Background <p>Advanced therapy medicinal products (ATMPs) are promising healthcare interventions which are transforming healthcare. Despite this, there is limited research exploring the value of these interventions with studies largely focussing on reimbursement challenges. The aim of this study was to conduct a comprehensive review of the existing literature on the value attributes of ATMPs. </p> Methods <p>A comprehensive targeted literature review was conducted to identify the literature on the value attributes for ATMPs. The databases searched included MedLine(R) ALL (1946 to current date) and Embase (1980 to 2024 July 27) via Ovid. Relevant MeSH terms related to advanced therapy, value, reimbursement, technology appraisal and health policy were used. Included studies were snowballed and where relevant, quality assessed. </p> Results <p>The review included 56 unique studies. Twenty-one (37%) were qualitative studies, 8 (14%) literature reviews, 14 (25%) perspectives/commentaries/viewpoints, 5 (9%) reviews of health technology appraisals, 6 (10%) reports, and 2 (3%) conference discussions. Identified value attributes were categorised into three themes. Direct treatment-specific consisted of safety/toxicity, efficacy, cure, treatment burden, value of hope, impact on future treatment choice, impact on daily activities, quality of life, uncertainty, mortality, disease severity, scientific spillovers, unmet need, quality-adjusted life year, age of onset, value of knowing, and innovation. The ‘Economic’ category included costs/financial burden, family/caregiver spillovers, insurance value, improvements to care processes, and productivity. Finally, the ‘societal’ category included ethics/equity and altruism.</p> Conclusions <p>This research contributes to the understanding of the concept of value of ATMPs. Additional workstreams will seek to finalise the list of value attributes.</p>

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Value attributes of advanced therapy medicinal products: a comprehensive literature review

  • Nermina Ferizović,
  • Caroline S. Clarke,
  • Rachael M. Hunter,
  • Ruth Plackett,
  • Nicholas Freemantle

摘要

Background

Advanced therapy medicinal products (ATMPs) are promising healthcare interventions which are transforming healthcare. Despite this, there is limited research exploring the value of these interventions with studies largely focussing on reimbursement challenges. The aim of this study was to conduct a comprehensive review of the existing literature on the value attributes of ATMPs.

Methods

A comprehensive targeted literature review was conducted to identify the literature on the value attributes for ATMPs. The databases searched included MedLine(R) ALL (1946 to current date) and Embase (1980 to 2024 July 27) via Ovid. Relevant MeSH terms related to advanced therapy, value, reimbursement, technology appraisal and health policy were used. Included studies were snowballed and where relevant, quality assessed.

Results

The review included 56 unique studies. Twenty-one (37%) were qualitative studies, 8 (14%) literature reviews, 14 (25%) perspectives/commentaries/viewpoints, 5 (9%) reviews of health technology appraisals, 6 (10%) reports, and 2 (3%) conference discussions. Identified value attributes were categorised into three themes. Direct treatment-specific consisted of safety/toxicity, efficacy, cure, treatment burden, value of hope, impact on future treatment choice, impact on daily activities, quality of life, uncertainty, mortality, disease severity, scientific spillovers, unmet need, quality-adjusted life year, age of onset, value of knowing, and innovation. The ‘Economic’ category included costs/financial burden, family/caregiver spillovers, insurance value, improvements to care processes, and productivity. Finally, the ‘societal’ category included ethics/equity and altruism.

Conclusions

This research contributes to the understanding of the concept of value of ATMPs. Additional workstreams will seek to finalise the list of value attributes.