FDA Accepts A New Endpoint

On The News Front – The FDA now will accept Metastasis-Free Survival (MFS) as a clinical trial endpoint for hormone sensitive cancer.

It’s good news for anyone with any type hormone sensitive metastatic cancer and its particularly great news for men with metastatic prostate cancer (Advanced Prostate Cancer).  Finally, it’s official; the FDA formally recognizes metastasis-free survival (MFS) as a valid clinical trial endpoint, even for men who are still hormone sensitive.  Given the extended survival times that many men who have advanced prostate cancer experience, this policy change will speed up the drug approval process for new prostate cancer treatments.  

The formal disclosure was written in a “perspective” piece written by FDA officials for

The New England Journal of Medicine.  The article described the characteristics of the endpoint, how it was developed, and how it was being used.

The piece was written by FDA Oncology Center of Excellence officials Julia Beaver, Paul Kluetz, and Richard Pazdur and published in the June 28 edition of NEJM.

“Whereas radiographic progression-free survival has been standardized as an endpoint in metastatic settings, there was previously no earlier endpoint for nmCRPC trials,” the article states. “The FDA has now recognized that a prolonged delay in development of metastatic disease is an objective and clinically relevant measure. Future agents may be approved on the basis of metastasis-free survival only if substantial effects on this transition are demonstrated and the safety profile is acceptable for a medication taken long-term.”

The article noted that earlier this year, on February 14, 2018, the agency approved a prostate cancer drug—apalutamide (Erleanda) (Janssen Pharmaceutical), an androgen receptor inhibitor—based on MFS.  Erleada was approved by the FDA for use with men with non-metastatic castration-resistant prostate cancer (nmCRPC). 

For many years many of us advocates have been complaining that the FDA needs to become more flexible in the drug approval process, especially in cancers like metastatic prostate cancer where the time to death can often be protracted causing protracted delays in drug approvals.  This significant policy change, recognizing the validity of delaying the onset of the development of metastatic disease, might not have any effect on survival, but it will have an impact on our quality of life (QoL).   

Hopefully, the next step will include the FDA considering patient-reported outcomes as an additional trial endpoint.

Joel T. Nowak, MA, MSW wrote this Post.  Joel is the CEO/Executive Director of Cancer ABCs. He is a Cancer Thriver diagnosed with five primary cancers - Thyroid, Metastatic Prostate, Renal, Melanoma, and the rare cancer Appendiceal cancer.