Considerations for successful clinical development for orphan indications

Clinical trials (programs) for orphan indications present unique challenges for the drug developer. The fact that the disease is often poorly characterised and under-researched makes it difficult to define suitable clinical parameters; the paucity of patients and specialist centres create logistical issues; the fact that the regulatory framework is not always well suited to novel therapies in a small and unstudied patient group requires innovative thinking and interaction with the regulators to achieve an appropriate study design. These factors all need to be given adequate consideration when developing treatments for orphan indications.