World Orphan Drug Congress: Speeding up Rare Disease Therapy Development

On April 28th, the virtual World Orphan Drug Congress USA 2021 was held. The program featured a variety of subjects relevant to the development of orphan drugs and the rare disease community. Patient Worthy had the privilege of attending some of the sessions during the day. One of these sessions was titled “How do We More Rapidly Advance Clinical Development for Rare Disease Advanced Therapy Products?”

This session featured Janet Lynch Lambert, who is CEO at the Alliance for Regenerative Medicine and Dr. Peter Marks from the US Food and Drug Administration (FDA). Dr. Marks leads the Center for Biologics, Evaluation, and Research at the agency. The session was conducted in an interview-style format, with Janet asking questions to Dr. Marks.*

Janet: So I know at a previous time when we spoke you said that you were devoting about 80 percent of your time to COVID. Thanks to those efforts we are now in a much more optimistic place, but not without its complexities. How much of your time is devoted to other things now?

Dr. Marks: Right now my time is split about 50-50 between COVID and everything else, including vaccines other than the COVID vaccines. Other areas include gene therapies and blood products. So overall I feel like that’s good news.

Janet: I know you have put some effort towards developing a public-private model of development for ultra rare disease gene therapies. Can you elaborate on this work and how these therapies will get to patients?

Dr. Marks: Well right now the approach for ultra rare diseases isn’t all that different from therapy development for common conditions. We have to find a way that leverages technology to help us address these rare diseases, particularly with the potential of gene therapy. It’s critical also for these products to be commercially viable.

Janet: Some have described the model that you are working on as a kind of “perpetual IND,” and these therapies wouldn’t really follow the traditional path. Is this accurate?

Dr. Marks: The hope is that we could take some of our statistical methods in order to get a lot of the information for a product approval using data from only ten or twenty patients. This could help encourage private insurance companies to cover these treatments. We can’t expect rare patients to rely on donations and GoFundMe to pay for treatment.

Janet: Could these be elements of a broader platform regulatory model?

Dr. Marks: We just have to have the same kind of data that will support their use and reimbursement.

Janet: We have talked a lot about gene and cell therapies for rare disease, but there are similar approaches being developed for less common conditions as well. Do you anticipate a divergence in the way the FDA regulates these therapies for rare vs common diseases?

Dr. Marks: Well ultimately standards of safety have to be universal, but the context in which these therapies are used will be different. In some cases more common diseases may already have multiple approved treatments. This can change where the therapy lies on the risk-to-benefit spectrum. Despite COVID-19, we saw a 15-20 percent increase in gene therapies during the last year.

Janet: I don’t think there’s ever been a lack of interest or commitment when it comes to collaboration, but obviously there are resource constraints. What can the rare disease community, especially those looking towards gene and cell therapies, expect in the near future? 

Dr. Marks: Well one way or the other we are going to need more staff, maybe around 100 more people. That’s what it will take for us to reach the level of responsiveness that I think we need. We have really been pushing for that, and we’ve also been trying to lean into our interactions with the community. The manufacturing process for these therapies is also always changing and developing.

Janet: Can you tell us about what’s at the top of your list for the rare disease space in terms of international regulatory harmonization?

Dr. Marks: Well my last international trip was in February 2020, which was a meeting of regulators from all over the world. There is definitely a desire for better education and harmonization. Many countries still need to be brought up to speed on what cell and gene therapies can do. The differences between different agencies from around the world have definitely decreased over time. This is going to help overcome access barriers.

Janet: Can you talk about the impact of non-sponsored stakeholders, such as patient groups?

Dr. Marks: Being able to interact with patients and hear what excites and bothers them is very motivating for us. It really helps us understand why we are here. These interactions are very important and keep us grounded in reality. We are in an ecosystem where there is a back and forth, and we are trying to maintain that.

*This interview was condensed and summarized for clarity.

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