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Urgent Need for Evidence-based Care Catered to Rare Diseases

Report by Economist Intelligence Unit (EIU) found only one in three rare disease patients receive best-available, evidence-based care.

Globally, there are between six to seven thousand known rare disease. Despite its “rare” classification, these diseases affect an estimated 258 million people in the Asia Pacific region with children making up approximate 50 percent of the patients.

A recent report by EIU, sponsored by CSL Behring was launched on 16 July 2020 to highlight priorities for improving the management of rare diseases. It also explores the challenges around diagnosis and management of rare disease across five Asia-Pacific economies; Australia, China, Japan, South Korea, and Taiwan.

Healthcare professionals who participated in this report shared that only one in three of these rare disease patients receives the best-available, evidence-based care due to a lack of clinical guidelines, regulatory-approved medicine and funding for testing or treatment. This represents a significant disease burden and unmet need that cannot be ignored.

“In Asia-Pacific, rare diseases are a challenge because of the staggered level of socio-economic disparities, low government and general awareness, and a lack of momentum in acknowledging and addressing the challenges of patients with rare diseases,” said Dr Ritu Jain, President, Asia-Pacific Alliance of Rare Disease Organisations.

Dr Ritu Jain also highlighted that the EIU report covers a number of areas where improvements are needed. She also stressed that commitment and urgent multi-party engagement are required to make a difference for the rare disease population.

Three high-profile priorities mentioned in the report for improving the management of rare diseases were; correct and timely diagnosis, improved financial assistance and greater consideration for non-medical support. On top of these three priorities, the report also highlights currently achievable or near-term goals that policy makers could prioritize to make further improvements.

Commenting on the findings, the editor of the report, Mr Jesse Quigley Jones said, “Our survey of HCPs across Asia-Pacific shows that they clearly need further support to better manage rare diseases, but are willing to collaborate with each other and the patient community to improve the care offered for those living with rare diseases. Health systems are slowly progressing towards more coordinated and integrated care, and a holistic approach in health policy that takes into account both the medical and social needs of the rare disease community is emerging in some economies.”

Peter Chow, Executive Director, Marketing, Medical Affairs and Market Access, Asia-Pacific, at CSL Behring said, “HCPs who participated in the report confirmed the deeply frustrating and complex challenges patients face in their pursuit of a correct diagnosis and treatment for their illness. In helping support this research, we wanted to shine a light on these disparities and unmet needs.

“We hope the report will spark constructive discussions among all stakeholders in the rare disease space and will go some way to honouring our promise to improve the quality of life for rare disease patients and their families in Asia-Pacific and beyond.” [APBN]