Plan Coverage of Orphan, Expedited Drugs Often More Narrow Than Approved Indication

Clinical prerequisites for the coverage of orphan drugs and drugs approved through expedited programs vary substantially across US commercial health plans, according to findings published in the Journal of Managed Care and Specialty Pharmacy. 

Such patient subgroup restrictions “tend to be consistent with eligibility criteria specified in pivotal clinical trials,” wrote researchers from Tufts Medical Center in Boston and Sarepta Therapeutics Inc., Cambridge, Massachusetts. “However, patient subgroup restrictions were inconsistent with the pivotal trial’s eligibility criteria roughly one-fifth of the time, raising questions about how health plans developed those criteria and the potential impact on patients’ ability to access a given therapy.”

Investigators used the Tufts Medical Center Specialty Drug Evidence and Coverage database to access coverage policies at 17 large US commercial health plans. The policies spanned 3786 orphan drugs and 4027 drugs included in US Food and Drug Administration (FDA)-expedited programs.

By health plan, the frequency of patient subgroup restrictions in policies varied from 11.7% to 36.6% for orphan drugs and 11.1% to 47.9% for FDA-expedited drugs, according to the study. Overall, health plans had patient subgroup restrictions in 20.2% of orphan drug policies and 21.8% of FDA-expedited drug policies.

Among 936 patient subgroup restrictions in orphan drug policies, 60.3% were consistent with the medication’s pivotal trial eligibility criteria, 7.3% used the same measure used in trial eligibility criteria but were more restrictive, 12% used the same measure but were less restrictive; and 20.5% were not consistent with trial eligibility criteria, researchers reported.

Among 1070 patient subgroup restrictions in FDA-expedited drug policies, 57.5% were consistent with pivotal trial eligibility criteria; 6.7% used the same measure but were more restrictive; 16% used the same measure but were less restrictive; and 19.8% were not consistent with trial eligibility criteria.

“The findings of this study suggest that plans often cover drugs more narrowly than the FDA’s approved indication, potentially preventing access to therapies for some patients for whom the therapies are indicated,” the authors wrote.

Reference:
Jenkins NB, Rucker JA, Klimchak AC, Sedita LE, Chambers JD. Commercial health plans use of patient subgroup restrictions: an analysis of orphan and FDA-expedited programs. J Manag Care Spec Pharm. Published online March 2, 2023. doi:10.18553/jmcp.2023.22363