REMS Assessments are now the hot topic in REMS. We’ve reached the 18-month mark and it’s not a coincidence that Draft Guidance includes a section on assessments and modifications. Companies need to begin to contemplate not just how to analyze and interpret their assessment findings, but they need to think about the best way to actually evaluate their REMS performance.
Of the total approved REMS (117 to date), performance assessments are the one predictable FDA requirement. It is the most frequent need of companies. Frequency cannot be less than 18, 36, and 84 months, but FDA often asks for more frequency (e.g., annually, semi-annually). The assessment type is dependent upon elements in the REMS –
- REMS with a MedGuide only require a survey of patient understanding of the serious risks and pharmacist compliance with MedGuide distribution. (The latter measurement is only necessary if the MedGuide is not packaged with a unit of use.)
- REMS with Communications Plans require a survey of healthcare professional understanding.
- REMS with an Element to Assure Safe Use require compliance reporting.
FDA communications about assessments have been very consistent. FDA is thorough in their review of protocols and surveys. The agency has edited survey questions (e.g., adding “True/False” question structures) and has suggested answers (e.g., include “I don’t know”). FDA often takes their full 90-day time allotment to review the protocols and instruments, which may cause a narrower window for recruiting for the first assessment. For that reason, it is recommended that Sponsors include their protocols and surveys with their REMS submission.
Sponsors are focused on meeting the minimal performance assessment requirements and may be missing early opportunities to learn how well their REMS is performing. FDA Draft Guidance encourages more frequent and/or comprehensive assessments. More frequent assessments could possibly include smaller sample sizes versus the larger sample sizes at the standard reporting intervals. More frequent assessments may equate to a continuous quality improvement of REMS.
The Assessment Plan of a REMS informs which of the REMS elements are meeting the specified goals and whether the goals or REMS elements should be modified. The Plan should include evaluation methods for assessing effectiveness, targeted values for each measure and timeframe, types of data to be collected, and a plan to evaluate unintended consequences.
FDA now anticipates upcoming assessments and the potential need for REMS modifications. Sponsors must submit an assessment report according to the agreed upon timetable, but may submit additional assessments at any time – with or without a modification. Proposed modifications may enhance or reduce REMS elements, or add to or modify assessment timetables, including eliminating an assessment after 3 years.
We recommend that Sponsors:
- Start early to develop their REMS assessment protocols and surveys to avoid short reporting intervals – submit these with REMS documentation.
- Consider ongoing, real-time assessment reporting (or more frequent implementation monitoring) to help flag early issues for program enhancements.