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Patients deserve better than a ‘Right to [Hope to] Try’

This section recently featured commentary I coauthored and a thoughtful response piece by Matthew Bellina, a patient and advocate for the ALS community, supporting and critiquing a legislative movement called “Right to Try.” Right to Try laws facially appear to enact a direct right to what’s commonly known as “Compassionate Use” access for individual patients to very early phase experimental drugs and devices, outside of clinical trials or existing pre-approval access routes. The Right to Try movement not only has swept through 31 states and is the subject of two pending federal Congressional bills, but in an important development, was recently adopted as part of the official GOP platform.

This unprecedented level of attention on possible ways of reforming how promising new therapies are tested, approved, and timely and appropriately made available patients, is both warranted and welcome. However, the almost singular focus of attention on legally-ineffective Right to Try legislation is a tragic waste of time and a squandering of opportunity to make any meaningful difference in these life and death issues.

{mosads}In our original piece, my colleague and I pleaded with federal and state legislatures to halt further consideration and passage of Right to Try laws, citing some of the many legal and ethical issues that have been raised upon reading the bills’ entire texts rather than just the high-level verbiage used to depict the bills’ goals. At base, however, one of the most important critiques arises from a simple legal conclusion:  these bills in NO WAY help patients gain access to otherwise unavailable interventions, or provide additional options for patients to determine their own course of treatment that don’t already exist under current rules. The bills merely assert that people have right to try TO REQUEST access to experimental therapies, but they do not, and legally cannot, actually effectuate a real right to try.

A “right” does not exist unless there is some obligation for others to recognize that right. For example, if I am granted a patent right to a new invention, another individual cannot use that invention without permission. Further, I can try to enforce and protect my patent right by appealing to a court of law to adjudicate my claim and to levy penalties against those who have violated my rights. In comparison, the so-called “right” to try laws assert that patients have certain access rights, but they impose no concomitant requirements on ANY person or entity to recognize this right, to act to protect this right, or to do anything to ensure fulfillment of this right in any way – for instance, by mandating provision of early access upon request or by allowing sanctions for failure to recognize another’s right to try. This makes the purported right to try non-existent. The legislation does nothing more than repeat the legal status quo:  patients only have the “right” to ASK for early access.

Worse, the legislation implies that state law somehow effectively allows companies, research institutions and others that are bound by federal regulation of experimental drugs to bypass federal mechanisms (it cannot), and that the law provides these entities with almost universal discretion to determine whether they will or must provide an experimental drug (it does not). These highly misleading legal assertions only further muddies the understanding of import and impact of these laws -or, the lack thereof.  

Mr. Bellina’s reply to our commentary both defended Right to Try laws, and went further to address some of the very challenging issues underlying the compassionate use area and provide some forward-thinking proposals. Unfortunately, Mr. Bellina read our critique of the bills as aimed at taking away or second-guessing a person’s right to make decisions regarding their own health care, but this is far from what we intended. In fact, an individual’s right to autonomous decision-making is one of the most central tenets of medical ethics. Rather, our goal was to stop ineffective and unethical legislation.

While ineffective laws may not normally be particularly problematic, here, the debate over these hollow laws has distracted from and effectively staunched meaningful dialog that might lead to impactful changes to the very real access problems raised. The issues surrounding compassionate use were already challenging prior to the tensions that have accompanied the Right to Try legislation. This is not to say that many stakeholders in the compassionate use arena could not do better:  there is a historic lack of transparency on how access decisions are made and by whom; apprehension about how and to what extent the FDA might factor adverse events that may be due to a patients’ co-morbidities when reviewing a drug for ultimate approval; questions of how decisions can be more fair and just for all patients in need and not just those who have access to learned clinicians who are aware of drugs in the pipeline and who understand how to make compassionate use requests.

We must also examine the many worthwhile proposals made by Mr. Bellina and others for reforms that may shorten the lengthy timelines drugs are made available, provide different or additional ways of gathering valid and comprehensive safety and efficacy data, analyze newer clinical trial designs and/or allow for adaption of designs when appropriate that provide active drug to participants earlier in the trials process, and a host of other mechanisms that might lead to beneficial improvements for both current and future patients.

It would be foolish to assert that even the most comprehensive reforms and changes would prevent the very understandable frustrations and agony of terminal patients and their loved ones, but we can certainly do better than we are now. And we will not arrive at “better” if we continue to spend precious time debating one ineffective piece of legislation – or, if we continue to allow this debate to devolve into increasingly divisive rhetoric that pits stakeholders who need to work together on these issues as against each other.

If patients, advocates, legislatures and others genuinely want to move the needle on compassionate use, we need to look beyond the misleading characterizations of these Right to Try bills, roll up our sleeves and try to find meaningful solutions to these complex issues.

Patients in need do not have the time to spare on empty legislative measures.

Beth E. Roxland, J.D., M.Bioethics, is an Associate of the NYU Langone Medical Center’s Division of Medical Ethics, and serves on the NYULMC’s Working Group on Compassionate Use and Pre-Approval Access.  


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