FDA Seeks To Improve REMS Program For High Risk Drugs

Federal regulators are looking at ways to lessen the risks associated with certain medications that are already known to carry dangerous side effects, but which are still medically necessary.

In a statement issued on January 24, FDA Commissioner Scott Gottlieb announced two draft guidance documents, which he says the agency believes will make certain that Risk Evaluation and Mitigation Strategies (REMS) programs are working to make sure only patients who need the drugs get them, and that they and their doctors are fully aware of the risks.

One draft guidance lays out planning and reporting best practices for industry (PDF), and the other lays out survey methodologies (PDF) to assess whether the REMS programs are meeting their goals.

The REMS program is reserved for drugs that have known high risks of adverse events, but are medically necessary for some patients. In many cases, the drugs are one-of-a-kind treatments for severe health problems, or are second-line treatments for patients who, for whatever reasons, cannot take the normally prescribed drug.

In all cases, the program seeks to ensure that the right patients get the drugs and that their doctors are aware of how and when they should be prescribed, and what to do if there’s a problem.

“It’s critical that we develop and implement rigorous ways to assess the effectiveness of REMS in real-world settings. We need to make sure that the REMS are achieving their public health goals, and that we’re designing and implementing these approaches in ways that minimize burdens to patients and providers. We know there’s still much to learn about the science of risk management,” Gottlieb said in the statement. “Approaches and scientific methods for assessing the effectiveness of REMS continue to evolve. Our goal in providing this latest thinking on assessing REMS programs is to ensure we’re constantly improving the programs we have in place today, and establishing the most efficient and effective programs moving forward, to optimize patient care and keep Americans safe and healthy.”

Guidance are not hard and fast regulations which industry are required to obey, but are instead suggested practices the FDA feels is the best interpretation of existing regulations. While they are not legal rules, following them means companies are less likely to run afoul of existing regulations.