Policy Summit

Another Can’t-Miss Policy Summit Session: Trial Design Considerations for Gene Therapy for Rare Diseases

Emily Walsh Martin, Ph.D. - September 11, 2020

Dr. Marks will be speaking about how CBER plans to leverage new regulatory frameworks to help advance therapies in ultra-rare indications towards authorization.

<p>Peter Marks, M.D., Ph.D.,<strong> </strong>Director of FDA CBER, speaking at the 2019 ASGCT Policy Summit in Washington, D.C.</p>

As you may have already gathered, the ASGCT Policy Summit is going to cover a wide range of topics central to the advancement of new cell and gene therapies into the clinic. And while I’ve already convinced you to sign up on the basis of the CMC session alone on Wednesday September 23, I thought I might offer additional incentive!

The clinical trials session at 8:30am (CDT), Thursday, September 24, is also not-to-be-missed. Register now and mark your calendars.

In this session, Brad Glasscock, PharmD, Head of Global Regulatory Affairs at BioMarin, will speak to best practices for flexibly engaging the agency to advance novel therapies in rare diseases. Also, Daniel Chung, D.O., Medical Strategy Lead-Ophthalmology at Spark Therapeutics, will provide a case study about the development and use of a patient-focused functional endpoint for market authorization to get to the crux of how a therapy can impact patient’s daily living.

These two talks from deeply experienced therapeutic developers will almost certainly provide you with some new thoughts about how to evaluate the benefits of your gene therapy in the clinic.

However, I probably buried the lede.

This clinical session starts off with none other than Peter Marks, M.D., Ph.D., Director of FDA CBER.

If you have never seen Dr. Marks speak, you should. Apart from his obviously crucial role at CBER guiding new therapies in the clinic, he’s also just a fabulous communicator. For perspective, I’ve been in the field of drug development for 15 years and have worked on a number of gene and cell therapy and gene editing programs along the way. But I have never experienced someone who so aptly understands the challenges of advancing these therapies and yet can conceptualize the possible paths forward as Peter Marks does.

At the Summit Dr. Marks will be speaking about how CBER plans to leverage new regulatory frameworks to help advance therapies in ultra-rare indications towards authorization. However, if experience is any guide, he will probably be easily encouraged to opine on what he sees as the crucial role the FDA must play to partner with developers as the field of gene and cell therapy moves forward.

After these three presentations, Andrea Spezzi, M.D., Chief Medical Officer at Orchard Therapeutics, will lead the group in a question and answer session with the audience. Based on the working session preview I had, I expect this will cover wide ranging topics including, but not limited to, philosophies around how best to balance randomized control trials with novel ways of establishing evidence in rare diseases.

Dr. Walsh Martin is a principal at Tremont Therapeutics and a member of the ASGCT Communications Committee.

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