Rare Diseases
Partner with our experienced team to advance your rare disease study as we deliver personalized services designed around the specific needs of patients, caregivers, and sites.
Rare disease studies, for rare patients
Rare disease studies demand a high level of attention. From designing a protocol with the appropriate inclusion/exclusion criteria to finding eligible patients and securing experienced sites, we work to address your trial’s unique challenges.
As a nimble CRO, we’re built to support emerging and mid-sized drug developers as we extend the expertise of their teams with our medical, scientific, and clinical specialists. We share our experience navigating complexity in rare disease trials and collaborate to design custom solutions that guide development and improve the potential for success.
Rare disease experience that matters
We bring expertise that matters for rare disease research. Our track record of successfully advancing these complex studies reflects our deep medical, scientific, and operational expertise. Together, we’ve advanced 210+ rare disease studies with nearly 19,500 patients across 2,935+ sites.
Our experience in rare disease drug development spans many rare types of cancers, including multiple myeloma, hepatocellular carcinoma, leukemia, lymphoma, and skin cancer. Beyond oncology, we have also supported studies in immunological rare diseases, rare genetic disorders, rare blood disorders, and rare neurological diseases.
Partnerships through flexibility
Rare Disease trials are fundamentally different. You need a flexible approach backed by expertise to run them effectively. We bring a personalized approach and partner with our clients to deliver customized solutions for rare disease studies.
Advance your rare disease study with dedicated leadership
We take ownership of your trial to deliver the best outcomes for you and your patients. Our Rare Disease team of operational, clinical, and medical specialists is led by Jonathan Kornstein, Vice President, Rare Disease and Pediatrics. Jonathan oversees clinical trial services and applies his extensive experience supporting rare disease and pediatric clinical trials. Jonathan also delivers strategic consultation and direction to our clinical management, commercial development, and business development teams, helping facilitate success in clinical trials.
Our mission to advance life-changing therapies
“We’re driven to advance rare disease studies while reducing patient and caregiver burden. Working alongside drug development sponsors, we are working to accelerate these complex studies and help address urgent unmet medical needs for people living with a rare disease.”
Jonathan Kornstein, Vice President, Rare Disease and Pediatrics
Shape your rare disease regulatory strategy
As regulatory agencies and legislative acts have helped encourage and incentivize rare disease drug development, sponsors need to understand their regulatory pathway—from the start—to make informed decisions.
Our regulatory experts can shape your regulatory strategy, apply for orphan drug designations in the US and EU, and prepare for and attend regulatory meetings. It’s just one way we’re here to support every aspect of your rare disease study.
Find and retain patients for your rare disease study
By their very nature, rare disease studies create a high demand for specific patients in sparse populations. To help find patients and communicate the value of your study, we leverage our technologies and tap into our established relationships with key opinion leaders and patient advocacy groups.
Once enrolled, every patient counts. We focus on reducing patient burden and supporting retention by incorporating patient-centric practices that reflect the needs of people living with rare diseases and their caregivers. Our team also examines the patient journey, especially the journey of the pediatric patient, to balance the ease of participation with the requirement to collect high-quality data and inform your endpoints.
Apply our global reach and expertise
Many rare disease studies need a global reach to maximize patient recruitment. We bring a distinct global reach that empowers us to identify sites and reach rare patients—no matter where they are in the world.
However, finding sites is not the complete answer. Prospective trial sites may lack specific rare disease experience. Our clinical and medical specialists share their expertise to help engage and educate site staff so you can work with trained trial sites. Expert site guidance is extremely critical when working with a cell or gene therapy to handle study execution and logistics effectively.
Throughout this process, we provide “high-touch” site management—without creating undue burden—as we apply our best practices and deliver customized site support. As a result, you’ll gain access to the best-suited sites, clinicians, and patients to meet your trial’s unique needs.
Shared knowledge is power
We are fully invested in the outcomes of your trials, providing the resources and expertise when and how you need them and sharing therapeutic area expertise and knowledge to help you make the best decisions.
Speak to a global expert
Ready for the next step in your drug development journey? Learn more about how our personalized solutions can help you enable better healthcare outcomes.
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