What patient groups need to know about Early Access Programmes (EAPs)

Since many rare diseases are life-threatening or life-limiting, early access programmes (EAPs) can be a lifeline for patients who urgently need access to promising treatments before they receive regulatory approval in the country where the patient lives.

Early Access Programmes are complex and vary across countries.

This guide aims to help patient groups navigate these complexities, so they have a clear understanding of the regulatory requirements and processes involved in EAPs.

Gain the comprehensive information needed to make informed decisions about participating in and supporting early access programmes.

Enroll now to explore EAPs, especially if your group has a treatment on the horizon or one that’s currently available and wish to access it.

With thanks to:

John Lagus, Founder of Bluestem Pharma Consulting, LLC, who generously provided his expertise and pro-bono support to create this course for patient groups.

My name is John Lagus and I am passionate about helping patients around the world with unmet medical needs get access to medicines for serious or life-threatening diseases by supporting pharmaceutical companies implement the most effective global early access programme for their unique situation.

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In my career I have been involved with the design, set up, and implementation of more than 200 global early access programmes. I work with companies to leverage my subject matter expertise to implement best practices for all stakeholders. I also provide support to patient groups and the patients they serve. Very early in my career I was introduced to the rare disease space when in 1994 I joined a company called Orphan Medical to oversee development and FDA approval of a medicine called ANTIZOL. Patients outside the US were seeking access to several of our medicines approved by the FDA and we used an early access programme to get medicine to these patients. In 2006 I joined Idis, a company based in the UK, and the pioneer in the early access space. Over the next 18 at Idis, Clinigen, and Tanner Pharma I educated pharmaceutical companies about how early access programmes work, and then designing and supporting implementation of their global early access programmes. My passion to support the rare disease community continues over 30 years later.

With thanks to the Funders of The Empowerment Programme 2024

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Resource type

Course



Skill level

Advanced



Duration

1 hour



Last updated

April 2024



Resource type

Course



Skill level

Advanced



Duration

1 hour



Last updated

April 2024

What will you learn in this course?

What is an early access programme?
Why do pharmaceutical companies put in place early access programmes?
What are the different names for these programmes?
How is medicine in an early access programme provided?

Who is eligible for an early access programme?
What are the risks and benefits of early access programmes?
What is an early (or expanded) access policy and why is it important?
Who can communicate with the pharmaceutical company?

Who is this course for?

This course is designed for patient organisations who are beginning to think about getting involved in research and want to understand as much as possible before making that jump.

Your patient group may have a treatment on the horizon or one that’s currently available and wish to gain access to it.

This guide should help you maximise the potential benefits of early access programmes for your community so you can make informed decisions about participating in and supporting EAPs.