A third of rare disease patients unaware of early access programmes


One-third (32%) of rare disease patients are not aware they can access unlicensed medicines through Early Access Programmes, new research has revealed.

However, two-thirds (68%) of patients are comfortable receiving medicines before they’re licensed through their country’s healthcare system.

The new data was released by pharma company Clinigen to mark the start of Rare Disease Month.

The findings are from NaviGATE, Clinigen’s UK-based pilot educational programme for rare disease patient advocates and organisations.

The NaviGATE programme aims to equip the rare disease community with the tools they need to advocate on behalf of themselves and others more effectively. The goal is to facilitate greater participation in rare disease research and development and to enable increased access to medicines for more patients.

Dr Lorna Pender, Global Patient Engagement Lead at Clinigen, said: “Shockingly, it can take five to thirty years to get a diagnosis of a rare disease, and when they finally receive one, patients often struggle to cut through the complex healthcare industry to find a way forward. Patients need to be backed by knowledge and resources, and knowing where they can get access to medicines – even before they are on the market – is vital for people with undiagnosed or rare diseases.”

The data was taken from 50 rare disease patients, parents or carers, patient advocates, organisations and charities. Of the 50, 42% represent patients that are undiagnosed, which shows a significant white space in research and knowledge.

Only 15 respondents (30%) had heard of EAPs and have a basic understanding, while 69% wanted to know where they can access information about EAPs.

Rare Disease Month is recognised in February to raise awareness of the over 7,000 rare diseases that impact over 300 million people globally.

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