It’s here! The long awaited FDA “draft framework for implementation” of the real world evidence program required by Section 3022 of The 21st Century Cures Act (Cures Act) has been published.
Framework for FDA’s Real World Evidence Program
The Cures Act is designed to accelerate medical product development and bring new 
innovations and advances faster and more efficiently to the patients who need them. Among other provisions, the Cures Act added section 505F to the Federal Food, Drug, and Cosmetic Act (FD&C Act). Pursuant to this section, the Food and Drug Administration (FDA) has created a framework for evaluating the potential use of real-world evidence (RWE) to help support the approval of a new indication for a drug already approved under section 505(c) of the FD&C Act or to help support or satisfy drug post-approval study requirements – Framework for FDA’s Real World Evidence Program – December 2018
Section 3022 of the Cures Act – Real World Evidence
As a reminder, according to Section 3022 of the Cures Act:
The Secretary shall establish a program to evaluate the potential use of real world evidence:
- to help to support the approval of a new indication for a drug approved under section 505(c); and
- to help to support or satisfy post-approval study requirements.
- Not later than 2 years after enactment (13 Dec 2018), the Secretary must establish a “draft framework for implementation” of the real world evidence program.
- The “framework” must include information describing:
- the sources of real world evidence, including ongoing safety surveillance, observational studies, registries, claims, and patient-centered outcomes research activities;
- the gaps in data collection activities;
- the standards and methodologies for collection and analysis of real world evidence; and
- the priority areas, remaining challenges, and potential pilot opportunities that the program established under this section will address.”
The Secretary must implement the real world evidence program not later than 2 years after enactment (13 Dec 2018) to “evaluate the potential use of real world evidence.”
In addition, the Secretary must utilize the program and activities or pilots noted above to “inform” a guidance for industry on:
- the circumstances under which sponsors of drugs and the Secretary may rely on real world evidence to help support a new indication of an approved drug or to help support or satisfy post-approval study requirements; and
- the appropriate standards and methodologies for collection and analysis of real world evidence submitted for such purposes.
The Secretary must issue a draft guidance on these topics not later than 5 years after enactment (13 Dec 2021), and must finalize such guidance not later than 18 months after the close of public comment.