Drugs

Traditional trial designs and regulatory processes face hurdles in keeping up with the pace of scientific discovery, artificial intelligence, big data and real-world data

Photo from patvocates.net

Jun 19, 2023, 20:15

Traditional trial designs and regulatory processes face hurdles in keeping up with the pace of scientific discovery, artificial intelligence, big data and real-world data

In a post by Jan Geissler on linkedin, it says, “I am attending the annual congress of the European Hematology Association (EHA) in Frankfurt. Today I chaired a session in the EHA-Patient Joint Symposium where we discussed with representatives from the French HTA agency HAS, the European Medicines Agency, pharma industry and clinicians about “novel clinical trials – the comparator challenge and the need for new formats”.
As the medical landscape evolves rapidly, traditional trial designs and regulatory processes face hurdles in keeping up with the pace of scientific discovery, artificial intelligence, big data and real-world data. Using existing data from earlier trials as “historical control arms” instead of randomizing masses of patients to control arms with therapies we should know well and used to love raises justified questions on robustness of the evidence, with many calling for more regulatory guidance to reduce uncertainty. However even if we wanted them, these historical control arms of studies are often wishful thinking, as due to competitive fears and academic career models, industry and academia sit on their data like hens on their own eggs, while patients on studies have shared their data for collective breeding and competition for the best conclusions.
This symposium served as a platform to address these challenge head-on, exploring how innovative study designs could generate robust data that is being shared widely and supports further research and robust access decisions. We need more work on pioneering novel clinical trials, breaking down barriers, and de-risking bold research.
Time was up when we were getting more beef to the bone how more collaborative models could be established — so we have a good basis to dig deeper next time.

Great to have such a great panel at EHA2023. A multidisciplinary approach needs to tackle complex issues, and I feel the patient community could have such a role in facilitating and driving such a dialogue.”

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