Discussions about potential solutions addressing respective evidence needs to support decision making
across the life cycle must acknowledge the difference in scopes between marketing authorisation,
including potential orphan maintenance applications as applicable, and HTA decision making.
That said, any discussions of the necessary clinical studies must also acknowledge the specific regulatory scope
for clinical trial applications and its implications for downstream evidence generation.
Overall, there was general agreement between HTAbs and regulatory participants that clear research questions of clinical interest need to be pre-specified, which together with context-specific feasibility concerns then
drives the choice of what constitutes adequate evidence generation.
Regulatory-HTAb collaboration early at the design stage and throughout the entire product lifecycle is
therefore key. This may include early clinical trials used to support conditional marketing authorisation,
‘pivotal’ clinical trials, to post-launch studies.
In many cases, HTA and regulatory research questions are distinct but overlapping; wherever possible, studies should be designed to meet both decision makers evidence needs simultaneously.