Variable | Values | Details |
---|---|---|
Recommendation | 0 if negative 1 if positive | • Negative: do not list • Positive: list, list with conditions, list with criteria, list if price reduced or cost-effectiveness improved |
Submission characteristics | ||
Year of recommendation | Continuous variable | • Year of final recommendation |
Type of submission | 0 if new submission 1 if resubmission | • Type of submission according to CADTH classification |
Presence of RCTs | 0 if no 1 if yes | • RCTs were included in the systematic review |
Therapeutic class of drugs | 0 if alimentary tract & metabolism 1 if Antineoplastic & immunomodulating 2 if other | • Classification of drugs based on ATC codes |
Characteristics of disease | ||
Type of condition | 0 if cancer 1 if non-cancer | • Classification based on ICD-10 |
Prevalence | 0 if ultra-orphan 1 if orphan | • Ultra-orphan: < 1 in 100,000 people • Orphan: < 1 in 2000 people |
Clinical need | 0 if no or not stated 1 if yes | • Need for alternative treatment options, no existing treatment or “unmet need” |
Clinical safety/efficacy | ||
Safety issues | 0 if yes 1 if no | • Concerns over potential serious life-threatening adverse events or unknown safety profiles |
Improvements in biomarker/ surrogate outcome | 0 if no, inconsistent or not measured 1 if yes | • Biomarker is “a defined characteristic that is measured as an indicator of normal biological process, pathogenic process, or responses to an exposure or intervention, including therapeutic interventions” [17]. • Surrogate outcome is “an endpoint that is used in clinical trials as a substitute for a direct measure of how a patient feels, functions, or survives” [17]. • Meaningful improvements defined as statistically significant differences or non-inferiority in biomarker/ surrogate outcomes (e.g. weight, 6 min walk test, progression-free survival) |
Improvements in clinical outcomes | 0 if no, inconsistent or not measured 1 if yes | • Clinical outcome is “an outcome that describes or reflects how an individual feels, functions or survives” [17]. • Meaningful improvements defined as statistically significant differences or non-inferiority in clinical outcomes (e.g. survival, transplantation) |
Improvements in PRO | 0 if no, inconsistent or not measured 1 if yes | • PRO is “a measurement based on a report that comes directly from the patient about the status of a patient’s health condition without amendment or interpretation of the patient’s response by a clinician or anyone else” [17]. • Meaningful improvements defined as statistically significant differences or non-inferiority in PRO (e.g. QOL, rating of pain intensity, SF-36) |
Quality of evidence | ||
Availability of comparative data | 0 if no 1 if yes | • Based on availability of direct head-to-head comparative studies (where comparators were available) |
Consistency between population in trials and indications | 0 if no 1 if yes | • Present when ‘final recommendation’ document stated that data from trials included all subgroup of the indicated population • Not present when for example submitted indication includes mild, moderate, and severe forms of disease but trial data limited to mild-moderate forms of disease |
Bias in outcome measures | 0 if yes 1 if no | • Present when indicated in the final recommendation document • Bias in outcome measurements (e.g., subjective outcomes classified by non-blinded investigators) |
Long term data | 0 if no 1 if yes | • Presence of long-term data where long-term data is important given the course of disease • Present when indicated in the final recommendation document |
Other study design issues | 0 if yes 1 if no | • Concerns over other aspects of study design (e.g., small sample size, carry-over effects associated with withdrawal trial methodology) • Present when indicated in the final recommendation document |
Cost/ cost-effectiveness | ||
Daily treatment cost | Continuous variable in $CDN/ patient | • Average daily treatment cost of drugs per patient |
ICER | Continuous variable in $CDN/QALY | • ICER calculated by CADTH or by manufacturer if no ICER calculated by CADTH was available |