Accelerating Patient Recruitment for a CAH Gene Therapy Trial
Case Studies
Biotech sponsor (CAH gene therapy)
Region
United States
Services
Patient Outreach, Data Management & Analytics
About
Challenge
Response
Targeting
Results
01 — About
About the project
MBL Health partnered with a U.S. biotech sponsor to accelerate patient recruitment for a Congenital Adrenal Hyperplasia (CAH) gene therapy clinical trial.
We developed a scalable patient recruitment ecosystem designed to identify and engage eligible patients, streamlining recruitment and improving study site efficiency.
02 — Challenge
Recruiting for a rare disease gene therapy is uniquely complex.
The sponsor required a scalable recruitment strategy capable of expanding patient reach while delivering high-quality, trial-ready referrals.
01
Limited and geographically dispersed patient population
02
Low awareness of gene therapy clincal trial options
We launched omnichannel digital outreach campaigns that identified and engaged patients living with CAH, creating a scalable recruitment pipeline that supported patient enrolment into the CAH gene therapy trial
01
IRB-approved Patient Education
Developed IRB-approved educational materials that translated complex CAH and gene therapy concepts into clear, accessible language for patients and caregivers.
02
Digital Patient Experience
Designed and launched a study website with integrated online pre-screening, providing a seamless patient journey from education to study referral.
03
Omnichannel Outreach
Launched targeted campaigns across search, social media and patient communities to identify and engage individuals living with CAH.
04 — Precision Patient Recruitment
Finding eligible patients beyond traditional investigator networks
Our recruitment strategy combined real-time campaign optimisation, patient behaviour insights and recruitment analytics to continuously improve referral quality throughout the study.
Data-driven optimization
Continuously refined patient journeys, campaign performance and eligibility pathways using real-time recruitment data.
DupCheck+ Verification
Automatically detected duplicate registrations to improve recruitment quality and trial integrity.
JAVIS
Centralised patient data, recruitment analytics and referral workflows, providing sponsors with real-time operational visibility.
RESULTS AND IMPACT
Outcomes that moved the trial forward.
RESULTS AND IMPACT
430+
Patients recruited through digital campaigns
MBL Health's digital-first approach gave us a quality patient pipeline our traditional channels couldn't match — and at a pace our timeline required.
Clinical Operations Lead
Biotech sponsor, CAH program
Next chapter
Bringyour trialforward.
We help biotech and pharma teams build digital-first recruitment ecosystems for rare disease and gene therapy programs.