Advancing Clinical Trials through Expanded Patient Input | Aplastic Anemia and MDS International Foundation

Advancing Clinical Trials through Expanded Patient Input

AAMDSIF is committed to improving the clinical trials process for the benefit of patients and researchers alike, and wants to help government, industry and the nonprofit sector contribute to this goal. Dr. Ellen Salkeld, our Director of Research, recently offered some comments on the Foundation’s progress on the PACCT+ program since it was established a year ago.  Learn more about PAACT+ and also what the pharmaceutical industry is doing to spur recruitment for clinical trials.

“PACCT stands for Patient Advisory Committee on Clinical Trials, and the + sign in the abbreviation indicates health services research, a distinct and additional part of the program.  Last year, PCORI (Patient Centered Outcomes Research Institute) funded our PAACT+ proposals. It has been focused on improving patient-centered research in all areas of medicine including clinical trials and also making sure that things that patients are genuinely interested in and concerned about make it into the research process.

Our PAACT+ program began late last year by building a committee of three patients, each having either aplastic anemia, MDS or PNH, a clinical researcher and a pharmaceutical industry rep. The committee meets on a monthly basis to plan an upcoming training seminar for the eventual full committee.

We have just finished recruiting 20 patients who will serve on the PAACT+ committee. These were selected from over 40 responses to our call for participants. This group is almost evenly balanced by disease, gender and geography. They do not have to be current participants in clinical trials, but some of them are. They’ll all be attending an upcoming full-day training session in September. There will be two faculty members for this training. One of them is also a patient who has a doctorate in a related field, and wanted to be involved in assembling the educational protocol for this training session. The other faculty member is a nurse advisor who has collaborated with us many times before.

We want the training process to be such that the participants already have some background and familiarity with the subject before they attend. So we have been providing information through articles, videos and internet content for them to become familiar with before the day-long training session. So they will be at least somewhat prepared for what they will be learning even more about. The two sessions that day will be recorded and edited into segments that can be used on the AAMDSIF web site for general patient education purposes.

After the September training session, another goal is to create or find an interactive web site/portal for use by patients who we train to evaluate proposals. So we would build or adapt an existing platform where researchers and patients can interact with each other ---and a way for proposals to come in for patients to access them and communicate about them in a private, secure setting. This idea is just to facilitate a part of the process we feel is very important.

Our vision is to attract researchers from academia and industry who would like to have patient input on their proposals at the development stage, before it is even submitted to a funder, when it’s just at the idea stage. It’s a crucial juncture at which to get qualified patient input on their proposed trial. We’ve learned from research that the FDA and various funding entities are strongly encouraging researchers to have patient input prior to issuing funding. This also appears to facilitate the IRB (Institutional Review Board) process – when a proposal can show input from patients with the actual disease being studied.

Getting patients involved in clinical trials has always been a hurdle for companies trying to develop new drugs. There’s literature showing a low percentage of doctors who mention clinical trials during their office visits. Likewise, patients are worried if the doctor brings it up. There hasn’t been a culture of discussion of clinical trials in routine office visits.

The drug companies want to get drugs to the market faster, especially in the rare disease area. There has been some progress in consortia arrangements for rare diseases, such as our MDS Clinical Research Consortium where one trial can be conducted at a number of institutions, and thus accrue participants and results faster.”

Learn more about what industry is doing to attract patients to clinical trials.


Ellen J. Salkeld, PhD

Position / Title: 
Senior Director of Research and Health Professional Programs

Ellen works with the Foundation's professional affiliates and Medical Advisory Board to provide management and oversight to the MDS Clinical Research Consortium, the annual grants review process, and professional symposia. After earning her doctorate in medical anthropology and completing a postdoctoral fellowship in clinical research, she gained experience in health services research, program evaluation, and mixed methods research. Most recently she has worked with clinical researchers in academic medicine to enhance collaborative efforts around implementation and translational science, and research consortia.