The first ten years of Conitec's operation featured growth in terms of patient and public involvement as evidenced by the number of public consultations conducted and contributions received over the years; in addition, 13% of recommendations changed after the public consultation, and the significant participation of patients and their families was strongly associated with changes in preliminary recommendations regarding the adoption of technologies into the SUS. Previous investigations, which featured shorter follow-up times or analysed specific subgroups, found the frequency of change in recommendations following a public consultation to 18.8% in the case of medicines up to June 2016 (13), 8% in the case of medicines, products and procedures by 2018 (2) and 19% in the case of rare diseases by June 2019 (14).
Despite the fact that the participation mechanism adopted had only an advisory nature, the results indicate that people who are affected directly by the health condition or the technology being evaluated have a greater influence on decisions regarding the adoption of technologies in the SUS than other stakeholders.
Although the causal relationship associated with the association thus identified cannot be confirmed, some characteristics of this study strengthen the presented conclusions. First, all processes associated with the incorporation of medicines, products or health procedures analysed by Conitec during the period of 10 years under study into the SUS were included in the regression model, which mitigates concerns related to possible sample bias or a lack of internal validity.
Second, the analysis considered the temporal relationships among the events by reference to objective measurements of the outcomes (recommendation changes) preceded by the exposure variable (public consultations). Third, the effect exhibited a large magnitude (OR 3.87) and the positive and significant results concerning the associated exhibited high consistency, with a confidence interval that assumes the possibility of even greater effects of the association (CI 95% 1.33–13.35).
Finally, the analysis was characterized by specificity in terms of the subgroup in which the effect was observed (exclusively for the subgroup of patients and family members), the dose‒response relationship with regard to the division of groups into those with higher and lower volumes of participation, and the plausibility of the hypothesis that people who are affected directly by the decision have a greater influence on the decision.
Impact evaluations of PPI strategies remain scarce and have found limited or inconsistent results. The systematic review conducted by Boivin and collaborators (2018) to investigate PPI assessment instruments indicated a greater focus on the impacts perceived and self-reported by the participants, with the majority of such assessments being focused on contexts and processes and fewer assessments emphasizing impacts that are observable by external evaluators (15). In health decisions informed by HTA, PPI impact assessments have been identified with the result of providing subsidies for HTA studies, such as willingness-to-pay measures or discrete choice questionnaires (11, 16). In the systematic review of international HTA public engagement experiences conducted by Gagnon et al. (2021), the authors concluded that there is a paucity of strong evidence regarding the impact of PPI initiatives, especially in the long term (16). The impact assessment presented in this paper therefore represents an important advance in our knowledge concerning the influence of PPI on health decisions.
Silva et al. (2019) presented a timeline of the PPI actions taken in the processes of technology incorporation implemented by Conitec until December 2017, which can improve our understanding of the increase in the number of contributions made in the context of public consultations during the period under analysis (5). The main actions were readjustment of the public consultation forms in 2014, with the provision of different forms for contributions pertaining to experience or opinions and for those concerning technical and scientific points; an increase in the availability of reports in a summarized format and using simplified language that were specifically targeted at the lay public in 2015; and the publication of a guide called “Understanding the incorporation of health technologies in the SUS: How to get involved” in 2016 with the objective of informing and guiding the PPI processes implemented by the institution (5). It can be inferred that these actions may have contributed to the increase in engagement observed in subsequent years, especially during the period between 2015 and 2019.
With regard to the decline in the volume of participation in the following years, i.e., 2020 and 2021, two coincident processes occurred during this period: the introduction of a government that was less aligned with participatory practices and the coronavirus disease (COVID-19) pandemic. Four months after the start of the mandate, in April 2019, President Bolsonaro decreed the revocation of the National Policy of Social Participation, which had been instituted in 2014, and eliminated all participatory collegiate bodies that had not been created by law (17). Since the onset of COVID-19 in Brazil, the federal government has also refrained from conducting an adequate and coordinated response to the pandemic, instead focusing on discourses and practices contrary to science-based recommendations (18, 19). In December 2020, Brazil had the second highest rate of the coronavirus worldwide (19), having accumulated a total of more than 34 million confirmed cases and 688,000 deaths by November 2022 (20). As of December 2022, there remain no national guidelines for the provision of outpatient care to people with COVID-19 following disagreements between Conitec and the Ministry of Health on this subject (21). Other health procedures that were unrelated to COVID-19 suffered drastic reductions in 2020 (19). This context may have been responsible for the negative impact on social participation in decisions at the federal level, as observed in this work for the years 2020 and 2021, with respect to both the decrease in confidence in the actions of the federal government, on which Conitec is based, and the dismantling of participatory movements for political and sanitary reasons.
Other PPI mechanisms that become available in the process of incorporating technologies into the SUS include i) demanders who focus on the adoption of technologies and ii) representatives of the “public” in the plenary of Conitec (2, 6). It was observed that the presence of “social representatives” in the position of demander remains incipient, representing only 4% (n = 17) of all requests analysed during the period. Conitec's receipt of a request for incorporation depends on the presentation of studies that exhibit a high degree of technical complexity in the form of systematic reviews and analyses of cost-effectiveness and budgetary impacts, which may represent an impediment to the ability of this group of stakeholders to access this process.
The representative of the “public” in the Conitec plenary holds a fixed appointment regardless of the topic under analysis and participates directly in the deliberative process involved in the task of evaluating each demand; this representative also has the right to vote. The nomination of this representative is freely chosen by the National Health Council, which represents users, health professionals and the private sector. That is, the person appointed to represent the “public” may have diffuse interests and may not be directly related to each demand. Thus, it is evident that the main mechanism for patients’ social participation is public consultations, which lead to high levels of engagement and allow patients to make themselves heard and to have important impacts on the decision at hand.
However, the question of whether the interests presented by patients reflect (exclusively) their own interests or whether patients can serve as spokespersons for other actors remains up for debate. The category of patients and family members referenced in this study grouped the contributions sent both individually and collectively through patient groups and associations. The volume of contributions sent separately by patient associations during the period under analysis corresponds to only 1.4% of the category of patients and family members or 0.7% of total contributions, and it was not possible to determine whether the content of contributions from associations and that of contributions from individual patients matched.
Patients' associations aim to take actions both in direct support of patients and their families by providing educational, health and emotional support as well as to articulate public policies to promote access to treatments and other support services, and in this context, strong appeals have been made via social media in recent years (22, 23). These associations are financed largely by the pharmaceutical industry and tend to reflect the positions of their sponsors (24, 25). Therefore, it is necessary to investigate not only the influence of the volume of participation in this context but also the manner in which the contributions of patients represent their interests and needs and the way in which the content thus presented impacts the corresponding decisions.
Additionally, other aspects that could enrich our understanding of this subject include the categorization of indications into rare diseases and nonrare diseases and the decisions made based on the cost of the technologies in question. The volume of contributions sent by patients in response to public consultations may reflect the prevalence of the health condition in question, and therefore, a differentiation based on the rarity of the disease may be necessary. On the other hand, greater engagement can be motivated by the adoption of technologies that families are unable to acquire directly for economic reasons.
Despite their frequently use as a PPI mechanism, public consultations are limited by their nature as a one-way form of communication between the public and decision makers. In accordance with the vision of patient-centred health, PPI practices are encouraged to focus more on dialog and to emphasize the involvement of all stakeholders at all stages of the decision-making process, rather than merely at the end of the process (26).
Although the rationale for PPI is becoming increasingly consolidated in the scientific literature, its operationalization by institutions that conduct HTA has continued to be challenging (8). Ivani & Dutilh-Novaes (2022) proposed a three-tiered model that can facilitate the analysis of the conditions under which public engagement would be successful in the context of epistemic exchange (26). According to these authors, the following factors should be considered: 1) the attention or exposure of the actors, in this case, the public institution and society; 2) mutual trust among actors; and 3) the form and content of the information included in the epistemic exchange (26).
If a decision regarding the allocation of resources or a recommendation for the incorporation of technologies are to be considered fair and reasonable, it is necessary not only to consider the methods that are used and the information that is analysed but also the stakeholders who are included in the process (8). However, Street et al. (2020) defended clear distinctions among stakeholders because these stakeholders represent different roles and interests, especially with regard to health decisions informed by HTA (7). In this work, we also found that different groups not only organize themselves and participate in different degrees but also have different influences on the corresponding decisions.