In May, the US Food and Drug Administration (“FDA”) released a draft guidance outlining its current thinking on real-world data (“RWD”) and real-world evidence (“RWE”), and how such data and evidence can be used in submissions. Since May, a number of stakeholders have submitted comments to the draft guidance with mixed results, as some caution against the use of RWD and RWE, while others encourage the FDA to expand the scope of RWD and RWE.
The draft guidance, entitled “Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drugs and Biologics,” is “intended to encourage sponsors who are using [RWD] to generate [RWE] as part of a regulatory submission … to provide information on their use of RWE in a simple, uniform format.” The guidance defines RWD and RWE, as follows:
RWD are “data relating to patient health status and/or the delivery of health care that are routinely collected from a variety of sources.” Sources of RWD include, for example, electronic health records, medical claims and billing data, product and disease registries, patient-generated data, data gathered from mobile devices.
RWE is “the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD.” Sources of RWE include, for example, randomized clinical trials or observational studies.
A submission including RWE should so note in the cover letter, as well as identify the purpose of using the RWE, the clinical study design that uses the RWE, and the RWD sources of the RWE.
A number of trade associations and biopharmaceutical companies have since commented on the draft guidance. Gilead Sciences, Inc., proposed broadening parts of the guidance, including expanding the list of submissions to include supplemental new drug applications and supplemental biologics license applications, including lab data as a source of RWD, and including retrospective analysis of RWD collected outside of clinical trials to generate RWE. Novartis Pharmaceutical Corporation similarly called for broadening the scope of the guidance, for example by including pharmacy claims as a source for RWE, and to expand the sources of clinical evidence to include patient reported outcomes and quality of life scores.
Other stakeholders, however, voiced concerns about the proposals. The Biotechnology Innovation Organization (“BIO”) noted that the proposals “may be delayed or impeded” by implementation challenges, such as a lack of experience at the review division level. BIO also suggested that the FDA should share “learnings and best practices” with other regulators to promote harmonization. The Pharmaceutical Research and Manufacturers of America (“PhRMA”) echoed BIO’s concerns and encouraged harmonization to increase consistency and “regulatory predictability.” PhRMA also suggested that the FDA implement tracking and reporting aggregate data about applications containing RWD and RWE and how “submitted RWD/RWE was used to inform … regulatory decision-making.”
The draft guidance is just the latest effort by the FDA to “integrate more effective ways to leverage electronic tools to gather and use [the] vast amounts of health-related data” being generated in the health care system. The FDA notes that utilizing RWD and RWE is a “top strategic priority.”