Rare Diseases

PPD is committed to advancing the development of medicines for adults and children with rare diseases and we have developed solutions to reach, recruit and retain small, globally dispersed patient populations. By leveraging our medical, operational, regulatory and real-world expertise, we design and operationalize studies with the customized approach that each rare disease requires. We successfully execute trials with organizations of all sizes. In the past five years, we have conducted more rare disease studies than any other contract research organization (CRO).

Rare Disease Framework

We deploy a four-part framework to support and conduct rare disease studies that ensures the application of best practices in study development. The key elements of this framework include:

  • Innovative and executable development plans – Deploying innovative, patient-centric development plans to navigate challenges and support clients in the development of clinical programs
  • Rigorous feasibility and study planning – Identifying the right sites and finding and enrolling patients crucial to a study’s success
  • Study execution – Providing unmatched patient focus to retain and consistently engage patients and sites
  • Proactive post-study planning – Leveraging key partnerships to plan for future development

Rare Disease and Pediatric Center of Excellence

The Rare Disease and Pediatric Center of Excellence team provides tailored thought leadership and innovation in the design and execution of rare disease trials, as well as aligned oversight of program delivery and clear escalation paths for operational teams. The center of excellence is the most comprehensive offered by any CRO and provides strategic insights that can inform and optimize clinical and regulatory strategy development. Additionally, the center of excellence facilitates access to a broad array of key stakeholders, including medical experts, patients and patient advocacy groups.

Learn more about PPD’s Rare Disease and Pediatric Center of Excellence

Extensive Cross-therapeutic Expertise

We have extensive global experience working with drug developers to design and execute successful clinical trials focused on rare disease indications across a broad spectrum of therapeutic areas. In the past five years, we have conducted more than 290 studies involving more than 65,000 patients in more than 65 countries.

Pediatric Investigator Network

PPD’s Pediatric Investigator Network (PIN) is a network of 14 pediatric centers of excellence established to accelerate and optimize the development of therapies specifically for pediatric populations. The PIN allows PPD to:

  • Provide clients access to disease experts across all pediatric therapeutic areas
  • Facilitate rapid feasibility and protocol input to facilitate development of clinical trials that are practical and operationally sound in the pediatric population
  • Provide immediate access to research-experienced sites to serve as key centers to support evaluation of new pediatric drugs
  • Assist the drug development industry in the goal to protect the child by the trial, not from the trial

View our rare disease resources

Patient Advocacy Groups

PPD’s rare disease team cares deeply about the rare disease patients in our clinical trials and strives to better understand their experiences. By collaborating with patient advocacy groups, we are able to incorporate patients’ voices into our study designs and gain valuable insights about the patient journey as we strive to enhance the clinical trial experience for patients and their caregivers.

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