Dr Robert Rifkin Explains the Importance of Real-world Data in MM Patient Populations

Phase 3 clinical trials are very expensive, and using real-world evidence to aid with trial design and increase representation can help benefit patients with multiple myeloma, said Robert Rifkin, MD, FACP, a medical oncologist and hematologist at Rocky Mountain Cancer Centers.

Transcript:

Can you introduce yourself and describe the work you do?

I'm Robert Rifkin, MD, FACP. I'm a medical oncologist at Rocky Mountain Cancer Centers in Denver, Colorado. My areas of interest include multiple myeloma, with a special attention to large disease-specific registries and the development of new drugs. In addition, I have a significant interest in biosimilars with a goal to increase access and decrease cost, and advocate strongly for their introduction to the United States market.

Why did you decide to research real-world treatment patterns and burdens in triple-class refractory multiple myeloma?

I think this sort of abstract really paves the way for what's coming in the future. As you review large phase 3 clinical trials, what everybody knows is that the entry criteria are quite specific and restrictive. As a result, you get a pretty highly selected patient population. By exploring disease-specific registries such as Connect MM, we get a real-world evidence look. So if you look at this particular registry, it's been in existence now for almost a decade and accrued somewhere around 3000 patients. That allows us a very unique opportunity to do all kinds of exploratory things.

We know for example, that less than 50% of our patients would have qualified for an ongoing clinical trial, so we're clearly missing a significant portion of multiple myeloma patients. As the readers will know, the FDA has just issued new guidance for real-world evidence research for manufacturers and others to use. It's now very, very expensive to do phase 3 randomized clinical trials, and if you can start to use real-world evidence to help us with trial design and make things more representative, I think it'll greatly improve the care of myeloma patients.

In this particular exploration, what we're now finding is with the introduction of novel agents, myeloma patients are surviving for a lot longer. When I started my career as a fellow, the median survival was one-and-a-half years. And if you now look at current data, it's 7 to 10 years. So we've made a lot of advances. There's a lot of new drug classes. And in particular, when patients are exposed to the IMiDs [immunomodulatory drugs] proteasome inhibitors, and monoclonal antibodies, we're creating a triple-class refractory population that we really need to look at.

These are patients in later stages of disease. As they become sicker, and perhaps responses are less durable, there's an increasing burden upon the patients and their caregivers. In addition, there's also an economic burden. In the Medicare era, we're all doing the oncology care model. We're now scored on cost per episodes of care and not so much on the old fee-for-service model. So it's very important for us to make things efficient and keep people out of our overcrowded hospitals and emergency rooms, and deliver really good care to our triple-class refractory patients.