Sunday, June 16, 2013

LPAD - The Full Court Press

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.

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