Faculty Bibliography

Hundreds of gene therapies are currently in various stages of research and development. A subset of these involve gene editing technologies such as CRISPR. In this hypothetical case, a patient with chronic pain has initiated a CRISPR-based intervention obtained from a clinic in the Cayman Islands. His physician doubts it is approved by the US Food and Drug Administration and worries about its safety. The case presents ethical questions about potential violations of US regulations regarding the sale of products intended to affect human health, patients' lack of understanding about risks of unproven drugs, and suboptimal support for and management of patients with chronic pain. We discuss how physicians should address these questions.

In the U.S., there is no requirement for research sponsors to compensate human research subjects who experience injuries as a result of their participation. In this article, we review the moral justifications that compel the establishment of a better research-related injury compensation system. We explore how other countries and certain institutions within the U.S. have adopted various systems of compensation. The existence of these systems demonstrates both that the U.S. lags behind other nations in its protection of human research subjects and that the establishment of a compensation system is both practical and feasible. We then examine factors which have prevented the U.S. from establishing its own compensation system. We consider possible alternatives for the U.S. by examining the advantages and disadvantages of both established and proposed systems. We offer a new proposal that addresses the justice concerns which compel the establishment of a national compensation system, distributes the burdens of such a system on multiple stakeholders that benefit from research, and has the additional advantage of minimizing the administrative and logistical challenges associated with initiating such a system.

Facial transplantation has gained increasing acceptance as a treatment option to improve quality of life (QoL) for persons suffering from severe facial disfigurement. Despite its growth, the field has yet to establish a consistent approach to assessing QoL in face transplant candidates and recipients that includes integration of meaningful patient-reported outcomes. The published literature suggests that face transplant programs currently use a wide variety of assessment tools and strategies. Moreover, confusion remains as to how best to weigh patients' lived experiences and incorporate them into QoL assessments. Qualitative research can illuminate the dimensions of QoL that are meaningful to face transplant candidates and recipients. Coupled with collaboration and data sharing across face transplant programs, qualitative research will help to bring conceptual clarity and transparency to the assessment process.

OBJECTIVE:To evaluate the availability of information regarding patient access to investigational treatments through clinical trials and non-trial pre-approval access pathways from a sample of patient advocacy organization (PAO) websites in the United States. RESULTS:We systematically analyzed the content of 118 randomly selected PAO websites to assess whether they contained information on clinical trials and non-trial pathways-e.g., the U.S. Food and Drug Administration (FDA) expanded access (EA) program and right to try-over the course of two months from February to March 2019. A majority (81%, n = 96) of PAOs provided a link to ClinicalTrials.gov, and 73% (n = 86) had their own clinical trial finder or list of relevant trials. 23% (n = 27) mentioned EA, with 8% (n = 9) providing specific resources for FDA's EA program. 8% (n = 10) provided a statement on the passage of the federal right to try law. A majority of PAO websites contained information on clinical trials, but a minority discussed non-trial pre-approval access. The lack of information on the latter highlights an area in need of improvement.

Objective: Posthumous assisted reproduction (PAR) raises complicated ethical and legal issues. ASRM recommends that assisted reproductive technology (ART) and fertility preservation (FP) programs develop written policies regarding cases of PAR, though little is known about adoption of such policies and how they have been implemented. Our objective was to assess the presence and content of policies toward PAR using oocytes and embryos amongSociety for Assisted Reproductive Technology (SART) member clinics in the U.S.
Design(s): Cross-sectional questionnaire-based study.
Material(s) and Method(s): Our study consists of three phases of communication: email-, postal mail-, and phone-based survey. We report on the first phase of anonymous email survey responses. Surveys were emailed to ASRM-member medical directors of all SART member clinics (n=332) during March and April 2019 using a modified Dillman Method; contact information was acquired from SART and ASRM membership data. The survey included 23 multiple-choice and 3 opened-ended questions assessing practice characteristics (practice type, location, IVF cycle volume), presence of a clinic policy towards PAR, and the content of such policy. Descriptive data are presented as %, with Fisher's exact test used where appropriate, and thematic content analysis was applied to open-ended responses.
Result(s): The first phase of the study received 39 clinic responses (12% response rate). Respondents were distributed across the U.S.; average volume of IVF cycles per year ranged from < 250 to > 1500. More than one-third (35.9%, n=14) of clinics reported participating in any cases of PAR over the past five years, and 5.1% (n=2) reported participation in more than five cases. Participation in cases of PAR was not significantly associated with practice type or IVF cycle volume (p>0.05). 57.9% (n=22) had written policies towards PAR using oocytes or embryos, while 36.8% (n=14) reported they did not have a policy. Practice type, IVF cycle volume, FP volume, and prior participation in cases of PAR were not significantly associated with the presence of a policy (p>0.05). Of those with a policy, 52.4% (n=11) reported they had used that policy, 66.7% (n=10) without a policy reported they had considered adopting one, and 60.0% (n=9) reported they had received a request for PAR services. Only 44% (n=15) of clinics specified that patients not expected to survive to use oocytes due to terminal illness were eligible for oocyte cryopreservation, while 50.0% (n=17) did not specify. Open-ended comments suggested need for case-by-case appraisal and firm consent polices regarding gamete disposition.
Conclusion(s): Our preliminary results suggest that SART programs are receiving an increasing number of requests for PAR services, but many SART programs lack PAR policies, and those with policies do not always follow ASRM recommendations. As PAR cases become more common, clinics should be equipped to manage the complexities of PAR. More data are needed as this study continues, and future research is needed to understand barriers to the creation and implementation of these increasingly needed policies.
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