The FDA is set to expedite drug approvals for multiple myeloma following a committee decision to allow minimal residual disease (MRD) as a clinical trial endpoint. This decision, influenced by research led by Dr. C. Ola Landgren at the Sylvester Myeloma Research Institute, could reduce drug development timelines by up to a decade.
The FDA committee's ruling, based on a 12-0 vote in April 2024, permits the use of MRD, which detects minute numbers of cancer cells, to support accelerated drug approval in multiple myeloma. The research demonstrated a correlation between MRD negativity and clinical outcomes such as overall survival and progression-free survival.
Impact on Drug Development
According to Dr. Landgren, the adoption of MRD as an endpoint will significantly accelerate drug approval timelines. "Drug approval will now happen much faster for multiple myeloma," he stated. This expedited process could potentially save numerous patient lives by bringing effective treatments to market sooner.
The EVIDENCE Meta-Analysis
Dr. Landgren and his colleagues initiated a study over 10 years ago to assess MRD as a potential alternative endpoint. This effort culminated in the EVIDENCE meta-analysis, which stands for "evaluating minimal residual disease as an intermediate clinical endpoint for multiple myeloma." The study analyzed data from over 12 clinical trials and more than 6,000 patients.
The EVIDENCE study, along with a separate meta-analysis, convinced the FDA committee to accept MRD as an endpoint for accelerated approval, an expedited pathway for treatments for serious diseases.
Challenges and Future Directions
While MRD testing is a powerful tool, challenges remain in standardizing methods across trials. Dr. Landgren advises researchers in other cancer fields to follow a rigorous process to gain FDA approval for MRD as an endpoint. This includes ensuring the timing is right for the disease, working closely with regulatory authorities, and gathering data from all key sources.
Key Considerations for Researchers
Dr. Landgren offers three key pieces of advice for researchers aiming to use MRD as an endpoint:
- Timing is crucial: Existing therapies must be effective enough to yield deep clinical responses.
- Collaboration with regulators: Understand FDA guidelines and develop a statistical plan in consultation with the agency.
- Comprehensive data collection: Gain the trust of biopharma companies to access their proprietary data.
With the FDA's decision, drug companies are already modifying their protocols to incorporate the MRD endpoint, and there is increased incentive to develop improved MRD measurement technologies. "There are many new drugs on the horizon for multiple myeloma. It's an exciting time," Dr. Landgren noted.
