Designed delays versus rigorous pragmatic trials: lower carat gold standards can produce relevant drug evaluations.

BACKGROUND: Centralized administrative databases enable low-cost pragmatic randomized trials (PRTs) of drug effectiveness and safety. We simplified the PRT strategy by using designed delays (DD) to evaluate drug policies.
OBJECTIVES: To reassess our DD trial of a cost-saving nebulizer-to-inhaler conversion policy and a proposed DD trial of reduced restrictions on Cox-2 inhibitors. RESEARCH
DESIGN: We randomized 52 pairs of communities and clusters of physician practices to the policy either on time or after a 6-month delay. Our 2-stage qualitative reassessment comprised: (1) applying criteria for reporting PRTs and (2) assessing DD trials in 3 domains of responsibility: policymakers' decisions, researchers' decisions, and joint decisions involving negotiation. MEASURES: A draft checklist of 22 Consolidated Standards of Reporting Trials (CONSORT). Researchers' recollections of their degree of influence on decisions.
RESULTS: DD trials deviated from ideal PRTs in the policymakers' domain: the policies affected mixtures of drugs, users, and illnesses, and implementation was not by strict protocol. Aspects negotiated by researchers and policymakers also deviated from ideal: length of delay; size and location of control group; unit of randomization; additional data collection; and communications to physicians. The DD trials complied better with CONSORT in the researchers' domain of analysis and interpretation.
CONCLUSIONS: DD trials can be negotiated with policymakers. Low cost and simplicity of DD trials partly compensate for some limitations for evaluating drug safety and effectiveness. The ethics question of whether a DD is routine evaluation or research depends on its purpose and generalizability.