HTA Reshaping: Rethinking the Health Technology Assessment Framework in Portugal

Abstract

<b><i>Introduction:</i></b> Health technology assessment (HTA) aims to provide decision makers with relevant data to make informed choices. Recent changes in the Portuguese HTA framework have altered substantially the assessment methodology with regard to economic evaluation, with potential impacts on access and process efficiency. The HTA Reshaping Project had as its main objective informing the debate on HTA in Portugal, thereby identifying improvement opportunities and solutions to the HTA framework that address future challenges. <b><i>Methods:</i></b> The project comprised several phases, i.e., (1) mapping and evaluation of different HTA frameworks across Europe, identifying best practices and key variables to consider in future frameworks; (2) conduction of in-depth interviews with relevant stakeholders (<i>n</i> = 11); and (3) development of 2 workshops – one with young professionals (<i>n</i> = 12) and another with relevant HTA stakeholders (<i>n</i> = 19) – to consolidate and further explore vital elements of HTA, aimed at brainstorming ideas and developing solutions to improve some of the most critical points, with a view to addressing future challenges. <b><i>Results:</i></b> The comparison of HTA frameworks showed that their purpose and sophistication vary across European countries. For example, the need for economic evidence is not unanimous, and reimbursement agreements vary considerably. Among the stakeholders interviewed there was a high level of agreement on priorities that should be addressed, e.g., expanding and creating national level registries and assuring patient participation throughout the HTA process. The possibility of using managed entry agreements to enhance patients’ access, applying multi-indication pricing for medicines with different therapeutic values per indication, and improvement of registry/system interoperability gathered a moderate level of agreement. <b><i>Conclusions:</i></b> The Portuguese HTA framework might be further adapted to upcoming challenges and should evolve to improve access to innovative therapies. There is still a long path towards the convergence of HTA frameworks in EU member states.

Not one, but many: developing a multi-indication pricing model for medicines in Belgium

“…The literature review identified 77 hits, excluding duplications. After review of abstract, and full paper when in doubt, 16 references were included (seven references from Web of Science and nine references from the grey literature) (Pearson et al, 2016;Campbell, 2017;Baker et al, 2018;Cookson, 2019;Lawlor et al, 2021;Pereira et al, 2021;Creating;Adida, 2022;Michaeli et al, 2022a;Michaeli et al, 2022b;Gill et al, 2022;Ha et al, 2022;Pani et al, 2022;Preckler and Espin, 2022;Vokinger and Kesselheim, 2022;Zhang et al, 2022). This section summarises the main messages from the literature review, focusing on MIP typologies, (dis)advantages and implementation challenges.…”

“…Netherlands, for example, opted to establish a price for the first indication, which is used to anchor the price of all further indications. If further indications are not cost-effective (at the prevailing price), either a lower price is negotiated or the medicine may not be reimbursed (Pereira et al, 2021). Italy uses a large registry with individual patient data (thus requiring extensive data systems), and the UK reviews each indication separately and recommends it if it is shown to be clinically and cost-effective (Pereira et al, 2021).…”

“…Belgian stakeholders argued that the initial price should be set lower than the volume-and value-weighted average price across indications as, in their opinion, value at launch can sometimes be difficult to estimate. The implementation of this MIP model requires that a legal Pearson et al, (2016) ;Campbell, (2017); Baker et al, (2018) ;Cookson, (2019); Lawlor et al, (2021); Pereira et al, (2021) ;Adida, (2022); Michaeli et al, (2022a); Michaeli et al, (2022b); Gill et al, (2022); Ha et al, (2022); Pani et al, (2022); Preckler and Espin, (2022).…”

“…Additionally, it was thought that MIP model 5 is less attractive for pharmaceutical industry and might lead to fewer market introductions, except for medicines with high uncertainty. [Michaeli et al, (2022a); Lawlor et al, (2021); Michaeli et al, (2022b); Preckler and Espin, (2022); Adida, (2022); Baker et al, (2018) ;Campbell, (2017) ;Cookson, (2019); Gill et al, (2022); Ha et al, (2022); Pani et al, (2022); Pearson et al, (2016); Pereira et al, (2021); Creating].…”

“…Disadvantages of MIP modelsPearson et al, (2016);Lawlor et al, (2021);Pereira et al, (2021); Adida, (2022);Michaeli et al, (2022a);Michaeli et al, (2022b);Gill et al, (2022);Ha et al, (2022);Pani et al, (2022);Preckler and Espin, (2022).…”