Clinical trials are an expensive business. With the cost of bringing a drug to market in the billions efforts are continuously being made to reduce costs where possible.
Whilst compensation and tax breaks from regulators have reduced the average cost of taking an orphan drug through clinical trials to just under a billion, costs could surely be reduced further if trial designers, CROs and sponsors can streamline the aspects of trials unique to orphan drugs. Aspects such as: small patient populations spread sporadically across nations, continents and globe; difficulty in securing clear data formatted universally between multiple clinical sites; engaging with winning the trust of the often reluctant rare disease patient population.
The importance of reducing the cost of clinical trials, perhaps the single most expensive part of development (especially Phase III), is in the knock on effect on the potential to make profit on a drug, whilst driving down the cost of treatment for patients. Orphan drugs command a high price on the market, but it is one that is often criticised for making it difficult for patients to afford or be granted treatment. Any way to reduce the costs of clinical trials for for drugs with such a small market is a necessity.