Rare disease treatments: Important elements for orphan drug clinical development

In Clinical Development by Matt Turner

rare disease, orphan drug, clinical development, orphan drug congressTreating rare diseases and developing orphan drugs has the potential to be more difficult to achieve due to unknown or barely known disease mechanisms, slow trial enrolment and challenging endpoint validation to satisfy both regulators and payers alike.

In our latest industry survey, we delved into which challenges and considerations of orphan drug clinical development were the most important to be educated on. Over 50% of the surveyed audience expressed that the following areas of concern were “critical” to their personal portfolio development.

  • How to design your clinical trial based on rare disease specific challenges?
  • How can you do adaptive study design and maintain flexibility throughout?
  • How to integrate, evaluate and refine innovative approaches to your design?
  • Why expanding into different indications following market approval is not guaranteed
  • How to harness protocol assistance and scientific advice when in the clinic?
  • Success and failures: why are some orphan drugs successfully developed and others not?
  • How to create compelling natural history knowledge for an un-researched rare disease
  • Sustainability of current procedure: the need for regulatory flexibility to positively impact drug development
  • How to leverage centres of Expertise and European reference networks

The 2012 Industry Survey is one of many sources used to shape the 2012 World Orphan Drug Congress taking place in Geneva on the 29th-30th November. So if some or all of the points raised above directly relate to the successful clinical development of your orphan drug portfolio, the World Orphan Drug Congress may be the event to attend to drive your innovations forward towards commercialisation.

You can download the full industry survey here and to learn more about the 2012 congress, take a look at the brochure by downloading it here.

While you are here, why not read this blog on what the challenges are when translating rare disease research into clinical development?

You might also like to read what common mistakes should be avoided during late stage orphan drug development