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Ethical Issues in Gender-Affirming Care for Youth

Kimberly, Laura L; Folkers, Kelly McBride; Friesen, Phoebe; Sultan, Darren; Quinn, Gwendolyn P; Bateman-House, Alison; Parent, Brendan; Konnoth, Craig; Janssen, Aron; Shah, Lesha D; Bluebond-Langner, Rachel; Salas-Humara, Caroline
Transgender and gender-nonconforming (TGNC) youth who suffer from gender dysphoria are at a substantially elevated risk of numerous adverse physical and psychosocial outcomes compared with their cisgender peers. Innovative treatment options used to support and affirm an individual's preferred gender identity can help resolve gender dysphoria and avoid many negative sequelae of nontreatment. Yet, despite advances in these relatively novel treatment options, which appear to be highly effective in addressing gender dysphoria and mitigating associated adverse outcomes, ethical challenges abound in ensuring that young patients receive appropriate, safe, affordable treatment and that access to this treatment is fair and equitable. Ethical considerations in gender-affirming care for TGNC youth span concerns about meeting the obligations to maximize treatment benefit to patients (beneficence), minimizing harm (nonmaleficence), supporting autonomy for pediatric patients during a time of rapid development, and addressing justice, including equitable access to care for TGNC youth. Moreover, although available data describing the use of gender-affirming treatment options are encouraging, and the risks of not treating TGNC youth with gender dysphoria are evident, little is known about the long-term effects of both hormonal and surgical interventions in this population. To support ethical decision-making about treatment options, we encourage the development of a comprehensive registry in the United States to track long-term patient outcomes. In the meantime, providers who work with TGNC youth and their families should endeavor to offer ethically sound, patient-centered, gender-affirming care based on the best currently available evidence.
PMID: 30401789
ISSN: 1098-4275
CID: 3520072

Fetal Therapies and Clinical Research: Beyond Risk and Benefit

Shah, Lesha D.; Lantos, John; Hunt, Cara; McFadyen, Andrew; Escandon, Rafael; Bateman-House, Alison
ISSN: 1526-5161
CID: 5189112

Somatic Gene Therapy Research in Pediatric Populations: Ethical Issues and Guidance for Operationalizing Early Phase Trials

Bateman-House, Alison; Shah, Lesha D; Escandon, Rafael; McFadyen, Andrew; Hunt, Cara
Currently, pediatric research involving investigational gene therapies (GT, used without intending to imply a therapeutic effect) targets a broad range of indications (including rare and ultra-rare diseases) that vary in severity and availability of approved disease-modifying therapies. Because of this diversity of circumstances, there is no one-size-fits-all list of ethical concerns relevant to all uses of investigational GTs in children. Here, we review the main ethical issues, specifically those surrounding the current state of knowledge about GT product-related immunogenicity, toxicity, duration, irreversibility, informed consent/assent, trial design (including the question of who 'goes first'), participant and caregiver burdens, and equity in diagnosis and access to research opportunities. Ethical issues that can be anticipated to arise in pediatric GT clinical trials, e.g., the uncertainty and risk of this research, the resultant preclusion of GT trial participants from other research, the length of follow-up monitoring, and the urgency often felt by caregivers dealing with dire, rapidly progressive conditions, should be proactively identified, addressed in accordance with existing best practices, and transparently discussed among all stakeholders.
PMID: 36527677
ISSN: 1179-1993
CID: 5382622

Ethically Allocating COVID-19 Drugs Via Pre-approval Access and Emergency Use Authorization

Webb, Jamie; Shah, Lesha D.; Lynch, Holly Fernandez
Allocating access to unapproved COVID-19 drugs available via Pre-Approval Access pathways or Emergency Use Authorization raises unique challenges at the intersection of clinical care and research. In conditions of scarcity, prioritization approaches should minimize harm, maximize benefit, and promote fairness. To promote continued data collection, patients seeking access to unproven COVID-19 drugs should receive lower priority for allocation when they decline to participate in clinical trials, either of the requested drug or other investigational products, offering a comparable balance of risks and benefits; special attention should be paid to concerns of voluntariness and distrust. In addition, institutional treatment protocols that can contribute more robust real world data should be preferred to single patient requests for access, with priority for inclusion based on traditional clinical allocation criteria relying on available evidence. Fairness demands distribution of these protocols across a diverse range of sites, particularly those serving marginalized populations, among other protections.
ISSN: 1526-5161
CID: 4611882

Implementation of a gene therapy education initiative by the ASGCT and Muhimbili University of Health and Allied Sciences [Letter]

Cornetta, Kenneth; Kay, Samantha; Urio, Florence; Minja, Irene Kida; Mbugi, Erasto; Mgaya, Josephine; Mselle, Teddy; Nkya, Siana; Alimohamed, Mohamed Zahir; Ndaki, Kinuma; Bonamino, Martín; Koya, Richard C; Shah, Lesha D; Mahlangu, Johnny; Drago, Daniela; Rangarajan, Savita; Jayandharan, Giridhara Rao
There has been rapid growth in gene therapy development with an expanding list of approved clinical products. Several therapies are particularly relevant to patients in low- and middle-income countries. Moreover, investing in research and manufacturing presents an opportunity for economic development. To increase awareness of gene therapy, the American Society of Gene and Cell Therapy partnered with the Muhimbili University of Health and Allied Sciences, Tanzania, to create a certificate-bearing course. The goal was to provide faculty teaching in graduate and medical schools with the tools needed to add gene therapy to the university curriculum. The first virtual course was held in October of 2022, and 45 individuals from 9 countries in Africa completed the training. The content was new to approximately two-thirds of participants, with the remaining third indicating that the course increased their knowledge base. The program was well received and will be adapted for other under-resourced regions.
PMID: 37595584
ISSN: 1525-0024
CID: 5598452

Siblings and Discordant Eligibility for Gene Therapy Research: Considering Parental Requests for Non-Trial "Compassionate Use"

Webb, Jamie; Shah, Lesha D.; Bateman-House, Alison
Deciding whether to grant an expanded access request for a child whose sibling is enrolled in a gene therapy trial involves a number of complex factors: considering the best interests of the child, the psychosocial and economic impact on the family, and the concerns and obligations of researchers. Despite the challenges in coming to a substantively fair outcome in cases of discordant eligibility, creating a procedurally fair decision-making process to adjudicate requests is essential.
ISSN: 1477-7509
CID: 4768902

A survey of pediatric hematologists/oncologists' perspectives on single patient Expanded Access and Right to Try

Chapman, Carolyn Riley; Belli, Hayley M; Leach, Danielle; Shah, Lesha D; Bateman-House, Alison
INTRODUCTION/UNASSIGNED:Physicians in the United States play an essential role guiding patients through single patient pre-approval access (PAA) to investigational medical products via either the Food and Drug Administration (FDA)'s Expanded Access (EA) or the federal Right To Try (RTT) pathways. In this study, we sought to better understand pediatric hematologist/oncologists' attitudes about seeking PAA, on behalf of single patients, to investigational drugs outside of clinical trials. METHODS/UNASSIGNED:A cross-sectional survey was developed and sent to pediatric hematologist/oncologists via St. Baldrick's Foundation's email distribution list. RESULTS/UNASSIGNED: = 46) had prior experience with single patient PAA. Respondents were most concerned about the unknown risks and benefits of investigational drugs and financial implications of PAA for patients. One hundred percent and 91.1% of respondents indicated a willingness to support patients through EA and RTT pathways, respectively. When asked about their most recent experience with PAA, 40 out of 46 indicated that they used the FDA's EA pathway to seek PAA and 4 out of 46 indicated that they used the RTT pathway. Of 44 respondents who had used the EA or RTT pathway, 43 indicated that the biotechnology or pharmaceutical company they solicited granted access to the requested product. CONCLUSION/UNASSIGNED:Survey results support other findings suggesting a need for additional physician support and education about PAA and that physicians may have unequal access to information about investigational drugs and concerns about financial implications of PAA for their patients.
PMID: 36204503
ISSN: 2399-2026
CID: 5427792

No Easy Answers in Allocating Unapproved COVID-19 Drugs Outside Clinical Trials [Letter]

Webb, Jaime; Shah, Lesha; Lynch, Holly Fernandez
PMID: 32840448
ISSN: 1536-0075
CID: 4614422

Preapproval Nontrial Access and Off-Label Use: Do They Meet Criteria for Dual-Deviation Review?

Chapman, Carolyn Riley; Folkers, Kelly McBride; McFadyen, Andrew; Shah, Lesha D; Bateman-House, Alison
PMID: 31135320
ISSN: 1536-0075
CID: 3898892

Morbidity and Mortality in Hospitalized versus Outpatient Lung Transplant Recipients [Meeting Abstract]

Rudym, D.; Benvenuto, L.; Kim, H.; Shah, L.; Aversa, M.; Robbins, H.; Hook, J.; D\Ovidio, F.; Bacchetta, M.; Sonett, J.; Arcasoy, S.
ISSN: 1053-2498
CID: 3979842