Articles and publications

Our latest insights and news on rare disease, and how we’re helping drive change through clinical research.

May 2020

Sticking to the medicine in rare disease

We all know how important it is for rare disease patients to take their medications as prescribed. Many rare diseases have no cures and the treatment options available are usually for managing symptoms, rather than treating the cause. Not taking the right medications at the right time can lead to symptoms returning.

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Blog

March 2020

Keeping rare disease communities and trial participants engaged during the COVID-19 outbreak

It’s safe to say COVID-19 has disrupted all our lives and the work that we do. Research staff are working from home, clinical trials are being suspended or delayed, and patients taking part in studies are facing travel disruptions and potential lockdowns.

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Rare Opinion

February 2020

After February 29th

Rare Disease Day is a much-needed fixture in the calendar, but the job of delivering new treatments cannot be achieved in a big-bang, once-a-year burst of activity.

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Media Article

February 2020

Rare Disease R&D

Our founder Julie Walters speaks to Contract Pharma about finding the hard-to-reach and leveraging big data and machine learning in drug development

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Rare Opinion

December 2019

Alnylam’s bold plan to insure health insurers

A novel pricing mechanism might help payers warm to pricey, ultra-orphan drugs.

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Rare Opinion

October 2019

Priority review vouchers start to lose their shine

PRV sales have been useful in funding companies’ rare disease-focused ambitions, but perhaps not for much longer.

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Rare Opinion

September 2019

What’s missing from the FDA’s draft guidance on natural history studies in rare disease

The regulator got a lot of things right, but this is how its thinking could be improved.

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White Paper

May 2017

A new approach to shortening the diagnostic odyssey

Patient-powered medical education promises to address knowledge gaps in rare disease.

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White Paper

February 2017

Tackling rare disease with big and small data

Why we need workable models for combining very large datasets with the very small

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Get in touch

Interested in hearing more? Feel free to reach out to us, and we’ll make sure to get back to you as soon as possible.