The True Price of Gene Therapy: Breaking Down Beqvezโs $3.5M Cost and What Really Drives It
Scott Jeffers, Chief Technology Officer at GenSight Biologics, has shared a post on LinkedIn:
“The Real Cost of Gene Therapy Development: Beqvez as a Case Study
Iโm often asked about the โoutrageousโ price of gene therapies. Letโs look at the real cost structure.
Beqvez (Pfizer’s hemophilia B gene therapy) launched at $3.5M.ย Hereโs where that money actually goes:
๐ ๐ฎ๐ป๐๐ณ๐ฎ๐ฐ๐๐๐ฟ๐ถ๐ป๐ด ๐ฅ๐ฒ๐ฎ๐น๐ถ๐๐
Per batch cost (including QC): ~$2.0M
Drug Substance testing: $172K
Drug Product release: $123K
In-process testing: $135K
Stability testing: $227K
QC alone: $657K
Yes, nearly $700K per batch โ and still not the biggest driver.
๐ง๐ต๐ฒ ๐๐ถ๐ด๐ด๐ฒ๐ฟ ๐ฃ๐ถ๐ฐ๐๐๐ฟ๐ฒ
Total development cost: ~$316M
Clinical trials: $78M (25%)
Preclinical: $58M (18%)
Post-approval surveillance: $160M (51%)
CMC/Regulatory: $16M (5%)
Manufacturing per dose: $0.75M (<1%)
๐ง๐ต๐ฒ ๐ฆ๐๐๐ป๐ป๐ถ๐ป๐ด ๐ง๐ฟ๐๐๐ต
Manufacturing + CMC = <6% of total costs.
The real spend is in:
Demonstrating safety & efficacy
15+ years of patient monitoring
Regulatory navigation
Building evidence for a novel therapy
๐ช๐ต๐ ๐๐ ๐ ๐ฎ๐๐๐ฒ๐ฟ๐
Pricing debates often blame manufacturing, but the economics come from:
Replacing $300Kโ$1M annual treatments with one-time interventions
Decades of risky R&D
Transformative patient outcomes
Note: Beqvez was discontinued in early 2025 โ proof of how tough this market is, even when the science works.
๐๐ฒ๐ ๐ง๐ฎ๐ธ๐ฒ๐ฎ๐๐ฎ๐๐
Manufacturing optimization wonโt fix pricing.
Clinical efficiency & regulatory innovation = biggest ROI.
Post-market surveillance costs are underestimated.
Platforms matter when manufacturing is <6%.
๐ช๐ต๐ฎ๐โ๐ ๐๐ผ๐๐ฟ ๐ฒ๐ ๐ฝ๐ฒ๐ฟ๐ถ๐ฒ๐ป๐ฐ๐ฒ? Are you seeing the same patterns?”
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