Free to Choose: A Moral Defense of the Right-to-Try Movement

The claim that individuals legitimately differ with respect to their values seems to be uncontroversial among bioethicists, yet many bioethicists nevertheless oppose right-to-try laws. This seems to be due in part to a failure to recognize that such laws are intended primarily to be political, not legal, instruments. The right-to-try movement seeks to build political support for increasing access to newly developed drugs outside of clinical trials. Opponents of right-to-try laws claim that increasing access outside of clinical trials would undermine evidence-based medicine. They seek to maximize overall gains to patients by protecting them from adverse drug reactions and ensuring that drugs are more effective on average. In contrast, right-to-try activists have a point that regulatory judgments of drug safety and effectiveness impose one set of trade-offs on all individuals, regardless of their different values. That might be acceptable if determinations of safety and effectiveness were black and white, but that does not seem to be the case. This article argues that judgments of safety and effectiveness are in an important respect normative and reflect the perceived value of those ends relative to others. Such judgments, when universally imposed, harm patients who would readily make do with less knowledge of drug safety and effectiveness in exchange for more time and self-determination. The relevant moral principle is that of respect for individual autonomy. Just as that principle should lead one to substitute collective decisions for individual ones to regulate a natural monopoly, the same principle should lead one to substitute individual decisions for collective ones to avoid a government monopoly on access to newly developed drugs. It is argued that reforms should increase the number of treatment options available to patients outside of clinical trials. The final section of the article discusses ways in which current regulations might be reformed so as to provide more treatment options outside of clinical trials, without undermining evidence-based medicine.

New Non-Validated Practice: An Enhanced Definition of Innovative Practice for Medicine

A significant part of the literature on innovative practice in medicine relates to seizing opportunities and curbing harms for patients in desperate situations. Unfortunately, the term innovation has multiple meanings and a rich rhetorical flourish that adds confusion and misunderstanding to an already difficult debate. This paper aims to enhance the current definition of innovative practice for medicine. First, we replace the term innovation with the more literal of new non-validated practice. To identify this meaning, we analyse the traditional research ethics’ distinction between research, validated practice, and innovation in the Belmont Report. Second, we propose the following explicit definition of new non-validated practice: the first or recent use of diagnostic, therapeutic or preventive interventions that introduce a significant change, with an insufficient level of evidence of safety or efficacy for regular healthcare, and with the main aim to benefit individual patients. This definition is a promising conceptual tool to inform empirical research, ethicists, and the harmonization of its regulation and legislation (e.g. right-to-try laws).

Seizing Opportunity at the Limits of Medicine: An Enhanced Definition of Innovative Practice

The activity of innovative practice relates to seizing opportunities now for patients in desperate situations and the ethical dilemmas around it. Sadly, the term innovation has multiple meanings, adding confusion and misunderstanding to an already difficult debate. This paper aims to enhance the definition of innovative practice in two ways. Fist, we propose to curb ambiguity by replacing the term "innovative practice" with the more precise of "new non-validated practice" and by distinguishing the specific meaning of innovation at stake. To identify this meaning, we analyse the traditional research ethics’ distinction between research, validated practice, and innovation. Second, we propose the following unified definition of "new non-validated practice", that is, the first or recent use of diagnostic, therapeutic or preventive interventions that introduce a significant change (new); with an insufficient level of evidence of safety or efficacy for regular healthcare (non-validated); and with the main aim to benefit patients (practice). To avoid objections to our definition, we analyse its three core elements (significant novelty, insufficient validation for, and patients' best interests) and we present a 2D classification of medical practice that follows from our definition. If sound, our enhanced definition of "new non-validated practice" allows comparing exceptional activities in different fields of medicine that previously have been considered unconnected (e.g. compassionate use of investigational drugs, humanitarian uses of devices, novel off-label uses, etc.).  In turn, it also provides a promising conceptual tool to inform empirical research, discuss responsible access to innovative care and evaluate the regulation of innovative practice (e.g. right-to-try laws).

Estimating Causal Effects of New Treatments Despite Self-Selection: The Case of Experimental Medical Treatments

Providing terminally ill patients with access to experimental treatments, as allowed by recent “right to try” laws and “expanded access” programs, poses a variety of ethical questions. While practitioners and investigators may assume it is impossible to learn the effects of these treatment without randomized trials, this paper describes a simple tool to estimate the effects of these experimental treatments on those who take them, despite the problem of selection into treatment, and without assumptions about the selection process. The key assumption is that the average outcome, such as survival, would remain stable over time in the absence of the new treatment. Such an assumption is unprovable, but can often be credibly judged by reference to historical data and by experts familiar with the disease and its treatment. Further, where this assumption may be violated, the result can be adjusted to account for a hypothesized change in the non-treatment outcome, or to conduct a sensitivity analysis. The method is simple to understand and implement, requiring just four numbers to form a point estimate. Such an approach can be used not only to learn which experimental treatments are promising, but also to warn us when treatments are actually harmful – especially when they might otherwise appear to be beneficial, as illustrated by example here. While this note focuses on experimental medical treatments as a motivating case, more generally this approach can be employed where a new treatment becomes available or has a large increase in uptake, where selection bias is a concern, and where an assumption on the change in average non-treatment outcome over time can credibly be imposed.

RxLegal: A Rapid Review of Right-To-Try

Right-to-try legislation is intended to allow patients with life-threatening illnesses access to investigational medical treatments without formal Food and Drug Administration (FDA) involvement. Currently, right-to-try laws have been enacted in 40 states. Despite the increased passage of right-to-try legislation at the state level, individuals have detailed arguments both for and against these laws. Proponents state that right-to-try removes regulatory burdens and improves timely access to potentially lifesaving medications for terminally ill patients, reduces inequalities regarding access, and improves patient-provider communication and decision making. Opponents argue that right-to-try does not really guarantee access, reinforces preexisting health care inequalities, prioritizes rapid access over safety and the interests of the individual over the public, and produces concerns regarding informed consent. Despite these issues, right-to-try has recently gained traction on the federal level with both Congressional chambers passing separate bills.