Patient Engagement

CTTI Charts New Pathways for Pediatric Antibacterial Drug Development

Posted on by Lorri Bingham

In an article recently published in the Journal of the Pediatric Infectious Disease Society (JPIDS), CTTI researchers lay out a roadmap for addressing an urgent public health issue: pediatric antibacterial drug development.

Antibacterial drugs are critically important for treating infectious diseases, but growing rates of antimicrobial resistance have made the development of new antibacterial therapies a priority for researchers and physicians. The problem is especially acute in pediatrics, because even when new drugs are developed for adults, it may be up to 10 years before the pediatric clinical trials needed to provide vital information about safety, effectiveness, and dosing in children are completed. The result of these delays is a chronic shortage of information to guide the use of new therapies in pediatric populations.

The JPIDS article, which was distilled from the results of CTTI research and expert stakeholder meetings, identifies five key areas for action aimed at overcoming barriers to conducting timely and efficient trials of new antibacterial drugs in children, including:

  1. Improving planning for pediatric drug development
  2. Streamlining processes for protocol development and trial design
  3. Refining approaches to seeking and obtaining informed consent
  4. Engaging with healthcare providers
  5. Emphasizing the rapid incorporation of new information into product labeling

The article reflects recommendations from CTTI’s Pediatric Antibacterial Drug Development Project, which focuses on creating efficient, evidence-based processes for developing, testing, and using antibacterial therapies in children. The “Peds Trials” Project itself is part of the larger CTTI Antibacterial Drug Development Program.

By fostering collaborative approaches that involve all stakeholders, CTTI hopes to accelerate the development of safe and effective pediatric antibacterial drugs—and to equip physicians and other healthcare professionals with the information they need to make the best possible decisions for the health of the children under their care.

Four Key Areas Where CTTI Has Transformed Clinical Trials

Posted on by Lorri Bingham

This year, CTTI marks its 10th year of working with a variety of stakeholders to positively change clinical trials, making them more streamlined, efficient, and patient-focused. To commemorate this milestone, we will #CelebrateCTTI throughout the month of January. Join us for the celebration—visit this blog, follow us on Twitter, and register to attend our 10 Year Anniversary Symposium on Tues., Feb. 6.

Since its inception in 2007, CTTI has made transformational shifts in the design and conduct of clinical research. Let’s take a look at the evolution of clinical trials in four key areas, among many, in which CTTI has played a critical role in moving the industry toward more efficient, high-quality, and patient-centric trials:

Trial Quality

Regulators have long acknowledged a need to change how the industry approaches clinical trial quality in order to focus on reducing errors that undermine data integrity and patient safety. Drawing on the expertise of key stakeholders, CTTI issued recommendations on effective and efficient trial monitoring in 2012. The following year, FDA issued final guidance on a risk-based approach to monitoring, reflecting principles from CTTI’s work. CTTI also developed the concept of Quality by Design, taking the focus from a reactive approach using audits to a proactive approach that builds quality into clinical trials at the outset. CTTI released its Quality by Design recommendations and toolkit in 2015, and its work in this area has been cited in FDA guidance and incorporated into GCP guidelines.

Patient Engagement

CTTI has been a pioneer in patient engagement, involving patient representatives in its organizational leadership and throughout its project teams. Spurred by the FDA’s Safety and Innovation Act of 2012, CTTI initiated the Patient Groups & Clinical Trials Project to establish best practices for engaging patients in clinical research. In 2014, CTTI released landmark recommendations that are currently used by multiple stakeholders to facilitate productive relationships with patient groups around clinical trials. In 2017, CTTI and FDA announced a partnership on a new initiative, the Patient Engagement Collaborative, to improve patient engagement across the FDA.

Central IRB

Although U.S. regulators are enthusiastic about the use of a central IRB to promote more efficient multi-site clinical trials, enterprise-wide implementation has lagged. CTTI initiated the Central IRB Project in 2010, creating recommendations and resources to help organizations identify and address barriers to the adoption of a central IRB. The NIH issued a draft policy in 2014 referencing CTTI’s recommendations and a final policy in 2016 requiring all NIH-funded multi-site clinical trials to use a central IRB effective in 2018.

Real-World Evidence

Since its inception, CTTI has recognized that “real-world evidence” from electronic health records, mobile devices, and other sources can provide unique insights into important clinical questions and could improve the efficiency of clinical trials. The FDA Amendments Act of 2007, which mandated FDA to develop a system to link safety data from multiple sources, paved the way for the use of connected datasets in clinical trials. After partnering with the FDA and Harvard Pilgrim Health Care Institute to assess the feasibility of using the Sentinel database for clinical research, CTTI supported the first trial using Sentinel. CTTI also launched a program in 2016 to support the use of mobile technology in clinical trials, issued recommendations in 2017 on how to use registries as reusable platforms for trials, and announced a Real-World Evidence Project in 2017.

CTTI Selected to Support FDA Efforts to Increase Patient Engagement Through New Collaborative

Posted on by Lorri Bingham

CTTI has been engaged by the U.S. Food and Drug Administration (FDA) to support its Patient Engagement Collaborative (PEC), an effort created in partnership with CTTI to bring together members of the patient community to discuss the best ways for patients to participate in the FDA’s regulatory discussions about medical products.

“Engaging patients as partners in the development of medical products is critical to the success of the clinical research enterprise, and CTTI is pleased to be hosting this new forum for patients and regulators to discuss and exchange ideas,” said Pamela Tenaerts, MD, MBA, CTTI Executive Director. “This is an important initiative that will benefit not only patients and regulators, but all stakeholders in the research enterprise.”

The Patient Engagement Collaborative is being created because of public feedback the FDA requested on Section 1137, Patient Participation in Medical Product Discussions, of the Food and Drug Administration Safety and Innovation Act (FDASIA). One suggestion was to create an outside group to give input on patient engagement across the FDA.

“The FDA is committed to expanding its efforts to engage patients in its regulatory decision-making processes and the new PEC will include a diverse group of representatives from outside the FDA who will help enhance the agency’s understanding of how to best engage across patient communities,” said Rachel E. Sherman, MD, MPH, FDA Principal Deputy Commissioner. “CTTI has a well-established network of patients and patient advocates, and we look forward to working with them in this important effort.”

The Patient Engagement Collaborative will bring 16 patients, caregivers, and patient group representatives together with the FDA several times a year to discuss topics such as communication, transparency, and new ways for patients and the FDA to work together. The new group will be hosted by CTTI, which has strong ties to the patient community.

Nominations to join the Patient Engagement Collaborative will be accepted until Jan. 29. Learn more about what is expected of members and how to apply in the Federal Register Notice. You are also welcome to join a call hosted by CTTI and the FDA on Fri., Jan. 12, at 2pm ET if you have questions about applying. Join by calling 800-988-9752 and use participant code 4753141.

Two CTTI Papers Explore Perceptions on Enrolling Children in Clinical Trials

Posted on by Lorri Bingham

Two papers from CTTI’s Peds Trials work on barriers to enrolling children in clinical trials were recently published in Contemporary Clinical Trials Communications. Despite the fact that pediatric trials are required for new drugs, these trials can be especially difficult to design, enroll, and complete. CTTI’s papers explore the challenges from both the provider and parent perspective and suggest ways to overcome them.

Based on feedback from parents, providers, and other stakeholders, CTTI developed actionable recommendations to address the challenges of enrolling children in clinical trials. These recommendations offer practical, evidence-based strategies that can be applied by research sponsors, investigators, and site staff to improve the quality and efficiency of pediatric trials.

CTTI Cited during the FDA’s Patient Engagement Advisory Committee Inaugural Meeting

Posted on by Lorri Bingham

Findings and recommendations from CTTI’s Patient Groups & Clinical Trials work were presented during the inaugural meeting of the FDA’s Patient Engagement Advisory Committee (PEAC) on Oct. 11-12, 2017.

At the meeting, Bray Patrick-Lake, director of stakeholder engagement, Duke Clinical Research Institute, discussed patient engagement efforts in the clinical trial enterprise and presented work conducted by CTTI to establish best practices for effective engagement with patient groups around clinical trials, which include:

  • Engage the “patient voice” by establishing partnerships from the beginning of the research and development program to improve trial design and execution
  • Clearly define the expectations, roles, and responsibilities of all partners, including the resources being committed, data being shared, and objectives of the program
  • Manage real or perceived conflicts of interest by establishing policies that require full disclosure, transparency, and accountability

The PEAC is composed of patients and caregivers who provide perspective to the FDA on complex issues relating to medical devices, the regulation of devices, and their use by patients. This first meeting brought together patients, patient organizations, FDA, industry, and other medical and scientific experts for discussion on incorporating patient perspectives in the design, conduct, and reporting of medical device clinical trials.

Meeting materials, including slide presentations, are available on the FDA website.

CTTI Shares Challenges and Solutions to Enrolling Participants in Pediatric Trials at AAP 2017

Posted on by Lorri Bingham

At the upcoming American Academy of Pediatrics National Conference, CTTI will offer insights into some of the biggest challenges currently affecting pediatric clinical trial enrollment and conduct. Through a podium presentation and poster sessions, CTTI will share survey-based findings that shed new light on obstacles affecting pediatric trials and discuss possible solutions that can help streamline these studies and reduce burdens on patients, families, and providers.

 

Although clinical trials are essential for ensuring access to safe and effective therapies for children, many studies struggle to enroll pediatric participants and families and providers often lack information to help them make the best possible treatment choices. This problem is especially acute for children and infants at risk of harm from bacterial infections, many of which are increasingly resistant to antibiotic treatment. For this reason, maintaining a strong pipeline of safe and effective therapies is an urgent priority.

 

CTTI’s Peds Trials project, part of the larger Antibacterial Drug Development Program, is actively addressing these gaps and has created evidence-based recommendations designed to improve and streamline pediatric trial development and conduct. We hope you will join us in Chicago this Sept. 16-19 as we share new insights into challenges affecting pediatric trial enrollment, including reasons that parents refuse trials and provider perceptions of obstacles.

 

Poster & Podium Presentation: Obstacles to Pediatric Clinical Trial Enrollment: Why Parents Refuse; Findings from the Clinical Trials Transformation Initiative

Date & Time: Monday, Sept. 18, 12:35 p.m.–12:50 p.m. (oral presentation)

Monday, Sept. 18, 1:00 p.m.–5:00 p.m. (poster session)

Presenter: P. Brian Smith (Duke University)

 

Poster: Barriers to Pediatric Clinical Trial Enrollment: Findings and Recommendations from the Clinical Trials Transformation Initiative

Date & Time: Monday, Sept. 18, 1:00 p.m.–5:00 p.m. (poster session)

Presenter: P. Brian Smith (Duke University)

Getting Down to the Bottom Line: The Financial Impact of Patient Engagement in Clinical Trials

Posted on by Lorri Bingham

CTTI publishes framework for estimating the impact of patient engagement on key business drivers

To fully realize the impact of patient engagement in clinical trials, there is a need for robust and rigorous study of its quantitative effects on research and development. To begin to address this gap, CTTI recently published an approach for assessing the financial value of patient engagement that accounts for the business drivers of cost, risk, revenue, and time.

In a set of recommendations published along with the financial model, CTTI’s Patient Groups & Clinical Trials Project team outlines “rules of engagement” for successful collaborations between research sponsors and patient groups around clinical research. Together, the articles in Therapeutic Innovation & Regulatory Science (TIRS) provide a blueprint for establishing effective patient group engagement in research programs.

While research sponsors are increasingly partnering with patients in the design and conduct of clinical development programs, sponsors may be reluctant to go beyond pilot initiatives because of uncertainty in the return on investment. The new CTTI article describes a novel approach to quantifying the financial impact of patient-centric initiatives using risk-adjusted financial modeling techniques that integrate key business drivers into a summary metric (expected net present value). Expected net present value (ENPV) is a widely used metric by industry for project strategy and portfolio decisions.

Drawing on published data to develop a case study for a typical oncology development program, the authors also show how patient engagement activities that avoid a protocol amendment or improve the patient experience can contribute considerable financial value.

Ongoing work by CTTI’s Patient Groups & Clinical Trials Project is examining how sponsors and patient groups characterize the benefits of patient engagement and investments required for engagement. Along with the ENPV model, the project aims to provide resources to assist in determining which of the many engagement opportunities provide the greatest value.

Webinar Recording Now Available: CTTI Recommendations for Improving Pediatric Antibacterial Drug Trials

Posted on by Lorri Bingham

Do you struggle with enrolling babies and children in clinical trials? Are you tired of not having the evidence you need to treat kids with serious infections?

A recording is now available of CTTI’s webinar discussing the new CTTI recommendations on improving antibacterial drug trials for children. Experts from FDA, academia, and pharma described the challenges of conducting pediatric antibacterial drug trials, along with practical, evidence-based strategies to improve the quality and efficiency of these trials. These strategies were developed with input from multiple stakeholders and can be used by research sponsors, investigators, and site staff to make pediatric trials more successful.

View the recording to learn ideas on how you can create better clinical trials for children, such as:

  • Importance of engaging with regulators early and throughout medical product development
  • Methods of streamlining trial design to decrease burden on sites and families
  • Special considerations for conducting trials with neonates
  • Approaches for improving the informed consent process
  • Ways to increase engagement with healthcare providers

 

These recommendations are a result of CTTI’s ABDD Peds Trials Project.

To view recordings of other CTTI webinars, click here.

CTTI Releases New Recommendations to Improve Studies of Antibacterial Drugs for Children

Posted on by Lorri Bingham

CTTI has released new recommendations to improve the quality and efficiency of research studies used to develop antibacterial drugs for children. In addition, many of the suggested strategies and practices could be applied to streamline clinical trials of other types of drugs and medical devices for children.

“Medically, children are not just little adults, and they need access to treatments that have undergone appropriate evaluation for safety and efficacy in children,” said Daniel Benjamin Jr., MD, PhD, MPH, a pediatric infectious diseases specialist at Duke University. “The CTTI recommendations address many of the common challenges of conducting this research, and if applied widely, can help deliver much-needed information and treatments to benefit our young patients.”

These recommendations resulted from a collaborative effort among research sponsors, parents, investigators, clinicians, and regulators from the US and the EMA (European Medicines Agency), who provided practical suggestions for the timing of pediatric trials, streamlining trial design, facilitating informed consent, and fostering global and community partnerships to conduct trials that can improve children’s health.

The time from approval of a new antibacterial drug for use in adults to pediatric labeling can be 5 years or longer, potentially delaying appropriate use of medicines for this vulnerable group. Antibacterial resistance is on the rise in children, and the very young can be particularly susceptible to severe illness or death from these pathogens. Despite the great need for more treatment options, many trial sponsors have challenges enrolling pediatric patients in antibacterial drug trials.

“These recommendations encourage consultation with the FDA on pediatric study plans early in drug development and emphasize the potential utility of global study networks and streamlining trials,” said Sumathi Nambiar, MD, MPH, Director of the Division of Anti-Infective Products at the U.S. Food & Drug Administration (FDA). “Our mutual goal is to provide data in the drug labeling that will better inform the safe and effective use of antibacterial drugs in children.”

The CTTI recommendations are meant to  help researchers design trials that are less burdensome for families, as well as to support  improved practices for approaching parents for consent during the stressful time of a child’s illness. These recommendations are based on research that showed 80% of clinicians surveyed identified parent concerns about their child participating in research to be a barrier for completing research studies with children. This emphasizes the need for better engagement with parents throughout a clinical trial, including during the initial design stage. “This work matters to the lives of families like mine,” said Breck Gamel, a parent participant in the CTTI effort. CTTI studied other clinician concerns as well, which helped to identify educational gaps in pediatric labeling and the need for better engagement with other healthcare providers.

*These recommendations are the result of CTTI’s ABDD Peds Trials Project.

**To read this press release in full, click here.