As part of a pro bono disease initiative exploring how multistakeholder collaboration can advance care for people living with rare diseases in Asia-Pacific (APAC), we are soliciting the views of patient groups, payers, clinicians and industry representatives. Complete this survey to share your perspective. 

Introduction 

Payers face significant challenges in reimbursing rare disease treatments, as conventional evaluation frameworks prioritize conditions with the greatest disease burden and treatment with the largest public benefit. Due to small patient populations and high costs of product development and commercialization, rare disease treatments require more flexible and nuanced evaluation approaches. Multi-stakeholder involvement in reimbursement deliberations helps ensure that these nuances are recognized, enabling the true value of rare disease interventions to be captured. 

Across Asia-Pacific, payers have started to incorporate diverse stakeholder perspectives into public funding decisions for health technologies, including rare disease treatments. In Taiwan, for example, the role of patient representatives in health technology assessment (HTA) and reimbursement decisions has progressively strengthened over the years. Along similar lines, Singapore’s Agency for Care Effectiveness (ACE) published a guide for patient involvement in HTA in 2023. The following year, Malaysia developed a multicriteria decision analysis (MCDA) framework for prioritizing rare disease reimbursement, developed through multi-stakeholder deliberation.  

While these initiatives represent meaningful progress in integrating more holistic perspectives in funding decisions, payers acknowledge that there remain opportunities for higher levels of collaboration where stakeholders contribute more comparable effort, resources, and decision-making power. For example, patient input is often treated as supplementary in HTA processes across APAC. To explore how multi-stakeholder collaboration in funding decisions can be strengthened to improve access to rare disease care, we spoke with payers and HTA experts in Malaysia, Singapore, and Taiwan: 

Institutionalizing Patient Engagement in HTA  

While health systems in APAC have initiated measures to strengthen patient engagement in HTA, the nature of these efforts vary across the region. Only a few systems currently involve patients in the appraisal process; Taiwan, for example, has made patient engagement a core component of its HTA reforms. Grace Huang, Senior Director of the HTA Division at Taiwan’s Center for Drug Evaluation (CDE), outlined how patient engagement in their HTA process has evolved over time. Following the 2013 amendment of the National Health Insurance (NHI) Act, Taiwan has progressively institutionalized mechanisms to ensure that patient experiences, lived realities, and value preferences meaningfully inform reimbursement appraisal and decision-making (see figure below). These developments reflect a broader shift toward greater transparency, inclusiveness, and patient-centered health policy, which is particularly important in rare diseases, where disease awareness is lower and patient experiences are highly heterogeneous.

Despite substantial progress, challenges remain, including uneven patient engagement across disease areas, time constraints, and limited resources for patient groups. To address these gaps, the CDE and NHI Administration continue to refine submission platforms, expand outreach to underrepresented groups, and explore opportunities for earlier patient engagement in the appraisal process. Overall, Taiwan’s patient engagement framework represents a mature and evolving model that enhances the fairness, relevance, and legitimacy of HTA-informed reimbursement decisions. 

 
Jasmine Pwu, former Director of the HTA Division at Taiwan’s CDE and current Chief Executive Officer at Fu Jen Catholic University’s Data Science Center, noted that the institutionalization of patient participation in Taiwan has enabled more substantive contributions from different stakeholders in reimbursement deliberations. As patient representatives take on a more active role in reimbursement appraisal and decision-making, collaboration among patient groups, clinicians, and other stakeholders has become increasingly important. In this context, manufacturers may play a complementary role by providing technical, evidentiary, or capacity-building support where appropriate, helping to strengthen the quality and sustainability of patient engagement within established frameworks.  

Engaging Stakeholders Early to Foster Buy-In and Maximize Impact 

Healthcare systems in APAC have traditionally adopted a top-down approach. However, as illustrated by Singapore’s experience with the Rare Disease Fund (RDF), higher levels of collaboration across stakeholders could have amplified the impact on access to care.  

The RDF operates as an endowment fund that pools community donations with matching government contributions. Since its inception in 2019, fewer than 15 individuals with rare diseases have received support due to limited available funds. As the RDF depends heavily on public donations, greater community engagement throughout its design and implementation–through public consultations, awareness of its purpose, transparency in funding priorities, and communication of patient outcomes–could foster public buy-in and shared ownership for greater impact on access to rare disease care. 

As payers and policymakers increasingly embrace collaboration and shared decision-making, it is important to recognize that this involves more than presenting stakeholders with a pre-developed solution for feedback; it involves stakeholders actively co-developing and co-refining solutions.  

Roza Sarimin​, Head of the HTA unit at the Malaysian Health Technology Assessment Section,​ noted that the development of Malaysia’s MCDA framework for rare disease reimbursement prioritization benefited from multi-stakeholder working groups involving clinicians (38%), health professionals (33%), patient representatives (16%), and industry representatives (13%). Potential criteria were defined in lay terms to ensure shared understanding before stakeholders identified the key criteria and their associated weights. Co-designing fostered trust and aligned stakeholder expectations, while deepening patients’ understanding of the challenges and complexity of decision-making, as well as the reality that finite healthcare resources necessitate careful prioritization of needs. The principle that public policy should protect the vulnerable members of society, rather than merely empower the strongest, constitutes a foundational principle of ethical governance and social justice.  

Notwithstanding the differing priorities among stakeholders, deliberation ultimately led to a shared consensus on the MCDA framework. As outlined in Malaysia’s 2025 National Policy for Rare Diseases, the framework has been incorporated into the rare diseases technology assessment process, demonstrating Malaysia’s commitment to incorporating the priorities and perspectives of different stakeholders into decision-making.   

Bridge Global Experience with Local Expertise to Address Heterogeneity in APAC 

Payers in APAC often seek proof of concept before adopting new approaches, making international case studies useful for understanding how higher levels of collaboration can be achieved to inform funding decisions. However, given the significant diversity across APAC in culture, health systems, and stakeholder readiness, it is never a one-size-fits-all solution. This is particularly relevant for rare diseases, where patient journeys vary across individuals, diseases, and contexts. As such, deep involvement of local stakeholders is essential to effectively adapt strategies that have been successful elsewhere. 

Fiona Pearce, co-chair of HTAi’s Patient & Citizen Involvement in HTA Interest Group, and Senior Advisor to Singapore’s ACE, shared that when ACE began incorporating patient involvement into its HTA processes to inform funding decisions, it reviewed processes from other HTA bodies, including the United Kingdom’s National Institute for Health and Care Excellence (NICE), Canada’s Drug Agency (CDA-AMC), and Australia’s Pharmaceutical Benefits Advisory Committee (PBAC) to understand key success factors and implementation challenges.  

To contextualize international learnings to local patients’ needs, ACE worked with local patient groups to co-develop a living patient involvement process guide, accompanying plain-English resources, targeted training materials, and a patient glossary. Success quickly followed: Between 2022 and 2024, ACE gathered input from more than 700 patients and carers across 40 patient groups, and more than 80% of ACE’s HTAs included lived experiences, which informed funding recommendations for drugs, gene therapies, and medical devices.  

Today, ACE continues to stay up to date with international and local best practices through its International Advisory Panel with HTA experts across five global markets, and a Consumer Panel comprising representatives from local patient groups, including Rare Disorders Society (Singapore), who provide strategic advice on strengthening patient participation in HTA processes and policy development. 

ACE also offers valuable lessons for other countries seeking to strengthen multi-stakeholder collaboration in HTA. While payers often favor quantitative and methodologically robust data, ACE recognizes that the ultimate purpose of the healthcare system is to serve patients’ needs. To operationalize this principle, they have introduced accessible and flexible approaches to collecting patient input – any patient, carer, or patient group can share lived experiences through structured patient surveys or simplified patient journey forms. All templates were initially piloted in English, with Mandarin versions subsequently developed to improve accessibility and response rates. Together, these mechanisms lower barriers to participation by accommodating different levels of health literacy and capacity. Such flexibility is particularly important in the context of rare diseases, where patient populations are small and experiences are often highly individualized.  

Conclusion 

As illustrated by developments in Malaysia, Singapore, and Taiwan, payers across APAC are increasingly incorporating perspectives of different stakeholders into rare disease reimbursement decision making. To sustain and build on this progress, it is essential to engage patients whose perspectives reflect real-world needs and lived experience, to involve stakeholders early in the process, and to draw on international experience.  

What other factors do you think are critical? Share your thoughts in our survey linked here.  

Note: All comments and opinions expressed by the interviewees in this article are their own and do not necessarily reflect the views or positions of their respective organizations.