Do value judgments play a role in evidence-based recommendations when information is based on proof of concept?

Article type
Authors
Hutton B1, Lee K1, O’Grady T1, Shukla V1
1Canadian Agency for Drugs and Technologies in Health, Canada
Abstract
Background: The Canadian Expert Drug Advisory Committee (CEDAC) delivers evidence-informed formulary recommendations for drug reimbursement based on data from clinical systematic reviews and critiques of health economic information. Past work suggests that value judgments play a role in evidence interpretation and recommendations.

Objectives: To explore the role of value judgments in CEDAC recommendations.

Methods: CEDAC recommendations from 2009-2010 were reviewed. Recommendations were evaluated which had (1) a partial or full listing, where evidence consisted of only placebo controlled trials; and (2) a do not list recommendation, where evidence consisted of only active comparator trials. Information was gathered on factors potentially influencing the recommendation, such as: drug novelty (i.e. first drug for indication or first in drug class); indication risk and rarity; insufficiency of evidence; study quality; economic considerations; and harms concerns.

Results: Nine partial or full listing recommendations were identified based on only placebo controlled trials, and 8 'do not list’ recommendations, based only on active comparator trials, were identified. Of the nine 'list’ recommendations, factors were related to therapeutic need (3/9) and potential for cost savings (6/9). Reasons underlying the 8 'do not listá recommendations related to cost/cost-effectiveness (8/8; increased cost or uncertainty around cost-effectiveness), insufficiency of evidence versus comparators (7/8; lack of studies versus key comparators, lack of meaningful improvements over other active therapies, inconsistency of findings across studies), and study quality (6/8; issues regarding blinding or withdrawals/missing data, appropriateness of non-inferiority margin, other design issues). For the 'do not list’ category, recommen- dations were largely made in consideration of ≥2 criteria.Conclusions: The absence of active comparator trials may not be the determinant of a recommendation as value judgments related to the reliability of clinical trial data, therapeutic need, and other factors play a role in CEDAC recommendations.