NIDDK Workshop on T1D Disease Modifying Therapy and Prevention

Event Details

Meeting Summary—NIDDK Workshop on T1D Disease Modifying Therapy and Prevention (PDF, 213.55 KB)

Background

In recent years, substantial advances have been made in identifying individuals at risk for the development of type 1 diabetes and in altering the course of the disease before and after clinical type 1 diabetes diagnosis. Specifically, seven immune therapies have been shown to alter the disease course after clinical diagnosis, and one therapy actually slowed disease progression prior to clinical diagnosis. In addition, people destined to develop type 1 diabetes now can be identified reliably, and close follow-up of at-risk individuals has been shown to reduce the incidence of diabetic ketoacidosis among those who do progress to diabetes. Type 1 Diabetes TrialNet, funded by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), has made many of these successes possible.

TrialNet’s achievements and capabilities highlight the promise of both immune therapy and prevention in type 1 diabetes and suggest that the field is poised to translate these findings into clinical practice. The NIDDK and TrialNet consider this to be an opportune time for a new framework for collaborative efforts among stakeholders to advance the field.

Meeting Objectives

The NIDDK is bringing TrialNet leadership together with representatives from the pharmaceutical industry, research-funding advocacy groups and nonprofits, and health care systems to discuss bringing disease-modifying and prevention therapies to clinical use. The overarching aim of this collaboration is to clarify specific challenges along the path to clinical translation, including identification of individuals at risk, trial conduct and facilitation of the pathway for clinical approvals, and implementation. Initial discussions will focus on understanding different perspectives, clarifying areas of interest for such collaborative efforts, and identifying champions or small groups to continue to develop specific activities and timelines. These initial efforts hopefully will result in an agenda by which energies can be combined to develop a comprehensive strategy to bring research results to the bedside and make diabetes prevention part of good clinical practice.

This workshop will be held in two sessions: Session 1 on May 14, 2021, and Session 2 on May 21, 2021. Each session will be held from 10:00 a.m. to 1:30 p.m. EDT. Session 1 will focus on identification of at-risk individuals, and Session 2 will emphasize disease-modifying therapies.

This workshop is open to the public, but active participation in discussion during the meetings will be limited to a small group of invited individuals. A meeting summary will be released shortly after the meeting is held.

Registration Deadline

May 05, 2021

Agenda

May 14, 2021

TOPIC: Identifying At-Risk Individuals

10:00 a.m. – 10:30 a.m.

Introduction

10:30 a.m. – 11:15 a.m.

Three concurrent break-out sessions. Each group is to address the same questions:
How do such goals as preventing diabetic ketoacidosis, enrolling in clinical trials, and assuring a market for therapy compete with or complement each other?
What are the scientific gaps in understanding and using different populations, algorithms, and assays?
What are the knowledge or education gaps of clinicians and individuals?
What information is needed by regulators, insurers, and clinical practice guidance groups to bring screening and monitoring to clinical use?

11:15 a.m. – 11:30 a.m.

Break

11:30 a.m. – 12:30 p.m.

Groups to report on their discussion (20 minutes each)

12:30 p.m. – 1:30 p.m.

Next steps: Specific projects for further discussion and prioritization of cross-stakeholder collaborative efforts to bring forward within or external to the framework of the Foundation for the (FNIH)

May 21, 2021

TOPIC: Disease-Modifying Therapy

10:00 a.m. – 10:30 a.m.

Introduction

10:30 a.m. – 11:15 a.m.

Three concurrent break-out sessions. Each group is to address the same questions:
What are the considerations (challenges and opportunities) of using a network to gain efficiencies in conducting clinical trials?
What information is needed by payors or those that issue clinical practice guidelines to allow disease-modifying therapy in clinical care?
What is needed to bring clarity to the pathway to regulatory approval pre- and post- clinical diagnosis?
What are the best ways to address the knowledge gaps by primary care and specialty physicians (e.g., endocrinologists) regarding the use of disease-modifying therapy?
How can data and samples be used to identify targets most effectively and understand response to therapy?

11:15 a.m. – 11:30 a.m.

Break

11:30 a.m. – 12:30 p.m.

Groups to report on their discussion (20 minutes each)

12:30 p.m. – 1:30 p.m.

Next steps: Specific projects for further discussion or prioritization of cross-stakeholder collaborative efforts to bring forward within or external to the framework of DNIH

1:30 p.m.

Adjournment