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Sign in to saveThe EMA has introduced new clinical requirements for the development of thalassaemia treatments, emphasizing the design of confirmatory phase 3 trials. Stakeholders have until January 31, 2027, to provide comments on the draft guidelines, which began public consultation on September 24, 2026.
The updated guidelines are critical for regulatory compliance in the development of thalassaemia therapies, impacting the design and execution of clinical trials and thus affecting market access and patient treatment options.
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PV teams should update workflows and SOPs to include the new section 19A approval processes and ensure compliance with the TGA's guidelines for managing medicine shortages.
PV teams should update their workflows regarding inspection readiness, review standard operating procedures (SOPs) to align with the new GCP inspection coordination processes, and ensure that all relevant personnel are trained on the updated guidance.
PV teams should review and update their Standard Operating Procedures (SOPs) related to real-world data collection methodologies and ensure alignment with the new guidance emerging from the workshop. Training may also be required to enhance understanding of RWD's impact on regulatory submissions.
Update workflows related to regulatory intelligence monitoring and ensure that all relevant SOPs reflect the new guidelines on clinical requirements for sickle cell disease treatments.
