Nermina Ferizovic | Health Economics Expert

For my latest blog post, I provide a perspective on an article forming part of a themed section in Value in Health. The article titled: “Evolving Concept of Value in Health Economics and Outcomes Research: Emerging Tools for Innovation and Access to Cell and Gene Therapies for Rare Diseases” which summarises potential solutions to improving access to cell and gene therapies. The paper can be found here.

The authors describe five potential solutions to improve patient access to these transformative medicines. These are grouped into the following: “collection of real-world data to generate real-world evidence, patient centricity and novel value assessment frameworks, evolution of economic evaluation methodologies, novel reimbursement models and management of affordability, and health equity and societal benefits”. I will discuss ‘patient centricity and novel value assessment frameworks’ with a focus on novel value assessment frameworks as this is where my research interests lie. 

On this point, the article highlights the lack of patient centricity in value assessment and the lack of assessment of long-term value. To that end, the authors highlight that the inclusion of novel elements of value (such as value of knowing, insurance value and disease severity) would account for patient and societal perspectives and (implicitly) lead to a more appropriate assessment of these transformative medicines. 

To extend on this viewpoint, the following considerations are key when developing a value assessment framework for cell and gene therapies:  

• It is imperative that any value assessment framework developed to assess the (long-term) value of cell and gene therapies includes all relevant value elements. This includes values from the perspective of all relevant stakeholders – including, but not limited, to patients. 
• The value elements must be relevant to cell and gene therapies. That is, there must be robust evidence of the applicability of value elements to these therapies before they can be included in a value assessment framework. Although potentially relevant to cell and gene therapies, there is limited specific research on the ‘value’ of novel elements of value to relevant stakeholders in relation to cell and gene therapies. 
• There is limited evidence that value elements may differ between disease areas particularly for advanced therapies, but differences should, and could be, accounted for in a comprehensive value assessment framework.
• The relative importance of value elements – and the extent to which they should be accounted for in value assessment – are likely to differ between healthcare systems. For instance, the ‘value’ of economic spillovers may be different in a publicly funded healthcare system compared to a private one. Of course, where a publicly funded healthcare system is in question, it is important to include a societal perspective. 

Any value assessment framework developed for cell and gene therapies should carefully account for these considerations.