King N M P, Bishop C E
Department of Social Sciences and Health Policy, Wake Forest School of Medicine, Center for Bioethics Health, and Society and Graduate Program in Bioethics, Wake Forest University, Winston-Salem, NC, USA.
Department of Pediatrics, Wake Forest School of Medicine, Winston-Salem, NC, USA.
Gene Ther. 2017 Sep;24(9):534-538. doi: 10.1038/gt.2017.32. Epub 2017 May 3.
Approval of Spinraza (nusinersen) for treatment of spinal muscular atrophy prompts consideration of a number of ethical issues that arise whenever a new treatment is proposed for a serious condition, especially one that is rare and can devastatingly affect children. Patients, families, clinicians, researchers, institutions and policymakers all must take account of the ways that newly available treatments affect informed and shared decision-making about therapeutic and research options. The issues to consider include: addressing what is still uncertain and unknown; the possibility that potential benefits will be exaggerated and potential harms underemphasized in the media, by advocacy organizations, and in consent forms and processes; the high cost of many novel drugs and biologics; the effects of including conditions of variable phenotype in state-mandated newborn screening panels; and how new treatments can change the standard of care, altering what is and is not known about a disorder and posing challenges for decision-making at both individual and policy levels. The good news that Spinraza brings thus requires additional attention to its ethical and policy implications, to improve counseling and shared decision-making about treatment and research options for patients and all involved in their care.
Spinraza（诺西那生钠）获批用于治疗脊髓性肌萎缩症，这引发了人们对一系列伦理问题的思考，每当针对一种严重疾病（尤其是罕见且会对儿童造成毁灭性影响的疾病）提出新的治疗方法时，这些伦理问题就会出现。患者、家庭、临床医生、研究人员、机构和政策制定者都必须考虑新出现的治疗方法如何影响有关治疗和研究选择的知情和共同决策。需要考虑的问题包括：解决仍不确定和未知的问题；在媒体、倡导组织以及同意书和同意过程中，潜在益处可能被夸大而潜在危害可能被低估的可能性；许多新型药物和生物制品的高昂成本；将具有可变表型的病症纳入国家规定的新生儿筛查项目的影响；以及新治疗方法如何改变护理标准，改变对一种疾病已知和未知的情况，并在个人和政策层面给决策带来挑战。因此，Spinraza带来的好消息需要额外关注其伦理和政策影响，以改善对患者以及所有参与其护理的人员在治疗和研究选择方面的咨询和共同决策。
