Advancing Decentralized Clinical Trials for Rare Diseases and Special Populations: Perspectives and Opportunities

Session Description

Decentralized clinical trials (DCTs) offer a shift in the field of clinical research, with potential to optimize the established clinical trial approach. DCTs can offer greater inclusivity and diversity for trial participants especially for patient populations with unique challenges. DCTs aim to empower participants to engage in research from the comfort of their own homes and reduce the burden of frequent site visits. Within rare diseases and special populations, DCTs offer unique potential to reduce patient burden and advance drug development more effectively.

Considering the emerging nature of DCTs, it is important to gain perspectives from multiple stakeholders to best navigate the implementation DCTs for rare diseases and special populations. Specifically, understanding both opportunities and challenges in the context of patient-centric DCT aspects can help industry sponsors create the most opportunities for diversity and inclusion while aiming to increase trial efficiency.
Perspectives from regulators and patient advocate groups should be incorporated into the strategies around DCT implementation for these patient groups.

Regulatory considerations play a crucial role helping sponsors to navigate guidelines and ensure compliance with regulatory authorities. However, it also stimulates conversation between regulators and industry when innovative areas within DCT elements are emerging. In conjunction, acquiring lessons learned from successful industry case studies helps bolster industry momentum around DCTs, specifically when challenges and respective mitigation strategies were implemented. Lastly, understanding the patient voice is critical to ensure sponsors are incorporating what matters to patients most when approaching DCT design, especially within rare diseases.

This session will provide attendees with insights around DCT principles from regulatory, industry, and patient-centric perspectives using case studies from rare and special populations. Connecting with the ASCPT theme "Patient-Centric Clinical Pharmacology: A Journey from Discovery to Recovery," this session underscores the importance of placing patients at the center of clinical research and pharmacological interventions.

Learning Objectives

1. Understand multi-stakeholder perspectives as it relates to DCT needs and implementation in special and rare populations.
2. Understand the application of DCT implementation in a special population through an industry case study example highlight success and challenges mitigated when incorporating DCT elements for a special population.
3. Understand perspectives from a patient advocacy perspective as it relates to DCT implementation. Patent advocacy perspectives will provide direct insights into patient burdens with a rare disease, and what DCT elements offer the most opportunity for dive

Session Schedule

Introduction
Jackson Burton, PhD and Katty Wan, PhD

Advancing Data Collection in Specific Populations through Decentralized Clinical Trials
Elimika Fletcher, PharmD, PhD

Embracing Innovation in Decentralized Clinical Trials: Industry Case Study
Laura Shaughnessy, PhD

Patient-Centricity and Engagement in Decentralized Clinical Trials: Patient Advocate Perspectives
Michelle Lorenz

Panel Discussion