Last Tuesday (March 13, 2018) in a gutsy, contentious vote, the Republican-controlled House failed to pass a "right-to-try" bill (H.R. 5247) that would have given terminally ill patients access to experimental drugs and medical devices without FDA authorization. The final 259-140 vote, which fell short of the necessary two-thirds support from the House chamber, represented a setback for the president, who called on Congress to approve the bill in his State of the Union address six weeks ago . . . as well as a small libertarian think tank (the Goldwater Institute) which has been the driving force behind the effort. The bill - which got to the House floor without having gone through a single committee hearing - would permit patients suffering from terminal illnesses, upon a request from their physician to a specific pharmaceutical company or medical device manufacturer, to get access to a non-approved medication or device available without having to go to or through the FDA. While at first glance the legislation would appear to be a compassionate no-brainer, this "right-to-try" legislation (RTT) is, in reality, a lie whose main beneficiaries are not terminally-ill men, women and children.
In his first State of the Union address, the only piece of legislation 45 specifically mentioned was this "right-to-try" bill, which had, in a slightly different form, unanimously passed the Senate. In the president's address, he said: "We also believe that patients with terminal conditions should have access to experimental treatments that could potentially save their lives. People who are terminally ill should not have to go from country to country to seek a cure — I want to give them a chance right here at home. It is time for the Congress to give these wonderful Americans the 'right to try.'" On the surface, right-to-try legislation seems like a no-brainer; after all, who but a heartless ghoul would deny terminally ill patients access to potentially life-saving drugs, treatments or devices? That's on the surface. However, descend a few steps beneath that surface and a plethora of problems begin to emerge.
First and foremost is the matter of safety. The proposed federal law would only require the successful completion of a Phase I study, which isn’t enough to ensure efficacy or safety on its own. (Phase I studies, which enlist healthy subjects, are primarily interested in determining what - if any - adverse events [bad side effects] a drug may have; what the maximum tolerated dose [MTD] might be, and how the body absorbs, metabolizes and excretes the drug [PK]. What a phase I study does not look for is whether or not the drug, device or procedure is beneficial - i.e. capable of having a curative effect. In order to find out if a drug or device works requires additional phases using subjects who actually have the disease or malady. The "gold standard" for a phase II or III study is called "Double-blind, placebo-controlled," in which neither the doctor nor the subject knows whether they are receiving the study drug or a dummy "sugar pill." All these phases (which can also include phase IV and post-marketing) take years and tens - sometimes hundreds - of millions of dollars to complete. But underlying all the research is the ethical mandate "First, do no harm." Contrary to our unfounded optimism about medical progress which insists that new drugs must be good drugs, fewer than 10 percent of drugs that enter Phase I end up being approved; for oncology, that figure falls to 5.1 percent. In other words, "right-to-try" drugs, far from having passed scientific or medical muster, can be unproved and worthless at best, lethal at worst.
In clinical trials, participants (and/or their insurance carrier) are only charged for "standard-of-care" procedures. The sponsor pays for everything else . . . especially the medication. Under terms of the "informed consent" (which all test subjects read and sign before entering a test phase), if the medicine(s) or procedures cause any harm, the sponsor is financially responsible and the participant does not lose any of their legal rights. Under terms of federal right-to-try legislation, the patient (or their insurance carrier) is on the hook for payment - which can be in the hundreds of thousands of dollars - and cannot sue. Then too, the very act of using right-to-try therapies can render terminally-ill patients ineligible for health insurance or hospice care when they need it most. Of the right-to-try laws (now on the books in 38 states), half allow insurers to deny patients hospice coverage should they require it after the use of right-to-try drugs. Several have made it clear that health insurers are not obligated to cover the costs of any complications that may arise. In Colorado, patients undergoing experimental treatment secured under terms of right-try legislation are denied coverage even six months after the treatment has ended.
Unbeknownst to many, the Food and Drug Administration (FDA) has long had an "Expanded Access Program," under terms of which a terminally ill patient’s treating physician, after first having determined that their patient ". . . has a serious or life-threatening condition and no comparable or satisfactory alternative therapy" . . . then approaches the pharmaceutical company to ask for its agreement that it will provide the drug being sought. The company has the right to approve or disapprove the physician’s request." If the company agrees to the physician’s request, the physician can then apply to the FDA for permission to proceed. Should they do so, they are highly likely to be allowed to proceed. (Between 2010 and 2015, the FDA approved fully 99% of these requests.) Opponents of this process (starting with the Goldwater Institute) claim that a terminally ill patient could be dead long before the paperwork has been completed. This is simply not true. Today, the FDA Expanded Access form takes 45 minutes to complete, and the FDA will reply to emergency requests within no more than 24 hours.
So once again, on the surface, federal "right-to-try" legislation seems to be as simple, logical and compassionate as anything under the sun. To libertarians, it is simply a matter of the government keeping the hell away from the individual's right to choose for themselves (except, of course, if that individual is a pregnant female). Who but a political Simon Legree could deny dying patients the right to try unproven medications . . . even if it turns out to be a "hail Mary pass?"
Who indeed? Days before the failing House vote (in which 2 Republicans crossed over and voted nay, and 24 Democrats yay), more than 75 patient groups, including the American Cancer Society Cancer Action Network, the American Lung Association and the Cystic Fibrosis Association, had sent a letter to House leaders saying the bill “would not increase access to promising therapies” because it didn't deal with the main barriers to experimental drugs — the cost of drugs and company restrictions on making therapies available outside of clinical trials. And by skirting the FDA, the letter added, the proposed right-to-try pathway would be “less safe” for patients than the agency's existing program (expanded access), for overseeing the use of unapproved therapies outside of trials. Reading between the lines, the goal of federal "right-to-try" legislation is not to make experimental drugs available to desperate patients. The goal is to weaken FDA oversight of the drug approval process. Weakening FDA oversight can easily open the gates, admitting a parade of medical charlatans to come storming through, preying on the already desperate, dangling "miracle cures" which may well contain nothing more miraculous than hot air and hollow promises.
423 days down, 1,036 days to go . . .
Copyright©2018 Kurt F. Stone