According to Peter Marks, an official of the U.S. Food and Drug Administration, rapid approval has to be used to advance gene therapy for rare disorders. Dr. Marks, the director of the agencys Center for Biologics Evaluation and Research, stated that accelerated approval is crucial for ultra-rare diseases since there are not enough individuals with these conditions to do placebo-controlled studies.

The so-called "accelerated approval" is given by the American health regulator primarily for medicines and therapies for uncommon illnesses or small patient populations that have previously lacked access to effective treatments. Last week, the FDA announced that it will convene a panel meeting to examine Sarepta Therapeutics Inc.s gene therapy for Duchenne muscular dystrophy, less than a month after stating that it would not.

Using the FDAs fast process, the business is requesting approval for its gene treatment. Lastly, Dr. Marks suggested a new regulatory norm as one of the steps to access gene treatments for those with uncommon diseases, as he explained that while making gene therapy for 10 to 20 people a year is feasible, the concept of a randomized clinical trial falls apart pretty quickly. 

(Source: https://health.economictimes.indiatimes.com/news/pharma/us-fda-official-says-agency-needs-to-start-using-accelerated-approval-for-gene-therapies/98852719 )