- Knowledge of rare diseases among healthcare professionals is lacking:
o 34% do not know if there is a definition for rare diseases
o 35% do not know if there is a national rare disease policy - 14% of healthcare professionals report never encountering a rare disease patient
- Better coordination between disciplines and across borders is needed to ensure every rare disease patient receives the best care available
CHINA – Media OutReach – 16 July 2020 – There are between 6,000 and 7,000 known rare diseases, which share little in common beyond their infrequency. This represents both a medical and policy challenge, often resulting in those with rare diseases receiving suboptimal support. While individual diseases may be rare, their collective burden is significant.
The Economist Intelligence Unit estimates that around 258 million people across Asia-Pacific are affected by rare diseases. While rare diseases are increasingly moving up the health policy agenda in Asia-Pacific, more work is needed to provide people with rare diseases the support they need.
The Economist Intelligence Unit report, Suffering in silence: Assessing rare disease awareness and management in Asia-Pacific, sponsored by CSL Behring, describes healthcare professional’s experiences around rare diseases–measured in a regional survey of over 500 people–and reviews policy responses in five economies: Australia, China, Japan, South Korea and Taiwan.
The research found that healthcare professionals do not feel adequately equipped with the knowledge or tools to diagnose and manage rare diseases across all economies, and obtaining a correct diagnosis within a reasonable timeframe is the greatest challenge. Fragmentation of care is another critical issue. Healthcare professionals reported that only one-third of rare disease patients receive the best-available care, and lack of clinical practice guidelines, regulatory-approved medicine and funding for testing or treatment were the reasons cited for why not. Finally, the provision of support for quality of life, autonomy and rights was the weakest aspect of care provision, according to survey respondents.
Policy responses to more comprehensively address rare diseases are emerging across the region, but are hampered by lack of comprehensive data on the incidence and prevalence of rare diseases and a lack of a unified rare disease definition. Meanwhile, the establishment of national undiagnosed disease programmes in some economies has demonstrated the power of increased collaboration and a multi-disciplinary approach to addressing the core issues around diagnosis and patient care. Currently-achievable or near-term goals identified by the research include better collection and use of data, enhanced education, broader dissemination of available knowledge and integration of patient care through increased national or international collaboration.
Jesse Quigley Jones, editor of the report said: “Despite the clear challenges in addressing rare diseases in Asia-Pacific, there appears to be slow and steady progress towards coordinated and integrated care. Adopting a holistic policy approach that makes provision for both the medical and social needs of the rare disease community–and doing so in collaboration with patient representatives–has the potential to make a real difference in this area.”
About the research
Suffering in silence: Assessing rare disease awareness in Asia-Pacific is a report by The Economist Intelligence Unit. It describes the results of a survey conducted in November-December 2019 of 503 healthcare professionals and in-depth interviews with 16 experts from across five Asia-Pacific economies: Australia, China, Japan, South Korea and Taiwan. Survey respondents comprised specialist physicians, general practitioners, nurses and pharmacists.
About The Economist Intelligence Unit
The Economist Intelligence Unit is the world leader in global business intelligence. It is the business-to-business arm of The Economist Group, which publishes The Economist newspaper. The Economist Intelligence Unit helps executives make better decisions by providing timely, reliable and impartial analysis on worldwide market trends and business strategies.
About CSL Behring
CSL Behring is a global biotherapeutics leader driven by its promise to save lives. Focused on serving patients’ needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, respiratory disease, and neurological disorders. The company’s products are also used in cardiac surgery, burn treatment and to prevent hemolytic disease of the newborn.
CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. The parent company, CSL Limited (ASX:CSL;USOTC:CSLLY), headquartered in Melbourne, Australia, employs more than 26,000 people, and delivers its life-saving therapies to people in more than 70 countries.
