11:03 AM
Dec 06, 2018
 |  BC Extra  |  Politics & Policy

FDA releases real-world evidence framework

FDA is creating a roadmap that will facilitate the integration of real-world evidence into drug development. FDA’s framework for real-world evidence envisions using data from electronic health records (EHRs), observational and pragmatic studies, and mobile or wearable devices to generate effectiveness data that would support approvals of new indications for approved drugs, and for satisfying postapproval study requirements.

Under the framework, real-world evidence (RWE) could be used to add or modify an indication, including by changing doses, dose regimens, or route of administration; or to update a label with a new population or data on comparative effectiveness or safety.

The framework focuses on supplemental approvals and does not address the use of real-world data to support initial approvals of new drugs.

In a statement, FDA Commissioner Scott Gottlieb said RWE is a “top strategic priority for the FDA.”

Gottlieb highlighted the potential for real-world data (RWD) and RWE to make drug development more efficient and to produce knowledge that is more relevant for patients and physicians than traditional clinical trials. “RWE provides us with a potential source of information that can complement, augment and expand our understanding of how best to use medical products -- improving what we know about our medical care.”

Technologies for collecting RWD and techniques for turning it into RWE will "gradually allow us to shift some studies and data collection to the point of care, making the collection of data and the development of actionable evidence more efficient,” Gottlieb said.

FDA has identified challenges that limit the use of RWD, and plans to launch initiatives to overcome them. For example, the agency said it will “explore strategies for filling gaps in data that may be difficult to obtain from currently used EHRs and medical claims data, including exploring the use of mobile technologies, electronic patient reported outcome tools, wearables, and biosensors.” It plans to review and publish guidance on strategies for overcoming such gaps.

FDA will also write guidance on incorporating data from routine clinical care, or “pragmatic elements,” into clinical trials that can generate effectiveness data to support regulatory decisions.

According to the framework, the agency will “explore pragmatic approaches to each stage of a clinical trial, including recruitment and enrollment of patients, strategies for facilitating interventions, and approaches to assessing outcomes.”

FDA plans to write additional guidance on the use of observational studies to generate effectiveness data for regulatory decisions.

Gottlieb highlighted some ongoing FDA initiatives to promote the use of RWE. These include “funding a project to validate the potential of RWE to replicate the results of 30 clinical trials -- both positive (23 trials) and negative (7 trials) -- using claims data.” The project will include trials of cardiovascular, endocrine, musculoskeletal, and pulmonary therapies. FDA hopes to learn "which RWE methods and trial designs can provide insight into the opportunities and limitations of using these designs in regulatory decisions,” Gottlieb said.

The framework, which fulfills mandates in the 21st Century Cures Act, was informed by ideas provided by academic researchers, the biopharma industry and patient advocates at public meetings. These included a Duke Margolis Center for Health Policy meeting, as well as workshops organized by the National Academy of Sciences, Engineering, and Medicine.

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