Rare Diseases
Activate the full potential of your scientific breakthroughs in rare diseases with clinical trials designed around the specific needs of your patients, their caregivers, and medical providers.
Successful development, delivery and clinical approval for rare disease treatments
Rare diseases affect over 300 million people worldwide, among over 6000 disorders. Performing clinical trials for rare diseases presents a unique set of challenges due to the small size of study populations, which can be further restricted by strict inclusion or exclusion criteria.
In addition, due to the scarcity of these diseases there are gaps in understanding of the natural history of the disease and its progression, making it more challenging to develop the right endpoints. Study design then, is critical to the successful development, delivery and clinical approval for rare disease treatments.
Our team has broad medical expertise spanning a wide range of rare diseases, including what type of information should be incorporated from rare disease registries to support the design of clinical trials. The patients with rare diseases are like gold – finding them, connecting them with the sites (if not already at the site), ensuring enrollment into the study and keeping them in the study to the end are critical for the success of your trial.
We share the knowledge of how to get this done through experience from the design of the protocol to the strategy of protocol implementation. We work closely with you to communicate the importance of the therapy and the study to the site personnel and the patients, and give you access to the best-suited trial sites, clinicians and patients to serve their unique needs.
We work to bring your science to life
With you across every step of the way from strategy and design to identifying suitable candidates and providing the training needed to research personnel, we work to bring your science to life.
Our dedicated global experts work with you to streamline the clinical trial process and help you hit your critical milestones. We have refined our processes and services to accommodate clinical trials involving small patient numbers and support the unique medical, scientific, clinical and commercial challenges of rare diseases.
We are passionate about advancing treatments for rare disease, and have developed expertise in three key areas, having run nearly 200 studies in over 22,000 patients:
Regulatory
Our experts will help shape your regulatory strategy, apply for orphan drug designations in the US and Europe, prepare for and attend regulatory meetings, and advise on establishing relations with important patient advocacy groups.
Patient Finding
Diverse and isolated patient groups are a core challenge for rare disease trials. We have an ecosystem of partnerships and various technologies to help identify patients combined with the understanding to effectively communicate the value of studies to recruit and retain trial participants.
Site Education and Training
Your prospective trial sites may not have any research experience, so we help engage, train and provide personnel to deliver studies. Having worked with nearly 2500 sites in rare disease trials, we use best practices to approach and support sites, patients and families, and clinical specialists to get and keep your trial running.
Our experience in rare diseases spans a wide variety of indications, including:
Rare disease experience
- Multiple myeloma
- Hepatocellular carcinoma
- Leukaemia (AML, CLL)
- Lymphoma (B cell, SLL, mantle cell, DLBCL)
- Mycosis fungoides
- Lymphoproliferative disorder
- Richter’s transformation
- Crigler-Najjar syndrome
- Myelodysplastic syndrome
- Graft vs. host disease
- Haemophilia
- Enterovirus
- Bird flu
- Enterovirus EV71
- Fibrinogen deficiency
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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04/01/2024