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Regulatory Focus™ > News Articles > 2020 > 3 > FDA Ends CSR Pilot, Plots New Approach for Disclosing Study Reports

FDA Ends CSR Pilot, Plots New Approach for Disclosing Study Reports

Posted 26 March 2020 | By Zachary Brennan 

FDA Ends CSR Pilot, Plots New Approach for Disclosing Study Reports

The US Food and Drug Administration (FDA) on Thursday officially ended a fledgling pilot program that was part of an effort to provide more transparency on the drug approval process. The agency said it’s working on a new approach to disclosing study reports.

Last June, the agency was already pushing away from the clinical study report (CSR) pilot because of lackluster participation (FDA noted that Janssen was the only sponsor that agreed to participate) and said it would focus its efforts to better communicate the basis for drug approvals on the development of new integrated review documents.

To illustrate the new integrated review template, which has three main components — an executive summary (including FDA’s benefit-risk determination), an interdisciplinary assessment and appendices (containing assessments and analyses that are supportive to key facts/data) — FDA rewrote the original review for Merck’s Pifeltro (doravirine) and Delstrigo (doravirine, lamivudine, and tenofovir disoproxil fumarate) to provide an example.

Still, more than 50 academics and researchers from Harvard, Yale, Johns Hopkins and other universities called on FDA to not replace its original reviews with integrated reviews because of the valuable information that would be lost.

But Peter Stein, director of FDA’s Office of New Drugs, assured Focus in an interview that the integrated reviews are moving forward and will not exclude information that was previously contained in the approval packages released by FDA.

Janet Woodcock, director of the Center for Drug Evaluation and Research, said in a statement Thursday that FDA has learned some useful information from the CSR pilot and may consider whether posting certain portions of the clinical summary data would improve transparency and enhance integrated review activities.

“For example, we found that there are significant inefficiencies in having multiregional disclosure requirements relating to often identical clinical data summaries. These inefficiencies multiply the transactional, administrative and redaction (because there are differing regional disclosure standards) costs, whether the costs are incurred by industry or a regional regulatory authority. These costs create barriers to programs to disclose clinical trial information which might be reduced if a centralized or regional approach could be achieved,” Woodcock wrote.

While noting the international nature of many trials, she said CDER identified a potential approach that could facilitate a harmonized system for disclosing study reports, which includes the following principles, some of which align with what the European Medicines Agency is doing:
  • “International Library: A centralized international library, managed by an independent organization, where information is made available to the public rather than each regulatory authority having its own system. MedDRA.org is an example of a successful model of international harmonization through use of an independent organization.
  • On-Demand: An on-demand system, where sponsors would automatically publish a limited number of documents in the international library, such as the summaries of clinical information and an index of study reports, should be considered. The public could request study reports of interest, and the sponsor would then prepare the report, protocol and statistical plan and add it to the library.
  • Standardized: Anonymization and disclosure standards would be established to satisfy the requirements of the participating regional regulatory authorities. Our ongoing, independent efforts to establish anonymization standards and best practices (e.g., PhUSE) will continue.
  • Voluntary: A sponsor could have the choice of committing to use the international library system to disclose study reports, protocols and statistical plans for all marketing applications or to follow the requirements of each region where they apply for authorization to market products.”

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