Acute stroke treatment trials in the United States. Rethinking strategies for success.

Abstract

In the recent Humana Lecture,1 Harold Adams quoted what might be the prevailing sentiment guiding the search for a treatment for acute ischemic stroke: “When we have a treatment for stroke, we will know it.” The implicit assumption is that there is a treatment that will dramatically improve acute stroke outcome. The current approach to finding a treatment for ischemic stroke is based on single-agent trials, sponsored mainly by pharmaceutical companies, that may produce a “winner,” to be followed by a “therapeutic cocktail” to combat the complex events set in motion by cerebral ischemia. Progress in ischemic stroke treatment trials over the last two decades has included improved statistical design, radically shortened entry windows, standardized rating instruments, and shared sponsorship between industry and government. We wish to raise concerns about the current approach to testing new stroke treatments and the impact that changes in healthcare delivery in the United States may have on the conduct of acute stroke trials. The success of clinical research in the treatment of acute stroke depends not only on trial design but on local, regional, and national factors that determine the environment in which acute stroke care is provided and that affect the feasibility of trial execution. In this editorial, we present some of the important issues facing those who design and implement acute stroke treatment studies. Our opinions pertain primarily to the medical environment in the United States. Our goal is to stimulate discussion and suggest a forum for the critical evaluation of trial design issues and also the development of a national strategy for stroke. We are concerned about the optimal use of critical resources needed to conduct stroke treatment trials. By “resources” we mean the investigators and stroke research teams; acute stroke patients available for enrollment; cooperative referring doctors and emergency-services …