Progress Your Rare Disease Trials with Confidence

Get expert biometrics support to overcome challenges, speed up timelines, and deliver rare disease breakthroughs.

Rare disease trials face unique challenges that can slow progress and delay life-changing treatments reaching patients.

These include small, geographically dispersed patient populations, complex endpoints and novel biomarkers, stringent regulatory scrutiny, evolving guidance, and the need for innovative trial designs and advanced methodologies.

With decades of experience supporting biometrics in trials for rare oncology, genetic, and immunological disorders, we can help you navigate these challenges. Our high-quality biometrics solutions accelerate timelines, protect data integrity, ensure regulatory compliance, and keep your studies moving efficiently from start to finish.

Our Expertise

We’ve supported rare disease trials across:

Oncology (e.g. ovarian cancer, multiple myeloma, head & neck cancers)

Genetic and neurodegenerative disorders (e.g. DMD, systemic inflammatory syndromes, Wiskott-Aldrich syndrome, trigeminal neuralgia)

Immunological conditions (e.g. IgA nephropathy)

Skin and joint diseases (HS, psoriatic arthritis)

Our Solutions

We work in true partnership, embedding seamlessly into your team, actively identifying and managing risks, and maintaining clear communication and governance to deliver reliable, high-quality results across global sites.

Choose from one of our flexible solutions to match your study needs:

Strategic & Technical Consulting

Specialized guidance across statistical design, data management, programming, and technology solutions

Functional Service Provider

Powering long-term success through close collaboration and bespoke integrated solutions.

Project-Based Solutions

Flexible, high-quality biometrics solutions to support clinical trials from start to finish.

Success stories

Phase III Rare Disease Trial: Veramed provided strategic statistical consulting to optimise study design and primary endpoint selection. Simulations were used to guide regulatory discussions and support protocol amendments.

Multiple Phase III rare disease programme in HS: Full biometrics reporting and project management across multiple studies run in parallel including DSMBs, open-label extensions and integrated summaries

Gene Therapy in DMD: Delivered full biometrics support from study design through submission, with a focus on PRO data and emerging endpoints.

Multiple Myeloma (Post-Approval): Supported market access and HTA activities through indirect treatment comparisons, targeted safety investigations, and publication planning.

IgA Nephropathy (Immunology): End-to-end support for a Phase II study, including Medrio EDC build, RTSM integration, CDISC deliverables, and PK modelling.

Discover Veramed

Your guide to the industry and our role at its cutting edge.

From Setback to Success: How a Biotech transformed their Rare Disease Clinical Study by leveraging Biostatistics and Programming expertise