Cost-effectiveness of Anti-CD19 chimeric antigen receptor T-Cell therapy in pediatric relapsed/refractory B-cell acute lymphoblastic leukemia. A societal view.

Introduction: In several studies, the chimeric antigen receptor T-cell therapy tisagenlecleucel demonstrated encouraging rates of remission and lasting survival benefits in pediatric patients with relapsed/refractory (r/r) acute lymphoblastic leukemia (ALL). We assessed the cost-effectiveness of tisagenlecleucel (list price: 320 000 EUR) among these patients when compared to clofarabine monotherapy (Clo-M), clofarabine combination therapy (Clo-C), and blinatumomab (Blina) from both a healthcare and a societal perspective. We also assessed future medical and future non-medical consumption costs.

Measurement and evaluation of quality of life and well-being in individuals having or having had fertility problems: a systematic review.

Objectives: The aims of this study were: (1) to identify which measurement instruments are used in practice to assess the quality of life or well-being of individuals with and without (sub)fertility; (2) to describe the design and outcomes of studies comparing quality of life or well-being of individuals with and without fertility problems; and (3) to determine which of the outcomes of the identified studies could be used in cost-utility studies.

Methods: A systematic literature review was performed of studies published before July 2018, using multiple databases. Included studies investigated (health-related) quality of life or well-being of individuals with fertility problems.

Structural validity and internal consistency of the Qualidem in people with severe dementia.

Background: Since its development, the Qualidem has had items that were considered unsuited for people with very severe dementia. This study attempted to investigate the applicability of all Qualidem items in people with all stages of dementia severity.

Methods: Four data sets that contained Qualidem observations on people with dementia were combined.

A Simple and Practical Index to Measure Dementia-Related Quality of Life.

Background: Research on new treatments for dementia is gaining pace worldwide in an effort to alleviate this growing health care problem. The optimal evaluation of such interventions, however, calls for a practical and credible patient-reported outcome measure.

Objectives: To describe the refinement of the Dementia Quality-of-life Instrument (DQI) and present its revised version.

Quantification of health by scaling similarity judgments.

Objective: A new methodology is introduced to scale health states on an interval scale based on similarity responses. It could be well suited for valuation of health states on specific regions of the health continuum that are problematic when applying conventional valuation techniques. These regions are the top-end, bottom-end, and states around 'dead'.

Quality of life in dementia: a study on proxy bias.

Background: Measurement of health-related quality of life (HRQoL) in dementia is difficult. At some point people with dementia become unable to meaningfully assess their own HRQoL. At such a point in time researchers need to rely on other types of information such as observation or assessments from informal caregivers (proxies).

Probabilistic choice models in health-state valuation research: background, theories, assumptions and applications.

Interest is rising in measuring subjective health outcomes, such as treatment outcomes that are not directly quantifiable (functional disability, symptoms, complaints, side effects and health-related quality of life). Health economists in particular have applied probabilistic choice models in the area of health evaluation. They increasingly use discrete choice models based on random utility theory to derive values for healthcare goods or services.

Validation study of the prototype of a disease-specific index measure for health-related quality of life in dementia.

Background: Index measures for health-related quality of life (HRQoL) quantify the desirability (utility) of a certain health state. The commonly used generic index measure, e.g.

Visual analogue scales: scale recalibration by patients with dementia and their proxies.

Background: Visual analogue scales (VAS) are often used to measure health-related quality of life (HRQoL). However, when such scales contain ambiguous anchors like "best imaginable health state," they produce answers that are difficult to interpret, as such anchors are interpreted differently by respondents of different age. This phenomenon that people's interpretation of subjective response scales changes in response to changing circumstances is known as scale recalibration.

Thurstone scaling revealed systematic health-state valuation differences between patients with dementia and proxies.

Background: Conventional techniques to measure health-related quality of life (HRQoL) in a single value or index are complex, require abstract reasoning skills, and are prone to biases (e.g., adaptation).