Before we can start discussing the issues with conducting clinical trials in rare diseases we can clarify what type of conditions will classify as rare diseases. Often rare diseases include genetically determined diseases which affect small part of the population. And although we say “small part of the population” it actually includes millions of people around the world. It is important to mention that some of these rare diseases do not have any treatment options at all and physicians have to adapt current standard of care practices.
In the recent years rare diseases have gained popularity in clinical research as part of the personalized care approach and now there are more efforts in finding treatment for some of them.
What are the challenges in setting up rare disease clinical trials?
- Finding the right hospital and the right physician – There are limited hospitals and consultants who are qualified to conduct clinical trials in rare diseases.
- Finding the right patients – Another challenge is to reach out and find patients with a specific rare condition who would be interested to participate in research.
- Setting the right timelines and expectations – Making sure the study will recruit the required amount of patients in a specific timeline could be a significant challenge.
But challenges exist not only for Sponsors but also for hospitals participating in clinical research.
What are the challenges for hospitals with rare disease clinical trials?
- Majority of the hospitals may not be able to run such studies due to lack of experience, patient population or qualified consultants.
- Financial loss – Setting up study and allocating resources for clinical trials is the same, however expected low recruitment rates (1-2 patients) may not justify the efforts from financial point of view.
- Resource issues – The low recruitment rates will require even more resources to identify patients. This may jeopardize the efforts of the clinical trials team to set up other studies.
- Loose study timelines – This is another significant issue for hospitals. Not knowing the exact close time for the clinical trials make it difficult to plan other studies and commit recourses elsewhere.
- Limited access – Having 1 or 2 potential patients for a study may not be very attractive to hospitals which would prefer more patients to have access to new treatment.
There are new obstacles with increased popularity of rare diseases and they will need to be addressed by the research community in a way that it will be favourable for both, Sponsors and research teams. Although all challenges with this type of clinical trials their popularity is surely a good news for patients and will bring new treatment options.
Author: Olga Peycheva
Olga is a clinical research professional who has been working in clinical research since 2005. She has extensive experience in clinical research in Eastern and Western Europe.
Originally published on 3 Apr 2017