3 March 2020
Cell and gene therapies are among the more promising innovations to treat cancer. Chimeric Antigen Receptor T-Cell (“CAR T” or “CAR T-cell”) treatments involve a combination of procedures that take place both in and outside of the body. Blood is taken from a patient, and T cells (a type of immune cell) are isolated and then modified, typically using a virus. The modified CAR T cells are cultured and multiplied, and a few weeks later, re-infused into the patient. The CAR T cells then bind to an antigen on the cancer cells and kill them. Several patents have been granted for CAR T therapies, but some governments are exploring the degree to which the technology is excluded from patentability. There are several problems associated with the CAR T monopolies. In June 2018, the European Medicines Agency recommended marketing authorizations for two CAR T therapies: 1) Kymriah (marketed by Novartis) – indicated for the treatment of acute lymphoblastic leukaemia, and 2) Yescarta (marketed by Gilead) – indicated for the treatment of diffuse large B-cell lymphoma. In Germany, Novartis introduced Kymriah at the price of 320,000 Euros. In Spain, the Hospital Clínic de Barcelona paid a unit price of 313,920 Euros per patient for Yescarta in 2019.
In the field of gene therapy, Zolgensma has emerged as a striking example of the dystopian future that confronts us now. The Novartis gene therapy Zolgensma is approved for the treatment of spinal muscular atrophy (SMA); Zolgensma costs 1.9 Euros million per patient. The early development of Zolgensma was funded by the US National Institutes of Health (NIH), the French charity Généthon, and US-based charities including Sophia’s Cure, Getty Owl Foundation, Fighting SMA, Jason’s Dream Foundation, the Gwendolyn Strong Foundation, and Miracle for Madison.
In light of the rise of these catastrophic health expenditures that drain health budgets, it is our view that the European Union may never achieve universal health coverage without the delinkage of R&D costs from the prices of drugs, vaccines and other health technologies.
To achieve the goals of reducing cancer incidence and mortality rates as well as expanding access to life-saving cancer treatments, we recommend that the European Commission takes specific actions and fine tune cost saving initiatives that do not involve the rationing of life saving treatments. To accomplish successful steps towards the delinkage of R&D cost from the price of drugs, the Commision should conduct a feasibility study to explore new market entry rewards as the incentive for development of new CAR-T and other cell and gene therapies, which would operate in countries where the patentability of such treatments will not be granted.
Furthermore, the Commission should create a working group to examine the implications of Article 27.3(a) of the WTO TRIPS Agreement on the patentability of gene and cell-based therapies including those involving autologous T-cell immunotherapy (CAR T), CRISPR and other technologies when treatment can be described as a service or a medical procedure, rather than a product. The working group should explore if certain cell and gene-based treatments are, in fact, exempt from patentability when a country has an exception that mirrors Article 27.3(a) of the TRIPS Agreement.
Since EU actions must be based on evidence and understanding of the impacts of regulations, we request that the Commission require the following information regarding cancer therapies be collected and made public:
a) annual reports on sales revenues, prices and units sold;
b) annual reports on marketing costs incurred for each registered product or procedure;
c) the costs directly associated with each clinical trial used to support the marketing authorization of a product or procedure, separately;
d) all grants, tax credits or any other public sector subsidies and incentives relating to the initial regulatory approval and annually on the subsequent development of a product or service.