Limited Population Antibiotic Development (LPAD) is a
proposal from the Infectious Diseases Society of America (IDSA) to streamline
antibiotic development for patients with “unmet Need” – that is those with
antibiotic resistant infections whose choices for therapy are very limited or
non-existent. Recently, Helen Boucher of
IDSA has published an opinion
piece on CNN where she makes a compelling case for the need for new antibiotics
with a moving case example. She notes that the LPAD legislation, which would
enshrine this regulatory approach for antibiotics, is currently moving through
congress. The New York Times also covered this topic
stating that the “administration” was pushing LPAD legislation. Of interest, the Times article also quoted an
opposing voice – that of John Powers – who stated that the limited trials
proposed by IDSA would not prove that the new antibiotics were effective and
that there would be an increased safety risk because the amount of patients
studied would be smaller and therefore the study would be less able to pick up
uncommon safety concerns. While what
John says is true, the alternative is to have no new antibiotics in the
pipeline. Which would you rather have; untreatable
infections or new antibiotics where the risk benefit ratio might be more like
what we currently have for cancer drugs?
Helen believes that this rapid regulatory pathway will
entice more pharmaceutical companies into antibiotic research – something that
is essential if we are going to bolster our pipeline. While LPAD is required, it unfortunately will
not be sufficient. We also need to
provide companies a return on their R&D investment. This debate has recently come to the fore
with the announcement
by Astra-Zeneca that antibiotic R&D would no longer be a priority area for
them. The bottom line is that a number of companies do not have confidence in
the increased reimbursement that has been discussed
for new antibiotics developed under LPAD.
It is clear to me that both things must occur. We must have a feasible and rapid regulatory
pathway to approval for antibiotics for resistant infection and at the same
time we must provide companies confidence that their R&D efforts will be
financially rewarded. Unfortunately, it is likely that some regulatory reform
will come first and confidence in pricing structures will only come later if at
all.
There are aspects of the regulatory discussion that I find
disturbing, however. It seems as if all progress towards bringing this pathway
forward at FDA has halted while they wait and see if congress will pass the
legislation. The FDA wants the legislation,
in my view, because they want congressional cover in the case that one of the
new antibiotics approved under this more risky pathway runs into trouble –
something I believe is likely to occur.
The FDA does not want to go through another Ketek experience with
congress. I do not necessarily disagree that having this legislation would be
nice from this point of view. But I’m
not at all convinced that even such legislation will provide the cover they
want. In addition, it seems highly controversial whether this legislation is
actually required on a statutory basis.
It seems to the non-regulatory-lawyer (me) that is it not. Yet we may
have to wait for new guidance while congress dithers. To me, this is unacceptable. If passage is imminent – before the summer
break – ok. If not – the FDA should just
bite the bullet and get new draft guidance (based on multiple discussions with
sponsors and at Brookings) out there.
Without new guidance, the industry is stuck in neutral. But even with new guidance, we will only get
to first gear when the financial issues are clarified.
No comments:
Post a Comment