In Search of Meaningful GAIN in Renewal of Prescription Drug Act

7/11/12

The repercussions of the Supreme Court’s recent ruling on the Affordable Care Act have largely overshadowed another key healthcare milestone—President Obama’s signing Monday of the Prescription Drug User Fee Act, also known as PDUFA V. Originally passed in 1992, PDUFA V re-authorizes the FDA to collect fees from drug sponsors to speed the review and approval of new drugs, and has been cited as a key reason why the U.S. is considered a world leader in providing patients access to innovative medicines. PDUFA is one of the rare pieces of legislation that has broad support among patients, the government, industry, and academia. Its timely reauthorization strengthens the U.S. healthcare system and facilitates access to potentially life saving new medicines.

One important area of life saving medicines highlighted in PDUFA V focuses on antibiotics, which I’ve followed closely as the CEO of San Diego’s Trius Therapeutics (NASDAQ: TSRX). The “Generating Antibiotic Incentives Now,” or GAIN Act, contains provisions that provide industry with incentives to develop innovative antibiotics to treat life-threatening infections caused by drug-resistant pathogens.

Why, you might ask, do we literally need an act of Congress to provide incentives to drug developers, especially in a political environment in which such actions have been viewed as “corporate welfare”? The answer lies in a near-perfect trifecta of ominous trends: the alarming growth of multidrug-resistant bacterial pathogens; the abandonment of antibiotic drug development by large pharmaceutical companies; and challenging … 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.