Sunday, January 1, 2017

The Perfect Storm Evolves

Here we are in 2017.  Happy New Year everyone!

Once again I find myself with mixed feelings going into the New Year. The perfect storm has evolved to become a mix of regulatory reform and political turmoil. I am optimistic on the regulatory front and on the prospects for government investment in antibiotic development, but uncertain on the future for new business models for antibiotics.

With the passage of the 21st Century Cures Act, the FDA in the US will now have legislative authority to look at smaller datasets to approve new antibiotics. This could allow for feasible pathways for approval of pathogen-specific antibiotics where the numbers of patients available to enroll in clinical trials will be extremely small and where their underlying illnesses will make these trials extremely challenging. I expect the FDA will respond with new guidance along these lines. In addition, the FDA is looking at the use of real world data (requires subscription) as a way to bolster their views on risk and benefit in a real world setting.  Such an approach could also provide new ways of using external control data to further support regulatory approval of pathogen-specific antibiotics. Given the leadership role that the European regulators at EMA have taken to looking at pathogen-specific antibiotics, I expect that these moves will also be welcomed there. So I expect further progress on the regulatory front during 2017.

BARDA has been very active over the past several years in providing large grants to companies to support the development of antibiotics.  Given their participation in the CARB-X initiative, I expect this to continue. But I do not expect BARDA to be able to participate in establishing a new business model for antibiotics (see below).

Storm clouds that could continue to threaten our ability to have a robust antibiotic pipeline are gathering. First, there is Brexit. I don’t know any better than anyone else whether Brexit will even occur. It looks like this may end up being decided at least in part by the British Supreme Court. But one of the consequences of the referendum is a serious consideration by Europe to move the European regulatory authority, the EMA, out of London where it has been based since its inception in 1995.  There is already a scramble among the other European nations to welcome the EMA.  One of the worries for me in particular is that the current Chair of the Infectious Diseases Working Group of EMA, a real leader in the regulatory approach to antibiotics, would not continue in that role.  This would be a tragic loss if it should occur.
The other issue with Brexit is the potential loss of the leadership position forged by David Cameron, George Osborne, Dame Sally Davies, Jim O’Neill and the AMR Task Force, the Wellcome Trust and others in the fight against antibiotic resistance. In so many areas, the United Kingdom has become a world leader in this fight – from showing the way with national antimicrobial stewardship to establishing the O’Neill task force to funding antibiotic research. I don’t see any other country or organization, not the US, not WHO, not the Gates Foundation, not anyone, who can replace this leadership on the world stage. It is impossible to predict what will happen to the UK’s leadership position if Brexit comes to pass.

It is clear that we need new business models to incentivize the establishment of a robust antibiotics pipeline while providing for appropriate stewardship at the same time (1,2). This will require action by various national authorities to actually spend money. I think of this as a capital investment that will save money later on as a way of return on this investment. But like most large companies, governments seem more interested in next year’s budget rather than cost savings over the next ten years. While there has been much more talk about new business models over the last two years, there has been precious little action. During 2016 there has been essentially no discussion of the coming crisis of antibiotic resistance by politicians running for office.  That certainly has been true in the US – but it is also true in the UK, France, Germany and elsewhere.  I’m not sure what is going on in Asia in this regard, but without a global effort, the business model will simply founder. This is a global problem requiring a global solution.  But the global solution requires individual nations to put up money – some more than others. In spite of the recent call by the United Nations, I don’t see progress on the horizon for 2017.

So, dear readers, my advice for 2017 is – fasten your seatbelts.

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