Gene therapy was a dream for a long period of time without concrete medical progress for patients with unmet needs. Since Mendel’s discoveries (1866), which jumpstarted a better understanding of epigenomics and translational medicine, a few manufacturers have been able to bring gene therapies to patients. Most of which achieved access in G7 countries. This has led to a growing number of gene therapies in the pipe utilizing a set of technologies (AAV Lentiviral vectors, CRISPR-Cas 9 technologies) aiming at achieving long-term remission for more than 100 different diseases. However, we have seen big public outcries on the cost of these medicines, as well as uncertainties on the viability of the business model for health plans and payers across rich countries. There is an emerging flexibility on value-based pricing and on recognizing the offset of expensive healthcare technology for severe conditions1, 2, 3. Health plans and governments will have to adjust their funding scheme and long-term real world data follow-up to allow patients to be treated in a safe and effective environment.
1. Améliorer la qualité du Système de santé et maîtriser les dépenses - ameli (2020) Assurance maladie. Available at: https://assurance-maladie.ameli.fr/sites/default/files/2020-07_rapport-propositions-pour-2021_assurance-maladie.pdf (Accessed: October 24, 2022).
2. Jönsson, B., Hampson, G., Michaels, J., Towse, A., Graf von der Schulenburg, J. M., Wong, O. (2018) “Advanced therapy medicinal products and Health Technology Assessment principles and practices for value-based and Sustainable Healthcare,” The European Journal of Health Economics, 20(3), pp. 427–438. Available at: https://doi.org/10.1007/s10198-018-1007-x.
3. Goodman, C., Berntorp, E. and Wong, O., on behalf of the International Haemophilia Access Strategy Council (2022) “Alternative payment models for durable and potentially curative therapies: The case of gene therapy for haemophilia A,” Haemophilia, 28(S2), pp. 27–34. Available at: https://doi.org/10.1111/hae.14425.