Hemophilia Care From the Payer Perspective

Hemophilia is a costly and complex disease, and while there has been “incredible growth” in treatment options—48 products are currently on the market—the increased product choice is not driving down costs, said Sam Leo, PharmD, director of specialty clinical programs at Magellan Rx Management in New York.

Dr. Leo and Gary Tereso, PharmD, a senior clinical pharmacist at Health New England in Massachusetts, discussed the roles of various stakeholders in hemophilia care, as well as spending considerations from a payer perspective at the Specialty Connect event yesterday.

The average cost of care for a patient with hemophilia is $300,000 per-member, per-year, with factor replacement products representing up to 94% of total costs for patients with severe disease. The rarity and genetic nature of hemophilia, as well as the wide range in cost per patient, can lead to substantial variation in the number of patients and total hemophilia-related costs for health plans.

More and more agents in the pipeline are highly complex, including more extended half-life agents and non-replacement therapies, as well as gene therapy. There are a lot of unanswered questions with these products, including “How will we afford this?” said Dr. Leo. It is essential for plans to understand their populations to evaluate how hemophilia is being managed, he added.

Management challenges, meanwhile, include fragmentation of care and lack of uniformity, limited transparency, robust and complex product selection, lack of individualized treatment, potential stockpiling and product waste, pharmacy and medical benefit utilization, and navigating the need for reinsurance programs for high-cost members.

Among stakeholders, payers have little insight into clinical data outside of factor product cost, and providers may have limited insight into product utilization, dispensed amounts, and total healthcare resource utilization. “We need to promote proactive communication between stakeholders to optimize patient care,” Dr. Leo said.

Pharmacokinetic (PK)-guided dosing and assay management may result in a better patient-tailored approach, he noted. PK-guided dosing can provide better and more accurate targeting of factor levels, better ability to adapt dosing to lifestyle, and a potential reduction in cost and units utilized without compromising efficacy. However, doing this is time-intensive for both patients and providers, and many facilities do not have the capability to conduct PK testing. Medical and Scientific Advisory Council guidelines recommend dispensing of factor products to be within plus or minus 10% of prescribed assays, and many specialty pharmacies will “guarantee” assay management within narrower ranges, but it is difficult for payers to measure and monitor.

Dr. Tesaro then discussed management strategies. First, utilization management ensures specific standards of care, improves transparency and data collection (e.g., bleed history, reason for treatment, product utilized), and improves care coordination. Pharmacy management should include standardized assay and inventory management, bleed history tracking, no auto-refills, standards of care for patient engagement and clinical contacts, and adherence monitoring and counseling. Product preferencing can also be used, but he noted that this concept is not intended for patients already on a certain therapy, but for those with “new starts.” Data collection and analysis is also very important to determine benchmarks and measure relevant outcomes.

Dr. Tesaro’s organization rolled out a hemophilia program in July 2017 that added a prior authorization to the medical benefit. This included PK testing for high-risk patients and dispensing pharmacies were required to manage assays to a standardized range and to report monthly drug inventory and bleed history. After six months, 50% of patients in the population had an average reduction in dose either through assay management or PK testing.

Presentation: Managing Patients with Hemophilia: A Payer’s Perspective. AMCP Annual Meeting 2018 Specialty Connect.