Presentation (CTI)

OBJECTIVES: Integrated scientific advice (ISA) continues to evolve as regulators, health technology assessment bodies and other stakeholders seek to align evidence requirements earlier in product development. Since our team’s development of a framework to describe ISA programmes in 2020, several new advice programmes have emerged. This study applied the framework to evaluate these newer programmes and identify how ISA has evolved.
METHODS: Publicly available information for four new or substantially expanded ISA programmes (CDA-AMC, ICER, JSC and NICE-MHRA) were compared using the previously developed five-domain framework: Eligibility and Type of Procedure; Process; Briefing Book Requirements; Meeting Advice Deliberations; and Advice Reporting & Follow-up. We also compared these new ISA programmes to those assessed in our prior work (including 17 programmes in Europe and North America).
RESULTS: The framework successfully characterised all four programmes and demonstrated its applicability to emerging ISA procedures. Collectively, the programmes illustrate four distinct developments in ISA: value-focused evidence planning (ICER), incorporation of real-world evidence (RWE) into scientific advice (CDA-AMC), permanent multinational HTA consultation under the EU HTA Regulation (JSC), and enhanced regulatory-HTA integration through a single-entry process and joint reporting (NICE-MHRA). Greater procedural standardisation and organisational coordination were observed; however, differences remain in eligibility, stakeholder participation, scope, timelines and reporting. When comparing to the previously reviewed programmes, the framework shows ISA has evolved from supporting pivotal trial design towards lifecycle evidence planning, with increased emphasis on RWE, post-launch evidence generation, patient-centred outcomes and multi-stakeholder alignment.
CONCLUSIONS: The ISA framework remains applicable to contemporary advice programmes and demonstrates a shift towards more coordinated, lifecycle evidence planning that integrates regulatory, HTA and value assessment perspectives. As ISA programmes continue to diversify, systematic comparison of programme characteristics will become increasingly important to help medicine developers select the most appropriate advice pathway and optimise evidence generation to support market access.