In Search of Meaningful GAIN in Renewal of Prescription Drug Act


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capital markets that make it nearly impossible for small biotech companies—the true innovators in this therapeutic area—to finance the late-stage development of new antibiotics under current regulatory guidance.

While the GAIN Act provides advantages for antibiotic drug developers, will it truly accomplish the goal of stimulating the development of new antibiotics that work against drug-resistant pathogens? Such antibiotics are called “Qualified Infectious Disease Products” or QIDPs under the GAIN Act. Let’s look at some of the key provisions:

1. Market exclusivity. QIDPs would get an additional five-year period of market exclusivity beyond the standard periods of exclusivity for which they would qualify. In the U.S., new small molecule drugs are granted 5 years of Hatch Waxman market exclusivity, so this provision could convey a total of 10 years of market exclusivity to a sponsor with a new drug application (NDA) for a QIDP. What this means is that, for a period of 10 years from FDA’s approval of a sponsor’s NDA application, a potential competitor would not be able to use the data generated by a sponsor of the QIDP in a regulatory filing for a generic version of the sponsor’s antibiotic. While not the same as a patent term extension, an additional 5 years of market exclusivity would be advantageous for a drug with a current patent expiry within 10 years of NDA approval. Also, for a drug with a patent expiry currently beyond 10 years from approval, the additional 5 years of market exclusivity provides a safety net in the event of a patent challenge and invalidation. As an example, under this provision of the GAIN act, coupled with the current Hatch-Waxman extension, a new antibiotic approved in 2014 could have guaranteed market exclusivity through 2024 even in the absence of a valid patent.

2. Priority review. Under the GAIN Act, NDAs for QIDPs would qualify for Priority Review by the FDA. As the FDA’s current goal for Priority Review is six months, this provision could cut … 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

    • 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.