Formats

Now Accepting Applications for FDA and CTTI Patient Engagement Collaborative (PEC)

Posted on by Hannah Faulkner

The FDA and CTTI are currently accepting applications from patient advocates interested in becoming members of the Patient Engagement Collaborative (PEC). The PEC is an ongoing, shared forum in which the patient community (PEC members), FDA, and CTTI discuss a variety of topics such as improving communication, education, and patient engagement related to medical product regulation.

Applicants will be selected based on their ability to meaningfully contribute to the PEC, represent and express the patient voice for their constituency, work in a constructive manner with involved stakeholders, and understand and navigate the clinical research system.

Successful applicants will include:

  • Patients who have personal disease experience
  • Caregivers who directly support patients (e.g., a family member or friend) and have personal disease experience through this caregiver role
  • Representatives from patient groups who, through their role in the patient group, have direct or indirect disease experience

PEC members participate in two to four virtual meetings per year. Other meetings may be organized, as needed.

The following PDF format documents are required to apply and must be submitted at the same time:

  • A current and complete one- to two-page resume or bio that summarizes your patient advocacy experience and related activities; and
  • A one-page professional letter of endorsement from a patient group with which you have worked closely on activities that are relevant to the PEC

A maximum of 75 applicants will be considered for PEC membership and up to 8 members will be selected. Interested applicants are encouraged to complete and submit the online form starting at 10:00 a.m. EDT on July 14, 2023. This announcement is open to receive a maximum of 75 applications.

Applications will be accepted until 11:59 p.m. Eastern Time on August 14, 2023, or until 75 complete applications are received, whichever happens first.

Those who are unable to apply electronically are encouraged to call FDA Patient Affairs at 301-796-8460 to arrange for a mail submission.

Please review the related Federal Register Notice for important information about the application process.

CTTI Publication Investigates Challenges and Solutions Surrounding Digitally Derived Endpoint Acceptance

Posted on by Hannah Faulkner

A new CTTI publication, published in Digital Biomarkers, investigates the challenges sponsors encounter when using digitally derived endpoints and offers potential solutions. Digital health technologies – including mobile apps, smart devices, sensors, and wearables – can capture robust data from participants’ daily lives. These digital tools allow researchers to collect data for novel endpoints that can provide a broader view of health and treatment effects using measures that matter to patients. Despite these benefits to clinical trial quality and efficiency, digitally derived endpoints are still underutilized as primary evidence in regulatory submissions.

To evaluate the barriers and challenges associated with digitally derived endpoints to support labeling claims, CTTI interviewed 20 sponsor representatives from 10 different organizations representing 11 individual clinical trials. During these interviews, conducted between November 2020 and March 2021, sponsor representatives were asked about their experiences related to digitally derived endpoints, their interactions with regulators, and the challenges they encountered. Based on these interviews, CTTI identified five key challenges to incorporating digitally derived endpoints in clinical trials:

  1. a lack of regulatory guidance specific to digitally derived endpoints;
  2. a qualification process for new clinical outcomes that is impractical for most industry sponsors;
  3. a lack of comparable clinical endpoints to validate novel digitally derived endpoints;
  4. a lack of validated DHTs and algorithms for a concept of interest; and
  5. a lack of operational support from technology vendors.

CTTI shared these interview findings with the U.S. Food & Drug Administration (FDA) and the European Medicines Agency (EMA) and during a multi-stakeholder expert meeting. Through these discussions, CTTI drafted and refined a set of recommendations and tools for developing novel digitally derived endpoints as part of the Digital Health Trials Hub. These resources provide a robust toolkit for sponsors, investigators, and operational partners navigating the regulatory landscape around digitally derived endpoints.

How Much Evidence Is Enough? Research Sponsor Experiences Seeking Regulatory Acceptance of Digital Health Technology-Derived Endpoints

Posted on by Hannah Faulkner

How Much Evidence Is Enough? Research Sponsor Experiences Seeking Regulatory Acceptance of Digital Health Technology-Derived Endpoints

Recording Now Available: CTTI Webinar Unveiling New Recommendations on Increasing Diversity in Clinical Trials

Posted on by Hannah Faulkner

A recording of CTTI’s May 18 webinar unveiling the new recommendations on increasing diversity in clinical trials is now available. 

The one-hour webinar includes a welcome from Sally Okun, CTTI; opening remarks from Richardae Araojo, U.S. Food and Drug Administration (FDA); a project overview from Luther Clark, Merck; a maturity model overview from Dawn Corbett, NIH; and a panel discussion moderated by Sara Calvert, CTTI. The panel discussion focused on stakeholder perspectives related to the integration of the of the new diversity recommendations and included Ruma Bhagat, Genentech-a member of the Roche Group; Tesheia Johnson, Yale; Jane Williams, Syneos Health; and Glendon Zinser, Susan G. Komen. 

The goal of creating these diversity recommendations is to increase the inclusion and adequate representation of women and historically underrepresented racial and ethnic groups in clinical trials to facilitate more accurate and generalizable trial results by enhancing and expanding our understanding of the safety and efficacy of investigational medical products. These recommendations were developed in collaboration with experts and key stakeholders from across the clinical trials enterprise using CTTI’s five-step methodology designed to ensure they are actionable, evidence-based, and consensus-driven. The first phase involved gathering input through qualitative, in-depth interviews with senior-level leaders at organizations conducting clinical trials to gather perspectives on strategies to support diversity and inclusion in clinical research. The recommendations were drafted and refined with input from a multi-stakeholder project team and through a 2-day Expert Meeting. 

View the slide deck to read more. 

 

CTTI to Launch New Diversity Recommendations at Free Public Webinar

Posted on by Hannah Faulkner

CTTI will host a free public webinar on Thursday, May 18 at 12:00 p.m. EDT to unveil new recommendations for increasing diversity in clinical trials.  

The webinar will include a welcome from Sally Okun, CTTI; opening remarks from Richardae Araojo, U.S. Food and Drug Administration (FDA); a project overview from Luther Clark, Merck; a maturity model overview from Dawn Corbett, NIH; and a panel discussion that will focus on stakeholder perspectives related to the integration of the new recommendations. 

The inclusion and adequate representation of women and historically underrepresented racial and ethnic groups in clinical trials leads to more accurate and generalizable trial results by enhancing and expanding our understanding of the safety and efficacy of investigational medical products. In addition, inclusion of all the populations who are affected by a condition can improve access to innovative and potentially life-extending and life-improving therapies, develop trust in clinical trial results, and facilitate uptake if the medical product is approved. 

As part of the Diversity Project, experts and key stakeholders from across the clinical trials enterprise developed the recommendations following CTTI’s five-step methodology designed to ensure they are actionable, evidence-based, and consensus-driven. The CTTI recommendations and supporting maturity model seek to build on the growing recognized need for long-term, transformative strategies that are rooted in a deep organizational commitment to developing clinical trial research infrastructure that is more responsive to the needs of historically underrepresented populations. 

Register for the free one-hour webinar. 

CTTI Holds Meeting to Discuss Engaging Stakeholders in Trial Design

Posted on by Hannah Faulkner

The Clinical Trials Transformation Initiative (CTTI) held a multi-stakeholder Expert Meeting on April 4 to discuss high-value approaches and situation-specific considerations for meaningful engagement of internal and external stakeholders in clinical trial design and implementation. Engaging all stakeholders during the earliest stages of study development is an important feature of quality by design (QbD). Aligned with CTTI’s QbD approach, the Engaging Stakeholders in Trial Design Project team is developing an engagement roadmap and recommendations to enable clinical trial designers to meaningfully and effectively engage all stakeholders across the trial design process. The project team is also collating resources surrounding the engagement of stakeholders to increase the efficiency of trial design to benefit patients faster. 

Experts from academia, clinical research organizations, patient advocacy groups, regulatory agencies, and the pharmaceutical industry discussed the challenges and opportunities, key strategies, and metrics for assessing holistic stakeholder engagement across the continuum of clinical trials. The following key themes were emphasized during the meeting: 

  • It is important to engage key stakeholders – including patients, site staff, and regulatory agencies – very early in the design of clinical trials. When planning study timelines, identify all internal and external stakeholders and the appropriate time and approach to solicit their input. 
  • Stakeholder engagement should be an iterative process throughout the life cycle of clinical trials. Internal and external stakeholders should be engaged, as appropriate, from the beginning of clinical trial design through the dissemination of results. 
  • Bring stakeholders and functional groups together to identify gaps between teams and brainstorm solutions to increase the quality and efficiency of trials. Effective communication and collaboration across all stakeholder groups is crucial to create a community around designing high-quality studies that meet the needs of patients and that generate reliable evidence with fewer amendments.  
  • Advanced methodologies and tools, such as artificial intelligence and machine learning, can help study designers develop innovative, high-quality trials and streamline processes. With databases of real-world and clinical trial data, artificial intelligence and machine learning can be used to inform the design of trials, explore potential treatments for patient subgroups, assess feasibility of enrollment, and identify sites. 
  • A collection of resources for designing clinical trials, including recommendations on how and when to meaningfully engage all stakeholders, is needed to help study designers plan innovative clinical trials more efficiently and in alignment with regulatory guidance.