We have previously discussed the social responsibility of the pharma community to producing drugs to treat rare diseases. But, at the risk of sounding callous, what about the economic side of things? Are orphan drugs economically viable, and are they a lucrative option for pharma and biotechs?
The answer is a resounding yes.
Thomson Reuters recently produced a report that states that "despite the smaller patient pool for rare disease R&D, the economics of orphan drug development shows great potential for commercialization." Whilst the patient population is smaller, this is offset by the higher price demand for orphan drugs because of fewer, if any, competitors and the high level of unmet need. Additionally, "economic drivers such as tax credits, grants, waived FDA fees, reduced timelines for clinical development and higher probability of regulatory approval, coupled with commercial drivers such as premium pricing, faster uptake, lower marketing costs and longer market exclusivity, further fuel the economic power of orphan drug development."
Whilst a few years ago orphan drugs were mainly developed by smaller biotech companies, recently more and more big pharma are seeing the benefits of orphan drug R&D.
According to Peter Saltonstall, chief executive of the National Organisation for Rare Diseases, "one-third of the new molecular entities approved by FDA last year were orphans. And some of the most innovative and interesting research being done at this time is in the orphan space. It's becoming increasingly evident that companies of all sizes may find a niche there.'
So, the conclusion is that not only are orphan drugs the socially responsible and ethical drug to focus your R&D efforts on, but they could also improve your bottom line!
For more information check out World Orphan Drug Congress USA.