Assessing medical devices: a qualitative study from the validate perspective.

OBJECTIVES: Our objective was to explore procedures and methods used at health technology assessment (HTA) agencies for assessing medical devices and the underlying views of HTA practitioners about appropriate methodology to identify challenges in adopting new methodologies for assessing devices. We focused on the role of normative commitments of HTA practitioners in the adoption of new methods. METHODS: An online survey, including questions on procedures, scoping, and assessments of medical devices, was sent to members of the International Network of Agencies for Health Technology Assessment. Interviews were conducted with survey respondents and HTA practitioners involved in assessments of transcatheter aortic valve implantation to gain an in-depth understanding of choices made and views about assessing medical devices. Survey and interview questions were inspired by the "values in doing assessments of health technologies" approach towards HTA, which states that HTA addresses value-laden questions and information. RESULTS: The current practice of assessing medical devices at HTA agencies is predominantly based on procedures, methods, and epistemological principles developed for assessments of drugs. Both practical factors (available time, demands of decision-makers, existing legal frameworks, and HTA guidelines), as well as commitments of HTA practitioners to principles of evidence-based medicine, make the adoption of a new methodology difficult. CONCLUSIONS: There is a broad recognition that assessments of medical devices may need changes in HTA methodology. In order to realize this, the HTA community may require both a discussion on the role, responsibility, and goals of HTA, and resulting changes in institutional context to adopt new methodologies.

The capability approach in rehabilitation: developing capability care.

PURPOSE: To develop a multidisciplinary outpatient rehabilitation intervention for people with neuromuscular diseases (NMD) based on the capability approach: capability care for persons with NMD. MATERIALS AND METHODS: The development process is described using a framework of actions for intervention development. It has been an iterative process consisting of a design phase based on theoretical insights and project group discussions, and a refine phase involving input from relevant stakeholders. RESULTS: Multidisciplinary efforts have resulted in the development of capability care for rehabilitation of persons with NMD. It can focus both on facilitating and achieving functionings (beings and doings), as well as looking for alternative functionings that fulfil the same underlying value, thereby contributing to the persons' well-being. To facilitate a conversation on broader aspects that impact on well-being, persons with NMD receive a preparation letter and healthcare professionals are provided with guiding questions and practical tools to use. CONCLUSIONS: We have shown that it is possible to develop a healthcare intervention based on the capability approach. We hope that rehabilitation professionals will be encouraged to use capability care and that other medical professionals will be inspired to develop capability care in their respective fields. REGISTRATION: Registered at trialregister.nl NL8946.

The capability approach can be used for development of healthcare interventions.Capability care in rehabilitation focuses on realising what is of real value to the person.The capability approach and the ICF are complementary and can both be used in rehabilitation.

Toward an Understanding of GSD5 (McArdle disease): How Do Individuals Learn to Live with the Metabolic Defect in Daily Life.

BACKGROUND: Glycogen storage disease type 5 (GSD) is an autosomal recessive inherited metabolic myopathy caused by a deficiency of the enzyme muscle glycogen phosphorylase. Individuals with GSD5 experience physical activity intolerance. OBJECTIVE: This patient-led study aimed to capture the daily life experiences of GSD5, with a focus on adapting to and coping with their physical activity intolerance. METHODS: An online survey was composed in close collaboration with patient organizations. It consisted of customized and validated questionnaires on demographics, general health and comorbidities, physical activity, psychosocial well-being and functioning, pain, fatigue and adapting to and coping with GSD5. RESULTS: One hundred sixty-two participants (16 countries) participated. The majority, n = 86 (69%) were from the Netherlands, USA or UK. We observed a high rate of misdiagnosis prior to GSD5 diagnosis (49%), surprisingly a relatively high proportion had not been diagnosed by DNA testing which is the gold standard. Being diagnosed had a strong impact on emotional status, daily life activities and important life choices. A large proportion had not received any rehabilitation (41%) nor medical treatment (57%) before diagnosis. Engagement in vigorous and moderate physical activity was reduced. Health related quality of life was low, most likely related to low physical health. The median Fatigue Severity Score was 4.3, indicating moderate to severe fatigue. Participants themselves had found various ways to adapt to and cope with their disability. The adaptations concerned all aspect of their life, including household chores, social and physical activities, and work. In addition to lack of support, participants reported limited availability of information sources. CONCLUSION: Participants have provided guidance for newly diagnosed people, including the advice to accept one's limited abilities and maintain an active lifestyle. We conclude that adequate counseling on ways of adapting and coping is expected to increase both health-related quality of life and physical activity.

Integrating Empirical Analysis and Normative Inquiry in Health Technology Assessment: The Values in Doing Assessments of Health Technologies Approach.

Health technology assessment (HTA) aims, through empirical analysis, to shed light on the value of health technologies (O'Rourke et al. [2020, International Journal of Technology Assessment in Health Care 36, 187-90]). HTA is, then, where facts and values meet. But how, where, and when do facts and values meet in HTA? Currently, HTA is usually portrayed as a sequential process, starting with empirical analysis (assessment), followed by a deliberation on the implications of the findings for a judgment of a health technology's value (appraisal). In this paper, we will argue that in HTA, empirical analysis and normative inquiry are much more closely entwined. In fact, as we hope to show, normative commitments act as an indispensable guide for the collection and interpretation of empirical evidence. Drawing on policy sciences, we will suggest a concrete methodology that can help HTA practitioners to integrate empirical analysis and normative inquiry in a transparent way. The proposed methodology can be conceived as a concrete means for conducting a scoping exercise in HTA. Moreover, it offers a distinct way of giving stakeholders a structural and constructive role in HTA. This paper outlines the approach developed by the values in doing assessments of health technologies project, a project funded by the Erasmus+ program (contract number 2018-1-NL01-KA203-038960), which is the European Union's program to support education, training, youth, and sport in Europe. The project has resulted in an E-learning course, an accompanying handbook, and a consensus statement, all freely available from the project's website www.validatehta.eu.

Learning and practicing more value-reflective, problem-setting health technology assessment: experiences and lessons from the VALIDATE project.

OBJECTIVES: To conduct a formative evaluation of applying the VALIDATE approach in practice by (i) assessing how students appreciated the e-learning course, (ii) exploring how, for what purposes and with what outcomes the acquired VALIDATE competences subsequently were used in internships in different institutional contexts, and how this was shaped by these contexts, and (iii) what this shows on real-world use of VALIDATE. METHODS: Comparative discussion of experiences of applying the VALIDATE approach via a semistructured survey and oral feedback from e-course students; final reports on internships in health technology assessment (HTA) practice, followed by semistructured interviews with interns and supervisors to complement and interpret results. RESULTS: All students considered the VALIDATE approach an enlightening and important addition to current HTA knowledge, especially regarding understanding the relation between empirical analysis and normative inquiry, identifying policy relevant questions and using the method of reconstructing interpretive frames for scoping. The latter appeared intellectually challenging and requiring some prior HTA knowledge. The use the VALIDATE approach in practice shows that interns productively redefined the HTA problem, based on appreciation of different stakeholders' definition of the issue; they experienced constraints from retrieving all relevant perspectives from existing literature as well as from institutional rules and routines. CONCLUSIONS: Some challenges in applying the VALIDATE approach deserve attention for its future use: currently used research approaches often assume a problem as "given"; and the data needed on different perspectives is often not reported in scientific literature. Finally, data gathering on and evaluation of value dimensions was experienced as challenging.

Prioritization of COVID-19 vaccination. The added value of the "VALIDATE" approach.

Prioritization of COVID-19 vaccines is one of the most relevant topics in the current pandemic emergency. Prioritization decisions are political decisions that are value-laden, and as such of ethical nature. Despite the clear political and ethical nature of this topic, prioritization decisions are often interpreted and presented as scientific decisions. The aim of this article is twofold. First, we aim to show critical points that characterize certain pandemic vaccination plans from the ethical viewpoint using four dimensions (problem definitions, incorporation of different perspectives, context, and specification). The four dimensions were drawn from findings of the European project "VALIDATE" (VALues In Doing Assessments of healthcare TEchnologies", https://validatehta.eu). Second, we aim to reframe the issue about prioritization itself in the light of the four dimensions mentioned. Our conclusion is that policy-problem definitions, incorporation of different perspectives, contextual considerations and specification of moral principles seem to be common critical points of some vaccination plan documents. The European project "VALIDATE" seems to be able to provide a useful and profitable approach to address many of these critical points.

Care for capabilities: Implementing the capability approach in rehabilitation of patients with neuromuscular diseases. Study protocol of the controlled before-after ReCap-NMD study.

BACKGROUND: High quality care of patients with neuromuscular diseases requires a personalised approach that focuses on achieving and maintaining a level of functioning that enables them to be in a state of well-being. The capability approach states that well-being should be understood in terms of capabilities, the substantial opportunities that people have to be and do things they have reasons to value. In this Rehabilitation and Capability care for patients with Neuromuscular diseases (ReCap-NMD) study, we want to investigate whether providing care based on the capability approach (capability care) has an added value in the rehabilitation of patients with neuromuscular diseases (NMD). METHODS: Two groups of 30 adult patients with facioscapulohumeral muscular dystrophy or myotonic dystrophy type 1 will be included. The first group will receive rehabilitation care as usual with a follow-up period of 6 months. Then, based on theory, and experiences of patients and healthcare professionals, capability care will be developed. During the following 3 months, the multidisciplinary outpatient rehabilitation care team will be trained in providing this newly developed capability care. Subsequently, the second group will receive capability care, with a follow-up period of 6 months. A mixed methods approach is used with both qualitative and quantitative outcome measures to evaluate the effect of capability care and to perform a process evaluation. The primary outcome measure will be the Canadian Occupational Performance Measure. DISCUSSION: The ReCap-NMD study is the first study to design and implement a healthcare intervention based on the capability approach. The results of this study will expand our knowledge on how the capability approach can be applied in delivering and evaluating healthcare, and will show whether implementing such an intervention leads to a higher well-being for patients with NMD. TRIAL REGISTRATION: Registered at Trialregister.nl (Trial NL8946) on 12th of October, 2020.

Mixed claims in Health Technology Assessment: The case of Non-Invasive Prenatal Testing.

Health Technology Assessment (HTA) uses explicit methods to determine the value of a health technology. This typically results in several claims regarding the effects that are expected to follow from the use of a health technology in a particular context. These claims seem to capture conclusions based solely on facts, but they often combine empirical information with normative presuppositions. Claims that have this character reflect (implicit) value judgments and have been labelled mixed claims. Not recognizing these normative components of such claims risks value inattention and value imposition, presenting results as self-evident and not in need of any moral justification. As proposed by Anna Alexandrova, to avoid these risks of value inattention and imposition we need rules to deal with mixed claims. According to her, when producing and evaluating mixed claims we need to unearth the invoked value presuppositions and check whether these presuppositions are invariant to disagreements. By applying these rules, the robustness of mixed claims can be checked: it can be evaluated whether their truth value is independent from the way in which their components, involving normative presuppositions, are conceptualized. This paper aims to illustrate the role of mixed claims in HTA, and expand upon the work by Alexandrova, by analyzing claims and recommendations presented in an HTA report on the introduction of Non-Invasive Prenatal Testing (NIPT) in The Netherlands. Our results show that the report contains mixed claims, and that a normative analysis of these claims can help to clarify the normativity of HTA and evaluate the robustness of claims on alleged effects of a health technology.

Core competencies for ethics experts in health technology assessment.

OBJECTIVES: There is no consensus on who might be qualified to conduct ethical analysis in the field of health technology assessment (HTA). Is there a specific expertise or skill set for doing this work? The aim of this article is to (i) clarify the concept of ethics expertise and, based on this, (ii) describe and specify the characteristics of ethics expertise in HTA. METHODS: Based on the current literature and experiences in conducting ethical analysis in HTA, a group of members of the Health Technology Assessment International (HTAi) Interest Group on Ethical Issues in HTA critically analyzed the collected information during two face-to-face workshops. On the basis of the analysis, working definitions of "ethics expertise" and "core competencies" of ethics experts in HTA were developed. This paper reports the output of the workshop and subsequent revisions and discussions online among the authors. RESULTS: Expertise in a domain consists of both explicit and tacit knowledge and is acquired by formal training and social learning. There is a ubiquitous ethical expertise shared by most people in society; nevertheless, some people acquire specialist ethical expertise. To become an ethics expert in the field of HTA, one needs to acquire general knowledge about ethical issues as well as specific knowledge of the ethical domain in HTA. The core competencies of ethics experts in HTA consist of three fundamental elements: knowledge, skills, and attitudes. CONCLUSIONS: The competencies described here can be used by HTA agencies and others involved in HTA to call attention to and strengthen ethical analysis in HTA.