Development of the First Value Assessment Index System for Off-Label Use of Antineoplastic Agents in China: A Delphi Study

Abstract

Objective: To develop the first value assessment index system for off-label use of antineoplastic agents in China. Methods: A modified two-round Delphi method was employed to establish consensus within a field to reach agreement via a questionnaire or doing interview among a multidisciplinary panel of experts by collecting their feedback to inform the next round, exchanging their individual knowledge, experience, and opinions anonymously, and resolving uncertainties. Results: Expert's positive coefficient was 94.74% in the first round and 100.00% in the second round. In the first round, expert's authority coefficient for a majority of 61 indicators was ≥ 0.80 (85.2%, ranging from 0.70 to 0.89, mean=0.84) and coefficient of variation for all the 61 indicators was ≥ 22% (ranging from 11.67% to 21.74%, mean=17.4%). In two rounds, the mean expert's authority coefficient raised to 0.85 (ranging from 0.75 to 0.90), and coefficient of variation for all indicators was < 20% (ranging from 10.49% to 19.71%, mean=15.97%). The P-values of Kendall's W test were all < 0.001 for each round. At the end of two rounds, W-value for concordance was 0.395 (c 2 =347.494, P<0.0001). The final value assessment index system comprised of eight domains, 21 subdomains, and 56 indicators. The weight and combination weight of each domain were 0.4211 for therapeutic value, 0.1678 for source and type of evidence, 0.0961 for public feedback/ comments, 0.0894 for novelty in drug discovery, 0.0689 for grading of evidence recommendation, 0.0578 for consistency of evidence results, 0.0561 for disease burden, and 0.0428 for ratio of composition/integration. Conclusion: Use of Delphi method to develop the proposed value assessment index system was found scientific and credible. This value assessment index system is highly appropriate for off-label use of antineoplastic agents in China.

Developing a Risk Assessment Tool for Cancer-related Venous Thrombosis in China: A modified Delphi-Analytic Hierarchy Process study

“…Generally, a coe cient exceeding 0.70 is considered reliable. Additionally, Kendall's concordance coe cient W was 0.190 and 0.132 in the two-round survey, respectively, indicating that all experts had a consistent and high opinion [21] .…”

Recommendations on Off-Label Drug Use in Pediatric Guidelines

“…As a result, it is critical that recommendations on the off-label use of drugs in pediatric guidelines are based on a reliable methodological pathway or framework ( Cheng et al, 2007 ). However, there is currently no standardized framework for creating recommendations for the use of off-label drugs in children ( Casali et al, 2015 ; Agrawal et al, 2019 ; Jiang et al, 2020 ; Stolbach et al, 2020 ; Remi et al, 2021 ). In general, the development of clinical guidelines should follow the GRADE Working Group’s “Five Paradigmatic Situations Warranting Strong Recommendation Despite Low or Very Low-Quality Evidence in Effect Estimates,” which has proven to offer a reliable framework ( Andrews et al, 2013 ).…”

Development of the Practice of Pharmaceutical Care for Cancer Pain Management in Outpatient Clinics Using the Delphi Method

“…The definitions of consensus were a combination of an average score ≥4, the percentage of experts rating the scores at >4 points, and the coefficient of variation of the scores. The experts were also asked to self-rate themselves on the authority (Cr) for each round, which was determined by the judgment criteria (Ca) and their familiarities (Cs) with the clinical issues ( Jiang et al, 2020 ). Ca includes four dimensions: work experience, theoretical analysis, understanding from domestic and foreign counterparts, and insights, and Cs include five levels: very familiar, familiar, generally familiar, unfamiliar, and very unfamiliar, which were quantified as 1.0, 0.8, 0.6, 0.4, and 0.2, respectively ( Supplementary Table S1 ).…”