T

he term “FDA approved” means a lot to those of us working in health care and the patients we treat. But if the 21st Century Cures Act becomes law — the House of Representatives approved it Wednesday and the Senate will vote on it next week — this mark of trustworthy stewardship will become a shadow of its former self.

Working in medicine and public health in Baltimore, we are witnesses to patients’ distrust of the health care system and the numerous barriers they face to quality, affordable care. We rely on the Food and Drug Administration to ensure that the treatments we provide are both safe and effective. Our patients have also placed their trust in this agency through us as their prescribers.

For example, in discussing medications to treat chronic pain with a 48-year-old single mother of five who works during the day, one of us suggested gabapentin as a treatment option. Although it is a commonly used drug, she had not heard of it. But she was reassured when told that it is FDA approved for this condition. She tried this medication, and it has provided her good pain relief.

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As members of the National Physicians Alliance (NPA), we have been closely following the 21st Century Cures Act legislation and its potential impact on what “FDA approved” would mean for new drugs and devices. Our interest in the 21st Century Cures Act emerged from overarching concerns among ourselves and other alliance members that the FDA’s mission to protect public health by “assuring the safety, efficacy, and security” of new treatments was being hijacked by the pharmaceutical and medical device industries. Reviewing the latest version of the bill, released the day after Thanksgiving, we are not sanguine.

The legislation does include some redeeming features, such as increases in funding for the National institutes of Health and for addressing mental health and the opioid epidemic. All of this much-needed funding, however, comes at the expense of gutting the only agency we as providers can rely on to tell us if the treatments we are prescribing to our patients will work and won’t harm them.

The act, for instance, would pressure the FDA to approve new antibiotics, antifungals, and possibly other drugs through a new and faster pathway called the “limited population antibacterial drug” pathway. Through this expedited pathway, new medicines would be approved and labeled for use in patients with “unmet medical needs,” even though these drugs had never been tested in these specific patient populations.

The act would also allow for new high-risk medical devices to be approved based on anecdotal case studies or reports in medical journals, rather than having to be held to the gold standard of testing in clinical trials. On top of that, manufacturers would be able to make changes to these high-risk devices without any FDA oversight. Instead, the changes would be monitored by third-party contractors — paid by industry.

The most recent version of the 21st Century Cures Act has a new addition: prompting the FDA to approve new regenerative therapies based on clinical anecdotes and surrogate endpoints like shrinkage of a tumor instead of clinically relevant outcomes like longer survival or improved quality of life.

These provisions would unravel the FDA, turning it from the treatment watchdog it is today into a puppet of the pharmaceutical and medical device industry. If the 21st Century Cures Act is passed as written, clinicians could be given potentially deadly drugs and devices to prescribe to their patients, blessed by this new version of FDA approval.

The good parts of the legislation — increased funding for mental health, opioid addiction, and the NIH — are not even guaranteed. Those funds would not be mandatory under the legislation and would be subject to the whim of Congress during appropriations battles each year. This horse trade between protecting the role of the FDA and critical funding has become unequal and instead represents a lose-lose proposition for patients and providers.

Despite all of their clinical and scientific knowledge, health professionals need a way to convey to patients that treatments are safe and effective. The imprimatur of the FDA is currently a kind of Good Housekeeping Seal of approval. Weakening the FDA’s standards would undermine our trust and leave patients at a loss. Without a strong FDA, who else will let providers and patients know if a treatment will work or is even safe? Pharmaceutical companies? Medical device manufacturers?

We don’t need #CuresNow. Instead, we need legislation that will let us all continue to trust the term “FDA approved” into the 21st century.

Reshma Ramachandran, MD, is an assistant scientist in the Johns Hopkins Bloomberg School of Public Health and cochair of the National Physicians Alliance FDA Task Force. Zackary Berger, MD, is an assistant professor of medicine at the Johns Hopkins School of Medicine and a core faculty member in the Johns Hopkins Berman Institute of Bioethics.

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  • I agree. My wife was injured and almost died because of 510k approval by the FDA for the Davinci surgical robot. They hid defects and took out a patent on the “fix” before they told the FDA or ANYBODY. Yet they remained on the shelf? 260+ have died and 18,000 injured like my wife. The “robot” is the 21st century snake oil and the biggest scam of the century!
    See: “unplug the robot” Facebook page (the truth is there)

  • How many rare disease patients have you seen over your careers? How many of these have you found an FDA-approved treatment for — much less been able to cure? How is their quality of life? The 21st Century Cures Act is NOT for the average patient. Even speeding antibiotics relates not to common diseases but those like and including Zika. The goal is not to weaken the FDA but to make it more possible for patient populations in which there is not the standard size sample for traditional endpoints and clinical trials to possibly see treatment within their lifetimes. These patients are willing to accept higher risks because they have no other options. I am one.

  • This is a good article I generally agree with, but the authors are not fully informed about the extent of harm from FDA approved medical and dental devices. Review and approval standards are much, much weaker than for Rx. They do not take account of allergies, immune reactions, and genetic variation in methylation that impacts tolerance vs toxicity.

    FDA is beefing up surveillance with a UDI system, but that is closing the barn door after the horse has bolted and run over a crowd of people. We need a real 21st Century Medical and Dental Device Safety Act.

    Along with two coauthors, we just submitted a piece to First Opinion. Not sure if and when it will run. Please call and tell your Senators now we need a real 21st Century Medical Device Safety Act.

  • I’d like to see the FDA and Pharmacitical Co. separated financially. I’d like to see drugs like Ketamine approved for rare diseases like mine CRPS that Pharma can’t patent and is already used for pain for other health issues. It’s already used in ERs for children and used on animals and safer to use ketamine troches than opiates. Off label uses because of non FDA approval is stupid when they know drugs will help. Take gabbapenten and lyrica for nerve pain being used for neuropathies and central pain disorders. This Opiate war also is causing Drs and administrators to run pain sufferers out of health care systems dumping pain patients for suspected abuse or drug seeking during flare ups to protect licenses. I know first hand having my medical records and even UA’s falsified in the ER and between Drs. I shouldn’t have to fly out of state, drive 5 hrs for PM appointments because I need pain meds to function and reduce physical suffering. Using drugs like Namenda another MNDA drug for centralized pain too. Low dose Naltrexone too. There is no drug FDA approved for CRPS which is rediculous, so we are left with antidepressants for Pain Management? How ignorant. Treat the underlying problem not the side effects from under treated pain conditions. Easier approval for procedures and medications. Less witch hunting over opiates. It’s driving chronic pain people out of health care to treat themselves by whatever means they can. Making law abiding citizens into criminals and forcing them to go underground. For example buying injectable lidocaine in Mexico to do superficial and IM injections to calm burning nerve pain. Compounded drugs being covered by insurance for neuropathic pain making it affordable and safer for central pain diseases and neuropathies, like ketamine topical coumponds. Why can’t we get these drugs and therapies FDA approved? Insurance Approved? Help Chronic pain sufferers with flexibility and tools to manage their own pain and reduce health care cost to everyone. Approve drugs used off label for conditions already approved for similar health conditions, including approving FDA approved drugs for rare diseases and disorders. There is so much over regulation of FDA approved drugs to diagnosis. Let Drs and patients do treatments that work for the individual situation. Trust medical Drs to treat health problems. Trust patients to work with Drs. to manage their conditions. Untie Drs. , open doors to cover medications from insurers for prescriptions for numerous rare disorders or similar syndromes and symptoms. Why is Cancer pain treatable but my neurological pain disorder isn’t? Until I got this disorder I had no idea how I would go from a patient to a drug suspect. It’s shameful. When I see the heroin epidemic it makes me wonder how many chronic pain patients have resorted to that because of another war on drugs by the government? Ketamine oral compounded troches terrify uneducated Drs. Because of misinformation correlating it to special K on the streets. Another war on drugs fallout. When is the gov going to stop this nonsense? This form of medication can be refilled 5 times and prescribed by nurse practioners. Why the unnecessary hype? It works better than opiates for burning nerve pain and is safer. Topical and sublingual and nasal drugs are safer than pills and endocrine systems. People with incurable diseases need safer long term choices for management and insurance approval for alternative support such as vitamin infusions, chiropractic, massage, physical therapy, nutrient support along with interventional procedures. I think this entire cures act is just a pork Bill with more disasterous consequences to an already broken medical system. Keep it up and we soon will have no Drs. Patients are already fed up with testing only and no result type medical care for the cost. Expand palliative care to pain disorders. I have had to be on deaths door to get hospitalized and get medical support during acute flare ups. Waiting until I lose 50 lbs and can’t eat or drink was unnecessary. There is no test for my disease. Wonder why people turn to marijuana? Break the law? It’s cheaper and at least if their illness cannot be cured they at least will be so drugged they won’t care. Better outcomes from self medicating and cheaper, even if they are labeled drug abusers and can’t get medical care. I took a woman to the ER with the same disease who was from out of state and had a medical MJ prescription and tested positive in an illegal state who couldn’t get female hormones prescribed because the Dr said why would she prescribe any meds to a criminal? I was educating nurses on our disease while there showing them my ketamine nasal spray and telling them the Miracle of this medication on burning nerve pain. They called my Dr and my meds were dropped on the spot. Really? I even went to an ER to get drug tested to prove I was not using MJ but it didn’t matter , I was now an official drug seeker. My neurology appointment was cancelled and they made me a phone appointment with a shrink. I already had one and a counselor. 6 years of wasted time. It never improved my pain or gave me better medical care. Instead I was offered electric shock therapy to cope with medical abandonment and limbic system issues caused by my illness. Really? In my opinion the entire health care system is broken. It’s been reduced to a 15 minute revolving door pill mill and test result doctoring instead of medical deduction and differential diagnosing. The so called evidence based medicine model is a complete failure as many disorders don’t fall in that category or take years of progression before diagnosis is measurable on test. At the end stages instead of early intervention through Dr diagnosis vs test results. I have a question too. Do you really believe this many people in the US need to be on all these neuro drugs and anti-depressants? Once on you can’t get off? How lucritive for Pharma. When your brain is fed by meds instead of making own chemicals you become a life long customer. Maybe this epidemic should be regulated and only used short term for support. If you have to taper off a neuro med what is the difference from an opiate? Feeding the brain chemicals or blocking pain signals both are dependence drugs. Something is causing autism and reproductive health issue rises. Diet maybe, but drugs have natoriously been implicated in birth defects.
    In my opinion health care and medicine are sorely disappointing today. Drs are just pill pushers not Drs. The medications the prescribe often cause the same symptoms people already suffer from. My favorite drug statement is ” believed to act on, bla, bla” Pharma doesn’t even really know why their drugs work or how. Dare I say “If”?
    I am over the entire system. I hope you get Pharma out of medical school finances too.
    Jerie Quinty

  • I agree with the other people here. As a physician practicing for 30 years,the FDA is not the be all end all the authors claim. They want government to hold their hands.We don’t get that anyway.We are always responsible for what we give our patients.The FDA needs to let the patients and their Docs decide more about new Meds and treatments.

  • The FDA as currently structured is a slow 20th Century agency , un- able to keep up with 21st century drugs . They take up to 10 years to approve drugs that are often genetic derivatives of previously safely tested drugs . The approvals are almost always conditional , with restrictions . The FDA has ” no time table ” to re- evaluate these restrictions . No wonder dugs then get prescribed ” off label ” . The authors sound like Dr. Pazdur , the FDA ‘s head of oncology drug reviews did back in 2009 . Back then , emboldened by a new , overly Big Govt. – friendly President , Dr. Pazdur pushed for overall survival data vs. the gold standard of progression free survival . He slowed down approvals just as new 21st century drugs came on line. The foremost being Kadcyla, now a life saver for thousands of breast cancer pt.’s. His dept. delayed it for 3 extra yrs., while approx. 15,000 Americans died from that type of cancer each of those yrs. His dept. tried to ban Iclusig , Lemtrada , (thankfully those 2 decisions were reversed ) revoked avastin for breast cancer when it could have just been ” black boxed ” . NO, we do not want to stay stuck in the 20th century . It’s time for change .

  • The article makes the point that only the government agency is able to determine what is safe and not safe. I know physicians, pharmacists, and nurses are all trained on how to read the medical literature and make patient specific evaluations. They have a professional responsibility to keep up on this literature too. Do we not trust our clinicians to make these responsible choices to evaluate the literature and make appropriate decisions for their patients?
    There have been plenty of FDA approved medications that get recalled because they were unsafe. So just having a FDA seal of approval does not necessarily mean it is always safe and appropriate. You still need clinician judgment.

  • Yea, I also found it funny that the author is trying to state that we need strict FDA approval to use meds by giving an example of using a med for a non-FDA approved indication.

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