Rare Disease Day

In 2024, Rare Disease Day falls on an appropriately rare calendar date: 29 February, a leap year.

Our CEO, Julie Matthews, gives an insight on Rare Diseases on PharmaTimes Online.

Sustainability challenges in rare disease trials remain, but change is happening

On Rare Disease Day 2024, Clinical Trials Arena investigates if and how rare disease trials can tackle environmental sustainability.

TMC is a global clinical research organisation dedicated to supporting the progression of life-saving treatment solutions for diseases that are often neglected.

We’re a specialised and experienced full-service CRO providing world-class support to pharma/biotech companies. To maximise success, we offer our clients a broad range of services with a focused and tailored approach for all their drug development requirements.

global partner

A Message From Our CEO

It’s hugely important that rare diseases are taken very seriously. Often a new company will reach out to us because a company founder has a family member living with a rare disease they’re seeking treatment for. These people are championing a particular orphan disease and we will guide them through the process of developing a treatment, proving to the authorities that the drug is safe and that it works, then getting it on the market so their loved one can be treated.

The heartbreak for a person with an orphan or rare disease is that they often feel neglected. It’s our job to turn that around and make those neglected groups feel looked after, assuring them we’re there to try and find treatments to make their lives better.

julie matthews

Our Expertise

Our development support activities span the early pre-clinical phase right through to post-marketing. We provide all the functions of a fully resourced pharmaceutical company, providing ‘bolt-on’ services that complement a client’s in-house resource, enabling them to access all the development capability appropriate for each stage of their project.


We only employ knowledgeable and trusted Associates who have worked in the pharma industry for at least 15 years. These experts can draw on their extensive experience to guide programmes and strategies — providing flexible, integrated and cost-effective solutions to meet our clients’ needs.


We specialise in orphan, rare and oncology diseases. Rare diseases affect 1 in 10 people, meaning more than 350 million people worldwide*. These people are often overlooked as there are significant challenges in the design and execution of clinical trials therapeutics. That’s why it’s our mission, indeed our passion, to offer drug development support in these disease areas.

*Source: https://www.rareundiagnosed.org/rare-disease-facts/


No two drug development programmes are the same — just as no two patients are the same. We help our clients understand the patient journey, facilitating their drug development pathway to bring treatments to market quickly and efficiently.

watch our latest recorded


Exploring the Differences in Medical Services for Rare Diseases

Milestones in Numbers

Championing the patients living with life-limiting orphan and rare diseases

0 years
since TMC was founded
associates worldwide
paediatric investigation plans (PIP)
orphan drug designation (ODD) applications