Patient Protocol Engagement Toolkit

Problem:

• The study team was unsure of both the number and timing of procedures.
• Were they out of sync with the practical realities of the health care system or patient lives?

Insight:

• Patients provided their comments on the acceptability of the proposed procedures and associated timings.

Action:

• Through patient and site collaboration, the sponsor redesigned the schedule of procedures.

Problem:

• The study team was considering requiring an invasive procedure in immunocompromised patients.

Insight:

• Patients informed sponsor that this would negatively impact willingness to participate.

Action:

• The protocol was changed to make the procedure optional.

Problem:

• Multiple pills involved in treatment therapy were similar in appearance.
• Presented a new opportunity for our drug manufacturing to explore how they might consider patient input in their work beyond the size and taste of a pill.

Insight:

• Patients helped to create solutions to better discern the different pills and any associated activities.

Action:

• Creative solutions were implemented to help improve drug compliance and prevent possible medication error.

Problem:

• Previous experience demonstrated patients are challenging to recruit to studies.

Insight:

• The proposed duration of visits and frequency of tests were burdensome.
• Patients appreciated the proposed ePRO.

Action:

• Reduced duration of visits and frequency of tests, as time burden was a key patient concern.
• The decision to proceed with ePRO in response to positive patient feedback.

Outcome:

• Reduced screen failures, reduced drop-outs, increased enrollment rate, zero (0) avoidable amendments.

Problem:

• The study team wanted to further understand what type of outcomes from their treatment mattered most to patients, including physical, emotional and social functioning.

Insight:

• Interviews & surveys highlighted the importance of the ability to function and partake in daily activities.

Action:

• Developed an instrument to capture these PROs during trials.
• Developed a clinical practice tool to capture patient experiences.

Outcome:

• Included objective data on the results for the MPFID endpoint in the label.

Problem:

• The study team wanted to determine how to meet the needs of a diverse patient population.

Insight:

• Gained information on patients’ overall perspective of clinical studies, their decision-making process, barriers, and motivating factors impacting participation and raising disease awareness.

Action:

• Focused efforts on educational initiatives related to patients’ general knowledge and perceptions of clinical trials and educated investigators on patient educational needs while enrolling.
• Worked to facilitate the use of more diverse imagery in clinical trial materials.
• Added as much specific information related to prior trials and the present trial to patient materials as possible.
• Included patient feedback during the development of patient brochures and other educational materials.

Problem:

• The study team wanted to understand the patient perspective on the clinical trial experience.

Insight:

• Important concepts and dominant trends in each interview were identified, and results were compared across interviews to allow for the generation of themes or patterns in participants’ responses.

Action:

• Qualitative information provided additional data to assist in interpreting the quantitative data from the trial, giving an in-depth understanding of patient experiences and can be used for future hypothesis generation.

Problem:

• Patients perceive a high burden of participating in a clinical trial.

Insight:

• Proposed duration of visits and frequency of tests were burdensome.
• Patients appreciated the proposed ePRO.

Action:

• Reduced duration of visits and frequency of tests, as time burden was a key patient concern.
• The decision to proceed with ePRO in response to positive patient feedback.

Outcome:

• Reducing burden and improving the patient experience led to reduced screen failures, reduced drop-outs, increased enrollment rate, zero (0) avoidable amendments.