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Improved Study Protocol Design for a Rare Disease through Patient and Caregiver Interviews

Featured Solution: Patient Protocol Engagement Toolkit (P-PET)


A sponsor company used parts of the Patient Protocol Engagement Toolkit (P-PET) for patient and caregiver (patient’s mother) interviews to obtain patient insights for a recent study protocol. The interviews were conducted virtually. The interview slide deck was developed with reference to the P-PET toolkit, and some questions were modified for this study. Before the interview, the patient expressed a desire to be interviewed separately from their caregiver. The patient said they would be unable to “speak honestly” if their mother (caregiver) was also in the interview. Based on this request, the interviews were conducted separately and at different times. This interview accommodation allowed the interviewers to have a more honest discussion with the patient.

Findings from the Interview

Below are some examples of the types of patient experiences and findings that were collected in the sponsors’ interviews with the patient and caregiver:

  • Disease and treatment
    • Since this is a rare disease, many physicians do not know the diagnostic methods, which can cause trial participation delays.  
      • Some of the procedures needed to diagnose this disease can burden patients, and patients don’t like these procedures.
      • For the patient, the worst symptoms were respiratory ones.
  • Clinical protocol and trial design
    • Patients often take nursing care in their homes. The trial design should consider the patient’s nursing care needs and schedule.
  • Recruitment, enrollment, and retention strategy
    • Patients have a community to exchange information. Collaboration with this community is one of the options for recruiting and encouragement for patients.
    • Many patients see clinical trials as a treatment option because the standard of care is limited. So, many patients want to participate even if the clinical trial site would be far from their homes.
  • Use of innovative trial solutions
    • A DCT option and a satellite site to gather patients would help reduce patient burden given their needs and the low number of people with the disease.     

Interview Impact

Ultimately, the patient experience interviews informed the sponsor’s development program by helping the study team gain new insights on symptomology from understanding the disease and treatment burden. This interview was the first case for the company to ask about patient experiences directly to the patient and caregiver. As this was the first case, the company didn’t have an original slide template for the interview, but the P-PET reminded them to add some information to the slide deck, such as the participant list. In addition, the patient discussion informed the study’s feasibility and gave the team many considerations for the overall study design. The P-PET question bank has many comprehensive questions that allowed the team to gather more high-quality patient data in the interview. Thanks to the P-PET, the team could imagine the patient interview general flow and general question sample.

[Sponsor Company]