In Search of Meaningful GAIN in Renewal of Prescription Drug Act

7/11/12

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in half the time to approval compared to the 12-month Standard Review goal most antibiotics are currently afforded.

3. Fast track review. Sponsors of QIDPs would be provided with early and frequent communications with the FDA, in addition to the typical review and communication opportunities, to expedite the review and potential approval process.

4. Updated guidance. The GAIN Act provides for a specific timetable for the FDA to develop and issue draft and final guidance, and provides sponsors with the opportunity to request written recommendations from the Secretary of Health and Human Services on the guidance for antibiotic trials if such guidance is lacking from the FDA.

5. Pathogen-focused drug development. Requires the FDA to issue guidance on pathogen-focused antibacterial drug development.

While all of these provisions are welcome improvements that will facilitate the development of drugs currently in the development pipeline, they are not sufficiently game-changing to encourage large pharmaceutical companies to re-enter antibiotic development—nor will they likely incentivize private or public institutional investors to create new companies or to double down on their current investments in biotech companies developing antibiotics.

Here’s why:

First, the market for hospital-based antibiotics does not scale appropriately to the growth needs of large pharmaceutical companies. The perverse reason for this is that antibiotics cure acute diseases, which limits their sales compared to drugs needed to treat chronic ailment.

For example, growing the nearly $67 billion in revenue that Pfizer reported last year by 5 percent would require an additional $3 billion in new revenues. Peak sales for a hospital antibiotic would likely top out at about $1 billion before patent expiry. Pfizer’s linezolid (Zyvox), approved to … Next Page »

Jeff Stein is the president and CEO of San Diego's Trius Therapeutics. He also has served as a Kauffman fellow and venture partner with Sofinnova Ventures, and as director of venture development at UC San Diego. Follow @

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  • mgw6

    If the need is for antibiotics to treat these resistant strains then one
    of the biggest challenges for antibiotic development is not just
    finding new antibiotics but finding new antibiotics that can prove they
    are better against these pathogens, with clinically meaningful better
    outcomes, than currently available antibiotics. Providing incentives to
    develop another mediocre (“me too”) antibiotic merely because it has
    MRSA or some other resistant pathogen in its in vitro (lab) spectrum is a
    waste of money. For new antibiotics to qualify for the benefits of
    GAIN, for example, shouldn’t they be required to demonstrate (in the
    clinic, not just in the lab) that they actually can treat multi-drug
    resistant pathogens that are resistant to currently available
    antibiotics?

    • Jeff Stein

      You are certainly correct about the importance of clinical demonstration of efficacy and safety and this is why the FDA is updating guidance for antibiotic clinical trials. Keeping guidance up to date with the current science is a key objective. The challenge in an antibiotic clinical trial setting, however, is how to design a trial that is ethical, feasible and generates statistically meaningful results. For example, as much as we would like to design a trial that targets patients with linezolid resistant strains, to demonstrate that tedizolid is effective and linezolid is not, we cannot ethically conduct such a study knowing that 50% of the patients in the double blind linezolid controlled study will likely fail treatment. However, we can conduct animal studies to demonstrate this and there is ample evidence that the results of such studies for antibiotics translate to human clinical efficacy. With respect to your question about the GAIN Act, the QIDP designation is only given to drugs that have an approved new NDA based on clinical efficacy, not in-vitro activity against MRSA (in this example). In the article, I advocated for a clinical trial design that is more consistent with how clinicians treat infections and this is entirely consistent with your comments. Points well taken.