Rare Disease CRO
Leveraging decades of experience in rare and orphan drug development, our dedicated specialists navigate the complexities of rare disease clinical trials, ensuring the success of your study.
Strategic Rare Disease CRO Solutions for Advancing Rare Disease Drug Development
TFS specializes in supporting rare disease and orphan drug clinical trials, offering tailored solutions to navigate the regulatory complexities, patient recruitment challenges, and specialized endpoint assessments unique to these studies. Our cross-functional teams, spanning dermatology, internal medicine, neuroscience, oncology, and ophthalmology, leverage deep therapeutic expertise and cutting-edge methodologies to bring transformative therapies to market.
With a proven track record of operational excellence and a commitment to patient-focused strategies, we help sponsors accelerate drug development for rare and ultra-rare diseases while ensuring regulatory compliance and high-quality data collection. Whether managing complete gene therapies, pediatric rare disease studies, or innovative targeted treatments, our rare disease CRO services ensure trial success.
Ready to drive breakthroughs in rare disease research? Connect with our rare disease clinical trial experts.
Comprehensive Phase I to IV Rare Disease CRO Services
Built on Precision and Expertise
With nearly 30 years of experience and over 70 rare disease clinical trials completed across all phases, TFS is a trusted partner in rare disease drug development. Our specialized teams navigate the unique challenges of orphan drug research, ensuring regulatory compliance, efficient patient recruitment, and high-quality data collection. Leveraging adaptive trial designs, decentralized trial solutions, and real-world evidence methodologies, we optimize study efficiency and accelerate the delivery of life-changing treatments. From pediatric rare diseases to gene therapies, our global rare disease CRO expertise supports sponsors in overcoming trial complexities while keeping patients at the center.
Global Reach
500
+
trial sites across nearly 15 countries
Broad Experience
40
+
rare disease indications spanning key therapeutic areas
Proven Expertise
70
+
completed clinical trials across phase I-IV and RWE studies
Patient Impact
3,000
+
patients enrolled in rare disease and orphan drug trials
Our rare disease and orphan drug expertise includes specialized pediatric capabilities, offering tailored strategies to manage the unique challenges of pediatric clinical trials, from regulatory compliance to patient recruitment and retention.
Decades of Rare Disease Clinical Research Experience and Proven Success
As a leading rare disease contract research organization, TFS brings decades of experience supporting orphan drug development across a broad spectrum of rare and ultra-rare diseases. Out reams understand the unique challenges of small patient populations, complex regulatory requirements, and the need for specialized trial methodologies. We ensure high-quality results through operational excellence, scientific precision, and patient-centric approaches. Our proven track record in successfully delivering rare disease clinical trials demonstrates our commitment to advancing transformative therapies for patients with high unmet medical needs.
See how we overcome obstacles to deliver results in rare disease research. Read our case study.
Exceeding Expectations: TFS Accelerates Patient Recruitment for a Global Phase III Prurigo Nodularis Study
Exceeding Expectations: TFS Accelerates Patient Recruitment for a Global Phase III Prurigo Nodularis Study
Leading Rare Disease CRO with Advanced Trial Methodologies
TFS employs innovative methodologies to optimize rare disease clinical trials, ensuring efficient patient recruitment, enhanced trial accessibility, and seamless regulatory navigation. Our expertise spans adaptive trial designs, which provide tailored strategies for small patient populations and evolving endpoints, as well as decentralized clinical trials (DCTs) that increase patient access and retention. We integrate precision. Biomarker strategies support patient selection and efficacy assessments, while real-world evidence (RWE) studies strengthen post-marketing surveillance and long-term outcomes research. As scientific advancements continue to reshape rare disease research, gene therapy is emerging as a transformative approach for rare neurological disorders.
Explore the latest breakthrough in gene delivery, preclinical studies, and clinical trial applications. Read our white paper.
Gene Therapy in Neurology: The Case for Optimism
Gene Therapy in Neurology: The Case for Optimism
Broad Indication Expertise in Rare Disease Clinical Studies
TFS provides end-to-end support for rare disease trials across multiple therapeutic areas, ensuring accelerated enrollment and operational efficiency through an extensive global side network and trusted investigator relationships.
Our expertise spans complex indications, demonstrating our commitment to advancing transformative therapies:
Dermatology
Epidermolysis Bullosa, Hidradenitis Suppurativa, Nevoid Basal Cell Carcinoma Syndrome, Prurigo Nodularis
Internal Medicine
Arginase 1 Deficiency, Barrett’s Oesophagus, Carnitine Deficiency, Dupuytren’s Disease, Eosinophilic Esophagitis, Giant Cell Arteritis, Hyperparathyroidism, Hypoparathyroidism, Peyronie’s Disease, Polyarticular Idiopathic Juvenile Arthritis, Urea Cycle Disorder
Neuroscience
Amyotrophic Lateral Sclerosis (ALS), Duchenne Muscular Dystrophy (DMD), Huntington’s Disease, Limb Girdle Muscular Dystrophy, Multiple Sclerosis (MS), including Relapsing Remitting MS (RRMS), Neuropathy (Multifocal Motor), Tuberous Sclerosis
Oncology & Hematology
Hemophilia, Leukemia (Chronic Myelomonocytic) & Myelodysplastic Syndrome, Transfusional Iron Overload
Ophthalmology
Keratoconjunctivitis, Limbal Stem Cell Deficiency
Rare Disease Insights
Leveraging AI to Transform Neurodegenerative Drug Development From Discovery to Clinical Research
Leveraging AI to Transform Neurodegenerative Drug Development From Discovery to Clinical Research
Precision Project Management in Action: Complex Gene Therapy Trial for Rare Neuromuscular Disease
Precision Project Management in Action: Complex Gene Therapy Trial for Rare Neuromuscular Disease
Pioneering Gene Therapy in Rare Diseases: A Path to Revolutionary Treatments
Pioneering Gene Therapy in Rare Diseases: A Path to Revolutionary Treatments
Expanding the Boundaries of Clinical Research: Multidisciplinary Expertise
At TFS, our rare disease expertise is part of a broader commitment to excellence across key therapeutic areas. From Dermatology and Internal Medicine to Neuroscience, Oncology, and Ophthalmology, our teams bring cross-disciplinary knowledge and specialized clinical research strategies to each study. We leverage insights across these fields to provide richer context, unique perspectives, and innovative solutions to enhance study success and accelerate the delivery of new treatments to market.
Supporting your Rare Disease Trial with Flexibility and Expertise
Beyond rare disease clinical development, TFS offers flexible Strategic Resourcing and Functional Service Provider (FSP) solutions that span every phase of the research lifecycle. Our flexible, full-spectrum approach ensures access to top-tier professionals from early discovery and pre-clinical stages through clinical development and into post-market phases.
Frequently Asked Questions About Our Rare Disease Clinical Trials
What makes TFS a trusted partner for rare disease clinical trials? TFS brings decades of experience managing rare and orphan drug trials, ensuring regulatory compliance, and patient-centric strategies.
What makes TFS a trusted partner for oncology clinical trials?
TFS brings nearly 30 years of specialized experience in rare and orphan drug trials, ensuring regulatory compliance, patient-centric strategies, and seamless execution.
Can TFS support pediatric rare disease trials?
TFS specializes in pediatric rare disease research, integrating patient advocacy, specialized endpoints, and regulatory navigation to ensure trial success.
What rare disease indications does TFS specialize in?
Our expertise spans dermatology, internal medicine, neuroscience, oncology, and ophthalmology, covering conditions like Huntington’s Diseases, ALS, DMD, and Epidermolysis Bullosa.
How does TFS optimize rare disease patient recruitment?
TFS employs AI-driven strategies, patient advocacy collaborations, and decentralized trial models to enhance recruitment and retention in rare disease studies.
Does TFS offer regulatory support for orphan drug designation?
TFS’s regulatory experts guide sponsors through orphan drug designations (ODD), breakthrough therapy designations, and accelerated approval pathways.
Partner with TFS HealthScience
Ready to advance rare disease research? Contact TFS today to explore how we can support your rare disease clinical trial needs with precision, expertise, and patient-centered solutions.