Articles
Patient registries and why they are essential for rare diseases

Patient registries and why they are essential for rare disea...

How essential are patient registries for rare diseases? Eileen O’Brien discusses, in the third article out of a five-part series from Siren Interactive. At a very basic level a registry is a systematic collection of standardized data on a group of patients. “There are different…

Articles
What’s the most important question in medicine?

What’s the most important question in medicine?

PatientsLikeMe founder Ben Heywood discusses the rise in online patient communities and the impact this has had on the awareness of rare diseases. I’m inspired daily by patient stories. They connect with others who have the same disease, often for...

Articles
Spotlight on…Rett Syndrome

Spotlight on…Rett Syndrome

This month’s spotlight into a disease or therapy area aligns with our rare disease focus month, as we look into how pharma can better support patients with Rett Syndrome. Our theme on pharmaphorum this month is on rare diseases and we’ve had a great response so far from rare disease patients, organisations and...

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Building a Phase I oncology centre

The question is not whether an institution should ultimately develop Phase Ia capability. It is when the organisation is ready.

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