Rare disease researchers have always contended with significant barriers posed by small patient populations, fragmented data, challenging endpoints and limited opportunities to gain efficiencies. But now, more than ever, there is a growing acknowledgment that solving rare disease challenges to expedite patients’ access to life-changing therapies requires regulatory, community and global stakeholder cooperation.
Although individually rare, rare diseases are not rare when viewed collectively. Approximately 300 million people globally live with a rare disease and many of them share common needs and experiences.
That is why global cooperation is so crucial to rare disease research. National, international and community partnerships and harmonised clinical trial strategies present opportunities to accelerate innovation and access to treatments. There are many benefits to pooling knowledge and resources.
- This recognition has led to the recent creation of collaborative efforts such as:
- The European Rare Disease Research Alliance (ERDERA)
- The EU-X-CT Initiative
- The US Food & Drug Administration (FDA) Rare Disease Innovation Hub
- The FDA Support for clinical Trials Advancing Rare disease Therapeutics (START) programme
- Caregiver-led groups like the Inchstone Project.
Such partnerships could be welcome resources for rare disease researchers – but researchers must be aware of the potential advantages of each organisation. Therefore, the following briefly describes each and how it can benefit the rare disease community.
The European Rare Disease Research Alliance
The European Rare Disease Research Alliance (ERDERA) is now carrying forward the work formerly done by the European Joint Programme on Rare Diseases (EJP RD) and other projects. ERDERA aims to help academic key opinion leaders (KOLs) drive science and innovation in their respective rare diseases through funding, research networks, data services and resource consolidation.
Its mission is threefold:
- Unify rare disease resources, knowledge and expertise under one roof
- Empower patients to be ‘findable’ and enrolled in appropriate clinical trials
- Foster collaboration and align strategies to make Europe a world leader in rare disease research.
More than 180 organisations from 36 countries are working together through ERDERA. As a result, the coalition should benefit patients by aligning some of the most well-respected KOLs from across the globe to aid the discovery of meaningful treatment options. One could consider ERDERA as something like a scientific advisory board – a hub of researchers devoted to identifying new treatments and treatment guidelines. Rather than driving incremental change, the desire is to drive scientific innovation.
Read the article in full here.
