Beyond direct health outcomes: The wider societal impact of innovative treatments

A common misconception is that evaluating societal and environmental outcomes demands an entirely separate analytical approach. In practice, the same modeling backbone used for standard health economic evaluation can accommodate these expanded outcomes within a single, consistent framework. Decision trees, Markov models, partitioned survival models, and discrete event simulations all lend themselves to the inclusion of clinical, economic, and societal endpoints as the core approach remains unchanged; only the outcome set is broadened.

Haemophilia A is a rare, inherited bleeding disorder caused by a deficiency or dysfunction of clotting factor VIII. Patients experience prolonged and excessive bleeding following injuries or surgeries, spontaneous bleeding episodes, and, in severe cases, life-threatening complications. The burden on patients and caregivers is substantial, encompassing the demands of administering frequent factor replacement, the emotional toll of constant vigilance against unpredictable bleeding events, significant financial strain due to the high cost of treatment, disruption to family routines from frequent medical appointments and emergency visits, and the ongoing responsibility of coordinating care across multiple specialists. This means haemophilia A profoundly affects not only patients but also the families and caregivers who support them.

A recently published study that we performed looked at the specific impact for patients and caregivers of obtaining their treatment. Until recently, patients in France could only access haemophilia A treatments at hospital pharmacies, requiring frequent travel. Our study found that this process resulted in the loss of 3 workdays, about €300, and 52 tons of CO₂ emissions per year per patient or caregiver. Though these numbers may seem minor, they can consume up to 2.6% of a family's disposable income, adding financial strain to obtaining necessary medication. The study also found that hospital-only access increased geographical inequalities for patients living far from the hospital in rural areas. Aware of these challenges, a leading haemophilia A drug maker partnered with hospitals and community pharmacists to offer their treatment at local pharmacies, easing access for patients and caregivers. Our study showed that these changes could substantially reduce the dispensation burden, giving families back time and money that they spend on the disease, while reducing the carbon footprint linked to transportation to and from the hospital. The innovative treatment's properties allowed for this shift in dispensing, resulting in measurable societal and environmental benefits relevant to policy.

Influenza causes, on average, over one million primary care consultations, 20,000 hospitalisations, and 9,000 deaths annually in France. Adults aged 50 to 64 years face elevated risks due to increasing chronic comorbidities, yet current vaccination recommendations begin only at age 65. We developed a susceptible, exposed, infectious, recovered (SEIR) compartmental model to evaluate the health and economic consequences of expanding vaccination to all adults aged 50 years and over (Béraud et al., 2025). In addition to healthcare resources and costs, we examined how influenza directly affects sick leave among working adults aged 50 to 64. We focused on sick leave frequency and duration, employment rate, and average daily economic output. 

The model demonstrated that achieving vaccination coverage among individuals aged 50 to 64 years, comparable to the current rates observed in the 65+ group, could annually prevent over 500,000 general practitioner consultations, 9,000 hospitalisations, and approximately 3,000 deaths. Evaluating cost-effectiveness from the payer perspective indicated that this approach is highly efficient, yielding an incremental cost-effectiveness ratio of €1,496 per quality-adjusted life-year (QALY). However, when accounting for the impact of reduced sick leave, this vaccination strategy resulted in substantial productivity gains (estimated at more than €300 million per year), thereby establishing it as a dominant strategy from a societal perspective. This represents a much higher value than just being cost-effective. Further work is being done to assess the additional environmental impact of this strategy. With a hospitalisation representing about 43 kg of CO₂ emissions, reducing thousands of hospitalisations per year could also be associated with a major reduction in CO₂ emissions.

This case demonstrates how restricting an evaluation to direct medical costs can substantially understate the true value of an intervention, and how adopting a societal perspective can transform the policy narrative.

Innovative treatments can generate value that extends across the full patient care pathway, with measurable societal and environmental consequences well beyond clinical endpoints. These impacts are increasingly quantifiable through robust health economic modeling, enriched by qualitative evidence that captures burdens overlooked by conventional assessments. The analytical framework is neither novel nor burdensome; rather, it builds on established modeling architectures, expanded to accommodate a broader, more realistic set of outcomes.

As it becomes more challenging to differentiate interventions based solely on clinical benefits, conducting thorough societal and environmental impact assessments provides a credible way to set innovations apart, making these factors central to the value proposition rather than secondary. Highlighting this differentiation and enhanced value is crucial to ensure that pricing and access align with the true value of the innovation.