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. Complete this survey to share your perspective.

Current Role of Patient Groups in Multi-stakeholder Collaboration

Patient groups are an indispensable partner in multi-stakeholder collaboration in APAC rare disease ecosystem. Positioned at the intersection of patients, clinicians, policymakers, payers, and industry, patient groups connect and coordinate stakeholders with different interests. In multi-stakeholder collaboration, patient groups also humanize healthcare decision making—from defining R&D priorities for manufacturers to supporting reimbursement decisions with payers and defining broader policy directions.

Patient groups have made notable progress in shaping awareness across the rare disease ecosystem. In China, for example, the Chinese Organization for Rare Disorders (CORD) recently collaborated with an Idiopathic Pulmonary Fibrosis (IPF) care center and a global pharmaceutical manufacturer to develop a comprehensive overview of the status of people with IPF and Progressive Pulmonary Fibrosis; the findings were subsequently highlighted in a national-level livestream program. Similarly, in 2024, Rare Cancers Australia (RCA) conducted over 120 meetings and speaking engagements, engaged 28 parliamentarians, and held an annual advocacy conference with strong media visibility.

Across APAC, there is also a growing number of channels for patient groups to provide input in the public reimbursement decision-making process, as seen in Australia, Singapore, and Taiwan. Patient groups are also actively ensuring that these channels are utilized adequately.

While these developments mark clear progress in collaboration with patient groups, resource constraints, rigid evidence frameworks, and changing policy priorities remain barriers to impactful collaboration, limiting opportunities for two-way collaboration.

To better understand how patient groups can amplify multi-stakeholder collaboration to achieve equitable access for people living with rare diseases, we spoke with representatives at three organizations:

Building a Collective Voice

When patients unite, rare diseases are not truly rare. By building a collective voice, patient groups can strengthen their advocacy and amplify their influence in multi-stakeholder collaboration. Umbrella patient organizations play a critical role in uniting and amplifying the voices of people living with a rare disease that does not yet have its own group. They also help emerging rare disease communities form organized groups, enabling patients to speak up and advocate for themselves.

Umbrella organizations can help unite patients in advocating for reimbursement. In China, CORD partnered with a global pharmaceutical manufacturer to support patient access to an innovative hereditary angioedema (HAE) therapy. With low awareness of the disease, frequent misdiagnosis, and no dedicated advocacy group, people with HAE were largely unorganized. CORD responded by identifying patients, incubating a unified community, and strengthening patient–physician engagement. When the first HAE therapy received regulatory approval in China, CORD’s groundwork enabled effective policy advocacy, contributing to the therapy’s inclusion in the basic medical insurance scheme and eventual inclusion in the National Reimbursement Drug List.

Umbrella organizations can also drive broader policy change. Recognizing the limited uptake and high out-of-pocket costs of genomic testing in Australia, RCA submitted written and oral evidence to the Senate Inquiry into equitable access to diagnosis and treatment for individuals with rare and less common cancers, including neuroendocrine cancers. The advocacy driven by RCA prompted government recognition of the need to expand genomic testing. In 2024, the Australian government announced its intention to legislate for a complete ban on genetic discrimination in life insurance.

Patient Groups as True Partners in the Decision-making Process

Building a collective patient voice is only half the battle. If the health system is not equipped to hear or act on that voice, the impact remains limited. Addressing systemic barriers—such as which types of evidence are considered useful—is therefore essential in enabling meaningful patient participation. This is particularly important in rare diseases, where the experiences of people living with rare diseases and their caregivers can be excluded or insufficiently included within existing HTA evidence frameworks.

Collaboration can occur at different levels depending on context, urgency, and resources, but deeper forms of collaboration allow patient groups to work alongside clinicians, payers, and policymakers to identify problems, co-design solutions, and refine initiatives over time. However, rigid evaluation frameworks and low receptiveness within the healthcare system frequently limit incorporation of patient perspectives into value assessment, delaying access to essential treatments.

Furthermore, even when formal processes exist, meaningful integration is not guaranteed. In Taiwan, for example, a review of the Pharmaceutical Benefit and Reimbursement Scheme (PBRS) Committee meeting records and the Online Patient Opinion Platform revealed that patient involvement in HTA remained limited in practice. This gap was a central theme of a white paper published by the TFRD in 2023, which called for strengthening patient participation, improving the integration of patient insights, and developing clearer guidelines for collecting and using patient input in decision making. The white paper ultimately enabled patients to share their perspectives during PBRS committee discussions, rather than simply attending as observers.

Exploring more flexible ways for patients to provide input will further empower patient groups to engage in higher levels of collaboration irrespective of literacy, geography, or resource constraints. For example, incorporating digital and decentralized approaches to collecting patient input— such as mobile-based surveys, virtual consultation platforms or social media channels —would not only encourage broader patient participation, but also help standardize collection of patient input. As the cofounder of RCA Richard Vines explained, “If you have a patient who is semi-literate living in a rural part of Asia, they should still be able to use a smartphone to record their experience and their frustration—and that contribution is valuable. We need to get much better at recognizing and using that [input].”

Ultimately, humanizing public reimbursement decision making requires an ongoing commitment to ensuring that patient groups are not only consulted, but are also empowered to shape the systems that govern access to rare disease care. Institutional mechanisms that formalize patient input, improve openness to patient-generated data, and facilitate thoughtful use of digital technologies can enable higher levels of collaboration with patient groups.

Conclusion

Patient groups serve as a vital bridge between individual patients and the wider healthcare ecosystem. They offer a formal, organized platform through which dispersed patient experiences can be consolidated into a collective voice that carries greater weight and negotiating power in policy and reimbursement discussions. This is especially critical in rare diseases, where patient populations are small, geographically dispersed, and often lack sufficient visibility to influence decisions on their own.

Further amplifying the potential of patient groups requires moving towards higher levels of collaboration. To this end, the collective patient voice needs to be meaningfully incorporated into decision making, and all stakeholders must be prepared to find common ground on what constitutes acceptable patient-generated evidence.

Patient groups can play a key role in gathering patient perspectives in a structured, consistent manner. At the same time, payers, health technology assessment bodies, and other decision makers can consider adapting existing evidence frameworks to accommodate less traditional or more qualitative data sources—while this is likely to require structural and systemic changes over time, patient groups can accelerate progress by seizing windows of opportunity, such as policy reforms, leadership transitions, or heightened media attention, to “ride the wave” and catalyze meaningful, sustained change. To learn more about Avalere Health’s work in this area, connect with us.

If you would like to contribute to our initiative, please complete this survey to share your views on the future of multi-stakeholder collaboration in rare disease care.

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.

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. Complete this survey to share your perspective.