What does the new FDA accelerated regulatory pathway mean for gene therapies?

U.S. Food and Drug Administration (FDA) commissioner Scott Gottlieb has announced that the drug agency is developing a system by which innovative gene therapies could be sped through the regulatory process. In a speech to board members of the Alliance for Regenerative Medicine, Dr. Gottlieb spoke about the challenges associated with reviewing cell and gene therapies in the same way as traditional drugs, and the steps that the FDA is taking to address these issues and improve access.

Gene therapies differ from more traditional medicines in that they are intended to be used as one-time treatments that permanently cure diseases by editing the genetic code within the patients’ cells. This poses significant challenges for regulatory bodies that review the drugs, as it is difficult to monitor the safety of such products many years after treatment.

For health regulators, the challenge is to find ways to get the new therapies to patients with critical need while balancing the need to monitor their long-term safety for years after approval, FDA Commissioner Scott Gottlieb said.

The scheme will initially be applied to gene therapy candidates to treat hemophilia which has been one of the focusses for gene therapy research. Pfizer, Spark, UniQure, Biomarin and Sanofi all developing products for this indication.

Traditionally, a novel drug to treat hemophilia would be reviewed on its ability to reduce bleeding episodes in patients. The new system would allow the approval of gene therapies without this evidence, provided there is evidence that the treatment increases the level of key proteins in the blood. However, it is likely that real-world evidence showing a reduction in bleeding will be required post-launch, according to Dr. Gottlieb.

Although this theory often makes sense, but from our experience at P4A, there are examples where chemical measures of drug efficacy do not translate into clinical benefits for patients.

The lack of substantial safety studies on such products may be seen as a drawback, as some cell and gene therapies, especially CAR-T cell (chimeric antigen receptor) therapies, can have significant safety issues.

These measures announced will  do little to address the main issue that has inhibited the uptake of gene therapies – cost. With Spark’s Luxturna costing $850,000 per treatment and the two approved CAR-T therapies also in the hundreds of thousands, there is a debate about whether these prices can be justified. Given the lack of long-term efficacy/safety data that will be required in this process, there will be no guarantees that the treatments are in fact a lifelong cure. In Europe, several cell and gene therapies have been withdrawn from the market either due to safety concerns or due to low market uptake. It remains to be seen if the US will suffer any such consequences due to the fast track procedure.

Investment in gene therapy research is growing now more than ever, with the FDA receiving over 100 investigational new drug applications involving gene therapies over the last 12 months. This move will be welcomed by companies working in this area, with the faster approval process leading to potentially faster access for patients in need of life-changing treatments. This move shows that the FDA is actively taking steps to prepare for the coming influx of gene therapies to the market and working with pharmaceutical companies to improve the regulatory process. Dr. Gottlieb spoke about the great potential of cell and gene therapies, acknowledging that in the future they “will soon become the mainstay of how we treat a wide range of illness.”

Partners4Access specialize in advising clients on market access of orphan and cell and gene therapies and orphan drugs. If you require further advice with your market access strategy please get in touch at contact@partners4access.

Author: Max Rex

Email Max at [email protected] 


  1. https://www.bloomberg.com/news/articles/2018-05-22/gene-therapies-that-could-transform-diseases-get-easier-fda-path
  2. https://www.statnews.com/2018/05/22/fda-gene-therapy-hemophilia/

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