Patient Perspectives in Pharmaceutical Policy: Information and influence in the diffusion of new medicines

Today’s patients are vocal, informed and eager to participate in processes which affect them. This also applies to the pharmaceutical policy arena – and to debates around the research and development of new drugs, their access and use. Patients and consumers play varied roles in pharmaceutical policy. They represent peers in dedicated platforms hosted by health authorities, provide input on their preferences and experiences in medicines’ use, advocate for reimbursement of new therapies, and can also be the target audience of awareness campaigns and pharmaceutical advertising. There are many instances for patient involvement throughout a drug’s life cycle which also represent unique opportunities for research. This thesis presents an analysis of challenging issues in pharmaceutical policy from a patient perspective untangling how the dissemination of information across stakeholders influences the uptake of new pharmaceutical products. Within the realm of pharmaceutical policy, we have explored initiatives or practices which are intrinsically perceived as positive developments but that, after thorough examination, demand further monitoring and action. Three key points stand out. First, the importance of applying rigorous standards for drug approval and reimbursement as a corner stone for appropriate medicine use. Placing public interests at the core means aligning clinical research with public priorities, designing clinical studies that explore outcomes relevant to patients and respond to questions which are valid from a societal perspective. The introduction of comparative requirements at the time of market approval could encourage manufacturers to go being incremental innovation and to focus on therapeutic areas with limited treatment options, thus addressing more pressing health problems and meeting societal needs. Second, the need to ensure proper representation in regulatory and pharmaceutical policy debates. Bearing in mind that the formation of pharmaceutical policy at the EU level relies on a multi-stakeholder approach, it is imperative that all sponsorship sources be publicly disclosed, as well asthe intended purpose of the funding, its value and the proportion of organisational revenue it represents. There are strong reasons in favour of creating a “sunshine” law to cover pharmaceutical industry payments to patient-advocacy organizations at European level.This could be a first step to help disentangle the complexity of the relationship between patient groups and corporate sponsors. Third, there are gaps in regulation of pharmaceutical promotion, especially in addressing new forms of promotion disguised as educational activities, such as the use of key opinion leaders and disease awareness campaigns. While there are some overall positive trends in some countries more systemic fundamental changes are needed to address current gaps. These require either an improvement or a better enforcement of current regulations. Finally, the focus of pharmaceutical policies has been changing over the last years from product-driven procedures to patient-driven decisions. This transition did not come naturally for many stakeholders who traditionally did not see patients as their peers. This thesis aimed to unfold challenging developments in pharmaceutical policy while providing guidance for the future, recognizing medicines as social goods which are instrumental to public health and embedding values such as transparency and public health driven policy. Sitting patients at the table must be more than a window-dressing exercise or the ticking of a box. Real inclusion of patients and consumers in health policy entails a commitment to apply democratic principles ensuring that those more directly affected and concerned by decisions are heard and actively engaged in the processes leading to those choices, be it in research, policy-making or practice