Patient and Caregiver : Study Concept

Patient and Caregiver : Study Concept

Trial Design Stage

Published Date: April 29, 2025

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Helpful Terms and Definitions

ClinicalTrials.gov Glossary Terms

NIH Glossary of Common Terms

FDA Drug Development and Review Definitions

Resources*

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*This list is not exhaustive, nor does CTTI officially endorse the resources created by other organizations.

Study Concept

Define Patients and Caregivers

Who makes up patients and caregivers?

  • Patient organization representatives across diverse demographics and, where appropriate, across regions or countries (e.g., age, race/ethnicity, gender, disease state)
  • Both experienced patients/caregivers and those with little or no clinical trial experience
  • To successfully inform on the trial design, a small, focused group (typically 3–4 individuals) should be convened to allow meaningful discussion

Where will you find them?

Start by mapping groups aligned with the research focus and building relationships over time. Maintain and refresh this mapping periodically.

  • Engage with patient organizations and advocacy groups relevant to the disease area
  • Consider treating patient representatives as subject matter experts based on lived experience
  • Allow adequate time to identify and build trust with these communities (timelines may vary depending on the disease area and access to groups)

To identify research-naïve individuals and broaden representation, consider engaging:

  • Federally qualified health centers (FQHCs)
  • Safety-net hospitals
  • Community-based organizations and groups serving underrepresented populations

Why involve patients and caregivers now?

Early engagement helps sponsors understand the needs, preferences, and lived experiences of people affected by the disease.

It also provides an opportunity to:

  • Build trust and ongoing relationships
  • Validate the study rationale from a patient perspective
  • Identify unmet needs and real-world care dynamics
  • Understand patient burden to better inform study objectives and design

Engaging patients and caregivers at the study concept phase helps ensure the study is relevant, feasible, and meaningful to the population it aims to serve.

Patients don’t just improve trial design—they expand what we believe can be designed.

Questions To Ask

open drawer
  • Why is this study appealing to the patient community?
  • How and when are patients typically diagnosed?
  • Is the study flow burdensome for patients?
  • Do the inclusion/exclusion criteria reflect the real-world patient population?
  • Are these the outcomes that matter most to patients?
  • How is patient health information being protected and used?
  • What does the risk–benefit profile look like from a patient perspective?
  • What types of control groups or treatments are being used, and are they acceptable to patients?
  • What options are available if a patient’s condition worsens during participation?
  • What is the long-term plan (e.g., open-label extension or continued access)?
  • What types of providers are involved in care?
  • What barriers exist to accessing treatment, and are there ways to address them?
  • Is there an opportunity to engage underrepresented patient and caregiver communities?
  • Are there deeper community connections available through disease-specific foundations or advocacy groups?
  • How can the burden of participating in the clinical trial be reduced for the patient and their caregiver?
  • If this were a study you were participating in, what would you change before enrolling yourself or someone you love?

Patient and Caregiver Resources

  1. Regulatory & Foundational Policy

These resources establish the "why" and the legal/ethical "how" for patient engagement. They are essential for briefing packages and regulatory interaction planning.

  1. Operational Planning & Co-Creation Toolkits

These tools are designed for protocol designers and clinical project leads to use during the study concept and synopsis stages.

  1. Patient Experience Mapping & Qualitative Research

These resources assist in identifying patient burdens and unmet needs to inform study endpoints and design features.

  1. Metrics, Valuation, & Specialized Standards

These resources allow you to measure the quality of your engagement and manage the financial/logistical aspects of collaborations.

  1. Digital & Decentralized Trial Implementation

Resources specific to the technical and operational shift toward modern, patient-centric trial delivery.