Nermina Ferizovic | Health Economics Expert

Advanced therapy medicinal products (ATMPs) are commonly described as having the potential to transform healthcare. Contributors to this transformational ability include their relatively high expense, one-off treatment administration, and of course, their potential to cure multiple health conditions. Additional contributors include challenges to both regulatory and health technology assessment.

Health technology assessment (HTA), such as that undertaken by the National Institute for Health and Care Excellence (NICE) in England, undertakes a value assessment of health interventions before providing a reimbursement recommendation. Due to the hefty price tag synonymous with ATMPs, positive recommendations are necessary for market access. Before a value assessment can be undertaken, value should be defined. With regards NICE, this definition is given as part of its reference case and manufacturers are expected to align their submissions to it. However, the question of whether value is defined correctly and appropriately with regards ATMPs becomes an important one which can be answered by asking how the value of ATMPs should be defined. In economics, it is accepted that in order to understand the value of products, it is necessary to consider the benefits they confer. Some of the key considerations include the following.

To begin with, the benefits of ATMPs need to be defined. This would also mean understanding the contributors to the benefits and how these contributors may interact. The benefits and their respective contributors are likely to differ between disease areas which would mean that a one-size fits all value assessment may not be appropriate.

An additional consideration is whether these benefits and their contributors be considered and defined only in relation to patients. Perhaps the relevant stakeholders extend further and include some or all of the following: clinicians, decision makers, legal, payers, the public, regulatory, and scientists? Given the transformative potential of ATMPs, perhaps a wider breadth of stakeholders is necessary; however, this should also be considered carefully and justified. This would also help to determine whose preferences should be considered as part of value assessments. 

This leads to a further consideration of how preferences should be defined and incorporated into HTA as a value assessment cannot be conducted if preference elicitation is not included. If benefits and relevant stakeholders differ from disease area to disease area, then a consistent approach may not be appropriate. 

These considerations have, so far, been missing from the narrative around value assessment of ATMPs. However, for their value to be defined, it is imperative that they be considered.