Presentation (CTI)

OBJECTIVES: Pregnant women with congenital thrombotic thrombocytopenic purpura (cTTP) are at increased risk of serious maternal and perinatal complications despite plasma-based therapies. Data from an international, multi-center, retrospective chart review showed that 57.8% (26/45) of ever-pregnant women with cTTP experienced ≥1 pregnancy-related complication (PRC) with fetal death or pregnancy loss occurring in 35.6% (16/45) of these patients, more than double the general population risk (15.7%). Health technology assessment (HTA) cost-effectiveness models (CEMs) typically focus on the patient perspective; however, conditions involving PRCs require a broader perspective to capture the full burden on maternal health and pregnancy outcomes, including quality-adjusted life year (QALY) losses due to pregnancy loss. Here, we assess how PRCs are incorporated in National Institute for Health and Care Excellence (NICE) HTAs/guidelines and estimate the potential QALY impact of PRCs in cTTP using precedent models and published chart review data.
METHODS: NICE guidelines and single technology appraisals (STAs) published between 2005 and 2025 that included pregnancy-related conditions were reviewed. QALY losses were derived from published NICE models by applying lifetime-discounted fetal QALYs for perinatal death.
RESULTS: Among 10 pregnancy-focused NICE publications, four guidelines and one STA incorporated PRCs in economic models, applying QALY losses based on discounted lifetime QALYs of the fetus. One guideline included live births and managed miscarriage outcomes and another used cost-minimization analysis. Applying QALY loss methodology to the cTTP chart review data suggests that approximately 9 QALYs may be lost per affected pregnancy, indicating a significant burden compared with the general population.
CONCLUSIONS: PRCs impose a substantial burden on maternal and fetal health. Accepted HTA methods exist to quantify this burden. Future HTA submissions for cTTP therapies should incorporate maternal and fetal QALY losses to better inform payer decisions and underscore the urgent need for effective treatments to reduce the impact of PRCs in this vulnerable patient population.