The Feasibility of Rare Disease Clinical Trials, Part 1

In Clinical Development by Freya Smale

This guest blog post was provided by Premier Research.

We’re excited to be seeing everyone at this year’s World Orphan Drug Congress next week! On Day One, Angi Robinson, Executive Director, Scientific Account Leader, will lead a discussion on Rare Disease and best practices when partnering with a CRO. Be sure not to miss it!

But, while we have you, last November, we were pleased to host a panel discussion at World Orphan Drug Congress Europe, where four leading specialists including senior executives from Aegerion and Lumena Pharmaceuticals, shared practical examples of their hands-on experience designing feasible studies, engaging regulators, and dealing with inexperienced sites.

Our panelists include 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 – each with insightful perspectives of fielding rare disease clinical trials.

In Study Design Considerations, our panelists discuss what goes into study design, including:

  • Communication. Why you need to talk to patients and their families early on, and what you need to learn from them.
  • Common ground. How to select end points that satisfy physicians, patients and regulators – all of whom may have different interests.
  • Addressing challenges. How to share the challenges with regulators early on, so they become part of the solution.

How to Successfully Engage Regulators provides insights on knowing exactly when to engage regulators – on both sides of “the pond.”

  • Early is good, but enlightened is better. You need enough information around your story to make it meaningful to regulators.
  • Regulators are not experts. When it comes to rare disease, you can’t expect regulators to make an informed decision about something they may have only read about in a textbook. Share the journey of learning with them.
  • Know your endpoint. Consider the primary and secondary endpoints for a given protocol—whether biochemical or clinical endpoints—when designing your study. 

In How to Help Inexperienced Sites Conduct Successful Studies  learn about how a clinical trials partner can help with:

  • Right place. Many centers are not turnkey solutions that are set up for clinical trials. Networks can help implement a study at a site that has already been chosen, or they can identify a site. Finding a site can be especially important for rare disease studies where only two or three hospitals in a country—or the world—can conduct the study.
  • Right people. You need the right staff on site, such as a clinical research coordinator and research nurses, to carry out the study. Even studies of only five patients might be intensive.
  • Right procedures. A network can help with applying for regulatory and ethics approval, explaining the required level of rigor, and ultimately helping to train up a site that is not experienced with conducting clinical trials.

Stay tuned for part 2 of this series, where we address the roles of advocacy groups and our top takeaways from this really informative panel.

And, of course, if you’d like to read more about our Rare Disease capabilities, please visit our resource page, where you can find our case studies, infographics, fact sheets and other videos.