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  • Developing an RWE-Driven Strategy for Formulary and Access Decisions

    Developing an RWE-Driven Strategy for Formulary and Access Decisions

    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

    1. 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.
    2. 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
    3. 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.
    4. 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
    5. 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

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  • Formulating Fit-for-Purpose Evidence Packages to Demonstrate the Value of Interventions for Rare Diseases

    Formulating Fit-for-Purpose Evidence Packages to Demonstrate the Value of Interventions for Rare Diseases

    Formulating Fit-for-Purpose Evidence Packages to Demonstrate the Value of Interventions for Rare Diseases

    The increasing emphasis on rare diseases brings with it both hope and challenges. With over 7,000 rare diseases detected globally, affecting millions of people, the need for novel treatments is crucial.(1) Yet, developing and determining the value of these interventions has some unique challenges. Conventional frameworks for evidence generation, typically developed for common conditions, may not align with the realities of small patient populations, heterogeneous disease progression, and lack of natural history data as in case of rare diseases. This makes it crucial to move beyond traditional methods and develop fit-for-purpose evidence packages that can successfully elucidate the value of rare diseases interventions.(1-3)

    One of the main concerns in rare disease research is the lack of patients, which hinders the feasibility of large randomized controlled trials (RCTs). Typical endpoints may also be difficult to apply due to high variability of disease manifestations that may not be effectively described by standard outcome measures. To address this, evidence packages for rare diseases must integrate diverse sources of information, right from clinical trial data to real-world evidence (RWE), natural history studies, and patient-reported outcomes (PROs). Such an integrated methodology enables stakeholders, including regulators, health technology assessment (HTA) bodies, and payers, to obtain an all-inclusive understanding of the value of an intervention.(2-4)

    Customizing the evidence package to the decision context is an equally important factor. For regulatory approvals, although the emphasis is on demonstrating safety and efficacy with the best available study designs; they are often supported by surrogate endpoints. However, for payers and HTA agencies, the focus is often on the long-term efficacy, cost-effectiveness, and the larger impact on patients and caregivers. This warrants cautious alignment of clinical data with economic models, quality-of-life (QoL) metrics, and studies on disease burden. By designing the evidence to meet the specific expectations of all the involved stakeholders, developers can more effectively generate the evidence required for regulatory submissions.(2-4)

    Another key factor in formulating fit-for-purpose evidence packages is the collaboration among stakeholders. Rare disease populations often play a central role in defining important endpoints, assisting with data collection, and allowing for the outcomes being evaluated to correctly depict important benefits to patients and caregivers. Collaborations across industry, academia, and patient groups can also enable the development of disease registries and natural history databases that act as crucial drivers of evidence generation. Optimizing advanced analytics, digital health tools, and novel study designs, such as basket trials or adaptive designs further enhances these evidence packages, offering the necessary flexibility in rare disease settings.(2-4)

    Finally, the goal is to secure market access for rare disease treatments while ensuring that patients receive well-timed and fair access to novel therapies that can transform their lives. By moving beyond unyielding, one-size-fits-all frameworks and emphasizing fit-for-purpose evidence generation, stakeholders can better encapsulate the multidimensional value of interventions for rare disease. This transformation in evidence strategy is instrumental in bridging the gap between scientific innovation and real-world patient benefit.

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    References

    1. Liu J, Barrett JS, Leonardi ET, et al. Natural History and Real-World Data in Rare Diseases: Applications, Limitations, and Future Perspectives. J Clin Pharmacol. 2022; 62 Suppl 2(Suppl 2):S38-S55.
    2. National Academies of Sciences, Engineering, and Medicine; Health and Medicine Division; Board on Health Sciences Policy; Committee on Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union; Shore CK, Worku TL, Smith CW, et al., editors. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington (DC): National Academies Press (US); 2024 Oct 30. 4, Alternative and Confirmatory Data. Available from: https://www.ncbi.nlm.nih.gov/books/NBK609394/
    3. Pai M, Yeung CHT, Akl EA, et al. Strategies for eliciting and synthesizing evidence for guidelines in rare diseases. BMC Med Res Methodol. 2019 Mar 28;19(1):67.
    4. ISPOR. Workshop: Development of Evidence Packages for Regulatory and Reimbursement Submissions in Rare Diseases: Real‐World Examples. 2015. (Accessed online on 28th August 2025). Available at: https://www.ispor.org/docs/default-source/presentations/597.pdf?sfvrsn=b428af52_1

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