OBJECTIVES:

In the advent of personalized advanced therapies, innovation continues to accelerate at unprecedent speed. The upcoming years will bring to the market new gene therapies (e.g. tumor agnostic), cell replacement therapies, gene modified cells (e.g. CAR-T). Over the years, Portuguese authorities (INFARMED) revised the national guidelines of Heath Technology Assessment (HTA). The latest update regarding HE (Health Economics) guidelines was in December 2019. The main goal of this study is to provide a systemized review by HTA experts from academia and industry about the implications and possible strategies to improve the Portuguese HTA framework.

METHODS:

The current HTA framework on HE was analyzed considering the challenges that innovation brings to go-to-market evaluation and approval. These findings were discussed in a Think Tank with a panel of experts to identify implications and recommendations to the HE methodology, evaluating its effect in accelerating or delaying patients’ access to new therapies.

RESULTS:

From this study, there were identified critical challenges for authorities and manufacturers to deliver innovation at a faster speed, such as, understanding the impacts and biases in considering the NHS perspective on cost-effectiveness assessment, the need to evaluate and minimize uncertainty in assessments, or the lack of standardized alternatives for cost-utility analysis when EQ-5D is not the most suitable tool. In this study, experts discussed and proposed a set of strategies to address current and future challenges of innovation.

CONCLUSIONS: There is a growing effort in developing, not only innovative therapies, but also new methodologies to evaluate them. In this study, there are discussed challenges and possible strategies to improve the HE of the Portuguese HTA process. By doing so, the authors expect to contribute to foster access of European and Portuguese patients to innovation.