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Pharmacyclics Inc. Message Board

  • riffissimo riffissimo Mar 21, 2012 4:19 PM Flag

    Marqibo Received a Postive Accelerated Approval Vote Today

    Sets a pretty low bar for precedence.

    Key Section of FDA Briefing Document
    =====================================
    Committee members commented that single arm trials may be used in the following situations: 1) rare diseases <<REMAINS TO BE IDENTIFIED FOR IBRUTINIB, BUT LIKELY TO BE DEFINED AS 65+ CLL PATIENTS) and 2) high level of activity of the agent or pronounced treatment effect <<EXACTLY IBRUTINIB'S PROFILE>>. It was also mentioned that the toxicity of the agent must be taken into account in a risk/benefit analysis in the situations in which single arm trials may be used <<EXACTLY IBRUTINIB'S PROFILE>>.

    Overall, members felt that a well designed development plan is needed prior to the application being filed. Most also preferred that the sponsor have studies already ongoing at the time of application. <<EXACTLY WHAT PCYC IS DOING>>

    Complete Section
    ================

    In 1992, the NDA and BLA regulations were amended (Subparts H and E, respectively) to allow for “accelerated approval” in diseases that are serious or life-threatening. Under accelerated approval regulations, for indications where the new product appears to provide benefit over available therapy, accelerated approval may be granted on the basis of a surrogate endpoint that is “reasonably likely” to predict clinical benefit. Under accelerated approval, the applicant is required to study the drug further to verify and describe its clinical benefit. Postmarketing studies would usually be underway at the time of accelerated approval. These post-marketing confirmatory studies (also known as post-marketing requirements) may be either a new trial or completion and final follow-up of patients on an existing trial. In either case, the required post-marketing study must show an effect on an endpoint that reflects clinical benefit. If those studies fail to demonstrate clinical benefit, or if the applicant does not show “due diligence” in completing the trial(s), the regulations describe a process for removing the product from the market.
    Previously Received Advice from the ODAC
    On February 8, 2011, the Office of Oncology Drug Products convened a meeting of the Oncologic Drugs Advisory Committee (ODAC) to discuss the status of drugs approved under the accelerated approval regulations; the following advice was offered from the committee:
    Overall, ODAC members agreed that randomized controlled trials should be the standard and that single arm trials should be the exception. Committee members commented that single arm trials may be used in the following situations: 1) rare diseases and 2) high level of activity of the agent or pronounced treatment effect. It was also mentioned that the toxicity of the agent must be taken into account in a risk/benefit analysis in the situations in which single arm trials may be used. Committee members noted that it would be helpful to have a definition of rare diseases. Members also noted that the bar for accelerated approvals should not be lowered to move products on to the market faster through single arm trials, but rather single arm trials should only be used in certain situations and randomized controlled trials should be the standard.
    Overall, members agreed that at least two controlled trials should be needed for accelerated approval commitments. Most members agreed with this statement with the caveat that in rare diseases and pediatrics this may not be feasible.
    Overall, members felt that a well designed development plan is needed prior to the application being filed. Most also preferred that the sponsor have studies already ongoing at the time of application.

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PCYC
118.54-1.86(-1.54%)Aug 21 4:00 PMEDT

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