OBJECTIVES: The past decades have seen an unprecedented growth of innovation in health technologies targeting smaller populations and rare diseases with biologics development, as well as new as faster health assessment programs. Today, regulatory authorities face increasingly complex processes, based on evidence models that support greater degrees of uncertainty, but at the same time require more specialized technical expertise to assess health technologies’ value. Over the years, INFARMED revised the national guidelines of Heath Technology Assessment (HTA), regularly. In this study, the evolutions of the Portuguese HTA framework are evaluated with the perspective of experts from academia and pharmaceutical industry, preparing them to accommodate the challenges of the future innovative therapeutics and, hence, provide greater patients’ access.

METHODS: The current and proposed revisions on HTA framework were analyzed considering the future innovative pipeline, identifying the key evolutions of HTA process, when compared with the previous ones and benchmarking. These findings were discussed in a Think Tank with a panel of experts to identify implications and recommendations to the ATV process, evaluating its effect in accelerating or delaying patients’ access to new therapies.

RESULTS: Based in the study, questions, such as, how to prove ATV with several subgroups and smaller populations, how to overcome the problem of limited evidence in advanced therapies were discussed and proposed a set of initiatives useful for regulators and manufacturers.

CONCLUSIONS: Balancing speed and accuracy in the HTA will prove to be key to deliver faster innovation to patients. Examples of these involve new studies designs, the collaboration between regulators and manufacturers in pre-assessment processes, the integration of patient-led evidence or re-evaluation HTA process are critical to remove uncertainty.