Conduct seamless, cost-effective clinical studies for rare diseases 

Conventional clinical study protocols and pathways don’t always apply when developing drugs for rare diseases. Limited patient populations make recruiting and retaining patients difficult, and regulatory submissions often require bespoke documentation and agency interactions.  

TMC supports clinical development and delivery — across all phases of clinical trials globally — for rare diseases, using both decentralised and hybrid trial models to improve access and engagement in difficult-to-reach communities. Our clinical experts provide scientific and strategic guidance throughout the drug development process, from Phase I safety assessments to Phase IV post-marketing surveillance.  

We ensure your clinical study is conducted timely and cost-effectively, taking advantage of incentive pathways like orphan drug designations (ODD) to reduce costs and extend your market exclusivity. By accelerating clinical development timelines, we drive your innovative, life-enhancing therapy to market (and the patients who need it) as quickly as possible, all while maintaining a rigorous focus on quality. 

Extensive therapeutic experience in rare indications 

As a specialist global clinical research organisation (CRO) for rare diseases, we probably have more product and therapeutic experience in this area than any other CRO. Our clinical experts have supported the progression of life-saving rare disease treatments for nearly 25 years.  

This experience includes biologics, cell therapies and gene therapies across our key specialisms:  

  • Oncology 
  • Neurological disorders 
  • Respiratory diseases 
  • Haematology 
  • Ophthalmological conditions 
  • Neonatology 
  • Hepatology 
  • Nephrology diseases 

Using our knowledge of rare diseases and clinical expertise, we ensure the successful clinical development of your product, including clinical trial design, managing patient recruitment in small populations and overseeing data integrity to keep your programme on track. We also hold orphan drug designations globally and can guide you through the application to unlock fee reductions, extend market exclusivity and de-risk your programme. 

Clinical governance is another crucial aspect of our clinical services. We facilitate data safety monitoring board meetings, prepare comprehensive clinical study reports and help you navigate complex international regulations to ensure seamless regulatory submissions for your study.  

A proven approach to clinical studies  

With TMC, you get a full-service CRO with access to our in-house team of clinical experts plus a global network (spanning 70+ countries) of highly skilled and experienced associates.  

Our average team tenure is 5+ years, so there’s no ‘B’ team here. You get a dedicated ‘A’ team throughout your clinical development project. This team combines our clinical research capabilities with your internal processes and goals to provide scalable, flexible resource solutions, integrated project management and tailored guidance. 

Whether you need assistance with your clinical trial design, patient recruitment, ODD application or maintaining clinical governance, we conduct and manage your clinical study from start to finish. Contact our team today to see how we can bring your new therapy to market faster and more effectively.