Spain incorporated in 2020 changes in its health technology assessment (HTA), pricing, and reimbursement system for medicines including publishing reports, development of networks of experts, or consultation with stakeholders. Despite these changes, it is unclear how deliberative frameworks are applied and the process has been criticized for not being sufficiently transparent. This study analyses the level of implementation of deliberative processes in HTA for medicines in Spain.

We review the grey literature and summarize the Spanish HTA, pricing, and reimbursement process of medicines. We apply the deliberative processes for HTA checklist, developed to assess the overall context of the deliberative process, and identify the stakeholders involved and type of involvement following the framework for evidence-informed deliberative processes, a framework for benefit package design that aims to optimize the legitimacy of decision making.

In the Spanish HTA, pricing, and reimbursement process deliberation takes place in order to exchange viewpoints and reach common ground, mainly during the prioritization, assessment, and appraisal steps. It is closed to the public, not clearly summarized in published documents and limited to the Ministry of Health, the regulatory agency, other Ministries, and experts with mostly clinical and/or pharmaceutical background. The views of stakeholders are only represented through consultation. Communication is the most commonly used form of stakeholder engagement.

Despite improvements in transparency of the Spanish HTA process for evaluating medicines, aspects related to stakeholder involvement and implementation of deliberative frameworks need further attention in order to achieve further legitimacy of the process.

To investigate the impact of the uncertainty stemming from products with European conditional marketing authorization (CMA) or authorization in exceptional circumstances (AEC) on the National Institute for Health and Care Excellence's (NICE) recommendations.

Products which received CMA/AEC by European Medicines Agency (EMA) up to 1 December 2016 were identified and matched with corresponding NICE decisions issued by August 2017, the status of which was then traced to August 2019. We assessed whether the conversion of CMA to full marketing authorization triggered a review of a NICE decision. The odds of a recommendation carrying a commercial arrangement for products with and without CMA/AEC were calculated.

Fifty-four products were granted CMA/AEC by EMA. NICE conducted thirty evaluations of products with CMA/AEC. Twelve products were recommended by NICE by August 2017 and fourteen by August 2019. All recommendations had an associated commercial arrangement. The odds of carrying a commercial arrangement were higher for products with CMA/AEC compared to those with full authorization. Conversions from conditional to full authorization among products not recommended by NICE did not trigger an appraisal review.

Uncertainty, stemming from the lack of robust clinical data of products authorized with CMA/AEC, has a substantial impact on HTA recommendations, frequently requiring risk mitigation mechanisms such as commercial and data collection arrangements. Further analyses should be conducted to assess whether the benefits of early access strategies outweigh the risks for patients and the healthcare system.