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First UK patients

Having opened recruitment at our two UK trial sites in April, we are thrilled to announce that the MCDS-Therapy team has successfully recruited three patients.

Recruitment will continue at our two UK sites - The Great North Children's Hospital in Newcastle and the Evelina Children's Hospital in London - until our UK target of 12 patients has been met.


Following a year's baseline observation in the UK, recruitment will open in sites across Europe and in Australia. For more information about the project's timeline and partners, please visit our MCDS-Therapy website.

This bulletin also includes:
  • What is 'health economics' and why are we interested in it?
  • MCDS-Therapy in the news
  • Spotlight on ERN-BOND - the European Reference Network for rare bone diseases
  • Helping students understand drug repurposing pathways

Health economics: what is it and why bother?

It may seem unusual that a team of economists is involved in a clinical trial. However, in order for a treatment to be approved and paid for by the NHS, you've got to show evidence that it is cost-effective - i.e. that its benefits outweigh its costs and risks.

The health economists involved in MCDS-Therapy are busy measuring cost-effectiveness through a variety of health economic models. Find out more about what they're doing and why in our blog.
Learn more about health economics!

MCDS-Therapy in the news

When MCDS-Therapy opened recruitment in April, we were delighted to see our story shared across mainstream media.

Our full press release is an excellent summary of the work we're doing and is available on the Newcastle University Press Office website. Please do share it with anyone who may be interested.
Read the full article

Spotlight on ERN-BOND

European Reference Networks (ERNs) are virtual networks for clinicians and researchers to share expertise and information across the EU.

ERNs were formed because no single country has the expertise and capacity to effectively diagnose and treat every rare disease. By connecting doctors across borders, ERNs enable knowledge - not patients - to travel. This means patients can access higher quality care and treatment from home.

There are 24 ERNs in total, each focusing on a different therapeutic area. For instance, Endo-ERN focuses on endocrine conditions and ERN LUNG focuses on respiratory diseases. The ERN that skeletal dysplasia falls into is ERN-BOND - the European Reference Network for Rare Bone Diseases.

Click the link below or watch the video above to find out more about ERNs. You may spot Luca Sangiorgi - Coordinator of ERN-BOND and MCDS-Therapy's Principal Investigator in Bologna - at work in the film.
Find out more about ERNs

Rick and Ronny spread the word in Berlin

MCDS-Therapy isn't just a clinical trial. It is a pioneering, investigator-led, EU-funded, international, drug repurposing and rare disease clinical trial.

We are so proud of everything we're doing that we're spreading the word far and wide. Earlier in June, Rick Thompson, from our partner Findacure, and Ronny Schmidt, from our partner Sciomics, travelled over to Berlin for a SPARK conference. Here they talked with academic researchers about the challenges and opportunities around repurposing drugs for rare diseases.

If you represent an academic body and would like to find out more about speaking opportunities, please do get in touch:

Thanks for reading! We hope you've enjoyed hearing about the MCDS-Therapy team's latest work. If you have any suggestions for future newsletters, please let us know by contacting

With best wishes,

The MCDS-Therapy team
Copyright © 2019 MCDS-Therapy, All rights reserved.

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