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Kohlhammer VW, author; Schildmann J, Buch C, Zerth J, editors. Defining the Value of Medical Interventions: Normative and Empirical Challenges [Internet]. Stuttgart (DE): W. Kohlhammer GmbH; 2021.
Discussions of how to define the value of medical interventions and the impacts concerning societal consequences have gained considerable attention during the last few decades. Different factors should be mentioned regarding that development. Firstly, a rectangularization of survival curve depicts the important impacts of a so-called demographic change in all industrialised countries. Not only have the proportions of elderly patients become more dominant, but the development of medical progress opens up new possibilities for diagnosis and treatment. Consequently, the so-called demographic effect can be explained endogenously by different factors which help patients to live longer and with a higher quality of life. Secondly, the progress of medical technologies reopens the discussion on the upper bounds a solidarity-based healthcare system is willing to bear. Finally, several new drugs and other interventions coming at high costs have been developed and approved in recent years. In Germany, for example the costs for anti-cancer drugs in 2017 were almost 6 billion EUR of around 38 billion spent on drugs by the statutory health insurance. Costs for anti-cancer drugs increased by more than 15 per cent compared to 2016.
Given the limited solidarity-based budget and the drivers of expenditures in healthcare mentioned previously, there is little surprise that the calls for investigating the value of healthcare interventions and the overall value of health itself have become more pressing. In this context, the term value-based healthcare has been used increasingly in debates about cost outcomes and necessities of health interventions. Value here refers to the regard we ascribe to something or the worth we think something should deserve. The US-American economist Michael Porter described the focus and goal of value-based healthcare in a contribution to the New England Journal of Medicine in 2009 as follows:
The central focus must be on increasing value for patients – the health outcomes achieved per dollar spent. Good outcomes that are achieved efficiently are the goal, not the false ‘savings’ from cost shifting and restricted services. Indeed, the only way to truly contain costs in health care is to improve outcomes: in a value-based system, achieving and maintaining good health is inherently less costly than dealing with poor health.
Accordingly, value, as outlined above, refers to the quotient of costs and benefit of a health intervention. While Porter's account of value-based healthcare (or “value-based system” as in the title of the respective article) is directed at a series of changes proposed to reform the US healthcare system, the concept has been taken up by many proponents especially in the debate about the costs and benefits of cancer treatment.
However, the concept of value as proposed in the context of value-based healthcare is not the only possible understanding of the term. Value or values also describe principles according to which we judge what is important in life. In this respect, values are held on an individual level, but they can also be shared by a group of people and, at least, values should be a main column of organised, institutionalized healthcare services. These understandings of the term can obviously be linked but, at the same time, it is important to clarify what we refer to when talking about the value of medical interventions. With this book, we provide a collection of articles which take up different perspectives on the meaning of value in healthcare.
This book is the result of a process which took place over two years, during which junior researchers from a range of normative and empirical disciplines contributed to the topic of “defining the value of medical interventions”. At the centre of this process was a conference at Wilhelm Löhe Hochschule in Fuerth, which took place in September 2019, near the ancient Franconian city of Nuremberg. The conference itself was based on the scientific workshop of the young scientists discussing their abstracts with each other combined with invited experts, reflecting the interdisciplinary approach. Moreover, some additional discourses took place in a broader sense. Two invited representatives of IQWiG (Institute for Quality and Efficiency in Health Care), Naomi Fujita-Rohwerder and Katharina Wölke, gave different insights into the German assessment strategy for valuating nondrug interventions and described the concept of early benefit assessment for pharmaceuticals. Michael Parker (University of Oxford) gave a short presentation on the first results of the Working Group “Research in global health emergencies”. Some discussions on the necessity for an adequate implementation of beneficiaries of healthcare interventions occurred. In order to stress the role of different caregivers in health and social care markets, Jürgen Zerth (Wilhelm Loehe University of Applied Science) also discussed the need to rethink the role of social entrepreneurship regarding the linkage of organised healthcare, long-term care markets and different institutional approaches to cover social care needs. At the end of the conference week, a public discussion with different stakeholders from the German healthcare sector took place in Nuremberg in order to give forms of participation for the public.
The preparations for the conference “Defining the value of medical interventions. Normative and empirical challenges” (funded by the German Federal Ministry of Education and Research (01GP1883)) started with a call for abstracts, followed by a selection of contributions based on the scientific quality and scope of the papers, and our aim to further the discussion on recent developments and different disciplinary perspectives on defining the value of medical interventions. All the participants invited received personalised feedback on their abstracts and information on the contributions of the other participants. Those presentations that were connected thematically close were grouped together as part of the workshop programme. The conference offered a unique opportunity not only for presentations by all the participants but also plenty of time for discussion on each contribution. In addition, three workshops with the scientific and healthcare specialists mentioned above provided an opportunity for further in-depth discussions. Subsequently, all the participants were invited to submit a manuscript of their presentation, taking into account the feedback they had received at the conference. All manuscripts have been reviewed by at least one conference participant and one editor with a focus on the content of each chapter and links between the different contributions.
One fundamental challenge in the debate about the value of medical interventions (and also a much-discussed question at the conference) is to find a theoretical consensus about how to define value(s) in healthcare, which also means the value of health, hence medical interventions. Against the background of several theoretical approaches to this question, the contributions in the first part provide insights into theoretical premises for understanding value(s) in healthcare.
Francisca Stutzin Donoso's article The concepts of ‘health’ and ‘disease’: underlying assumptions in the idea of value in medical interventions provides an introduction into the debate, in so far as it discusses the fundamental concepts of health and disease, which is an indispensable background when talking about the value of medical interventions. Her article presents multiple existing definitions of health and disease and points out potential deficiencies and general criticisms. Based on her analysis, she provides an account of elements of disease concepts, which allow contextualisation of health outcomes and the value(s) of medical interventions.
Karla Alex discusses the connection between the right to health and healthcare and the concept of sustainability in Ethical conceptualization of a sustainable right to health(care). Influenced by Thomas Nagel's defence of normative realisms, her line of argumentation starts with the objective instrumental value of health which follows from the objective intrinsic value of a human being. According to her subsequent analysis, a sustainable right to health(care) comprises an agent-relative and an agent-neutral right to health(care) and also tackles economic aspects. Furthermore, in the conceptualisation of a sustainable right to health, environmental aspects are encompassed.
Defining the value of medical interventions is relevant for proceeding through the regulated benefit basket and what the different stakeholders in the regulated healthcare markets face regarding the reimbursement process. The value assigned determines the monetary equivalent an individual or society is willing to pay for the intervention. However, without a standardised definition of what value is, the development of methodologies to assess the value of certain medical interventions is not an easy task. Multiple approaches exist at the macro level, and they differ a lot across different health systems depending on the political and legal framework and institutional characteristics. Different approaches can differ on a micro level even within one system according to the respective needs, preferences and stakeholders included. Against this background, the second part of the publication comprises contributions covering exemplary value assessments and financing approaches and their health economical and interdisciplinary analyses.
Paul Mark Mitchell starts with the presentation of the well-known quality-adjusted life years (QALYs) approach as a health-economical way of defining value in healthcare in his article The cost-effectiveness of what in health and care?. The author analyses the criticism of this approach in his work and introduces the evaluation of people's capabilities as an alternative approach to inform health and care decision-making. According to his analysis, shifting to a “sufficient capability” objective may help to address efficiency and equity concerns related to decisions about the value of medical interventions
Jasper Ubels provides an analysis which links well to the preceding chapter by taking up the introduced concepts of QALY and the capability approach in The assessment of value in health economics: utility and capability. Subsequent to a reflection of the multiple conceptualisations of utility and a critique of both QALY and the capability approach, he argues for a combined measure of the value of medical interventions which includes information about capability, functioning and utility.
Sebastian Himmler's article Estimating the monetary value of health: why and how presents a justification of why assigning a monetary value to health (QALY) is necessary and different methodologies to estimate such a value. The author outlines an alternative approach in his contribution for estimating the monetary societal valuation of a QALY: the well-being valuation approach. In addition, the author presents first results of applying this approach in two different contexts and provides an analysis of the methodological limits of the approach presented.
Charlotte Buch, Jan Schildmann and Jürgen Zerth address the risks for both the payer and the pharmaceutical company in terms of real-world effectiveness and, subsequently, financial predictability in their article Risk-sharing schemes to finance expensive pharmaceuticals. Interdisciplinary analyses. Value-based pricing is described as a possible approach to control pharmaceutical expenditures, whereby on a micro-perspective level, this is employed as risk-sharing agreements (either financial- or performance-based) between payers and pharmaceutical companies. The article provides a theoretical account and a case study of performance-based risk-sharing agreements.
An important, nevertheless, sometimes underestimated perspective when defining the value of medical interventions is the implementation of patients’ views, hence their values and preferences. As a “user” of medical interventions, however physician-induced, the patients are usually also the direct or indirect payer for healthcare. Patients are eventually the reason for all efforts regarding, for example, the definition of values, subsequent remunerations according to the value and fostering of continuous innovations. Therefore, the final part of this book focuses on the patients’ perspective on an individual and societal level.
Caroline Steigenberger, Petra Schnell-Inderst and Uwe Siebert analyse how the perspective of patients and the social perspective can be included in Health Technology Assessments (HTAs) in their article Integrating patients and social aspects into health technology assessment. The authors argue that such an integrative approach is highly relevant in order to understand the needs, values and preferences of the users of a medical intervention. In the light of lacking implementation, the authors present an approach to systematically elaborate the patients’ and social aspects within HTA taking the example of an HTA on integrative mistletoe therapy for patients with breast cancer.
Karolina Napiwodzka emphasises the importance of participatory decision-making for the eliciting of values on the micro-level in her article The shared decision-making model and practical discourse to foster the appreciation of patients’ value preferences in Polish healthcare. Discussing potentials and challenges. In her contribution, which places the Polish healthcare system at the centre of her investigation, she analyses cognitive and communicative skills, active engagement and participation as premises for the shared understanding of values and respective responsibilities.
The part and book concludes with a contribution by Jordan A. Parsons on Death or dialysis: The value of burdensome life-extending treatments for the cognitively impaired. Taking the example of decision-making about dialysis, he analyses the factors relevant for the evaluation of the burdens and benefits of the interventions with a focus on situations in which treatment may be ended. The evaluation of benefit and harm of life-sustaining treatment is usually based on the subject's perceptions and views. In his analysis, the author focuses on decisions in the particularly challenging cases of patients who are not able to communicate their preferences and values due to a lack of decisional capacity.
The brief outline above indicates that this book offers not only a wide range of topics but also disciplinary perspectives on the different facets of the value of medical interventions and reflects a guided interdisciplinary approach for discussion and induced political advice.
The editors hope that this book provides interesting multi- and, in parts, interdisciplinary perspectives on the value of medical interventions and related values. We are very grateful to all the authors for their articles and their willingness to contribute to this volume. We would like to thank Philip Saunders for proofreading and the Peter-Oberender-Stiftung for the support of a professional formatting service. Additionally, we gratefully thank Anna-Kathleen Piereth for different assistance during the conference. Finally, we thank the German Federal Ministry of Education and Research for funding the conference and this publication.
Halle (Saale) and Fuerth, December 2020
Jan Schildmann, Charlotte Buch, Jürgen Zerth
This is an open access article licensed under a Creative Commons Attribution 4.0 International License, which permits unrestricted use, distribution, reproduction and adaptation in any medium and for any purpose provided that it is properly attributed.
Monographs, or book chapters, which are outputs of Wellcome Trust funding have been made freely available as part of the Wellcome Trust's open access policy
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