participated in the conceptualization of this project, drafted portions of the manuscript, and contributed feedback to subsequent drafts; Dr Bluebond-Langner participated in the conceptualization of this project and contributed feedback to subsequent drafts; and all authors approved the final manuscript as submitted and agree to be accountable for all aspects of the work.
We report on the deliberations of an interdisciplinary group of experts in science, law, and philosophy who convened to discuss novel ethical and policy challenges in stem cell research. In this report we discuss the ethical and policy implications of safety concerns in the transition from basic laboratory research to clinical applications of cell-based therapies derived from stem cells. Although many features of this transition from lab to clinic are common to other therapies, three aspects of stem cell biology pose unique challenges. First, tension regarding the use of human embryos may complicate the scientific development of safe and effective cell lines. Second, because human stem cells were not developed in the laboratory until 1998, few safety questions relating to human applications have been addressed in animal research. Third, preclinical and clinical testing of biologic agents, particularly those as inherently complex as mammalian cells, present formidable challenges, such as the need to develop suitable standardized assays and the difficulty of selecting appropriate patient populations for early phase trials. We recommend that scientists, policy makers, and the public discuss these issues responsibly, and further, that a national advisory committee to oversee human trials of cell therapies be established. (Fertil Steril 2003;80:1077-85.
If stem cells fulfill their therapeutic promise, moving them from the laboratory into the clinic will raise several concerns about justice. One concern is that, for biological reasons alone, stem cell‐based therapies might not be available for every patient who needs one. Worse, depending on how we address the problem of biological access, they might benefit primarily white Americans. We can avoid this outcome—although at a cost—by carefully selecting the stem cells we make available.
In 2017, President Trump said that "one thing that's always disturbed" 1 him is that the US Food and Drug Administration (FDA) denies access to experimental drugs even "for a patient who's terminal… [who] is not going to live more than four weeks [anyway.]" 1 Fueled by emotionally charged anecdotes recirculated by libertarian political activists, 38 states have passed Right to Try laws. In 2017, the US Senate approved a bill that would create a national law (Box). 2 As of December 2017, the US House of Representatives was considering the bill.Although the FDA has an expanded access option for utilizing experimental drugs outside of clinical trials, Right to Try laws create an alternative pathway that bypasses the agency. Moreover, the term "Right to Try" is a misnomer: the legislation creates a right for a patient to ask a company to provide a product, the same right that currently exists. 3 As is the case with the FDA program, companies are not obligated to provide access.The proposed federal Right to Try legislation 2 sets the threshold for patients to access investigational drugs at the completion of phase I (dosage-determination) trials, grants companies and physicians broad immunity from liability, and largely blinds the FDA to safety or efficacy data from these therapeutic attempts. Such changes would upend the agency's expanded access program, which has worked successfully for decades to provide patients who are seriously ill and without therapeutic options access to investigational drugs and still assure the safety and efficacy of new drugs before these products gain marketing approval. The legislation does not solve actual concerns, such as lack of knowledge that the program exists and the very limited supply of many investigational products.A 2016 review 4 of 10 years of FDA records found that the Center for Drug Evaluation and Research receives over 1000 expanded access applications per year. The FDA reviews such requests in days-or hours when an emergency so requires-and approves over 99%. 5 This is no mere rubber stamp of proposals: unlike individual physicians, the FDA has access to proprietary data about the risks and benefits of the investigational product or others in the same class. In some instances, the agency can improve the proposed treatment via modifications of drug dosage, dosing schedule, or other aspects. 6 Eliminating the FDA's review of expanded access requests is perilous, because most of the drugs that succeed in phase 1 trials turn out to be too unsafe or ineffective for clinical use. Phase 1 trials are intended to find a reasonable dosage of a drug in a small number of subjects, who may not even have the disease in question.
Patients have received experimental pharmaceuticals outside of clinical trials for decades. There are no industry-wide best practices, and many companies that have granted compassionate use, or 'preapproval', access to their investigational products have done so without fanfare and without divulging the process or grounds on which decisions were made. The number of compassionate use requests has increased over time. Driving the demand are new treatments for serious unmet medical needs; patient advocacy groups pressing for access to emerging treatments; internet platforms enabling broad awareness of compelling cases or novel drugs and a lack of trust among some that the pharmaceutical industry and/or the FDA have patients' best interests in mind. High-profile cases in the media have highlighted the gap between patient expectations for compassionate use and company utilisation of fair processes to adjudicate requests. With many pharmaceutical manufacturers, patient groups, healthcare providers and policy analysts unhappy with the inequities of the status quo, fairer and more ethical management of compassionate use requests was needed. This paper reports on a novel collaboration between a pharmaceutical company and an academic medical ethics department that led to the formation of the Compassionate Use Advisory Committee (CompAC). Comprising medical experts, bioethicists and patient representatives, CompAC established an ethical framework for the allocation of a scarce investigational oncology agent to single patients requesting non-trial access. This is the first account of how the committee was formed and how it built an ethical framework and put it into practice.
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