What Matters Most to People Impacted by Rare Disease: An Inspire-FDA Collaboration

By Sara Ray
What happened when we compared research strategies used to obtain patient insights? We discovered that using multiple strategies is key to gaining usable understandings.

In a recent collaboration between Inspire and the FDA, we compared and contrasted patient and caregiver generated online communication with the results of one of FDA’s Voice of the Patient meetings.

The Data Sets

Inspire’s membership includes over 350,000 patients and caregivers living and coping with rare diseases. In the last three years, there has been 203% growth in the rare disease community on Inspire. When looking at Inspire’s membership population of rare disease, we find that in some cases, Inspire’s membership represents over half the US population living with that condition.

Audience (rare conditions) Members in U.S. % of U.S. population
Ehlers-Danlos 41,022 59%
Sarcoidosis 30,482 51%
Scleroderma 28,588 38%
Pancreatic Neuroendocrine Tumor 2,885 36%
Von-Hippel Lindau 2,069 35%
Pulmonary arterial hypertension 5,279 33%
Pulmonary Fibrosis 17,154 29%
Amyotrophic lateral sclerosis (ALS) 3,923 24%
Wilson Disease 2,323 23%
Leukodystrophy 2,286 17%
Neurofibromatosis 16,290 16%
Nephrotic Syndrome and FSGS 7,464 15%
Multiple myeloma 4,178 13%
Tuberous Sclerosis 3,817 12%
Glioblastoma 377 12%


Patients come to Inspire seeking support and asking for information from other patients who are having the same experiences. They write to each other, conversing in unstructured organic ways, driven by their desire to help each other and to find guidance on their disease journey.  Their posts are snapshots of their lives in real-time, describing in rich color, the details of what is happening to them at particular moments of their journey, discussing life experiences as they happen. The focus of their communications are not necessarily the specific rare disease but what is happening to them; therefore they may discuss other comorbidities that impact living.

FDA Voice of the Patient

In contrast, FDA’s VOP are one time events: meetings where patients came to the FDA to describe their life with rare disease.

FDA’s Voice of the Patient (VOP) initiative occurred between 2012 and 2017. Under the fifth authorization of the PDUFA, a series of 24 meetings took place during those five years to try to better understand patients’ experience of chronic, symptomatic conditions that impact daily living. FDA announced the initiative in the Federal Register, put in place a short comment period and held their first meeting to decide on the list of diseases that would be covered on October 10, 2012. Organizations that monitor the Federal Register as well as industry were among the attendees. A second meeting helped to develop the structure of each meeting, the result being a mix of prepared statements followed by a question-answer period in which attendees would provide their experiences with their disease.

FDA has made available detailed reports and transcripts of each of the meetings. One such meeting transcript, focused on a fatal rare disease, was the basis of our collaborative comparison.

Inspire-FDA Collaboration: The Methodology

To learn how social media conversations between members compared with the insights and data obtained in an FDA VOP, Inspire used a novel strategy, combining natural language processing (NLP) in an iterative process with hands-on curation and sociolinguistic analysis. From over 800,000 posts that were first obtained by NLP, the iterative process sorted and gleaned down to almost 2000 posts written by patients with the fatal rare disease covered during the FDA VOP. Sociolinguistic analysis compared and contrasted the two data sets to  identify similarities and differences.

This research shows how social media posts, using Inspire’s novel research strategies, can enhance the research obtained from patient advocates in question-guided discussions. It also illuminates the wealth of information that can be learned by combining different research strategies, like FDA VOP and Inspire’s novel methodologies, especially in the rare disease space.

Inspire hosted a webinar on November 8 which described this research.  If you could not attend the webinar explaining our research or would like to learn more about using several strategies to obtain patient insights, here is your chance to see the recording. Download, “What Matters Most to People Impacted by Rare Disease: An Inspire-FDA Collaboration”

Download our webinar “What Matters Most to People Impacted by Rare Disease: An Inspire-FDA Collaboration”

Download Webinar
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