The results of HTx can be outlined based on the key objectives:
1. One of the key HTx studies, based on the input of more than 20 European HTA bodies, demonstrated that advanced therapeutic medicinal products (ATMPs) and histology-independent therapies were considered to be the most challenging to evaluate based on the predened complex health technologies and case studies. It also concluded that most challenges in HTA of complex health technologies are rooted in data insufciencies rather than the complexity of health technologies. As the number of complex technologies grows, the urgency for new methods and policies to guide HTA decision-making was clearly highlighted in this study.
2. As a starting point, the current landscape of the average and individualised real-world effectiveness and cost-effectiveness of health technologies was reviewed. These reviews assessed:
- Quality assessment tools for non-randomised studies of interventions (NRSI).
- Studies assessing the value of diabetes monitoring systems compared studies that used RCT data and RWD.
In a next phase, a suite of Bayesian NMA and network meta-regression (NMR) models was developed, allowing for cross-design (RCT and NRS) and cross-format (IPD and AD) synthesis, and implemented these models in a new R package crossnma. Additionally, a framework for a personalised prediction modelling based on cross-design NMA was developed, and its importance to use in health economic evaluation was exemplified.
Finally, the Target Trial Emulation (TTE), a framework that uses causal inference from observational data and that can be viewed as an attempt to emulate a hypothetical randomised trial (i.e. the target trial), was further developed in HTx to allow for analysis of RWD to (try and) answer the clinical and HTA question of interest, for application in decision modelling for cost-effectiveness analysis.
3. Prediction models were developed to support an individualised treatment approach for all case studies.
4. Several policy instruments were developed under HTx. Key outputs were:
5. The activities for transferability of the models developped were organised
During the entirety of the project, we have published:
14 newsletters
27 YouTube videos (of which 17 are part of the HTx Patient Toolbox)
40 blog entries on the HTx website
120 posts on the project’s LinkedIn profile
181 tweets and retweets on the project’s Twitter / X profile
Published 45 articles in peer-reviewed scientific journals
Organised 30 HTx events
Reported presenting over 115 times at scientific meetings and conferences, including ISPOR (in 2019, 2022 & 2023), ISPOR Europe (in 2019, 2020, 2021, 2022 & 2023) and HTAi (in 2019, 2021, 2022 & 2024) annual meetings.
