How can rare disease market access benefit from cross-border and multi-stakeholder initiatives?

1 The need for collaboration

Rare diseases are complex and heterogeneous, requiring specialized expertise and data to understand and treat them. No single country or organization can address the unmet needs and gaps of rare disease patients alone. Therefore, collaboration across borders and sectors is essential to share knowledge, resources, and best practices. For example, the European Reference Networks (ERNs) are networks of healthcare providers that facilitate the exchange of information and expertise on rare diseases across the EU. The ERNs aim to improve the diagnosis, treatment, and care of rare disease patients, regardless of where they live.

2 The role of patient advocacy

Rare disease patients and their families often face multiple challenges, such as delayed diagnosis, lack of information, isolation, and discrimination. Patient advocacy groups play a vital role in raising awareness, empowering patients, and influencing policy and decision-making. Patient advocacy groups can also foster cross-border and multi-stakeholder initiatives by creating platforms for dialogue, partnership, and co-creation. For instance, the European Organisation for Rare Diseases (EURORDIS) is a non-governmental alliance of over 900 rare disease patient organizations that advocates for the rights and needs of rare disease patients at the European and international levels.

4 The importance of data

Data is the foundation of evidence-based decision-making for rare disease market access. However, data on rare diseases is often scarce, fragmented, and inconsistent, limiting the ability to generate robust and relevant evidence. To address this issue, cross-border and multi-stakeholder initiatives can help to collect, harmonize, and share data on rare diseases. For example, the European Joint Programme on Rare Diseases (EJP RD) is a co-funded initiative that aims to create a comprehensive and sustainable ecosystem for rare disease research and innovation in Europe. One of its objectives is to establish a virtual platform for data sharing and analysis on rare diseases.

5 The potential of digital health

Digital health technologies offer new opportunities to improve the access and delivery of rare disease therapies. Digital health can enable remote diagnosis, monitoring, and care, as well as personalized and patient-centered approaches. However, digital health also poses challenges for market access, such as regulatory uncertainty, ethical concerns, and data protection. To leverage the potential of digital health, cross-border and multi-stakeholder initiatives can help to create common standards, frameworks, and guidelines. For example, the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) is a collaborative project that aims to develop a platform for collecting and analyzing data from various sources to support the development and approval of rare disease therapies.

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?