Nermina Ferizovic | Health Economics Expert

Advanced therapy medicinal products (ATMPs) are transforming healthcare by providing patients with potentially curative one-off treatment options. Although non-ATMP therapies have also provided patients with immense benefits, the sheer contrast in terms of the development, manufacture and impact of ATMPs means that these therapies can be considered unique. 

Currently ATMPs are subject to the same value assessment frameworks used to inform decision making in healthcare as non ATMP health interventions. In England, The National Institute for Health and Care Excellence (NICE) has a defined framework as set out in its reference case which ultimately focusses on the cost per quality-adjusted life year (QALY)¹. In the United States, the Institute for Clinical and Economic Review (ICER)² and the Innovation and Value Initiative³ have developed value assessment frameworks for health interventions. 

There has been some discussion in the field regarding the appropriateness and relevance of value frameworks for assessing ATMPs with some researchers claiming that there are elements of value missing or not being considered.^4,5 As such, value assessment frameworks are often described as not capturing the value of ATMPs leading to concerns that current value assessment frameworks are not fit-for-purpose in that they do not capture what is relevant. 

However, in the development of alternative or new value assessment frameworks, it is important to discuss how these should be defined. This is particularly important as there has been limited, or no focus on the methodologies used to development value assessment frameworks for ATMPs and lack of attempts to align with economic theory. 

As part of this discussion, the following elements should arguably be considered:

• How should value elements be identified? 
• How should value elements be defined? 
• Should value be subjective or objective? 
• Which stakeholders’ values should be considered?
• If, and how, differences in stakeholder values should be accounted for? 
• How should the value elements be incorporated in value assessment frameworks? 
• Does the cost per QALY suffice? Can elements of value be incorporated into this framework? If so, how should they be incorporated?
• Finally, should value assessment be linked to increases in social welfare? 

The answer to these and other relevant questions is currently a key gap in health economics but will need to be answered in order to ensure that ATMPs are being appropriately assessed for their value. 

Subsequent blog posts will discuss these key questions.

1. NICE health technology evaluations: the manual. 2023: https://www.nice.org.uk/process/pmg36/chapter/introduction-to-health-technology-evaluation
2. ICER. Value assessment framework. 2023: https://icer.org/our-approach/methods-process/value-assessment-framework/
3. Innovation and value initiative. Principles for value assessment in the US: https://thevalueinitiative.org/principles-for-value-assessment-in-the-us/
4. Lakdawalla et al., Defining Elements of Value in Health Care—A Health Economics Approach: An ISPOR Special Task Force Report [3]: https://www.valueinhealthjournal.com/article/S1098-3015(17)33892-5/fulltext
5. Goring et al., 2021. Novel Elements of the Value Flower: Fake or Truly Novel? https://www.ispor.org/publications/journals/value-outcomes-spotlight/vos-archives/issue/view/navigating-the-changing-heor-publishing-landscape/novel-elements-of-the-value-flower-fake-or-truly-novel