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Right-to-Try Laws: Hope, Hype, and Unintended Consequences

Bateman-House, Alison; Kimberly, Laura; Redman, Barbara; Dubler, Nancy; Caplan, Arthur
PMID: 26413841
ISSN: 1539-3704
CID: 1882622

Should patients in need be given access to experimental drugs?

Caplan, Arthur L; Bateman-House, Alison
Patient access to experimental drugs outside of clinical trials is called compassionate use or expanded access. Compassionate use/expanded access presents a powerful ethical dilemma in that it involves competing claims that both have moral weight: specifically, an individual patient's very understandable desire to try to extend his or her life versus the orderly and efficient functioning of a drug development and clinical trial system that benefits much larger numbers of patients. Patient advocates, the FDA, pharmaceutical trade groups, and state and national legislators in the US are all currently weighing in on patient access to experimental drugs, and new guidelines and rules are being introduced. In this editorial, we discuss the impulse to rescue individual patients facing dire diseases and underscore the ethical questions that such rescue efforts raise.
PMID: 26001178
ISSN: 1744-7666
CID: 1591212

The Lived Experience of Pediatric Gene Therapy: A Scoping Review

Kimberly, Laura; Hunt, Cara; Beaverson, Katherine; James, Emma; Bateman-House, Alison; McGowan, Richard; DeSante-Bertkau, Jennifer
Little is known about patients' and families' lived experiences of participating in pediatric gene therapy (GT) clinical trials. Currently, pediatric GT research targets a broad range of indications--including rare and ultra-rare diseases--which vary in severity and in the availability of alternative therapies. Pediatric GT differs meaningfully from adult GT because the decision to participate involves a dyad of both the child and parent or caregiver/s. It is critical to understand patients' and caregivers' perceptions and experiences of social, emotional, physical, and logistical burdens or benefits of participating in such trials, and how they weigh and prioritize these factors when deciding whether to participate. We conducted a scoping review of the current literature in this subject area with objectives to (1) provide an overview of existing literature, (2) identify gaps and areas for further research, and (3) better understand the lived impact of pediatric GT research on patients and their parents/caregivers. Four themes emerged, including (1) weighing risks and benefits (2) timing of GT trial participation, (3) value of clear communication, and (4) potential impact on quality of life. Notably, our sample surfaced articles about how patients/parents/caregivers were thinking about GT-their understanding of its safety, efficacy, and risks-rather than accounts of their experiences, which was our initial intention. Nevertheless, our findings offer useful insights to improve the informed consent process and promote a more patient- and family-centered approach. Moreover, our findings can contribute to patient advocacy organizations' efforts to develop educational materials tailored to patients' and families' expressed informational needs and perspectives, and can inform more patient- and family-centered policies from GT clinical trial sponsors.
PMID: 37964764
ISSN: 1557-7422
CID: 5607822

Navigating the Expanded Access Pathway to Investigational Drugs as an Academic Oncologist

Fernandez Lynch, Holly; Salam, Tasnim; Gould, Patrick; Bateman-House, Alison; Kimberly, Laura
PMCID:9938427
PMID: 36800184
ISSN: 2574-3805
CID: 5421212

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

Implications of Pediatric Initiatives on CNS Drug Development for All Ages-2020 and Beyond: Second of Three Sets of Expanded Proceedings from the 2020 ISCTM Autumn Conference on Pediatric Drug Development

Pandina, Gahan; Busner, Joan; Horrigan, Joseph P; McSherry, Christine; Bateman-House, Alison; Pani, Luca; Kando, Judith
This article expands upon a session, titled "Implications of Pediatric Initiatives on CNS Drug Development for All Ages-2020 and Beyond," that was presented as part of a two-day meeting on pediatric drug development at the International Society for Central Nervous System (CNS) Clinical Trials and Methodology (ISCTM) Autumn Conference in Boston, Massachusetts, in October 2020. Speakers from various areas of pediatric drug development addressed a variety of implications of including children in drug development programs. The speakers wrote summaries of their talks, which are included here. The session's lead chair was Dr. Gahan Pandina, who wrote introductory and closing comments. Dr. Joseph Horrigan addressed the current landscape of pediatric development programs. Dr. Gahan Pandina addressed how the approach to research in pediatric populations affects the drug development process and vice versa. Dr. Alison Bateman-House discussed the ethical implications of research in the pediatric population. Dr. Luca Pani discussed some of the global regulatory issues and challenges concerning research in pediatric patients. Dr. Judith Kando served as a discussant and posed new questions about means of facilitating pediatric research. Finally, Dr. Gahan Pandina provided closing comments and tied together the presented issues. This paper should serve as an expert-informed reference to those interested and involved in CNS drug development programs that are aimed at children and/or required, through regulations, to include children as part of the approval process.
PMCID:10132276
PMID: 37122578
ISSN: 2158-8333
CID: 5544712

Perspectives of Academic Oncologists About Offering Expanded Access to Investigational Drugs

Gould, Patrick; Salam, Tasnim; Kimberly, Laura; Bateman-House, Alison; Fernandez Lynch, Holly
Importance/UNASSIGNED:The expanded access (EA) pathway permits patients to be treated with investigational medical products outside clinical trials. Because cancer care is a common indication for which EA is sought and these efforts require physician management, understanding oncologists' perspectives can help illuminate factors influencing patient access. Objective/UNASSIGNED:To learn how oncologists practicing at academic medical centers (AMCs) perceive EA and their role in offering it. Design, Setting, and Participants/UNASSIGNED:This qualitative study used data from semistructured interviews conducted from February 2020 to September 2021 with a purposive sample of oncologists recruited from large, urban AMCs in the northeast United States. Oncologists who had submitted at least 1 single-patient EA request to the institutional review boards at the University of Pennsylvania, Children's Hospital of Philadelphia, NYU Langone Health, and Dana-Farber Cancer Institute from January 1, 2014, through January 31, 2020, were eligible to participate. Data were analyzed from July 2021 to March 2022. Main Outcomes and Measures/UNASSIGNED:Interviews focused on oncologist practice demographics, experience with EA, factors relevant to decisions to pursue EA and comfort with those decisions, perspectives on oncologists' role in EA, perspectives on the FDA's role, and the Right to Try pathway to access investigational drugs. Results/UNASSIGNED:Eligible oncologists were interviewed until thematic saturation was reached, resulting in 25 interviews; most participants were women (15 participants [60%]), reported primarily treating adult patients (15 participants [60%]), had more than 10 years of clinical experience (16 participants [64%]), and had submitted at least 2 single-patient EA requests to their institutional review boards during the relevant period (14 participants [56%]). Oncologists viewed EA as an important tool for securing what they determined to be the best treatment option for their patients based on their own expert assessment of available data. Interviewees reported that they would rather access interventions as commercially available products or through clinical trials; however, if the preferred option was not available through these means, they viewed pursuit of EA as part of their obligation to patients, while often recognizing the potential for inequities in the broader patient population beyond their institutions. Participating oncologists felt confident pursuing investigational drugs for treatment use, despite the absence of FDA marketing approval, and did not necessarily view EA as a last resort. Conclusions and Relevance/UNASSIGNED:These findings indicate that oncologists practicing in large academic settings sought to treat patients with the interventions they deemed most likely to be beneficial, regardless of approval status. As such, they viewed EA as an unexceptional means to obtain promising products, although it remains unclear whether their confidence in evaluating investigational treatments was justified. Future research should examine whether oncologists outside large AMCs share this confidence, as differences may influence patient access to the EA treatment pathway.
PMID: 36318206
ISSN: 2574-3805
CID: 5358252

Individualized Therapeutics Development for Rare Diseases: The Current Ethical Landscape and Policy Responses

Bateman-House, Alison; Kearns, Lisa
The first individualized therapy was administered in the United States just 2 years ago, when milasen, a therapeutic adapted from a Food and Drug Administration (FDA)-approved antisense oligonucleotide technology, was developed for a young girl with an extremely rare genetic mutation associated with Batten disease. Since then there has been an explosion of enthusiasm in developing customized treatments for extremely rare genetic conditions. These interventions raise some of the ethics concerns characteristic of novel therapeutics while simultaneously challenging existing legal, regulatory, and ethical understandings. Their individualized aspect blurs to the point of erasing the historically distinct line separating research from treatment, leading regulators and ethics oversight bodies to reevaluate existing policies. As experimental therapeutics, they raise the potential for both compromised informed consent and conflicts of interest, and their considerable expense provokes serious justice concerns. This article examines these challenges, urges multidisciplinary stakeholder engagement to address them in a transparent and practicable manner, and recommends initial policy responses.
PMID: 34797685
ISSN: 2159-3345
CID: 5049712

Fetal Therapies and Clinical Research: Beyond Risk and Benefit

Shah, Lesha D.; Lantos, John; Hunt, Cara; McFadyen, Andrew; Escandon, Rafael; Bateman-House, Alison
SCOPUS:85126196374
ISSN: 1526-5161
CID: 5189112

Treating "N-of-Few" Genetic Conditions with Antisense Oligonucleotides (ASOs): Current State and Ethical Challenges [Meeting Abstract]

Massarelli, John; Bateman-House, Alison
ISI:000794043700214
ISSN: 1525-0016
CID: 5244122