On September 24, the AAMDSIF Patient Advisory Council on Clinical Trials (PACCT+) members met in Arlington, Virginia for an orientation seminar to meet fellow committee members, and learn from two experts about clinical trial design and the state of current bone marrow failure (BMF) research.
PACCT+ is a two-year demonstration project to develop and test a program to train patients in basic clinical and health services research concepts. PACCT+ was funded by the Patient Centered Outcomes Research Institute (PCORI) through AAMDSIF’s PCORI Engagement Award.
A panel of 18 trained patients and caregivers were trained to provide researchers in industry and academia with non-binding recommendations -- meaningful patient input in the design and implementation of clinical and health services research. PACCT+ participants are required to become familiar with clinical science research to ensure their ability to provide informed feedback that would benefit a study’s design and impact.
Greg Martin, Deputy Director of Stakeholder Engagement with the Patient Centered Outcomes Research Institute (PCORI) kicked off the meeting, speaking about PCORI’s mission – improved outcomes from research guided by patients, caregivers, and the broader healthcare community.
As luck would have it, PACCT+ Steering Committee member Dr. Phil Page (an aplastic anemia survivor) served an additional role by also delivering the presentation on clinical trials and clinical trial design.
The PACCT+ Council members then constructed a mock (sample) clinical trial using the principles described in the prior presentation, and built around bone marrow failure clinical scenarios, sometimes using actual approved drugs or ones currently being evaluated in actual clinical trials.
After lunch, Leslie Pettiford, RN, MS, presented an update on current research in bone marrow failure, taking questions from the audience at the conclusion. Then the groups reconvened to present and summarize their proposed trials from statement of purpose through patient consent forms.
Attendees spoke openly about their interest in contributing to the PACCT+ experiment and what they hope it can accomplish.
Thomas Coccagna, of Baltimore Maryland: “As an MDS patient and PACCT+ participant, I hope to contribute my experience in an actual clinical to be part of the process of developing more effective clinical trials. I feel patient input is going to change the face of clinical trials MDS, as well as for other diseases. This is just the beginning and I’m thrilled to be a part of it.”
Lani Alexander, Marietta, Georgia: “I was impressed by how engaged and enthusiastic the entire group was. It was rewarding to learn more about the process of a clinical trial, from planning to eventual market feedback. I hope that we are able to contribute in a way that pushes research forward, making it clearer and more accessible to patients and caregivers; and that we are able to be of value to the research process so that this program and others like it will proliferate.”