Nermina Ferizovic | Health Economics Expert

Advanced therapy medicinal products (ATMPs) are ground-breaking genetic and cell/tissue-based therapies with the potential to treat and cure myriad health conditions (1,2). However, they have faced multiple challenges in decision making with relatively low success in achieving reimbursement (3-7). As such, there may be a disconnect between existing value assessment frameworks which Health Technology Assessment (HTA) agencies employ and the potential value ATMPs bring to patients and society. 

Thus, understanding the attributes of value of ATMPs from a multi-stakeholder perspective may provide information which would help economists to understand whether current HTA processes capture the full value of ATMPs. 

As a first step in a research project exploring the value advanced therapies bring to society, a literature review was used to identify studies across MedLine, Embase, Allied and Complementary Medicine Database, Global Health, ProQuest Central, Social Sciences Premium Collection, Applied Social Sciences Index and Abstracts, Econ Lit, Web of Science and SCOPUS from inception to 3rd March 2024. Search terms for genetic and cell/tissue-based therapies were conducted separately. Studies were included if they were a peer-reviewed primary research study or systematic literature review using stated preference measures to elicit preferences for ATMPs.

Thirteen studies were included with 8 pertaining to genetic therapies and 5 to cell/tissue therapies. The quantitative genetic therapy studies were focussed on haemophilia (3 studies; 75%) and sickle cell disease (1 study; 25%) and were limited to discrete choice experiments and a threshold analysis. These studies identified that ‘clinical benefits’, ‘treatment burden’, ‘uncertainty/risk’, ‘quality of life’ were important considerations for stakeholders. The quantitative cell and tissue-based therapy studies were all focussed on oncology. Discrete choice experiments were the preference measure of choice in three of the studies with two utilising a time trade-off approach. These studies identified that ‘clinical benefit’ (including the potential for a cure), ‘uncertainty/risk’, and ‘treatment burden’ are the most important considerations for stakeholders. A comparison between the two categories of ATMP found that attributes are widely similar between the categories of ATMP but there is an indication that there are differences between them, namely in the importance of quality of life; however, this may be as a result of the small number of studies identified and with the cell and tissue-based therapy studies focussing on understanding the trade-offs between clinical benefits and adverse events.

In summary, ‘clinical benefit’, ‘uncertainty/risk’, and ‘treatment burden’, and ‘quality of life’ were found to be important value attributes to stakeholders with suggestions that other elements such as having an additional treatment option and being informed as part of treatment decisions are also important to stakeholders. A key finding is that attributes of value are consistent between genetic therapies and cell and tissue-based therapies. 

Further, the study also identified research and methodological gaps which are arguably contributing to the lack of advancement in this area. For instance, current studies aiming to understand the value of advanced therapies are limited to patients and clinicians. In order to understand the broader value and the attributes of value associated with these innovative therapies, it is important to include further stakeholders to ensure all relevant value attributes are considered in HTA. There is also a lack of quality assessment tools for preference studies with this limited to conjoint analysis. There is also the potential for preference studies needing to be adapted and for new preference quantification methods to be developed to account for the specifics of advanced therapies. 

To summarise, there is limited research on the preferences for ATMPs; but these findings suggest that the value attributes of ATMPs are consistent across gene and cell/tissue therapies with clinical benefit, uncertainty, risk, treatment burden, and quality of life being important. However, additional research is needed to confirm and expand on these findings. Finally, in order to support the field to advance, new tools and methods will need to be developed.  

The interested reader can find the link to the full paper here: https://journals.sagepub.com/doi/10.1177/00469580251390763

References:

1.         EMA. Advanced therapy medicinal products 2022 [Available from: https://www.ema.europa.eu/en/human-regulatory/overview/advanced-therapy-medicinal-products-overview.

2.         ABPI. Advanced therapy medicinal products: Transformative treatments 2022 [Available from: https://www.abpi.org.uk/value-and-access/advanced-therapy-medicinal-products-atmps/.

3.         Ronco V, Dilecce M, Lanati E, Canonico PL, Jommi C. Price and reimbursement of advanced therapeutic medicinal products in Europe: are assessment and appraisal diverging from expert recommendations? Journal of Pharmaceutical Policy and Practice. 2021;14(1):30.

4.         Remap Consulting. Why is the number of advanced therapy medicinal products (ATMP) withdrawals from the European market rising? 2022 [Available from: https://remapconsulting.com/atmps-in-europe/.

5.         Rothwell S, January D, Duda M, Gardner K, Michel S. Challenges and Future Perspectives on The Reimbursement of Advanced Therapy Medicinal Products. Value Health. 2017;20(9):A705.

6.         Hanna E, Marre C, Toumi M. PBI59 THE REIMBURSEMENT STATUS OF ADVANCED THERAPY MEDICINAL PRODUCTS IN EUROPE. Value in Health. 2019;22:S428.

7.         Jørgensen J, Kefalas P. Reimbursement of licensed cell and gene therapies across the major European healthcare markets. J Mark Access Health Policy. 2015;3:10.3402/jmahp.v3.29321.