Navigating Access:
A guide to Early/Managed Access Programs
Early Access Programs (EAPs) are initiatives designed to provide patients who have severe or life-threatening conditions access to treatments that are still in development or have not yet received full regulatory approval in their country.
These programs can be a crucial lifeline for patients with rare diseases, offering hope for effective therapies that might not otherwise be available.
EAPs allow patients to benefit from potentially life-changing treatments under strict compassionate use or managed access mechanisms that grant access to unauthorised medicines (often before that medicine is available to the general public) when no other treatment option is available, and patients are unable to participate in a clinical trial.
This resource will provide a comprehensive guide to navigating Early Access Programs (EAPs), designed to help patient groups support their communities in accessing critical treatments.
The guide aims to equip patient organisations with the knowledge and tools needed to effectively navigate and advocate for EAP access.
It is recommended that you first enroll in the following courses to gain a basic understanding of Early Access Programs (EAPs) so you can best utilise the tips and downloadable checklist offered in this guide.
• What patient groups need to know about Early Access Programs (EAPs)
• What does early access actually mean?
With thanks to:
BAP Pharma provided pro bono support in the creation of this course.
Debora de Paula
Katie Bowen
With thanks to the Funders of The Empowerment Programme 2025
Disclaimer
This guide was produced in collaboration with BAP Pharma, with the case study approved by Duchenne UK.
All decisions regarding the naming of companies were made independently by Beacon and do not reflect any judgment, criticism, or assumption about the organisations mentioned.
Resource type
Course
Skill level
Intermediate
Duration
1 hour
Last updated
May 2025
Resource type
Course
Skill level
Intermediate
Duration
1 hour
Last updated
May 2025
What will you learn in this course?
- An introduction to EAPs and their importance for rare disease patients.
- Guidance on how patient groups can conduct research into available EAPs and determine eligibility criteria.
- The role of treating physicians and tips for effective communication between the patient and physician.
- A downloadable patient checklist outlining key information to discuss with physicians before engaging with them regarding an EAP.
- Common challenges faced by patient groups in an EAP, and how to overcome them.
- Practical ways patient groups can advocate for their community to improve access to EAPs, as illustrated by a Duchenne UK case study.
Who is this course for?
This course is designed for patient organisations who want to gain the knowledge and tools needed to effectively navigate and advocate for EAP access.
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