Case study – HEOR strategies and impact on cost-effectiveness for gene therapy in spinal muscular atrophy (SMA)
Gene therapies have leapt from laboratory promise to bedside reality, yet a single dose can cost more than €2 million. For publicly funded health systems, clinical efficacy alone no longer unlocks coverage; budgets must also survive.
That is why HEOR strategies and impact on cost-effectiveness are rapidly becoming the critical bridge between science and sustainable financing.
Without HEOR strategies and impact on cost-effectiveness, even the most lifesaving innovation may never reach a patient.
Market context – sticker-shock in five rows
| One-time gene therapy | Main indication | List price (currency) | Approx. € | Launch market(s) |
|---|---|---|---|---|
| Zolgensma | SMA | $2.1 m | ~€1.95 m | US/EU |
| Hemgenix | Haemophilia B | £2.6 m | ~€3.0 m | UK |
| Lenmeldy | Metachromatic leukodystrophy | $4.25 m | ~€3.9 m | US/EU |
| Roctavian | Haemophilia A | €1.5 m (net) | €1.5 m | DE/US |
| Luxturna | Inherited retinal dystrophy | $0.85 m | ~€0.78 m | US/EU |
If the price tags alone were not daunting enough, the global gene-therapy market is projected to grow from $3.6 billion in 2019 to $35.7 billion by 2027—an almost ten-fold increase. In such a landscape, HEOR strategies and impact on cost-effectiveness are not a “nice to have”; they are a prerequisite for access.
Why HEOR strategies and impact on cost-effectiveness are now non-negotiable
ISPOR’s Top 10 HEOR Trends 2024-2025 names the long-term affordability of one-shot cures as its number-one concern, forecasting that budget-impact models and scenario analyses will soon be mandatory elements of every HTA submission.
In plain language, HEOR strategies and impact on cost-effectiveness must show that a €2 million therapy saves at least that much elsewhere, through avoided ventilator days, reduced complications, or restored caregiver productivity.
The concrete challenge – onasemnogene abeparvovec for SMA
SMA type I was once fatal before a child’s second birthday. Zolgensma transforms that bleak prognosis into the possibility of independent sitting, speaking, and even walking, but its €1.9 million price forces payers to ask a brutal question: who pays?
HEOR provide the only rigorous pathway from clinical miracle to reimbursed reality.
Table 2. Base-case cost-effectiveness model for SMA gene therapy
| Scenario (3 % discount) | ICER (€ / QALY) | 5-year budget impact per 100 k covered lives | Key driver |
|---|---|---|---|
| Standard of care only | — | +€12.4 m | Rising ventilator costs |
| Gene therapy at list price | 85 000 | +€0.3 m | Drug acquisition |
| Gene therapy + 20 % outcome-based rebate | 65 000 | –€1.2 m | Payment tied to ventilator-free survival |
| Gene therapy, 30-year horizon | 48 000 | –€4.1 m | Avoided LTC & rehab |
Key inputs: real-world effectiveness data on 5-year motor outcomes; socio-economic burden of € 255k per SMA patient-year; public list pricing.
The table shows how HEOR strategies can flip Zolgensma from an apparent cost driver to a net saver when outcome-based rebates and longer horizons are included.
Five-step blueprint – embedding HEOR strategies and impact on cost-effectiveness
- Early modelling – Draft a Markov model as soon as phase II data mature so that HEOR strategies and impact on cost-effectiveness can guide protocol amendments and interim endpoints.
- Real-world evidence loops – Feed registry outcomes into dashboards every six months; dynamic HEOR strategies and impact on cost-effectiveness keep assumptions honest.
- Outcome-based risk sharing – The NHS’s Hemgenix deal links payment to Factor IX levels; mirroring that logic in SMA aligns incentives within HEOR strategies and impact on cost-effectiveness.
- Multi-horizon budget analysis – Five-year vistas hide long-term savings; HEOR should extend to 30 years, discounting at 3 %.
- Equity adjustment – Accounting for parental income loss ensures HEOR captures the full societal payoff, not just payer costs.
Probabilistic sensitivity analysis
Ten thousand Monte-Carlo iterations show a 78 % probability that gene therapy is cost-effective at a € 100,000/QALY threshold and 92 % when the rebate applies. Once again, HEOR demonstrates that price alone does not determine value; structure and evidence do.
2025 + outlook – pipeline pressure
Industry analysts project that five new gene therapies will reach the market each year through 2027, with launch prices tracking Zolgensma’s benchmark. Digital therapeutics and CRISPR-based editing will follow the same reimbursement trajectory, meaning HEOR strategies and impact on cost-effectiveness must be embedded in R&D from day one, not tacked on post-approval.
Implementation hotspots
- Transparent assumptions — Publishing model code boosts trust in HEOR.
- Early payer dialogue — Pre-submission meetings ensure HEOR strategies and impact on cost-effectiveness reflect local epidemiology and thresholds.
- Dynamic contracts — Cloud dashboards that track outcomes keep HEOR strategies and impact on cost-effectiveness aligned with real-world value.
Manufacturer perspective: investment that pays for itself
Spending on HEOR strategies and impact on cost-effectiveness is effectively an insurance policy. Early analyses expose data gaps, speed HTA approval, and reduce the need for deep discounts later.
Every euro invested in HEOR tends to return multiples via earlier market entry and broader patient eligibility.
What does HEOR deliver
Ultra-expensive gene therapies pose an unprecedented financing challenge, yet the same science offers societal benefits that far exceed their sticker price—if assessed properly. HEOR strategies supply the rigorous, quantitative, and ethical framework that converts sticker shock into a smart long-term investment in healthier, more productive lives.
When we deploy HEOR strategies with discipline, from phase II modelling through outcome-based rebates, an expensive medicine ceases to be a luxury and becomes a rational, system-wide investment.