Risk Evaluation and Mitigation Strategies in the Specialty BioPharma Market


What each stakeholder needs to know about REMS in today's fast-paced climate.

What each stakeholder needs to know about REMS in today's fast-paced climate.


Heather Morel, MBA

Three years ago, Congress passed the FDA Amendments Act, which empowered the FDA to require Risk Evaluation and Mitigation Strategies (REMS) for new and existing products. The number of REMS required for products has steadily grown since the first programs were introduced in 2008.

Nearly 90% of these REMS have required a health professional communication plan and patient medication guide. For products with more serious safety concerns, the FDA requires pharmaceutical manufacturers to administer REMS with Elements to Assure Safe Use (ETASUs). These elements require manufacturers to institute post-market safety prescriber or patient registries, controlled distribution, and other tools.

While all REMS have the potential to improve safety, programs with ETASUs are most likely to have unintended consequences on health care stakeholders, including reduced access or increased burden. In the specialty market segment, REMS with ETASU have the highest prevalence compared with the rest of the market.

REMS’ Impact on Stakeholders Increasing

With more medication guides, communication plans, and REMS with ETASU, stakeholders—including physicians, pharmacists, specialty pharmacies, and distributors—have all had to alter their work flow and engage in activities supporting REMS on patients’ behalf. As the number of these programs increases, the need to indentify strategies for REMS’ success that enable patient access and minimize unnecessary disruption to business and clinical practice is intensified. The FDA explicitly states that it seeks to avoid what they term “unintended consequences” of REMS programs, including reduced access and increased burden to the health care system.

Nevertheless, the FDA can only advise on submitted REMS programs and is not directly involved in the program design details. The biotech and pharmaceutical company sponsors must design and prepare for the execution of their REMS, while hopefully minimizing the impact on stakeholders. Also, the FDA does not usually have any direct regulatory interaction over many of the stakeholders of REMS (eg, pharmacies) that are directly affected by REMS programs. REMS require many stakeholders to work together to increase the safe use of products and reduce the potential for adverse events.


The patient can have a variety of responsibilities, depending on the extent of the ETASU. At minimum, a patient medication guide is typically presented to the patient for review. Also, under ETASU, patients can be required to undergo training, testing or complete follow-up clinical counseling calls while on therapy.

Physicians and Other Health Care Providers

Physicians and other health care providers have a responsibility to understand the REMS requirements and engage with their patients to comply with them before initiating, and while maintaining, patients on therapy. The responsibilities that a physician has for REMS can vary greatly for each product. They can be as minimal as the distribution of a medication guide when the product is administered in the office, or as involved as completing a training course and providing baseline and ongoing follow-up status to the REMS program administrator on each patient on therapy.

Retail and Specialty Pharmacies

Pharmacies and pharmacists also play important roles in the administration and management of REMS programs. Most REMS require that a medication guide be given to patients, and often this responsibility is on the dispensing pharmacist. In REMS with ETASU, often the design of the program calls for limiting access of the product to a more narrow set of dispensing pharmacies that will agree to comply with the REMS program requirements.

These dispensing pharmacies may, in some of the most restrictive REMS, undergo special education and training and be required to ensure that each dispensed prescription is written by a registered prescriber and is being given to a registered patient. These more comprehensive programs are attempting to use the point of dispense as the final checkpoint to ensure that only those patients who qualify are given the product.


Specialty distributors and wholesale distributors also may have controls that they are responsible to administer in REMS programs. As part of becoming a certified distributor of REMS with ETASUs, distributors may need to ensure that product is only distributed to locations that are enrolled and registered in the REMS program. Also, distributors may be required to provide data to pharmaceutical manufacturers to complete their required data sets, which must be delivered to the FDA to stay in compliance with the agreed requirements of their REMS.

REMS Program Administrators

Most pharmaceutical manufacturers contract with a REMS Program Administrator to provide centralized regulatory- compliant services for the collection and maintenance of all data elements, which must be reported back to the FDA resulting from the agreed upon REMS. Services include designing and maintaining the registries of prescribers, pharmacies, distributors, and patients required by ETASUs.

Often there are also call center support services to help address the questions and needs of stakeholders impacted by the REMS. These Program Administrators are also involved in the strategy, development and implementation of surveys and assessments provided to the FDA to determine if patients are receiving and understanding the content of the medication guides. Periodically, REMS Program Administrators provide data reports back to manufacturers from validated databases and deliver data sets to the pharma manufacturers, who then reports results back to the FDA.

Responsibilities of the Pharmaceutical Manufacturer

The pharmaceutical manufacturer is responsible for designing the REMS program to meet the safety objectives for their product. They must ensure compliance with the REMS program, which means that a Program Administrator must be identified and periodic reports of data must be made to the FDA on progress against the objectives of the REMS. The manufacturer must also track and collect adverse events and report them to the FDA.

Manufacturers must also work to ensure that access is supported for their products beyond REMS. Often this includes reimbursement patient assistance programs (PAPS), patient support services for therapy initiation, and support adherence and persistence to therapy. These services are often contracted to support services call center hubs and can be synergistic to the Program Administration services provided as part of a REMS.


Many stakeholders must align and remain accountable to the delivery of safer product use in the market. REMS require more of each stakeholder group and can force changes in processes, work flow and data collection, and reporting. Ultimately, it requires a careful balance to limit reduction in access with mitigating risks.

About the Author

Ms. Morel is Vice President and General Manager of Reimbursement & Access Services for McKesson Specialty Care Solutions in Scottsdale, Arizona. Morel leads the development and execution of reimbursement strategies and support services, patient assistance programs, risk evaluation and mitigation strategies programs, and other patient- and physician-focused initiatives. She is a nationally recognized expert in health care reimbursement and marketing. With this “Focus on REMS,” Morel joins Specialty Pharmacy Times as the regular REMS columnist and a member of the Editorial Board.

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