OBJECTIVES: In the past few years cell and gene therapies have started to receive regulatory approval in the US and EU, with a substantial number of additional therapies in development. Many of these therapies have the potential to provide transformative benefits for patients in terms of extension of life and improved quality of life, though there is uncertainty about long term effects. Herein we compared estimated health gains for cell and gene therapies with estimated health gains for drugs and biologics.

METHODS: We identified published incremental health gain estimates measured in terms of quality adjusted life-years (QALYs) for drugs and biologics approved from 1999 through 2012 by searching the PubMed database. We limited attention to cost-utility and comparative effectiveness studies that evaluated treatments with alternative therapeutic approaches available at the time of FDA approval. We identified published incremental health gain estimates for cell and gene therapies by searching the PubMed database and HTA agency websites. Because of the nascent nature of cell and gene therapies, we included incremental health gain estimates without regard to publication date. In all cases, we excluded studies funded by product manufacturers. QALY gains for cell and gene therapies were then compared to corresponding gains for drugs and biologics using the Mann-Whitney U test.

RESULTS: We identified QALY gain estimates for 104 drugs, 31 biologics and 8 cell or gene therapies. Cell and gene therapies included treatments for hemophilia, spinal muscular atrophy, oncology, inherited retinal disease, and other genetic diseases. QALY gains for cell and gene therapies (median 3.39; interquartile range [IQR] [1.64–6.70]) exceeded corresponding QALY gains for drugs (median 0.05; IQR [-0.002–0.26]) and biologics (median 0.07; IQR [-0.001–0.37]) (p<0.01).

CONCLUSIONS: Our study suggests that, to date, cell and gene therapies offer substantially greater health gains than do conventional drugs and biologics.