The convergence of policy reform, the evolution of highly targeted therapies, and innovations in regulatory and development science has created the potential for a new era in pediatric drug development. Policy reform in the US and EU has led drug developers to gain extensive pediatric drug development experience over the last two decades. In parallel, the development of pharmaceutical pipelines to include highly targeted therapies that account for genetic variability to treat or prevent disease has led to exciting progress in the development of new therapeutic targets (such as CFTR modulators for cystic fibrosis) to address rare pediatric disease.
Development of highly targeted therapies for small research populations has led to unimaginable innovation in regulatory and development science and revolutionized how treatments for the most complex diseases can be developed. Tools to facilitate the evaluation of cutting-edge therapeutic discoveries are also evolving rapidly through the efforts of regulatory agencies. Leveraging pre-existing data for modeling and simulation, inclusion of pediatric patients within adult confirmatory studies when dosing is similar between adult and pediatric populations, or utilizing molecular targeting to allow tissue agnostic development approaches are just a few examples of pragmatic solutions that can address the complexities of pediatric drug development.
The DIA Pediatric Drug Development Workshop will bring together the pediatric research community (drug developers, regulators, and academic researchers) to evaluate and discuss how such solutions can be applied to advance the development of biopharmaceutical therapies for pediatric patients.
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Who should attend?
Professionals involved in:
- Regulatory, clinical, and drug development professionals from health authorities and within industry
- Employees from clinical research organizations (CRO) and individuals involved in pediatric clinical trials
- Representatives from academia, pediatric societies, and networks
- Parents and patient advocacy organizers
- Any stakeholder interested in the development of better pediatric research programs
- Describe how novel technologies such as gene therapy are being applied to the development of therapies for serious pediatric illnesses
- Explain how innovative uses of methods such as extrapolation and modeling and simulation can leverage existing adult data and decrease the need for pediatric data to facilitate the study of therapies in pediatric populations
- Discuss how the latest generation of clinical development plans, clinical trial designs, and organizational structures can enable accelerated development and approval of therapeutics for children
- Describe how companies make decisions regarding inclusion of pediatric development programs within their portfolios and the unique and evolving roles of large pharmaceutical, venture capital, and academic institutions in the advancement of new medications for children
Christina Bucci-Rechtweg, MD • Global Head, Maternal Health and Pediatric Regulatory Policy
Novartis , United States
Samuel C. Blackman, MD, PhD • Head of Clinical Development
Mavupharma, United States
Edward Connor, MD, MS • Professor Emeritus Pediatrics, Microbiology, Immunology, Tropical Medicine
George Washington University School of Medicine and Health Sciences, United States
Thomas Miller, PhD • Vice President and Global Head, Pediatrics
Bayer, United States
Andrew Mulberg, MD • Senior Vice President
Neurogene, United States
Yeruk (Lily) Mulugeta, PharmD • Associate Director, Division of Pediatric and Maternal Health, CDER
FDA , United States
Robert "Skip" Nelson, MD, PhD • Senior Director, Pediatric Drug Development (CHILD)
Johnson & Johnson, United States
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Industry Perspective: Master Protocols as an Option to Design & Conduct Pediatric Oncology Studies under FDARA
Check out our 2019 Program!