Darvadstrocel: real-world clinical outcomes and economic impact in the Spanish national health system

Abstract

Background Complex perianal fistulas in Crohn’s disease (CD) represent a therapeutic challenge. Darvadstrocel has demonstrated efficacy in clinical trials, but evidence from real-life clinical practice is limited. This study evaluated the effectiveness and safety of darvadstrocel in real-life clinical practice, and assessed the economic impact associated with the outcome-based payment model (OBPM) linked to its funding within the Spanish National Health System.

Methods An observational, descriptive, retrospective study was conducted on patients treated with darvadstrocel in the Servizo Galego de Saúde (SERGAS) between December 2019 and December 2024. Data were collected from the Therapeutic Value of Medicines Information System (VALTERMED), including demographic, clinical, safety and effectiveness variables at 6 and 12 months post-treatment. Descriptive statistics and Fisher’s exact test were used for subgroup analyses.

Results A total of 26 patients were included (50.0% female; median age: 38.4 years). Combined remission was achieved in 69.2% (n=18) at 6 months and 57.7% (n=15) at 12 months. No significant differences were observed among subgroups. No treatment-related adverse events were reported. Regarding sustainability, the OBPM resulted in SERGAS covering 81.5% of the total treatment costs, as the second payment installment was not made for non-responders.

Conclusions Darvadstrocel demonstrated high effectiveness and safety in real-world clinical practice for patients with CD and complex perianal fistulas, with remission rates consistent with previous studies. The implementation of the OBPM linked to health outcomes proved to be a valuable tool for funding innovative therapies.

Keywords Darvadstrocel, mesenchymal stem cells, Crohn’s disease, remission induction, safety

Ann Gastroenterol 2025; 38 (6): 661-666