With over 90% of rare diseases having no approved medical treatment, clinical trials for new therapies are of paramount importance. And with over 3,500 individuals diagnosed with a rare disease globally, therapies for these conditions should be a golden opportunity for any pharmaceutical company. Add to that the significant financial incentives from the federal government in the Orphan Drug Act of 1983, it would seem that pharma would be diving into rare disease enthusiastically and often.
However, the process is slowed by several challenges on the clinical trial front. First of all, although the overall rare disease population is large, the individual populations of each condition ranges from small to miniscule. Consequently, each therapy potentially delivers a smaller Return on Investment than traditional medicines, even considering the government incentives. The smaller sizes also mean that it will be harder to meet regulatory thresholds in the trials for safety and efficacy.
Finding providers who can facilitate the trials is another hurdle. As with all aspects of treating rare disease, there are precious few providers with sufficient clinical knowledge to treat and monitor rare disease patients, and centers that have capable staffs are few and far between. That makes it harder for patients to travel to the centers to participate.
Additionally, traditional clinical trial endpoints may not be appropriate for rare disease therapies. The diseases are not well understood, so cures and symptom eradication may not be easily reached. It is probably more appropriate to partner with rare disease patients to discover what outcomes they want from a therapy.
How can we design trials to make them efficient enough to attract rare disease patients and produce therapies that enhance quality of life? Tune in next week!