Seminar

Orphan drugs are used for the diagnosis, prevention or treatment of life-threatening or very serious conditions. Although there is EU orphan medicine legislation in place to provide incentives for researching, developing and marketing, access to such treatments across Europe is likely to be variable. In this conference we explore the extent to which pricing and reimbursement arrangements accommodate orphan medicinal products.

The focus of the seminar is twofold. First, we want payers in selected European countries to describe the P&R and decision making processes around orphan drugs if they exist. In particular, we are interested in understanding whether they treat them differently from other drugs and, if so, how. Second, we want to generate a constructive discussion about affordability of orphan drugs in the medium/long term with key stakeholders including third party payers.

For the first objective in particular, we are interested in learning about the following issues regarding orphan drugs, with case studies where appropriate:

• Is there any specific national policy in favour of orphan drugs e.g. creation of specific programmes or institutions dealing with orphan drugs?
• What type of evidence (clinical and/or cost effectiveness) is currently being used to determine the price and/or reimbursement status of orphan drugs? Are there any other criteria required for the decision-making process?
• What type of special reimbursement controls, if any, are currently targeting orphan drugs when launched (e.g. are they included in national reimbursement lists or reimbursed on a case by case basis)?
• Are there any market access issues regarding orphan drugs in particular are there any additional barriers to entry such as specific budgetary constraints?;
• Are third party payers requiring post-launch studies for orphan drugs?

Below is the full OHE briefing.