For many years, the CF Foundation has worked to build a robust drug discovery and development “pipeline” of promising therapies to treat cystic fibrosis. Thanks to the efforts of countless volunteers, donors and scientists, there are now nearly 30 drugs in the pipeline. With this expansion of critical research, the Foundation now faces a new challenge: recruiting more CF patients to participate in clinical trials to test potential drugs.
It is estimated that 10 percent of CF patients are already involved in clinical trials, but this number must grow two- or three-fold over the next three to five years if new drugs are to be evaluated and become available to patients. Currently, several large Phase 3 trials are recruiting volunteers. To successfully complete these trials, more than a thousand people with CF will need to step forward and volunteer to participate. If there are not enough participants to complete these trials, some new therapies will never become available.
“We are recognized for our ability to engage companies in the CF drug discovery and development effort, but the real measure of our success is how effective we are in getting drugs evaluated and into the marketplace,” said Robert J. Beall, Ph.D., president and chief executive officer of the CF Foundation. “In every part of our community—among caregivers, people with CF, and their families, we must increase awareness of how vitally important it is to take part in clinical trials. People with CF who join clinical trials are among our most important contributors to CF research. The development of new treatments is absolutely dependent upon these key contributors. This year, as we work to achieve our mission to enhance and extend the lives of those with CF, increasing participation in clinical trials is a top priority.”
People with CF who join clinical trials are among our most important contributors to CF research."
Robert J. Beall, Ph.D.
President and Chief Executive Officer
To increase awareness about the importance of clinical trials and help boost participation, the Foundation recently launched the Clinical Trials Initiative.
As a first step, the CF Foundation conducted a series of surveys to determine what factors contribute to concerns about participating in clinical trials. Two primary obstacles became apparent: patients and their caregivers have inadequate knowledge about how clinical trials work and how important they are; and they lack information about where the trials are being conducted and how they can participate in them.
To raise awareness of the critical need for clinical trial participation, the CF Foundation has produced educational materials for patients, launched a toll-free clinical trials hotline at (877) 8CF-JOIN and is creating a special section on its Web site to heighten patient awareness about where trials are taking place and explain how they can become involved.
“It is essential that our patient and family community recognize that clinical trial participation cannot be left to someone else,” stresses Bruce Marshall, M.D., vice president of clinical affairs for the CF Foundation. “Unless enough people take part in Phase 1 and 2 trials, which evaluate safety and dosage, then Phase 3 trials may be delayed or may not occur. Without these clinical trials, new drugs cannot become available.”
In addition to changing patient attitudes about clinical trials, the CF Foundation is also increasing the number of care centers capable of conducting high-quality clinical trials over the next two years. To promote clinical research within the CF Foundation’s Care Center Network, and to prepare for an expansion of the Therapeutics Development Network (TDN), the CF Foundation invited all care centers to apply for Clinical Research Facilitation Awards. The awards provide financial support for those centers positioning themselves to become more involved in CF clinical research.
Training courses also are available so that care centers can enhance their capabilities for conducting clinical research under certified Good Clinical Practice parameters, and recruit patient volunteers. To date, 46 care centers have been selected to receive a Clinical Research Facilitation Award.
“Concern for the health and well-being of people with cystic fibrosis is behind every decision the CF Foundation makes,” said Preston W. Campbell, III, M.D., executive vice president for medical affairs of the CF Foundation. “And while the Foundation’s mission has remained constant over the years, the ways we achieve that mission are ever-evolving. To meet new and pressing needs in CF research as they arise, the CF Foundation relies upon the continued participation and dedication of the CF community. With the Clinical Trials Initiative, essential progress will continue toward our shared goal of finding new treatments and a cure for this disease.”