Endpoints and Outcomes Conference 2009:
Optimizing Clinical Trials in FGID
On April 15-16, 2009, the Rome Foundation hosted at the Pfister Hotel in Milwaukee, WI the Rome Endpoints & Outcomes Conference: Optimizing Clinical Trials in FGID The meeting provided a unique opportunity to bring together representatives from the academic scientific community, practicing clinicians, the National National Institutes of Health (NIH), regulatory agencies from the USA, Europe and Japan, and several pharmaceutical companies. All interested participants shared their commitment the care of patients with functional gastrointestinal disorders (FGID) and looked to this meeting to learn about and help improve the clinical trial methodology for patients having these disorders. This “global meeting of the minds” included speakers and participants from the United States, Canada, Europe, Japan, Australia, and Latin America. The conference was held at an important time given the Food and Drug Administration’s (FDA) recent patient reported outcomes (PRO) guidance draft and their call to develop valid, reliable and meaningful endpoints for clinical trials for FGID.
Led by Lin Chang, MD, Chair, the planning committee created a program (download here) that examined the broad range of topics relevant to outcomes in FGID. While the conference specifically presented data on IBS, gastroparesis and pediatric FGID, much of the discussion applied generically to other FGID and gastrointestinal motility disorders.
The Outcomes Conference included presentations on:
- • Clinical trial primary and secondary endpoints
- • Meaningful clinical outcomes
- • Health Related Quality of Life
- • Risk-benefit assessment
- • Severity of FGID
- • Pediatric FGID endpoints
- • Perspectives from Industry, Regulatory Agencies and NIH about Drug Regulation and Development in FGIDs
In addition, brief lectures were given on important areas relative to clinical trials and research in the FGIDs. Following the presentations, breakout groups developed consensus statements that were presented to the entire group the following day. The topics included:
- • Role of biomarkers
- • Psychological symptoms and co-morbidities
- • Health behaviors, economic outcomes, HRQOL
- • Clinical trial endpoints for gastroparesis
At the end of the conference, a roundtable discussion was held by members of the Rome Foundation and the FDA Patient Reported Outcomes Consortium: Proposed Framework for a Public-Private Partnership
The success of the conference went beyond expectation, due to the outstanding commitment and contributions by the conference faculty We expect that the products of this conference will help in the approval and availability of treatments for our patients affected by these disorders.
Special thanks go to the members of our program planning committee: Lin Chang (USA), Chair, Carlar Blackman (USA), Douglas A. Drossman (USA) Jan Irvine (Canada), Nancy Norton (USA), Robin Spiller (UK), and William Whitehead (USA), for their effort in developing this meeting, and also to Kathy Haynes Johnson and George Degnon (Degnon Associates, USA) for organizing this meeting.
In addition, the Rome Foundation thanks the sponsors of the conference who provided unrestricted educational grants, without which the conference would not have been possible.
Ironwood Pharmaceuticals, Inc.
Procter & Gamble Pharmaceuticals
Takeda Pharmaceuticals, North America
The Rome Foundation plans to develop a consensus document from the meeting proceedings for publication.