Real-World Evidence (RWE) and Its Impact on Scottish Medicines Consortium (SMC) Decisions: A Retrospective Review
Author(s)
Fatene Abakar Ismail, PhD, Alex Henriquez, MSc, Pauline McGuire, MSc, Yvonne Semple, PhD.
Scottish Medicines Consortium, Glasgow, United Kingdom.
Scottish Medicines Consortium, Glasgow, United Kingdom.
OBJECTIVES: The SMC advises NHSScotland on the value of newly licensed medicines. Recognising that randomised controlled trials (RCTs) are not always feasible, particularly for rare/ultra-rare or rapidly evolving therapeutic areas, SMC acknowledges that RWE can complement early-phase or single arm studies. This project examined how RWE has been used in SMC submissions, and its influence on SMC decision-making.
METHODS: A retrospective review (2018-2025) of all company submissions containing RWE was conducted. Eligible submissions specified use of RWE (control cohort studies, patient’s registry, observational studies). Submissions were excluded if SMC advice was unpublished, deferred or withdrawn. Submission characteristics including therapeutic area, prevalence of disease, source and purpose of RWE were collected from SMC’s internal database. A descriptive analysis was conducted to explore a correlation between the use of RWE and SMC’s final decision.
RESULTS: Of 675 submissions, 55 contained RWE; 39 with published SMC advice were analysed. RWE use in submissions peaked in 2022 (n=10). The majority (79%) of submissions were for orphan (n=23) or ultra-orphan (n=8) conditions; oncology (n= 14) and rare genetic conditions (n=13) were the most common therapeutic areas. Patients’ registries and observational cohort studies were the most common sources of RWE used to support the evidence. Base-case analyses were comprised of phase I/II studies (n=19), patients’ registry (n=6), indirect comparisons (n=12) or RCT (n=7). The SMC Committee accepted for use or restricted use 26 (67%) of the 39 submissions.
CONCLUSIONS: Use of RWE in SMC submissions has increased since 2018, adding value to decision-making. RWE has contributed by providing additional evidence in areas where clinical trial data have limitations, especially for conditions with high unmet need such as rare diseases and oncology. SMC recognises the value of RWE to inform the health technology assessment of medicines and has recently published a position statement on its use.
METHODS: A retrospective review (2018-2025) of all company submissions containing RWE was conducted. Eligible submissions specified use of RWE (control cohort studies, patient’s registry, observational studies). Submissions were excluded if SMC advice was unpublished, deferred or withdrawn. Submission characteristics including therapeutic area, prevalence of disease, source and purpose of RWE were collected from SMC’s internal database. A descriptive analysis was conducted to explore a correlation between the use of RWE and SMC’s final decision.
RESULTS: Of 675 submissions, 55 contained RWE; 39 with published SMC advice were analysed. RWE use in submissions peaked in 2022 (n=10). The majority (79%) of submissions were for orphan (n=23) or ultra-orphan (n=8) conditions; oncology (n= 14) and rare genetic conditions (n=13) were the most common therapeutic areas. Patients’ registries and observational cohort studies were the most common sources of RWE used to support the evidence. Base-case analyses were comprised of phase I/II studies (n=19), patients’ registry (n=6), indirect comparisons (n=12) or RCT (n=7). The SMC Committee accepted for use or restricted use 26 (67%) of the 39 submissions.
CONCLUSIONS: Use of RWE in SMC submissions has increased since 2018, adding value to decision-making. RWE has contributed by providing additional evidence in areas where clinical trial data have limitations, especially for conditions with high unmet need such as rare diseases and oncology. SMC recognises the value of RWE to inform the health technology assessment of medicines and has recently published a position statement on its use.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA278
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Neurological Disorders, Oncology, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain), Urinary/Kidney Disorders