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2023 | March / April 2023

Next-generation approaches to statistical significance

by cyb2025
Biopharma Excellence
alternative therapies
biopharmaceutical
cell and gene therapy
clinical research
Clinical trials
MHRA
rare diseases

Biopharma Excellence
Denmark

ABSTRACT

Biopharma Excellence recently hosted a panel discussion featuring experts in biotech and the evolving regulatory environment to discuss the challenge of achieving statistical significance with novel and highly targeted therapies.

Daniel O’Connor of the UK’s MHRA, with a remit of Innovation Acceleration within a regulatory context, noted that the challenge of achieving statistical significance when target populations are tiny is nothing new, but conceded that randomised trials aren’t always viable – especially with very rare conditions. He urged drug developers to seek scientific advice before finding workarounds.

WHEN TRIAL DATA IS LACKING
The panel considered the potential for supplementary data sets – such as pharmacodynamic read-outs, histological evidence and historical controls – in enabling regulators to reach robust decisions.

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