Issues, Challenges and Opportunities for Economic Evaluations of Orphan Drugs in Rare Diseases: An Umbrella Review
ConclusionsThe results highlighted issues around a considerable paucity of evidence for economic evaluations and few cost-effectiveness analyses, supporting the notion that a paucity of evidence makes economic evaluations of rare diseases more challenging compared with more prevalent diseases. Furthermore, we provide recommendations for more sustainable approaches in economic evaluations of rare diseases. (Source: PharmacoEconomics)
Source: PharmacoEconomics - April 14, 2024 Category: Health Management Source Type: research

A Blueprint for Multi-use Disease Modeling in Health Economics: Results from Two Expert-Panel Consultations
ConclusionsMUDMs would improve on current decision support regarding cost-effectiveness information. Given feasibility challenges, this would be most relevant for diseases with multiple treatments, large burden of disease and requiring more complex models. The current overview offers policy makers a starting point to organize the development, use, and maintenance of MUDMs and to support choices concerning  which diseases and policy decisions they will be helpful for. (Source: PharmacoEconomics)
Source: PharmacoEconomics - April 13, 2024 Category: Health Management Source Type: research

Barriers and Facilitators of Using R for Decision Analytic Modeling in Health Technology Assessment: Focus Group Results
ConclusionsThere is a need for standardization, which can facilitate decision model sharing, coding homogeneity, and improved country adaptations. The creation of training materials and tailored workshops was identified as a key short-term facilitator. Increased communication and engagement of stakeholders could also facilitate the use of R by identifying needs and opportunities, encouraging HTA agencies to address structural barriers, and increasing incentives to use R. (Source: PharmacoEconomics)
Source: PharmacoEconomics - April 12, 2024 Category: Health Management Source Type: research

Is Risk-Stratifying Patients with Colorectal Cancer Using a Deep Learning-Based Prognostic Biomarker Cost-Effective?
Conclusions Risk-stratifying patients with CRC using Histotyping prior to the administration of adjuvant chemotherapy is likely to be a cost-effective strategy in Norway. (Source: PharmacoEconomics)
Source: PharmacoEconomics - April 7, 2024 Category: Health Management Source Type: research

Value of Information for Clinical Trial Design: The Importance of Considering All Relevant Comparators
We present two hypothetical examples to demonstrate that VOI methods are only accurate when (1) all feasible comparators are included in the decision model when designing research, and (2) all comparators are retained in the decision model once the data have been collected and a final treatment recommendation is made. Omitting comparators from either the design or analysis phase of research when using VOI methods can lead to incorrect trial designs and/or treatment recommendations. Overall, we conclude that incorrectly specifying the health economic model by ignoring potential comparators can lead to misleading VOI results...
Source: PharmacoEconomics - April 7, 2024 Category: Health Management Source Type: research

Is Risk-Stratifying Patients with Colorectal Cancer Using a Deep Learning-Based Prognostic Biomarker Cost-Effective?
Conclusions Risk-stratifying patients with CRC using Histotyping prior to the administration of adjuvant chemotherapy is likely to be a cost-effective strategy in Norway. (Source: PharmacoEconomics)
Source: PharmacoEconomics - April 7, 2024 Category: Health Management Source Type: research

Value of Information for Clinical Trial Design: The Importance of Considering All Relevant Comparators
We present two hypothetical examples to demonstrate that VOI methods are only accurate when (1) all feasible comparators are included in the decision model when designing research, and (2) all comparators are retained in the decision model once the data have been collected and a final treatment recommendation is made. Omitting comparators from either the design or analysis phase of research when using VOI methods can lead to incorrect trial designs and/or treatment recommendations. Overall, we conclude that incorrectly specifying the health economic model by ignoring potential comparators can lead to misleading VOI results...
Source: PharmacoEconomics - April 7, 2024 Category: Health Management Source Type: research

Childhood Transitions Between Weight Status Categories: Evidence from the UK Millennium Cohort Study
ConclusionsOur models generated estimates of bodyweight status transitions in a representative UK childhood population. Compared to our scenario models (i.e., time-homogeneous transition rates), our base-case model fits the observed data best, indicating a non-time-homogeneous pattern in transitions between bodyweight categories during childhood. Transition hazards varied significantly by age and across subpopulations, suggesting that conducting subgroup-specific cost-effectiveness analyses of childhood weight management interventions will optimize decision-making. (Source: PharmacoEconomics)
Source: PharmacoEconomics - April 3, 2024 Category: Health Management Source Type: research

Challenges and Opportunities in Interdisciplinary Research and Real-World Data for Treatment Sequences in Health Technology Assessments
AbstractWith an ever-increasing number of treatment options, the assessment of treatment sequences has become crucial in health technology assessment (HTA). This review systematically explores the multifaceted challenges inherent in evaluating sequences, delving into their interplay and nuances that go beyond economic model structures. We synthesised a ‘roadmap’ of literature from key methodological studies, highlighting the evolution of recent advances and emerging research themes. These insights were compared against HTA guidelines to identify potential avenues for future research. Our findings reveal a spectrum of c...
Source: PharmacoEconomics - April 1, 2024 Category: Health Management Source Type: research

Quality Appraisal in Systematic Literature Reviews of Studies Eliciting Health State Utility Values: Conceptual Considerations
ConclusionsAchieving this consensus marks a pivotal step towards developing a QA tool specific to systematic reviews of studies eliciting HSUVs. Future research will build on this foundation, identify QA items, signalling questions and response options, and develop a QA tool specific to studies eliciting HSUVs. (Source: PharmacoEconomics)
Source: PharmacoEconomics - March 29, 2024 Category: Health Management Source Type: research