Results From Outcomes-Based Agreement Analysis in Patients With Multiple Sclerosis

Outcomes-based agreements, also called value-based contracts, are agreements made between pharmaceutical manufacturers and payers in which payments received for medications and treatments are contingent upon patient outcomes. Such arrangements allow both parties to share risks associated with the costs of the medications. As healthcare entities shift from volume-based to value-based care for patients, outcomes-based agreements are gaining some traction in the U.S.

These contracts aim not only to improve patient access and outcomes but also to reduce healthcare utilization costs and improve patient-reported outcomes. It is not surprising that multiple sclerosis (MS) is a prime therapeutic area for adopting outcomes-based agreements. Close to 1 million people in the U.S. are living with MS. Disease-modifying therapies, which have been shown to reduce relapses in a large number of patients with MS, rate among the top 10 costliest medication classes, with annual expenditures adding up to almost $19 billion.

According to an analysis published in the November 2024 issue of the Journal of Managed Care and Specialty Pharmacy, there are 17 known MS outcomes-based agreements. These mainly focus on outcomes such as MS-related emergency department visits, medication adherence, hospitalizations, and relapse rates, which can be extracted from insurance claims and electronic health records.

Elizabeth Swart, M.P.H.

However, in a previously conducted survey, a research team led by Elizabeth C.S. Swart, M.P.H., from the University of Pittsburgh Medical Center for High-Value Health Care, 100% of participants agreed that “worsening physical disability’ is the outcome most meaningful to patients with MS. Patient-reported disability is also commonly used in randomized clinical trials to gauge disability status in patients with MS.

Using this information, Swart and her colleagues chose patient-reported disability as a primary outcome in their prospective real-world analysis of an outcomes-based agreement for patients with MS using interferon β-1a (sold under the brand names Avonex and Rebif) or dimethyl fumarate (sold under teh brand name Tecfidera). Interferon β-1a and dimethyl fumarate are both disease-modifying therapies FDA-approved to treat MS.

The analysis included 184 patients, of whom 107 took dimethyl fumarate and 77 used interferon β-1a. Using the patient-determine disease steps (PDDS) scale, the researchers obtained a baseline disability score and follow-up scores between 90 and 180 days after baseline. The PDDS is a surrogate of the commonly used Expanded Disability Status Scale. Scores range from 0 to 8, where 0 indicates no disability, 1 is mild disability, 2 corresponds to moderate disability, 3 indicates gait disability, 4 equals early cane use, 5 is late cane use, 6 corresponds to bilateral support needed, 7 equals use of a wheelchair or scooter, and 8 means bedridden.

The mean baseline PDDS score for patients taking dimethyl fumarate was 0.95. The mean score for patients in the interferon group was 0.86. Upon completion of the contract period, 11.4% of participants had confirmed disability progression. Of these, 5.6% were in the dimethyl fumarate group, and 19.5% were in the interferon group.

Swart and her colleagues concluded that patient-reported outcomes, such as disability progression, are a viable marker to use with MS outcomes-based agreements.