How can multi-stakeholder collaboration improve rare disease access and affordability?

1 The value of collaboration

Collaboration can bring multiple advantages for rare disease access and affordability, such as sharing knowledge and expertise, leveraging resources, aligning incentives and objectives, and advocating for policy changes. This collaboration can accelerate innovation and evidence generation, reduce duplication and inefficiencies, foster trust and transparency, and ensure patient-centered outcomes. In addition, it can support the recognition, prioritization, and integration of rare diseases into health systems.

3 The best practices for collaboration

To maximize the value of collaboration and overcome any potential barriers, stakeholders should adopt some best practices, such as establishing a clear vision, mission, and goals for the collaboration with defined roles and responsibilities for each partner. It is also important to engage patients and patient organizations as active partners in the collaboration from design to evaluation. Furthermore, creating a governance structure and legal framework will ensure accountability, quality, and compliance between partners. Building a culture of trust, respect, and mutual learning among partners is also essential, with regular communication and feedback channels. Lastly, measuring and demonstrating the impact and value of the collaboration both qualitatively and quantitatively for patients, health systems, and society is key.

4 The examples of collaboration

Successful multi-stakeholder collaboration initiatives in the rare disease field have been numerous. The International Rare Diseases Research Consortium (IRDiRC) is one example, aiming to coordinate and accelerate global rare disease research efforts, with the goal of developing 200 new therapies and diagnostic tools by 2027. The European Reference Networks (ERNs) were established to connect health professionals, patients, and centers of excellence across Europe for better diagnosis, treatment, and care of rare diseases. Additionally, the Global Commission to End the Diagnostic Odyssey for Children with a Rare Disease brings together industry, academia, patient groups, and health systems to create recommendations and solutions to improve the diagnosis of rare diseases in children.

5 The future of collaboration

Collaboration is an ongoing process that requires adaptation and improvement. As the rare disease field evolves, so do the opportunities and challenges for collaboration. Digital technologies such as artificial intelligence, blockchain, and telemedicine are being increasingly used to improve data sharing, analysis, and access. Additionally, there is a growing demand for patient empowerment, involvement, and co-creation in rare disease research, innovation, and decision-making. There is also a rising pressure for health system transformation, value-based healthcare, and health equity in rare disease policies and practices.

6 Here’s what else to consider

This is a space to share examples, stories, or insights that don’t fit into any of the previous sections. What else would you like to add?