Author: Hannah Faulkner
New CTTI Project to Develop Common Framework to Assess Progress Towards TT2030 Vision
Clinical trials are critical components of the evidence generating system in research. Launched in 2021, CTTI’s Transforming Trials 2030 vision outlines five pillars for how clinical trials should be performed by 2030. This vision establishes that clinical trials need to be patient centered and easily accessible, fully integrated into health processes, designed with a quality approach, maximally leveraging all available data, and improving population health. Currently, the TT2030 vision lacks metrics to demonstrate progress towards the vision’s five central goals. The Measuring Trials Transformation (MTT) Project will develop a common framework to evaluate and assess the progress made towards achieving the five main goals of the TT2030 vision, a framework that can be used to share accountability across the clinical trials enterprise.
After launching the TT2030 vision, CTTI conducted concept elicitation interviews to develop key concepts metrics should address within each pillar. In 2022 and early 2023, CTTI conducted surveys of experts to assess the value of individual metrics for assessing progress towards the vision.
To deploy a set of relevant metrics, CTTI will utilize a variety of iterative strategies, including interviews with key stakeholders on their experiences with each pillar and ideas for related metrics, expert surveys on draft measures for each pillar, and an online public forum on the measurement framework. The selected metrics will be further refined by integrating public comment, expert input, and project team feedback and discussions. CTTI will perform a case study using the framework of metrics to measure progress towards the TT2030 vision by collecting and reporting an initial set of metrics using recent clinical trials.
CTTI understands that some goals of the TT2030 vision will be achieved sooner than others, and so the framework created by the MTT Project will establish key milestones to assess progress made by the program over time. CTTI will use this vision to guide their priorities, and they will encourage others to do the same, facilitating accountability across the entire clinical trials industry.
The Metrics Framework is currently open for public comment until mid-October 2023. Please view the dashboard and provide your suggestions and feedback here.
FDA, CTTI Convening Virtual Public Workshop on Enhancing Clinical Study Diversity
The U.S. Food and Drug Administration (FDA), in collaboration with the Clinical Trials Transformation Initiative (CTTI), is convening a virtual public workshop to solicit input on increasing the enrollment of historically underrepresented populations in clinical studies and encouraging clinical study participation that reflects the prevalence of the disease or condition among demographic subgroups.
The free two-day virtual public workshop will be held on November 29 and 30, 2023, from 10 a.m. – 2 p.m. EST on both days. Registration for the workshop is now open and an agenda for the workshop can be viewed here.
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.
Representatives from academia, clinical research organizations, drug sponsors, medical device sponsors, patients, and other interested parties will share their experiences and approaches for increasing enrollment and encouraging participation of historically underrepresented populations in clinical studies.
The workshop fulfills a requirement under section 3603 of the Food and Drug Omnibus Reform Act of 2022 (FDORA).
CTTI Executive Director Sally Okun Announces Retirement
I wanted to let you know that after much consideration and reflection I am retiring from my position as Executive Director of the Clinical Trials Transformation Initiative by the end of 2023.
My time at CTTI has been incredibly rewarding both professionally and personally and my decision to retire was not an easy one. I am grateful for the support, opportunities, and camaraderie I have experienced throughout my time here. As I prepare to embark on a new chapter after five decades in healthcare, I feel a mixture of emotions, including excitement, as I contemplate the future for me and my loved ones.
When I arrived at CTTI in October of 2021, we were navigating our way into a post pandemic world and changing clinical trial ecosystem. Our CTTI team and members stepped up to meet these challenges and opportunities with creativity, curiosity, and resilience. It has been a privilege to work alongside this talented team and the many dedicated professionals, patients, and caregivers from across our membership who demonstrate their commitment to CTTI’s mission and bold vision each day.
Over the next several months, I am committed to ensuring a smooth transition of my responsibilities to my successor. I’m working closely with Duke and CTTI’s Executive Committee leadership – Chair Mark McClellan, Duke Co-chair John Alexander, and FDA Co-chair Khair ElZarrad – on a thoughtful and mindful succession plan that ensures the highest level of team and organizational integrity.
In addition, the CTTI team is actively involved in developing a transition process that includes their voices and reflects their perspectives about the next generation of leadership for our organization. The job posting for the Executive Director of CTTI has posted to Duke’s career webpage. I welcome any thoughts, ideas, and suggestions you wish to share as we begin this process.
And finally, I want to express my deepest gratitude to our CTTI community for making my experience with CTTI unforgettable. It gives me great pride to have been a part of CTTI’s work and achievements during my tenure. Our exciting and important work continues this fall and rest assured we won’t miss a beat with this transition.
FDA, CTTI Convening Virtual Public Workshop to Discuss FDA Emergency Recommendations to Mitigate Clinical Study Disruptions
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)
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
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:
- a lack of regulatory guidance specific to digitally derived endpoints;
- a qualification process for new clinical outcomes that is impractical for most industry sponsors;
- a lack of comparable clinical endpoints to validate novel digitally derived endpoints;
- a lack of validated DHTs and algorithms for a concept of interest; and
- 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.