Skip to main content

Table 4 Respondent propensity to prescribe off-label therapy

From: Scenario drafting for early technology assessment of next generation sequencing in clinical oncology

Level of evidence Adjuvant (n = 16) Metastatic (n = 26)
At least validated by an RCT3 in another type of cancer and an observational study for the type cancer you intent to treat 6/16; 37,5 % 7/26; 27,0 %
At least validated by a RCT3 for another type of cancer 2/16; 12,5 % 3/26; 11,5 %
At least validated by an observational study in another type of cancer 4/16; 25 % 6/26; 23,1 %
Other, namely never 2/16; 12,5 % 1/26; 0,04 %
Other, namely descriptive: “tissue-based labelling should be changed” (1/16; 6,25 %), “Bayesian approach should be used” (1/16; 6,25 %) “only as part of a trial” (2/26; 0,08 %) and 7 individual comments (0,04 % each) including “Based on RCTII data”, “Based on RCTII with molecularly selected patients”, “Casuistic evidence from other disease entities”, “Any time”, “tissue-based labelling should be changed”, “Bayesian approach should be used”, “Depending on costs”
  1. Respondents were presented with the following hypothetical situation: “A NGS gene panel was only able to identify one molecular target in a patient’s tumour. However, the corresponding targeted therapy has not been registered for that type of cancer yet, thus off-label treatment may be the only option” They were then asked based on what level of evidence and stage of disease they would prescribe the therapy. The question regarding the metastatic setting was asked in all versions of the questionnaire, while the question for the adjuvant setting was only posed in the physicians and policy version. Therefore, the number of respondents per column differs