FDA Commissioner Scott Gottlieb Issues Statement With Gene Therapy Guidelines

According to a story from the U.S. Food and Drug Administration (FDA), Commissioner Scott Gottlieb has released a press release which discusses in detail the agency’s efforts in the development of gene therapies. The announcement also includes drug development and manufacturing guidelines for specific categories of illness, such as retinal disorders, hemophilia, and rare diseases in general. Many diseases that are considered rare could potentially respond to gene therapy treatment approaches.

About Gene Therapy

Gene therapy is still an emerging medical technology in the medical field overall, and the research and development that has happened so far suggests that regulating and testing gene therapies in the same way as other types of drugs may not be the most effective. In the last year, the FDA has approved three gene therapy products. The potential of gene therapy is significant, and it will not be long before the number of experimental gene therapy products begins to increase in the agency’s regulatory pipeline.

Gene Therapy Development

Some of the primary concerns about gene therapy include how to test the durability of the response in the long term and the need for precise quality and manufacturing standards. The agency concedes that, due to these challenges, there may be a greater degree of uncertainty involved for some gene therapy drugs that get approved. However, the agency has committed to continued clinical trials to test long term outcomes after these therapies become available.

As many of the first gene therapies are aimed at treating severe, deadly medical problems, a degree of uncertainty is seen as reasonable, especially for rare diseases that have no current treatment options.

New Draft Guidelines and Updates

The new guidelines for developing gene therapies for different kinds of diseases are still not entirely complete. These guidelines will address subjects such as pre-clinical considerations for new gene therapies, the design of clinical trials, and information for company’s that hope to seek accelerated approval for their products.

The announcement also updates some existing guidelines that are relevant to the quality, manufacture, and chemistry of gene therapies. Additionally,they include guidelines for the testing of gene therapies that use retrovirus as the delivery system and long term follow up with patients after treatment. These guidelines were last updated a decade ago.

The announcement highlights the agency’s commitment to realizing the full potential of this new generation of therapeutic technology.


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