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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