FDA Proposals Would Shed Light on Drug Approval Process

Agency Seeking Public Input on Transparency Issues

June 09, 2010 03:45 pm News Staff

The FDA is considering a proposal that would provide physicians and consumers with more data about the safety and effectiveness of medical products with pending applications.

The agency is seeking public comment(www.fda.gov) on that issue and 20 other draft proposals related to its public disclosure policies.

Joshua Sharfstein, M.D., the FDA's principal deputy commissioner and chair of the agency's transparency task force, said during a May 19 media briefing that the proposals are intended to help consumers, stakeholders and others understand the agency's operations and decision making -- all while balancing the need for transparency with manufacturers' desire to protect their proprietary information.

The proposals(edocket.access.gpo.gov) were published May 21 in the Federal Register, kicking off a 60-day comment period that ends July 20. The task force will review comments and decide which proposals to recommend to FDA Commissioner Margaret Hamburg, M.D.

Hamburg launched the first phase of the agency's transparency initiative(www.fda.gov) last summer. The FDA received more than 1,500 comments -- which were the basis for the draft proposals -- after two public meetings conducted by the task force.

The draft proposals are part of the transparency initiative's second phase. Draft proposals regarding a third phase that covers the agency's transparency to regulated industry are expected this summer.

Sharfstein said during the briefing that some of the 21 draft proposals being considered in phase two might not be adopted. He also said some may require substantial increases in FDA resources, and some may require changes in laws or regulations to give the agency new or expanded authority.

"We didn't want to be sidetracked into a legal discussion," he said of the decision to move ahead with drafting the proposals despite uncertainty about the agency's regulatory authority in certain areas. "We really focused on what's the right thing to do."

If the proposals are adopted, the FDA could disclose when a drug or medical device is being studied and for what indication, when an application for a new product has been submitted or withdrawn, whether there were safety concerns associated with a product that led to withdrawal of an application, and why the agency did not approve an application.

Sharfstein said such transparency could accelerate the development process because manufacturers would be able to learn from the successes and failures of other companies.

For example, when an application for a so-called orphan drug -- one aimed at treating a rare disease or a condition with few treatment options -- is withdrawn or terminated by a sponsor for business reasons, one proposal would allow the FDA to clarify that the application was not withdrawn because of safety or effectiveness issues. Such clarification might encourage other companies to continue research on that product.

Some other highlights of the proposals:

  • the agency could disclose summary safety and effectiveness information from investigational applications and pending marketing applications if such a disclosure would be in the interest of the public health, such as correcting misleading information about a product;
  • the agency could disclose details of its inspections of clinical trial investigators, institutional review boards and manufacturing facilities;
  • the agency could share information about the most common observations of objectionable conditions or practices that are made during inspections; and
  • the agency should release more information in a more expeditious fashion during product recalls.