FDA’s Recent Outlook and New Policies for Cell and Gene Therapies

Mon, Feb 11, 2019 | By Tom Heinzen, VP, Corporate Development & Investor Relations, Cryoport

On January 15th FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D, Director of the Center for Biologics Evaluation and Research released a public statement which outlined new policies and the FDA’s outlook for cell and gene therapies.

The FDA is anticipating that they will receive more than 200 new investigational drug applications (IND) per year by the year 2020.  For some perspective, today there are a total of approximately 800 active IND’s on file with the FDA.  Additionally, the statement predicts that the FDA will be approving 10 to 20 new cell and gene therapy products per year by 2025.

To handle the expected increase in filings, clinical trials, and biologic license applications (BLA) the FDA has a goal to add 50 new clinical reviewers to their staff.  Additionally, the FDA is reviewing their policies, developing new guidelines, and working to continually streamline their processes. 

One of the relatively new regulatory pathways for these ground-breaking new therapies is the regenerative medicine advanced therapy (RMAT) designation.  The RMAT offers manufacturers a very expedited route to approval for therapies that treat disease states are have serious or life-threatening consequences.  If the cell or gene therapy is showing “meaningful” efficacy in clinical trials over the current treatment options and has an acceptable safety profile the RMAT designation can shave years off of the traditional timeline for commercial approval.

The RMAT designation also offers the FDA a way to require post approval follow up studies.  There remain significant questions about the durability and possible off target effects of these new therapies and trying to address those issues during the clinical trials would cause major delays and significant increased costs in getting the products to the market.  With many patients already in a life-threatening state, any and all delays could be a death sentence for some patients.

Many doctors and scientists believe that cell and gene therapies have tremendous potential to address diseases that have so far proven to be incurable.  The FDA is clearly attempting to help patients by providing a faster way to approval while also adhering to their mandates for safety.

Written by: Tom Heinzen, VP, Corporate Development & Investor Relations, Cryoport  

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