In the coming years, a significant increase is expected in the number of extremely expensive cancer treatments that are approved for marketing. National public healthcare systems encounter difficulties in arranging or even justifying reimbursement of these treatments using collective funds. For a research project funded by the Dutch Cancer Society, we are examining ethical dilemmas that arise when newly approved expensive cancer treatments have received marketing authorization but are not (yet) reimbursed within the national healthcare system. When patients could possibly benefit from non-reimbursed cancer treatments, physicians and patients might try to arrange access to these treatments in other ways, such as asking health insurers or pharmaceutical companies for leniency, requesting the use of hospitals budgets, or letting patients pay out of pocket. Based on differing weighing of values at stake, such as beneficence, liberty, autonomy, solidarity, and justice, physicians may make different choices with respect to pursuing such routes to seek access or supporting patients in doing so. Variability in practices among individual doctors or institutions could potentially lead to unequal access to expensive medical treatments for patients. While in many European countries, equal access to healthcare is widely seen as highly valuable, denying patients to make out-of-pocket payments interferes with their liberty. In this symposium, we discuss ethical dilemmas of access to and funding of extremely expensive medical treatments that are not (yet) reimbursed under national health insurance schemes.
Programme: the symposium will start with a brief introduction by the Chair (2 min), and two short presentations (8 min) to set the scene. Then, there will be three more extensive presentations (15 min). We will have 25 minutes for a discussion with participants in the audience, moderated by the Co-Chair.
Stakeholders´ perspectives on ethical issues regarding access to expensive cancer treatments: an interview study in the Netherlands
Charlotte Bomhof | Erasmus MC | Netherlands
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Charlotte Bomhof | Erasmus MC | Netherlands
While legally, physicians may prescribe treatments that are approved but not (yet) reimbursed in the Netherlands, an interview study we are currently conducting among physicians suggests that perspectives of physicians on access to non-reimbursed cancer treatments differ greatly. Little is known about current practices and policies regarding access to non-reimbursed treatments in the Netherlands, and it is not clear whether or not patients are allowed to make out-of-pocket payments to access cancer treatments, and on what grounds. In this symposium, we will present the result of an interview study on the perspectives of stakeholders, including policy-makers, hospital managers, health technology assessors, representatives of pharmaceutical companies and patient organizations on practices and policies regarding access to non-reimbursed cancer treatments in the Netherlands. It is important to gain more insight into stakeholders’ perspectives to be able to design fair and fitting models for access to effective but (too) expensive cancer treatments in the future.
Ethical analysis of access and reimbursement models for CAR-T cell therapy Yescarta
Dr. Jilles Smids | Erasmus MC | Netherlands
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Dr. Jilles Smids | Erasmus MC | Netherlands
CAR-T cell immunotherapy is a clear example of an extremely expensive personalized medicine approach in oncology. One CART-T cell therapy, Yescarta, is a treatment for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), two aggressive subtypes of non-Hodgkin lymphoma, after 2 or more lines of systemic therapy. Yescarta is an extremely expensive (€327.000 for a one-off treatment), yet effective therapy that is potentially curative for some patients and has an estimated median survival gain of 11 months. Yescarta was not immediately reimbursed by Dutch health insurers after approval by the European Medicines Agency in 2018 (Zorginstituut Nederland 2019). Following price negotiations with the manufacturer, from May 2020 onwards, Yescarta is currently reimbursed for the indications mentioned until 31-12-2021. In this case study, we will report on the various modes of access and funding of Yescarta that have been employed by patients and physicians during the period in which the treatment was not being reimbursed under standard health insurance. Furthermore, we will provide a normative analysis of these modes’ implications for the moral values of beneficence, liberty, autonomy, solidarity and justice. We will identify the value conflicts and discuss the ways in which they might be resolved.
Opening the backdoor to public funding for rich people or providing necessary and effective care? A case challenging epistemology and ethics
Prof.dr. Bjørn Hofmann | University of Oslo | Norway
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Prof.dr. Bjørn Hofmann | University of Oslo | Norway
Hofmann has written extensively on the role of ethics of Health Technology Assessment (HTA) and on methodological approaches to ethical assessment within HTA. When reimbursement decisions have been made in ways that are procedurally sound, and ethical aspects have been adequately addressed, should there be room in universal healthcare systems for individual patients to pay for expensive cancer treatments out of pocket? In Norway, societal commitment to the value of equity (and a general hesitance towards allowing for privately funded healthcare) may be stronger even than in the Netherlands. In this symposium Hofmann will present a case of expanded access in Norway, and discuss how the use of privately paid investigational treatments ends up getting reimbursed by health insurers, posing problems for priority setting in solidarity- and equity-based healthcare systems.
Does solidarity have a place in personalized medicine?
Prof.dr. Barbara Prainsack | University of Vienna | Austria
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Prof.dr. Barbara Prainsack | University of Vienna | Austria
Prainsack has written seminal work on the concept of ‘solidarity’. This concept that is brought to the fore frequently in discussions on access to personalized medicine, including expensive cancer treatments. Interestingly, we observe that the concept is used as an argument both for and against granting patients access to expensive treatments. On the one hand, solidarity with patients who are severely ill and in urgent need of treatment might require collective efforts to help them to effective treatments. On the other hand, the use of collective means to fund treatments that are effective but expensive/not cost-effective might be seen as undesirable, as it may undermine a healthcare system built on ‘solidarity and justice’. What is the relationship between solidarity and justice in the context of access to personalized medicine, including expensive cancer treatments? Prainsack will discuss what solidarity would require healthcare systems, physicians, or patients to do when medical treatments are considered effective but too expensive to reimburse. Could solidarity demand self-exclusion, i.e. could it demand that well-off patients forego treatment using private funds, as long as others may not be able to pay for treatment either?
Sharing the benefits and burden in expensive drugs research fairly?
Prof. Dr. Hans-Jörg Ehni | University of Tübingen | Germany
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Prof. Dr. Hans-Jörg Ehni | University of Tübingen | Germany
Building on his paper ‘Expensive cancer drugs and just health care’ (2014), Ehni will discuss a new issue: extremely high drug prices are challenging some of the basic principles of the ethics of clinical research. Fair selection of participants has always required that those who take the risks and suffer the burdens of clinical research should either benefit personally from participation or belong to a group of patients who will benefit from the resulting medical treatments. Also, as very costly new medical interventions are increasingly exceeding the available resources, they might no longer be covered by public health insurance or public health care systems. Therefore, access to newly developed treatments – and the associated benefits – is no longer guaranteed. Also, people who then can not afford these drugs through either private health insurance or their own funds, might consequently only have access to such new drugs by taking part in research, which might undermine the principle of voluntariness. Extremely high costs of medical treatments might thus undermine the ethical legitimacy of clinical research as a whole.