Stressing the need for speed and efficiency, FDA Commissioner Scott Gottlieb on Monday offered a look at FDA’s plans around real-world data (RWD) and real-world evidence (RWE) in 2019.
The speech at the Bipartisan Policy Center built off a framework, released in December, that sought to begin the conversation on how RWD and RWE can support changes to labeling, including adding or modifying an indication, changes in dose, dose regimen or route of administration; changing or adding new populations; or the addition of comparative effectiveness or safety information.
“Traditional postmarket studies typically require years to design and complete and cost millions of dollars. By using RWD and RWE, we may be able to provide patients and providers with important answers much sooner – identifying a broader range of safety signals more quickly, and following up on them much more effectively,” Gottlieb said, noting this is especially important for treatments for uncommon conditions, such as rare tumor types.
Gottlieb also laid out four additional activities the FDA is taking in connection to RWE and RWD this year.
First, to support the integration of digital technologies in clinical trials, the FDA is planning to convene a stakeholder meeting to help develop a framework on how digital systems can be used to enhance the oversight of clinical trials.
“To take one example, remote- and risk-based monitoring can provide better regulatory oversight,” he said. “These approaches may lower development costs, and enable more trial sites to answer important scientific and clinical questions as a way to improve patient care.”
He also said the FDA will use digital technologies to bring clinical trials to the patient, rather than always requiring the patient to travel to the investigator.
“To support the development and adoption of decentralized trials, the FDA established a formal working group on decentralized trials, and we’re working towards writing a guidance document to outline our approach. We’ll
have much more to say about this in the next month,” he said.
And third, the FDA is exploring how reviewers can gain more insight into how labeling changes inform provider prescribing decisions and patient outcomes. The FDA’s Information Exchange and Data Transformation (INFORMED) is using RWD to examine the impact of a recent FDA labeling change for two approved products from weight-based dosing to flat-dosing of immune checkpoint inhibitors.
In 2019, INFORMED is going to be working with FDA’s medical product centers to develop an FDA curriculum on machine learning and artificial intelligence in partnership with external academic partners.
“The agency will also pilot a competitive fellowship program under INFORMED in artificial intelligence and machine learning that allows post-doctoral fellows from leading academic centers to join the FDA for two-year fellowships to develop high-impact AI-based regulatory science tools by working closely with mentors from the agency’s medical product centers,” Gottlieb said.
He also noted that in oncology, the FDA currently has new drug applications under review where RWD and RWE are helping to inform ongoing evaluations.
The FDA’s Oncology Center of Excellence (OCE) is working with Friends of Cancer Research, the National Cancer Institute and other stakeholders to harmonize reference standards for assessing tumor mutational burden to help identify cancer patients who are more likely to respond to immunotherapy.
“OCE is also working on a project exploring whether it’s possible to use real world endpoints, like time to treatment discontinuation (TTD) from in legacy datasets as a potential real-world endpoint for pragmatic randomized clinical trials for FDA-approved therapies in the off-label setting, and whether synthetic control arms based on prior clinical trials can be used for tumor types where the standard of care has not changed in decades, and the prognosis is especially poor,” he added.
Scott Gottlieb at a 2016 event. BIPARTISAN POLICY CENTER
First published here. Regulatory Focus is the flagship online publication of the Regulatory Affairs Professionals Society (RAPS), the largest global organization of and for those involved with the regulation of healthcare and related products, including medical devices, pharmaceuticals, biologics and nutritional products. Email email@example.com for more information.
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