Expert Elicitation for Estimation

IntroductionIn 2018, the National Centre for Pharmacoeconomics (NCPE) was commissioned to conduct a health technology assessment (HTA) of one of the first commercially available chimeric antigen receptor (CAR) T-cell therapies, tisagenlecleucel. CAR T-cells are a major advance in personalized cancer treatment, demonstrating promising outcomes in relapsed/refractory pediatric acute lymphoblastic leukemia (pALL). However, the results are based on short-term follow up, limiting their value in predicting long-term survival and leading to uncertainty about the most appropriate survival modeling method to employ. This study aimed to address these limitations by means of expert elicitation.MethodsAn expert elicitation method, the histogram technique, was employed. A predefined discrete numerical scale was presented in Microsoft Excel® and the expert was asked to place twenty crosses on a frequency chart. These crosses represented the expert's beliefs about the distribution of particular quantities. Each cross represented five percent of the probabilistic distribution. Individual distributions were then aggregated across experts using linear pooling.ResultsA total of seventeen experts were invited to take part; eight agreed to participate and five completed the exercise. Three experts did not consider tisagenlecleucel to be a “curative” therapy because patients had a higher risk of death, compared with the age- and sex-matched general population. The aggregated distributions indicated the five-year overall survival rate to be thirty-three percent (95% CI 8.65–56.88) in patients who do not receive a subsequent stem cell transplant and twenty percent (95% CI 2.38 -52.04) in those who do.ConclusionsThe results of this study will be used to calibrate CD19 CAR T-cell therapy survival estimates presented in HTA submissions to the NCPE to ensure more robust assessments. They will also be used to inform the construction of a de novo cost-utility model for examining the cost effectiveness of CD19 CAR T-cell therapies for relapsed/refractory pALL in the Irish healthcare setting.

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A Framework for Risk-Based Cost–Benefit Analysis for Decision Support on Hydrogeological Risks in Underground Construction

Geosciences ◽

10.3390/geosciences11020082 ◽

2021 ◽

Vol 11 (2) ◽

pp. 82

Author(s):

Johanna Merisalu ◽

Jonas Sundell ◽

Lars Rosén

Keyword(s):

Decision Support ◽

Cost Benefit Analysis ◽

Risk Mitigation ◽

Ground Surface ◽

Cost Benefit ◽

Expert Elicitation ◽

Benefit Analysis ◽

Underground Construction ◽

Groundwater Leakage ◽

Risk Reducing

Construction below the ground surface and underneath the groundwater table is often associated with groundwater leakage and drawdowns in the surroundings which subsequently can result in a wide variety of risks. To avoid groundwater drawdown-associated damages, risk-reducing measures must often be implemented. Due to the hydrogeological system’s inherent variability and our incomplete knowledge of its conditions, the effects of risk-reducing measures cannot be fully known in advance and decisions must inevitably be made under uncertainty. When implementing risk-reducing measures there is always a trade-off between the measures’ benefits (reduced risk) and investment costs which needs to be balanced. In this paper, we present a framework for decision support on measures to mitigate hydrogeological risks in underground construction. The framework is developed in accordance with the guidelines from the International Standardization Organization (ISO) and comprises a full risk-management framework with focus on risk analysis and risk evaluation. Cost–benefit analysis (CBA) facilitates monetization of consequences and economic evaluation of risk mitigation. The framework includes probabilistic risk estimation of the entire cause–effect chain from groundwater leakage to the consequences of damage where expert elicitation is combined with data-driven and process-based methods, allowing for continuous updating when new knowledge is obtained.

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The burden of cancer in Belgium, 2004-2017

European Journal of Public Health ◽

10.1093/eurpub/ckaa165.1012 ◽

2020 ◽

Vol 30 (Supplement_5) ◽

Author(s):

A Cornez ◽

G Silversmit ◽

V Gorasso ◽

I Grant ◽

G M A Wyper ◽

...

Keyword(s):

Colorectal Cancer ◽

Lung Cancer ◽

Burden Of Disease ◽

Disease Burden ◽

Expert Elicitation ◽

Years Of Life Lost ◽

Cancer Burden ◽

Life Years ◽

Belgian Population ◽

The Impact

Abstract Background Monitoring the health status of a population requires consistent and comparable data on the morbidity and mortality impacts of a disease. The Disability-Adjusted Life Year (DALY) is an increasingly used disease burden indicator, combining healthy life years lost due to living with disease (Years Lived with Disability; YLDs) and due to dying prematurely (Years of Life Lost; YLLs). In Belgium, as in many other developed countries, cancer is a major contributor to the overall burden of disease. To date, however, local estimates of the burden of cancer are lacking. Methods We estimated the burden of 48 cancers in Belgium from 2004 to 2017 in terms of DALYs, using national population-based cancer registry data and international disease models. We developed a microsimulation model to translate incidence- into prevalence-based estimates, and used expert elicitation to integrate the long-term impact of surgical treatment. Results In 2017, in Belgium, breast cancer was the cancer with the highest disease burden among women, followed by lung cancer and colorectal cancer. Among men, lung cancer had the highest disease burden, followed by colorectal cancer and prostate cancer. Between 2004 and 2017, the burden of lung cancer increased by more than 50% in women, while in both sexes, significant increases were observed in melanoma and skin cancer burden. The majority of the cancer burden remained linked to premature mortality. Conclusions Cancer maintains a major impact on the health of the Belgian population. Current resources allocated for their prevention and treatment will need to be maintained to further reduce the cancer burden. Lung cancer remains a crucial challenge, among both men and women, calling for strengthened tobacco control policies. Integrating the current study in the Belgian national burden of disease study (BeBOD) will allow monitoring the burden of cancer over time, highlighting new trends and assessing the impact of public health policies. Key messages Burden of disease studies allow assessing and monitoring the impact of diseases and risk factors in a comparable way. Cancer maintains a major impact on the health of the Belgian population; lung cancer in particular remains a crucial challenge.

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Response (minimum clinically relevant change) in ASD symptoms after an intervention according to CARS-2: consensus from an expert elicitation procedure

European Child & Adolescent Psychiatry ◽

10.1007/s00787-021-01772-z ◽

2021 ◽

Author(s):

Lucie Jurek ◽

Matias Balthazar ◽

Sheffali Gulati ◽

Neda Novakovic ◽

María Núñez ◽

...

Keyword(s):

Autism Spectrum Disorder ◽

Rating Scale ◽

Expert Knowledge ◽

Scientific Evidence ◽

Autism Spectrum ◽

Expert Elicitation ◽

Spectrum Disorder ◽

Initial Finding ◽

Elicitation Process ◽

Core Symptoms

AbstractThe lack of consensual measures to monitor core change in Autism Spectrum Disorder (ASD) or response to interventions leads to difficulty to prove intervention efficacy on ASD core symptoms. There are no universally accepted outcome measures developed for measuring changes in core symptoms. However, the CARS (Childhood Autism Rating Scale) is one of the outcomes recommended in the EMA Guideline on the clinical development of medicinal products for the treatment of ASD. Unfortunately, there is currently no consensus on the response definition for CARS among individuals with ASD. The aim of this elicitation process was to determine an appropriate definition of a response on the CARS2 scale for interventions in patients with Autism Spectrum Disorder (ASD). An elicitation process was conducted following the Sheffield Elicitation Framework (SHELF). Five experts in the field of ASD and two experts in expert knowledge elicitation participated in an 1-day elicitation workshop. Experts in ASD were previously trained in the SHELF elicitation process and received a dossier of scientific evidence concerning the topic. The response definition was set as the mean clinically relevant improvement averaged over all patients, levels of functioning, age groups ***and clinicians. Based on the scientific evidence and expert judgment, a normal probability distribution was agreed to represent the state of knowledge of this response with expected value 4.03 and standard deviation 0.664. Considering the remaining uncertainty of the estimation and the available literature, a CARS-2 improvement of 4.5 points has been defined as a threshold to conclude to a response after an intervention. A CARS-2 improvement of 4.5 points could be used to evaluate interventions' meaningfulness in indivudals. This initial finding represents an important new benchmark and may aid decision makers in evaluating the efficacy of interventions in ASD.

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