Sometimes it really is a zebra! Listening to the patient voice without burdening the rare disease community

Industry Insights



We hear so much about the importance of “the patient voice”, but what does this really mean, and do we truly understand how best to incorporate this into drug development work? We reflect on these questions in relation to rare disease and look at what we can do to leverage available evidence to minimize the burden on rare disease communities.

At Sprout, we conceptualize the patient voice as insights provided directly by the patients orally, written or visually, or by a family member or caregiver when the patient is unable to communicate these insights themselves.

Listening to the patient voice is important from early in the drug development process. Strategy work should begin prior to Phase 2 to identify the concepts important to patients and the availability of clinical outcome assessment (COA) measures to assess those concepts in a valid and reliable way. In our rare disease work, we hear from advocacy organizations that patient communities can sometimes feel tired from multiple requests for them to fill in yet another questionnaire or take part in yet another research interview. To minimize burden on patient communities it is therefore crucial to always look to see if there are any opportunities for secondary data insights gathering before deciding that a new interview or survey study is needed.

A hugely valuable resource is the FDA-led Patient-Focused Drug Development (PFDD) series of public meetings, that the FDA has hosted to systematically obtain the patient perspective on the experience of specific diseases and the currently available treatments1. These publicly available meeting reports include testimonials from patients, caregivers and other stakeholders in the therapeutic area (e.g., clinicians, pharma, regulators) and are a rich source of insight into the patient voice in relation to symptoms, impacts and unmet needs in these patient communities. Social media research can also be a helpful approach to minimize burden on rare disease communities and can help capture the voice of those who may not often choose to participate in more formal research methods. Industry guidelines are available for ethical best practices for social media research approaches2.

Finally, it is important that patient-centered work is not kept within silos at the pharma companies who commission this work. Efforts to share reports cross-functionally ensures that insights from patients can be leveraged along the whole drug development pathway. For example, qualitative work from the early pipeline setting related to patient-centered outcomes can be designed to also inform beyond-the-pill solutions to support daily functioning, treatment adherence and emotional wellbeing in phase 3 and post-marketing settings.




  1. FDA-led Patient-Focused Drug Development (PFDD) Public Meetings (accessed 23 February 2021)
  2. British Healthcare Business Intelligence Association (BHBIA). Guidelines for Social Media Research. Available from: (accessed 23 February 2021)