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August 4, 2025HealthcareOpen Access

Pharmacoeconomic Profiles of Advanced Therapy Medicinal Products in Rare Diseases: A Systematic Review

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Authors

MSMarianna SerinoMKMilana KrstinSMSara Mucherino

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Overview

Systematic review analyzes cost-effectiveness of advanced therapy medicinal products in rare diseases, suggesting economic potential but variability in outcomes.

Key Points

  • ATMPs showed promising clinical outcomes, yet cost-effectiveness varies significantly across different rare diseases.
  • Sixty-one studies were included, with ATMPs for rare blood diseases often being cost-effective across many evaluations.
  • Incremental QALYs for Tisagenlecleucel and Axicabtagene Ciloleucel ranged from 1.5 to 10 per patient, indicating substantial benefits.
  • Policymakers need to balance innovation with sustainability, taking into account long-term patient outcomes and system costs.

Cite This Study

Serino et al. (2025) studied this question.

synapsesocial.com/papers/689a0f86e6551bb0af8d0a69https://doi.org/10.3390/healthcare13151894
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  2. 2Why promising ATMPs fail to reach patients: a qualitative stakeholder-informed analysis of cell therapy access in Europe2026 · 1 citations
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  5. 5Improving access to gene therapy for rare diseases2024 · 38 citations