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Program Training School 6

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​Regulatory aspects of personalized medicine development:
Global harmonization, advanced therapies & patient relevant outcomes

Date​ Time   Program/event
March 22, 2021​Global Medicines Regulation – the basics of ICH
​13:00 -13:30
​Drug development: Global alignment
ICH guidelines
Advanced therapies drug development
​13:30 - 14:30​Haemophilia drug development
  • Short overview of new & recent developments
Patient relevant outcomes & patient reflections
14:30 - 15:00Break
​15:00 - 16:00Non clinical developments – too advanced for the animal model
  • Organoid models in CF / T1DM
Regulatory reflection Safety pharmacology (ICH S-guidelines)
​ ​Introduction to case studies
​16:00-17:00​Teplizumab for prevention of T1DM
  • The study results (NEJM 2019; 381:603-613)**
  • Teplizumab as a Priority Medicine (PRIME)
Introduction to the case studies
​March 23, 2021
​ ​
​9:00 – 10:30​Developing Advanced Therapeutic Medicinal Products (ATMP’s)
  • CAR-t – glioblastoma
  • ATMP approval (the case of Jescarta) – CAT/CHMP
  • Discussion of a dossier
​10:30 - 11:00
​Break
​ ​​11:00 - 18:00​Case Study (three groups)
Design a drug development program for a targeted therapy in TxDM:
a) prevention of T1DM,
b) focus on symptomatic benefit (less hypo’s),
c) slowing renal decline
​19:00 - 20:30​Social event (virtual)
​March 24, 2021​10:00 - 12:00​Case presentation & discussion
​June 7, June 9 & June 11, 20219:00 – 12:30​​Entrepreneurship & Valorization