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Doctors/Dr. TREMBLAY Gabriel

Dr. TREMBLAY Gabriel

Abstract Gabriel TREMBLAY - cohort 1

Thesis topic:

The Economic value in the decision making process for innovative health care products:

The case of orphan diseases and GENOMICS


Key words: Health economics, Health care, Decision-making, Value proposition, Economic value


Market access for innovative health care technologies is at the center of a dilemma. While stakeholders and groups wish for a greater access to novel health care treatments and technologies, the resources (e.g., capital, financial, economic, human, technological) that can be allocated to health care are limited in both private and public systems. Over the past few decades, the rising cost of health care has increasingly put pressure on health care payers to identify and choose technologies which generate the greatest value for patients. More recently, the recession of 2008 has constrained, on the one hand, private payers to keep health care premiums low, and on the other hand, public payers to reduce the burden of health care costs on tax payers. This has led either to an increased emphasis on the notion of value for money, or to the introduction of cost-saving initiatives. During the health care decision process, payers must weigh potential benefits and costs of new treatments for a specific patient population, while balancing the overall budget impact of new technology funding. In this emerging value-driven environment, the need to demonstrate the value of innovative products prior to getting payers’ funding is common practice and key to market access. Health care value can include the efficacy, safety, quality of life, or any other dimensions that have an impact on patient care. In addition, assessing the value for money of novel technologies is becoming an integral part of the value proposition, and is termed: economic value. Divergences on the definition and perspective of health care value amongst professionals can arise in the decision-making process as they may hold and have different priorities, incentives or personal values. This divergence in values, referred to as multirationality amongst the participants involved, can have important impacts on that decision-making process. Understanding the convergence and divergence of the value perspective held amongst decision-makers is therefore critical to truly understand what health care value based is on its underlying dimensions.

The recent interest of the health care industry for personalized medicine and genomics, in addition to massive investment to discover treatments for orphan or rare diseases, is expected to lead to the development of numerous novel technologies over the coming years. These technologies will be directed toward small subgroups of the overall patient population and it is to be expected these new treatment possibilities will be onerous. To ensure proper market access for these technologies, the health care value assessment process will need to be ready to evaluate both benefits and costs of these new products. Consequently, understanding differences in the value dimensions observed across these innovative fields (personalized medicine and genomics, orphan diseases) compared to modern medicine, will be key to properly demonstrate the overall health care value of these technologies.

An understanding of multirationality amongst professionals involved in that assessment process, could lead to a better match between the health care value assessment techniques and the value concept. Therefore, the goal of this research was to identify convergences and divergences in the value perspective held amongst different health care expert profiles (physicians and clinicians, health economists, health technology assessors, and market access experts and payers), health care settings (modern medicine, personalized medicine and genomics, orphan diseases), and a few countries (United States, Canada, United Kingdom). In addition, understanding the positioning and structuring of economic value within the overall health care value proposition is important to better examine the evolving role of the economic analysis in the health care value assessment process.

To capture the perspective of experts on health care value, the group concept mapping (GCM), a mixed methods-based approach introduced by Trochim (1989), was used to map the dimensions of health care value. The GCM relies on detailed qualitative data in addition to robust multivariate statistical techniques (multidimensional scaling analysis, agglomerative hierarchical cluster analysis). The GCM method as applied in this research includes in-depth interviews, survey design, data collection and analysis as main steps. GCM results in concept map representations, matching pattern diagrams and descriptive statistics on the perception of participants regarding various scales such as the relative importance of the health care value dimension across settings. The interpretation of the results can help draw conclusions regarding the convergence and divergence amongst professional profiles for the concepts identified.

The identification of convergences and divergences of the health care value concept helped generate eight main recommendations for health care value demonstrations and the payers’ decision-making process. First, the research allowed a better understanding of the health care dimension positioning, and generated data showing the clinical, patient impact, and burden of the disease clusters to be systematically the most important clusters of dimensions, reinforcing their relative importance at the core of the value proposition. Second, the research showed the relative absence of multirationality between the expert profiles and the strong presence of convergence, in addition to, third, a low bias of experts toward their own field of expertise (exposure effect). These conclusions show that strong societal values are present between health care decision-makers and outweigh the influence of a particular expertise or professional profile. Fourth, a stronger multirationality was detected between countries and, thus provided a weaker basis for applying the same value concept across different countries (transferability). Fifth, the research results also stressed that modern medicine, and personalized medicine and genomics share a common value concept, while the orphan disease setting was considered as a distinct one by expert profiles. Sixth, the research also reported that a systematic low importance was attributed to ethical considerations. A possible explanation for this result is that relatively more important ethical dimensions would be internalized in other dimensions that have an impact on equity or ethical consideration (e.g., efficiency). Seventh, the importance of budget impact analysis that includes all health care costs also proved to be highly rated in the value concept and outweighs other economic dimensions, including the value for money. Finally, the rich diversity of dimensions that composed the health care value concept proved to be core to the perception of value by experts. Hence, the value concept is perceived as multidimensional which indicated that a unique criterion can hardly be used to demonstrate the value of a product. Therefore, the sole use of the cost-per-QALY criterion is potentially not a perfect match for the value concept and when more criteria are considered. This may reflect the presence of other dimensions which should be included in the economic decision-making process.

A better understanding of health care value is paramount to decision-making in value-driven environments and allows for the identification of convergent values and of multirationality. The application of the knowledge generated in this research could allow for the development of new tools or bring adjustments to current value demonstration techniques, and decision-making processes, in order to better match the true health care value concept as perceived by decision-makers.