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Global Regulatory and Public Health Initiatives to Advance Pediatric Drug Development for Rare Diseases.
Epps, Carla; Bax, Ralph; Croker, Alysha; Green, Dionna; Gropman, Andrea; Klein, Agnes V; Landry, Hannah; Pariser, Anne; Rosenman, Marc; Sakiyama, Michiyo; Sato, Junko; Sen, Kuntal; Stone, Monique; Takeuchi, Fumi; Davis, Jonathan M.
  • Epps C; Office of Pediatric Therapeutics, Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD, 20993, USA. carla.epps@fda.hhs.gov.
  • Bax R; Paediatric Medicines, Scientific Evidence Generation Department, European Medicines Agency, Amsterdam, The Netherlands.
  • Croker A; Office of Paediatrics and Patient Involvement, Health Products and Food Branch and Director General's Office, Biologic and Radiopharmaceutical Drugs Directorate Health Canada, Ottawa, ON, Canada.
  • Green D; Office of Pediatric Therapeutics, Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD, 20993, USA.
  • Gropman A; Neurodevelopmental Disabilities and Neurogenetics, Children's National Medical Center, Washington, DC, USA.
  • Klein AV; Office of Paediatrics and Patient Involvement, Health Products and Food Branch and Director General's Office, Biologic and Radiopharmaceutical Drugs Directorate Health Canada, Ottawa, ON, Canada.
  • Landry H; Office of Paediatrics and Patient Involvement, Health Products and Food Branch and Director General's Office, Biologic and Radiopharmaceutical Drugs Directorate Health Canada, Ottawa, ON, Canada.
  • Pariser A; Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD, USA.
  • Rosenman M; Mary Ann & J. Milburn Smith Child Health Outcomes, Research, and Evaluation Center, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.
  • Sakiyama M; Pediatric Drugs Working Group, Pharmaceuticals and Medical Devices Agency, Tokyo, Japan.
  • Sato J; Pediatric Drugs Working Group, Pharmaceuticals and Medical Devices Agency, Tokyo, Japan.
  • Sen K; Neurodevelopmental Disabilities and Neurogenetics, Children's National Medical Center, Washington, DC, USA.
  • Stone M; Advanced Therapies Unit, Prescription Medicines Authorisation Branch, Therapeutic Goods Administration, Department of Health, Woden, ACT, 2606, Australia.
  • Takeuchi F; Pediatric Drugs Working Group, Pharmaceuticals and Medical Devices Agency, Tokyo, Japan.
  • Davis JM; Department of Pediatrics, Tufts Medical Center and the Tufts Clinical and Translational Science Institute, Boston, MA, USA.
Ther Innov Regul Sci ; 56(6): 964-975, 2022 11.
Article in English | MEDLINE | ID: covidwho-1803265
ABSTRACT
The literature thoroughly describes the challenges of pediatric drug development for rare diseases. This includes (1) generating interest from sponsors, (2) small numbers of children affected by a particular disease, (3) difficulties with study design, (4) lack of definitive outcome measures and assessment tools, (5) the need for additional safeguards for children as a vulnerable population, and (6) logistical hurdles to completing trials, especially with the need for longer term follow-up to establish safety and efficacy. There has also been an increasing awareness of the need to engage patients and their families in drug development processes and to address inequities in access to pediatric clinical trials. The year 2020 ushered in yet another challenge-the COVID-19 pandemic. The pediatric drug development ecosystem continues to evolve to meet these challenges. This article will focus on several key factors including recent regulatory approaches and public health policies to facilitate pediatric rare disease drug development, emerging trends in product development (biologics, molecularly targeted therapies), innovations in trial design/endpoints and data collection, and current efforts to increase patient engagement and promote equity. Finally, lessons learned from COVID-19 about building adaptable pediatric rare disease drug development processes will be discussed.
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Full text: Available Collection: International databases Database: MEDLINE Main subject: Biological Products / COVID-19 Drug Treatment Type of study: Cohort study / Prognostic study / Randomized controlled trials Limits: Child / Humans Language: English Journal: Ther Innov Regul Sci Year: 2022 Document Type: Article Affiliation country: S43441-022-00409-w

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Full text: Available Collection: International databases Database: MEDLINE Main subject: Biological Products / COVID-19 Drug Treatment Type of study: Cohort study / Prognostic study / Randomized controlled trials Limits: Child / Humans Language: English Journal: Ther Innov Regul Sci Year: 2022 Document Type: Article Affiliation country: S43441-022-00409-w