The Rare Disease Action Forum holds an international event on the topic of “value assessment of orphan drugs in Switzerland and the EU” on 31 October 2018 in Basel, Switzerland Experts from authorities, patient organizations and the industry will provide insights to assessment models for orphan drugs in Switzerland, the EU and specific individual countries such as Germany, Austria and France.

A timely and predictable value assessment process for orphan drugs plays an important role in enabling access to treatment for patients with rare diseases. Due to the characteristics of rare diseases and related clinical trials (e.g. very low patient numbers, no active comparator) standard value assessment processes are especially challenging for orphan drugs. An additional hurdle to the reimbursement and access for patients is imposed by the fact that value assessments are mostly conducted at the time of marketing authorization without consideration of growing evidence from use in clinical practice.

The RDAF event will give participants the opportunity to exchange knowledge and best practices on this topic.