5 Topic Commentaries

Roving Reporter Asks: How Should We Redefine Value, Access, and Affordability in Cell and Gene Therapy?

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  Peter Marks, MD, PhD

  Hematology/Oncology; Regulatory Science

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  U.S. Food and Drug Administration

  manufacturers, particularly small ones, can't afford to make additional doses of their therapies.

  Source
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  Gary H. Gibbons, MD

  Cardiology

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  National Heart, Lung, and Blood Institute (NHLBI), National Institutes of Health

  The cost of gene therapies is concerning: by some estimates they could be as high as $2 million per patient.

  Source
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  Julie A. Panepinto, MD, MSPH

  Pediatric Hematology/Oncology

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  National Heart, Lung, and Blood Institute (NHLBI), National Institutes of Health

  the cost of one-time gene therapy is likely to be cost-effective.

  Source
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  Holly Tabor, PhD

  Bioethics

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  Stanford University School of Medicine

  The high costs of gene-targeted therapies pose additional ethical challenges related to access and allocation of scarce resources, said Tabor.

  Source
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  Philip O. Ozuah, MD, PhD

  Pediatrics; Health System Leadership

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  Albert Einstein College of Medicine / Montefiore Medicine

  the cost of the treatment, which is likely to be more than $1 million per patient

  Source