FDA’s REMS Drug Safety Program Sometimes Confusing To Patients, And A Barrier To Drug Access: Study

A new study highlights the high levels of confusion surrounding a key drug safety program, indicating that most consumers believe the risk reduction efforts are intended to benefit drug manufacturers, not patients, or find information about potential drug side effects difficult to understand.

The FDA’s Risk Evaluation and Mitigation Strategy (REMS) program was implemented to monitor medications with a high potential for serious side effects, authorizing the FDA to impose safety requirements on drugs with important risks.

Requirements can include doctor certification, routine laboratory testing, or a requirement to use specific pharmacies. The REMS program requirements apply to specific prescription drugs and can include requirements for the patient to fulfill as well, as part of an effort to ensure that the benefits of using drugs with potentially severe side effects outweigh the risks.

Researchers with Brigham and Women’s Hospital and Harvard Medical School conducted a survey of patients who encountered the REMS program, and found patients were often confused about what the program was for, what they were supposed to do, and who was benefitting from it. The findings were published January 20, in the journal JAMA Network Open.

The researchers conducted a study of 63 participants in the REMS program. This included some patients who received one of four drugs subject to REMS program with Elements to Assure Safe Use (ETASU) requirements mandated by the FDA. It also included caregivers of patients taking those drugs.

The study included phone interviews conducted between 2016 and 2017, focusing on four drugs: natalizumab, also known by the brand name Tysabri used to treat multiple sclerosis and Crohn’s disease; riociquat, known as Adempas used to treat pulmonary high blood pressure; sodium oxybate, known by the brand name Xyrem used to treat narcolepsy; and vigabatrin, known by brand name Sabril; an anticonvulsant used to treat seizures.

Many patients and their caregivers reported difficulty understanding program educational materials and difficultly accessing medications easily. They also had concerns regarding data-sharing, with some participants saying the enrollment forms were overwhelming or vague.

Patients reported that they knew obtaining the medication required meeting specific conditions, completing regular monitoring, or using centralized pharmacies. Yet, most participants said they did not know it was part of an FDA-mandated safety program.

The study also indicated REMS requirements made some patients more likely to initiate treatment, but many reported difficulties accessing the necessary medication, including having to travel to certified prescribers or pharmacies.

Some patients believed the REMS program was intended to help drug manufacturers or doctors, not patients. They also believed doctors were reimbursed for referring patients for enrollment, not as part of a mandatory program. Other patients thought the program was a way for a drug company to “cover its back if something went wrong,” such as patients suffering severe side effects.

Many patients said the program did not provide sufficient or clear information about the risks of the drugs or the reasons why the program exists to begin with. For example, progressive multifocal leukoencephalopathy is a side effect of taking Tysabri; but the information provided was too confusing or not clear enough for patients without a medical background.

Furthermore, some patients said they were not sufficiently updated on new findings about their drug. For example, patients taking Tysabri were not informed when the program was significantly modified for that drug, like making vision testing voluntary instead of mandatory.

“This qualitative study found that REMS programs with ETASU reassured patients and their caregivers about drug safety and helped support medication initiation,” the researchers concluded. “However, steps are needed to improve the quality of REMS educational materials, promote efficient medication access, and protect patient privacy.”

The study’s findings come after the FDA halted its REMS program for the antipsychotic Clozapine last month, after patients reported difficulty accessing the drug and delays in treatment when healthcare professionals were not certified in the program by the specified due date. Additionally long call wait times have made it difficult for patients to access care. As a result, the FDA placed the REMS program for Clozapine on hold.