Don’t let limited patient pools, fragmented referral pathways, diagnostic delays, and inconsistent natural history data keep your therapy from the people who need it.
Partner with Caidya for a patient-first approach, tailored planning, flexible execution, and deep therapeutic and operational expertise that keep your rare disease clinical research moving at the speed of science.
Cookie-cutter approaches won’t cut it in rare disease clinical research. We collaborate with you to design custom solutions that:
We find needles in haystacks with targeted clinical trial feasibility, refined country and site selection, and patient pathway identification built for ultra-small populations.
Let us make your study easier on patients and caregivers with visit-schedule optimization, decentralized components, and advocacy-informed planning.
Our medical, operational, and data experts understand how to handle phenotype variability, evolving clinical trial regulatory requirements, and the absence of validated endpoints in rare disease trials.
Our proactive oversight and customized site training are tailored to conditions where prior experience is limited.
From detecting early efficacy signals and powering protocols for meaningful comparisons to grounding trial design in the patient journey, our rare disease approach is designed to make a difference.
Our rare disease subject matter experts (SMEs) contribute early and continuously to project design, endpoint evaluation, risk mapping, and operational planning. With demonstrated success in highly varied environments spanning neuromuscular, metabolic, genetic, ophthalmic, pediatric, immune, and many other rare indications, our SMEs provide support specifically aligned to address rare disease development complexities.
Our Rare Disease team of operational, clinical, and medical specialists is led by Jonathan Kornstein, Vice President, Rare Disease and Pediatrics.
Vice President, Rare Disease and Pediatrics
Jonathan Kornstein leads our team of operational, clinical, and medical specialists. Jonathan has 30 years of industry experience and oversees clinical trial services, applying his experience supporting rare disease and pediatric clinical trials.
Jonathan Kornstein - Vice President, Rare Disease and Pediatrics
We’re personally invested in your rare disease program and the patients you want to help.
As your experienced, flexible partner, we’ll:
We leverage precise feasibility tools, global site intelligence, and SME-driven protocol evaluation to develop data- and pathway-driven feasibility models that reduce delays tied to underpowered enrollment assumptions or misaligned country strategies.
Our cross-functional oversight is critical for studies with limited natural history, heterogeneous phenotypes, or evolving regulatory precedent.
We simplify participation, reduce care fragmentation, and give sites tools to elevate consistency and retention in extremely small patient populations.
With critical medical, scientific, and operational expertise, we’ll
Enrollment is constrained by delayed diagnosis, fragmented care, small patient pools, and limited specialist concentration. Caidya improves predictability by using pathway-based feasibility, referral-network mapping, targeted country selection, historical site performance insights, and advocacy-aligned engagement plans. By leveraging its geographic footprint, Caidya can extend study enrollment into underserved regions and reach untapped patient populations.
Many sites lack prior exposure to rare disease indications, particularly in regions where patients are located, but formal clinical trial experience is limited. Caidya mitigates this risk through customized site education, operational readiness assessments, enhanced monitoring, and ongoing SME-backed oversight, enabling less-experienced centers to execute studies consistently and enroll patients without increasing variability.
Limited natural history often leads to unclear endpoints or inappropriate assessments. Caidya’s medical and data SMEs evaluate endpoint feasibility, refine protocol assumptions, and align expectations with available evidence to improve clarity and reduce redesign risk.
Global dispersion complicates site placement and travel expectations. Caidya’s feasibility models incorporate country-level incidence, regulatory timelines, referral networks, and site performance history to create a realistic activation plan that avoids geographic misalignment.
Caidya takes a patient-first approach to rare disease studies, starting with a deep understanding of the patient pathway and designing trials around the patient rather than the data to be collected. High visit frequency, long travel distances, and complex procedures make participation difficult. Caidya simplifies engagement by optimizing visit schedules, integrating decentralized components where appropriate, and developing communication and support plans that respond to caregiver needs.
Rare disease SMEs provide early risk assessment, guide endpoint strategy, evaluate operational constraints, and advise on data quality and safety oversight. Their involvement reduces ambiguity and helps align the protocol with real-world clinical and operational conditions.
Phenotypic heterogeneity (the existence of variation in observable characteristics among genetically identical individuals or cells) can complicate assessments and introduce noise. Caidya addresses this through standardized site training, proactive monitoring, centralized data review, and endpoint coaching from medical and biometrics SMEs.
Retention is critical because each participant meaningfully affects statistical power. Caidya reduces drop-off through burden-mitigation planning, caregiver communication, site engagement tools, and clear expectation setting early in the study.
Regulatory precedent may be limited in first-in-population or novel mechanism studies. Caidya’s SMEs help align design assumptions with available guidance, evaluate precedents from adjacent indications, and ensure data collection supports future discussions.
Traditional feasibility often fails in rare disease because it relies on broad epidemiology rather than real referral patterns. Caidya applies a pathway-driven model that evaluates diagnostic steps, specialist flow, regional access constraints, and historical performance to produce more accurate enrollment projections.