REMS: Pathways to Drug Safety

An effective REMS program improves patient access to medication.

REMS: Pathways to Drug Safety

The EXL Risk Evaluation & Mitigation Strategy Summit, held at the end of January, was an opportunity for colleagues across the industry to discuss many topics related to REMS, including benefit-risk counseling between patients and providers, integrating REMS with healthcare systems and pharmacies, and evaluating the impact of REMS programs on stakeholders.

In my presentation, Evaluating REMS Programs for Effectiveness and Value, I shared insights I’ve gained from working with sponsors on more than 100 risk management plans and REMS programs. Below are some of the main points from my presentation.

Factoring in the Role of External Constituencies

In 2007, the Food and Drug Administration Amendments Act (FDAAA) heightened the industry’s approach to drug safety by giving the FDA the authority to require REMS. Since that time, I’ve seen an increasing emphasis on input from patients, healthcare professionals, payers, and regulators when designing and redesigning REMS programs.

  • Patients and advocacy groups bring key insights that can reduce barriers to access and help measure effectiveness. In addition, patients’ input during REMS planning can help create educational materials that are clear, impactful, and actionable.
  • Healthcare professionals can help assess the “ease of use” of a REMS and pinpoint unnecessary burdens on time and resources.
  • Payers have made their concerns clear related to the cost of specialty drugs. Including payers early in REMS planning can provide understanding on how a REMS may influence formulary placement or pre-authorization requirements.
  • Regulators have not always been considered partners in REMS planning, but they may be able to provide key findings from other products with REMS, identifying what types of data may be needed in the approval process and what factors may influence the benefit-risk balance.

Establishing Evidence of REMS Effectiveness: When designing a REMS program, sponsors should be looking beyond risk management strategies necessary for product approval. Once a product is on the market, sponsors will need to establish evidence of REMS effectiveness with patient and prescriber access in mind. Consider these questions:

  • Are the correct stakeholders utilizing the tools?
  • Is the REMS creating barriers to access?
  • Can the measures or tools be improved? Reduced? Removed? Technology advances and regulatory changes should continually be assessed to help answer these questions and modify the REMS program when possible.

Evaluating REMS Effectiveness: Sponsors have a variety of options when it comes to assessing the effectiveness of REMS programs. The challenge comes in identifying which tools will provide the most accurate assessment. Tools may include:

  • Knowledge, Attitude and Behavior Surveys to measure understanding of stakeholder knowledge.
  • Chart Reviews to evaluate pre- and post-REMS behavior.
  • Epidemiologic studies in large databases to identify patients and healthcare providers, allowing population-based estimates of risk and possibly determining appropriate patient selection.
  • Drug Utilization Studies to better understand treatment patterns

Assessing with RE-AIM: The RE-AIM framework, developed in 19991 for purposes of assessing the public health impact of an intervention, continues to be an effective outline for organizing an effective REMS assessment. RE-AIM categories are:

  • Reach into the target population. (Who will benefit from the intervention?)
  • Effectiveness or efficacy. (How favorably will the intervention perform in practice?)
  • Adoption by target settings, institutions, and staff. (How efficiently will the intervention be incorporated into healthcare providers’ workflow?)
  • Implementation consistency and cost. (How will the intervention be delivered and received?)
  • Maintenance of intervention effects in individuals and settings over time. (Does the intervention produce desirable outcomes? How can this be sustained?

Ten years after FDAAA, REMS requirements and best practices continue to evolve, and the FDA is currently drafting new guidance on the assessment of REMS programs. Be sure to keep in touch to learn more as the guidance becomes finalized.


Like all risk management solutions, “one size fits all” does not apply.  Contact us to learn more about our services and to help you meet any requirements that may result from the new guidance.

1. Evaluating the public health impact of health promotion interventions: the RE-AIM framework. Glasgow, Am. J. Public Health, Sept. 1999, Vol. 89, No. 9.