OBJECTIVES: To understand European Union (EU) and Australian 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 payer decision makers representing Australia and the EU-5: France, Germany, Italy, Spain, and the United Kingdom from the RTI Health Solutions Payer Advisory Panel.

RESULTS: 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. Understanding the disease and its burden and unmet needs was consistently highlighted. All 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. Clawbacks in France will be common; employment of the rule of rescue in Australia and pricing pressures in Italy, Spain, and the UK are to be expected. 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 will not be captured in traditional health economic analyses, thus increasing uncertainty. Bridging the clinical evidence with other robust data will be critical. The cost to the systems was highlighted as a consistent concern. More emphasis on showing the value, affordability, comparative effectiveness, and cost offsets is critical. 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 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, with increasing resistance to high prices.