Skip to main content Back to Top


FDA Issues Risk Management Guidance for Drug Makers

Donna Young

When planning risk assessment and risk minimization activities, drug makers should seek input from pharmacists and other health care providers, consumers, and payers, the Food and Drug Administration (FDA) counseled in three draft guidance documents it released today.

Under the Prescription Drug User Fee Amendments of 2002, or PDUFA III, FDA agreed to meet certain performance goals, including developing formal guidance for the drug industry on risk management activities, in exchange for user fees—payments the agency collects from firms to reduce the approval time of their new drug and biological license applications.

The agency released concept papers in March 2003 outlining its proposed approach for the documents and heard comments from the public at a three-day workshop held April 9–11, 2003, in Washington, D.C.

FDA is accepting comments on the draft documents until July 6 and plans to release the final guidances by September 30.

Risk management—the process of assessing a product's benefit-risk balance, developing and implementing tools to minimize its risks while preserving its benefits, evaluating tool effectiveness and reassessing the benefit-risk balance, and making adjustments to the risk minimization tools to further improve the benefit-risk balance—should be continuous throughout a product's lifecycle, with the results of risk assessment informing the manufacturer's decisions regarding risk minimization, according to FDA.

The guidance documents do not require drug companies to take any action regarding risk assessment and minimization, said Steven K. Galson, acting director of FDA's Center for Drug Evaluation and Research.

"Like with all of our guidance, these are not requirements," he said. "There's nothing specific that we expect to see."

Instead, he said, the guidances provide "more information and transparency about how we are thinking about these issues in the agency," and assist drug makers "as they think about putting together their approval packages, creating risk management programs, and looking at their data after approval."

"Overall what we're trying to achieve is more benefit from products with less risk," Galson said.

FDA has coined a new name for a strategic safety plan that a drug company submits to the agency to minimize the risks associated with a product: risk minimization action plan, or RiskMAP.

"One of the critiques we received on the concept paper and at the public meeting was that readers were confused as to the use of the terms risk management and risk management program," said Christine Bechtel, FDA science policy analyst.

However, the name change for safety programs will not be official until the final document is issued, she added.

A RiskMAP targets one or more safety-related health outcomes or goals and uses one or more tools to achieve those goals, according to FDA.

The guidance documents do not specifically address challenges health-systems pharmacists face, such as the difficulties of dispensing isotretinoin, which requires pharmacists to dispense a 30-day supply of the drug only when presented with a prescription that bears a yellow qualification sticker, when orders are submitted by a prescriber using a computerized order entry system.

But, Galson said, "we do have language that talks about the necessity of really making sure that plans that are laid out can be implemented across different sectors in the health care community. This is one of the issues that we heard in the comments, that depending on the circumstance of how drugs are actually prescribed, it's more difficult for some sectors to comply with the plans, and this is something that we do think is an important factor."

FDA's guidance about developing, implementing, and evaluating RiskMAPs suggests that manufacturers should seek input from health systems and providers about the feasibility of implementing and accepting a risk minimization tool.

"Acknowledge the importance of using tools with the least burdensome effect on health care practitioner–patient, pharmacist–patient, and/or other health care relationships," the guidance states.

RiskMAPs should be designed to be compatible with a health-system's current technology, applicable to both outpatient and inpatient use, accessible to patients in diverse locales, and consistent with existing tools and programs that have achieved positive results, the agency advised.