Clinical Research: Phase 1 - Phase 4

Tales from the Road – World Orphan Drug Congress

Last week, I attended World Orphan Drug Congress in Belgium. It was my first WODC, and it was a productive three days in Brussels – even if it was held at the airport hotel, and we never left the building!

The congress agenda covered a wide range of topics in the rare and orphan disease – with a 360-degree view from patients, pharma, and research. However, I particularly enjoyed the roundtable on the challenges and mitigation of drug development in neuro-orphan diseases, led by representatives from another CRO.

The roundtable included people from biotechs and patient organizations, and we had a very good discussion about orphan diseases in general, and about neuro-orphan diseases in particular. I was thrilled to find that my rare disease work at Premier has given me much relevant experience to draw upon, allowing me to provide more insight on topics we discussed, such as:

  • the complexity of using rater scales at research-naïve sites
  • the importance of partnering with advocacy groups in early stages of study planning, not just for patient recruitment
  • identifying endpoints in rare diseases that can be used both pre- and post- marketing approval.

All in all, it was an interesting 45 minutes, and a very good discussion around the nuances of neuro-orphan studies.

A great conference from the sessions to meeting the speakers and attendees, to even the food – so happy I had the opportunity as a PM to get out and about with the rare community.