Rare Disease CRO Services
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
Make the Right Decisions Earlier
The wrong decision can upend your study in rare disease research — especially for clients developing an orphan drug. That’s why PPD takes a holistic approach from the outset, accelerating study start-up by providing an upfront understanding of data, cost and necessary clinical expertise for your trial. The advance planning in our CRO services accounts for:
- Site costs
- Indication-specific experience and available staff
- Disease diagnosis and progression
- Clear endpoints
Your project is assigned a dedicated, cross-functional team from the start, meaning you’ll have consistency as our experts design and deliver your clinical trial with rapid patient recruitment, retention and experience in mind.
We also run efficient natural history studies in parallel with therapeutic studies, which:
- Generate the real-world evidence required by the FDA and other regulatory bodies to better characterize patient populations and delineate target populations
- Focus on describing disease frequency, features and evolution by collecting real-world data from patients
- Are often performed early in the clinical development process to support and guide the design of rare disease clinical trials
Rare Disease And Pediatrics Center Of Excellence
The PPD Rare Disease and Pediatrics Center of Excellence (COE) focuses on shaping trial design, strategy and delivery for adult and pediatric rare indications. The COE has performed more than 240 pediatric studies and supported over 545 total rare disease studies across all phases, indications and therapeutic areas. Our cross-functional rare disease experts deliver innovative, patient-centric solutions and train our study teams to skillfully address the complex challenges faced by these young patients and their caregivers.
To decrease the participation burden for pediatric patients and their loved ones, we aim to increase trial flexibility. PPD can design hybrid study approaches that include both in-clinic and at-home endpoints by leveraging novel trial design solutions, our bespoke digital platforms, and the widespread availability of decentralized clinical trial options.
Rare Neurological Disorders
Rare Metabolic Disease
PPD Heroes in Rare Disease
Our PPD Heroes program is dedicated to sharing the stories of individuals facing significant health conditions like rare diseases. These patients have benefitted from the therapies that are made possible by clinical research performed with CROs like PPD.