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Statement of Need: Before a drug is approved for marketing, FDA must determine that the drug is safe and effective for its intended use. This determination is based in part on clinical study data (CSD) that are submitted as part of the marketing application. At present, FDA accepts CSD in paper and electronic formats. When CSD are submitted to FDA only on paper, the information must be transcribed by hand into electronic form for review and analysis. This process is extremely time consuming and is prone to data entry error. CSD submitted to FDA in electronic format have generally been more efficient to process and review. FDA’s proposed rule would require the submission of CSD in a standardized electronic format. The standardized CSD format would improve patient safety and enhance health care delivery by enabling FDA to process, review, and archive CSD more efficiently. Standardization of CSD would also enhance the ability to share study data and communicate results. Investigators and industry would benefit from the use of standards throughout the lifecycle of a study--in data collection, reporting, and analysis. The proposal would work in concert with ongoing agency and national initiatives to support increased use of electronic technology as a means to improve patient safety and enhance health care delivery.

Summary of the Legal Basis: Our legal authority to amend our regulations governing the submission and format of CSD for human drugs and biologics derives from sections 505 and 701 of the act (U.S.C. 355 and 371) and section 351 of the Public Health Service Act (42 U.S.C. 262).

Alternatives: FDA considered issuing a guidance document outlining the electronic submission and the standardization of CSD, but not requiring electronic submission of CSD in the standardized format. This alternative was rejected because the agency would not fully benefit from standardization until it became the industry standard, which could take up to 20 years. We also considered a number of different implementation scenarios, from shorter to longer time-periods. The two-year time-period was selected because the agency believes it would provide ample time for applicants to comply without too long a delay in the effective date. A longer time-period would delay the benefit from the increased efficiencies, such as standardization of review tools across applications, and the incremental cost saving to industry would be small.

Anticipated Costs and Benefits: FDA estimates that the costs to industry resulting from the proposal would include some one-time costs and some potential annual recurring costs. One-time costs would include, among other things, the cost of converting CSD to standard structures, terminology, and cost sets (i.e., purchase of software to convert CSD); the cost of submitting electronic CSD (i.e., purchase of file transfer programs); and the cost of installing and validating the software and training personnel. Additional annual recurring costs may result from software purchases and licensing agreements for use of proprietary terminologies. The proposal could result in many long-term benefits for industry and for the agency, including improved patient safety through more efficient, comprehensive, and accurate data review; enhanced communication among sponsors, clinicians, and FDA through improved access to and sharing of CSD; and reduced data management costs through the standardization of data formats.

Risks: None.