This week, the ‘super bowl’ of biotech J.P. Morgan 2017 kicked off in San Francisco and Senate confirmation hearings on Trump nominees began in DC. While there is no official nominee for FDA chair, Jim O’Neill and Scott Gottlieb are under discussion. Either way, the new Chair is likely to oversee much needed changes to our drug approval process.
National Cancer Institute Drug Formulary
At J.P. Morgan, Vice President Joe Biden spoke in favor of transparency and cooperation among cancer researchers as the fastest way forward and highlighted the National Cancer Institute Drug Formulary. The formulary is a public-private partnership with 20 – 30 pharmaceutical and biotech companies intended to facilitate research on new purposes, new compounds, and new combinations. The NCI Formulary is set to open next week.
No FDA Nominee on the Senate Confirmation Hearings Schedule YET
Trump’s intent for the FDA is not yet clear but he has indicated a focus on reform with an eye toward innovation and patient need which is consistent with the recent 21st Century Cures Act. Tech Pay Pal mogul Peter Thiel has been identified as someone involved in vetting the candidates.
Current estimates show that it costs over $2.5B and 12 years to move an innovative new pharmaceutical through the testing and approval process. No only do patients not gain access to life-saving drugs during these lengthy cycle-times, but these costs are translated into the very controversial pricing of drugs. So, with Trump’s focus on encouraging innovation while reducing overall drug prices (and costs of overall healthcare), it is no wonder that more progressive candidates are being considered.
In a 2014 speech, Jim O’Neill, a colleague of Peter Thiel’s and an unconventional FDA Commissioner candidate, outlined the following perspective: “We should reform FDA so there is approving drugs after their sponsors have demonstrated safety–and let people start using them, at their own risk, but not much risk of safety. Let’s prove efficacy after they’ve been legalized.”
Current regulators may struggle to translate what this type of wild, wild west concept for drug approvals would really look like. Yet, rare disease patient groups have been advocating for this model for many years. Sarepta’s Duchenne drug approval in 2016 shows encouraging transformation of the agency towards a more progressive model; Janet Woodcock is truly the hero.
While O’Neill is currently managing director at Mithril Capital Management, this is not his first trip to Washington. He is the the former HHS Deputy Secretary under George W. Bush.
Many are voicing concerns over O’Neill’s revolutionary thoughts and lack of medical background for the FDA post. If he were to be confirmed, it is predicted that it would cause disturbances at the agency and limit his effectiveness to do the transformative work that is needed.
OR Scott Gottlieb
Fan favorite Dr. Scott Gottlieb is also being vetted. Gottlieb has already done a tour of duty at the FDA and has been actively influencing policies regarding the recent 21st Century Cures Act. During a speech to the Energy and Commerce Committee, Gottlieb stated that “regulators made the barriers higher, not lower” and encouraged “as we improve our scientific understanding of rare diseases, and our ability to target their molecular origin, we’ll have more opportunities to treat and even cure the rarest disorders.”
A partner at venture capital firm New Enterprise Associates, fellow at the conservative think-tank the American Enterprise Institute, Gottlieb is an outspoken critic of the Affordable Care Act and has deep ties to the pharmaceutical industry.
Some feel he is the better choice. He will push for and encourage change at the Agency. But, will it be transformative or fast enough for Trump?