Submissions and denials

Even more significant transparency proposals aim to disclose when drug-product manufacturers seek FDA approval to launch clinical trials or to bring to market a new drug, biologic, or medical device. FDA also wants to announce when such applications are withdrawn, rejected, or delayed, and to release Complete Response (CR) letters that describe why the agency is not approving a submission and what the sponsor needs to do to get its product to market.

Sponsors often elect to announce when they file an investigational new drug (IND) application to demonstrate that a development project is moving forward, and the FDA Amendments Act of 2007 requires manufacturers to post information about ongoing clinical trials and their results on the ClinicalTrials.gov/ website. However, current FDA regulations prohibit the release of most information regarding the status or substance of investigational products.

The task force further proposes that FDA provide more information on the status of market applications for new drugs and medical products. Under current policy, FDA only announces when it approves a medical product for market, but not when a manufacturer files a new drug application (NDA) or regulatory submission for a generic drug, biologic, animal drug, or medical device. Manufacturers often acknowledge application filings in financial reports, and FDA posts a good deal of information on products scheduled for advisory-committee review.

As part of Phase 2, the agency seeks authority to disclose when a company files a market application or efficacy supplement for a new use of an existing product. FDA also wants to announce when such applications are withdrawn or abandoned, especially when such action arises because of safety issues. In a related proposal, the task force would like FDA to be able to explain to the public that safety concerns or regulatory issues are not responsible when a sponsor abandons or withdraws an application for an orphan drug purely for business reasons; the aim here is to encourage other parties to continue development of a needed therapy for a rare disease.

An important, and controversial, proposal is for FDA to release refuse-to-file notices and CR or not-approvable letters for applications and efficacy supplements for human and animal drugs and medical devices. Sponsors often issue a summary of a CR letter to acknowledge a delay in approval and outline a scenario for reaching the market in the future. Manufacturers are wary that posting CR letters might reveal proprietary information, but FDA feels that company releases seldom fully explain agency decisions. Former FDA official Scott Gottlieb, now with the American Enterprise Institute, says that drug companies should support the release of CR letters because the prospect of explaining to the public why it is delaying approval of a needed therapy may make it "harder for the agency to make bad decisions," particularly with diseases that lack approved treatment. Investors similarly like the idea of having access to the full details of FDA's statements, and the agency feels that the information would be useful to doctors and patients.

However, the transparency panel decided not to seek disclosure of refusal or CR letters to generic-drug manufacturers or for manufacturing and labeling supplements. The hundreds of chemistry, manufacturing, and controls supplements filed each year contain a great deal of trade secrets that would be difficult to redact and, the task force notes, "would provide little insight about the rationale underlying FDA's drug-review process."