Cost Analysis on High-Profile Specialty Medicines Awaiting FDA Decision

Pipeline presentations are always well attended at AMCP meetings, and none draw more attendees than the sessions addressing the specialty drug sector. Two pharmacists from Prime Therapeutics—Nicole Kjesbo, PharmD, BCPS, clinical program development director, and Catherine Starner, PharmD, BCPS, senior principal health outcomes researcher—delivered the latest update and cost analyses on some high-profile specialty medications at AMCP 2021.

One of the key medications facing an FDA decision is Biogen’s aducanumab, which has a PDUFA date of June 7, 2021. This monoclonal antibody attacks amyloid-beta in patients with Alzheimer’s disease, and it has been heavily scrutinized. In November 2020, an FDA Advisory Committee voted that clinical trials did not provide strong evidence of effectiveness of aducanumab for the treatment of Alzheimer’s disease, according to the presenters. Its estimated cost is $40,000 per year, and they based their forecasting on uptake by 20% to 40% of patients with Alzheimer’s disease. This yielded a commercial per-member per-month (PMPM) cost of up to $0.30. For Medicare coverage, the PMPM cost could be as high as $14.89.

Another important medication mentioned was roxadustat, the first oral medication for the treatment of anemia in patients with chronic kidney disease. Produced by partners AstraZeneca and FibroGen, this drug is expected to receive an FDA decision in the third quarter of 2021.

“A small percentage of patients with non–dialysis dependent anemia is considered treatable with an erythropoietin-stimulating agent,” said the presenters, adding that this product may fit an important niche. In clinical trials, roxadustat had a robust effect on hemoglobin levels in this patient subset but without any noticeable effect on major adverse cardiovascular events. Hemoglobin levels were increased by much lower amounts in patients with dialysis-dependent kidney disease. An ICER Evidence Report concluded that roxadustat would likely have a net clinical benefit over placebo for patients with non-dialysis-dependent kidney disease but not necessarily for the dialysis-dependent variety. The presenters calculated a PMPM cost of $0.17 if the drug was priced at $8,000 per year.

Avacopan, a product by ChemoCentryx to treat the rare disease antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis, is a C5a receptor inhibitor, and oral avacopan may be an add-on treatment to immunosuppressants. The incidence of ANCA-associated vasculitis, according to the presenters, is about 4,000 new cases each year in the U.S. In clinical trials, avacopan attained sustained remission at 52 weeks in 66% of patients versus 55% of those taking prednisone-based standard of care. Patients taking avacopan had lower glucotoxicity index scores (calculated as least-squares means) compared with prednisone treatment. Based on presumed uptake of up to 40% of patients with ANCA-associated vasculitis, PMPM costs were calculated as much as $0.16, or as low as $0.03, depending on which diagnosis codes were included in the patient population.

Kjesbo N, Starner C. Specialty Pipeline and Forecasts 2021. Presentation K12. Presented at AMCP 2021; April 12-16, 2021.