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In the context of ERS Congress 2023, Dr Mayer-Hamblett from Seattle Children’s Hospital focused her presentation on the changing landscape in trial design for new CF therapies.
How to deal with the new paradigm after Kaftrio?
- It is time to consider a global partnership to ensure the continued therapeutic development of modulators and a reasonable sample size.
- A continued willingness to participate is necessary.
- More sensitive endpoints need to be established, with a need to rationalise their clinical significance.
- Look for opportunities to combine with other disease groups.
- To consider the advancement of multiple therapeutic programmes in people who do not have modulators.
- Inclusion of historically under-represented populations in research.
- Create global prioritisation strategies and innovative trial designs.
#TogetheragainstCF
