Panel Perspectives on Hemophilia Management

To conclude the Specialty Connect, a panel was convened to discuss various perspectives on the current climate of hemophilia care and projections for the future. Previous speakers Steven W. Pipe, MD, of the University of Michigan in Ann Arbor, Gary Tereso, PharmD, a senior clinical pharmacist at Health New England in Massachusetts, and Susan Trieu, PharmD, director of enterprise specialty clinical solutions at MedImpact Healthcare Systems, Inc., in Texas, were joined by Michelle Rice, senior vice president of external affairs at the National Hemophilia Foundation in Indiana. Ms. Rice has two children with hemophilia, and she spoke as a representative of the patient perspective.

Ms. Rice began by discussing the hemophilia patient community, which is very active and engaged with their care and treatment. “As a patient community, we know what we cost,” she said, noting that many—including herself—want and try to help payers save money, “although there are barriers to that,” she said. She argued for patient “access,” noting that the use of the words “patient choice” conveys an entitlement. It is important that payers create metrics and tools for costs, which can include inventory and assay management, methods for adherence, and data sharing. “This is something we all need to work on together,” she said.

The discussion turned to patient care, and Ms. Rice said she advises patients to know what is prescribed and track what they receive each month. “If you always see an overage [in the dose], call your specialty pharmacy provider and ask, ‘Can we get this closer to the dosage,’” she said. “Be the steward of your own healthcare.

On the topic of received dosage, Dr. Pipe reminded, “We are talking about biologic agents where there is variability.” He said while there is not a need for 20 different factor VIII (FVIII) products, “I don’t think one [preferred agent] per product category is enough,” he said. As long as access to treatment is not impeded, the preferred agents and formulary tiers are not as big an issue as it is made out to be, he said, noting a need for a pathway to the right product.

Turning to gene therapy, Dr. Pipe said that an important part of the gene therapy clinical trial data that are being published is the product pool from different serotypes. “We want to choose gene therapies appropriately because patients in these clinical trials will not [currently be eligible for] retesting of other gene therapies,” he said.

“It’s an exciting time for hemophilia community,” with regard to new treatment options, said Ms. Rice, although she noted that the hemophilia community will not just jump into the “new, shiny” therapy. “Don’t assume that everyone who’s eligible for gene therapy will go for it,” she advised. She noted that patients will likely have concerns that switching to a new treatment will lead to the development of an inhibitor—which is something that “hangs over the [hemophilia] patient’s head,” she said. “[The development of] inhibitors are greatest fear” in this patient population, she said.

With that, Dr. Pipe noted that the vast majority of inhibitors occur within first 10 exposures to FVIII, and these patients tend to present themselves fairly quickly. He said refractory inhibitors is an area where innovative treatment options are needed. He said emicizumab was an agent that helped this patient population significantly. “I believe gene therapy will have something to offer for patients with inhibitors,” he said, “but not in the first wave.”

Dr. Trieu pointed to some important factors of hemophilia care, including assay management, auto-refills on treatments, and patient education. With gene therapy she said, “We are potentially not going to [see] inhibitors, and patients may not need to be logged for every bleed and target joint. These are things payers [should] think about.”

Presentation: Perspectives in Hemophilia Management. AMCP Annual Meeting 2018 Specialty Connect.