Chapter 16: Possible Solutions to Accelerate Access to Rare Disease Treatments Check Access

The introduction of legislative incentives to promote rare disease research, along with changes to regulatory policy, has resulted in increases in the number or orphan drug designations. Despite this, the number of drugs reaching marketing approval across OECD countries remains frustratingly flat. Research into rare diseases faces inherent challenges throughout clinical drug development and regulatory approval. Tailor-made regulatory and access solutions are needed to overcome these problems. Here I suggest areas for consideration that could have an immediate impact in facilitating regulatory approval and access to treatments for rare diseases.