US Specialty Drug Market Access

High-touch specialty drugs for complex, chronic, high-cost, or rare diseases are an increasingly significant component of the US pharmaceutical market. A sharp growth in regulatory approvals accompanied by high prices has driven rapid increases in expenditure on specialty therapies by both public and private payers.

These factors, coupled with subsequent price rises and strong uptake of some specialty drugs, have prompted payers to adopt stringent cost- and utilization-management strategies in categories such as oncology, multiple sclerosis, rheumatoid arthritis, and hepatitis C.

The specialty drugs trend has also brought new players, such as specialty pharmacies, distributors, and drug hubs, into the pharmaceutical value chain to address unique challenges encountered in the distribution, administration, and reimbursement of specialty products. These challenges include restricted distribution channels that provide specialized services such as fulfillment of Risk Evaluation and Mitigation Strategies or patient support programs, as well as closed networks of specialty pharmacies to drive cost efficiencies, consistency of care, and optimal clinical outcomes. Some payers are also “carving out” specialty pharmacy benefits to focus attention on issues such as patient support, drug adherence, or the generation of outcomes data.

While payers are becoming more adept at managing the costs of specialty drugs reimbursed under medical benefits, a shift from medical to pharmacy coverage, or to integrated specialty drug coverage, may offer more clarity and options to control utilization and costs. In particular, these include prior authorization requirements, step edits, quantity restrictions, diagnostic tests, tiered formularies with high cost-sharing for specialty drugs, discounts, and rebates secured from manufacturers or pharmacists. Some payers also encourage the use of lower-cost sites of care for provider-administered specialty drugs.

These strategies are increasingly likely to be supplemented with innovative cost-management techniques such as risk-sharing and performance-based pricing or reimbursement, with an emphasis on bundled services and outcomes. The emerging US biosimilars market will present significant opportunities to lower the costs of premium-priced specialty biologics, even with relatively modest discounts against the originator brands. Patient-centric services will remain an important part of the equation, although manufacturer support programs to offset the often substantial cost-sharing for specialty drugs will continue to be viewed in some quarters as a pretext to circumvent formulary restrictions and maintain premium prices.