− The founding of the ASEAN Rare Disease Consortium (ARdC) by five rare disease patient advocacy groups in Southeast Asia will be the catalyst to drive health equity for rare disease communities in the region.
− Stronger collaboration between public and private sectors and patient empowerment have been identified as crucial elements to improve access and quality of care in the five-year development roadmap of ARdC.
− The 2^nd Southeast Asia (SEA) Rare Disease Summit 2024 emphasized the critical need for implementation of patient-centric access policies to ensure health equity in rare disease communities across the region. 

BANGKOK, Thailand, April 1 (Bernama) -- Rare diseases impact the lives of millions of people despite being rare. In Southeast Asia, an estimated 45 million people, approximately 9% of the region's population, suffer from rare diseases. [i] The Medical Genetics and Genomics Association, Medical Association of Thailand and Thai Rare Disease Foundation joined hands with Takeda to host the 2^nd Southeast Asia Rare Disease Summit in Bangkok to address the complex challenges posed by rare diseases and ensure improvement for patients. Key stakeholders from diverse sectors, ranging from patient advocacy groups and caregivers to policymakers, clinicians, researchers, industry representatives, payers, and regulators, gathered once again for the summit. Held for the second year, the summit serves as a pivotal platform for stakeholders to address pressing issues, exchange insights and strategize meaningful advancements to improve diagnosis, access to treatment, health equity as well as patient empowerment with the aim of amplifying the patient voice for sustainable change.
 
“In Malaysia, based on the findings of a recent study conducted by the School of Pharmaceutical Sciences, Universiti Sains Malaysia and University Malaya Medical Centre, only 60% of rare disease patients are receiving treatment.[ii] For those rare disease patients who are diagnosed, funding for treatment remains a main obstacle as the payment of treatments is contingent on charities, public and industrial subsidy or out-of-pocket payments by patients.[iii] To date, key challenges like the lack of awareness, inadequate clinical expertise, facilities for diagnosis and treatment of rare diseases, as well as high treatment costs and limited pathways for reimbursement [iv] remain prevalent in Malaysia. The Rare Disease Summit highlighted the necessity for different stakeholders to come together to discuss tangible steps that can ensure rare disease communities in Southeast Asia can enjoy equitable and sustainable access to medicines. No single company or organization can do this alone. It is important for all of us in the society to create opportunities to broaden access to medicines for rare diseases patients to ensure that no one is left behind in Malaysia,” said Dr. Tae Sok Kun, Head of Genetics and Metabolism Unit, Department of Paediatrics, University Malaya.
 
Patient empowerment through regional collaboration is the key to enhancing health equity
Across the region, common challenges faced by the rare disease community include the lack of disease awareness, inadequate clinical expertise, facilities for diagnosis and treatment of rare diseases, as well as high treatment costs and limited pathways for reimbursement. Some rare diseases are so uncommon that they can affect as few as one in two million people [v] – and often, there are no viable treatment options. For patients living with rare diseases, this results in a wide set of challenges that starts with the diagnostic journey, with the average patient waiting for around four years before receiving an accurate diagnosis.[vi] To address these challenges, five rare disease patient advocacy groups – Thai Rare Disease Foundation, Malaysia Rare Disorders Society, Philippines Society for Orphan Disorders, Indonesian Rare Disease Foundation and Rare Disorders Society (Singapore) – have come together to form the ASEAN Rare Disease Consortium (ARdC) to share best practices and champion much-needed conversations with healthcare stakeholders. Plans are in place to invite more patient advocacy groups in the region to galvanize efforts with the ARdC.
 
As the first step to address challenges faced by rare disease communities across the region, the ARdC will work with patient advocacy groups, top healthcare professionals and other key healthcare stakeholders to encourage cross-country best practice sharing around patient support, patient resources, peer-to-peer exchanges, as well as experiences round fundraising programs and policy maker communications. In addition, the ARdC will conduct collaboration and partnership discussions with payers and governments to ensure sustainable funding for reimbursement of medicines and treatments.
 
“To ensure sustainable change, we need to take a leap of faith. The 2^nd Southeast Asia Rare Disease Summit is indeed a milestone in Rare Disease discussions among patient leaders from Thailand, Malaysia, Indonesia, the Philippines and Singapore. It is my honor to witness the seed that was sown at the 2014 Rare Disease Asia Conference 10 years ago is finally bearing fruit, with the formalization of the ARdC. At ARdC, we believe that the future of progress is in collaboration and focus on in-country best practices and solutions. To address the affordability gap among patients, we will continue to proactively discuss medicine access and reimbursement with government agencies and payers to ensure that some of the most vulnerable patients are being well taken care of in the region,” said Kenneth Mah, Founder of Rare Disease Orders Society (Singapore).
 
Public-private collaboration is a catalyst to driving systematic change
Since many treatments for rare diseases are specifically developed to treat rare medical conditions and often come at a high cost, patient accessibility to potentially vital treatments is often affected. Given that around 70% of rare diseases begin in childhood, there is often a big impact on the family’s financial health and the local economy due to loss of productivity.[vii]
 
“Due to the low level of diagnosis, rare disease patients are one of the most vulnerable communities in the healthcare sector of the region. To support rare disease communities in Southeast Asia to overcome patient barriers, at Takeda, we are committed to bringing medicines to patients. To do so, we have been working closely with public and private stakeholders in the healthcare sector to explore ways to strengthen healthcare systems. While systematic changes take time, since 2017 we have been helping to bridge the affordability gap of rare disease patients via our Patient Assistance Programs. Though the programs can provide short-term support, we will continue conversations with governments and organizations to explore sustainable funding for patient reimbursement and access to needed treatments. As we do our best to improve access to medicines in Southeast Asia, we will continue to forge new partnerships in the region to drive change and bring rare diseases into the spotlight,” said Dion Warren, Head of Multi-Country Organization, India and Southeast Asia at Takeda Pharmaceuticals.
 
Despite the lengthy road to accessing transformative treatments, several regions and countries in Asia have already begun to implement programs and policies to facilitate reimbursement for the treatment of rare diseases. For instance, Taiwan and the Philippines have introduced financial subsidies and discounts on healthcare services and medicine. In Taiwan, rare disease patients are now able to receive up to 100% reimbursement on orphan drugs necessary for their treatment.[viii] Innovative financing programs and other localized health economic solutions will be key to driving change for rare disease communities.
 
 

For more information about Southeast Asia Rare Disease Summit 2024, please visit: https://seararediseasesummit.org/   

 
About Takeda

Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit www.takeda.com.