kid with rare disease hugging dad

Rare Disease CRO Services

Alleviating the burdens of rare diseases

There are no U.S. Food and Drug Administration (FDA)-approved treatments for 95% of rare disorders, creating an immeasurable physical and mental toll on patients and their families. Whether performing clinical trial therapeutic research for adult or pediatric rare diseases, PPD recognizes the person behind the condition.

Patients are at the heart of our rare disease CRO services. By incorporating the experiences of trial participants, their caregivers and investigators, we enable a patient-centric approach for our clients’ transformational therapies and for positive change in the lives of people with rare diseases.

Rare disease development is a priority for many drug developers, but they face persistent challenges when trying to advance treatments for these often-complicated disorders. These barriers to rare disease treatment and orphan drug development include:

  • A lack of consensus on endpoints
  • Inexperienced, understaffed, or unavailable research sites
  • A shortage of regulatory precedents
  • Geographically dispersed patient populations
  • Finding and retaining enough patients to participate in a rare disease clinical trial

PPD enables clients to navigate these difficulties and overcome obstacles that arise in the clinical development, testing and regulatory approval of treatments for rare diseases.

Committed to rare disease research

Of all the rare diseases that have been identified globally, few have an approved treatment or therapy. Only five percent. This number inspires us to drive research forward and to develop new rare disease therapies that change lives. Through our Rare Disease and Pediatric Center of Excellence, wide-ranging study experience and specialized rare disease trial framework, we deliver innovative solutions to overcome the challenges associated with orphan drug development.

PPD rare disease clinical trial services

Patients and the drug developers working to fight rare disorders face a number of challenges — PPD, Thermo Fisher Scientific’s clinical research business, designs and conducts rare disease studies that solve for those challenges. Our focus is to decrease patient burden and help them and their caregivers navigate a complex health care system.

PPD’s patient services build on long-standing partnerships in the rare disease community to provide innovative solutions for increasing access to clinical trial participation and patient retention. These services include:

  • Booking and coordinating patient transportation to and from study sites, providing convenience and travel expense verification for patients
  • Offering flexible patient reimbursement options in over 150 currencies
  • Connecting patients to options for home health care, including remote staff partnerships

Draw on global rare disease study CRO experience

In the past five years, we’ve partnered with rare disease developers across all phases, indications and therapeutic areas.

0 +

Rare disease studies






Global patients

Rare disease trials

To enable you to create and test your rare disease therapy, our team creates an unmatched, customer approach for deep understanding of the diagnostic journey, care landscape and patient experience.

Understanding the patient journey webinar

Gain insights into the challenges of accessing global patient populations, manufacturing, regulations and patient-centered protocol design in rare disease clinical trials.

Recruit rare disease clinical trial patients

Finding the right patient for your rare disease trial can be a challenge. PPD offers flexible, innovative protocols for reaching, recruiting and retaining the small, globally dispersed populations of patients with rare diseases. Patients also benefit from our decentralized trial services, which help them stay engaged while ensuring that clinical trial data and materials are handled appropriately and in a timely fashion.

Virtual rare disease trials

By offering virtual trials, the patient experience can be optimized with minimal delays and inconvenience by allowing patients to visit a local care hub or arranging for a nurse to make home visits. Participants are assigned a PPD patient concierge, who can proactively help them with resources and solutions to reduce the burden for patients and their caregivers.

You can employ our digital and decentralized trial technologies that help bring clinical research to the patient, meeting them where they are and simplifying participation through the use of:

  • Mobile sites
  • Wearables
  • Telehealth
  • E-consent
Explore the latest insights on DCTs in rare disease