Rare Disease CRO Services
Alleviating the burdens of rare diseases
Rare diseases pose significant challenges for patients and their caregivers, with 95% of these disorders lacking U.S. Food and Drug Administration (FDA)-approved treatments. At the PPD™ clinical research business of Thermo Fisher Scientific, we prioritize alleviating the burdens of rare diseases and recognize the individuals affected by these conditions.
Our rare disease contract research organization (CRO) services center the patient. By incorporating the experiences of trial participants, caregivers and investigators, we enable a patient-centric approach to develop transformational therapies and bring positive change to the lives of people with rare diseases.
Persistent challenges drug developers face in advancing treatments for rare diseases include:
- A lack of consensus on endpoints
- Inexperienced or understaffed research sites
- Regulatory precedents
- Geographically dispersed patient populations
- Finding and retaining enough participants for clinical trials
We enable clients to navigate these obstacles and overcome the difficulties that arise in the clinical development, testing and regulatory approval of rare disease treatments.
Enabling Successful Rare Disease Clinical Trials
Committed to rare disease research
Our Rare Disease and Pediatric Center of Excellence delivers innovative solutions to overcome challenges associated with orphan drug development. We offer rare disease clinical trial services that decrease patient burden and help them navigate the complex health care system. Our patient services include coordinating patient transportation, providing flexible reimbursement options and connecting patients to home health care resources.
Recruit rare disease clinical trial patients
Finding the right patients for rare disease trials can be challenging due to the small, globally dispersed populations. We offer flexible and innovative protocols for reaching, recruiting and retaining these patients. Additionally, our decentralized trial services ensure that clinical trial data and materials are handled appropriately and in a timely fashion, while keeping patients engaged.
Virtual rare disease trials optimize the patient experience by minimizing delays and inconvenience. Patients can visit local care hubs or receive home visits from nurses. They are assigned a patient concierge who proactively empowers them with resources and solutions to reduce the burden for both patients and caregivers. Our digital and decentralized trial technologies, such as mobile sites, wearables, telehealth and e-consent, bring clinical research to the patient and simplify their participation.
Patient-centered rare disease research
We leverage patient-centered partnerships to drive treatments forward. Our strategic relationships with advocacy groups, academic institutions, health care providers and pharma industry groups offer firsthand insight into patients’ journeys. We take a holistic approach from the outset — which ensures that decisions made in rare disease research are the right ones — and we accelerate study startup by providing upfront understanding of data, cost and necessary clinical expertise. Our cross-functional team ensures consistency and delivers clinical trials with rapid patient recruitment, retention and positive experiences in mind.
Rare Disease and Pediatrics Center of Excellence
Our Rare Disease and Pediatrics Center of Excellence shapes trial design, strategy and delivery for adult and pediatric rare indications. With extensive experience in conducting rare disease studies, we deliver innovative, patient-centric solutions and train our study teams to address the complexities young patients and their caregivers face. Decrease the participation burden for pediatric patients and their loved ones with our hybrid study approaches that include both in-clinic and at-home endpoints.
Rare disease expertise across therapeutic areas
Rare disease trials span many indications. Explore our dedicated rare disease expertise in neurology, metabolic and neuromuscular clinical research development.
With more than 100 studies in therapies for rare neurological conditions, including neuromuscular diseases, genetic disorders, cerebral cavernous malformations and lysosomal storage disorders, we excel in designing and executing trials from orphan drugs to gene therapies.
We offer extensive experience and patient-centered development strategies for rare metabolic diseases, such as Gaucher and glycogen storage diseases.
Our global team tackles complex clinical development challenges for therapies treating rare neuromuscular conditions like ALS.
Our heroes in rare disease
Our dedication to rare diseases is further exemplified through our heroes program, which shares the stories of individuals facing significant health conditions who benefit from therapies made possible by clinical research.