Insights Blog

From Vision to Value: Leveraging Health Economics to Maximize the Translational Potential of Your Research

From Vision to Value: Leveraging Health Economics to Maximize the Translational Potential of Your Research

From Vision to Value: Leveraging Health Economics to Maximize the Translational Potential of Your Research

Insights from the Network

June 2025

By Jason Robert Guertin, Associate Professor, Faculty of Medicine, Université Laval, & Lucie Germain, Professor, Faculty of Medicine, Université Laval
Dr. Jason Robert Guertin
Dr. Lucie Germain

 

In late February 2025, the Stem Cell Network (SCN) hosted two webinars, one in French and one in English, in which we presented key health economic considerations which could help regenerative medicine researchers bring their innovations to patients.

The webinars were broken down into three sections: 1) introducing key concepts of health economic evaluations; 2) describing the expected life-cycle of regenerative medicine technologies through a hypothetical case study; and 3) illustrating, using the same hypothetical case study, how regenerative medicine researchers and health economic experts can work together to maximize the chance these technologies make it to market.

In this Insights blog, we provide a brief summary of these webinars and key take aways for the SCN community.

Key Concepts in Health Economic Evaluations

Health economic evaluations are often misunderstood by fundamental and clinical researchers. Yet, it is important that these researchers understand these evaluations as many public funding agencies ask that researchers not only assess a treatment’s effectiveness profile but its cost-effectiveness profile as well.

Briefly, an economic evaluation is a “comparative analysis of the costs and consequences of two or more possible options” and they aim to help determine if the adoption of a treatment by a healthcare system represents the best use of limited resources. Although various types of economic evaluations exist, each with their own strengths and weaknesses, cost-utility analyses (CUA) are commonly favoured in Canada and abroad when examining technologies that have long-term impacts on patients’ survival and/or quality of life. Consequences within a CUA are generally represented using quality-adjusted life years (QALYs) which both quantifies the amount of life gained and patients’ preference (assessed with the use of utility scores) for the health state(s) in which they spend it. Once researchers have assessed both the cost and consequences (i.e., the total number of QALYs gained) of treating patients with both their novel treatment (NTx) and the standard(s) of care (SOC), they can estimate the CUA’s result: i.e., the incremental cost-effectiveness ratio (ICER) of the new treatment versus its comparator(s).

equation

Though simple to estimate, a treatment’s ICER often fails to define if a new treatment provides enough benefits to justify its added cost (i.e., if it’s cost-effective or not); in such circumstances, one must oppose the resulting ICER with the Payer’s (or Society’s) willingness to pay threshold (Figure 1). Unfortunately, as mentioned in the webinar, no official threshold exists within Canada (nor in most countries), but health economic experts will commonly refer to the generally cited $50,000 to $100,000 per QALY gained as reasonable thresholds to aim for.

Co-Designing a Treatment to Account for its Economic Value

Following the introduction of key concepts, the remainder of the webinar focused on first explaining how regenerative medicine treatments go from benchside to bedside by referencing Health Canada’s Health Product Vigilance Framework (1) and how to integrate various economic inputs that will be required to assess their economic value.

As detailed during our talks, discovering a treatment is just the beginning of a long series of potentially complex and harsh steps (e.g., pre-clinical studies, regulatory authorizations by Health Canada, in patient clinical trials, market authorization). While fundamental and clinical researchers tend to recognize the importance of these various steps, they often fail to recognize the benefit of collecting valuable economic inputs alongside these.

In a sense this is understandable, researchers often overspecialize within sub-specialties and economic inputs (and epidemiological inputs) are rarely the main focus of those designing the regenerative medicine treatments. However, this needs to change since economic inputs that are relevant to the Canadian regenerative medicine space are rare and, those that do exist, will often be outdated.

In hopes of helping research teams address this issue, we highlighted various types of study designs and data sources that they could plan and utilize. Specific examples include assessing patients’ typical care pathways through epidemiological studies, early economic evaluations (also referred to as headroom analyses) of the proposed or hypothesized treatment (2), and cost analyses from various economic perspectives. Planning and conducting such work will be needed if we want to bring novel regenerative medicine technologies to patients. Furthermore, doing so earlier will likely allow us to obtain these inputs in a quicker and less costly manner than if we wait until after clinical trials have shown the treatment to be effective.

Breaking the Research Silos

In conclusion, these webinars and the points raised within them reflect our shared thoughts on the importance of the greater collaboration between regenerative medicine researchers and health economists. Although sometimes complex, especially as the jargon between both fields rarely align, we believe that the benefits that arise from these collaborations greatly outweigh any challenge that can be faced. Learning from each other help us co-design studies that will provide the data required to answer the questions we will eventually need to face.

Figure 1: Cost-effectiveness plane

Cost-effectiveness plane
Watch the full webinar here

 

References

  1. Health Canada. Health Product Vigilance Framework. Health Canada; 2012 September 12, 2012. Report No.: H129-12/2012E-PDF.
  2. Bubela T, McCabe C, Archibald P, Atkins H, Bradshaw SE, Kefalas P, et al. Bringing regenerative medicines to the clinic: the future for regulation and reimbursement. Regen Med. 2015;10(7):897-911.