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This weekly column offers opinions on the latest pharmaceutical industry news.

Imagine being prescribed a medicine when neither your doctor nor the manufacturer has any clue whether it will actually work — because the government never required it to be tested for effectiveness.

That’s not how things are done now, because federal law requires drugs to undergo clinical trial testing to gauge benefits and risks. But the incoming Trump administration may seek to undo a decades-old standard of evaluating drugs for effectiveness — to the detriment of every American who takes a prescription medicine.

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  • I would like to see better pathways to investigational drugs for patients who are completely out of other options. But to think this could work as national policy is just so wrong. Patients don’t get to decide what drugs they take. Even our doctors get overruled – by our insurance companies. You think one of those companies is going to approve anyone to take medications that aren’t proven effective? Unlikely.

  • The FDA needs a complete overhaul. Their slow , 20th Century, 7 -10 yr. approval system is costing thousands of lives each yr. Just as it did during the AIDS crisis . My wife’s miracle cancer drug , kadcyla, is a case in point. It appears to be a cure for many terminally ill breast cancer pt.’s but was held up for 3 extra yrs. in the slow approval system – as 15,000 Americans , mostly women died from that specific type of breast cancer EACH of those yrs. Iclusig , a leukemia drug , also appears to be cure for thousands – yet the same FDA tried to deny it approval . The nit picking over the duchennes drug , the current slowness over the SMA drug , nusinersen – we pt. activists have had it !! 21st century cures is a step forward but it’s compassionate use reforms were gutted – but we are not giving up – we want real life evidence to be utilized . we want the whole FDA/ Pharma system overhauled . from speedier trials to quicker expansion of restricted approvals, to real compassionate use . these pharma co.’s need to wake up too – they are not making a new snazzy smart phone — they are producing 21st century cure drugs. My wifes’ stage iv cancer melted away in 2010 – but no one listened to her testimony – the FDA of that time didn’t listen to the top breast cancer researchers at the time either . Enough is enough .

  • Because of statutes and bureaucratic resistance, the most Jim O’Neill as FDA Commissioner would be likely to achieve is a relaxation of efficacy standards when there is scientific support for that relaxation. Such relaxation required a dramatic public movement more than a quarter of a century ago with HIV-AIDS meds. I was then a leader of the drive to speed FDA approval of AIDS drugs. A tipping point was reached in 1996 with the approval of protease based ARV cocktails. FDA resisted early approval proposing to run instead “large simple trials” which would have given better efficacy data while it delayed approval of the cocktails 3-5 years. That delay would have cost hundreds of thousands of lives. It was only because AIDS activists across the country, supported by leading doctors such as 1996 Time Man of the Year David Ho, pushed hard and were backed by Newt Gingrich and others in Congress that the drugs were approved in a timely fashion. Had FDA’s penchant for ever more rigorous efficacy testing prevailed, the loss of life and the increased suffering would have been far greater than thalidomide combined with all the other mistaken approvals FDA has made.

    There are dangers in both speed of drug approval and in delay: but history shows that the delay in treatments results in more suffering and lost lives. Additionally, delays increase the cost of drug development resulting in fewer products being developed. Costly delays mean that products for small disease populations are simply not developed. Unless your disease has a big population and market, you’re out of luck.

    Moreover, we must not forget that under the current system patients and insurers must pay for drugs even when they do not work. As a Crohns patient, I learned that lesson the hard way. When I first came down with a life-threatening Crohns attack 7 years ago my MSA was quickly exhausted with just the copays on drugs that failed to work and, indeed, made me sicker. These may have worked for some, but for me and many others they were neither safe nor effective, only God-awful expensive.

    The author, along with other Jim O’Neill critics, appears to have forgotten the lessons of AIDS activism along with its achievements. Like O’Neill we challenged FDA orthodoxy and found it to be fundamentally a self-serving defense of the bureaucratic status quo. If O’Neill’s critics had a better grasp of history and more respect for the signal achievements of AIDS activists, they might be able to see that O’Neill could prove to be just the antidote to bureaucratic sloth and inflexibility that is needed to open new doors to innovation.

    Again seeing with the eyes of a longtime AIDS activist, I cannot but wonder if, in view of O’Neill’s close ties with his mentor Peter Thiel, the disrespect evident in this article and others like it is not rooted in the same homophobic biases that AIDS activists had to fight two and three decades ago in our life and death struggles to get through to a hidebound FDA and to incognizant Washington policy makers.

    • This is insightful. People who have become ill from FDA-approved medical and dental devices are ignored as collateral damage in the risk-benefit calculation, and we are enraged. We are harmed by “standard of care” medical and dental care. Yet because of vested interests that promote these devices, we are not believed because there are not yet enough reports in the “credible” medical and dental journals, which are run by associations with conflicts of interest.

      Imagine fighting for removal and reassignment of installed devices that are fueling inflammation in your body, because your system cannot handle an FDA-approved neurotoxin.

      You are not a substance abuser, for which there are programs and treatments. You are substance abused. You could get your gender reassigned to match your gene type, but not your dental fillings reassigned to match your gene type. The FDA, your dental insurer and your health plan says Go Fix Yourself if mercury accumulates to toxic levels from amalgams. Others face difficulties getting approval, need for step-wise treatments, and high copays and deductibles to remove uterus and ovaries if they cannot tolerate nickel or PET fibers in Essure, polypropylene mesh in Mesh, or other materials in implants, stents and more.

      All it takes is screening for biocompatibility of each patient before any medical or dental device is installed. Something the FDA recommmends for hair color.

      We need civilian control over the FDA, just as we need civilian control over the military. And a reboot of the system to a Precision Device framework to insure they are “right for you” before being installed. Today’s badly dated rules are causing a world of hurt, harm and health care costs.

    • All good points. Thanks to groups like ACT-UP and the New York Gay Men’s Health Crisis AZT was approved in 25 months based on a single trial, a record at the time.

      Unfortunately some other politicians were the biggest obstacles to getting effective treatments to the most vulnerable. The biggest culprit of all was New York City Mayor Ed Koch, who, as a tortured way of denying his own homosexuality refused to lend assistance or funding to the GMHC.
      The Feds can get it done when they want to. I worked for Abbott labs when they received FDA approval for the first AIDS blood test in another record breaking time. Unfortunately their pharmaceutical division turned around and reamed the patients by raising the price of Norvir to unconscionable levels

  • We need a radical new approach to medical device safety at the FDA. It is pretty simple: pretest people for tolerance vs. toxicity to the metals, plastics and other ingredients dentists and doctors install in our teeth and our bodies. It is what the FDA recommends for hair color.

    Why is this so hard, when it is so simple?

    Second, tell people without exception what each device is made of, potential side effects, risk factors, and how to report harm using the new MedWatcher app, download at Why do we get labels on drugs, OTCs, personal care products, food products, and pet food, but not devices installed in us 24/7/365?

    Third, the “learned intermediary doctrine” is an artifact from past centuries, and should be retired into history. It is outdated and risky in the era of conflict-driven medicine.

    Fourth, allow patients recourse when a device causes harm. FDA, device manufacturers, and groups such as the ADA which have historical product, commercial and trade ties enjoy a get out of liability free card due to a court decision. Who is responsible for fixing the harm? The patient, of course.

    You can’t make this stuff up…

  • “Imagine being prescribed a medicine when neither your doctor nor the manufacturer has any clue whether it will actually work.”
    This is already happening according to “The hidden side of clinical trials” by Sile Lane at TEDx [email protected]://

  • Running clinical trials to prove effectivenss has become extremely time consuming and expensive. If this trend continues unabated innovation will suffer and drug prices will continue to spiral out of control. Investing in companies dedicated to pharmaceutical innovation has become a game of Russian roulette as a slew of FDA reviewers, often with little understanding of the medical condition and the technology employed to treat it, dig their heels in, inflict delays, misinterpret results, and ask for ever more and bigger studies. The proposal here isn’t new, and it is worth consideration. Reverse direct-to-consumer advertising for prescription drugs and let patients and their physicians decide whether or not a (safe) new product is right for them. The industry would have to agree to price controls, which, in the absence of proven effectiveness, would also be needed to get on insurer’s formularies of covered products. IMO, no serious drug company would risk its reputation by pushing non-effective products and lose a lot of money on a dud that still had considerable development costs.

    One thing is for certain: Pharmaceutical innovation has become so risky and expensive that many patients can no longer benefit from it. Case in point: Hepatitis C. A cure is withheld from many Americans who are infected because of its high price. Something has to be done, and all ideas should be on the table.

  • Republicans may have a “penchant for deregulation,” but as their failure, after eight years of fulminating against the “failed” Affordable Care Act makes abundantly clear, they have no competence at conceiving or drafting the complex, detailed legislation necessary to effect an alternative policy. The Kefauver Harris Amendments took three years, from initial hearings to signing, to craft…and that was when Congress had members with the skills and commitment required for actual governance.

  • That’s how Trump is going to reduce the cost of drugs in the US. By allowing companies to sell sugar pills and phosphate buffered solutions (homeopathy style) at a fraction of a cost of today’s drugs. And they will be safe!

    The guy is a f***ing genius!

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