Without a strict set of safety guidelines in place, some much-needed therapies would not be available for patients who need them most.
Currently, 66 drugs on the market require a Ensured Patient Safety With REMS Expertise to ensure that the benefits of a drug or biologic outweigh its risks. Approximately half of those drugs with a REMS include Elements to Assure Safe Use (ETASU), which ensure additional safety measures are in place before patients receive their medication.
The Need for REMS
With the growing complexity of new medications, the Food and Drug Administration has increasingly required manufacturers to institute REMS programs. REMS may be put in place for a new medication, or a new safety issue might be identified in an already-marketed drug, causing the FDA to impose a REMS requirement on an older product. As pharmaceutical companies focus on narrower patient populations for specific drugs and biologics, the need for REMS programs to monitor the real-world effect of these new medications is greater than ever before.
REMS requirements may include medication guides, easy-to-understand pamphlets for patients with information about serious side effects, or communication plans that involve notifying and educating healthcare professionals about a drug’s safety information. In addition, REMS with ETASU may require doctor and pharmacy training and certification; patient monitoring to ensure safe use conditions while the medication is being used; or patient enrollment and long-term follow-up as long as the patient remains on the medication.
United BioSource Corporation helps pharmaceutical manufacturers design, implement and evaluate REMS programs. While the Food and Drug Administration Amendments Act (FDAAA) of 2007 gave the FDA the authority to require REMS, UBC’s influence on drug safety goes back much further.
Prior to REMS, a small number of pharmaceutical products included Risk Minimization Action Plans (RiskMAPs) that were put in place to enhance appropriate and effective use of drugs and medical devices, and to prevent adverse events. UBC has worked with many manufacturers during the transition from RiskMAPs to REMS.
Before RiskMAPs were in place, the industry used performance-linked access systems (PLAS) to ensure necessary restrictions were in place for the safety of patients. UBC developed one of the first PLAS instituted in 1999. That program, for a drug that treats patients with schizophrenia, is still in effect today.
Through many industry changes, UBC continues to work with all stakeholders — patients, pharmacies and prescribers — to ensure patients receive safe and timely access to the therapies they need.
Proactive Versus Reactive Approach
In the early days of REMS, the process was largely reactive and only considered after the FDA’s feedback and request. Today, however, REMS have become an established part of the pharmaceutical landscape, and manufacturers are developing proactive strategies knowing that bringing a product to market may require a REMS.
At times, some manufacturers have been concerned that REMS programs restrict access to the very products they’ve spent much time and resources developing. In addition, they are concerned patients and prescribers might presume that a product is dangerous, reducing a product’s marketability. These are valid concerns, and that’s why a proactive plan — early in the drug development process — will help minimize obstacles to drug approval and integration into the healthcare system.
The FDA is working to establish rules or direct guidance to provide insight into the circumstances under which they might impose a REMS. Some of the factors that influence their decision-making process include:
- Estimated size of the patient population
- Seriousness of disease or condition
- Expected benefit of the drug
- Expected duration of treatment
- Seriousness of known or potential adverse events
- Whether the drug is a new chemical entity
The Best Indicator of Future REMS Is Past REMS
Insights into the kind of REMS program, if any, that may be required can most likely be found by looking at approved REMS in the marketplace. Whether a drug is in the approval process or already on the market, the FDA often relies on precedents when evaluating REMS requirements.
That’s why UBC’s experience has played a significant role in the REMS industry. UBC teams, made up of epidemiologists, safety scientists, data analysts, software developers and patient educators, to name a few, have designed, implemented and evaluated more than 100 REMS programs for drugs to treat everything from diabetes to organ transplant rejection.
Working with many stakeholders and patient populations across a wide variety of therapeutic areas has taught us many valuable lessons about REMS programs and drug safety, including:
Drug safety starts early: Proactive risk management should begin early in the drug development process, allowing for opportunities to:
- Begin compiling the safety profile of a drug.
Identify and quantify potential unique benefits of a drug, which ultimately may keep it on the market to treat patients who need it.
Understand how the clinical trial population may differ from the post-approval target population that will include patients on multiple concomitant medications and patients in special demographic groups, such as the elderly or women of reproductive potential.
Communication is key: Determining which risks may impact the benefit-risk balance of a product requires input from many different groups within a pharmaceutical company as well as from regulatory agencies, patient advocacy groups and healthcare providers.
Patients matter most: Appropriate safety guidelines are needed. However, if requirements are too complicated and cumbersome for patients and their prescribers to follow, it’s possible another drug or course of therapy may be chosen. This can result in unintended consequences if the alternative treatment also has risks.
The ultimate goal of a successful REMS program is to get the right therapy to the right patients at the right time.
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