2013 was an eventful year in the antibiotics world – not
all good but not all bad either.
Both the FDA
and Europe
released new guidance on developing anti-bacterials for highly resistant
infections and other unmet needs. The EU
guidance is much more user-friendly than that from FDA – but both provide
feasible ways forward for very small trials for antibiotics targeting limited
populations of patients with such unmet needs.
In the context of the FDA reboot, we began a discussion in
earnest and with payers around the necessity for value-based pricing for new
antibiotics active against resistant strains.
These new antibiotics would be developed along the lines of what the FDA
calls anti-bacterials for unmet needs in their guidance
released in July. The trials would be very small and the clinical data would be
supported heavily by preclinical data and both clinical and preclinical PK/PD.
Because the populations targeted by these drugs would be small, the industry
would need to charge a high price for each treatment course in order to make a
return on investment. This will be true
in spite of the smaller, less expensive trials required for achieving market
approval. The reduced expense will not
be enough to allow sales at currently accepted prices. The meeting at the Pew
in January confirmed that payers are willing to take on these high prices for
products that can be shown to work.
Payers seem used to this idea based on their experience in oncology
where drugs offering very little in terms
of value are already given high prices.
Further support for the concept comes from a paper
published by Rex and Spellberg analyzing the cost-benefit of this approach and
clearly showing that even very high prices can be supported by the benefits of
new antibiotics that work where others don’t (if you don’t have a subscription
to Nature – check my blog).
The development of feasible regulatory pathways for small,
rapid and less expensive trials to register antibiotics targeting resistant
infections plus value based pricing for such drugs provides the boost we need
to get large pharma interested again in antibiotics. Since these events, Cubist
has purchased both Trius with its tedizolid being submitted to FDA and EU and
its preclinical programs and Optimer with dificid for C. difficile infections.
The two deals amounted to up to $1.6 billion. Roche
entered into a licensing agreement with Polyphor for their new peptimomimetic
antibiotic that is highly specific for Pseudomonas
aeruginosa. This represents the first real venture into antibiotics (with
one minor exception) for Roche since 1999 when they were the first large pharma
company to announce they were quitting the field. I fully expect this trend to continue in 2014 with increased investment in antibiotic R&D from venture capital and other private sources of funding as well as additional large pharma companies re-entering the field.
In spite of all these activities, Astra-Zeneca,
which has been the stalwart company for antibiotic R&D over all these
years, announced that it would limit its investment in the area while awaiting
further developments. Basically, the CEO
does not believe that value-based pricing will occur to the extent required for
his company to make a sufficient return on their investment. Going forward, I
am optimistic and believe that the CEO will realize the error of his ways – but
nothing would surprise me from AZ at this point.
The low point of the year for me was the loss of John
Quinn. I hope to have more to say about
this in 2014. But I know that John lives on and, like me, awaits the awakening
of the sleeping giant that will be a new era of antibiotic R&D to provide
the medicines that we need now and that we will need in the future.
The blog will continue.
I am retiring from my consulting business (see below), but not from
blogging. 2013 and 2012 were both big years for the blog. Since its inception,
the blog has seen 138,000 page views from 38,000 different visitors. 2012 and
2013 were the biggest years for the blog with around 23000 page views each year
compared to 12,000 in 2011. The most popular blogs were Lew
Barrett’s piece on the market for a limited use antibiotics (4500 page
views), the tribute
to John Quinn (3800 page views), the FDA
and CABP (3600 page views), rebooting
hospital acquired pneumonia (3500 page views) and my piece on antibiotics
in 2012 looking forward from 2011 (3028 page views).
I’m looking forward to 2014 and my retirement from
consulting. I apologize to all those of
you who wanted me to work with you this year and in the future – but all good
things must come to an end sometime. I am hoping, though, as I noted in a
previous blog,
to be able to work more closely with the FDA to continue to implement their
reboot of antibiotic development in a way that makes sense. I hope to be able
to tell you more about that early in 2014. I’m now working hard on my next
book, my guitar, and the blog, but I also have time to spend with my family and
for travel that does not involve work.
To everyone out there in blog-land – many thanks for your
continued support! Have a great New Year!
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