Gene therapy has recently emerged as a treatment option for some rare diseases with unmet needs. The FDA approved the first gene therapy in 2017 and 19 gene therapies as of June 2024, many of which are for rare diseases. Clinical trials that support approval of gene therapy products provide the needed information about the risks and benefits of the product so that patients and care partners can make informed decisions regarding treatment.  

After a product is approved, FDA and manufacturers continue to collect data about the safety of gene therapies. These data come from long-term observation of patients who participated in clinical trials and post-marketing studies as well as from reports from patients, manufacturers, and health care providers. Long-term studies of patients treated with gene therapies can help detect adverse events that may not be observed during clinical trials and can help develop a more complete understanding of the benefits and risks of the therapy. 

As the body of knowledge about the risks and benefits of gene therapies continues to grow and the early molecular diagnosis of some progressive rare genetic diseases is becoming more common, patients and their care partners have expressed a willingness to participate in gene therapy clinical trials in the pre-symptomatic or very early stages of their disease. FDA recognizes a need for the development of earlier treatment for both children and adults with progressive rare diseases.   

Patient engagement has a lasting impact on the work we do, enhancing FDA's clinical and regulatory understanding of gene therapies and our ability to effectively monitor their safety. Patients, care partners, and advocates are essential partners in our efforts to gather this information, as they are directly impacted by what we learn.  

The FDA is holding two listening meetings with patients with rare diseases and their care partners to hear their perspectives on short-term and long-term risks of approved gene therapy products, to learn what types of information patients would find helpful in their decision-making when considering gene therapy, to learn about their considerations and experience with participating in long term post-market studies, and to help inform patient-centered protocols for long-term studies of gene therapies.