CTTI News

Improving Human Health through Large Simple Trials

Posted on by Lorri Bingham

“Randomized clinical trials remain the most reliable means of identifying the drugs, devices, and treatment strategies that will improve human health.”
– Zubin J. Eapen, MD, MHS, Michael S. Lauer, MD, and Robert J. Temple, MD

On May 13-14, 2013, CTTI hosted an expert meeting on the need to streamline the conduct of large simple trials (LSTs) in the U.S. This meeting convened a broad array of engaged stakeholders including regulators, government sponsors of clinical research, members of academia and industry, patient advocates, clinical investigators, and other interested parties. The primary objective of this meeting included discussing the following:

  • Findings from a survey of practices
  • Strategies that companies are using to implement LSTs
  • The challenges to LSTs

The summary of this expert meeting is now available on the CTTI website.

Additionally, the key takeaways from this expert meeting were captured in a recent JAMA Viewpoint publication, The Imperative of Overcoming Barriers to the Conduct of Large, Simple Trials. Co-authored by three of CTTI’s LST Project Team Members (Zubin J. Eapen, MD, MHS, Michael S. Lauer, MD, and Robert J. Temple, MD), this perspective piece highlights how the U.S. healthcare system stands to benefit from larger, simpler, less expensive randomized trials. This article also touches on the possibility of leveraging existing data collection platforms to enhance patient enrollment in LSTs, as well as the ability of funding opportunities to encourage streamlined trial design.

The authors concluded, “With the current opportunities to streamline trials and leverage existing clinical data, conducting large, simple trials is more possible than ever and presents a path forward for advancing human health.”

For more information about CTTI’s LST Project, CLICK HERE.

Rethinking Clinical Research: Central IRB Advancement Webinar

Posted on by Lorri Bingham

On Friday April 25, 2014, the NIH Collaboratory’s free webinar series known as the Grand Rounds: Rethinking Clinical Research will host the web conference titled CTTI Advancing the Use of Central IRBs Project: Academic Institution and Government Sponsor Perspectives. The focus of this webinar will be the adoption of a single IRB of record for multi-center clinical trials, as encouraged in CTTI recommendations. Two speakers, both of whom serve as team leads for CTTI’s Central IRB Advancement Project, will be featured:

  • Cynthia Hahn, VP, Clinical Research and Regulatory Affairs, North Shore-LIJ Health System
  • Petra Kaufmann, M.D., M.Sc., Director, Office of Clinical Research, National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH)

We invite you to attend this presentation, and encourage you to share this opportunity with your colleagues.

Time: 1:00-2:00 p.m. ET

Webinar Login:

  1. Log in to the URL first: https://dukemed.webex.com/dukemed/j.php?MTID=m30760324061a749812cd1c966d1bfde0
  2. Click “Audio Conference”
  3. Choose “I will call in” and select the Toll Free number.
  4. Dial in using the information from the dialog box that appears. Be certain to use the Access Code AND the Attendee ID.

*Note: If the URL above does not work, go to dukemed.webex.com and enter:
Meeting Number: 739 773 050
Meeting Password: 1234

For additional webinar details, please visit the NIH Collaboratory’s Grand Rounds Calendar.

We would like to thank Cynthia Hahn, Petra Kaufmann, and the rest of the Central IRB Advancement Team for their dedicated efforts to encourage implementation of CTTI’s recommendations. It is through these dissemination activities that methods to increase the quality and efficiency of the clinical trials enterprise are shared, and changes in practice are effected.

 

CTTI Tackles Practical Applications of Quality by Design at the DIA’s 50th Annual Meeting

Posted on by Lorri Bingham

DIA Meeting

This summer, the DIA will host their 50th Annual Meeting in San Diego, CA, bringing together “a global network of life sciences professionals to foster innovation that will lead to the development of safe and effective medical products and therapies to patients.” We are proud to announce that CTTI will be hosting a one-day, pre-conference tutorial, titled Quality by Design: From Theory to Practice, in conjunction with this event. This Quality by Design (QbD) tutorial is intended to give representatives from a broad cross-section of the clinical trials enterprise practical insights into applying QbD principles into their work. Emphasis will be placed on:

  • Developing an understanding of risk-based QbD for clinical trials, from general principles, real-world examples, and hypothetical case studies;
  • Gaining confidence in the application of building quality into the clinical trials process;
  • And identifying perceived barriers to the adoption of Quality by Design principles.

The QbD tutorial will be held on Sunday June 15, 2014, from 9 am – 5 pm, and will be co-instructed by Ann Meeker-O’Connell (Janssen), John Alexander (Duke), and Jean Mulinde (FDA/CDER). This pre-conference tutorial is open to any individual registered for the DIA conference. (To register for the DIA conference, CLICK HERE.)

*Note: There is limited availability for the pre-conference tutorial. To assure your seat, please sign up as soon as possible.

To view more information on CTTI’s ongoing QbD project, CLICK HERE.

CTTI’s Founding Executive Director Retires

Posted on by Lorri Bingham

In February of 2014, CTTI’s founding Executive Director Judy Kramer retired and today we’d like to pay tribute to her legacy. She most recently served as CTTI’s Senior Scientific Advisor and as Professor Emerita of Medicine at Duke. In addition to establishing this CTTI, Judy provided scientific guidance on individual CTTI projects and she played a significant role in communicating the results of these projects through publications and presentations at national and international meetings. She also advanced our mission by participating in the efforts of other non-profit organizations with similar goals.

Judy is known for her genuine passion and unfaltering conviction to improve and transform clinical trials, as well as her ongoing commitment to patient-centeredness and clinical trial safety issues. These attributes permeated through all of our interactions with her and aside from her valuable professional contributions to CTTI and Duke, Judy had a lasting positive personal influence on her colleagues.

Luckily for the clinical trial enterprise, Judy will continue to stay involved with FDA advisory committees. She will also likely have additional interactions with CTTI through her Professor Emerita role at Duke. Please join me in wishing Judy all the best in this next phase of her life, which will include a larger proportion of time devoted to travel, taking beautiful pictures, and her family. She and her husband, Lloyd, are looking forward to spending more time with their four granddaughters.

– Pamela Tenaerts, MD, MBA
Executive Director, CTTI

As a tribute to Judy’s years of service, we’d like to share a recent video featuring our retired founding Executive Director’s explanation of the importance of randomization in clinical trials:

Materials Available for Device-Focused Workshop in QbD Project

Posted on by Lorri Bingham

On January 29-30, 2014, CTTI’s QbD Project held a workshop focused on clinical trials that evaluate medical devices (diagnostic and therapeutic). The objectives for this workshop were similar to previous QbD Workshops:

  • To develop understanding of risk-based Quality by Design (QbD) for clinical trials, from general principles, real-world examples, and hypothetical case studies;
  • Gain confidence in the application of such concepts to clinical trials;
  • Identify obstacles to the adoption of this approach;
  • And identify opportunities for dissemination of these principles and practices to a broad array of stakeholders.

 

Executive Summary of the CTTI-Hosted Expert Meeting on Good Clinical Practices Now Available Online

Posted on by Lorri Bingham

GCP Training Expert Panel

On January 31, 2014, CTTI convened a diverse array of thought leaders from the clinical trials enterprise to discuss good clinical practice (GCP) training. The goal of this meeting was to seek consensus on the key elements of GCP training, as well as the frequency, format, and competency of training required to conduct clinical studies in the United States. We are now pleased to share the executive summary from this meeting online. For more information on this expert meeting, including the participant list and presentations, CLICK HERE.

This meeting is a part of CTTI’s ongoing GCP Training Project. For more information on this project, CLICK HERE.

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.