Abstract
In 2011 we reported that 153 drug, vaccines and in vivo diagnostics which received FDA approval prior to September 1, 2009 had been discovered in whole or in part in research carried out at U.S. public sector research institutions (PSRI's). A parallel study found that non-U.S. PSRI's also contributed to the discovery of 15 of these drugs and in addition discovered a further 19 FDA-approved drugs.
In the current study, we update the earlier study to include drugs which received FDA approval up to December 31, 2016. Our methodology included a significant new source of information, the Sunshine Act, which requires the disclosure of all payments by medical providers to U.S. physicians and teaching hospitals. This allowed the identification of additional drugs discovered by physicians at PSRI's, some of whom owned the underlying intellectual property in their own names, and also revealed several drugs discovered by physicians in private practice.
We have now identified 252 FDA-approved drugs that were discovered in whole or in part by U.S. PSRI's, and an additional 79 discovered by non-U.S. PSRI's, for a total of 331 drugs discovered at PSRI's worldwide.
We examine in detail two cases of the discovery of new classes of oncology drugs: checkpoint inhibitors, specifically ipilimumab, nivilumab and pembrolizumab; and androgen receptor inhibitors, specifically enzalutamide and apalutamide.
The checkpoint inhibitor case illustrates the challenges in translating radical new treatment modalities from academic lab to patient bedside. Ipilimumab went through the hands of nine companies and was first developed in four combination products over 16 years before receiving FDA approval as a single agent in 2010.
The enzalutamide / apalutamide case illustrates the massive private investment needed to successfully develop drugs whose discovery was publicly funded. Scientists at UCLA received grants totaling $1.3 million, which resulted in the discovery of enzalutamide, apalutamide and a breast cancer drug whose development was discontinued after Phase 2, an average public investment of $433,000 per drug discovered. The cost to develop just enzalutamide worldwide cost around $1.8 billion, some four thousand times more than the cost to discover it.
