Once an expedited pathway is granted, the therapeutics dossier – a thorough collection of documents submitted to the FDA, often ranging from 5,000 -15,000 pages – undergoes intense scrutiny. Just like standard reviews, experts from the agency evaluate data across a treatment’s research and development journey, but at a quicker pace. From pharmacodynamics studies that look at how different doses of drugs are processed within the body, to pre-clinical data from cell lines, to manufacturing quality and control, to clinical trial results, a huge amount of information is evaluated in parallel, sparking questions that require rapid answers.
“We typically receive a minimum of 200 questions during the review,” Lipp says, noting that number can easily swell to 1,000 queries that the cross-functional team is committed to answering quickly. “As a part of the Commissioner’s National Priority Voucher (CNPV) program, for example, we made a commitment to the FDA that our teams would work 24 hours a day, seven days a week.” And on that project, there was indeed a sprint: “We received questions at 11pm on Saturday and answers were due Sunday midday.”
Despite the quicker pace of evaluation, the FDA applies the same rigorous standards for demonstrating drug safety, quality and efficacy. The review process still brings together experts from diverse scientific backgrounds to assess distinct aspects of the therapeutic, evaluate clinical data, inspect manufacturing sites, and carry out thorough analyses to reach a conclusion.
The expedited approval, however, isn’t the end of safety and efficacy research – we are required to continue to monitor the drug usage to prove long-term clinical benefit for the drug to retain its approved status.