Rare Disease Research
Rare disease market research: insights from patients, caregivers & physicians
testing healthare sub heading
MedPanel is a specialist in rare disease market research — connecting life science companies, CROs, and healthcare consultancies with verified rare disease patients, caregivers, and the physicians who treat them. We turn hard-to-reach populations into high-quality research insights.
10+ years
Peer-reviewed
Global reach
What makes rare disease research different
Rare diseases present unique research challenges that standard market research approaches aren’t built to handle. Patient populations are small and geographically scattered. Diagnoses are often delayed by years. Standard panel providers simply don’t have access to these respondents.
MedPanel was built for exactly this problem. Our physician network, advocacy partnerships, and verified patient community give clients access to respondents that are genuinely difficult to reach — with the diagnosis verification and compliance controls that rare disease research demands.
The challenge
Small, dispersed patient populations with complex diagnostic histories — standard panels can't reach them.
The MedPanel approach
Physician partnerships, advocacy outreach, and a verified respondent community built specifically for rare conditions.
Useful external resources
Research capabilities
We support the full range of market research methodologies across rare disease populations:
Qualitative research
In-depth interviews and focus groups with patients, caregivers, and treating physicians — across rare and ultra-rare conditions.
Quantitative surveys
Large-scale patient surveys with statistically meaningful samples, even for conditions with small global populations.
Patient-reported outcomes
Real-world data on disease burden, treatment satisfaction, quality of life, and unmet needs — directly from patients.
Physician & KOL research
Access to specialists, rare disease experts, and key opinion leaders through our verified expert network.
Longitudinal panels
Track patient and physician attitudes over time with a consistent, verified respondent panel for your condition area.
Advisory boards
Convene patient or physician advisory boards with verified, engaged participants who bring genuine rare disease experience.
Conditions we have researched
Our rare disease research experience spans metabolic disorders, neurological conditions, hematology, autoimmune diseases, and oncology. Recent published and client studies include:
Fabry disease
Hemophilia A & B
Sickle cell disease
Hereditary angioedema
Epidermolysis bullosa
Tuberous sclerosis complex
Alpha-mannosidosis
Ocular myasthenia gravis
Schizophrenia
Idiopathic pulmonary fibrosis
Polycythemia vera
Parkinson's disease
Published research
MedPanel’s work has contributed to peer-reviewed publications in rare disease and specialty medicine. Selected publications:
-
2025
Disease burden and psychological well-being in patients with ocular myasthenia gravis: insights from a US patient panel
Ocular myasthenia gravis -
2024
Patient-reported experience with Fabry disease in the real-world setting: results from a double-blind, cross-sectional survey of 280 respondents
Fabry disease -
2020
US haematologist survey on disease management of haemophilia A patients treated with emicizumab
Haemophilia A -
2020
Patient-reported symptom worsening and disease monitoring in Fabry disease: results from a cross-sectional survey
Fabry disease -
2018
Real-world practice patterns for prevention and management of adverse events with pirfenidone in idiopathic pulmonary fibrosis
Idiopathic pulmonary fibrosis
Frequently asked questions
Rare disease market research involves gathering insights from patients, caregivers, and physicians about conditions affecting fewer than 200,000 people in the US (or roughly 1 in 2,000 in Europe). It covers topics like disease burden, treatment experience, unmet needs, and quality of life — and requires specialist recruitment and verification methods that standard market research providers don’t have.
Our clients include pharmaceutical and biotech companies, contract research organizations (CROs), healthcare consultancies, patient advocacy groups, and academic medical centers. Studies range from early drug development to post-launch market assessment.
We work through three channels: our physician partner network (who refer verified patients), relationships with rare disease advocacy organizations, and our own community of opt-in rare disease patient respondents. All participants are verified before inclusion in any study.
Yes. Many rare disease studies benefit from triangulating patient-reported experience with physician treatment patterns. We run parallel patient and physician tracks within the same project, ensuring consistency and enabling direct comparison of perspectives.
Yes, and we’re transparent about feasibility. For ultra-rare conditions (fewer than 10,000 patients globally), we conduct a feasibility review before engagement and will tell you honestly what sample sizes are achievable and on what timeline.