Strategy developments for providing clinical and economic evidence to support formulary decisions are increasingly adopting carefully planned real-world evidence (RWE). As payers move toward value-based assessments, evidence submissions must go beyond efficacy in trial settings to show treatment impact in routine practice and on resource utilization, and alignment with decision criteria revolving around budget, access, and unmet clinical needs. RWE helps bridge these gaps by supporting the value narrative in real-world behaviours of patients, providers, and systems.(1, 2)
Clarity on the specific formulary questions the submission aims to impact is the foundation of an effective strategy. Payers are usually interested in knowing where a treatment fits in the pathway, which populations benefit most, how it compares with existing options, and what monitoring or authorization conditions may be necessary.(3) Early strategizing around these questions ensures that RWE generation emphasizes outcomes that may directly impact formulary and pharmacy and therapeutics (P&T) committee decision-making, including comparative effectiveness, safety in different populations, adherence, maintenance, and utilisation patterns.(3-5)
Selection and validation of applicable real-world data (RWD) sources is the next crucial step.(4) Every source, including claims databases, electronic health records (EHRs), registries, and digital health platforms, offers unique strengths. However, the emphasis should be on ensuring that selected datasets represent actual clinical practice, offer meaningful endpoints, and link to healthcare utilisation and costs. Performing feasibility evaluations, determining data completeness and representativeness, and transparently documenting data provenance reinforce the methodological defensibility of analyses and help payers examine external validity and potential bias.(4)
Once relevant data sources are chosen, the focus moves to developing fit-for-purpose analyses to support the clinical trial package. Thorough observational methods, including matching techniques, confounding adjustment, and sensitivity analyses, facilitate evaluation of real-world efficacy and safety. Incorporating these results into cost-effectiveness and budget impact models enables reflection of real-world utilisation, uptake, discontinuation patterns into economic evaluations, which further benefits designs. The aim is to convert complex analytics into clear, decision-ready insights that show clinical value, total cost of care propositions, and the expected budget impact under realistic implementation scenarios.(4, 5)
Ultimately, an efficient submission depends on well-communicated evidence. A comprehensive value story seeks to bridge the gap between payer expectations and disease burden, clinical and patient-relevant outcomes, and economic performance. Communicating RWE within the structure of a standard dossier format, supplementing with clear summaries, transparent assumptions, and specific acknowledgement of limitations, improves credibility. Outlining results around real clinical and operational challenges, such as lower hospitalisations, optimized care pathways, or enhanced adherence, helps place the findings within system preferences rather than abstract metrics.(3-5)
Finally, an impactful strategy identifies that RWE is not a one-time input but part of continuous evidence generation throughout the product lifecycle. Early and proactive engagement with payers, HTA authorities, clinicians, and patient groups helps facilitate alignment on evidence requirements before the analyses begin. Also, a plan for apprising evidence as new data accumulates supports commitment to monitoring real-world performance and adapting value communications over time. This lifecycle-oriented methodology prioritizes RWE as a dynamic decision support tool that strengthens formulary considerations long after initial submission.
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References
- Alipour-Haris G, Liu X, Acha V, et al. Real-world evidence to support regulatory submissions: A landscape review and assessment of use cases. Clin Transl Sci. 2024; 17(8):e13903.
- Gettman D. Using Real-World Evidence Platforms to Guide Formulary Decisions in Health Systems. Conference: Pharmacy Management (PMD714)At: Buffalo, New YorkAffiliation: D’Youville College. Accessed online on 3rd December 2025 at: https://www.researchgate.net/publication/393377304_Using_Real-World_Evidence_Platforms_to_Guide_Formulary_Decisions_in_Health_Systems
- Jansen MS, Dekkers OM, le Cessie S, et al. Multiple Perspectives on the Need for Real-World Evidence to Inform Regulatory and Health Technology Assessment Decision-Making: Scoping Review and Stakeholder Interviews. Pharmacoepidemiol Drug Saf. 2025 Jan;34(1):e70074.
- Format for formulary submissions. Accessed online on 3rd December 2025 at: https://www.amcp.org/sites/default/files/2019-03/Format%20Version%201%200%20Final%2010.2000.pdf
- CIOMS Working Group report. Real-world data and real-world evidence in regulatory decision making. 2020. Accessed online on 3rd December 2025 at: https://cioms.ch/wp-content/uploads/2020/03/CIOMS-WG-XIII_6June2023_Draft-report-for-comment.pdf