Presentation (CTI)

OBJECTIVES: Evolving evidence requirements across stakeholders, driven by changing policy, pathways, and guidance, are reshaping evidence generation approaches. Using the UK as an example, we assessed evolving evidence requirements across key stakeholder groups and access milestones to identify how integrated evidence plans (IEPs) can remain strategically sequenced and adaptable, as evidence requirements continue to evolve.
METHODS: Targeted desk research of the UK access landscape was conducted including published journal articles, policy documents, and relevant stakeholder websites. Evidence requirements were mapped across regulatory (MHRA), HTA (NICE, SMC, and AWMSG), commissioning (NHS/Integrated Care Boards [ICBs]), implementation (local formulary decision makers, clinicians) and patient/caregiver stakeholders, at key access milestones to identify alignment, stakeholder-specific evidence requirements, and opportunities to optimise evidence planning throughout the product lifecycle.
RESULTS: Our analysis identified that shared requirements included efficacy, safety, HRQoL, comparative effectiveness, endpoints, subgroup analyses, eligible population size, and anticipated uptake. Stakeholder-specific requirements included MHRA benefit-risk and pharmacovigilance, NICE comparative clinical and cost effectiveness, NHS/ICB affordability and expected use, pathway integration, capacity/resource implications, implementation readiness, clinician workflow integration, and patient and caregiver experience, burden, preferences, and support needs. These insights informed the development of an adaptive roadmap, illustrating how use of IEPs can harmonise evidence needs into strategic plans, promoting de-duplication, milestone-based sequencing and agility in response to changing pathways, policy, guidance, and stakeholder requirements.
CONCLUSIONS: IEPs can transform discrete evidence-planning activities into a lifecycle strategy adaptable to evolving regulatory, HTA, payer, and implementation needs amid policy, pathway, and guidance changes. Our roadmap illustrates how structured mapping of shared and unique requirements can be designed with purposeful utility for multiple stakeholders in the UK. This framework provides a basis for application to other national and global contexts.