TY - JOUR
T1 - Accelerating Pediatric Cancer Drug Development
T2 - Challenges and Opportunities for Pediatric Master Protocols
AU - Khan, Tahira
AU - Stewart, Mark
AU - Blackman, Samuel
AU - Rousseau, Raphaël
AU - Donoghue, Martha
AU - Cohen, Kenneth
AU - Seibel, Nita
AU - Fleury, Mark
AU - Benettaib, Bouchra
AU - Malik, Raleigh
AU - Vassal, Gilles
AU - Reaman, Gregory
N1 - Publisher Copyright:
© The Author(s) 2018.
PY - 2019/3/1
Y1 - 2019/3/1
N2 - Although outcomes for children with cancer have significantly improved over the past 40 years, there has been little progress in the treatment of some pediatric cancers, particularly when advanced. Additionally, clinical trial options and availability are often insufficient. Improved genomic and immunologic understanding of pediatric cancers, combined with innovative clinical trial designs, may provide an enhanced opportunity to study childhood cancers. Master protocols, which incorporate the use of precision medicine approaches, coupled with the ability to quickly assess the safety and effectiveness of new therapies, have the potential to accelerate early-phase clinical testing of novel therapeutics and which may result in more rapid approval of new drugs for children with cancer. Designing and conducting master protocols for children requires addressing similar principles and requirements as traditional adult oncology trials, but there are also unique considerations for master protocols conducted in children with cancer. The purpose of this paper is to define the key challenges and opportunities associated with this approach in order to ensure that master protocols can be adapted to benefit children and adolescents and ensure that adequate data are captured to advance, in parallel, the clinical development of investigational agents for children with cancer.
AB - Although outcomes for children with cancer have significantly improved over the past 40 years, there has been little progress in the treatment of some pediatric cancers, particularly when advanced. Additionally, clinical trial options and availability are often insufficient. Improved genomic and immunologic understanding of pediatric cancers, combined with innovative clinical trial designs, may provide an enhanced opportunity to study childhood cancers. Master protocols, which incorporate the use of precision medicine approaches, coupled with the ability to quickly assess the safety and effectiveness of new therapies, have the potential to accelerate early-phase clinical testing of novel therapeutics and which may result in more rapid approval of new drugs for children with cancer. Designing and conducting master protocols for children requires addressing similar principles and requirements as traditional adult oncology trials, but there are also unique considerations for master protocols conducted in children with cancer. The purpose of this paper is to define the key challenges and opportunities associated with this approach in order to ensure that master protocols can be adapted to benefit children and adolescents and ensure that adequate data are captured to advance, in parallel, the clinical development of investigational agents for children with cancer.
KW - clinical trial design
KW - drug development
KW - master protocol
KW - pediatric cancer
KW - regulatory policy
UR - http://www.scopus.com/inward/record.url?scp=85047406254&partnerID=8YFLogxK
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U2 - 10.1177/2168479018774533
DO - 10.1177/2168479018774533
M3 - Article
C2 - 29759018
AN - SCOPUS:85047406254
SN - 2168-4790
VL - 53
SP - 270
EP - 278
JO - Therapeutic Innovation and Regulatory Science
JF - Therapeutic Innovation and Regulatory Science
IS - 2
ER -