OBJECTIVES: With rapidly increasing numbers of orphan new product molecules (NMEs) approved in the US (167% between 2016 (9) and 2018 (24)), the sustainability of access and reimbursement for these products is no longer a certainty. The objective of this study was to assess the rationale for and potential effect of key payer uncertainties around ongoing orphan drug access. Additionally, this research assessed current US payer management tactics for orphan drugs.

METHODS: Utilizing divergent scenario planning qualitative research methodology (DSP), 8 US payers (representing more than 63 million healthcare lives) were interviewed about key uncertainties that will impact orphan drug access in the coming 3-5 years, and were asked to describe the range of market scenarios for each uncertainty. Respondents subsequently ranked the relative impact of each uncertainty. Additionally, currently managed orphan product categories were discussed, and a frequency analysis of collated scenarios was conducted.

RESULTS: Uncertainties identified by respondents included alternative payment models (6), durability of curative therapies (4), “orphanization” of large markets (2), legislative uncertainty (2), orphan indication expansion (2). Orphan categories receiving increased utilization management include PAH, HAE, SMA, enzyme deficiency diseases, hemophilia, vascular edema, and some oncology indications.

CONCLUSIONS: The divergent scenario planning methodology describes the market access scenarios that payers, manufacturers and patients will be facing in 3-5 years. Payers are increasingly applying traditional management tactics to orphan categories, identifying orphan indications with sufficient product selection as to allow payer restrictions, and assessing novel payment mechanisms. The scenarios defined in this research highlight the importance for orphan manufacturers to align data development and product value with the market access environment. Historical payer utilization management tools and payment mechanisms employed today must be improved to address near-term orphan access uncertainties.