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The inherent normativity of HTA can be conceptualized as a result of normative commitments, a concept that we further specify to encompass moral, epistemological and ontological commitments at play in the practice of HTA. Based on examples from literature, and an analysis of the example of assessing Non-Invasive Prenatal Testing (NIPT), we will show that inevitable normative decisions in conducting an assessment commits the HTA practitioner to moral (regarding what makes a health technology desirable), ontological (regarding which effects of health technology are conceivable), and epistemological (regarding how to obtain reliable information about health technology) norms. This highlights and supports the need for integrating normative analysis and stakeholder participation, providing guidance to HTA practitioners when making normative choices. This will foster a shared understanding between those who conduct, use, or are impacted by assessments regarding what are conceivable and desirable outcomes of using health technology, and how to collect reliable information to assess whether these outcomes are (going to be) realized. It also provides more insight into the implications of different normative choices.
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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.
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INTRODUCTION: Cochlear implantation (CI) is a (cost-)effective intervention for people with severe or profound hearing loss. Since its introduction experience increased and the technology evolved, leading to better results and relaxation of CI eligibility criteria. Meanwhile, with national healthcare costs increasing there is a need for evidence of healthcare technology's value. This protocol describes a study to investigate clinical and participatory outcomes after CI for the currently (expanded) eligible hearing impaired population. The study adds to the current evidence base through its multicentre design, long-term follow-up and use of participatory outcomes alongside standard clinical outcomes. METHODS: This multicentre prospective observational cohort study will include at least 156 adult patients with severe-to-profound hearing loss, approximately evenly divided into two groups (1, ages 18-65 years and 2, age >65 years). The measurements consist of audiometry, cognition tests, listening effort tests and multiple generic and disease specific questionnaires. Questionnaires will be administered twice before CI, soon after inclusion at CI referral and shortly before CI surgery, with an annual follow-up of 3 years after CI. The Impact on Participation and Autonomy questionnaire will be used to assess participation. Generalised models (linear, logistic, Poisson) will be used. Mixed effects models will be used to investigate changes over time while exploring differences in subgroups and the influence of covariates. ETHICS AND DISSEMINATION: The study has received ethical approval from the Medical Ethical Committee of all participating centres. The results could provide valuable insights into changes in participatory outcomes of people with severe-to-profound hearing loss after CI. Results will be disseminated through peer-reviewed journals, scientific conferences and professional and patient organisation meetings. TRIAL REGISTRATION NUMBER: NCT05525221.
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Implantación Coclear , Sordera , Pérdida Auditiva Sensorineural , Pérdida Auditiva , Adulto , Anciano , Humanos , Pérdida Auditiva/cirugía , Pérdida Auditiva Sensorineural/cirugía , Estudios Multicéntricos como Asunto , Estudios Observacionales como Asunto , Estudios Prospectivos , Calidad de Vida , Adolescente , Adulto Joven , Persona de Mediana EdadRESUMEN
In the context of its program 'Appropriate Care', the Healthcare Institute of the Netherlands has suggested that scarcity in healthcare should also be discussed between patient and healthcare provider. In this paper the desirability of this proposal will be explored. This will be done on the basis of a new and expensive drug for the treatment of children with spinal muscle atrophy, risdiplam.
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Atención a la Salud , Pacientes , Niño , Humanos , Evaluación de Programas y Proyectos de Salud , Países Bajos , Personal de SaludRESUMEN
We investigated 34 deaf and hard-of-hearing children with hearing devices aged 8-12 years and 30 typical hearing peers. We used the capability approach to assess well-being in both groups through interviews. Capability is "the real freedom people have to do and to be what they have reason to value." Speech perception, phonology, and receptive vocabulary data of the deaf and hard-of-hearing children, that were used retrospectively, showed a large variability. The analysis of the relation between clinical quantitative outcome measures and qualitative capability interview outcomes suggests that at this age, differences in clinical performance do not appear to translate into considerable differences in capability, including capability did offer insight into the factors that appeared to ensure this equivalence of capability. We argue that capability outcomes should be used to determine the focus of (auditory) rehabilitation and support, in line with the United Nations Convention on the Rights of the Child.
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Audición , Percepción del Habla , Niño , Humanos , Estudios Retrospectivos , Lingüística , Grupo ParitarioRESUMEN
Background: Engaging with the arts can enrich medical education by fostering transformative learning, reflection, and a holistic view of the patient. Objective: To explore the development of professional competence of residents in prolonged arts-based medical education. Methods: We followed residents (n=99) of various specialties as they engaged in arts-based learning through creative and reflective assignments such as painting, sculpting, and formal analysis. Participants were interviewed about their learning process and experiences, one-on-one and in small groups, by independent researchers using short, semistructured interviews. We used grounded theory to inform an iterative process for data collection and analysis over the course of 3 years (2016-2018). Results: Seven themes were constructed, which showed that (1) slowing down education provides room for reflection; (2) absence of judgment and rules sparks experimentation; (3) engaging with emotions fostered reflection and motivation; (4) the artists' methods provided a perspective change; (5) a holistic view on the patient emerged; (6) residents understood the need to take control over their professional development; and (7) there were barriers to overcome in terms of hierarchy and expectations. Our investigation shows that interns and residents undergo a perspective transformation. Key to the development of the physicians in training is the open and affective nature of the arts in education. Conclusions: Arts-based learning results in a new perspective for physicians in training in line with patient-centered health care and self-directed learning.
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Arte , Educación Médica , Internado y Residencia , Médicos , Humanos , Aprendizaje , Médicos/psicología , Educación Médica/métodosRESUMEN
OBJECTIVES: Eligibility criteria for cochlear implantation (CI) are shifting due to technological and surgical improvements. The aim of this study was to explore the impact of further expanding unilateral CI criteria in those with severe hearing loss (HL) (61-80 dBHL) in terms of number of CI recipients, costs, quality of life, and cost-effectiveness. METHODS: A dynamic population-based Markov model was constructed mimicking the Dutch population in three age categories over a period of 20 years. Health states included severe HL (61-80 dBHL), profound HL (>81 dBHL), CI recipients, and no-CI recipients. Model parameters were based on published literature, (national) databases, expert opinion, and model calibration. RESULTS: If persons with severe HL would qualify and opt for CI similar to those with profound HL now, this would lead to a 6-7 times increase of new CI recipients and an associated increase in costs (550 million) and QALYs (54.000) over a 20-year period (incremental cost utility ratio: 10.771 euros/QALY [2.5-97.5 percentiles: 1.252-23.171]). One-way-sensitivity analysis indicated that model outcomes were most sensitive to regaining employment, utility associated with having a CI, and costs of surgery and testing. CONCLUSION: Our findings suggest that expanding eligibility for CI to persons with severe HL could be a cost-effective use of resources. Clearly, however, it would require a significant increase in diagnostic, operative, and rehabilitative capacity. Our quantitative estimates can serve as a basis for a wider societal deliberation on the question whether such an increase can and should be pursued. LEVEL OF EVIDENCE: NA Laryngoscope, 133:924-932, 2023.
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Implantación Coclear , Pérdida Auditiva Sensorineural , Pérdida Auditiva , Humanos , Adulto Joven , Adulto , Pérdida Auditiva Sensorineural/cirugía , Calidad de Vida , Análisis Costo-Beneficio , Pérdida Auditiva/cirugíaRESUMEN
The unique task and contribution of health technology assessment (HTA) is to help identify those health technologies and their uses that are most likely to preserve and restore a population's health in a way that is consonant with its values, including, for example, equity and access to high-quality care (1). Such a task is challenging for at least two reasons. First, because of the vast and constantly evolving number and diversity of health technologies and their applications. Second, because of the usual wide variety of competing views within communities and their stakeholders regarding what strategies are likely to be conducive to the goal of preserving and restoring population health. Although perhaps tempting, it would be a grave mistake to hold that the controversies that result from such competing views can be resolved by taking recourse to the facts only. For such controversies are usually fueled by different notions of health and disease and different specifications of values such as equity and individual and collective responsibility for health. For this reason, they cannot be resolved in a satisfactory way without also addressing those normative issues and their interplay with empirical analysis.
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Tecnología Biomédica , Evaluación de la Tecnología BiomédicaRESUMEN
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.
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Tecnología Biomédica , Evaluación de la Tecnología Biomédica , Europa (Continente) , Evaluación de la Tecnología Biomédica/métodosRESUMEN
In the Western world, for deaf and hard-of-hearing children, hearing aids or cochlear implants are available to provide access to sound, with the overall goal of increasing their wellbeing. If and how this goal is achieved becomes increasingly multifarious when these children reach adolescence and young adulthood and start to participate in society in other ways. An approach to wellbeing that includes personal differences and the relative advantages and disadvantages that people have, is the capability approach, as developed by Nobel Prize laureate Amartya Sen. Capability is the set of real opportunities people have to do and be things they have reason to value. We interviewed 59 young people, aged 13 through 25, with cochlear implants (37) or hearing aids (22) to capture their capability. We found that their hearing devices enabled them to actively participate in a predominantly hearing society, with few differences between cochlear implant and hearing aid recipients. They did, however, report challenges associated with prejudices and expectations, and with feeling poorly understood, all of which appeared to impact their capability. Through the lens of capability, alleged differences between hearing aid and cochlear implant recipients began to fade. We discuss the implications for initiatives focused on the long-term support young recipients of hearing devices to meet their specific requirements over time.
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BACKGROUND: Bumetanide is a selective NKCC1 chloride importer antagonist which is being repurposed as a mechanism-based treatment for neurodevelopmental disorders (NDDs). Due to their specific actions, these kinds of interventions will only be effective in particular subsets of patients. To anticipate stratified application, we recently completed three bumetanide trials each focusing on different stratification strategies with the additional objective of deriving the most optimal endpoints. Here we publish the protocol of the post-trial access combined cohort study to confirm previous effects and stratification strategies in the trial cohorts and in new participants. METHOD/DESIGN: Participants of the three previous cohorts and a new cohort will be subjected to 6 months bumetanide treatment using multiple baseline Single Case Experimental Designs. The primary outcome is the change, relative to baseline, in a set of patient reported outcome measures focused on direct and indirect effects of sensory processing difficulties. Secondary outcome measures include the conventional questionnaires 'social responsiveness scale', 'repetitive behavior scale', 'sensory profile' and 'aberrant behavior scale'. Resting-state EEG measurements will be performed at several time-points including at Tmax after the first administration. Assessment of cognitive endpoints will be conducted using the novel Emma Tool box, an in-house designed battery of computerized tests to measure neurocognitive functions in children. DISCUSSION: This study aims to replicate previously shown effects of bumetanide in NDD subpopulations, validate a recently proposed treatment prediction effect methodology and refine endpoint measurements. TRIAL REGISTRATION: EudraCT: 2020-002196-35, registered 16 November 2020, https://www.clinicaltrialsregister.eu/ctr-search/trial/2020-002196-35/NL.
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Bumetanida , Trastornos del Neurodesarrollo , Bumetanida/farmacología , Bumetanida/uso terapéutico , Niño , Estudios de Cohortes , Humanos , Trastornos del Neurodesarrollo/tratamiento farmacológico , Proyectos de Investigación , Resultado del TratamientoRESUMEN
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.
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Tecnología Biomédica , Evaluación de la Tecnología Biomédica , Humanos , Conocimiento , Proyectos de Investigación , Evaluación de la Tecnología Biomédica/métodosRESUMEN
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.
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Vacunas contra la COVID-19 , COVID-19 , COVID-19/prevención & control , Humanos , Pandemias/prevención & control , VacunaciónRESUMEN
OBJECTIVES: Early assessments of health technologies help to better align and integrate their development and assessment. Such assessments can take many forms and serve different purposes, hampering users in their selection of the most appropriate method for a specific goal. The aim of this scoping review was to structure the large set of methods according to their specific goal. METHODS: A scoping review was conducted using PubMed and reference lists of retrieved articles, to identify review studies with a methodological focus. From the included reviews, all individual methods were listed. Based on additional literature and examples, we extracted the specific goal of each method. All goals were clustered to derive a set of subclasses and methods were grouped into these subclasses. RESULTS: Of the 404 screened, 5 reviews were included, and 1 was added when searching reference lists. The reviews described 56 methods, of which 43 (77%) were included and classified as methods to (1) explore the nature and magnitude of the problem, (2) estimate the nature and magnitude of the expected (societal) value, (3) identify conditions for the potential value to materialize, and (4) help develop and design the type of research that is needed. CONCLUSIONS: The wide range of methods for exploring the societal value of health technologies at an early stage of development can be subdivided into a limited number of classes, distinguishing methods according to their specific objective. This facilitates selection of appropriate methods, depending on the specific needs and aims.
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Proyectos de Investigación , HumanosRESUMEN
BACKGROUND AND OBJECTIVES: To perform a systematic review of published N-of-1 trials (e.g., single patient crossover trials) in neurologic disorders, including an assessment of methodologic quality and reporting. METHODS: We searched PubMed, MEDLINE, and Embase from inception date to the December 1, 2019, for reports on N-of-1 trials in neurologic disorders. Basic trial information on design, disease, intervention, analysis, and treatment success was extracted. Strengths and weaknesses of the N-of-1 trials were assessed with the Consolidated Standards of Reporting Trials extension for N-of-1 trials (CENT) 2015 criteria checklist and the Jadad score as measures of quality and reporting. RESULTS: We retrieved 40 reports of N-of-1 trials in neurologic disorders (19 individual N-of-1 trials, 21 series of N-of-1 trials). Most N-of-1 trials were performed in neuromuscular and neurodegenerative/movement disorders. Unlike the majority of trials that studied the main symptom(s) of a chronic stable condition, 9 N-of-1 trials studied a stable chronic symptom of a progressive or acute neurologic disorder. Besides pharmacologic interventions, electric stimulation protocols and nutritional products were studied. A mean total CENT score of 20.88 (SD 9.10, range 0-43) and mean total Jadad score of 2.90 (SD 2.15, range 0-5) were found as methodologic measures of quality and reporting across all N-of-1 trials. DISCUSSION: N-of-1 trials have been reported in numerous neurologic disorders, not only in chronic stable disorders, but also in progressive or acute disorders with a stable symptom. This indicates the emerging therapeutic area of N-of-1 trials in neurology. Methodologic quality and reporting of N-of-1 trials were found to be suboptimal and can easily be improved in future trials by appropriately describing the methods of blinding and randomization and following CENT guidelines. Because most N-of-1 trials remain unreported in medical literature, this systematic review probably represents only the tip of the iceberg of conducted N-of-1 trials in neurologic disorders. In addition to conventional trial designs, N-of-1 trials can help to bridge the gap between research and clinical care by providing an alternative, personalized level 1 evidence base for suitable treatments.
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Lista de Verificación , Neurología , Enfermedad Aguda , Enfermedad Crónica , HumanosRESUMEN
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.
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Enfermedades Neuromusculares/rehabilitación , Adulto , Estudios Controlados Antes y Después , Femenino , Humanos , Masculino , Distrofia Muscular Facioescapulohumeral/diagnóstico , Distrofia Muscular Facioescapulohumeral/rehabilitación , Distrofia Miotónica/diagnóstico , Distrofia Miotónica/rehabilitación , Enfermedades Neuromusculares/diagnóstico , Evaluación de Resultado en la Atención de Salud , Grupo de Atención al PacienteRESUMEN
Preparing future professionals for highly dynamic settings require self-directed learning in authentic learning situations. Authentic learning situations imply teamwork. Therefore, designing education for future professionals requires an understanding of how self-directed learning develops in teams. We followed (bio-)medical sciences students (n = 15) during an 8-month period in which they worked on an innovation project in teams of 4-6 students. Template analysis of 39 transcripts of audio-recorded group meetings revealed three mechanisms along which group dynamics influenced self-directed learning behaviour. First, if expressions of emotions were met with an inquisitive response, this resulted in self-monitoring or feelings of responsibility. Second, openness in the group towards creativity or idea exploration stimulated critical thinking. Third, disputational talk frustrated learning, because it adversely affected group cohesion. We conclude that emotions, openness, and relatedness are important drivers of self-directed learning in teams and hence should be given explicit attention in designing collaborative learning for future professionals.
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Prácticas Interdisciplinarias , Estudiantes de Medicina , Curriculum , Evaluación Educacional , Humanos , AprendizajeRESUMEN
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.