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FDA to Allow Gene Therapies Accelerated Approval Pathway Route


Sita Somara, PhD, MBA
RTI International
United States

FDA has recently shared the news that it is taking steps to help gene therapies get accelerated approval. According to Dr. Marks, the director of FDA's Center for Biologics Evaluation and Research, "the FDA views gene therapy as an excellent opportunity to expedite the delivery of potentially life-saving therapies to patients with rare diseases".

What is accelerated approval pathway?

Accelerated approval pathway is a regulatory avenue commonly used to expedite the development and availability of drugs and biologics that treat serious or life-threatening conditions. It is an approval for the market entry of drugs for unmet medical needs by using surrogate endpoints to cut the approval time.

It is important to note that the FDA's accelerated approval program is intended for use in situations where the benefit of a therapy is likely to outweigh its risks, even if there is uncertainty about its clinical benefit. The U.S. health regulator grants the accelerated approval mainly for drugs and therapies targeted at rare diseases or diseases with small patient populations that have had no effective treatments available to them. Companies however are still required to conduct studies to confirm the anticipated clinical benefit.

Accelerated approval may be appropriate for certain gene therapies, particularly those that treat rare or life-threatening diseases for which there are few or no other treatment options.

How can it be applied to gene therapies?

To qualify for accelerated approval, a gene therapy must demonstrate a meaningful advantage over existing therapies or show the potential to address an unmet medical need. To gain accelerated approval, a gene therapy must undergo clinical trials that demonstrate its safety and effectiveness. The FDA may also require post-marketing studies to confirm the therapy's clinical benefit.

For certain gene therapies, there may be a need to "accept some level of uncertainty" at the time of approval around questions like side-effects over long-term and safety during administration. Adding that post-marketing tools such as safety monitoring and the possible use of extra clinical trials are going to be key.

Biomarkers, such as pulse and blood pressure, are characteristics of the body that are measurable. According to Peter Marks, the FDA will support using measurable biomarkers as substitutes for other biological indicators in gene-therapy clinical trials to help developers secure "accelerated approval”.

If a gene therapy receives accelerated approval, the FDA may require the manufacturer to conduct additional studies to confirm its clinical benefit. If these studies fail to demonstrate clinical benefit or confirm safety concerns, the FDA may withdraw the therapy from the market.

What are the advantages of gene therapies with accelerated approvals?

The FDA's accelerated approval program for gene therapy offers several advantages, including:

·       Faster access to potentially life-saving treatments: The accelerated approval program allows patients with serious or life-threatening diseases to access gene therapies sooner than they would with traditional approval pathways. This can be especially important for patients with conditions for which there are few or no effective treatments available.

·       Flexibility in clinical trial design: The program allows for more flexible clinical trial designs, which can speed up the development and approval of gene therapies. For example, clinical trials can use surrogate endpoints that may be easier and faster to measure than traditional endpoints, such as overall survival.

·       Continued monitoring and evaluation: Gene therapies approved through the accelerated approval program are subject to continued monitoring and evaluation. This allows the FDA to collect additional data on the therapy's safety and effectiveness, and to take action if new safety concerns arise.

·       Encouragement of innovation: The accelerated approval program can encourage innovation in the development of gene therapies. Companies may be more willing to invest in developing gene therapies for rare or difficult-to-treat diseases if they know there is a pathway to expedited approval.

Overall, the accelerated approval program for gene therapy allows for more rapid access to potentially life-saving treatments while maintaining rigorous standards for safety and effectiveness.


Accelerated Approval Program | FDA