School of Population Health, Faculty of Medicine and Health, UNSW Sydney, Sydney, New South Wales, Australia.
Discipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Medicine & Health, UNSW Sydney, Sydney, New South Wales, Australia.
Pediatr Blood Cancer. 2024 Nov;71(11):e31147. doi: 10.1002/pbc.31147. Epub 2024 Aug 26.
Accessing compassionate access schemes to obtain novel therapeutic agents for children with hard-to-treat cancers can be fraught with challenges such as regulatory barriers and limited resources. This study aimed to explore clinician perspectives on the barriers, impacts and ethical considerations of accessing novel therapeutic agents within the context of a paediatric oncology precision medicine trial.
We gathered data from 37 semi-structured interviews with paediatric oncologists participating in the PRecISion Medicine for Children with Cancer (PRISM) study, a precision medicine clinical trial in Australia. The interviews, conducted over 2 years, focused on paediatric oncologist's experiences with the PRISM trial. Interviews were re-analysed to identify themes related to access pathways and any challenges in obtaining novel agents through thematic analysis. The resulting thematic framework was discussed and refined by a multidisciplinary team.
Three main themes were identified: (i) barriers to access, including poor drug availability, lack of evidence and the time burden of the application process; (ii) impacts of inaccessibility, encompassing medical consequences and financial burden on families; and (iii) ethical considerations, centred around balancing realistic expectations and providing compassionate care to patients and families. Paediatric oncologists expressed frustration with the complex regulatory landscape and the lack of systematic reporting on applications and outcomes of obtaining novel agents. Lengthy wait times for decision notifications were also highlighted, raising concerns about missed therapeutic opportunities for patients.
This study provides insight to the challenges faced when seeking access to novel therapies for paediatric oncology patients. There is a clear need for improved communication, streamlining processes and increased resources to facilitate access to novel agents. Further resource development is necessary to address these complexities in accessing novel therapy agents to ultimately ensure equitable and timely access.
为难以治疗的癌症儿童获得新型治疗药物，通过有同情心的途径获取可能充满挑战，如监管障碍和有限的资源。本研究旨在探讨临床医生在儿科肿瘤精准医学试验背景下获取新型治疗药物的障碍、影响和伦理考虑。
我们从参与澳大利亚 PRISM 研究（儿童癌症精准医学试验）的 37 名儿科肿瘤学家的 37 次半结构化访谈中收集了数据。这些访谈在 2 年的时间里进行，重点关注儿科肿瘤学家参与 PRISM 试验的经验。通过主题分析，对访谈进行重新分析，以确定与获取途径相关的主题和任何获取新型药物的挑战。由此产生的主题框架由多学科团队进行讨论和完善。
确定了三个主要主题：（i）获取途径的障碍，包括药物供应不足、缺乏证据和申请过程的时间负担；（ii）无法获得药物的影响，包括对家庭的医疗后果和经济负担；（iii）伦理考虑，围绕平衡现实期望以及为患者和家庭提供同情关怀。儿科肿瘤学家对复杂的监管环境以及缺乏关于新型药物获取申请和结果的系统报告表示不满。决策通知的漫长等待时间也引起了人们的关注，这使得患者错失了治疗机会。
本研究提供了在为儿科肿瘤患者寻求新型治疗方法时面临的挑战的深入了解。显然需要改进沟通、简化流程和增加资源，以促进新型药物的获取。需要进一步开发资源，以解决获取新型治疗药物的复杂性，最终确保公平和及时的获取。
