Paper short abstract:

Drug exceptional access programs (DEAPs) in Canada to help address access gaps to pharmaceuticals. They entail circumventing the standard procedures which raises economical, social and political issues. This presentation helps understand DEAPs’ procedures and associated issues.

Paper long abstract:

Drug exceptional access programs (DEAPs) in Canada to help address access gaps to pharmaceuticals. They entail circumventing the standard procedures which raises economical, social and political issues. This presentation helps understand DEAPs’ procedures and associated issues.

Recent pharmaceutical industry development towards immunotherapies, gene therapies and more broadly personalized medicine have led these drug exceptional access programs (DEAPs) to play a key role, allowing access to personalized treatments under conditions not yet approved, thus creating a form of parallel market. The programs and dynamics surrounding exceptional access to pharmaceuticals are under documented. How do these programs work in practice? What are the health and political issues generated by DEAPs in the era of gene therapies, precision medicine, and ultra-expensive therapies? Addressing these issues requires delving into the links between pharmaceutical exception programs and the new economic and scientific dynamics of pharmaceutical development.

Paper short abstract:

The high costs and social repercussions related to oncology drugs highlight the necessity for a thorough examination of the choices and biases in pharmacoeconomic studies. This study aims to analyse reports from INESSS to understand the factors defining these methodological choices and decisions.

Paper long abstract:

Over the last decade, many authors have highlighted significant methodological discrepancies found in health technology assessments (HTA) regarding oncology drugs. These variations persist in the uncertainty of therapeutic value and in the assessment of quality of life, among others. As HTA agencies advise policymakers on reimbursement decisions, this raises significant concerns regarding the choices and biases inherent in both the pharmacoeconomic analyses carried out by manufacturers and the decisions made regarding HTAs.

The case of Québec is noteworthy, as the province spent $1.2 billion on oncology drugs in 2021 through its public insurer program, the Régie de l'Assurance Médicament du Québec (RAMQ). The province also possess its own HTA agency, the Institut National d'Excellence en Santé et Services Sociaux (INESSS), which operates separately from agencies in other canadian provinces. The significant rise in innovative treatments and associated expenses underscores the importance of analyzing Quebec's pharmacoeconomic practices in oncology. The critical consideration lies in whether these high costs genuinely reflect innovation or primarily serve to generate profits.

The examination of methodological choices and methodologies in health technology assessments, particularly in oncology, emphasises the importance of understanding factors contributing to the high costs of innovative therapies, as demonstrated by Quebec's substantial expenditures on oncology drugs. This study's objective is to examine publicly available reports from Québec's INESSS, to understand, describe, and to analyze factors shaping methodological choices andthe decision-making process of a HTA agency.

Paper short abstract:

From 1975, independent Mozambique adopted a pharmaceutical policy to rationalise drug provision and purchasing, by restricting drugs to 300 products, with generic names only. This paper will revisit the Mozambican experience to address if it could be a model for current Global South health policies.

Paper long abstract:

When Mozambique achieved independence in 1975, health was among the main priorities of the new socialist government. The provision of pharmaceutical products went hand in hand with nationalisation and socialisation of medicine to bring health care to the people. A Technical Commission on Pharmacy and Therapeutics was set up two months into independence to rationalise the drug provision. Its first mission was to comb through the 13,000 drugs previously registered in Colonial Mozambique during Portuguese domination. However, the country had only one graduated pharmacist and a limited health budget. Thus, rationalisation aimed to select a restricted list of drugs by combining both medical and economic reasoning, adopting mandatory generic nomenclature and creating a new system of provision of drugs for the country. The health authorities in Mozambique also got engaged at the WHO and other fora, actively participating in the debates of that period on Essential Medicines Lists and advocating technical cooperation to foster local production of drugs in the Third World. In the early 1980s, several journal publications and book chapters authored by Mozambican health authorities and foreign medical internationalists working in Mozambique promoted the success of these policies in achieving substantial price reductions for medicines and increasing the volume of purchases, with only 300 drugs selected. This communication will interrogate if at the time Mozambique became a model on pharmaceutical policies for low-income countries, and how this experience could be revisited regarding the current challenges on health provision for the Global South.

Paper short abstract:

Analysis of the deficits in reasonableness experienced when approving or refusing exceptional access to drugs for individual patients in Quebec. Here, an epistemic concept of democratic reasonableness is used as a heuristic to reveal issues of fairness at the systemic/programmatic level.

Paper long abstract:

This communication will present an analysis of the deficits in reasonableness experienced when approving or refusing exceptional access to drugs for individual patients in Quebec, under a specific regulatory statute called "particular medical necessity" (PMN). As elsewhere, Quebec is under pressure as drug therapies target ever more specific conditions while marketed at significant costs.

Here, the implications of this pressure are examined at a specific locus, as the NMP mechanism has become a vehicle by which the traditional logic of regulating access through indications gives way to a logic of judging access on a case-by-case basis. Together, local NMP committees bear the weight of decisions with major ethical and societal implications.

In examining the results of research carried out with NMP committees, we will use an epistemic concept of democratic reasonableness, which locates the anchoring of reasonableness within the intellectual practices that enable NMP committees to appreciate the desirability of responding to an access request relative to other requests. Hence, the distinctiveness of this normative approach is as a heuristic to reveal issues at the systemic/programmatic level. Our analysis will show how the difficulties experienced by NMP committees in making decisions they deem fair and reasonable go beyond classic issues of evaluation methodology, as they also point to structural origins (e.g., incoherent epistemic basis for defining the ‘particular’, proposed drug uses without an institutionalized path to access, etc.). This analysis will contribute to the international discussion on more integrated and reasonable access to drugs within the current pharmaceutical paradigm shift.