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Rare Diseases Require Rare Recruiting Methods

Research among patients is vital to closing the information asymmetry between drug producers and end users.  For many diseases, finding patients willing to share their experiences is no issue at all.  However, conducting research among rare disease patients is a unique challenge due to, you guessed it, the rarity of their disease.  In a recent study, we recruited n=75 hereditary angioedema (HAE) patients for a quantitative survey to understand changes in preferences and prescribing behavior based on two recent product launches.  The estimated prevalence of HAE in the US is 1 in 50,000 individuals1 and we used a multimodal approach to recruit a robust sample within our client’s budget.  Here are a few novel methods to access rare disease patients like those with HAE.

Social media – People of all types use the internet to connect with one another.  For the HAE research, we found youtubers, bloggers, and Facebook groups discussing the patient journey.

Referrals – If the research includes a physician component, one may offer interested physicians links to the screening survey to share with their patients.  Additionally, patients may refer other patients.  Because HAE is a genetic disorder, most sufferers know one or more family members with the disease.  Incentives can be provided to the referrer.

Advocacy groups – Non-profit patient advocacy organizations serve many rare disease populations and are often a great source for engaged patients.  In many cases, these organizations will share information about a study in their newsletter.  One must be mindful of representativeness when recruiting through patient groups.  In a past segmentation study, we found that patients involved with their advocacy group had different attitudes and perceptions than patients found elsewhere.  Both patient types are important parts of the market and should be included.

We offer larger honoraria for rare disease studies than for general population studies and validation is necessary to ensure high quality responses.  In the HAE research, we separated the screener and main survey and validated patients’ responses with a phone conversation.  While on the phone, we were able to ask for referrals and gauge interest in other types of research, such as telephone interviews.

While accessing rare disease patients can be difficult, we find that participants are knowledgeable, engaged, and want to share their story.  The methods described in this article help us achieve readable sample sizes among cohorts not reachable with online sample providers.

1Ghazi, A., & Grant, J. A. (2013, May 3). Hereditary angioedema: Epidemiology, management, and role of icatibant. Retrieved February 11, 2019, from