37. That’s then number of breakthrough therapy designations (BTDs) that have been awarded since the US Food and Drug Association (FDA) introduced the new designation mid 2012.
But back in 2012 when the FDA announced BTD as part of their Safety and Innovation Act, what seemed to distinguish BTD from other designations such as their fast track designation, was the assistance on offer to small cap biotech firms. Through providing clinical trial design assistance, regular meetings and specialised expert advice, manufacturing help and commercial-scale-up advice, the FDA looked to be targeting small biotech firms who are strapped for cash and cannot accelerate product development to 100%.
But a glance over a list of the 37 designations shows that the vast majority of companies who have been granted BTD are mid to large pharma companies. Only 20% of designations went to small-cap biotechs. Perhaps more telling is that none of the designations took place during phase I trials, after which the value of clinical trial design help from the FDA becomes dubious.
So where are the small biotechs?
Perhaps, as rejection and approvals for breakthrough therapy designation do not have to be made public for individual products and companies, we cannot see the drove of small biotechs who have applied but not achieved the designation. But even if these small developers are simply not making things public (so far only 2 of 72 companies have publicized their rejections) why are they not being approved?
Perhaps the application process is keeping small firms at an arms length? In which case we may well see a surge in the number of small biotechs being approved as regulatory advisers and consultants begin to offer the relevant services. But this somewhat jibes with comments made by Scioderm’s (one of the successful small biotechs) COO, Robert Coull, who describes the application process as “relatively straightforward” having produced their application entirely in-house.
So is the designation the problem? Currently there isn’t enough information to show that BTD pushes products through approval any faster than products with other designations, can we conclude that so far the designation has failed to show any real benefits outside of ‘prestige’? (as there’s no evidence that BTD effects company share price)
If this is the case we can explain the sizable presence of big pharma through the ‘prestige’ the designation bestows onto the product. In the rare disease and orphan drug space bigpharma are constantly trying to show the world that they are making headway. If breakthrough therapy designation is just another trophy for the mantel piece and offers little in the way of actual support, no wonder small developers aren’t wasting time on applications.
Find us on Google.