The increasing Importance of Gene Therapies - FDA Statement and New Guidelines

Gene Therapies have an increasing importance to combat fatal diseases. Amongst others this importance is demonstrated by an official statement from FDA Commissioner Scott Gottlieb, M.D., on the agency's efforts to advance the development of gene therapies and by the publication of  six draft guidelines on gene therapies:


Gene therapy-related research and development in the United States as well as in Europe continue to grow at a fast rate, with a number of products advancing in clinical development. The first first treatments in the U.S. came to market in 2017 after receiving approval from the Food and Drug Administration. Therefore, scientific advice and regulatory guidance gain in importance, for the involved companies as well as for the competent rapporteurs. 

In the first part of his statement, Scott Gottlieb said:

 "In the future, we expect this field to continue to expand, with the potential approval of new treatments for many debilitating diseases. These therapies hold great promise. Our new steps are aimed at fostering developments in this innovative field.

Gene therapies are being studied in many areas, including genetic disorders, autoimmune diseases, heart disease, cancer and HIV/AIDS. We look forward to working with the academic and research communities to make safe and effective products a reality for more patients. But we know that we still have much to learn about how these products work, how to administer them safely, and whether they will continue to work properly in the body without causing adverse side effects over long periods of time. In contrast to traditional drug review, some of the more challenging questions when it comes to gene therapy relate to product manufacturing and quality, or questions about the durability of response, which often can't be fully answered in any reasonably sized pre-market trial. For some of these products, we may need to accept some level of uncertainty around these questions at the time of approval. For example, in some cases the long-term durability of the effect won't be fully understood at the time of approval. Effective tools for reliable post-market follow up, such as required post-market clinical trials, are going to be one key to advancing this field and helping to ensure that our approach fosters safe and innovative treatments.

Even when there may be uncertainty about some questions, we need to make certain we assure patient safety and adequately characterize the potential risks and demonstrated benefits of these products. In part because of the added questions that often surround a new technology like gene therapy, these products are initially being aimed at devastating diseases, many of which lack available therapies, including some diseases that are fatal. In such cases of devastating diseases without available therapies, we've traditionally been willing to accept more uncertainty to facilitate timely access to promising therapies. In such cases, drug sponsors are generally required to conduct post-marketing clinical trials, known as phase 4 confirmatory trials, to confirm clinical benefit of the drug. This is the direction Congress gave the FDA by creating vehicles like the accelerated approval pathway."

Additionally, he emphasized that the FDA is taking a step forward with these new regulations to come to a modern and up-to-date regulatory framework and to keep a safety and effectivness gold standard for such innovative products. For further information please read the complete statement at "Agency's efforts to advance development of gene therapies".  

One step on that way for additional support was the issue of the above mentioned guidance drafts. They cover different potential fields of  Gene Therapy Products - like hemophilia, Retinal Disurders or rae diseases as well as more general topics like CMC information or the long term follow-up after administration of GTPs. An overview about FDA's guidances on Gene therapy can be found on the FDA website - section  "Cellular & Gene Therapy Guidances".

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