Dr Ruben Mesa Discusses Balancing Treatment Access, Cost-Effectiveness for Patients With Rare Cancers

It is important that practices provide patients with a treatment structure that really helps support an individual with any type of cancer and meets their unique needs, says Ruben A. Mesa, MD, FACP, president and executive director of Atrium Health Levine Cancer Institute (LCI) and Atrium Health Wake Forest Baptist Comprehensive Cancer Center.

This transcript has been lightly edited for clarity.

Transcript

How can health care systems like Atrium Health effectively balance the treatment of rare diseases, such as myeloproliferative neoplasms, with more common types of cancer in their value-based care approaches?

As we learn more about precision cancer care—in many ways, every cancer is rare these days as we really look at the differences between patients, the different molecular mutations, different presentations, and issues regarding each individual patient—I think it starts with us really having a structure that helps to support the individual patient and their needs.

The prior days when we treated everyone with breast cancer the same as everyone with lung cancer; those days are long gone. So, I think as we have both the ability to support the full range of things in terms of being able to support clinical trials—more clinical trials, and more nimbly for smaller groups of patients—the ability to identify how patients are different and be able to support them is really crucial.

Given your extensive experience in developing FDA-approved drugs for chronic leukemias, what strategies do you believe are essential to ensure access to treatments for patients with rare cancers while remaining cost-effective?

One of the main drivers of approval for the FDA these days is really the clinical benefit to the patient. Drugs clearly have to have good data in terms of safety and effectiveness, as well as a strong rationale for their use in terms of mechanism of action and impact on the disease. FDA clearly looks at what is the overall benefit for the patient when one weighs risk and benefit.

And to some degree, I think as we look at the final piece, clinical benefit also includes the ability to access a medication. So, I think increasingly, that becomes an important piece—perhaps not the FDA regulatory piece, but broader in terms of our health care system piece. We can't have a great therapy that but then not have the patients who might benefit be able to receive that therapy. So that's another critical piece.

It clearly takes partnership between both the manufacturers of these medications and the many philanthropic groups around the country that make an important impact. I've been involved as a leader at the Leukemia Lymphoma Society, where they have worked hard to maintain access to critical therapies for patients with hematologic malignancies.