Regulatory Submissions + Approvals

CTTI Seeks to Speed Use of Mobile Technology in Clinical Trials through Novel Endpoint Recommendations

Posted on by Lorri Bingham

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New CTTI Recommendations and Tools Equip Stakeholders to Integrate Mobile Technology into Clinical Trials

Mobile technologies hold enormous promise for clinical research, but uncertainty about how to use the data captured by these devices has slowed progress. CTTI’s newly released recommendations and tools aim to change this by providing a pathway for using information gathered from mobile technologies to accelerate the development and evaluation of urgently needed therapies.

Mobile technologies such as remote sensors and wearables can be used to make trials faster, more efficient, and more inclusive. They can also spare patients from burdensome clinic visits while capturing new kinds of data that offer a better picture of how patients experience their disease or condition in their daily lives.

“Technology-derived endpoints offer the benefit of capturing information about patients’ experience in ‘real-world’ settings,” said Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research. “These tools have the potential to capture data that can be used to develop endpoints and evaluate therapies in the patient population.”

Responding to this potential, CTTI experts have crafted a set of recommendations and tools designed to help diverse stakeholders identify and develop novel endpoints based on data from mobile technologies for use in regulatory clinical trials.

“By engaging with experts who have been early champions of mobile technology in trials and combining that with patient insights, CTTI has created practical recommendations and action-oriented tools that have the potential to really accelerate the use of mobile technology in clinical trials,” noted Craig Lipset, Pfizer’s head of clinical innovation. “In particular, the use cases provide a realistic pathway for incorporating novel endpoints through technology into clinical development programs. CTTI’s recommendations show we may be closer than previously believed to realizing the benefits of these novel endpoints, creating a sense of urgency to act.”

In partnership with a group of investigators, regulators, patient representatives, technology developers, and research sponsors, four use cases were written to better guide the development and use of novel endpoints as part of clinical trials for Parkinson’s disease, heart failure, diabetes, and Duchenne muscular dystrophy.

“The Michael J. Fox Foundation was pleased to participate in CTTI’s efforts to develop novel mobile endpoints for use in clinical trials,” said Lauren Bataille, senior associate director of research partnerships at the Foundation. “We look forward to leveraging these assets to support Parkinson’s research collaborations and speed the developments of measures that matter to patients.”

CTTI’s Novel Endpoints recommendations are the first to be released as part of a larger body of work to address multiple challenges to using mobile technology in clinical trials. CTTI’s Mobile Clinical Trials Program includes additional projects on legal and regulatory considerations, stakeholder perceptions, and scientific and technical issues related to the use of mobile devices. Recommendations from these projects will follow over the next year.

Click here to view more information on CTTI’s Novel Endpoints Project.

To view a recording of the webinar that unveiled these recommendations, click here.

Accelerating the Use of Mobile Technology In Clinical Trials: Webinar Recording Now Available

Posted on by Lorri Bingham

EXPERTS UNVEIL NEW RECOMMENDATIONS AND TOOLS FOR INTEGRATING NOVEL ENDPOINTS DERIVED FROM MOBILE TECHNOLOGY INTO CLINICAL TRIALS

Mobile technologies are reshaping the landscape of clinical trials and making it possible to capture new kinds of patient data. Learn how novel endpoints derived from these technologies can be leveraged for more efficient, less burdensome clinical trials that generate the high-quality data needed to address the outcomes that matter most to patients.

In this webinar, a group of expert presenters and panelists describe CTTI’s new recommendations and tools for identifying and developing technology-based novel endpoints and incorporating them into clinical trials. Drawn from backgrounds spanning academia, industry, technology development, patient advocacy, and regulatory agencies, CTTI’s expert stakeholders provide a “guided tour” of these resources. They also discuss key strategies—including early engagement and discussion with regulators—for successfully integrating novel endpoints into clinical trials that enable a better understanding of the effects of diseases and their treatments on patients’ daily lives. CTTI’s MCT Novel Endpoints recommendations, which were developed from a multi-stakeholder expert meeting that applied four “real-world” use cases (heart failure, diabetes, Parkinson’s disease, and Duchenne muscular dystrophy), address the following key areas:

  • Identifying and developing high-quality novel endpoints that best reflect patients’ priorities and needs
  • Selecting the best outcome measures and matching those measures with appropriate technologies
  • Developing products that address unmet needs and adapting solutions that meet the demands of the clinical research and patient care environments
  • Supporting trial sponsors and researchers in working effectively with regulators to develop evidence-based, high-quality novel endpoints that can be used to support regulatory review and approval

FOR MORE INFORMATION ON THIS WEBINAR, INCLUDING POWERPOINT SLIDES, PRESENTER INFORMATION, AND MORE, CLICK HERE.

The MCT Novel Endpoints Project is part of CTTI’s larger Mobile Clinical Trials Program.

Webinar June 26: Launching New CTTI Recommendations to Accelerate the Appropriate Use of Mobile Technology in Clinical Trials

Posted on by Lorri Bingham

CTTI Webinar

Mobile technologies offer unique opportunities for making clinical trials faster, more efficient, and more representative of actual patient experiences. By addressing unmet needs for quality outcome measures, enabling more efficient alternatives to traditional clinical research models, and reducing dependence on data that represent only a tiny snapshot of patients’ experience of disease, mobile technologies allow us to capture new kinds of information while reducing the burdens of trial participation. However, realizing these benefits requires a clear understanding of how to successfully select, develop and incorporate technology-derived novel endpoints and incorporate them into clinical trials—a need that CTTI is addressing through its MCT Novel Endpoints Project.

Join us for a special webinar in which CTTI will present new recommendations and tools to support the selection, development, and inclusion of technology-derived novel endpoints in clinical trials.

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Topic: Developing novel endpoints generated using mobile technology for use in clinical trials
Date: Monday, June 26, 2017 12:00 – 1:30 PM EDT (GMT-04:00)
Webinar Link: http://bit.ly/2rAJ9K7
Presenters:

  • Martin Landray (University of Oxford)
  • Marc Walton (Janssen)

Panel members sharing their perspectives on adoption of the recommendations:

  • Leonard Sacks (US Food and Drug Administration)
  • Lauren Bataille (Michael J. Fox Foundation)
  • Wendy Snyder (Amgen)
  • Rob Wilson (ActiGraph)

The webinar will include a first look at evidence-based approaches and tools that can be applied by research sponsors, investigators, technology developers, and patient groups to facilitate the use of novel endpoints derived from mobile technology in clinical trials. You will learn about a set of tools developed by CTTI’s expert stakeholders to guide the identification, development and inclusion of appropriate novel endpoints.

Our presenters and panel members will show how:

  • The clinical research enterprise can benefit from the appropriate inclusion of technology-derived novel endpoints in clinical trials
  • Sponsors and investigators can work effectively with regulators to develop high-quality novel endpoints to support regulatory submission and approval
  • Patient groups can both drive the identification of novel endpoints that measure symptoms that matter and participate in their development
  • Tech developers can better match product development to unmet needs and adapt solutions for the demands of clinical trials and the patient care environment
  • Stakeholders can collaborate to avoid common pitfalls in the development process

You will be introduced to four “real-world” use cases that shaped the development of these tools and recommendations, with key lessons to be carried forward to other therapeutic areas. You will also learn how these tools and recommendations can be applied in practice from our panel of early adopters.

This webinar is open to the public. Please feel free to share this invitation with your colleagues.

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.

Webinar February 16: Improving Pediatric Antibacterial Drug Trials

Posted on by Lorri Bingham

Challenges in conducting antibacterial drug trials for pediatric patients can delay the safe and effective use of treatments for this vulnerable group.  There are now evidence-based, consensus-driven solutions. Are you ready to do better clinical trials for children?

Join us for a special webinar in which CTTI will unveil new recommendations for improving pediatric antibacterial clinical trials:

Add to Calendar

Title: CTTI Recommendations from the Antibacterial Drug Development (ABDD) Peds Trials Project
Date: February 16, 2016 12:00 – 1:00 PM EST (GMT-05:00)
Webinar Link: http://bit.ly/2kYGm7j
Speakers:

  • Sumathi Nambiar, U.S. Food and Drug Administration
  • John Bradley, University of California, San Diego
  • Gary Noel, Johnson and Johnson Pharmaceutical Research and Development

The webinar will include practical, evidence-based strategies that can be applied by research sponsors, investigators, and site staff to improve the quality and efficiency of pediatric antibacterial trials.

Learn these tips and more for making your trials more successful:

  • 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

Although developed in the context of antibacterial drug development, many of the recommendations can be applied to improve pediatric clinical trials in multiple therapeutic areas.

This webinar is open to the public. Please feel free to share this invitation with your colleagues.

Expert Meeting Materials Now Available: Developing Novel Endpoints Generated by Mobile Technology for Use in Clinical

Posted on by Lorri Bingham

View Expert Meeting MaterialsOn September 29-30, 2016, CTTI hosted a multi-stakeholder expert meeting to begin to develop recommendations for how to develop mobile technology–derived novel endpoints for use in clinical trials. Mobile technologies such as remote sensors have the potential to facilitate continuous, high-quality data acquisition not typically possible with traditional data collection methods. However, it is currently unclear how data from these devices may be used to generate novel endpoints for use in clinical trials.

Meeting attendees drafted four use cases to clarify the pathway for developing novel endpoints derived from data captured using mobile devices:

  • Use of accelerometers to measure treatment benefit in heart failure, Parkinson’s disease, and muscular dystrophy trials
  • Use of continuous glucose monitors to measure treatment benefit in diabetes trials

Highlights from this meeting are now available in the meeting summary. As a next step in the MCT Novel Endpoints Project, the team will use the evidence from these four use cases, along with findings from a systematic review that is underway, to inform general recommendations to help promote widespread adoption of mobile technologies in clinical trials.

Meeting attendees included investigators, patient representatives, engineers, algorithm experts, regulators, nonprofit consortia, and statisticians who had experience with the diseases and/or devices discussed.

CTTI Publishes Findings on Patient & Physician Perceptions of Streamlined Development for Antibacterial Drugs

Posted on by Lorri Bingham

Over 2 million people in the United States are estimated to become infected with drug-resistant bacteria each year, so new antibacterial therapies are desperately needed. Streamlined drug development approaches have the potential to accelerate the availability of new antibacterial drugs for patients with serious unmet need who have limited or no treatment options. CTTI has published findings on patient and physician attitudes regarding streamlined development approaches for antibacterial drugs in BMJ Open.

Through a series of interviews and focus group discussions, CTTI found that patients and physicians agreed on the usefulness of streamlined approaches in situations of unmet need, but both groups also emphasized the need for careful oversight, transparency in risk communication, and continuous monitoring and reporting of safety and efficacy post-approval.

These findings, which resulted from CTTI’s Unmet Need in Antibiotic Development Project, can help inform the future use of streamlined drug development approaches and communication with stakeholders. For example, patients described information they would like to know when treatment with a drug developed using a streamlined approach is being considered. They also expressed a preference to have early conversations with their providers around the potential use of these treatments before they become seriously ill, when their decision-making may be impaired.

CTTI gathered input on these findings and their impact in a multi-stakeholder expert meeting; a meeting summary is available for additional context. CTTI would like to thank the interview and focus group participants, expert meeting attendees, and project team members who contributed to this work.

Patient and physician attitudes regarding risk and benefit in streamlined development programmes for antibacterial drugs: a qualitative analysis

Posted on by Hannah Faulkner

Patient and physician attitudes regarding risk and benefit in streamlined development programmes for antibacterial drugs: a qualitative analysis

Parent & Provider Input on Opportunities to Improve Antibacterial Clinical Trials: Findings Presented at Cystic Fibrosis

Posted on by Lorri Bingham

Breck Gamel presents CTTI posterOn October 27, CTTI presented findings from its Pediatric Trials in Antibacterial Drug Development Project at the North American Cystic Fibrosis Conference. Conducting antibacterial trials in pediatric populations has unique challenges, yet safe and effective therapies are desperately needed for this vulnerable group. We conducted surveys and interviews with pediatric providers, investigators, and parents to determine barriers to conducting these trials and obtain suggestions for strategies to overcome them.

View the poster to learn more about our findings:

  • Perceived barriers to conducting pediatric antibacterial drug trials
  • The need for better engagement with parents
  • Approaches for making studies child- and parent-friendly
  • Strategies for improving communication and enrollment

These findings, along with multi-stakeholder input, are being used to generate recommendations to improve the quality and efficiency of pediatric antibacterial drug trials.