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.