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pSivida Corp. Message Board

  • mickmack50 mickmack50 Dec 16, 2013 5:27 PM Flag

    Would A Sustained Release Platform Qualify As "Breakthrough" Designation?

    I've always thought so....what do you think? This information is from the FDA:

    ection 902 of the FDASIA articulates two general criteria according to which the FDA may designate an investigational drug as a breakthrough therapy. First, this designation can be applied only within the context of a “serious or life-threatening disease or condition.” Second, it must be predicated on “preliminary clinical evidence indicat[ing] that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints.” The FDA has interpreted the second criterion to mean that data from studies in animals or conducted in vitro showing that a drug has promise are not sufficient to justify this designation; data from clinical trials in humans are needed.
    Once a drug is designated as a breakthrough therapy, the FDA commits to working particularly closely with the drug sponsor to devise the most efficient pathway for generating additional evidence needed about safety and efficacy. The amount of additional data needed will vary, depending on the disease, the magnitude and robustness of the initial data, and the availability of alternative therapies. The statute also calls for reducing exposure of patients to a potentially less-effective active control drug (i.e., when clinical equipoise is not present). Although this ethical principle is applicable to all development programs, it is especially pertinent to drug development under the breakthrough-therapy program in cases in which impressive early clinical data are available. In such cases, the immediate needs of patients must be balanced on an ongoing basis against the need to generate reliable data to inform therapy. In addition, rapid clinical development for breakthrough-therapy drugs will put more pressure on other components of drug development, such as drug manufacturing: development of a final formulation and scale-up of processes will have to occur more rapidly than they traditionally do. It is possible that manufacturing will become the rate-limiting step in some breakthrough-therapy drug-development programs.
    It is not expected that all products designated as breakthrough therapies will in fact turn out to have the potential suggested by the early clinical data. Subsequent trials may reveal a smaller treatment effect, or unacceptable adverse effects may occur. It is also important to recognize that a breakthrough-therapy designation is not a drug approval. Like all drugs in development, drugs designated as breakthrough therapies will be reviewed by the FDA to determine whether they are safe and effective for the intended use before they can be approved for marketing. This review will be expedited for drugs designated as breakthrough therapies, if the clinical findings warrant doing so. Since enactment of the FDASIA about 1 year ago, more than 80 requests for a breakthrough-therapy designation have been submitted to the FDA, and 26 have been granted. The designations that have been publicly announced by drug sponsors, along with the conditions being studied, are listed in Table 1TABLE 1
    Drugs with Breakthrough-Therapy Designations Announced as of September 30, 2013.

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