Real-World Evidence Integration into Cancer HTA: Scotland’s Success Story
How is real-world evidence integration a routine part of Health Technology Assessment (HTA) for cancer medicines in Scotland? Through a successful collaboration between the Cancer Medicines Outcomes Programme–Public Health Scotland (CMOP-PHS) and the Scottish Medicines Consortium (SMC), structured real-world evidence (RWE) generated from national healthcare data is being used to supplement clinical trial findings and reduce uncertainty in decision-making.
Delivering Timely Evidence at Scale
Between February and July 2025, the SMC requested RWE for 13 cancer medicine submissions. CMOP-PHS successfully delivered reports for eight (61.5%) of these, providing essential data on the number of patients treated in the prior 12 months, current comparator regimens used in Scottish practice, and baseline patient characteristics. Feedback from SMC assessors was unanimously positive, with all respondents describing the reports as informative.
Addressing Critical Evidence Gaps with Real-World Data
Real-world evidence integration has proven particularly valuable in resolving common HTA uncertainties around patient population generalisability, choice of relevant comparators, and longer-term outcomes. The delivered reports enabled SMC assessors to directly compare company-submitted trial data against actual Scottish treatment patterns. Information on patient numbers and current comparators received the highest usefulness ratings, while baseline characteristics were also highly valued. Even simple descriptive analyses from the National Systemic Anti-Cancer Therapy (SACT) Dataset have demonstrated meaningful impact when delivered through a transparent, standardised process.
Building a Robust Collaborative Framework
Scotland’s HTA system relies primarily on company submissions to the SMC, often limited by single-arm trials, surrogate endpoints, or accelerated approvals. In response, SMC published a 2025 position statement recognising the role of RWE as supplementary evidence. The CMOP-PHS partnership, established in 2024, brings together NHS Health Boards, Public Health Scotland, and the University of Strathclyde to harness Scotland’s rich linked national datasets.
The pathway follows a rigorous, multi-stage process: early notification from SMC, feasibility assessment, protocol development, analysis using validated R statistical scripts, and internal clinical and academic review. Reports are delivered in time for New Drugs Committee meetings, supported by staff training workshops and structured feedback mechanisms. This approach ensures methodological robustness while fully complying with UK GDPR requirements.
Strategic Implications for HEOR and Market Access
This Scottish model demonstrates how real-world evidence integration can be sustainably embedded into national HTA processes, offering a practical blueprint for other health systems. For HEOR professionals and pharmaceutical companies, it signals that submissions to SMC will increasingly be evaluated against real Scottish patient numbers, treatment patterns, and outcomes.
The rapid uptake within just six months and consistently positive assessor feedback highlight the potential for RWE to reduce decision uncertainty and support more efficient allocation of resources in cancer care. As the programme evolves to include richer data on treatment indications and genomic information, its value is expected to grow further.
