A town hall meeting was held on February 7th of this year to discuss designs for clinical trial gene therapies. According to a report in Pharmaceutical-Technology, the FDA experts were on hand to suggest ways to create clinical effectiveness despite the difficulty in recruiting a satisfactory number of patients with rare diseases.
This issue was glaring as it involved the need for gene therapy data from a small patient population.
The Challenges of Research for Rare Diseases
· Clinical trials have been growing increasingly complex.
· The industry is heavily regulated.
· Spiraling costs; the cost of conducting a clinical trial is at an all-time high.
Branch Chief of DCEPT, Elizabeth Hart, M.D. noted that clinical trials should be designed to illustrate how patients function, feel, and survive. Yet research into rare diseases usually centers on very few patients thus presenting challenges in the conduct, design, interpretation, and analysis of clinical studies.
Although there are no particular methods of coping with such a small number of patients, there are still different types of approaches that will increase the utility and efficiency of clinical trials. Rigorous planning is therefore required to encourage each patient in a trial to provide adequate information.
FDA’s Industry Guidance Document
The FDA released an industry guidance document in 2020 that contained recommendations for sponsors that were developing rare disease therapies. The document contained aspects that were related to preclinical and clinical trial design and manufacturing for every phase of clinical development.
On a positive note, 54% of drug approvals for 2022 were for rare diseases. Dr. Melanie Blank at the FDA’s CDER Division noted that they are seeing several new applications every week for gene therapies.
Even more encouraging is the news from Global Data that there are 113 gene therapies in Phase III trials and more than half are for rare diseases.
The FDA’s OTAT office approved its first cell and gene therapy in 2017. Since that time the OTAT has approved 27 such therapies. Further to the town hall discussion, leaders spoke about the importance of pediatric patients’ participation in trials that focused on gene therapy. The topic moved on to discussing the difficulties that may occur in getting consent for early trials. Despite these concerns, clinicians should be encouraged to enroll patients who would most likely benefit noting that the early trials present the most safety risks.
Dr. Hart commented that the Agency recognizes the urgent medical need to treat fully progressive rare diseases.
