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The American Journal of Bioethics Volume 20, 2020 - Issue 4
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CASE REPORT

Ethical Drug Development for Rare Childhood Diseases: When There Are Limited But Promising Data in Adults, How to Choose Between Safety or Efficacy Studies?

Liza-Marie JohnsonSt. Jude Children's Research Hospital; ORCID Iconhttp://orcid.org/0000-0002-7124-979XView further author information
ORCID Icon,
Devan M. DuenasSeattle Children's Research Institute; Correspondence[email protected]
ORCID Iconhttp://orcid.org/0000-0003-1630-1417View further author information
ORCID Icon &
Benjamin S. WilfondSeattle Children's Research Institute; ;University of Washington School of MedicineView further author information
Pages 111-113 | Published online: 24 Mar 2020

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Read on this site (3)

Devan M. Duenas, Benjamin S. Wilfond & Liza-Marie Johnson. (2020) Clearing Muddy Waters: The Need to Reconceptualize Minor Increase over Minimal Risk in Pediatric Rare Disease Research. The American Journal of Bioethics 20:4, pages 8-10.
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Kathryn M. Porter, Anne Stevens & Benjamin S. Wilfond. (2020) Flexibility Required: Balancing the Interests of Children and Risk in Drug Development for Rare Pediatric Conditions. The American Journal of Bioethics 20:4, pages 116-118.
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Robert M. Nelson. (2020) The Use of Pediatric Extrapolation to Avoid Unnecessary Pediatric Clinical Trials. The American Journal of Bioethics 20:4, pages 114-116.
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Articles from other publishers (1)

Debbie S. Gipson, Joshua Tarnoff, Lauren Lee, Marina Vivarelli, Elena Levtchenko, Jun Oh, William E. Smoyer, Hailey Desmond, Samara Attalla & Howard Trachtman. (2021) A pediatric gateway initiative for glomerular disease: introducing PIONEER. Kidney International 99:3, pages 515-518.
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