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Accelerating Pediatric Cancer Drug Development: Challenges and Opportunities for Pediatric Master Protocols

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Abstract

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.

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Authors and Affiliations

  1. Genentech Inc, a member of the Roche Group, South San Francisco, CA, USA

    Tahira Khan PhD & Raphaël Rousseau MD, PhD

  2. Friends of Cancer Research, 1800 M St NW Ste. 1050s, Washington, DC, 20036, USA

    Mark Stewart PhD (Senior Science Policy Analyst)

  3. Silverback Therapeutics, Seattle, WA, USA

    Samuel Blackman MD

  4. Food and Drug Administration, Silver Spring, MD, USA

    Martha Donoghue MD & Gregory Reaman MD

  5. Department of Pediatrics and Oncology, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, MD, USA

    Kenneth Cohen MD, MBA

  6. USA National Cancer Institute, Bethesda, MD, USA

    Nita Seibel MD

  7. American Cancer Society Cancer Action Network Inc, Washington, DC, USA

    Mark Fleury PhD

  8. Celgene Corporation, Summit, NJ, USA

    Bouchra Benettaib MD

  9. Drug Information Association, Washington, DC, USA

    Raleigh Malik PhD

  10. Department of Clinical Research, Institut Gustave Roussy, Paris-Sud University, Paris, France

    Gilles Vassal MD, PhD

Authors
  1. Tahira Khan PhD
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  2. Mark Stewart PhD
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  3. Samuel Blackman MD
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  4. Raphaël Rousseau MD, PhD
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  5. Martha Donoghue MD
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  6. Kenneth Cohen MD, MBA
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  7. Nita Seibel MD
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  8. Mark Fleury PhD
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  9. Bouchra Benettaib MD
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  10. Raleigh Malik PhD
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  11. Gilles Vassal MD, PhD
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  12. Gregory Reaman MD
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Corresponding author

Correspondence to Mark Stewart PhD.

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Khan, T., Stewart, M., Blackman, S. et al. Accelerating Pediatric Cancer Drug Development: Challenges and Opportunities for Pediatric Master Protocols. Ther Innov Regul Sci 53, 270–278 (2019). https://doi.org/10.1177/2168479018774533

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  • DOI: https://doi.org/10.1177/2168479018774533

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