Sources of Innovation in Gene Therapies — Approaches to Achieving Affordable Prices

From the New England Journal of Medicine.

By Kerstin N. Vokinger, MD, JD, PhD, LLM; Jerry Avorn, MD; Aaron S. Kesselheim, MD, JD, MPH.

SUMMARY: This NEJM Perspective examines a fundamental contradiction in the pricing of gene therapies: their prices are exceptionally high, yet the key scientific innovations underlying their development were largely driven by academic and other publicly funded research institutions rather than by the pharmaceutical companies now charging for them. The authors argue that this disconnect — between the public sources of innovation and the private capture of pricing power — raises important questions about fairness and sustainability. The piece reviews how gene therapy innovations moved from academic laboratories into commercial pipelines and considers policy approaches that could achieve more affordable prices without undermining the incentives needed to continue translating research into treatments.

BACKGROUND: Gene therapies have been developed at exceptionally high prices. Their underlying scientific innovations were heavily driven by academic and other research institutions rather than the pharmaceutical companies that commercialized them.

KEY FINDINGS: Prices charged for gene therapies are exceptionally high even though key innovations were predominantly publicly generated. This creates a disconnect between the public sources of innovation and private pricing power.

IMPLICATIONS: Policy approaches are needed to achieve affordable gene therapy prices in ways that reflect the public investment underlying these innovations, without undermining legitimate commercial incentives to bring therapies to market.