{"title":"Ethics of gene and cell therapy development for neurologic disorders.","authors":"Jonathan Kimmelman","doi":"10.1016/B978-0-323-90120-8.00002-2","DOIUrl":null,"url":null,"abstract":"<p><p>In this chapter, I provide a condensed overview of nine recurring policy and ethical challenges encountered with the development of gene and cell therapies for neurologic disease. These include the question of when to initiate first-in-human trials, the ethics and policy of expanded/special access, the conduct of individualized therapy trials, subject selection in trials, designing trials for negative results, unintended effects of interventions on personal identity, comparator choice in randomized trials, consent and therapeutic misestimation, and cost and access for effective therapies. Broadly speaking, I argue that early in their development, the justification of risk in trials of gene and cell therapies derives from the social and scientific value of a trial and not the therapeutic value for trial participation. This generates strong imperatives to justify, design, and report trials appropriately and select patient populations that incur the least burden and opportunity cost for trial participation. Late in intervention development, policy makers must contend with the fact that proven effective interventions will almost certainly amplify strains in healthcare budgets as well as the ethical justifications standing behind reimbursement decisions.</p>","PeriodicalId":12907,"journal":{"name":"Handbook of clinical neurology","volume":"205 ","pages":"111-121"},"PeriodicalIF":0.0000,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Handbook of clinical neurology","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1016/B978-0-323-90120-8.00002-2","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"Medicine","Score":null,"Total":0}
引用次数: 0
Abstract
In this chapter, I provide a condensed overview of nine recurring policy and ethical challenges encountered with the development of gene and cell therapies for neurologic disease. These include the question of when to initiate first-in-human trials, the ethics and policy of expanded/special access, the conduct of individualized therapy trials, subject selection in trials, designing trials for negative results, unintended effects of interventions on personal identity, comparator choice in randomized trials, consent and therapeutic misestimation, and cost and access for effective therapies. Broadly speaking, I argue that early in their development, the justification of risk in trials of gene and cell therapies derives from the social and scientific value of a trial and not the therapeutic value for trial participation. This generates strong imperatives to justify, design, and report trials appropriately and select patient populations that incur the least burden and opportunity cost for trial participation. Late in intervention development, policy makers must contend with the fact that proven effective interventions will almost certainly amplify strains in healthcare budgets as well as the ethical justifications standing behind reimbursement decisions.
期刊介绍：
The Handbook of Clinical Neurology (HCN) was originally conceived and edited by Pierre Vinken and George Bruyn as a prestigious, multivolume reference work that would cover all the disorders encountered by clinicians and researchers engaged in neurology and allied fields. The first series of the Handbook (Volumes 1-44) was published between 1968 and 1982 and was followed by a second series (Volumes 45-78), guided by the same editors, which concluded in 2002. By that time, the Handbook had come to represent one of the largest scientific works ever published. In 2002, Professors Michael J. Aminoff, François Boller, and Dick F. Swaab took on the responsibility of supervising the third (current) series, the first volumes of which published in 2003. They have designed this series to encompass both clinical neurology and also the basic and clinical neurosciences that are its underpinning. Given the enormity and complexity of the accumulating literature, it is almost impossible to keep abreast of developments in the field, thus providing the raison d''être for the series. The series will thus appeal to clinicians and investigators alike, providing to each an added dimension. Now, more than 140 volumes after it began, the Handbook of Clinical Neurology series has an unparalleled reputation for providing the latest information on fundamental research on the operation of the nervous system in health and disease, comprehensive clinical information on neurological and related disorders, and up-to-date treatment protocols.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
