The regulatory landscape in tuberculosis integrated research platforms.

BACKGROUND

Tuberculosis (TB) is the infectious disease with the highest mortality burden globally. Despite a recently enriched drug pipeline, there has been limited advance in improving its treatment. Integrated research platforms (IRPs) provide a novel research framework which, by hosting adaptive platform trials, can streamline the evaluation of several new TB regimens, whilst reducing costs. However, there is no clear, evidence-based guidance on the use of IRPs for regulatory drug development in TB.

METHODS

This consensus statement summarizes the discussion and recommendations produced during the European Union Patient centric clinical trial platforms (EU-PEARL) project. A multidisciplinary task force reviewed the literature regarding regulatory pathways for IRP with special focus on TB and identified current gaps and needs, which were discussed by an extended panel in two consultations. Here we summarise the output of this process and propose possible ways to advance in the implementation of such platforms.

RESULTS AND

CONCLUSION

IRP coordinators are advised to seek an early contact with national regulators and supranational organisms, including regulatory collaboration initiatives. To ensure regulatory acceptance, IRP must prioritize careful design, including clear decision rules for phase transitions, and the development of surrogate makers. Furthermore, data leverage and stratification across groups could enhance IRP success. These efforts will not only strengthen the scientific validity of the IRP but also enhance its regulatory acceptability and impact on global TB treatment strategies.