A doctor speaking with her young patient and her parents

Reaching Rare Disease and Pediatric Patients

“Over 300 million people are affected worldwide by rare diseases, and half of those are children.

In a population with underserved and unmet needs, developing targeted therapies for these conditions is not only essential, but also urgent. That’s why PPD has been committed to serving these patients for so long. We established a Rare Disease and Pediatric Center of Excellence well before rare diseases became a focal point for the industry. We continue to maintain our deep commitment to advancing rare disease research across the globe.”


PPD seeks to understand rare and low-prevalence indications and then determine how patients can be accessed and enrolled in trials. When studying unique patient populations, forethought in study design and precise data generation strategies are essential.

PPD also maintains relationships with world-class academic medical centers for pediatrics through our Pediatric Investigator Network.

ppd’s EXPERIENCE in the past five years:

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Rare disease studies across 70+ countries, enrolling 110,000+ patients in those trials

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Pediatric studies across 95+ countries, enrolling 100,000+ patients in those trials

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Therapeutic areas across which PPD has executed rare disease studies, including oncology, immunology and neurology


Centers of excellence in PPD’s Pediatric Investigator Network, established to accelerate and optimize the development of therapies specifically for pediatric populations

a patient in physical therapy

The Care Ecosystem

The challenges of developing rare disease therapies are numerous and include the need to engage research-naïve sites and access small, widely dispersed patient populations.

We field direct input from patients, providers and caregivers to understand the patient burden of a disease and determine feasible therapeutic regimens. This early assessment of the patient’s care environment guides study and protocol design, while also positioning your clinical research program as a worthy care option – not as an obstacle along the way to a promising therapy.

We work with rare disease research organizations and advocacy groups while also connecting directly with physicians to identify patients.


Our Rare Disease and Pediatric Center of Excellence team provides tailored thought leadership and innovation in the design and execution of trials, as well as aligned oversight of program delivery.

Our team of more than 20 rare disease experts from across the organization brings a wealth of experience across all therapeutic areas to provide strategic insights that inform and optimize clinical and regulatory strategy development.

Rare Disease Therapeutic Expertise

Find out how we can help you reach extraordinary rare disease patients.

Pediatric Therapeutic Expertise

Explore our pediatric drug development experience and expertise.