Lecturers:
Marc Van Dijk (UCB Pharma)
Luis Pinheiro (European Medicines Agency)
During this webinar speakers, panelists and participants discussed:
- How to plan for the data needs for the future of medicine development for rare diseases.
- How to anticipate the scope, depth, and quality of data that will be required to generate reliable evidence suitable for regulatory use cases.
- The tools that are available to make data collection accessible for these uses.
Video timestamps:
- Keynote presentations
00:00:01 Marc Van Dijk (UCB): Data and technologies that offer solutions for challenges in rare diseases
00:22:15 Luis Pinheiro (EMA): From Data to Decision
- Panel discussion (highlights on some questions discussed)
(Jose/patient, Cécile/regulatory science, Meelis/tech developer, Dinko & Tom/drug developers, Luis/regulator)
00:48:05 Points to consider when using new technologies to generate robust data
01:01:27 Solutions to develop better data (technologies and examples)
01:03:30 Synthetic data (part 1: context of use, value)
01:06:40 AI doctor (scientific and regulatory validation)
01:07:22 Synthetic data (part 2: regulator’s perspective)
01:08:27 AI doctor (acceptability of technologies, patient perspective)
01:10:35 Conventional methods vs. New technologies and methods
01:12:02 Data protection aspects when generating regulatory evidence
01:13:36 Pre-competitive collaboration to de-risk and accelerate progress
01:15:55 Evolution of technology and its limitation
01:18:12 Blockchain in drug development
01:19:20 Key principles/imperatives from understanding the disease up to regulatory context
01:28:45 Interplay between medicines and medtech and AI regulations
01:31:50 Patient perspective on digital health technologies
01:33:05 Take home message – recommendations from the panel
