Case study · Recruitment & patient survey
Hearing from a rare-disease population: treatment preferences in PH1
MedPanel collaborated on a peer-reviewed web survey that reached U.S. adults living with primary hyperoxaluria type 1 to learn what they value most in treatment.
Primary hyperoxaluria type 1 (PH1) is an ultra-rare genetic disorder — with a genetic prevalence of roughly 1 in 150,000 births — that causes excess oxalate to build up and damage the kidneys. Patients have few treatment options and carry a heavy administration and travel burden. This real-world study set out to understand which treatment attributes matter most to them. The hardest part of a study like this is simply finding and enrolling enough diagnosed adults, which is where MedPanel’s recruitment collaboration came in.
Understanding patients who are hard to find
Because PH1 is so rare, real-world evidence about how patients actually experience treatment has been scarce. Current therapies can mean frequent trips to a medical facility and significant time away from work, school, and family — yet little had been documented about how patients weigh attributes like dosing frequency, who administers the injection, and whether it can be done at home.
Generating that evidence first required reaching a dispersed, low-incidence population of diagnosed adults across the country and enrolling them in a survey they could complete on their own devices. Recruiting that audience is precisely where MedPanel collaborated on the study.
Among adults living with PH1, which treatment attributes — frequency, location, who administers, complexity, and time — matter most when choosing therapy?
What the research set out to learn
Five treatment attributes, plus the real challenges of day-to-day administration.
Understand preferences around frequency and place of administration — how often treatment is needed, and where it happens.
Explore who administers treatment and regimen complexity — self vs. healthcare provider, and how simple the regimen feels.
Capture the time required for treatment and the real-world travel burden it creates.
Characterize the specific challenges patients face when administering current PH1 treatments.
Recruit, survey, analyze
A virtual, cross-sectional survey design built for a rare, dispersed population.
Recruitment
U.S. adults (18+) with a confirmed PH1 diagnosis were recruited through provider panels and community collaborations, screened against clear eligibility criteria. MedPanel collaborated with Stratevi to reach this rare population.
Web survey
Participants completed a 20–25 minute cross-sectional questionnaire on their own devices via the inVibe Labs platform, fielded October–December 2023, covering demographics, treatment history, and preferences.
Analysis
Descriptive statistics summarized participant and treatment characteristics, the travel and time burden of care, and preferences across each treatment attribute and combination.
Convenience and autonomy came first
Patients consistently valued treatments that fit their lives.
preferred self-injection
Given the choice, most participants preferred self-injection over having a healthcare provider administer their treatment — underscoring the value placed on autonomy.
chose home, even if more frequent
Two-thirds preferred monthly injections at home over quarterly injections at a medical facility — the convenience of home outweighed the desire for less frequent dosing.
ranked frequency most important
How often treatment is needed was the single most important factor when choosing a therapy, ahead of self-administration, time, location, and regimen complexity.
per year for facility-based care
Most participants (87%) made at least one trip to a facility for treatment — on average 6.4 trips a year at about 4.1 hours each, a burden patients were keen to reduce.
Reaching a rare population so their voice could be heard
In rare disease, the participants simply aren’t easy to find. By collaborating on recruitment, MedPanel helped the team reach and enroll diagnosed PH1 adults from across the country — turning a hard-to-study population into real-world evidence.
The resulting dataset gave a clear picture of what patients value — convenience, autonomy, and treatment that fits a busy, mobile life — and was published as an open-access, peer-reviewed paper. It’s a strong example of how MedPanel’s recruitment reach supports rigorous rare-disease research.
Rare population reached
Diagnosed PH1 adults recruited and enrolled despite a very low incidence.
Real-world preference signal
Clear evidence that home-based, self-administered care drives patient choice.
Peer-reviewed outcome
Findings published open access, adding to the rare-disease evidence base.
Published, peer-reviewed, open access
The study MedPanel helped make possible — available in full.
Peer-reviewed publication · 2025
Treatment Preferences Among Individuals with Primary Hyperoxaluria Type 1 (PH1): A Real-World Study
A cross-sectional web survey of U.S. adults living with PH1, examining how patients weigh treatment attributes such as dosing frequency, who administers the injection, location, regimen complexity, and time. MedPanel recruited the patient participants for this study.
Read the full study
MedPanel provided patient recruitment for this peer-reviewed study. Figures shown are drawn from the published article.