摘要
The paper is to look at the affordability of orphan medications across the globe and whether payer attitudes to high-price medications are changing in the face of rising healthcare expenditure and tighter budgets. We conducted an online semi-quantitative survey of 10 European markets, the United States and Japan （Q1-Q2 2014） to understand how payers＇ views and attitudes are changing in response to new treatments coming to market for rare and ultra-rare conditions. The payers selected for the survey hold or have held senior positions within their respective market institutions. The United States and Japan were included in the study to provide international context to the European results. Responses were anonymised in accordance with good market research principles. The research shows that 75% of respondents surveyed believe that the current approach to orphan drug pricing is unsustainable in the future and 92% predict a tougher approach from payers going forward. 75% of payers do not believe that patent expiry alone will free up the necessary space for innovative orphan and ultra-orphan products. 83% of the payers surveyed believed that less than half of all orphan and ultra-orphan drugs coming to market are supported by an adequate evidence base for reimbursement. The environment for orphan medicines across the globe is changing; and as the financial performance of countries begins to diverge, so do attitudes towards the funding of orphan medicines. The increasing number of rare diseases, and treatments available, is forcing payers to view orphan drugs in a new light and they are becoming increasingly sceptical about the prices charged in relation to the clinical benefit offered. As rare disease spending becomes a higher proportion of pharmaceutical spending, payers will need to take action to curb this trend.
