There were several key presentations from various funding
agencies including BARDA, NIH, IMI and the Wellcome Trust. With the exception of the Trust, it was
abundantly clear to everyone that without additional funding, the efforts of
these agencies to go beyond their current funding levels or even to maintain
them will be threatened. The funding
from these agencies goes to everything from early discovery all the way through
phase III clinical development and has been very important for academia and
both small and large pharma in the pursuit of new antibiotics. Any diminution or even an inability to expand these efforts would be a terrible tragedy.
Several non-antibiotic approaches were discussed including
antibodies, peptides and virulence. Some
are being examined for prevention while others are thought of as adjunctive
therapy. All of these approaches will
mean superiority trials. How to set control levels of response within these
trials then became a topic of conversation.
More later.
Diagnostics, both rapid and not, were discussed by FDA. On
the more usual antimicrobial susceptibility testing devices, it is clear that
the delays in availability of these devices after antibiotic launch is not
being well addressed by FDA. Until this
occurs, the delays will continue to impede acceptance of new antibiotics by
end users – not a good state of affairs with continuing increases in resistant
infections.This will require some sort of preliminary breakpoint from FDA that would allow device manufacturers to begin their process prior to approval. Everyone will have to work at risk. Will this ever occur???
Stava Epstein gave a wonderful and elegant talk on the iCHIP.
There was nothing really new in his talk, but it was exciting to hear him speak
about this wonderful new technology that may revolutionize the way we look for
new drugs.
Dr. Sumathi Nambiar provided a discussion on the QDIP
designation and on market authorization for products to treat resistant
infections. In the latter portion of her
talk, she admitted that the FDA remains somewhat lost on how to proceed in
pathogen specific trials. She noted that the FDA prefers a hybrid approach with
non-inferiority trials plus some additional data on activity against resistant
infections.
Later, Dr. Nambiar was roped (my fault) into a panel
discussion where the topic of an orphan drug designation for such unmet needs
antibiotics was raised. As I discussed in a previous blog,
the orphan drugs group is still back in the 1990s of antibiotic
development. They only think in terms of
broad clinical indications like urinary tract infection, pneumonia, etc. In
this thinking, the affected populations are much too large to be considered for
orphan drug status (200,000 individuals or less in the US). But a pathogen
specific indication where the drug is indicated only for those with few or no
alternatives (4500-7500 per year in the US for carbapenem-resistant
Enterobacteriaceae for example) has not been considered even though this is
addressed by the unmet needs guidance both in the EU and in the US. As a result
of this discussion, Dr Nambiar agreed to raise this issue with her FDA
colleagues. We await, once again,
further developments.
Finally, the economics of the antibiotic markets was
broached by Kevin Outterson and Barrett Thornhill. The proposals for so-called de-linking, where there would be an attempt by governments to provide a guaranteed return on investment for companies pursuing antibiotics for resistant pathogens seem to
be in limbo in the US since we have no congress. In Europe there is hope that such an approach
will bear fruit – but whether that will occur soon or with the usual tectonic
EU speed is hard to predict. The precise method of implementation of such payments also remains nebulous. These
discussions reminded everyone that, at the end of the day, to progress, we need
money. And, especially in the US
congress, that remains a dirty word.
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