D

ear Vice President Pence,

Two weeks ago, you met with several families and encouraged them to lobby Congress for a federal “right-to-try” law, which would allow people with fatal illnesses to gain access to experimental medicines, even though they are not enrolled in a clinical trial.

Your goal is admirable. Unfortunately, though, you’re peddling false hopes.

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Here’s why: From the start, the right-to-try movement has been misguided.

The effort grew out of mounting frustration with the Food and Drug Administration, which does have a program called expanded access that allows seriously ill people to obtain an experimental medicine. In fact, the agency has historically approved 99 percent of requests.

Yet critics complain the program is unwieldy and sometimes capricious. And they argue that, if only the FDA were sidelined, more dying patients could more readily gain access to potentially life-saving treatments. Not coincidentally, this argument mirrors general criticism of the FDA’s drug approval process.

Two years ago, as governor of Indiana, you signed a right-to-try bill into law. Now, 33 states have such laws. But there are outstanding questions about the extent to which the FDA has a legal right to preempt, or override, the state laws.

This explains why lawmakers introduced right-to-try bills in the House and Senate in recent weeks. The impulse to offer patients hope is understandable; after all, not everyone can get into a clinical trial. But these proposals have the same fundamental flaw as state laws — at the end of the day, requests must be granted by drug makers, not just the FDA.

And that’s always been the rub.

Drug makers may have several legitimate reasons for denying a request for access to an experimental drug they’ve been testing. They may not want to risk giving the drug to a patient outside of a controlled clinical trial, because if the patient has a bad reaction, that could undermine their effort to get the drug approved by the FDA.

And smaller companies, in particular, may lack sufficient supplies to grant a large number of requests.

“The legislation makes it seem there’s a single roadblock — the FDA,” said Ken Moch, who heads Cognition Therapeutics. He previously ran a biotech called Chimerix that created an outcry by initially denying an experimental drug to a dying boy. “In my personal experience, that’s untrue.”

Starlee Coleman, a spokeswoman for the Goldwater Institute, a conservative nonprofit that has drafted model right-to-try bills, acknowledged that some drug makers may avoid granting requests from patients.

“A drug company can still be an obstacle,” she told me. “But we think there will be scenarios where a drug company will be comfortable providing a drug outside the FDA process and we want to make sure they have a pathway to do this. We don’t have any expectation that every sick person would be helped if the law passed tomorrow. But we do want to create options quickly without bureaucratic delay.”

Hoping to alleviate some concerns, the federal bills say drug companies — as well as physicians — cannot be sued if something goes wrong. This provision may, perhaps, encourage a drug maker to provide its experimental medicine, but the complete lack of accountability is troubling.

Significantly, the bills also contain a provision that blocks the FDA from using information about any unintended consequences, such as a severe patient reaction, against the company when it comes time to consider the drug for approval.

This is a bad idea. The simple reason is that the FDA would be precluded from considering the impact of a serious side effect or later placing that information in the product labeling.

“Removing the FDA from one segment of the process is short-sighted, and puts a simple, but ineffective, solution on a much bigger problem, [which is] the long time frame [needed] … to gather sufficient safety and efficacy data,” said Beth Roxland, a law and health policy expert at the division of medical ethics at the NYU Langone Medical Center and a former bioethics and strategy leader at Johnson & Johnson.

She has a point. As public health goes, cutting the FDA out of the loop is simply bad policy.

In supporting such laws, you might mean well, but the federal legislation assumes there will never be any need for oversight. Beyond expanding access, right to try may wind up expanding uncertainty.

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  • I have worked with big PharmaCos since my career started some 35++ years ago. From an advisor to a independent consultant to a full fledged project director and I have so far seen one common thread with all of them. They are all risk averse.
    This new program won’t make one bit if difference in them freeing up their drugs. The ability to claim no liability is shear fiction. As long as there are lawyers there will be lawsuits. Without the final FDA approval to withstand the basic jury scrutiny the risk is FAR greater then the PharmaCos are going to tolerate on anything but a VERY small basis
    In my own case I have a VERY rare stage 4 cancer. I know the PharmaCo who has the potential drug I want/need and I know the VP of R&D personally and yet even with the FDA’s approval the PharmaCo is still not willing to allow me access to the drug. Why you might ask? SIMPLE they are right now in their final phase of approval and if they give it to me and somehow something goes wrong even with this new bill stating that the FDA can’t use the data surely they are in jeopardy. Do we really want drugs approved that have been found to have outside spectrum effects that have to be ignored by the FDA?
    Lets say they give it to me and 30 others with my cancer and lets say half of us get some horrific unseen before effect? Is it really proper to force the FDA from using that as a criteria to review the drug? Who wins in that situation? I lose because I got the effect the population loses because the effect is now known but can’t be used and even noted to physicians as a possibility and the company loses because it now knows it is going to get a deluge of complaints that will have to be dealt with post approval that might get them a black box warning.
    What about the stock holders? They are expecting smooth sailing and now are not aware that Dr Dave and 20 others are having effects that could be a financial issue down the road
    Forcing anyone to do anything is not a good thing, PERIOD! The process we currently have is a real PITA the FDA needs to make it much easier to leap thru the hoops but to force the PharmaCos to provide their drugs one for free (they can’t charge since the drugs are unapproved) and two in an uncontrolled manner that only has a negative spin for them is insanity defined. There is NO win for the drug companies if the drug works great they gave it away for free. If the drug in ANY way loses they take the hit and might even lose the drug in its entirety
    We too often forget Laetrile and Thalidomide and yet the FDA is based on the Thalidomide debacle of the 1950’s and will never forget it as neither should we.
    Yes I wish I could get the drug and I might not be here when it finally arrives but if the drug helps all the others with this instead then I am thankful. Atleast I didn’t get the process messed up for my own sake
    Dr Dave

    • Dr Dave, personal relationships aside I think you’re getting a line of BS from the company. In the industry 34 years I know how the baloney gets made. In reality, if the drug has been submitted for approval to FDA it will win or lose based on the Phase III studies that were performed PRIOR to NDA submission and NOT based your putative experience were you to be given the drug in Phase IIIB. Furthermore, it is unusual for a brand new side effect heretofore unseen to pop up de novo in Phase IIIB if the drug has been properly studied up through NDA submission. Even if there were some de novo side effect, unless it was on a large scale, serious, life threatening or cause a multiplicity of deaths, those events would not influence FDA approval but would be added to the package insert. Even if the drug company wanted to have a “clean” package insert, such adverse events would ultimately come out post approval since the company must periodically update the labeling after launch with new side effect information.

  • I have ALS (Lou Gehrig’s Disease) and I see desperate patients make poor choices every day. RTT is a state solution to a federal issue. A state may pass an RTT law but if the drug crosses to another state for delivery to the patient, that automatically trips the Interstate Commerce law and makes it a federal matter. Passing a federal version of RTT is still meaningless because giving patients the “right to try” means nothing without a pharmaceutical company willing to provide their product. And even if one does, the patient very rarely has sufficient funding to purchase the drug, and nothing in any of the RTT bills compels the companies to provide their product for free. Then there is the matter of how the drug is administered. Many drugs require injection or transfusion, and almost no patient has the credentials or training to obtain and use the required equipment. That means trying to enlist qualified medical personnel, none of whom would risk their license to practice or being summarily dropped by their malpractice insurance.

    Basically, Republicans are using us, the patients, as mere tools to further their objectives of eroding all government regulation of industry. The “jobs” and “economy” excuses weren’t working fast enough so they found this new strategy. FDA, along other regulating agencies, have very important jobs to do to protect consumers from unscrupulous businesses. Read up on the history of the early 20th Century. A great book demonstrating the predations rampant during the time is “The Jungle” by Upton Sinclair.

    The current process of drug approval was created in 1962 and definitely requires updating, taking into account 21st Century technology and techniques. But anarchy is never the answer. Refining the process is required, not removing it. I co-founded an advocacy group dedicated to updating the old system using new 21st Century techniques (Hope Now For ALS – http://www.hopenowforals.org). These techniques can remove the need for the placebo group in trials, and analyze data better so that smaller trial patient populations are required. This makes trials more humane. It also makes trials shorter and less-expensive, meaning drugs are delivered to patients more quickly or futile drugs rejected more quickly so that the search for truly effective ones can be continued.

    THAT is useful change and a strategy in which FDA has already expressed willingness to use as a secondary endpoint in order to test it. HNFA has had multiple meetings with FDA on this subject. Fix what’s broken, don’t throw the baby out with the bath water.

    • Correction, there are no issues with interstate commerce laws. It would only apply if the every component of the drug, from active ingredient, to final product were made, packaged and sold in the same state. In reality drugs are sourced from many places and ingredients cross state lines on a regular basis, so there is no violation of Interstate commerce law.

    • Laertril was advertised as a “miracle” by unscrupulous clinics in Mexico. And only by the resistance of FDA “bureaucrats” who were placed under enormous pressure by pharmaceutical companies, was Thalidomide kept out of the US.

      “Right to Try” is an ethically thorny issue, and one that may not have a clean and simple resolution. I work with chronic pain patients every day in social media. Many of them would try ANYTHING to get their pain under control. And many of them are victimized by their own desperation every day, manipulated by quacks and charlatans in the so-called “alternative medicine” movement.

      The FDA drug approval process likely does need to be shortened and made more efficient. But the devil is in the details.

    • Dear Jeanne,

      Thanks for the note. I’m not sentencing anyone to death. I’m simply trying to point out that the bills – and the effort – are misguided. Yes, the FDA must approve requests, but does so nearly all of the time and has attempted to simplify the process. Companies remain ultimate arbiters. You can read the bills if you wish; the links are there. This is a complicated issue and patients do need consideration, but blaming the FDA appears to be a straw man.

      Regards
      ed at pharmalot

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