Regulatory Submissions + Approvals

Disease Progression Modeling Expert Meeting

March 6, 2023

CTTI Project: Using Disease Progression Modeling to Advance Trial Design and Decision Making

MEETING OBJECTIVES:

  • Discuss disease progression modeling (DPM) and its current applications 
  • Explore opportunities, barriers, and best practices for advancing the use of disease progression modeling to aid in decision making 
  • Brainstorm relevant metrics to monitor and evaluate the recognition, value and consistent use of disease progression modeling  

Meeting Location: Mayflower Hotel, Washington, D.C.

Meeting Summary

Meeting Agenda

List of Meeting Attendees

Full Presentation Set            

Meeting Materials:

The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.

CTTI Conducting Survey on Registering and Reporting Clinical Trial Information on ClinicalTrials.gov

Posted on by Hannah Faulkner

The ClinicalTrials.gov Reporting Challenges Project is conducting a survey following in-depth interviews that were conducted in late 2021. We invite individuals who provide oversight, register, and/or report trial results information on ClinicalTrials.gov to complete the survey. You may also forward this survey to others who you know register and/or report clinical trial information on ClinicalTrials.gov.  

Additional information for potential survey participants is provided below. 

What are you being invited to do?  

CTTI is conducting a survey to learn more about organizations’ experiences with registering and reporting clinical trial information on ClinicalTrials.gov. 

We invite individuals at organizations such as academic institutions, medical product companies, government agencies, and non-government organizations who provide oversight, register, and/or report trial results information on ClinicalTrials.gov to complete the survey.  

The survey focuses on policies and approaches your organization implements to comply with ClinicalTrials.gov regulatory requirements, as well as the advantages and disadvantages of your approaches, challenges faced, and suggested strategies for addressing challenges. 

How will CTTI use the information? 

CTTI will outline useful practices for improving the reporting of timely, accurate, and complete registration and results information on ClinicalTrials.gov.  

Your name and organization will not be included when CTTI reports the survey findings. CTTI will send the FDA an aggregated summary of survey findings to inform their approach to supporting more complete reporting of clinical trial information. 

Interested in participating? 

If you provide oversight, register, and/or report clinical trial information on ClinicalTrials.gov, please click on this link to take the survey.

You may also forward this survey to others who you know register and/or report clinical trial information on ClinicalTrials.gov. 

Have questions? 

For questions about this survey, please email CTTI Project Manager Kelly Franzetti.

New CTTI Project Aims to Promote the Use of Disease Progression Modeling to Advance Trial Design and Decision Making

Posted on by Hannah Faulkner

Modeling and simulations are powerful tools that can be leveraged to inform clinical trial design, support regulatory decision making, and accelerate the process of bringing treatments to patients. Despite the benefits of these methods, their use is still not widely accepted among sponsors, investigators, and regulators. Recently, there has been growing interest across the clinical trials ecosystem in advancing the use of disease progression modeling to improve clinical trial quality and efficiency and inform regulatory decision making. Disease progression modeling leverages data from a variety of sources, improving trial diversity and combining many different models to help inform decision making. 

To promote these methods, CTTI has started a new project engaging a variety of stakeholders and experts in the development of recommendations and resources for the application of disease progression modeling in clinical trials. Through this project, CTTI will generate case studies detailing successful applications of disease progression modeling, a framework for the execution of disease progression modeling in clinical trials, a set of recommendations for the implementation of disease progression modeling in clinical trials, and a review paper detailing relevant disease progression modeling applications. This project aims to improve trial and clinical development efficiency by increasing the recognition, value, and consistent use of disease progression modeling. CTTI’s work in this area will also help to advance the broader application of modeling and simulation for trial design and regulatory decision making. 

To generate recommendations and resources supporting the application of disease progression modeling in clinical trials, CTTI will utilize a variety of iterative evidence generating strategies—including a scoping review assessing the landscape of disease progression modeling applications and an expert meeting discussing the barriers to disease progression modeling application and avenues for advancement. 

Through this work, CTTI will create recommendations and resources for designing clinical trials with a quality approach that maximally leverages available data—in alignment with CTTI’s Transforming Trials 2030 vision.

 

Using Disease Progression Modeling to Advance Trial Design and Decision Making

Topics Included: Innovative Trials, Regulatory Submissions + Approvals

Using modeling and simulations, researchers can make more informed decisions when planning and executing clinical trials. In particular, disease progression modeling (DPM) leverages multiple types of data from various sources to inform trial design and support regulatory decision making. To help advance the use of DPM in medical product development, CTTI has released recommendations that highlight its unique value and provide practical guidance on when and how to apply it effectively. These resources support efforts to bring treatments to patients more efficiently and promote broader adoption of modeling and simulation approaches in clinical research.

What is the Issue?
What CTTI project has been inititated to address the issue?
What are the objectives of CTTI’s work on using disease progression modeling to advance trial design and decision making?
What is the anticipated impact of CTTI’s work on this topic?
How will evidence be gathered to support our findings?
What products will result from this project?
Project Team Members

Resources

Regulatory Submissions + Approvals | Expert Meetings

Disease Progression Modeling Expert Meeting

March 6, 2023 CTTI Project: Using Disease Progression Modeling to Advance Trial Design and Decision Making MEETING OBJECTIVES: Discuss disease progression modeling (DPM) and its current applications Explore opportunities, barriers,...
Regulatory Submissions + Approvals | CTTI News

CTTI Conducting Survey on Registering and Reporting Clinical Trial Information on ClinicalTrials.gov

The ClinicalTrials.gov Reporting Challenges Project is conducting a survey following in-depth interviews that were conducted in late 2021. We invite individuals who provide oversight, register, and/or report trial results information on...
Site Planning | Case Studies

Accelerating eConsent Adoption During COVID-19

Accelerating eConsent Adoption During COVID-19
Regulatory Submissions + Approvals | Resources

Regulatory Engagement Opportunities when Developing Digitally Derived Endpoints

Regulatory Engagement Opportunities when Developing Digitally Derived Endpoints
Regulatory Submissions + Approvals | Resources

Question Bank for Identifying Meaningful Outcome Measures

Question Bank for Identifying Meaningful Outcome Measures
Regulatory Submissions + Approvals | Resources

Preparing a Digitally-derived Endpoint for Key Endpoint Use

Preparing a Digitally-derived Endpoint for Key Endpoint Use
Regulatory Submissions + Approvals | CTTI News

New CTTI Project Aims to Promote the Use of Disease Progression Modeling to Advance Trial Design and Decision Making

Modeling and simulations are powerful tools that can be leveraged to inform clinical trial design, support regulatory decision making, and accelerate the process of bringing treatments to patients. Despite the...
Regulatory Submissions + Approvals

Using Disease Progression Modeling to Advance Trial Design and Decision Making

Using modeling and simulations, researchers can make informed decisions when planning and executing clinical trials. In particular, disease progression modeling leverages multiple types of data from various sources to inform...
Regulatory Submissions + Approvals | CTTI News

Recording Now Available: “Next Steps for Obtaining Novel Endpoint Reliability & Acceptance” Webinar

A recording of CTTI’s public webinar on “Next Steps for Obtaining Novel Endpoint Reliability & Acceptance” is now available. During the webinar, presenters Lindsay Kehoe (CTTI) and Alicia Staley (Medidata)...
Regulatory Submissions + Approvals | CTTI News

CTTI to Host 10/5 Webinar on “Next Steps for Obtaining Novel Endpoint Reliability & Acceptance”

On Oct. 5 at noon ET, the Clinical Trials Transformation Initiative (CTTI) will host a public webinar on “Next Steps for Obtaining Novel Endpoint Reliability & Acceptance” – the third...

Formats

Stage of Trial

Recording Now Available: “Next Steps for Obtaining Novel Endpoint Reliability & Acceptance” Webinar

Posted on by Hannah Faulkner

A recording of CTTI’s public webinar on “Next Steps for Obtaining Novel Endpoint Reliability & Acceptance” is now available.

During the webinar, presenters Lindsay Kehoe (CTTI) and Alicia Staley (Medidata) discussed CTTI’s current work to obtain reliability and acceptance of meaningful, digitally derived novel endpoints – a project that builds on CTTI’s existing novel endpoint development recommendations and resources from 2017. They also provided:

  • Highlights from a recent Expert Meeting of leaders and stakeholders from across the clinical trials ecosystem
  • Insights from in-depth interviews with sponsors
  • A preview of the forthcoming set of CTTI novel endpoint recommendations and resources

This webinar built on recent webinars by DiMe, which focused on the best resources currently available for using digitally derived endpoints, and TransCelerate, which demonstrated key considerations and challenges for developing novel digital endpoints through a hypothetical case study.

“As the novel endpoints space continues to evolve, CTTI, DiMe, and TransCelerate are committed to working together to help stakeholders across the clinical trials enterprise evolve with it,” said Kehoe. “By encouraging greater collaboration and innovation among technology companies, academic investigators, patients, regulators, and trial sponsors and sites, we can accelerate progress to the benefit of everyone involved.”

For more on CTTI’s work around Novel Endpoints, please visit CTTI’s website.

CTTI to Host 10/5 Webinar on “Next Steps for Obtaining Novel Endpoint Reliability & Acceptance”

Posted on by Hannah Faulkner

On Oct. 5 at noon ET, the Clinical Trials Transformation Initiative (CTTI) will host a public webinar on “Next Steps for Obtaining Novel Endpoint Reliability & Acceptance” – the third in a series of webinars on using novel digitally derived endpoints in clinical trials for medical product development from CTTI, Digital Medicine Society (DiMe), and TransCelerate.

Presenters Lindsay Kehoe, CTTI, and Alicia Staley, Medidata, will discuss CTTI’s current work to obtain reliability and acceptance of meaningful, digitally derived novel endpoints – a project that builds on its existing novel endpoint development recommendations and resources from 2017.

Attendees will hear:

  • Highlights from a recent Expert Meeting of leaders and stakeholders from across the clinical trials ecosystem
  • Insights from in-depth interviews with sponsors
  • A preview of the forthcoming set of CTTI novel endpoint recommendations and resources.

This webinar builds on recent webinars by DiMe, which focused on the best resources currently available for using digitally derived endpoints, and TransCelerate, which demonstrate key considerations and challenges for developing novel digital endpoints through a hypothetical case study.

“As the novel endpoints space continues to evolve, CTTI, DiMe and TransCelerate are committed to working together to help stakeholders across the clinical trials enterprise evolve with it,” said Kehoe. “By encouraging greater collaboration and innovation among technology companies, academic investigators, patients, regulators and trial sponsors and sites, we can accelerate progress to the benefit of everyone involved.”

Register to attend “Next Steps for Obtaining Novel Endpoint Reliability & Acceptance” today.