(20) Trends in commercial payer coverage of accelerated approval therapies

Background: : The FDA’s Accelerated Approval (AA) Pathway allows for expedited approval of therapies for serious conditions based on surrogate endpoints. Payers face challenges balancing timely access with managing high-cost therapies that often lack comprehensive data. Coverage decisions are complex, especially when alternatives are limited.

Objective: : This study explores how accelerated approval drugs are covered by commercial payers.

Methods: : We identified all ongoing AA indications (excluding vaccines and hospital-only drugs) from the FDA AA list as of January 1, 2025. Orphan Drug Designation (ODD) was assessed via the FDA ODD database. We reviewed 80 non-oncology (N-O) and 156 oncology (O) policies from 4 of the largest U.S. commercial payers between March 1 and May 27, 2025.

Results: : Among AA indications, 83% had an ODD. Commercial coverage varied: some policies aligned with FDA labeling, while others were more restrictive or excluded coverage.
In N-O:
• 82.5% (n=66) required prior authorization (PA)
• 2.5% (n=2) of policies considered the drug investigational
• 15% (n=12) lacked a PA policy
Of those requiring PA, 45% aligned with FDA labeling, 36% were more restrictive than the FDA label, and 18% included step therapy (ST). Of the 12 without policies, 4 were not publicly available, 4 covered the drug with no utilization management (UM), and 4 were non-formulary with no PA.
In O:
• 70% (n=109) applied PA [40% (n=63) aligned with FDA labeling; 26% (n=40) required additional criteria; 4% (n=6) required ST]
• 30% (n=47) lacked policies; 5 of those were non-formulary with no PA.
Initial and renewal durations also varied. N-O drugs were more likely to have shorter approval durations: 25% had initial approvals ≤6 months and 11% had renewals ≤6 months. No O policies had durations ≤6 months.

Conclusions: : Most AA indications carry an ODD, underscoring the challenge of balancing access with evidence uncertainty. While most AA therapies are covered and have UM, gaps in policy transparency remain. Some plans may use exceptions or appeals to manage access. Many align with FDA labeling, but others apply more restrictions that may prevent or delay access to care. Furthermore, N-O drugs are more likely to face shorter approval periods, which may increase administrative burden and delay care.

Sponsorship:: None