When Rusfertide May Become Accessible for Those with Polycythemia Vera

Dr. Aaron Gerds sat down for an interview with CURE to explain which factors will determine if treatment with rusfertide will become an accessible treatment option for patients with a myeloproliferative neoplasm like polycythemia vera. This conversation came following a presentation of data from the phase 3 VERIFY trial.

Overall, investigators shared that the investigational agent was able to reduce or eliminate the need for phlebotomies (a procedure which removes extra blood cells from the body by using a needle to take blood from the vein), according to Gerds, which would improve the quality of life of patients and help with symptom control.

In the interview with CURE, Gerds, who is an assistant professor of medicine at Cleveland Clinic Taussig Cancer Institute, also highlighted what patients should know about the VERIFY trial, as well as how rusfertide affects patient symptoms.

Transcript

What factors will determine whether rusfertide becomes a widely accessible option for patients, and how can individuals advocate for access to emerging treatments like this one?

That will certainly take more time. These are just the initial, top-line results — the week 32 data. The week 52 data is expected [this year (2025)], so you can mark that on your calendars. I believe the longer-term data will be helpful in answering those questions.

The company that makes the drug is certainly in conversations with regulatory authorities, asking, “What else is needed to have a good chance of approval to submit that new drug application?” That's going to be the next step: gathering all the data, the entire packet, and putting together a new drug application to submit to the regulatory authorities, or the U.S. Food and Drug Administration (FDA) in this case. Then, that packet or application, if you will, gets reviewed by the FDA. There's a number of reviews, hearings, and all kinds of processes that go along with that. At the back end of that, that's when we'll have a decision on regulatory approval.

Regulatory approval is just that next step. When you say, “access and widely available,” the step after that is insurance coverage. Here in the United States, our healthcare system is often run by insurance companies. What will that regulatory approval look like? What will it include? What levels of disease and what parameters? Then there will be guidelines that come out, for example, from the NCCN, which will make guideline recommendations on the use of rusfertide.

These two factors will be taken together, along with pricing and other factors. Then the insurance companies will decide what they will and won't cover, and that may actually impact access for a lot of patients.

Reference

1. “Protagonist and Takeda announce positive topline results from phase 3 VERIFY study of rusfertide in patients with polycythemia vera,” by Protagonist Therapeutics and Takeda. News release.

Transcript has been edited for clarity and conciseness.

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