OBJECTIVES : It is unclear to what extent US commercial health plans treat drugs indicated for orphan diseases (diseases affecting fewer than 200,000 individuals in the US) and non-orphan diseases differently. The object of our research was to compare the frequency that US commercial health plans apply coverage restrictions to orphan and non-orphan drugs.

METHODS : We relied on the Specialty Drug Evidence and Coverage (SPEC) Database, which includes information on 17 of the largest US commercial health plans’ specialty drug coverage policies. We compared how plans covered drugs with the drug's corresponding FDA label and categorized coverage as follows: (i) coverage with no restrictions (the plan does not apply restrictions on coverage beyond the FDA label), (ii) coverage with restrictions, (iii), or not covered. Further, we categorized coverage restrictions as follows: (i) step therapy protocols, (ii) patient subgroup restrictions, (iii) prescriber restrictions (a certain type of physician, e.g., an oncologist, must prescribe the drug), or (iv) other restrictions.

RESULTS : We examined 2,168 orphan drug coverage policies and 2,832 non-orphan drug coverage policies. We found that 67% of orphan drugs were covered without restrictions, 32% with restrictions, and 1% were not covered. We found that 47% of non-orphan drugs were covered without restrictions, 47% with restrictions, and 6% were not covered. Plans most often applied patient subgroup restrictions (45%) to orphan drugs, followed by step therapy protocols (43%), prescriber restrictions (35%), and other restrictions (5%). 24% of orphan drug decisions included multiple restrictions. Plans most often applied step therapy protocols (78%) to non-orphan drugs, followed by patient subgroup restrictions (24%), prescriber restrictions (26%), and other restrictions (3%). 27% of non-orphan drug decisions included multiple restrictions.

CONCLUSIONS : Plans cover orphan drugs more generously than non-orphan drugs. Plans tend to apply different types of coverage restrictions to orphan and non-orphan drugs.