Rare Disease CRO Therapeutic Research

Therapeutic Expertise 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.

Connect with our rare disease experts

Rare diseases survey report

Identifying clients’ needs and the challenges they are facing in conducting rare disease clinical trials.

Read the findings

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.

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Rare disease studies

Sites

Countries

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.

Watch the webinar on demand

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

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.