The Feasibility of Rare Disease Clinical Trials, Part 2

In Clinical Development by Freya Smale

This guest blog post was provided by Premier Research.

Welcome back for part two of our WODC video series, where we hosted a panel discussion at World Orphan Drug Congress Europe, where four leading specialists including senior executives from Aegerion and Lumena Pharmaceuticals, discussed the role of advocacy groups and shared some stop takeaways when it comes to the feasibility of rare disease clinical trials.

We address the Role of Advocacy Groups, Networks and Compassionate Use Programs, recognizing that advocacy groups come in all shapes and sizes. Some have adequate financial support and others may simply operate around a kitchen table. Whatever the scope or size, these groups have vast potential to assist with clinical trials for rare diseases. Our panelists help us better understand the many hats that advocacy partners wear, including: 

  • Champion. You won’t find a group more dedicated to the cause. Advocacy group members may have children or other family with this condition, and they want more than anything to find a therapy.
  • Evaluator. It’s important to involve advocates early as they can help with many important aspects of study feasibility. Understanding issues early enables you to adapt the study, rather than face bigger issues later.
  • Liaison. Patient support is critical, and not just during a clinical trial. Advocacy groups can help post-trial to ensure medicines are paid for, or that patients receive proper reimbursement. They can also help keep families informed—especially when they are in a different country.

And, finally, don’t forget Top Takeaway Tips from the Experts, where we revisit:

  • Examination. Being under the magnifying glass in a rare disease study. Get it right the first time, if you can.
  • Advocacy. Learning from patient advocacy groups and be your own advocate by educating regulators.
  • Tested ground. Following a common pathway with regulators, patients and stakeholders to make orphan drugs sustainable.

A great big thank you goes to our panelists, of course:  Dr. Colin Hayward, Vice President, Medical Affairs, Premier Research; Dr. Susan Tansey, Medical Director, Pediatrics, Premier Research; Dr. Ciara Kennedy, Chief Operating Officer, Lumena Pharmaceuticals;  and Dr. Massimo Boriero, President for Europe Middle East and Africa, Aegerion Pharmaceuticals.

We learned a lot from their insights, and hope you did as well.

We have a wealth of Rare Disease resources and capabilities, so please take a moment to visit our resource page, where you can find our case studies, infographics, fact sheets and other videos.

And don’t forget, we’ll be seeing you soon at this year’s World Orphan Drug Congress! Stop by our booth (#13) and meet some of our experts, but also, don’t miss Angi Robinson, Executive Director, Scientific Account Leader, on day one, who will lead a discussion on Rare Disease and best practices when partnering with a CRO.

See you in DC!