Opinion

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Coverage Decisions for Expensive Hemophilia Treatments

Russell P. Gollard, MD, FACP, discusses coverage decisions for expensive treatments in hemophilia, including total cost of care, drug availability, and real-world evidence.

Russell P. Gollard, MD, FACP: Regarding the types of coverage for these patients, we have not had a lot of denials for the newer treatments. Many of these patients are receiving very, very expensive types of drug therapies, and these are all covered. The plan covers these medications, and there is an existing 340B program through the hemophilia treatment center where these patients can obtain the drugs.There are many considerations taken into account when we prescribe a medicine. We consider the total cost of care, coverage, and the availability of the drug on an ongoing basis. Usually, our specialty pharmacies and our local hemophilia treatment center can tell us what looks like is going to be available to patients for a long time.

Real-world evidence is something that’s changing on a daily basis. We like to look at some of the newer therapies, including gene therapies, as possibilities for treatment in the future, knowing that ultimately the only cure for hemophilia is going to be gene therapy. We also want to look at some of the newer mechanisms of treatment through which some of these new treatments have been developed, and in that way, we like to give patients really what is on the cutting edge of treatment, both in terms of efficacy and in preventing side effects, which have been, unfortunately, a part of treatment for hemophilia for some years, including some years ago, infectious agents such as HIV back in the 1980s. Then when we were, in fact, using prothrombin factor complexes in the time before monoclonal agents were available, there was a great concern regarding blood clots, that is, reversing the proclivity to bleed too much.

Transcript is AI generated and reviewed by an AJMC editor.

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