P

resident Donald Trump wants to remake the Food and Drug Administration. And he happens to have a “fantastic person” in mind to do it, he said Tuesday — someone who will turn the agency into an industry-friendly shop that cranks out new cures on the double.

But does anyone really want a deregulated FDA? STAT canvassed biopharma insiders, physicians, Wall Street analysts, and FDA veterans. Their verdict: Trump’s plan sounds like a solution in search of a problem.

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  • No need to blow up the FDA. The FDA needs a return to sanity, though. Right now they are rejecting good drugs based on messed up, insane, stupid logic. The FDA needs more money, transparency, and brains.

    For an in-depth dissection of one horrible FDA decision, see my article entitled “Cempra’s Solithromycin and the Great FDA Echo Chamber” here (warning: bit of a long read):
    http://seekingalpha.com/article/4044221-cempras-solithromycin-great-fda-echo-chamber

    • They also are approving ineffective and even dangerous drugs that are marketed for years before dangerous side effects are acknowledged.

    • Steve: I agree. But sometimes there is no other way; in any case, the whole process currently favors Big Pharma, big time (they can weather all these things, and maybe even pay people off). Transparency and accountability is very much needed.

      For example, they shouldn’t invite someone into an advisory committee meeting regarding a 505(b)(2) submission who doesn’t understand what a 505(b)(2) is! (see: KMPH adcom mid-2016)

  • I think you were referring to Solithromycin from Cempra. The P2, P3 and NDA were all executed in line with FDA guidance. Soli is being used in Pediatric trails extensively and a single pill cures Gonorrhea. As a Macrolide it has a pretty safe profile.

    http://investor.cempra.com/releasedetail.cfm?ReleaseID=959020

    From the company’s website:

    “Based on their review of the NDAs, the CRL stated that the FDA determined the risk of hepatotoxicity had not been adequately characterized. The FDA noted the size of the safety database is limited to 920 patients who received solithromycin at the proposed dose and duration, and is too small to adequately characterize the nature and frequency of serious hepatic adverse effects.

    To address this deficiency, the FDA is recommending a comparative study to evaluate the safety of solithromycin in patients with CABP. Specifically, the CRL recommends that Cempra consider a study of approximately 9,000 patients exposed to solithromycin to enable exclusion of serious drug induced liver injury (DILI) events occurring at a rate of approximately 1:3000 with a 95 percent probability.”

    Now, whose fault it is if the risk is not adequately characterized? The company followed instruction and guidance to the letter. But is now asked to do another P3 with 9000 patients. WOW.

    But, was there even a risk in the first place? They just have a doubt that there was DILI. There was not a single case of DILI in any patient in the trials (no case of Hy’s law). The summary on Cempra site states:

    “The CRL noted that while the FDA reserves comment on the proposed labeling until the NDAs are otherwise adequate, even in the absence of a case of Hy’s Law or of another form of serious DILI in future studies, labeling will need to include adequate information about the potential for hepatotoxicity, limiting use to patients who have limited therapeutic options and limitations regarding duration of therapy. A comprehensive plan for post-marketing safety assessment including an enhanced pharmacovigilance program would also be required.”

    Dr. Bhavneesh Sharma MD’s detailed analysis on safety:

    http://seekingalpha.com/article/4018412-cempra-inc-take-fdas-question-liver-enzyme-elevation-due-solithromycin

    https://twitter.com/funky_omnivore/status/814501824346603520

    They destroyed this company. Was Cempra pulling a fast one on them? Or, did the company invent a better antibiotic than the current standard of care (which is 60% resistant) and is desperately needed the world over? Moxifloxacin, the current standard of care, has so many lawsuits. Just google. Even, Soli pricing was so good that it was unbelievable! But all dead, thanks to FDA.

    Also, you stated: “FDA does take too long in many cases.”

    The fact is that FDA takes too long in all the cases. When efficacy and safety have been adequately proven, why force a P3 and then take another year for an NDA?

    They take one whole year!!! And then they find something minor (relating to GMP) and the company stands in the NDA queue again.

    WOW, FDA.

  • FDA does take too long in many cases. Multiple successful phase 2 and phase 3 and successful adcom, recently for an antibiotic. Then on padufa date they issue an arbitrary CLR, all this while the world suffers because of antibiotic resistance.
    Many companies prove both safety and efficacy in phase 2 but multiple phase 3 are mandated. After that, they review it for another year. While the world awaits medicine for important cures. Something does need to be done.
    Trump plan is not an solution looking for a problem. Yes, there needs to be validation, but fda is known to be a corrupt organization where a good medicine can be given a CLR in guise of a unsubstantiated adverse effect, possibly to favor another Pharma. They know that lower dose doesn’t cause adverse but is still efficacious, but they give CLR and destroy not only a cure but 10 s ofyyears of drug makers efforts.
    FDA needs to be quick and clean. Change can be a good thing if done right.

  • Just as the FDA has set up offices in China and India for inspections / investigations – it will be worthwhile to have expert dossiers’ review staff stationed in India (including few Indians from the industry). They can have a better control & hold on review to expedite regulatory approvals. They can quickly visit and check raw data as well at factory site.
    Director – GMP PHARMA (I) Pvt. Ltd.

  • Of course established industry players love the FDA. It’s the largest barrier to entry ever erected in any industry. There is a reason that Pharma has returned 23% on invested capital since 1963 (12% higher than market average returns – and the highest ROIC of any industry over that time period).

    Pick 15 countries, allow drugs approved in those countries to be sold in the USA. Then to lower drug costs, allow imports from those countries. Hold the FDA accountable by making it compete.

  • The FDA suffers from one major flaw- Croneyism. The agency promotes long-time FDA employees with no degrees into senior-level positions, simply because they have stuck around for so long. I know of several GS15 level Directors that were promoted up from the former secretarial pool decades ago. They joined the FDA right out of high-school, never took a single college course, and eventually made Division Director. These people get these positions because they band together with others just like them and hold jobs for each other. As they rise through the ranks, they are not trying to make the FDA better, they are trying to figure out how to get the best yearly bonuses and retire with the most money possible. They piss on their territories; they don’t let their employees talk to other FDA employees outside of their croney group, they don’t let other FDA’ers collaborate on projects outside of the croney group, they do the minimum amount of work they can get away with, and they don’t hire from outside unless they think the person is controllable and will abide within their established boundaries. If I were on the FDA Leadership team, I would order desk audits for anyone in one of these positions and all their employees- find out what they do every day. After the fourth time my Director came to me for help with the daily Word Jumble and I caught another watching a portable TV she hid in her desk, I think I have a good idea of exactly what they are doing. Don’t blow it up, just do a massive re-org and evaluation of all the employees. If they have been at the FDA for more than about 10 years, send them on a detail to another agency to have their work capabilities critically evaluated by people outside of their circle. There needs to be some way to determine if an employee is incompetent. And if they are found to be such a person, get them out!

    • Well said. I have seen this in FDA. Not only these uneducated people get promoted, but they obstruct hard working individuals with advance degrees (PhD MD) and wealth of scientific Knowles.

    • I do support stricter regulation as medicines are for health of mankind. Regulation should be stringent for drugs which are irrationally introduced for plain business reason (which are not really for mankind’s gain). In the past there have been many drugs introduced through FDA approval which later were found to be irrational or ineffective or unsafe. While new drugs may take time for approval – why should essential drugs under NDA regime and the generic drugs under ANDA regime take a long time for approval. There needs to be an even balance. FDA needs to be supported by expert reviewers and not just promotees with inadequate or inappropriate education and experience. Experts can understand the science behind product development, Quality systems, associated Risks and GMPs required for the product manufacturing. Ordinary reviewers send silly queries seeking explanation and additional information – delaying the process of approval. Conclusively, Trump should look for reorganizing FDA with experts.

  • I support Mr President on many things but strongly oppose this proposal. FDA is our safeguard and their current regulation works well so far. patients safety should be the priority, far more important than jobs and profit. It’s always good to improve the efficiency of the agent, which they are keep doing, but such a storm change is surely a bad thing.

  • These articles i keep reading have a surprising hint of liberal thinking
    . If change doea not occur , we will have no future to dream of .. A Veteran of the United States of America . Lets certainly not upset the politicians and Drs and large Pharma ..how would they survive ? My God ! Change ? Post this . I dare you

    • It’s a blog focused on healthcare professionals, who tend to be liberal-leaning on healthcare funding. And big pharma, thanks to iterations of PDUFA, has developed a working cadence with the FDA–they benefit from regulation through reduced competition and a marketplace who believes FDA approval=safe and effective. No surprise they have a perspective.

  • The point about FDA changes resulting in a skewing of the market towards big players with YUGE marketing budgets is well made.

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