Author: Hannah Faulkner

FDA, CTTI Convening Virtual Public Workshop to Discuss FDA Emergency Recommendations to Mitigate Clinical Study Disruptions

Posted on by Hannah Faulkner

The Food and Drug Administration (FDA), in collaboration with the Clinical Trials Transformation Initiative (CTTI), is convening a virtual public meeting on Oct. 18 and 19, 2023 to discuss recommendations provided by the FDA during the COVID-19 public health emergency (PHE) to mitigate disruption of clinical studies, including recommendations detailed in the guidance entitled “Conduct of Clinical Trials of Medical Products During the COVID-19 Public Health Emergency, Guidance for Industry, Investigators, and Institutional Review Boards.”

Registration for the meeting is now open.

We are inviting those who register to submit questions for our speakers and panelists in advance of the meeting. Please click here to submit your questions.

The meeting will also include discussions with representatives from the FDA, pharmaceutical and medical device industries, academia, research study teams, patients, and patient advocacy groups, and other federal agencies, who will share the experiences, best practices, and approaches they used to effectively navigate disruptions to clinical studies during the COVID-19 PHE. An agenda for the meeting can be viewed here.

The meeting fulfills a requirement under section 3605 of the Food and Drug Omnibus Reform Act of 2022 (FDORA) and will discuss potential strategies for advanced planning to mitigate clinical study disruptions in the future.

The free two-day virtual public meeting will be held on Oct. 18 and 19 from 10 a.m. – 1:30 p.m. ET on both days.

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.