Gatekeepers for pragmatic clinical trials.

To successfully implement a pragmatic clinical trial, investigators need access to numerous resources, including financial support, institutional infrastructure (e.g. clinics, facilities, staff), eligible patients, and patient data. Gatekeepers are people or entities who have the ability to allow or deny access to the resources required to support the conduct of clinical research. Based on this definition, gatekeepers relevant to the US clinical research enterprise include research sponsors, regulatory agencies, payers, health system and other organizational leadership, research team leadership, human research protections programs, advocacy and community groups, and clinicians. This article provides a framework to help guide gatekeepers' decision-making related to the use of resources for pragmatic clinical trials. Relevant ethical considerations for gatekeepers include (1) concern for the interests of individuals, groups, and communities affected by the gatekeepers' decisions, including protection from harm and maximization of benefits; (2) advancement of organizational mission and values; and (3) stewardship of financial, human, and other organizational resources. Separate from these ethical considerations, gatekeepers' actions will be guided by relevant federal, state, and local regulations. This framework also suggests that to further enhance the legitimacy of their decision-making, gatekeepers should adopt transparent processes that engage relevant stakeholders when feasible and appropriate. We apply this framework to the set of gatekeepers responsible for making decisions about resources necessary for pragmatic clinical trials in the United States, describing the relevance of the criteria in different situations and pointing out where conflicts among the criteria and relevant regulations may affect decision-making. Recognition of the complex set of considerations that should inform decision-making will guide gatekeepers in making justifiable choices regarding the use of limited and valuable resources.

Comparative effectiveness research in Ontario, Canada: producing relevant and timely information for health care decision makers.

CONTEXT: Comparative effectiveness research is increasingly being recognized as a method to link research with the information needs of decision makers. As the United States begins to invest in comparative effectiveness, it would be wise to look at other functioning research networks to understand the infrastructure and funding required to support them. METHODS: This case study looks at the comparative effectiveness research network in Ontario, Canada, for which a neutral coordinating committee is responsible for prioritizing topics, assessing evidence, providing recommendations on coverage decisions, and determining pertinent research questions for further evaluation. This committee is supported by the Medical Advisory Secretariat and several large research institutions. This article analyzes the infrastructure and cost needed to support this network and offers recommendations for developing policies and methodologies to support comparative effectiveness research in the United States. FINDINGS: The research network in place in Ontario explicitly links decision making with evidence generation, in a transparent, timely, and efficient way. Funding is provided by the Ontario government through a reliable and stable funding mechanism that helps ensure that the studies it supports are relevant to decision makers. CONCLUSIONS: With the recent allocation of funds to support comparative effectiveness research from the American Recovery and Reinvestment Act, the United States should begin to construct an infrastructure that applies these features to make sure that evidence generated from this effort positively affects the quality of health care delivered to patients.

Deliberative Engagement Methods for Patient-Centered Outcomes Research.

There is growing emphasis on eliciting and incorporating stakeholder perspectives into health research and public policy development. The deliberative engagement session (DES) method provides one approach to elicit informed preferences from patients and other stakeholders on policy issues. DES involves day-long interaction with participants, including short plenary presentations followed by small group discussion. While interest in DES methods is expanding, practical guidance for researchers on this method remains limited. In this paper, we describe the DES method and its contemporary relevance for health policy research, illustrate how to conduct a DES using an example of a recent patient-centered outcomes research (PCOR) study with which we were involved, and discuss strengths and challenges of using this approach. DES methods generate rich data, reduce the risk of eliciting uniformed preferences or non-attitudes, and increase the likelihood of eliciting informed, reflective preferences. However, they are resource-intensive, and thus generally require trading away a larger, more representative sample. Despite these limitations, the DES method, when carefully designed, is well-suited for engaging stakeholders in research on complex health policy issues.

Ethical Issues in Patient Safety Research: A Systematic Review of the Literature.

As many as 1 in 10 patients is harmed while receiving hospital care in wealthy countries. The risk of health care-associated infection in some developing countries is as much as 20 times higher. In response, in many global regions, increased attention has turned to the implementation of a broad program of safety research, encompassing a variety of methods. Although important international ethical guidelines for research exist, literature has been emerging in the last 20 years that begins to apply such guidelines to patient safety research specifically. This paper provides a review of the literature related to ethics, oversight, and patient safety research; identifies issues highlighted in articles as being of ethical relevance; describes areas of consensus regarding how to respond to these ethical issues; and highlights areas where additional ethical analysis and discussion are needed to provide guidance to those in the field.