Jill Wechsler

Although manufacturers are willing to implement costly and cumbersome programs to meet FDA postmarketing safety requirements, doctors, pharmacists, and other healthcare providers are alarmed by the proliferation of new requirements for drug prescribing and dispensing. The most restrictive REMS programs are intended to apply only to those very high-risk products that cannot come to market without special safety controls, but healthcare providers, as well as manufacturers, feel that the program is being overused, raising costs and interfering with patient treatment. Health providers regard all the new educational and patient registration programs as a significant burden on their operations and on the healthcare system. Similarly, payers are concerned about added costs, while consumers are caught between their desire for access to treatment and the need to avoid adverse events.

These issues have been debated at recent advisory committee meetings to evaluate proposed drugs, as well as at a two-day FDA public meeting in July on the REMS program. The shape and scope of REMS requirements has become a central topic related to revising the Prescription Drug User Fee program (PDUFA), and significant changes are likely to be included in the user fee renewal legislation slated for enactment by 2012.

At the July public meeting, Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research (CDER), acknowledged that the REMS program established by the FDA Amendments Act of 2007 (FDAAA) "is not perfect" and that the FDA is looking for guidance from all stakeholders on "how we might remodel our house." The FDA also published a draft guidance in October 2009 on what information manufacturers should include in REMS proposals, and the FDA is reviewing comments on this plan.

Everyone at the meeting acknowledged the importance of REMS in ensuring safe drug use, while highlighting the unintended consequences of added postmarketing requirements at both ends of the REMS spectrum: more diverse medication guides, as well as a growing number of highly restricted REMS with Elements to Assure Safe Use (ETASU). The general complaint is that there are too many REMS of all kinds, and that the FDA should more clearly explain the need for special risk management requirements. So far, the FDA has approved more than 120 REMS, including 15 with ETASU.

Pharmacists, physicians, and healthcare leaders outlined a host of burdens and costs imposed by multiple REMS requirements. Physicians cited multiple educational and training programs that they consider redundant and unnecessary. Oncologists have been particularly vocal in opposing added educational requirements, noting that they are well trained in using highly toxic chemotherapies. Pharmacists described how training, tracking compliance, and patient counseling takes time and interferes with pharmacy operations.

Probably the most objectionable REMS feature for pharmacists and providers is the limited distribution systems for high-risk therapies that permit prescribing by only certain well-informed specialists and dispensing by specialty pharmacies that can verify appropriate use and patient understanding of risks. Retail pharmacists complain that such systems steer patients away from regular pharmacies, while hospitals and health plans cite added costs and difficulties in obtaining therapies from restricted sources.