In a recent development, experts in oncology have endorsed a new measure to hasten the approval process for drugs targeting multiple myeloma. A panel of FDA advisers, consisting of 12 members, unanimously voted in favour of using minimal residual disease (MRD) as a surrogate endpoint for accelerated approvals of therapies in multiple myeloma.

The decision follows extensive discussions on the potential benefits of employing MRD as a marker for treatment efficacy. MRD, which involves detecting minimal traces of cancer cells in patients' bodies after treatment, is believed to offer insights into the long-term effectiveness of therapies.

The endorsement is based on compelling evidence from two separate meta-analyses presented during the meeting. These analyses demonstrated a strong correlation between MRD negativity post-treatment and prolonged progression-free survival in myeloma patients.

Unlike traditional clinical endpoints like progression-free survival or overall survival, MRD assessment provides quicker results, allowing for expedited drug approvals. This approach is particularly crucial given the challenges of assessing treatment outcomes with existing measures due to the high efficacy of current therapies.

While the panel acknowledged some uncertainties surrounding the use of MRD as a surrogate endpoint, including varying trial-level associations, they emphasised the importance of considering additional endpoints during the approval process.

One concern raised during the meeting was the potential impact of prioritizing MRD as an endpoint on drug development strategies. There's a risk that focusing solely on MRD could lead to the premature abandonment of potentially effective treatments that do not show immediate MRD signals.

To address these concerns, the FDA encouraged biopharmaceutical companies to adopt a "single trial" approach, which involves utilising the same study to support both accelerated and full approvals. This approach ensures continued evaluation of treatment efficacy beyond intermediate endpoints like MRD.

Despite the uncertainties, the decision to support MRD as a surrogate endpoint marks a significant step towards expediting the introduction of new therapies for multiple myeloma patients. Biopharmaceutical companies, such as Sanofi, are poised to benefit from this decision, with ongoing trials demonstrating promising results using MRD as a primary endpoint.

As the FDA moves forward with integrating MRD into regulatory practices, the focus remains on ensuring patient safety while accelerating access to innovative treatments for multiple myeloma.