What do regulators look for when granting #orphandrug approval #raredisease

In Uncategorized by Matt Turner

gene therapy, jorn aldag, amsterdam molecular therapeutics, ultra orphan drug, rare diseasesResearching into a rare disease?

There are significant challenges facing orphan drug development and some of these challenges increase when developing an “ultra-orphan drug”. Clinical trial design and development need to be adapted to clearly and statistically demonstrate the purpose of the treatment, especially in light of a very widespread and niche patient populations. Moreover, the interaction and understandable dialogue with the regulatory authorities is paramount in order to receive their ongoing feedback and adjust future clinical phases and data accumulation accordingly.


· But could communication between industry and the regulatory authorities be improved to avoid clinical setbacks?

· What channels are in place for large and small pharmaceutical organisations to engage and discuss the development of orphan drugs with the regulatory authorities?

· How have organisations experienced real life interactions with the regulators and what lessons were learnt from initial setbacks in order to re-evaluate and ultimately achieve market approval?

Jӧrn Aldag, CEO, Amsterdam Molecular Therapeutics discussed the challenges for clinical trials in small patient populations and their experiences with the regulatory authorities during the development of Glybera, a gene therapy for LPL deficiency at the World Orphan Drug Congress 2011 in Geneva.

We hope you enjoy the video below.




Looking for more examples and information on the challenges of orphan drug development?

Try our latest blog on How Genzyme created the critical elements of orphan drug success

Why not hear how Lysogene discussed and validated their approach into clinical trials by attending the World Orphan Drug Congress Europe.