Author: Lorri Bingham

Patient Perspective: Why We Need Reform of the Clinical Trial Process

Posted on by Lorri Bingham

“Duchenne muscular dystrophy (DMD) is a, rare, progressive and ultimately fatal pediatric genetic disease that affects male children. Parents usually learn that their seemingly healthy son has DMD at age 4 to 5. By age 9 to 10, afflicted boys typically lose the ability to walk, eat and care for themselves, eventually becoming reliant on a ventilator. These patients commonly die in their 20s of respiratory complications or cardiomyopathy. There are currently no effective treatments.

Fortunately, there are very promising clinical candidates entering phase II and III trials. The problem is that the only validated endpoint for DMD drug approval is the Six Minute Walk Test, a measure that is most sensitive at a time when the boys are still ambulatory but beginning to lose the ability to walk. Furthermore, any time spent in the placebo arm of a trial for an effective treatment represents a lost opportunity for those participants. Some boys in the placebo arm may plateau at a much lower functional level after they are crossed over to the drug, or they may lose the ability to walk altogether. This loss of ambulation can mean that the patient is no longer eligible for future trials, and can lead to a loss of hope for the family involved.

We need to think about creative trial designs to minimize the amount of time spent in the placebo treatment, as well as different endpoints with faster, more efficient ways to validate them.”

– Sharon Hesterlee, PhD, VP Research, Parent Project Muscular Dystrophy

This piece was featured in CTTI’s 2013 Annual Report. To view this document, CLICK HERE.

CTTI’s 2013 Annual Report is Now Available Online

Posted on by Lorri Bingham

Would you like to see how innovation is being achieved within the clinical trials enterprise? In the following video CTTI’s Executive Director, Dr. Pamela Tenaerts, summarizes the trends that mark CTTI’s work in 2013, including an increased patient voice and expanded dissemination efforts in the pursuit of identifying and promoting practices that will increase efficiency and quality in clinical trials.

To view CTTI’s 2013 Annual Report, CLICK HERE.

CTTI Executive Director Speaks on Engaging Patients to Drive Innovation at Recent ACRO Meeting

Posted on by Lorri Bingham

At the 2014 ACRO Annual Meeting, CTTI’s Executive Director Pamela Tenaerts spoke on the Innovation Panel. Comments focused on how increased patient involvement could be the disruptive change needed to improve the clinical trials system. Other experts featured on this panel included Patricia Leuchten, President and CEO of the Avoca Group Inc., Dalvir Gill, CEO of TransCelerate Biopharma, Luke Williams, professor of innovation and author of “Disrupt”, and John Lewis, Vice President of ACRO. The resulting speech was recorded and is now available online:

We’d like to thank ACRO for creating this video and sharing this message. At CTTI, patient engagement is at the core of several of our initiatives, such as the Patient Leadership Council, the Recruitment Project, and the Patient Groups & Clinical Trials Project. Through the collaboration of all stakeholders, including patients, we can move toward a high quality clinical trial system that is patient-centered and efficient, enabling reliable and timely access to evidence-based prevention and treatment options.