholders for around two decades
and was a contributor to the 2000 Rare
Disease Control and Orphan Drug Act.
TFRD also plays additional roles such as
collaborating with health authorities inscreening activities.3

3 Dharssi et al. Orphanet Journal of Rare Diseases (2017) 12:63 DOI 10.1186/s13023-017-0618-0

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 findings and views
expressed in the report do not necessarily reflect the views of the sponsor.
© The Economist Intelligence Unit Limited 20204