A few weeks ago, I encouraged a mother of three young children who is fighting an uphill battle against cancer to try to enroll in an immunotherapy clinical trial, placing hope in a medical field that is rapidly advancing. It has already produced a miraculous seven-year recovery for one of my relatives who turned out to be a “super-responder.”
For a few days, my intervention generated new optimism in a distraught family. But the woman did not qualify for a trial. Doctors at a leading cancer institute told her to stick to conventional therapies, even though they are little more than a delaying measure against an aggressive disease that has already spread through much of the patient’s body. At least for now, the unproven but potentially more promising therapies built around her body’s own immune system remain out of reach.
So I find myself supporting the “right to try” legislation currently pending in the Senate, even though I have written in the past about my strong skepticism when politics intrudes on medical science to determine what treatments are appropriate and when.
Though they are largely toothless on their own, some form of right to try law has passed in 38 states, signaling an appetite for the national version that has been working its way through Congress. President Trump mentioned the policy with approval in this year’s State of the Union Address, suggesting he will be inclined to sign such legislation if it arrives on his desk. The House of Representatives passed its version of the bill in late March, sending it to the Senate, which passed a slightly different version last summer. The timing for the Senate’s consideration of the new bill is unknown, and it still faces real opposition, but many observers expect it will eventually pass.
The national version of right to try allows terminal patients to request access to experimental medicines without government oversight. The drugs must have cleared the Food and Drug Administration’s Phase I safety trials in order to be eligible, though the federal bill would protect doctors and drug companies from liability if something should go wrong with the therapy. For patients to be eligible, under the House bill’s rules, they must also have exhausted other treatment options and be ineligible for clinical trials.
There is no question that for research purposes, clinical trials need to be carefully structured to control the types of patients who are enrolled and the types of treatments they receive. Our drug-approval laws are based on the idea that treatments should not be offered until they are proven to be both safe and effective. Promoting a harmless but useless treatment can lead patients away from therapies that would be of greater benefit, and there is certainly no shortage of charlatans who are prepared to do just that in the interest of profit.
As a young man, I recoiled when Mexican clinics promoted a therapy called Laetrile as a cure for cancer, when it did nothing of the sort and was, in fact, potentially harmful. I watched as the Montana Legislature symbolically legalized the compound, which was a meaningless step since federal law prohibited its shipment in interstate commerce and it could not be produced in-state. Many years later, another group of lawmakers in Helena took the Laetrile law off the books.
But I think the federal right to try legislation is different. I will grant that there is a chance I am rationalizing due to my personal connections to the topic, but I don’t think so. For one thing, the bill passed by the House would only make therapies available if they have passed the first step in the FDA’s existing approval process, which determines that they are safe enough to test for therapeutic value. The old Laetrile legislation included no such hurdle.
In addition, doctors would still need to prescribe the unproven treatments, and drug companies would still need to agree to provide them. Such treatments are unlikely to be covered by most insurance, so profit motive to victimize otherwise-treatable patients should be a less prominent consideration.
If right to try passes, unproven therapies will still be a form of research, although not rigorous enough to be relied upon alone for regulatory approvals. Besides offering some hope to patients who otherwise might have none, this approach can advance the knowledge frontier in a fast-moving and unpredictable area of research. Some immune therapies have, in fact, been tried in patients where they were not expected to work, with surprisingly positive results. And immune therapies are hardly the only ones that will be employed when patients and doctors are allowed to consider options outside the clinical textbooks.
Yes, the FDA already has a procedure to allow compassionate use of unproven therapies, and that is a good thing. Its expanded access program, sometimes called “compassionate use,” allows doctors to request a medical product still under investigation for a patient who cannot access it through a clinical trial, either because the patient is not eligible or because no such trial is underway. If right to try legislation passes, doctors who prefer to use the FDA’s expanded access program can continue to do so. But sometimes there is not enough time, or not enough information, to go through the existing process. It’s not realistic to expect time-pressed experts in cancer or other diseases to push back against the bureaucracy on more than rare occasions.
I don’t want to raise false hope for anyone. I don’t want patients to forgo useful therapies in favor of pipe dreams. I don’t want needless side effects to compromise whatever quality time might remain for someone in desperate circumstances. I don’t really disagree with any of my past views on the subject of politics and medicine.
But I know a mother out there who has three young children and a husband who want and need her to stay in their lives. I would never presume to second-guess highly trained doctors and researchers if they honestly believe there is no hope in trying a particular therapy. I just don’t think the law should get in the way in situations where there is little to lose in trying to put the latest medical science to work in places where it has not yet been tried.
Posted by Larry M. Elkin, CPA, CFP®
A few weeks ago, I encouraged a mother of three young children who is fighting an uphill battle against cancer to try to enroll in an immunotherapy clinical trial, placing hope in a medical field that is rapidly advancing. It has already produced a miraculous seven-year recovery for one of my relatives who turned out to be a “super-responder.”
For a few days, my intervention generated new optimism in a distraught family. But the woman did not qualify for a trial. Doctors at a leading cancer institute told her to stick to conventional therapies, even though they are little more than a delaying measure against an aggressive disease that has already spread through much of the patient’s body. At least for now, the unproven but potentially more promising therapies built around her body’s own immune system remain out of reach.
So I find myself supporting the “right to try” legislation currently pending in the Senate, even though I have written in the past about my strong skepticism when politics intrudes on medical science to determine what treatments are appropriate and when.
Though they are largely toothless on their own, some form of right to try law has passed in 38 states, signaling an appetite for the national version that has been working its way through Congress. President Trump mentioned the policy with approval in this year’s State of the Union Address, suggesting he will be inclined to sign such legislation if it arrives on his desk. The House of Representatives passed its version of the bill in late March, sending it to the Senate, which passed a slightly different version last summer. The timing for the Senate’s consideration of the new bill is unknown, and it still faces real opposition, but many observers expect it will eventually pass.
The national version of right to try allows terminal patients to request access to experimental medicines without government oversight. The drugs must have cleared the Food and Drug Administration’s Phase I safety trials in order to be eligible, though the federal bill would protect doctors and drug companies from liability if something should go wrong with the therapy. For patients to be eligible, under the House bill’s rules, they must also have exhausted other treatment options and be ineligible for clinical trials.
There is no question that for research purposes, clinical trials need to be carefully structured to control the types of patients who are enrolled and the types of treatments they receive. Our drug-approval laws are based on the idea that treatments should not be offered until they are proven to be both safe and effective. Promoting a harmless but useless treatment can lead patients away from therapies that would be of greater benefit, and there is certainly no shortage of charlatans who are prepared to do just that in the interest of profit.
As a young man, I recoiled when Mexican clinics promoted a therapy called Laetrile as a cure for cancer, when it did nothing of the sort and was, in fact, potentially harmful. I watched as the Montana Legislature symbolically legalized the compound, which was a meaningless step since federal law prohibited its shipment in interstate commerce and it could not be produced in-state. Many years later, another group of lawmakers in Helena took the Laetrile law off the books.
But I think the federal right to try legislation is different. I will grant that there is a chance I am rationalizing due to my personal connections to the topic, but I don’t think so. For one thing, the bill passed by the House would only make therapies available if they have passed the first step in the FDA’s existing approval process, which determines that they are safe enough to test for therapeutic value. The old Laetrile legislation included no such hurdle.
In addition, doctors would still need to prescribe the unproven treatments, and drug companies would still need to agree to provide them. Such treatments are unlikely to be covered by most insurance, so profit motive to victimize otherwise-treatable patients should be a less prominent consideration.
If right to try passes, unproven therapies will still be a form of research, although not rigorous enough to be relied upon alone for regulatory approvals. Besides offering some hope to patients who otherwise might have none, this approach can advance the knowledge frontier in a fast-moving and unpredictable area of research. Some immune therapies have, in fact, been tried in patients where they were not expected to work, with surprisingly positive results. And immune therapies are hardly the only ones that will be employed when patients and doctors are allowed to consider options outside the clinical textbooks.
Yes, the FDA already has a procedure to allow compassionate use of unproven therapies, and that is a good thing. Its expanded access program, sometimes called “compassionate use,” allows doctors to request a medical product still under investigation for a patient who cannot access it through a clinical trial, either because the patient is not eligible or because no such trial is underway. If right to try legislation passes, doctors who prefer to use the FDA’s expanded access program can continue to do so. But sometimes there is not enough time, or not enough information, to go through the existing process. It’s not realistic to expect time-pressed experts in cancer or other diseases to push back against the bureaucracy on more than rare occasions.
I don’t want to raise false hope for anyone. I don’t want patients to forgo useful therapies in favor of pipe dreams. I don’t want needless side effects to compromise whatever quality time might remain for someone in desperate circumstances. I don’t really disagree with any of my past views on the subject of politics and medicine.
But I know a mother out there who has three young children and a husband who want and need her to stay in their lives. I would never presume to second-guess highly trained doctors and researchers if they honestly believe there is no hope in trying a particular therapy. I just don’t think the law should get in the way in situations where there is little to lose in trying to put the latest medical science to work in places where it has not yet been tried.
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