Nick Kenny, PhD, Chief Scientific Officer at Syneos Health talks about the challenges in developing clinical trials for rare conditions.

Dr. Kenny said there are a myriad of challenges. First among them is the limited number of patients that can qualify for a clinical trial. “You may have a lot of people trying to develop molecules in those diseases for which is a very limited number of patients and as a physician of a patient, how do you accurately assess what is the right trial?” noted Dr. Kenny.

A second concern is that there are over 7,000 rare diseases but “we often don’t understand the natural history of those diseases, so even looking to get a diagnosis made to begin with can be challenging,” stated Dr. Kenny, adding, “then once you’ve found those patients to actually design trials to collect the right data because sometimes the endpoints are not particularly clear and you’re actually creating things from scratch.”

A third concern is the issue of the placebo group. “Certainly, in many instances, it’s unethical to do a control arm in a trial. There simply aren’t enough patients or standard care may not exist or it may be ineffective — so why would we put the very few patients with that disease through some control?” asked Dr. Kenny.

Dr. Kenny is hopeful however about the state of rare disease clinical trials and noted that curated real-world evidence to help develop clinicals is showing promise

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