duke-margolisIn December, after the American Society of Hematology (ASH) meeting in Orlando, Florida, I wrote a blog on Patient Power called “Impatient Patients”. I highlighted four patients who aren’t sitting around, waiting to be told about the next best treatment for their blood cancers. Instead, they are mobilizing networks of patients to push for more effective medicines, urging participation in clinical trials, and raising money to direct research beyond the efforts of pharma and government.

On September 7th, I had the opportunity to deliver the message about “impatient” patients to a room filled with clinicians, statisticians and FDA experts. The general subject was “MRD (minimal residual disease) as a surrogate end point in hematologic cancer.” I represented Patient Power on the CLL panel headed by CLL Specialist Dr. John Byrd of the Ohio State University. The newly formed Washington, DC-based Duke-Margolis Center for Health Policy sponsored and organized the discussion.

At issue is whether a minimal amount of detectable CLL can be considered a therapeutic end point for treatment. In other words, does 21st century technology allow MRD to be an acceptable measure of treatment success for CLL patients?

You gather a room filled with docs, data crunchers, and policy experts and – you guessed it—there’s no consensus yet on how to proceed. Wisely, caution remains a watch word, especially where patient safety is concerned. However, the question hovering over the discussion: Is the FDA being too cautious in its regulatory approach to drug development and treatment? Can it take advantage of molecular blood testing to refine its procedures and reach conclusions more quickly and accurately about new meds?

carol-dukeSkeptics of MRD as a therapeutic end point recognize that this is not your father’s technology or your father’s patients. We patients want therapies made available to allow us to live full lives, even if that means the drug won’t cure us. I’m almost 67 years old. Do I need a cure or will an MRD measure provide the treatment I need to enjoy the next 15 to 20 years? Many of us cannot afford to wait another 10 years for development of the next best drug or for the results of clinical trials, which attract only 3 to 5 percent of all patients. A serious, thoughtful discussion is underway. But with no consensus, your voice is needed to move the needle on what comes next.

If you are interested in the replay of Sept. 7th forum, you’ll find it on the Duke-Margolis YouTube Channel. By mid-month, you should be able to access the individual sessions, which include multiple myeloma (MM), acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) along with CLL.

Wishing you good health and great questions,

Carol Preston