When rare disease trials stall, Leapcure delivers. On an ultra-rare Phase 2 rescue, we brought in 8 of the final 10 participants.

Leapcure has recruited across multiple rare and ultra-rare indications, including pediatric, reaching small, dispersed communities through trusted advocacy and a dedicated Patient Success team. On an ultra-rare Phase 2 rescue we delivered 8 of the final 10 participants.

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530+
Patients pre-screened
on an ultra-rare Phase 2 rescue
~70%
Avg enrollment contribution
on Phase 2 rare programs
205
Interested families
in one ultra-rare pediatric study
40+
Advocacy partnerships
program standard

Understanding Rare Disease as a Clinical Trial Area

In rare disease, the eligible pool is so small that a recruiter either carries most of the enrollment or the study stalls. Populations are tiny and dispersed, many patients are undiagnosed, and in pediatric conditions a protective parent is the decision-maker, not the patient. These are not the conditions of generic, volume-driven recruitment. They are the conditions of small, tight-knit communities reached through the organizations they already trust.

A disease is defined as rare in the US when it affects fewer than 200,000 people (Orphan Drug Act, 1983). There are an estimated 7,000 or more known rare diseases, affecting roughly 30 million people in the US, about 1 in 10 Americans (NIH/GARD; NORD). About half of those affected are children, and the large majority of rare diseases still have no FDA-approved treatment, which is what drives the trial pipeline (NIH; commonly cited literature).

Leapcure reaches these tiny, dispersed communities through the advocacy organizations patients already trust and a dedicated Patient Success team, and we contribute the majority of enrollment. We have delivered across the rarest populations, from an ultra-rare Phase 2 rescue where we brought in 8 of the final 10 participants to ultra-rare pediatric programs. As the umbrella across our rare-disease work, this page routes sponsors with a specific indication to the deeper rare sub-pages: Rare Autoimmune Neuromuscular and Pediatric Rare Disease.

The Challenge

Rare disease trials are hard for reasons that compound: tiny and dispersed populations, communities reached only through trusted voices, complex and heterogeneous eligibility, and pools so small that a recruiter must carry the majority of enrollment.

Diagnosed populations are tiny and dispersed, many undiagnosed

Eligible patients are few, spread across many regions, and frequently undiagnosed, so reaching enough of them often means reaching them globally and helping diagnosed and at-risk patients find the trial at all.

On an ultra-rare pediatric program, 205 eligible or interested families were generated and 72 physicians engaged across 18 states. On an osteogenesis imperfecta program, a network of roughly 50 sites widened an extremely narrow pool beyond the US.

Patients are reached through trusted communities, not list buys

Small, tight-knit rare disease communities gather in the advocacy organizations they already trust. Cold digital and purchased lists do not reach them. Recruitment works when research reaches patients through the voices they already rely on.

30+ advocacy partners were mobilizable in two days on an ultra-rare Phase 2 rescue. On an ultra-rare pediatric program, 23 advocacy groups were engaged, driving roughly a 70% increase in awareness.

Eligibility is complex and heterogeneous

Rare conditions carry complex, heterogeneous eligibility, and patients vary widely in presentation and history. Generic campaigns surface patients who fail screening, spending scarce specialist-site time on people the protocol cannot use.

Real human conversations and a dedicated Patient Success team support every patient. On the ultra-rare Phase 2 rescue, 530+ patients were pre-screened and 220 assigned to sites.

In tiny pools, you carry the majority of enrollment or the study stalls

When the eligible pool is tiny, a recruiter either contributes most of the enrollment or the study does not finish. The question a sponsor should ask is how much of enrollment a partner actually carries, not how many referrals they generate.

Across rare programs, Leapcure's enrollment contribution averages about 60% on Phase 1, about 70% on Phase 2, and about 40% on Phase 3.

How Leapcure recruits across rare disease

We activate the trusted community for each indication, pre-screen and support every patient and family through real human conversations, and optimize in real time when the diagnosed pool proves too small.

1

Activate the trusted advocacy communities for each indication

We engage the advocacy organizations patients already trust for the specific indication, alongside precision digital and physician outreach mapped to your eligibility and geographies. These communities are mobilizable within two days, so research reaches patients through sources they rely on.

2

Pre-screen and support every patient and family

Each patient and family is engaged through real human conversations, not automated screening, supported by a dedicated Patient Success team before any site referral. In pediatric conditions that means working with the parent who makes the decision, so families arrive informed and prepared.

3

Optimize in real time when the pool is too small

When a diagnosed pool proves too narrow, we adjust in flight: widening to a global site network to reach patients beyond the US, or adjusting messaging to reach hereditary carriers. This in-flight optimization is how we keep enrollment moving in the rarest populations.

Rare Disease Results and Metrics

Ultra-rare Phase 2 rescue: Delivery

Brought in to rescue a stalled Phase 2 study, Leapcure delivered 8 of the final 10 participants in the closing months, preventing further delays.

Ultra-rare Phase 2 rescue: Reach and readiness

530+ patients pre-screened and 220 assigned to sites, with 30+ advocacy partners mobilizable in two days, supporting 10 active sites.

Ultra-rare pediatric program: Community reach

205 eligible or interested families generated through Leapcure campaigns for an ultra-rare pediatric condition (children ages 2 to 11), 31 in-depth conversations with parents, 72 physicians engaged across 18 states, and 23 advocacy groups engaged, driving roughly a 70% increase in awareness within that community.

Ultra-rare pediatric program: In-flight optimization

On an osteogenesis imperfecta program, a network of roughly 50 global sites widened an extremely narrow eligible pool beyond the US, and mid-campaign messaging was adjusted to reach hereditary carriers when the diagnosed pool proved too small.

Average enrollment contribution

Across rare programs, Leapcure contributes about 60% of enrollment on Phase 1, about 70% on Phase 2, and about 40% on Phase 3. In tiny populations, carrying the majority of enrollment is the difference between a study that finishes and one that stalls.

Four channels. Built for the rarest populations

Leapcure reaches small, dispersed rare disease communities through trusted advocacy, real human pre-screening, proven enrollment delivery, and answer-engine visibility delivered to your trial.

Channel 1: Advocacy communities patients trust

  • •We reach patients through the advocacy organizations they already trust, with the National Organization for Rare Disorders (NORD) as the cross-rare credibility anchor plus condition-specific anchors for each indication.
  • •Partnerships are active and co-developed, not list buys.
  • •40+ advocacy partnerships is the program standard.

Channel 2: Patient Success and human pre-screening

  • •Every patient and family is engaged through real human conversations, not automated screening, including those who do not qualify.
  • •A dedicated Patient Success team supports each patient and family from first contact through enrollment.
  • •This reduces site burden and screen fails on scarce, specialized sites.

Channel 3: We carry the majority of enrollment

  • •Across rare programs, our enrollment contribution averages about 60% on Phase 1, about 70% on Phase 2, and about 40% on Phase 3.
  • •When a study stalls, we deliver: on an ultra-rare Phase 2 rescue, we brought in 8 of the final 10 participants.
  • •In tiny populations, carrying the majority of enrollment is the difference between a study that finishes and one that stalls.

Channel 4: Answer-engine and AI-search visibility

  • •Patients increasingly ask ChatGPT, Perplexity, and Google's AI Overview which trials they may be eligible for. We make your trial's information genuinely retrievable for those engines.
  • •We do this with crawlable, server-rendered content, first-party data backed by sourced stats, a named reviewer, and a last-updated date, plus presence in trusted third-party sources such as advocacy orgs and ClinicalTrials.gov.
  • •This is not schema tricks or markup promises. Because AI engines cite trusted third-party sources more often than any single brand domain, our advocacy partnerships and ClinicalTrials.gov presence are the structural advantage we bring.

Built on the rare-disease communities patients trust

Leapcure's partnerships are active and co-developed, not list buys. 40+ advocacy partnerships is the program standard. We name only confirmed partners and confirm others before logos go live.

National Organization for Rare Disorders (NORD)

Cross-rare credibility anchor across the rare disease community

Condition-specific rare disease communities

Indication-specific advocacy organizations, confirmed before logos go live

Rare disease caregiver and family groups

Communities supporting caregivers and families, including parents of pediatric patients

Rare disease KOLs and community leaders

Trusted clinical and community voices who reach dispersed rare disease populations

Living with a rare disease, or caring for someone who is? A clinical trial may be an option to explore.

If you are living with a rare disease, or caring for a child or loved one who is, a clinical trial may be an option to explore alongside your existing care, never instead of it. We know these are rare, complex conditions, and we will never pressure you. Leapcure's team answers your questions honestly, explains what a trial involves, and supports you and your family step by step.

Explore trials in your rare disease community
Leapcure kangaroo mascot

Leapcure has delivered across the rarest populations, from an ultra-rare Phase 2 rescue where we brought in 8 of the final 10 participants to ultra-rare pediatric programs. We reach small, dispersed communities through the advocacy organizations patients already trust, support every patient and family with a dedicated Patient Success team, and carry the majority of enrollment. We can mobilize quickly. If you have a specific indication, we can point you to the right rare sub-page.

Talk to our team