Search

Background: Due to rising healthcare expenditures, countries with publicly funded healthcare systems face challenges when providing newly approved expensive anti-cancer treatments to all eligible patients. In the Netherlands in 2015, the so-called Coverage Lock (CL), was introduced to help safeguard the sustainability of the healthcare system. Since then, newly approved treatments are no longer automatically reimbursed. Previous work has shown that as policies for access to CL treatments are lacking, patient access to non-reimbursed treatments is limited and variable, which raises ethical issues. The ethics of access were discussed in a series of multi-stakeholder dialogues in the Netherlands. Methods: Three dialogues were held in early 2023 and included physicians, health insurers, hospital executives, policymakers, patients, citizens, and representatives of pharmaceutical companies, patient and professional organizations. In advance, participants had received an ‘argument scheme’ featuring three models: 1) access based on third-party payment (e.g., by pharmaceutical companies, health insurers or hospitals) 2) access based on out-of-pocket payments by patients 3) no access to CL treatments. During the dialogues, participants were asked to discuss the merits of the ethical arguments for and against these models together, and ultimately to weigh them. The discussions were audio-taped, transcribed, coded, and thematically analyzed. Results: Generally, most stakeholders were in favour of allowing access–at least when treatments are clearly beneficial–to treatments in the CL. When discussing third-party payment, stakeholders favoured payment by pharmaceutical companies over payment by health insurers or hospitals, not wanting to usurp collective funds while cost-effectiveness assessments are still pending. Largely, stakeholders were not in favour of out-of-pocket payments, emphasizing solidarity and equal access

Some psychiatric patients have exhausted all approved treatment options. Numerous investigational drugs are currently being developed and tested in clinical trials. However, not all patients can participate in clinical trials. Expanded access programs may provide an opportunity for patients who cannot participate in clinical trials to use investigational drugs as a therapeutic option outside of clinical trials. It is unknown to what extent expanded access occurs in psychiatry. We conducted a systematic literature search on PubMed, Embase, and PscyInfo, with additional information from ClinicalTrials.gov, the WHO International Clinical Trials Registry Platform and FDA/EMA approvals, in order to find all expanded access programs ever conducted, globally, in the field of psychiatry. This resulted in a total of fourteen expanded access programs ever conducted in psychiatry. Given the prevalence of psychiatric disorders, the activity in clinical research in psychiatry, the regulatory framework enabling expanded access, and the impact of psychiatric disorders on patients, their families, and society, we had expected a higher utilization of expanded access. We propose that the psychiatric community, with pharmaceutical industry, should consider establishing and optimizing expanded access programs.

When the cost-effectiveness of newly approved cancer treatments is insufficient or unclear, they may not (immediately) be eligible for reimbursement through basic health insurance in publicly funded healthcare systems. Patients may seek access to non-reimbursed treatment through other channels, including individual funding requests made to hospitals, health insurers, or pharmaceutical companies. Alternatively, they may try to pay out of pocket for non-reimbursed treatments. While currently little is known of these practices, they run counter to a deeply held egalitarian ethos that is prevalent in many publicly funded healthcare systems. In this article, we investigate to what extent this ethos can be grounded in theories of justice, notably egalitarianism and prioritarianism. We argue that allowing out-of-pocket payments by patients themselves, in principle, is not unjust from the perspective of either of these theories, provided that it does not raise in-practice justice-based concerns, for instance by displacing more cost-effective care, to the detriment of other patients, or by failing to treat patients equally. In contrast, we conclude that the practice of making exceptions for individual patients by health insurers or healthcare providers does run counter to the justice-based requirements of equal treatment.

The field of regenerative medicine offers potential therapies for Type 1 Diabetes, whereby metabolically active cellular components are combined with synthetic medical devices. These therapies are sometimes referred to as “bioartificial pancreases.” For these emerging and rapidly developing therapies to be clinically translated to patients, researchers must overcome not just scientific hurdles, but also navigate complex legal, ethical and psychosocial issues. In this article, we first provide an introductory overview of the key legal, ethical and psychosocial considerations identified in the existing literature and identify areas where research is currently lacking. We then highlight two principal areas of concern in which these discrete disciplines significantly overlap: 1) individual autonomy and 2) access and equality. Using the example of beta-cell provenance, we demonstrate how, by harnessing an interdisciplinary approach we can address these key areas of concern. Moreover, we provide practical recommendations to researchers, clinicians, and policymakers which will help to facilitate the clinical translation of this cutting-edge technology for Type 1 Diabetes patients. Finally, we emphasize the importance of exploring patient perspectives to ensure their responsible and acceptable translation from bench to body.

Background: Metastatic cancer is often experienced by patients as a death sentence. At the same time, translational scientists approach metastasis also as an interesting phenomenon that they try to understand and prevent. These two sides of the same coin do not mask the considerable gap that exists between the laboratory world of scientists and the life world of patients. Funding agencies nowadays increasingly demand researchers to be responsive to the values and priorities of patients and public. One approach to bridge this gap and to increase the impact of science is patient and public involvement (PPI). A concise literature review of PPI research and practice in this paper revealed that although PPI is often deployed in translational health care research, its methodology is not settled, it is not sufficiently emancipatory, and its implementation in basic and translational science is lagging behind. Here, we illustrate the practical implementation of PPI in basic and translational science, namely in the context of HOUDINI, a multidisciplinary network with the ultimate goal to improve the management of metastatic disease. Methods: This paper reports on a societal workshop that was organized to launch the holistic PPI approach of HOUDINI. During this workshop, societal partners, patients, and physicians discussed societal issues regarding cancer metastasis, and contributed to prioritization of research objectives for HOUDINI. In a later stage, the workshop results were discussed with scientists from the network to critically review its research strategy and objectives. Results: Workshop participants chose the development of metastasis prediction tools, effective therapies which preserve good quality of life, and non-invasive tissue sampling methods as most important research objectives for HOUDINI. Importantly, during the discussions, mutual understanding about issues like economic feasibility of novel thera

Public healthcare systems are increasingly refusing (temporarily) to reimburse newly approved medical treatments of insufficient or uncertain cost-effectiveness. As both patient demand for these treatments and their list prices increase, a market might arise for voluntary additional health insurance (VHI) that covers effective but (very) expensive medical treatments. In this paper, we evaluate such potential future practices of VHI in public healthcare systems from a justice perspective. We find that direct (telic) egalitarian objections to unequal access to expensive treatments based on different ability to afford VHI do not stand up to scrutiny. However, such unequal access might lead to loss of self-respect among individuals, or loss of fraternity within society, rendering it more difficult for citizens to interact on equal moral footing. This would be problematic from a relational egalitarian perspective. Moreover, the introduction of VHI might turn out to have negative consequences for the comprehensiveness and/or the quality of the public healthcare services that are offered to all patients equally through basic health insurance. These consequences must be weighed against potential health gains and the value of liberty. We conclude that governments should be careful when considering the introduction of VHI in public healthcare systems.

Drawing on ethical and legal frameworks in the Netherlands, the United States and France, we examine whether physicians are expected to inform patients about potentially relevant opportunities for expanded access to investigational drugs. While we found no definitive legal obligation, we argue that physicians have a moral obligation to discuss opportunities for expanded access with patients who have run out of treatment options to prevent inequality, to promote autonomy, and to achieve beneficence.

The development of new effective but expensive medical treatments leads to discussions about whether and how such treatments should be funded in solidarity-based healthcare systems. Solidarity is often seen as an elusive concept; it appears to be used to refer to different sets of concerns, and its interrelations with the concept of justice are not well understood. This paper provides a conceptual analysis of the concept of solidarity as it is used in discussions on the allocation of healthcare resources and the funding of expensive treatments. It contributes to the clarification of the concept of solidarity by identifying in the literature and discussing four uses of the concept: (1) assisting patients in need, (2) upholding the solidarity-based healthcare system, (3) willingness to contribute and (4) promoting equality. It distinguishes normative and descriptive uses of the concept and outlines the overlap and differences between solidarity and justice. Our analysis shows that the various uses of the concept of solidarity point to different, even conflicting, ethical stances on whether and how access to effective, expensive treatments should be provided. We conclude that the concept of solidarity has a role to play in discussions on the accessibility and funding of newly approved medical treatments. It requires, for instance, that healthcare policies promote and maintain both societal willingness to contribute to the care of others and the value of providing care to vulnerable patients through public funding.