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Product Applications (including Investigational Applications) - Draft Proposal 13

View PDF of the Product Applications (including Investigational Applications) Section (142 KB)


Summary of Public Comments


View All Product Applications (including Investigational Applications) Draft Proposals

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Draft Proposal

FDA should disclose the fact that the Agency has issued a refuse-to-file or complete response letter in response to an original NDA, BLA, or an efficacy supplement for an NDA or BLA at the time the refuse-to-file or complete response letter is issued, and should, at the same time, disclose the refuse-to-file or complete response letter, which contains the reasons for issuing the letter. 

Reasoning: There are significant public benefits associated with disclosing information about letters FDA issues when the Agency does not accept a marketing application or approve or clear a marketing application for a medical product.  Disclosing that FDA has sent the sponsor one of these letters would inform the public about the current status of an application and the fact that FDA cannot approve or clear the application in its current form.  Disclosing the letter would provide the public with FDA’s reasons for its actions.  Disclosing that the letter has been sent, and the letter itself, may be valuable to patients and health care providers who want to know whether, and when, new medical treatments will become available.  If the application at issue seeks approval of a new indication for a product that is currently used off-label for that indication (i.e., for an indication that has not been approved by FDA), health care professionals and patients would have access to information that could influence decisions about whether to use the products for that off-label use, if such use is not otherwise prohibited.  Investors may also be provided with information that may allow for the most efficient use of limited research dollars.

Companies sometimes disclose the receipt of these letters from FDA to the public.  But even when it is publicly reported that FDA has not approved or cleared an application, or has not filed or accepted the application for review, FDA’s reasons for its action may not presented.  FDA does not say anything to the public about the basis for its decision.  And in cases where a sponsor provides an explanation to the public about FDA’s determination, the information provided may not give the public a complete picture of FDA’s rationale for its decision. 

FDA should publicly explain its rationale for not filing or receiving an application to market a product, not approving a medical product, or not clearing a medical device.  Disclosure of the letter sent to sponsors, which sets forth those reasons, enhances the credibility of FDA's decisions by revealing the basis for the Agency's decisions not to permit the marketing of a product and furthers the goal of more transparent and open government called for by the Administration.  FDA will also be able to explain its concerns about an application to the public in more detail, even in cases where the company provides information to the public about FDA’s action.  And by allowing FDA to explain its decision-making process to the public, the public may get a better understanding of FDA’s application review process.  Disclosing the letters FDA issues when the Agency does not accept a marketing application, or approve or clear a marketing application for a medical product will provide the public with reasons for FDA’s decision about the application.

The EMA currently makes available online the reasons for denying initial marketing authorization applications (i.e., marketing applications), denying applications for new indications, or denying applications for an extension of a marketing authorization (e.g., changes to the active substance or a new route of administration).  As noted above, given the global nature of the industry, some companies operating in the U.S. are currently subject to the EMA’s disclosure policy.

The Task Force concluded that when FDA issues letters to sponsors in response to ANDAs, ANADAs, chemistry, manufacturing, and controls (CMC) supplements, or labeling supplements FDA’s rationale for not approving the application primarily relates to how the drug was made, or to labeling negotiations between the sponsor and FDA.  Additionally, CMC information contains a great deal of trade secret information, which the Task Force supports redacting.  Disclosing these letters would provide little insight about the rationale underlying FDA’s drug review process.

The public health interests set forth above strongly favor disclosure when FDA has made a decision not to accept a marketing application or to approve or clear a marketing 
application for a new medical product, or for a new use for an existing product.  In most cases, those letters will include deficiencies relating to the safety and efficacy of the product.  The Task Force is not proposing disclosure of letters issued to sponsors in response to ANDAs, ANADAs, chemistry, manufacturing, and controls (CMC) supplements, or labeling supplements because those letters will yield little public benefit, particularly in light of the need to protect trade secret information and maintain incentives for innovation.

Comments for this proposal 

(Total Received: 1)

Comment: It is troublesome that although the FDA is willing to focus on transparency related to the efforts or inability of drugs to reach the market, such as the existence of an IND (Proposal 8), or an IND being placed on hold, terminated or withdrawn (Proposal 9), or the submission of an NDA (Proposal 10), or the withdrawal or abandonment of an NDA (Proposal 11), or the refusal to file an NDA (Proposal 13), or relevant safety and effectiveness information on a pending IND or NDA (Proposal 16), there are no draft proposals related to why drugs were approved for marketing and any safety-related issues OND wrestled with in reaching its decision to approve. Since 1992, access to drugs has been given priority over safety as drugs are rushed to market to meet deadlines. Why not give the public a heads-up on potential risks of newly-marketed drugs and why OND believed the benefits outweighed those risks?
Date Submitted: 6/01/2010