All Hail No Cattle

Authors: Alison Bateman-House, Arthur L. Caplan
Division of Medical Ethics
NYU Langone Medical Center

Are right-to-try laws a good idea? In 2014, they began appearing throughout the United States, first in Colorado and now in 23 states, with several more considering passing versions of this popular legislation.

At first glance these bills may seem worth enacting. Right-to-try (RTT) laws promise terminally ill patients that they can obtain experimental drugs or medical devices in their quests to save or extend their lives. These investigational medical products are still in development and have not yet met the requirements set by the Food and Drug Administration (FDA) in order to be approved for sale or use in the United States. In some cases, the products are still being tested and patient access would primarily be by enrolling in a clinical trial – something terminally ill people may be too unwell to do. Less commonly, the products have completed clinical testing and are waiting for an approval verdict from the FDA. In either case, RTT laws are about providing terminally ill patients access to unapproved drugs or devices.

Of course we want to help people who are dying, so legislation that would give patients access to possible treatments would appear laudable. However, this conclusion is based on several assumptions. The first is that RTT legislation grants a patient a new right of some kind. Assumption two is that state legislation has jurisdiction over when a patient may access an investigational medical product. Assumption three is that use of an investigational medical product will help, or at least do no harm, to a patient. And the fourth assumption is that use of the experimental medical product is intended to benefit the patient. We argue that none of these assumptions are universally valid. Even more troubling, RTT legislation may harm the very patients that those backing the legislation claim to be helping.

How can this be? The answer lies in what the legislation, both passed laws and pending bills, actually says. RTT legislation is quite similar in content from state to state, as it is all based upon a model bill put out by the Goldwater Institute, a libertarian think tank based in Arizona. According to the language of this model bill:
“A manufacturer of an investigational drug…may make available and an eligible patient may request the manufacturer’s investigational drug…under this act. This act does not require that a manufacturer make available an investigational drug, biological product, or device to an eligible patient.”(1)

So, patients may ask, and companies may provide access to their investigational medical products – but only if the companies want to do so. And elsewhere in the model bill it says, “A manufacturer may…[R]equire an eligible patient to pay the costs of, or the costs associated with, the manufacture of the investigational drug, biological product, or device.”(1) So assumption one, that RTT legislation grants a dying patient a new right of some kind, is false. Patients have no right to investigational medical products. Under RTT laws, they may only ask for them.

How does this differ from the situation facing the dying today? It doesn’t. Patients do not need new laws in order to be able to make “compassionate use” requests for pre-approval medical products. All patients, regardless of age, gender, income level, access to health insurance, or state of residence may request access to an investigational drug or device from the company developing it. The rules for such non-trial provision of unapproved medical products are governed by the FDA’s “expanded access” policy.(2)

Ironically, the new RTT laws pertain only to dying patients (and, in a few cases, to patients deemed permanently unconscious and unlikely to recover). That makes them more restrictive than the current system in which patients may seek compassionate use access to investigational drugs and devices in hopes of staving off blindness, paralysis, or intractable pain – all serious but not fatal problems.

The current system is far from perfect. As requests for pre-approval medical products are normally channeled by doctors to the company developing the new product, patients who do not have access to healthcare providers that stay abreast of the newest drugs or devices in development may never be informed of the possibility of compassionate use. RTT laws offer no solution to this problem, stating only that doctors will not get in trouble for recommending or being involved in the use of an investigational product. This is a red herring: we know of no such cases. If a doctor gets in trouble, something else is going on, such as fraudulent claims about the investigational product’s curative power.

If patients cannot find doctors to channel their requests, they can make their own compassionate use requests, provided they find some means – on the phone, through their connections, on social media, through the local news media – to contact the company. Again, this poses problems: not everyone is able to navigate the media or the Internet successfully or has access to helpful contacts. But again, RTT laws do not amend this situation.

In states that have passed right to try laws, the key difference is that investigational medical products that have only gone through Phase I testing – safety testing, normally conducted only on a small number of healthy people – are suddenly considered quasi-therapeutic agents, even though they have not been tested for efficacy. This has potentially dire consequences. While terminally ill patients would have to sign a form testifying that they understand that the treatment they seek is experimental and has not been shown to work in humans – which would indicate that it is not therapeutic – several RTT laws and bills have language stating that “eligibility for hospice care may be withdrawn if the patient begins curative treatment with the investigational drug.” Thus, this legislation is inherently contradictory. How can a medical product be simultaneously experimental and curative? Perhaps a drug that has gone through the testing required of the FDA would fit this category – where enough evidence has been collected to indicate that the drug can help patients but the FDA has not yet rendered its decision on approval. But no medical products in the early phases of their development can claim to be both experimental and curative – yet RTT laws require only that the experimental drug have gone through Phase 1 testing. As such, investigators might know what the most common harms of the drug are but would have no idea if the drug will work in humans. As such, how could a patient make (as required by RTT legislation) an informed decision about using the experimental product when he or she lacks information about its possible benefits and has only very limited information about its possible harms? And, should a patient choose to knowingly take this leap into the unknown, how could she or he be denied access to hospice on account of it? Colorado’s law goes even further, stating that patients in such cases might lose in-home healthcare or certain types of insurance coverage.

The baseline assumption that use of an investigational medical product will help or at least not harm a patient is wrong. Whether the medical product will help or harm a patient would be a matter of chance, with the only safeguard being how far along in development the product is. Likewise, the assumption that use of an experimental medical product is intended to benefit the patient is also arguably wrong. No doubt some knowledgeable and sincere physicians may see right to try legislation as a way to help their patients get access to treatments that they need. But the legislation varies from state to state in what, if any, safeguards are put in place to prevent an unscrupulous or downright criminal health provider from trying out pet therapies or expensive placebos on patients who have no other options. In the best case, an RTT law would mandate at least a second opinion from a physician qualified to understand the patient’s condition, prognosis, and available therapeutic options. In many cases, however, these laws contain no such protections; instead, they guarantee physicians immunity from penalty so long as they “act in good faith.”

A final flaw with RTT legislation pertains to the assumption that the state has a say in when a patient may access an investigational medical product. In the United States, use of – and, indeed, even the shipping of – investigational medical products is regulated by the FDA. The FDA is a federal entity, and under a legal doctrine known as preemption, states cannot claim for themselves the ability to control something already controlled by the federal government. The federal government may choose not to exert its authority, as is currently the case in states that have legalized marijuana use, sale, or cultivation. Thus far, the federal government has not acted against the RTT laws that have sprung up. However, numerous legal commentators have predicted that, should a suit be filed, the FDA would win, given its longstanding authority to regulate investigational medical products.(3,4)

It seems that the ultimate purpose of RTT laws is not to assist terminally ill patients – recall that the laws grant them no new rights – but rather to strip the FDA of its power. The libertarian think tank that created the RTT movement and that has effectively lobbied for its model bill to be adopted by state governments has acknowledged that its legislation has not enabled a single patient to access investigational medical products unavailable to them under the FDA’s current rules. However, RTT advocates carry on, flogging inaccurate and misleading talking points such the oft-repeated claim that it takes 100 hours or more to complete the FDA’s paperwork for compassionate use.(5) Not only is there no data to back up such a claim, there are numerous accounts of the FDA working to expedite compassionate use decisions where time was of the essence.

Nobody wants people to die, and everyone is in favor of terminally ill patients having hope of recovery or a remission. If RTT laws saved people, we would support them. However, save their FDA bashing and rhetorical flourishes, the bills offer nothing new. Right to try is an appealing slogan, but it is hollow, offering patients no new rights and only false hope. We think it is – at best – unkind to offer the terminally ill and those who love them false hope. Furthermore, it is fundamentally wrong to penalize dying patients simply because they wish to try an experimental treatment. The false hope that these laws generate, coupled with the fact that hospice or other kinds of care may be withdrawn from those who need it, makes it clear that right to try laws do nothing to help the dying.

We would like to acknowledge the editorial and research assistance of Lisa Kearns, MS, MA

References:

1. Goldwater Institute. Right to try model legislation. https://goldwater-media.s3.amazonaws.com/cms_page_media/2015/1/28/RIGHT%20TO%20TRY%20MODEL%20LEGISLATION%20%282%29_1.pdf (accessed 1 August 2015).

2. Code of Federal Regulations 2014. FDA 21CFR312.305. Washington: U.S.FDA; 2014. Available from: http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=312.305

3. Farber D, Pinto P, Caplan A, Bateman-House, A. How state right-to-try laws create false expectations. Health Affairs Blog. http://healthaffairs.org/blog/2015/05/22/how-state-right-to-try-laws-create-false-expectations (accessed 1 August 2015).

4. Adriance S. Fighting for the “right to try” unapproved drugs: law as persuasion. Yale Law Journal 2014;124(148). http://www.yalelawjournal.org/forum/right-to-try-unapproved-drugs (accessed 1 August 2015).

5. Goldwater Institute. Everyone deserves the right to try: empowering the terminally ill to take control of their treatment. http://goldwaterinstitute.org/en/work/topics/healthcare/right-to-try/everyone-deserves-right-try-empowering-terminally- (accessed 1 August 2015).

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