Patient and Caregiver
Investigator
Regulator
IRB
Tech, Device,
PayerTrial Design Stage
Published Date: April 29, 2025
Share to:
Helpful Terms and Definitions
Study Concept
Is there an opportunity to engage underrepresented patient and caregiver communities?
Are deeper community connections available through foundations specific to the disease area?
Questions To Ask
- Why is this study appealing to the patient community?
- Is the flow burdensome for patients?
- Are the inclusion/exclusion criteria accurate to the current patient population?
- Are these the endpoints and outcomes that matter most to the patients?
- How is patient health information being protected and used?
- What does the risk to benefit analysis look like?
- What types of control groups and treatments are being used?
- What are the options for patients whose disease worsens during treatment?
- What is the long-term strategy - is there an open label extension period?
- How and when are patients diagnosed?
- What type of providers are giving care?
- What are barriers to accessing treatment? Are there solutions?
Define Patients and Caregivers
Who makes up Patients and Caregivers?
- Patient organization representatives across demographics and, if applicable, from multiple disease types and countries (consider, as appropriate, age, race/ethnicity, gender, disease-state, etc.)
- Both expert patients/caregivers and patients/caregivers with little to no clinical trial experience
- This group should be comprised of a very limited number of representatives (3-4 people)
Where will you find them?
Start by mapping groups that have interest in the particular research focus, establishing a relationship with these groups - make sure to periodically refresh the mapping.
- Begin to treat these groups as key opinion leaders
- Dedicate 6 months to identifying and establishing relationships
To find research naïve individuals and a representative group, consider also mapping less traditional partners such as federally qualified health centers and safety net hospitals, as well as organizations like the Urban League Health Ministry.
Why involve Patients and Caregivers now?
Early engagement of patients and caregivers helps sponsors understand the needs and perspectives of the specific patient/caregiver group affected by the disease area. It's also an opportunity to develop a relationship with patients and caregivers that will facilitate further patient engagement throughout the present study and future studies. Sponsors should begin to engage patients and caregivers at the study concept phase to ensure their unmet needs are being addressed, validate the treatment rationale and understand the real-world setting, and obtain a deeper understanding of the patient burden to guide selection of primary and secondary objectives.
Patient and Caregiver Resources
Patient Focused Drug Development Guidance
Patient Protocol Engagement Toolkit
Practical How-to Guides for Patient Engagement
Paradigm Patient Engagement Toolbox
Patient Engagement Quality Guidance Tool
Framework for Patient Engagement in Cancer Network Group Studies
Representativeness in Patient Engagement
Assessing Meaningful Patient Engagement in Drug Development: A Definition, Framework, and Rubric
Assessing the Financial Value of Patient Engagement
Streamlining Patient Recruitment with AI-driven Site Identification
CTTI: Patient Group Engagement
Complete Guide to Remarkable Patient Engagement