High-Precision Therapies vs. Collective Impact: The Case of Hemgenix and National Productivity

In June 2025, the United Kingdom marked a milestone by becoming one of the first countries to publicly fund an ultra-high-cost gene therapy for hemophilia B: Hemgenix. With a list price of approximately USD 3.3 million per patient, the treatment is a one-time solution that, according to current data, keeps patients free from bleeding episodes for at least three years. The NHS (National Health Service) fully covered the treatment through the Innovative Medicines Fund, at no cost to the patient.

It is important to note that this cost structure and public reimbursement are specific to the United Kingdom. In the United States, where healthcare services are priced significantly higher and often reimbursed through private insurance, the cost of managing hemophilia B over a lifetime — or even accessing gene therapies — would likely be far greater.

The event generated justifiable excitement: it is a transformative therapy, based on a logic of gene replacement that could eliminate the need for frequent and costly chronic treatments. But it also raises an uncomfortable question from the perspective of public economics:

—Is this the best way to allocate healthcare budgets if the goal is to maximize a society’s productivity and growth?

Clinical Context: Who Can Receive Hemgenix?

To date, Hemgenix (etranacogene dezaparvovec) has only been evaluated and approved for adult males over 18 years of age with moderate to severe hemophilia B. The pivotal HOPE-B clinical trial, which led to the product’s approval, included patients aged 18 to 75 and showed sustained efficacy for up to three years. However, there are currently no registered clinical trials involving Hemgenix in pediatric populations or adolescents under 18.

Some institutions, such as the Children’s Hospital of Philadelphia (CHOP), are investigating gene therapies for hemophilia B in minors, but these involve different products and platforms from Hemgenix. In practice, this means that access remains limited to a narrow cohort of adults with years of accumulated treatment and already past their productivity peak.

This scenario opens a yet-undeveloped strategic angle: the possibility of extending clinical trials to pediatric populations. Clinically, this would improve quality of life from an early age, preventing irreversible joint complications, school absenteeism, and healthcare dependency. Economically, it would also mean substantially increasing the return on investment (ROI), since the productive potential of individuals who have not yet entered the workforce would be preserved.

Rather than treating accumulated damage at age 40, the root cause would be addressed from the outset, maximizing the health, economic, and social impact of the intervention.

Comparative Cost: What a Cure Really Costs

Treating 50 patients with Hemgenix requires a total expenditure of USD 165 million. In return, symptoms are controlled for three years in individuals who, for the most part, have already reached age 40 and have lived with partial disability for decades.

From a national productivity (GDP) perspective, these individuals have likely contributed less than average:

• A healthy person between ages 20 and 40 contributes around USD 980,000 to the UK’s GDP.

• A patient with severe hemophilia B would likely contribute only half: ~USD 490,000.

• Moreover, the state has invested up to USD 6 million to sustain their health through recombinant factors, hospitalizations, and ongoing care.

The per capita GDP estimates used here are illustrative and do not account for individual variability by sector, class, gender, or employment status, but are useful to indicate the general magnitude of impact.

Another critical and often underestimated factor must be added: patients with severe hemophilia B require decades of frequent blood product transfusions to survive. This not only represents a significant additional cost, but also carries cumulative risks of blood-borne infections, such as hepatitis C or HIV. Although safety measures have improved significantly, the risk is not zero, and the costs associated with treating these infections can be substantial over the patient’s lifetime.

In short: the net balance for a severe hemophilia patient at age 40 is typically -USD 5.5 million for the public system, not fully accounting for transfusion-related complications.

From this starting point, Hemgenix may be seen as a way to reduce long-term healthcare liabilities, but not as a generator of new economic growth. Even if the patient regains 100% functionality and works for an additional 15 years, the return in GDP terms would be USD 735,000—far from justifying the therapy’s cost on its own.

Redefining Healthcare Efficiency

This analysis does not aim to delegitimize the use of Hemgenix. In fact, if it avoids 15 years of conventional treatment, it already represents a structural saving. But it does compel a revision of the dominant narrative:

Not everything that is technologically astonishing is economically rational when the goal is to optimize collective well-being.

In a scenario of limited resources, healthcare prioritization must consider not only individual effectiveness but also structural impact. Curing 50 people with a rare condition is commendable. But changing the life course of 5 million children, women, and youth through simple, underutilized interventions could be transformational.

Moreover, if resources come from taxes paid by society at large, publicly funded innovative treatments should aspire not only to provide individual clinical relief but also to deliver tangible structural returns in public health and national productivity.

Final Reflection

The true value of a health policy lies not only in how much suffering it alleviates, but in how much it expands the possibility for an entire generation to grow up healthy, fulfilled, and productive.

Between genetic miracles and structural justice, perhaps the real breakthrough is not choosing between them, but designing policies capable of sustaining both in intelligent balance.

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