Catherine Kielar
PRINCIPAL MARKET ACCESS CONSULTANT
STAATZ BUSINESS DEVELOPMENT & STRATEGY

An increasing amount of new treatments obtain orphan designation from the European Medicines Agency (EMA 2019). Since 2000, the number of applications for orphan medicinal product designation reached 3,443 with 2,247 of these achieving positive opinions and 169 orphan marketing authorizations. Orphan drugs are usually highly priced but there is more uncertainty around their data (i.e., short-term trials, placebo controlled, small numbers of patients, etc.) which causes a challenge from a reimbursement and health technology assessment (HTA) perspective. Orphan drugs are generally assessed by national bodies through HTA with the corresponding requirements varying by country. The purpose of HTA is to demonstrate the value of a new technology, against currently available alternatives. Different HTA bodies will use HTA to negotiate price reductions and save money. Therefore, understanding how the appraisal requirements and processes vary between countries is paramount for a successful orphan product launch.

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