Project description:BackgroundEconomic evaluation of one-time therapies during reimbursement decision-making is challenging due to uncertain long-term outcomes. The availability of 5-year outcome data from the ELIANA trial and real-world evidence of tisagenlecleucel, the first chimeric antigen receptor T-cell (CAR-T) therapy, presents an opportunity to re-evaluate the predictions of prior cost-effectiveness analyses (CEAs).ObjectiveTo conduct a systematic literature review (SLR) of prior CEAs of tisagenlecleucel for pediatric/young adult relapsed or refractory acute lymphoblastic leukemia (r/r ALL) and evaluate the impact of recently available 5-year efficacy data from ELIANA and advances in CAR-T manufacturing in an updated CEA model.MethodsOVID MEDLINE/Embase and health technology assessment (HTA) databases were searched for full-text economic evaluations in English reporting cost-effectiveness results for tisagenlecleucel for r/r ALL. Evaluations with publicly reported incremental cost-effectiveness ratios (ICERs) were included in the SLR. Study screening and data abstraction were conducted following PRISMA guidelines. Data extracted included the country/currency, perspective, clinical trial evidence, model structures, long-term efficacy extrapolation approaches (i.e., overall survival [OS]), time horizon, discount rates, and outcomes (i.e., life years [LY], quality-adjusted LY [QALY], and ICERs). The CEA model reported in Wakase et al. was updated using 5-year OS data from ELIANA and the CAR-T infusion rate informed by real-world practice.ResultsSixteen records corresponding to 15 unique studies were included in the SLR (11 publications and 5 HTA reports); all were conducted from the health care system perspective of the respective countries. Most studies found tisagenlecleucel to be cost effective, but all studies' projected 3- and 5-year OS rates for tisagenlecleucel were lower than the observed 3- and 5-year rates, respectively, derived from 5-year ELIANA data. When applying updated OS projections from the most recent ELIANA data cut and higher infusion rates of 92.5% (per the real-world infusion rate)-96.0% (per the manufacturer success rate) to the CEA of Wakase et al., the associated QALYs for tisagenlecleucel increased from 11.6 to 14.6-15.0, and LYs increased from 13.3 to 17.0-17.5. Accordingly, the ICERs for tisagenlecleucel decreased from ¥2,035,071 to ¥1,787,988-¥1,789,048 versus blinatumomab and from ¥2,644,702 to ¥2,257,837-¥2,275,181 versus clofarabine combination therapy in the updated CEA model.Conclusions and relevanceProjections at launch of the likely cost effectiveness of tisagenlecleucel appear to have underestimated its ultimate economic value given more recent trial and real-world data. To balance uncertainty in initial valuation with the need to provide access to novel oncology therapies, payers can consider flexible reimbursement policies alongside ongoing assessments as new data emerge.

Project description:ObjectiveGuidelines for a structured assessment in community paramedicine home visit programmes have not been established and evidence to inform their creation is lacking. We sought to investigate the relevance of assessment items to the practice of community paramedics according to a pre-established clarity-utility matrix.DesignWe designed a modified-Delphi study consisting of predetermined thresholds for achieving consensus, number of rounds of for scoring items, a defined meeting and discussion process, and a sample of participants that was purposefully representative.Setting and participantsWe established a panel of 26 community paramedics representing 20 municipal paramedic services in Ontario, Canada. The sample represented a majority of paramedic services within the province that were operating a community paramedicine home visit programme.MeasuresDrawing from a bank of standardised assessment items grouped according to domains aligned with the International Classification on Functioning, Disability, and Health taxonomy, 64 previously pilot-tested assessment items were scored according to their clarity (being free from ambiguity and easy to understand) and utility (being valued in care planning or case management activities). Assessment items covered a broad range of health, social and environmental domains. To conclude scoring rounds, assessment items that did not achieve consensus for relevance to assessment practices were discussed among participants with opportunities to modify assessment items for subsequent rounds of scoring.ResultsResulting from the first round of scoring, 54 assessment items were identified as being relevant to assessment practices and 3 assessment items were removed from subsequent rounds. The remaining 7 assessment items were modified, with some parts removed from the final items that achieved consensus in the final rounds of scoring.ConclusionA broadly representative panel of community paramedics identified consensus for 61 assessment items that could be included in a structured, multidomain, assessment instrument for guiding practice in community paramedicine home visit programmes.Trail registration numberISRCTN58273216.

Project description:BackgroundThe number of e-mental health apps is increasing rapidly. Studies have shown that the use of some apps is beneficial, whereas others are ineffective or do not meet users' privacy expectations. Individuals and organizations that curate, recommend, host, use, or pay for apps have an interest in categorizing apps according to the consensus criteria of usability and effectiveness. Others have previously published recommendations for assessing health-related apps; however, the extent to which these recommendations can be generalized across different population groups (eg, culture, gender, and language) remains unclear. This study describes an attempt by Canadian stakeholders to develop an e-mental health assessment framework that responds to the unique needs of people living in Canada in an evidence-based manner.ObjectiveThe objective of our study was to achieve consensus from a broad group of Canadian stakeholders on guiding principles and criteria for a framework to assess e-mental health apps in Canada.MethodsWe developed an initial set of guiding principles and criteria from a rapid review and environmental scan of pre-existing app assessment frameworks. The initial list was refined through a two-round modified Delphi process. Participants (N=25) included app developers and users, health care providers, mental health advocates, people with lived experience of a mental health problem or mental illness, policy makers, and researchers. Consensus on each guideline or criterion was defined a priori as at least 70% agreement. The first round of voting was conducted electronically. Prior to Round 2 voting, in-person presentations from experts and a persona empathy mapping process were used to explore the perspectives of diverse stakeholders.ResultsOf all respondents, 68% (17/25) in Round 1 and 100% (13/13) in Round 2 agreed that a framework for evaluating health apps is needed to help Canadian consumers identify high-quality apps. Consensus was reached on 9 guiding principles: evidence based, gender responsive, culturally appropriate, user centered, risk based, internationally aligned, enabling innovation, transparent and fair, and based on ethical norms. In addition, 15 informative and evaluative criteria were defined to assess the effectiveness, functionality, clinical applicability, interoperability, usability, transparency regarding security and privacy, security or privacy standards, supported platforms, targeted users, developers' transparency, funding transparency, price, user desirability, user inclusion, and meaningful inclusion of a diverse range of communities.ConclusionsCanadian mental health stakeholders reached the consensus on a framework of 9 guiding principles and 15 criteria important in assessing e-mental health apps. What differentiates the Canadian framework from other scales is explicit attention to user inclusion at all stages of the development, gender responsiveness, and cultural appropriateness. Furthermore, an empathy mapping process markedly influenced the development of the framework. This framework may be used to inform future mental health policies and programs.

Project description:ObjectivesTo evaluate whether the Morphological Uterus Sonographic Assessment (MUSA) features of adenomyosis need to be better defined and, if deemed necessary, to reach consensus on the updated definitions.MethodsA modified Delphi procedure was performed among European gynecologists with expertise in ultrasound diagnosis of adenomyosis. To identify MUSA features that might need revision, 15 two-dimensional (2D) video recordings (four recordings also included three-dimensional (3D) still images) of transvaginal ultrasound (TVS) examinations of the uterus were presented in the first Delphi round (online questionnaire). Experts were asked to confirm or refute the presence of each of the nine MUSA features of adenomyosis (described in the original MUSA consensus statement) in each of the 15 videoclips and to provide comments. In the second Delphi round (online questionnaire), the results of the first round and suggestions for revision of MUSA features were shared with the experts before they were asked to assess a new set of 2D and 3D still images of TVS examinations and to provide feedback on the proposed revisions. A third Delphi round (virtual group meeting) was conducted to discuss and reach final consensus on revised definitions of MUSA features. Consensus was predefined as at least 66.7% agreement between experts.ResultsOf 18 invited experts, 16 agreed to participate in the Delphi procedure. Eleven experts completed and four experts partly finished the first round. The experts identified a need for more detailed definitions of some MUSA features. They recommended use of 3D ultrasound to optimize visualization of the junctional zone. Fifteen experts participated in the second round and reached consensus on the presence or absence of ultrasound features of adenomyosis in most of the still images. Consensus was reached for all revised definitions except those for subendometrial lines and buds and interrupted junctional zone. Thirteen experts joined the online meeting, in which they discussed and agreed on final revisions of the MUSA definitions. There was consensus on the need to distinguish between direct features of adenomyosis, i.e. features indicating presence of ectopic endometrial tissue in the myometrium, and indirect features, i.e. features reflecting changes in the myometrium secondary to presence of endometrial tissue in the myometrium. Myometrial cysts, hyperechogenic islands and echogenic subendometrial lines and buds were classified unanimously as direct features of adenomyosis. Globular uterus, asymmetrical myometrial thickening, fan-shaped shadowing, translesional vascularity, irregular junctional zone and interrupted junctional zone were classified as indirect features of adenomyosis.ConclusionConsensus between gynecologists with expertise in ultrasound diagnosis of adenomyosis was achieved regarding revised definitions of the MUSA features of adenomyosis and on the classification of MUSA features as direct or indirect signs of adenomyosis. © 2021 The Authors. Ultrasound in Obstetrics & Gynecology published by John Wiley & Sons Ltd on behalf of International Society of Ultrasound in Obstetrics and Gynecology.

Project description:BackgroundTelehealth is becoming more prevalent in physical therapy, involving a whole host of clinical services. These services are often provided without structured training in telehealth, and no formal curricula currently exist for this purpose.ObjectiveTo develop a set of educational competencies (ECs) to guide instruction of telehealth-related skills in entry-level programs (i.e., Doctor of Physical Therapy), existing programs (i.e., residencies and fellowships), and potential future post-graduate programs specific to telehealth physical therapy.MethodsPhysical therapists and physical therapist assistants from diverse geographic locations and practice areas were invited to participate on an expert panel. A modified Delphi process was then used to evaluate the acceptability of draft ECs gathered from the extant literature by a steering group. Draft ECs were presented to the expert panel on a questionnaire, which asked expert participants to rate each draft EC according to applicability and clarity. Draft ECs were accepted if they met a priori established criteria for acceptability and clarity. Unendorsed ECs were revised by the steering group according to open-ended comments from respondents and presented during a subsequent round. Three rounds of surveys were undertaken.ResultsThirty-eight participants formed the expert panel; 38 participants completed the Round 1 survey, 28 participants completed the Round 2 survey, and 24 participants completed the Round 3 survey. Delphi group members approved 48 ECs in the first round, 23 ECs in the second round, and 2 ECs in the third round. There were 4 ECs that remained unendorsed after the modified Delphi process. Endorsed ECs spanned 7 conceptual areas. Distinct sets of ECs characterized expected end points of first professional degree, existing residency and fellowship, and potential future telehealth physical therapy post-graduate program.ConclusionsConsensus-based ECs identified in this study may guide instruction in knowledge and skills relevant to physical therapy telehealth.

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The evolving value assessment of cancer therapies: Results from a modified Delphi study

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