OBJECTIVES: To understand United States (US) payer perceptions and challenges in the evaluation of emerging health technologies with orphan and ultra-orphan designations. METHODS: In-depth, qualitative, one-on-one interviews were conducted with US payer decision makers from the RTI Health Solutions US Commercial Payer Advisory Panel.

RESULTS: In the US, patient access to orphan and ultra-orphan technologies is seldom denied due to the rarity of the diseases, unmet needs, and lack of alternative treatments. Payers identify the biggest challenges as lack of clinical and comparative efficacy data and pressures from advocacy groups, patients, and prescribers to fund the ever-increasing numbers of orphan and ultra-orphan technologies, which are often very expensive and have limited clinical evidence. Payers estimated that spending for orphan and ultra-orphan technologies will increase significantly in the next 5 years, leading to concerns over future funding and budgets. Payers were interested in data that could have an impact on costs, cost offsets, resource utilization, readmissions, and real-world outcomes in their settings and patient/member populations. Payers wanted to see better-defined patient populations and unmet needs accompanied by well-defined treatment courses (e.g., when to stop treatment). Benefits of new technologies may not be captured in traditional health economic analyses, thus increasing uncertainty. Bridging the clinical evidence with other robust data will be critical, because payers will be passing on more risk to patients and prescribers in an effort to manage budget constraints. CONCLUSIONS: Payers are seeking more value-based information to better inform decision making in the evaluation of new orphan and ultra-orphan technologies. The challenge to payers lies with the value of the new technology and who is judging that value. Rising costs of orphan and ultra-orphan technologies will have more impact on market access in the future; over time there will be increasing resistance to high prices.