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Rare Diseases

AllieviatING The BurdenS Of Rare Diseases

For 95% of rare diseases, there are no treatments approved by the U.S. Food and Drug Administration (FDA). This creates an immeasurable physical and mental toll on patients and their families. While rare disease development is a priority for many drug developers, they face persistent obstacles. These barriers include:

  • lack of consensus on endpoints,
  • inexperienced research sites,
  • a shortage of regulatory precedents,
  • geographically dispersed patient populations, and
  • access to enough patients to participate in a rare disease clinical trial.

PPD Rare Disease Clinical Trial Services

The PPD clinical research business of Thermo Fisher Scientific designs and conducts rare disease studies that solve for the challenges that patients and drug developers face. Our focus is to decrease patient burden and help patients and caregivers navigate a complex health care system.

Leveraging innovative methods and long-standing partnerships in the rare disease community, we increase enrollment and retention in cutting-edge rare disease clinical trials. We’re continually investing in enhancements to the investigator, patient and caregiver experience.

Draw On Global Rare Disease Study Experience

In the past five years, we’ve partnered with rare disease developers across all phases, indications and therapeutic areas. Our profound understanding of the diagnostic journey, care landscape and patient experience allows us to create an unmatched approach for each client.

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Recruit Rare Disease Clinical Trial Patients

Innovative, flexible protocols – combined with digital and decentralized trial technologies – offer new ways to reach, recruit and retain the small, globally dispersed populations of patients with rare diseases. With trials being reimagined to bring clinical research to the patient, we are able to meet them where they are and simplify participation with tools such as mobile sites, wearables, telehealth and e-consent.

Virtual Rare Disease Trials

Virtual trials enable us to optimize the patient experience with minimal delays and inconvenience – whether patients visit a local care hub or a nurse makes in-home visits. A patient concierge – a team member who is a dedicated resource for patients and their caregivers – is another way that study teams can elevate the rare disease clinical trial research experience. This keeps patients engaged and ensures that all data and materials are handled appropriately and collected on time.

Explore the latest insights on DCTs in rare disease

Patient-centered Rare Disease Research

Patient-centered partnerships are also key to our approach. We actively build strategic relationships with patient advocacy groups, academic institutions, health care providers and pharma industry groups. Initiating this comprehensive engagement alleviates patient burden and assures compassionate insight of the patient journey. We also tap into our own network of PPD employees who have volunteered to share their experiences as patients and caregivers.

Make The Right Decisions Earlier

In rare disease research, the wrong decision can upend your study. PPD accelerates study start-up with a holistic approach that provides an upfront understanding of data, cost and clinical expertise. Our advance planning accounts for

  • site costs,
  • experience in the indication,
  • disease diagnosis and progression, and
  • clear endpoints.

Our customers enjoy cross-functional teams dedicated to their projects from the start, which delivers consistency as the teams design and deliver your trial with rapid recruitment, retention and patient experience in mind.

We plan for efficient natural history studies to run in parallel with therapeutic studies. Natural history studies:

  • 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

PPD’s Rare Disease and Pediatrics Center of Excellence (COE) focuses on rare and pediatric indications to shape trial design, strategy and delivery. The cross-functional experts of this COE deliver innovative, patient-centric solutions and train our study teams to skillfully address the most complex challenges of these young populations.

Our aim is to decrease burden and increase flexibility for pediatric patients and their loved ones. Bringing children into a clinic is challenging, and it can be even more difficult for children with rare diseases. With the widespread availability of decentralized options, we can design hybrid approaches, including a mixture of in-clinic and at-home endpoints. These options are supported by bespoke digital platforms and novel trial design solutions. Access to the Rare Disease and Pediatrics Center of Excellence can help you give pediatric rare disease patients the best possible experience.

 

Rare Disease Expertise Across Therapeutic Areas

Rare diseases cover many indications. Explore our dedicated rare disease expertise in neurology, metabolic and neuromuscular clinical research development.

PPD Heroes in Rare Disease

PPD Heroes is a program dedicated to sharing the inspiring stories of individuals facing significant health conditions, including rare diseases. These patients have benefitted from therapies that are made possible by clinical research. Learn more about PPD Heroes.