Patient and Caregiver Experiences With and Perceptions of Risk Evaluation and Mitigation Strategy Programs With Elements to Assure Safe Use

Abstract

IMPORTANCEThe US Food and Drug Administration (FDA) Amendments Act of 2007 authorized the FDA to impose safety requirements on drugs with important risks, such as prescriber certification or routine laboratory testing, to ensure that the benefits of use outweighed the risks. However, little is known about patient and caregiver experiences with these Risk Evaluation and Mitigation Strategy (REMS) programs with Elements to Assure Safe Use (ETASU). OBJECTIVE To understand patient and caregiver experiences with and perceptions of REMS programs with ETASU. DESIGN, SETTING, AND PARTICIPANTS This qualitative study included semistructured qualitative phone interviews conducted between 2016 and 2017, with initial analysis performed in 2017 and reanalysis performed in 2021. Adult patients prescribed natalizumab or sodium oxybate, adult patients or caregivers of adult patients prescribed vigabatrin, and adult female patients of reproductive age prescribed riociguat were included. MAIN OUTCOMES AND MEASURESAssessment of knowledge, decision-making, medication access, and perceptions of medical privacy. RESULTS Among 63 participants, 46 (73%) were female. Twenty-five participants (40%) had taken natalizumab, 10 (16%) riociguat, 15 (24%) sodium oxybate, and 10 (16%) vigabatrin. One participant had taken both natalizumab and vigabatrin; 4 (6%) were caregivers of patients using vigabatrin. Most participants expressed knowledge of REMS program requirements, but many lacked the insight that these requirements were part of an FDA-mandated special safety program and expressed difficulty understanding program education materials. REMS requirements made some participants more likely to initiate treatment. However, many reported burdens accessing medication, including the need to travel to certified prescribers or pharmacies. Manufacturer access to personal health information was also controversial, although some participants expressed an altruistic desire to assist others. CONCLUSIONS AND RELEVANCEThis qualitative study found that REMS programs with ETASU reassured patients and their caregivers about drug safety and helped support medication initiation.However, steps are needed to improve the quality of REMS educational materials, promote efficient medication access, and protect patient privacy.

How Do Risk Evaluation and Mitigation Strategies Impact Clinical Practice? A National Survey of Physicians

“…Across all REMS programs, just over half of physicians reported that the safe use requirements delayed patients in getting their medications. Problems with access have also been reported by patients 23 . To better integrate REMS programs into clinical workflows, the FDA has partnered with the MITRE Corporation for the development of a “proof‐of‐concept prototype,” incorporating REMS activities into standard health information technologies 24 .…”

“…Problems with access have also been reported by patients. 23 To better integrate REMS programs into clinical workflows, the FDA has partnered with the MITRE Corporation for the development of a "proof-of-concept prototype, " incorporating REMS activities into standard health information technologies. 24 Routine re-evaluation of REMS programs can help determine whether to remove certain safe use requirements.…”

“…In interviews with prescribers of one of four REMS-covered drugs (natalizumab, riociguat, sodium oxybate, and vigabatrin), some physicians reported that the REMS programs facilitated discussion with patients and increased comfort with prescribing. 10 However, the generalizability of these findings to other physicians and to prescribers of other REMS-covered drugs is unknown. In addition, anecdotal reports have emerged in the medical literature expressing concern over the time and resources required to comply with REMS programs, 11 the value of their certification processes, 12 and the need for their safe use requirements.…”

“…REMS programs may affect clinical practice. In interviews with prescribers of one of four REMS‐covered drugs (natalizumab, riociguat, sodium oxybate, and vigabatrin), some physicians reported that the REMS programs facilitated discussion with patients and increased comfort with prescribing 10 . However, the generalizability of these findings to other physicians and to prescribers of other REMS‐covered drugs is unknown.…”

Analysis of risk evaluation and mitigation strategies for teratogenic drugs: Variation in primary and secondary prevention measures

“…For secondary prevention, the heightened frequency of testing before treatment initiation and during the first month of treatment for some REMS may pose cost and access issues for patients without meaningfully contributing to risk mitigation [ 25 ]. In addition to streamlining these testing requirements, REMS should ensure that females are effectively counseled on emergency contraception and provided with take-home materials to foster patient autonomy in minimizing teratogenic risks.…”