Alison Bateman-House
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View article: Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UK
Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UK Open
Patient and caregiver treatment preferences should be incorporated into the drug development process. We updated a 2018 survey to reflect current knowledge about gene therapy for Duchenne muscular dystrophy (DMD) and obtained new data in t…
View article: From roadmap to a sustainable end-to-end individualized therapy pathway
From roadmap to a sustainable end-to-end individualized therapy pathway Open
The field of individualized, or N-of-1, therapy development is growing and increasingly gaining attention as a novel option for people with serious diseases, caused by unique genetic variants for whom approved therapies are not available. …
View article: Lived Experience of Patients and Caregivers in Rare Genetic Neurological Gene Therapy Clinical Trials in Children
Lived Experience of Patients and Caregivers in Rare Genetic Neurological Gene Therapy Clinical Trials in Children Open
To date, sparse attention has been paid to the importance of the "lived experience" of participants and their caregivers in pediatric gene therapy (GT) trials for rare genetic neurological disorders. Pediatric GT studies differ meaningfull…
View article: Somatic Gene Therapy: Ethics and Access
Somatic Gene Therapy: Ethics and Access Open
Manipulation of a patient's genome for therapeutic ends is being attempted through numerous methods, some of which have resulted in disease-modifying interventions. The much anticipated promise of somatic gene therapy is starting to pay of…
View article: Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy
Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy Open
Background: Duchenne muscular dystrophy (DMD) is a progressive, life-limiting, neuromuscular disorder. Clinicians play an important role in informing families about therapy options, including approved gene therapies and clinical trials of …
View article: ACT To Sustain: Adoptive Cell Therapy To Sustain Access to Non-Commercialized Genetically Modified Cell Therapies
ACT To Sustain: Adoptive Cell Therapy To Sustain Access to Non-Commercialized Genetically Modified Cell Therapies Open
View article: Navigating the Expanded Access Pathway to Investigational Drugs as an Academic Oncologist
Navigating the Expanded Access Pathway to Investigational Drugs as an Academic Oncologist Open
This qualitative study explores academic oncologists’ needs and satisfaction with expanded patient access to investigational drugs.
View article: 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.
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. Open
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 So…
View article: Perspectives of Academic Oncologists About Offering Expanded Access to Investigational Drugs
Perspectives of Academic Oncologists About Offering Expanded Access to Investigational Drugs Open
Importance 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…
View article: Gene therapy companies have an ethical obligation to develop expanded access policies
Gene therapy companies have an ethical obligation to develop expanded access policies Open
In the US, gene therapy companies, as with all biopharmaceutical companies, are not required by law or regulation to provide their investigational products to patients via non-trial preapproval (expanded) access. However, we believe that g…
View article: Siblings and Discordant Eligibility for Gene Therapy Research: Considering Parental Requests for Non-Trial "Compassionate Use”
Siblings and Discordant Eligibility for Gene Therapy Research: Considering Parental Requests for Non-Trial "Compassionate Use” Open
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 t…
View article: A survey of pediatric hematologists/oncologists’ perspectives on single patient Expanded Access and Right to Try
A survey of pediatric hematologists/oncologists’ perspectives on single patient Expanded Access and Right to Try Open
Introduction: 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 …
View article: Transparency is key to ethical vaccine research—Response
Transparency is key to ethical vaccine research—Response Open
Estep and Church depict as their end goal the development of a safe and effective vaccine. We have a different goal: the production of a safe and effective vaccine that the majority of possible recipients will choose to use and that has re…
View article: Emergency Approvals for COVID-19: Evolving Impact on Obligations to Patients in Clinical Care and Research
Emergency Approvals for COVID-19: Evolving Impact on Obligations to Patients in Clinical Care and Research Open
There currently is debate regarding whether U.S. institutions and clinicians may or should restrict patient access to COVID-19 drugs and vaccines that have been granted emergency use authorization by the U.S. Food and Drug Administration. …
View article: Fecal microbiota transplantation: Uses, questions, and ethics
Fecal microbiota transplantation: Uses, questions, and ethics Open
View article: Is Right to Try Being Tried? Using Crowdfunding Data to Better Understand Usage of Nontrial Pre-Approval Access Pathways
Is Right to Try Being Tried? Using Crowdfunding Data to Better Understand Usage of Nontrial Pre-Approval Access Pathways Open
Aim: The US FDA has two nontrial pre-approval access pathways: expanded access (EA) and right to try (RTT). Reports of successful RTT use are scarce, and the FDA has not yet published RTT usage data, yet proponents tout its utility.…
View article: Mandatory Bicycle Helmet Laws in the United States: Origins, Context, and Controversies
Mandatory Bicycle Helmet Laws in the United States: Origins, Context, and Controversies Open
This article examines the origins and context of mandatory bicycle helmet laws in the United States. Localities began to enact such laws in the early 1990s, having experimented with helmet laws for motorcycles previously. As cycling became…
View article: Acknowledgments
Acknowledgments Open
The journey we have taken over the past decade writing about the lead wars has given us the opportunity to meet and work with an extraordinary group of dedicated people.We have gotten to know and to learn from public health scientists who,…
View article: Addressing the dichotomy between individual and societal approaches to personalised medicine in oncology
Addressing the dichotomy between individual and societal approaches to personalised medicine in oncology Open
View article: Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials
Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials Open
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 investigationa…
View article: “Alien” Health Care
“Alien” Health Care Open
The article discusses U.S. government policies regarding access to health care for migrants (illegal immigrants) as of 2017, and it provides a response to the article Effect of U.S. Health Policies on Health Care Access for Marshallese Mig…
View article: Corrigendum: Free to Consume? Anti-Paternalism and the Politics of New York City's Soda Cap Saga
Corrigendum: Free to Consume? Anti-Paternalism and the Politics of New York City's Soda Cap Saga Open
View article: Even without written codes, ethical standards for human research existed before World War II
Even without written codes, ethical standards for human research existed before World War II Open