Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.
Advanced therapy medicinal products (ATMPs) are a heterogeneous class of medicinal products that by offering the potential of cure represent a paradigm shift in the approach of many life-threatening diseases. Although a common regulatory framework for ATMPs has been established in the EU, the health technology assessment (HTA) and financing decisions remain local. The aim of this article is to present an integrated analysis of the current status of the value judgment of ATMPs and the integration of ethical evaluation in the HTA process. It has been identified that approaching the specificities of ATMPs in terms of market access will require a broadening of the definition of value to be able to systematically capture elements of value not traditionally considered. Outcomes modelling will play an important role in the pricing and reimbursement of ATMPs, providing a way to bridge the gap caused by the absence of data from clinical studies or real-world data. Given the nature and disruptive consequences of ATMPs the assessment and adoption of these medicinal products raises important ethical questions, both at a policy and at society level that should be properly addressed. HTA can be made more transparent and reliable, and simultaneously promote robust and accountable decision making, by turning explicit the value judgments implicit in HTA. Ultimately, there should be no core conflict between ethical requirements and HTA in a scenario where the goal is to promote equity and access of patients to truly innovative therapies such as ATMPs, while assuring the sustainability of healthcare systems.
Reflection paper on classification of advanced therapy medicinal products, EMA/CAT/600280/2010 rev.1 (2015)
Jönsson, B., Hampson, G., Michaels, J., Towse, A., von der Schulenburg, J.M.G., Wong, O.: Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare. Eur J Heal Econ 20(3), 427–438 (2019) CrossRef
Juškevičius, J.: Classification and ethical issues regarding advanced therapy medicinal products. Med Ethics Bioethics 20, 2–7 (2013)
European Parliament and the Council of the European Union: Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004. Off J Eur Union 324, 121–137 (2007)
Kleiderman, E., Boily, A., Hasilo, C., Knoppers, B.M.: Overcoming barriers to facilitate the regulation of multi-centre regenerative medicine clinical trials. Stem Cell Res Ther 9(1), 1–9 (2018) CrossRef
European Medicines Agency. https://www.ema.europa.eu. Accessed Oct 31 2019.
European Medicines Agency. Orphan designation: overview. https://www.ema.europa.eu/en/human-regulatory/overview/orphan-designation-overview. Accessed Oct 31 2019.
Jørgensen, J., Kefalas, P.: Reimbursement of licensed cell and gene therapies across the major European healthcare markets. J Mark Access Heal Policy 3(1), 29321 (2015) CrossRef
Legault, G.-A., Béland, J.-P., Parent, M., Bédard, K., Bellemare, C.A., Bernier, L., et al.: Ethical evaluation in health technology assessment: a challenge for applied philosophy. Open J Philos 09(03), 331–351 (2019) CrossRef
Kristensen, F.B., Husereau, D., Huić, M., Drummond, M., Berger, M.L., Bond, K., et al.: Identifying the need for good practices in health technology assessment: summary of the ISPOR HTA council working group report on good practices in HTA. Value Heal 22(1), 13–20 (2019) CrossRef
Garrison, L.P., Jackson, T., Paul, D., Kenston, M.: Value-based pricing for emerging gene therapies: the economic case for a higher cost-effectiveness threshold. J Manag Care Spec Pharm 25(7), 793–799 (2019) PubMed
Refolo, P., Sacchini, D., Brereton, L., Gerhardus, A., Hofmann, B., Lysdahl, K.B., Mozygemba, K., Oortwijn, W., Tummers, M., van der Wilt, G.J., Wahlster, P., Spagnolo, A.G.: Why is it so difficult to integrate ethics in health technology assessment (HTA)? The epistemological viewpoint. Eur Rev Med Pharmacol Sci 20(20), 4202–4208 (2016) PubMed
Da Cruz Freire, J.E., De Medeiros, S.C., Neto, A.V.L., Júnior, J.E.M., Sousa, A.J.S., Rocha, A.J., et al.: Bioethical conflicts of gene therapy: a brief critical review. Rev Assoc Med Bras 60(6), 520–524 (2014) CrossRef
Hunt, S.: Controversies in treatment approaches: gene therapy, IVF, stem cells, and pharmacogenomics. Nat Educ 1, 222 (2008)
European Network for Health Technology Assessment (EUnetHTA). https://eunethta.eu. Accessed Oct 31 2019.
- Advanced therapy medicinal products: value judgement and ethical evaluation in health technology assessment
- Springer Berlin Heidelberg
The European Journal of Health Economics
Print ISSN: 1618-7598
Elektronische ISSN: 1618-7601