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Benefit Corporation: a path to affordable gene therapies?

Nature Medicine volume 25, pages 1813–1814 (2019)Cite this article

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With the growing number of efficient gene therapies on the market, now is the time to take actions to ensure reasonable pricing of gene therapy products. Among these, we propose to incentivize gene therapy companies to adopt a status that translates their corporate social responsibility into concrete commitments.

Pia, a 9-month-old baby with spinal muscular atrophy, was the first patient to receive Zolgensma gene therapy in Belgium. The product was injected on 9 October 2019, 3 weeks after an unprecedented crowdfunding campaign that raised €1.9 million in a few days. It was a last-resort initiative launched by Pia’s parents after the Belgian health payer denied reimbursement because the product was not yet approved by the European Medicines Agency (EMA). Doctors of another Belgian child with adenosine deaminase deficiency (ADA-SCID) have not succeeded in obtaining reimbursement for EMA-approved Strimvelis, the gene therapy product that represents the best possible therapeutic option for this child. Such situations will be faced by increasing numbers of patients who could benefit from either ex-vivo retrovirally mediated or in vivo adeno-associated viral vector–mediated gene transfer1. The number of products based on gene therapy on the market or in the pipeline—for the treatment of common diseases such as β-thalassemia, sickle cell disease and hemophilia, or autologous chimeric antigen receptor (CAR) T cells for leukemia and lymphoma—continues to grow. However, the cost of these potentially lifesaving therapies is prohibitive for most patients, and this is true on a worldwide scale, regardless of the healthcare system (Table 1). Indeed, although the European Union preceded the United States in approving the first gene therapies, it has yet to put a system in place to provide affordable access to these treatments. The clinical applications of genome editing relying on engineered nucleases or base-editing enzymes that are under development will most likely raise the same issues.

Table 1 Approved gene therapy products in the US and Europe
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Authors and Affiliations

  1. Collège de France, Assistance Publique des Hôpitaux de Paris, Hôpital Necker Enfants Malades, Unité d’Immunologie-Hématologie Pédiatrique, and Imagine Institute, INSERM UMR 1163, Paris, France

    Alain Fischer

  2. Ecares, Solvay Brussels School of Economics and Management, Université libre de Bruxelles, Brussels, Belgium

    Mathias Dewatripont

  3. I3h Institute, Université libre de Bruxelles, Brussels, Belgium

    Mathias Dewatripont & Michel Goldman

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  1. Alain Fischer
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  2. Mathias Dewatripont
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Correspondence to Alain Fischer or Michel Goldman.

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Fischer, A., Dewatripont, M. & Goldman, M. Benefit Corporation: a path to affordable gene therapies?. Nat Med 25, 1813–1814 (2019). https://doi.org/10.1038/s41591-019-0676-z

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