
Dates: October 29 – 30, 2024; 8:00 AM – 4:00 PM
We are delighted that you will be attending the MRCT Center’s invitation-only two-day workshop, Advancing Pediatric Platform Trials: Streamlining Development, Maximizing Impact, on October 29-30, 2024, at The Ven at Embassy Row, Washington, DC. You will be part of a diverse group of multi-stakeholder participants, each with deep expertise in critical areas related to the design and conduct of pediatric platform trials.
In addition to keynote addresses and panel discussions, three disease areas have been selected to address various challenges and opportunities in pediatric drug development: major depressive disorder, multi-drug-resistant tuberculosis, and pediatric hematological malignancies.
This solution-focused workshop aims to reinforce the strengths of platform trials by advancing the principles, ethical foundations, and operational considerations for conducting these trials in registration studies of pediatric investigational products. The goal is to promote efficiencies in FDA and EMA approval processes, including labeling. The workshop will include carefully structured small and large group discussions, exploring opportunities such as sharing control data, incorporating patient-centric approaches in the design phase, reducing participant enrollment numbers, and streamlining trial conduct.
Additional Workshop Objectives:
- Understand how study design parameters—such as eligibility criteria, outcome measures, endpoints, data and information sharing, and the handling of company-confidential information—are managed and could be optimized.
- Identify and develop approaches to address statistical, regulatory, and operational challenges associated with multi-sponsor pediatric platform trials.
- Explore regulatory perspectives on the feasibility and acceptability of multi-sponsor pediatric platform trials.
Johnson & Johnson, AstraZeneca, and Sanofi have provided sponsorship grants for this independent program. Sanofi is part of the Steering Committee of the MRCT Center. Johnson & Johnson and AstraZeneca are part of the Executive Committee.
Post-workshop materials
Agenda
Venue and Travel
Book using your group rate for MRCT Center Pediatric Platform Block
- The Ven at Embassy Row, Washington DC, A Tribute Portfolio Hotel
2015 Massachusetts Avenue NW, Washington, DC 20036
Virtual Attendees
Workshop materials and pre-reads
Platform Trials
MDR-TB
- FDA DRAFT Guidance: Pulmonary Tuberculosis: Developing Drugs for Treatment
- Nachman: Towards earlier inclusion of Children in Tuberculosis (TB) drug trials
- Master Protocols for Drug and Biological Product Development: Guidance for Industry
- ICH E11A: Guideline on pediatric extrapolation
- EMA DRAFT: Addendum to the ‘guideline on the evaluation of medicinal products indicated for the treatment of bacterial infections’
Hematologic Oncology
- A Potential Paradigm for the Robust and Systematic Priorisation of Assets in Academic-Led, Multi-Industry Collaborative Trials in Rare Populations (Glo-BNHL)
- EMA: Letter of Support for the Global Platform Study of Novel Medicines in Paediatric and Adolescent Relapsed and Refractory B-cell Non-Hodgkin Lymphoma
- A Global Study of Novel Agents in Paediatric and Adolescent Relapsed and Refractory B-cell Non-Hodgkin Lymphoma (Glo-BNHL)
MDD
MDD: Why Pediatric Platform Trials
- Penkov: Pediatric Medicine Development: An Overview and Comparison of Regulatory Processes in the EU and US
- Nelson: The Role of Master Protocols in Pediatric Drug Development
- Miller and Campo: Depression in Adolescents
- Meyer: The Evolution of the Master Protocol CIinical Trial Designs: A Review
- Li: Unlocking the Potential: A Systematic Review of Master Protocol in Pediatrics
- FDA: Major Depressive Disorder – Developing Drugs for Treatment – Guidance for Industry
- Fava: How Should we design future mechanistic and/or efficacy clinical trials?
- EMA: Draft Guidline of clinical trial investigation of medicinal products in the treatment of depression
- Carmack: Pediatric Drug Policies Supporting Safe and Effective Use of Therapeutics in Children
- Butlen-Ducuing et al: The New EMA Guideline on Antidepressants: A Guide for Researchers and Drug Developers
MDD: Pediatric Platform Design
- Gupta: Annual Research Review: Puberty and development of anhedonia
- Cuijpers: The effects of psychological treatments of depression in children and adolescents on response, reliable change and deterioration
- Dhaenens: Optimizing expert and patient input in pediatric trial design
- Drevets: Immune targets for therapeutic development in depression
- FDA: Master Protocols for Drug and Biological Product Development
- Feeney: Antidepressants in children and adolescents with major depressive disorder and the influence of placebo response.
- Findling: The Relevance of Pharmacokinetic Studies
- Gamalo: Extrapolation as a Default Strategy
- Hiemke: Consensus Guidelines for Therapeutic Drug Monitoring in Neuropsychopharmacology
- ICH E11A: Pediatric Extrapolation
- Jones: Precision Psychiatry: Biomarker-Guided Tailored Therapy
- Kim: Symptom clusters in adolescent depression and differential responses
- Koenig: Current state-of-the-art and gaps in platform trials
- Lee: Molecular Biomarkers for Pediatric Depressive Disorders
- Li: Impact of Placebo Response Rates on Clinical Trial Outcomes
- Meister: Placebo response rates and potential modifiers in double-blind trials
- Mossman: Impact of Failed Antidepressant Trials
- Namerow: Editorial: Beyond Red Light, Green Light
- Nguyen: Regulatory Issues of Platform Trials
- Okubo: Factors Related to Placebo Response
- Pergolizzi: Pirates of Clinical Trials
- Perna: Biomarker-Guided Tailored Therapy
- Radecki: Safety and Efficacy of Levomilnacipran Extended Release
- Rice: Adolescent and adult differences
- Scott: Association of Single-Blind Placebo Run-In Periods
- Shibata: Population pharmacokinetics in duloxetine
- Stone: Response to acute monotherapy
- Strawn: Impact of age on antidepressant response
- Strawn: Pediatric Therapeutic Drug Monitoring
- Suseelan: Heterogeneity in major depressive disorder
- Tini: Therapeutic drug monitoring of sertraline in children and adolescents
- Walkup: Antidepressant Efficacy for Depression in Children and Adolescents
- Wasan: Drug development process and COVID-19 pandemic
- Winter: Systematic Evaluation of Machine-Learning-Based Biomarkers
- Wu: An electroencephalograpic signature
- Yang: Changes of tPA/PAI-1 system
- Zwolinska: Biomarkers of Child and Adolescent Depression