Estimated Spending on Beremagene Geperpavec for Dystrophic Epidermolysis Bullosa.
Level 4 - case-series / case-control
Economic evaluation and budget impact simulation model based on registry data (graded by design analogy).
PubMed 38294784 · doi:10.1001/jamadermatol.2023.5857
What was done
This economic evaluation modeled projected US healthcare spending on beremagene geperpavec (B-VEC), a topical gene therapy approved by the US FDA for both autosomal recessive and autosomal dominant dystrophic epidermolysis bullosa (DEB). Using prevalence data from the National Epidermolysis Bullosa Registry, the authors estimated the eligible US patient population and projected total spending over 1-year and 3-year periods from an all-payers perspective, assuming a base-case cost of $300,000 per patient per year, alongside sensitivity analyses and per-patient lifetime cost estimations.
What was found
An estimated 894 US patients with DEB were eligible for B-VEC in the first year post-approval. Projected spending figures included: - Total 1-year US spending: $268 million (range, $179 million–$357 million). - Total 3-year US spending: $805 million (range, $537 million–$1.1 billion). - Lifetime cost per patient: $15 million (range, $10 million–$20 million) for autosomal recessive DEB and $17 million (range, $11 million–$22 million) for autosomal dominant DEB.
Why it matters
The broad FDA approval of B-VEC for both autosomal dominant and recessive DEB—despite limited trial data in dominant disease—is projected to create a substantial financial burden on healthcare payers, reaching hundreds of millions of dollars within years of approval.
Limits
This was an economic projection model, not an empirical analysis of observed healthcare claims or expenditures. Calculations rely on registry-based prevalence estimates and an assumed manufacturer base price ($300,000/year), without accounting for real-world rebates, treatment adherence, discontinuation, or comparative clinical cost-effectiveness.
Cited by
- supports Gene therapies and palliative care for rare genetic diseases typically cost millions of dollars per person over a lifetime, whereas preconception genetic carrier screening costs in the low hundreds of dollars.