What does the nomination of Dr Scott Gottlieb mean for drug development and approval in the US?

Since his inauguration in January, US President Donald Trump has nominated like-minded individuals to his cabinet and to head federal agencies. Sharing his probusiness and deregulation agenda, they spent considerable parts of their careers either in the fi nancial industry or at large corporations. Controversially, many lack the relevant expertise to lead the departments or agencies for which they are heading. Trump’s choice of Dr Scott Gottlieb as Commissioner at the Food and Drug Administration (FDA) follows a very similar pattern, but only to a certain extent.

Who is Dr Scott Gottlieb?

Dr Gottlieb, age 44, shares many common conservative beliefs as Trump’s other nominees, namely being pro-business and deregulation. However, unlike them, he possesses relevant experiences which place him uniquely to lead the FDA. A physician by training, he went on to work at Wall Street, followed by an appointment as an FDA deputy commissioner before returning to work as a fi nancier. In the meantime, he has secured independent board directorships and consultancy work with numerous biotech and pharma companies, ranging from start-ups to titans like GlaxoSmithKline, Bristol-Myers Squibb and Daiichi-Sankyo. He is a partner at a large venture capital fund, New Enterprise Associates, and at T.R. Winston, a merchant bank; both having signifi cant investments in the bio-pharmaceutical industry.

Dr Gottlieb has authored dozens of articles in leading medical journals including the British Medical Journal and Journal of the American Medical Association, alongside mainstream publications like Wall Street Journal, New York Times, National Aff airs and Forbes. He also appears regularly as a commentator on CNBC and Fox News.

Of particular interest is his penchant for writings on FDA-related issues; in his own words, “analysing developments in policy and regulation and their impacts on innovation, medical care, and public health”.

He is a Resident Fellow at the American Enterprise Institute, a conservative thinktank, focusing on “healthcare reform and political and clinical trends in medicine”.

On a personal note, Dr Gottlieb made a complete recovery from Hodgkin’s Lymphoma in 2006 and acts as an advisor to the National Coalition of Cancer Survivorship.

His time at the FDA

Dr Gottlieb served two stints at the FDA: from 2003-2004 as a senior advisor to the FDA Commissioner, and from 2005-2007 as a deputy commissioner for medical and scientific affairs. In between these two postings, he was a senior advisor at the Centers for Medicare and Medicaid Services working on the Medicare Drug Benefit programme, advising on policy changes including coverage and payment decision making and the uptake of new medicines by the Services.

What will Dr Gottlieb do if he gets appointed?

Unlike the other of Trump’s nominees, Dr Gottlieb has written prolifically on matters that come under the remit of the FDA.

He has called on numerous occasions for the FDA to reduce the time taken to approve drugs. He feels that the agency should demonstrate more flexibility in granting product licences and that indicative efficacy evidence should be suffcient for an initial approval, as long as there is a commitment to generate definitive evidence at a later date. In addition, he has called for the faster approval of complex generics in order to compete with the higher-priced originals. In that respective, he echoes the Trump campaign rhetoric to bring down drug prices.

Dr Gottlieb has certainly reflected deeply during his time at the FDA. In a seminal paper which he had written in 2012 for National Affairs, a conservative journal, Changing the FDA’s Culture, he set out his persuasive arguments for cutting the bureaucracy at the agency while advocating for a complete change in the philosophical outlook of approving medicines, especially rarer diseases. He felt that over the decades, the agency has become increasing dogmatic and excessively risk-adverse, leading to an “increasingly unreasonable hunger for statistical certainty” for registration trials to become unnecessarily complex and longer than needed. Although the US Congress has passed two Acts recently to speed up the drugs approval process, he feels that until there is a fundamental structural change at the agency, the rigid ‘review culture’ will continue to act as an impedance. He proposed that the FDA continues to do what it does best, which is to Dr Stephen Huang is pharmaceutical medicine consultant at SCP Medical conduct a scientific review of drugs by teasing out their benefits and risks, but then hand over the ‘policy’ decision to another body to weigh these benefits and risks before making the judgemental call to grant a licence. Interestingly, he suggested that the US could look to the EU as an example. He observed that the scientific review of drugs falls under the remit of the European Medicines Agency while the granting of a licence sits with the European Commission, a politically accountable body. Dr Gottlieb is a barometer of what Trump’s still uncrystallised healthcare policies are likely to be, as he has shared his opinions widely for over a decade now.

Will there be any opposition to Dr Gottlieb’s plans?

However, Dr Gottlieb does have his detractors who pointed out his glaring conflicts of interests. Michael Carome, research director at Public Citizen, a liberal consumer advocacy group, wanted the nomination to be rejected due to his entanglement with the pharmaceutical industry; making him unfit to act as an independent regulator whose primary role should be the welfare of patients rather than serving the narrow commercial objectives of the life sciences sector.

Other critics argued that Dr Gottlieb’s advocacy for a shortened drug approval process will compromise drug safety, pointing out that the FDA is already one of the fastest regulatory agency in the developed world. As expected, his nomination was highly partisan and it drew wholehearted support from the Republicans while the Democrats were largely opposed due to his overwhelming conflict of interests. Dr Gottlieb tried to assuage his critics with the reassurance that, if appointed, he will ‘recuse’ himself from decisions that could affect over 20 companies with whom he had dealings.

Dr Gottlieb’s confirmation was heard by the US Senate and on 27th April, its health committee ‘favourably reported out’ his nomination for consideration by the full Senate. The committee chairman, Senator Lamar Alexander (Republican) commended, “Dr Gottlieb is the right person to lead the FDA in its vital mission and move the agency forward. He has impressive qualifications from nearly every perspective; he has been a practising physician, held three roles at FDA, and is a cancer survivor.” When endorsed by the Senate as expected, his appointment will give us an indication of President Trump’s likely policies regarding the development and approval of medicines. We can expect major policy shifts at the FDA in the coming years rather than the glacial incremental changes that are often associated with federal agencies.