Methodological Guidance for the Design of Pharmaceutical Pragmatic Clinical Trials:

A major goal of comparative effectiveness research (CER) is to provide health care decision makers, particularly patients, clinicians and payers, with better evidence for use in making informed health care decisions.  To meet this goal, comparative effectiveness or patient-centered outcomes research must consider a comprehensive array of health-related outcomes across diverse patient populations. (Federal Coordinating Council, 2009) The concept of pragmatic clinical trials (PCTs) is closely associated with the goal of CER. (Luce et al., 2009)  PCTs are prospective studies designed with the specific objective of producing information that will assist patients, clinicians and payers in making informed decisions about alternative drug and other health care therapies.

In order to understand the barriers to conducting PCTs to gain regulatory approval for drugs and opportunities for increasing the use of PCTs at this phase of drug development, the Center for Medical Technology Policy (CMTP) brought together a multi-stakeholder workgroup to characterize recurring gaps in evidence that are often not sufficiently addressed in regulatory trials, explore the reason for these gaps, and begin to elucidate potential solutions.   Jodi Segal, MD, MPH, from Johns Hopkins University School of Medicine provided a background paper for the attendees on “The Prospect of Early Pharmaceutical Pragmatic Clinical Trials: A Background Paper.
Segal PCT Background Paper (pdf) (281.95 kB) .”

At the conclusion of the meeting, the stakeholders determined that clear guidance on how to approach designing PCTs is probably something that can only really be addressed in the context of specific therapeutic class or more specifically on a drug by drug basis or the combination of drug and indication. However, there are some general principles that are applicable across different therapeutic areas that became apparent during the course of this meeting.  Therefore, CMTP, in collaboration with meeting participants, developed an Effectiveness Guidance Document outlining these general principles in the form of study design recommendations.

A meeting summary is available here,
PCT Meeting Summary (pdf) (1.16 MB)