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Client Success

Designing a rare disease study with patient input

rare disease advisory board


A medical affairs team within a leading pharmaceutical company wanted to gather patient perspectives about living with a chronic, but treatable, rare disease.

The team had three primary objectives:

  1. Understand patient perspectives on a gene therapy clinical trial
  2. Obtain feedback on patient materials
  3. Gather input on patient education needs


The medical affairs team decided to reach out to patient advisors via the Within3 insights management platform, which would not require the patients to travel or all be available to attend a webcast at the same time. This was especially important given that patients were located in the United States, Europe, and Canada.

Patients answered multiple-choice and open-ended questions, including How willing do you think people would be to participate in the study? and How do you educate yourself and your community about your condition? Participants also used Within3’s document annotation features to provide input on patient-facing materials. During the sessions, moderators used the platform’s built-in tools to remind or encourage patients to respond to questions or contribute to the conversation.


By using the Within3 platform, the medical affairs team achieved its objectives for the rare disease patient advisory board. With the virtual engagement approach, the medical affairs team was able to:

  1. Receive robust patient feedback on the observational study
  2. Obtain detailed responses pertaining to brochures and other patient materials
  3. Gather valuable insight to update patient-facing educational content

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