Barriers to access of precision guided therapies for children with high-risk cancer.
compassionate use
novel agents
paediatric oncology
precision medicine
therapeutic drugs
Journal
Pediatric blood & cancer
ISSN: 1545-5017
Titre abrégé: Pediatr Blood Cancer
Pays: United States
ID NLM: 101186624
Informations de publication
Date de publication:
26 Aug 2024
26 Aug 2024
Historique:
revised:
06
05
2024
received:
02
01
2024
accepted:
31
05
2024
medline:
26
8
2024
pubmed:
26
8
2024
entrez:
26
8
2024
Statut:
aheadofprint
Résumé
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.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e31147Subventions
Organisme : Cancer Institute NSW
Organisme : Translational Program
ID : 2021/TPG2112
Organisme : Zero Childhood Cancer National Personalised Medicine Program
Organisme : NHMRC of Australia
ID : APP2008300
Organisme : Kids Cancer Foundation
Organisme : Luminesce Alliance-Innovation for Children's Health
Informations de copyright
© 2024 The Author(s). Pediatric Blood & Cancer published by Wiley Periodicals LLC.
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