The Role of Collaboration in Advancing Rare Disease Research

In a recent video interview with Applied Clinical Trials, Derek Ansel, vice president, therapeutic strategy lead, rare disease, Worldwide Clinical Trials, discussed the unique challenges in rare disease R&D, including competing priorities among researchers, difficulty in diagnosing and treating patients, and defining endpoints due to varying symptoms. Ansel also touched on the potential of technology and artificial intelligence to address some of these challenges and improve rare disease research.

ACT: Is there anything you would recommend to help advance rare disease research from a policy or strategic perspective?

Ansel: Another great question. I think it's really about collaboration. Now more than ever, I think the rare disease community and its various stakeholders need to really join forces to support and maximize really every resource available to the community. As I mentioned earlier, if you think about finances, there’s only so much money to go around. For example, if rare disease advocacy organizations could join arms, link together, and share additional funding to support research initiatives or research projects or clinical trials or registries that overlap with other groups might warrant financial benefit to those groups. I think coming together also supports conversations with regulatory authorities. By having a unified front, by working together, there's greater strength in greater numbers, and I think that's particularly true for regulatory authorities. From data sharing infrastructures, I think it's important that even our biotech partners and pharmaceutical companies, who spend a lot of money, time and energy developing programs in are disease, have some sort of exit strategy, whether, if the drug is not as safe and efficacious as we thought it was, using that data and other venues, are there patient advocacy organizations who would ingest that data and can use it to support subsequent clinical trials, so that another biotech company who's looking to research in the same disease doesn't have to start from square one at the very beginning. I think the lens of true collaboration, at least in industry, could really benefit all communities, I think, for drug development, at least for the time being.