Case Studies
/ CAH Gene Therapy Trial
Patient Recruitment
Gene Therapy
Rare Disease
U.S. Market
Accelerating patient recruitment for a CAH gene therapy trial in the U.S.
Case Studies
Biotech sponsor (CAH gene therapy)
Region
United States
Services
Digital recruitment, Pre-screening,
Multi-channel media
Multi-channel media
About
Challenge
Response
Targeting
Results
01 — About
About the project
MBL Health partnered with a biotech sponsor to support patient recruitment for a gene therapy clinical trial in Congenital Adrenal Hyperplasia (CAH) across the United States.
The project focused on building a scalable digital ecosystem to identify, engage, and pre-screen patients — enabling direct referral to study sites at the pace clinical timelines demand.
02 — Challenge
Recruiting for a rare disease gene therapy is uniquely complex.
The sponsor required a solution to expand reach while maintaining high-quality, trial-ready patient referrals.
01
Limited and geographically dispersed patient population
02
Low awareness of gene therapy options
03
Strict eligibility criteria leading to high drop-off during screening
04
Heavy reliance on traditional site-led recruitment
03 — Response
A full-stack digital recruitment strategy.
We designed an integrated ecosystem — content, conversion, media, and engagement — calibrated for a rare disease audience.
01
IRB-approved patient content
Simplified complex gene therapy messaging into accessible language patients and caregivers could understand.
02
High-conversion study site
Designed and launched a study website with integrated pre-screening, optimized for clarity and completion.
03
Continuous optimization
Refined eligibility questions, reduced friction, and iterated on CTAs based on live performance data.
01
IRB-approved patient content
Simplified complex gene therapy messaging into accessible language patients and caregivers could understand.
02
High-conversion study site
Designed and launched a study website with integrated pre-screening, optimized for clarity and completion.
03
Continuous optimization
Refined eligibility questions, reduced friction, and iterated on CTAs based on live performance data.
04 — Precision Targeting
Reaching self-referring patients beyond traditional channels.
Our targeting strategy was built around patient intent, not investigator geography — and refined every week against performance data.
Search-first
Targeted high-intent search queries related to CAH and treatment options.
Adaptive audiences
Continuously refined audience targeting using campaign performance data.
Channel reallocation
Shifted investment toward top-performing channels as signal strengthened.
Patient language
Adapted messaging to align with how patients actually describe their condition.
RESULTS AND IMPACT
Outcomes that moved the trial forward.
RESULTS AND IMPACT
435+
Patients registered through digital campaigns
Pipeline
Consistent monthly
qualified patient flow
Reach
Beyond investigator
networks, nationwide
Referrals
Consistent monthly
supporting recruitment goals
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
Bring your
trial forward.
We help biotech and pharma teams build digital-first recruitment ecosystems for rare disease and gene therapy programs.