March 2020: FDA Ends CSR Pilot, Plots New Approach for Disclosing Study Reports

The FDA on March 26th officially announced March 26th the end of the Clinical Data Summary Pilot Program that was part of an effort to provide more transparency on the drug approval process. FDA’s Center for Drug Evaluation and Research (CDER) had planned to release portions of the clinical study reports (CSRs) from the sponsor’s NDA along with the action package following approval.

Janet Woodcock, CDER Director, said in a March 26th statement 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, Woodcock 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 (EMA) 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. 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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