Digital health, targeted medicines, and regenerative medicine are just a few relatively new technologies pushing the life science industry forward at an accelerated pace. These technologies offer transformative opportunities but are also challenging the U.S. Food and Drug Administration (FDA) to modernize its approach to evaluating medical products.¹ A new FDA Voice blog post on the Agency’s website looks back at recent efforts made by FDA to modernize its evaluation of new medical technologies and looks ahead to initiatives planned in the coming years.
FDA Commissioner Scott Gottlieb uses the blog post to reflect upon the agency’s efforts to “[create] a new operating system for innovation by modernizing clinical trials, [streamline] the FDA’s organization and processes to advance regulatory science, expand the FDA’s capacity to analyze complex real-world data streams to detect early safety and efficacy signals… and to describe the new policies we plan to announce to advance these goals.”¹
Modernizing clinical trials for medical technologies
Gottlieb discusses several actions the agency has taken to modernize clinical trials for drugs and devices. Decreasing time and cost to conduct clinical trials can yield greater competition within the market, help reduce drug prices, and put medical products into the hands of patients faster. Gottlieb describes a trend where second and third-to-market competition for drugs targeting unmet needs is taking longer to reach patients. One reason Gottlieb points to for this trend is “the difficulty of conducting traditional clinical trials in settings where there is an available therapy, but still significant unmet medical need – for instance, in some rare diseases.”¹
FDA investigated these trends and plans to publish a full analysis soon. One key finding FDA uncovered when analyzing the number of drugs or biologics that CDER approved in the same class is this: when a novel sole source drug wins approval, it does not face competition from drugs in the same class. Follow-on drugs/biologics that would compete with the first-in-class drug are arriving more slowly. “For non-orphan pharmaceuticals, which treat conditions affecting larger patient populations, 41 percent of the first-in-class products approved between the years of 1991 and 2000 had at least one competitor in the same class within five years. This rate dropped sharply over the next decade.”¹
Additional topics covered in the blog post include: tactics being utilized to modernize FDA’s organization and breaking down outdated silos (e.g. standing up the Oncology Center of Excellence, proposed changes to CDER’s Office of New Drugs), harnessing real world evidence (RWE) to help answer questions that are relevant to broader patient populations, and FDA’s role in curating standards for novel technologies (e.g. the Pre-Cert program to facilitate and regulate the use of artificial intelligence in product development, electronic capture of patient-reported outcomes (PRO) data).
As biopharmaceuticals, medical devices, and diagnostics become more sophisticated, the methods for developing and evaluating new products to ensure efficacy and patient safety must adapt. Gottlieb’s post highlights the strides the agency has taken thus far to achieve this goal, but there is still a lot of work to be done. Our consultants average 25 years of experience and hail from different sectors within the life science industry. Learn more about our team.