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left2rightdoor 63 posts  |  Last Activity: Apr 17, 2014 12:50 PM Member since: Jul 16, 2012
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  • Reply to

    Washington Post

    by greblok Apr 17, 2014 12:26 PM
    left2rightdoor left2rightdoor Apr 17, 2014 12:50 PM Flag

    How about a simple message:

    No Health and Human Services Secretary confirmation without DMD medication!

  • Reply to

    Michael Astrue article in Weekly Standard

    by greyzone513 Apr 14, 2014 1:19 PM
    left2rightdoor left2rightdoor Apr 14, 2014 2:02 PM Flag

    In 1991 the FDA approved Genzyme’s Ceredase for a very rare fatal condition called Gaucher disease in a non-blinded trial of only 12 patients; the drug has allowed almost all patients to lead almost normal lives.

  • And every member of the Senate including Senators Harkin of Iowa and Enzi of Wyoming, the sponsors of S3187, FDSIA of 2012, should be prepared to ask about the Duchennes boys and whether FDA is fully implementing Sec 506 regarding fast track, compressed trials, smaller groups, and accelerated approval

    Still, Burwell is relatively unknown outside the White House, as one of the few actions that got her widely noticed was her ordering the first government shutdown in 17 years, NBC reports.

    But the White House is confident in its pick, who will no doubt face a contentious confirmation process as Republicans will be sure to pepper her with questions — as they did Sebelius — over the shortcomings and technical glitches of the Obamacare rollout.

    Battling with Republicans isn't new for her either. She often butted heads with Republicans for delaying spending bills in 2000, telling them "we will sign short-term [continuing resolutions] as long as it takes to get the job done," The Wall Street Journal reported.

    How about "we will sign petitions and letters as long as it takes to get Eteplirsen and follow on exon skipping quickly to all DMD patients"

  • —The actions to expedite the develop- ment and review of an application under subparagraph (A) may include, as appropriate— ‘‘(i) holding meetings with the sponsor and the review team throughout the development of the drug; ‘‘(ii) providing timely advice to, and interactive communication with, the sponsor regarding the development of the drug to ensure that the develop- ment program to gather the nonclinical and clinical data necessary for approval is as efficient as prac- ticable; ‘‘(iii) involving senior managers and experienced review staff, as appropriate, in a collaborative, cross- disciplinary review; ‘‘(iv) assigning a cross-disciplinary project lead for the Food and Drug Administration review team to facilitate an efficient review of the development pro- gram and to serve as a scientific liaison between the review team and the sponsor; and ‘‘(v) taking steps to ensure that the design of the clinical trials is as efficient as practicable, when sci- entifically appropriate, such as by minimizing the number of patients exposed to a potentially less effica- cious treatment.’’; and

  • The FDSIA also included subsection(c) Guidance; Amended Regulations, requiring issuing draft guidance to implement the FDA amendments within 1 year and requiring the Secretary to "specifically consider issues arising under the accelerated approval and fast track processes under Sec 506" of the FDA. The Draft Guidance was issued June 2013, with questions to be directed to the CDER Office of Medical Policy 301-796-2500, or CBER Office of Communication 800-835-4709. Comments, including comments from DMD advocacy groups were made. (2) Final Guidance Not later than 1 year after the issuance of draft guidance....the Secretary shall (A) Issue final guidance. So the clock is now running for the Final Guidance to come out in June 2014 under the law.

    Meantime the FDA sends out letters to interested parties: We recognize the huge unmet medical need in Duchenne muscular dystrophy, the devastating nature of the disease for patients and their families, and the great urgency to make new treatments available.

    FDA is clearly on the hot seat here. If their guidance is serious, they must recognize the DMD illness as a long term illness, serious need, and unmet medical need requiring treatment, not new, because there are no existing treatments.

  • Reply to

    Hamburg quote about new drug application

    by mitchpark74 Apr 6, 2014 11:02 PM
    left2rightdoor left2rightdoor Apr 7, 2014 12:51 AM Flag

    As previously posted, Sec 901 of the FDSIA modify section 506 of the Food and Drug Act to provide for review of incomplete applications, and the FDA beginning early, if the sponsor can then provide a schedule for additional information which may be needed. This seems to map pretty well to this situation where there is early indication of effectiveness in multiple patients, a serious, life threatening long term condition, and zero available treatments if the fast track product is denied.

    (c)Review of incomplete applications for approval of a fast track product
    (1)In general
    If the Secretary determines, after preliminary evaluation of clinical data submitted by the sponsor, that a fast track product may be effective, the Secretary shall evaluate for filing, and may commence review of portions of, an application for the approval of the product before the sponsor submits a complete application.

  • left2rightdoor left2rightdoor Apr 5, 2014 6:48 AM Flag

    Sec 506 damn tiny keyboards

  • sec 901 of S 3187 amended Sec 596 to provide dor review of incomplete applications if there was a schedule for further info

    (c)Review of incomplete applications for approval of a fast track product
    (1)In general
    If the Secretary determines, after preliminary evaluation of clinical data submitted by the sponsor, that a fast track product may be effective, the Secretary shall evaluate for filing, and may commence review of portions of, an application for the approval of the product before the sponsor submits a complete application. The Secretary shall commence such review only if the applicant—

    (A)provides a schedule for submission of information necessary to make the application complete; and
    (B)pays any fee that may be required under section 736.

  • Feds are still worried, very worried. Excerpt from change April 2

    In addition to the critical need for new classes of antivirals and immunotherapeutics, the recent H1N1 pandemic highlighted the need for drugs that can be used later in the course of infection to treat severely ill, influenza infected patients who are hospitalized. Currently, there are no treatments approved for severely ill, hospitalized influenza patients. Many of these patients also suffer from secondary bacterial infections or increased sickness due to an overactive immune response. To make available effective treatments for this population BARDA will support the development of host-directed therapeutics, such as small molecule immune modulators, anti-inflammatory agents, and regulators of innate immunity that have been shown to be effective against influenza.

  • left2rightdoor left2rightdoor Apr 3, 2014 1:31 AM Flag

    Vs this?

    Severe influenza A H7N9 pneumonia with rapid virological response to intravenous zanamivir
    Pak-Leung Ho1, Wai-Ching Sin2, Jasper #$%$-Woo Chan1, Vincent Chi-Chung Cheng1 and Kwok-Hung Chan1

  • left2rightdoor by left2rightdoor Apr 2, 2014 9:57 AM Flag

    (C)As a result of these remarkable scientific and medical advances, the FDA should be encouraged to implement more broadly effective processes for the expedited development and review of innovative new medicines intended to address unmet medical needs for serious or life-threatening diseases or conditions, including those for rare diseases or conditions, using a broad range of surrogate or clinical endpoints and modern scientific tools earlier in the drug development cycle when appropriate. This may result in fewer, smaller, or shorter clinical trials for the intended patient population or targeted subpopulation without compromising or altering the high standards of the FDA for the approval of drugs.

  • left2rightdoor left2rightdoor Mar 30, 2014 1:01 PM Flag

    Has the FDA ever published the "draft guidance"promised at p.21 of Driving Biomedical Innovation released by Hamburg and FDA in Sep2011

    To respond to these challenges, FDA will hold a series of scientific meetings with academic investigators, patient groups, drug developers, statistical and methodological experts, and ethicists to achieve a common understanding of steps that can be taken when an investigational drug being studied for a serious disease with no acceptable treatment option shows exceptional promise. CDER will then publish a draft guidance on an expedited development pathway based on the outcome of these meetings.

  • The early discovery of a potential breakthrough therapy raises ethical and trial design issues. It is important to gain confidence that the effects seen in the early trials are real, and to understand the safety risks of the new drug. On the other hand, from an ethical standpoint, it is important to make sure that people with serious diseases are getting the best possible therapy. In these situations, the clinical trials for the drug's development must be compressed and the evidence about its effects gathered in the most efficient manner possible; however, there is not a good understanding in the biomedical community about how to accomplish this.

    Additionally, there are questions surrounding the use of an expedited drug pathway, such as:

    Can FDA, drug developers, and investigators agree on a threshold to determine when a treatment poses “exceptional promise” and should thus be treated in an expedited fashion?
    Can seamless drug development programs be created to utilize natural history data or adaptive trial design concepts to compress drug development time?
    What are the ethical issues involved in identifying a promising intervention? How should the needs of all patients with the disease be balanced against the need for better therapy for an individual?
    Can surrogate outcome measures that could be used for accelerated approval be rapidly identified?
    Can we arrive at a consensus view of the goal of monitoring commitments companies will make once a product is on the market after such a development program, such as scientific expectations and timelines?
    To respond to these challenges, FDA will hold a series of scientific meetings with academic investigators, patient groups, drug developers, statistical and methodological experts, and ethicists to achieve a common understanding of steps that can be taken when an investigational drug being studied for a serious disease with no acceptable treatment option shows exceptional promise. CDER will then publish a draft guidance

  • left2rightdoor left2rightdoor Mar 27, 2014 12:17 AM Flag

    How do you find stuff like this = you check the FDA Advisory Committee website every so often to see what meetings and topics are scheduled, and what new releases FDA has made. Obviously, FDA is feeling heat because A) They have authority to use accelerated approval but B) AA is necessarily based on early data
    for a disease with severe moribidity consequences, no alternative effective treatment, but uncertainty. So they
    can issue an AA but with labeling that says, here's what this drug is indicated for, based on these clinical studies, but it is uncertain whether it will be effective, so the Accelerated Approval is conditional, based
    on either further studies or confirmatory trials. 3 possible outcomes after AA with labels: Approval after confirmatory trials, withdrawal of approval after problems or lack of effectiveness shown, or continuing "contingent" approval. So for DuchennesMD, and exon skipping, AA with acknowledgement of limited trials,subject to possible withdrawal after confirmatory trials, still makes the treatment available to those pediatric patients who desperately need it. FDA should label all they want, and issue AA to give the DMD community access while laying out the limited,but very positive, data on which AA is based.

  • The following is an example of how these three elements would be represented in the indications and usage statement in the full prescribing information:
    Drug X is indicated for {state indication}. This indication is approved under accelerated approval based on {state effect on surrogate endpoint or clinical endpoint that supported the accelerated approval} [see Clinical Studies (14.X)]. An improvement in {identify the specific clinical benefit that remains to be established} has not been established. Continued approval for this indication may be contingent upon {either (“verification and description of clinical benefit”) or (“demonstration of” followed by identification of the particular expected clinical benefit(s) that will be the objective of the postmarketing study)} in confirmatory trials.

  • See FDA 3/24 Guidance for labeling to let PATIENTS and FAMILIES decide whether to use an AA drug

    Guidance for Industry Labeling for Human Prescription Drug and Biological Products Approved Under the Accelerated Approval Regulatory Pathway

  • Discusses potential values and risks, competition, censorship differences between China and US on legal remedies for shareholders in dual share structures

  • left2rightdoor left2rightdoor Mar 19, 2014 10:45 AM Flag

    Possible timeline By Mar 31 Completion of stability testing for peramivir if held up to 10 years; Presolicitation notice by Jun following review of stability and discussions with FDA on regulatory status; Acquisition proposal in 3Q for SNS at 5-20% of Tamiflu level - this is all speculation, but Tamiflu solicitation indicates stockpiling is continuing for pandemic preparedness DYODiligence

  • Solicitation Number:
    2014-N-16420
    Notice Type:
    Presolicitation
    Synopsis:
    Added: Mar 18, 2014 10:56 am
    This is notice of intent to negotiate and award a sole source contract and is not a request for competitive proposals. The Centers for Disease Control and Prevention, Office of Public Health and Preparedness, intends to negotiate and enter into a contract with Genentech as a result of receipt of an unsolicited proposal for the distribution of proprietary FDA licensed antiviral drugs during a pandemic emergency. The Government intends to negotiate and award to only one contractor using other than full and open competition procedures pursuant to the authority of USC 2304(c)(1) pursuant to FAR 6.302-1(2)(i), supplies or services needed by the agency are available from only one responsible source and no other type of supplies or services will satisfy the needs of the agency. A determination not to compete this requirement is based on market research and is solely within the discretion of the Government. This announcement is not a request for competitive quotations.

  • Coming 2015 on youtube with Charlie Brown and Snoopy

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