How It Started
One of our key focuses at BAP Pharma is to actively engage with patient advocacy groups (PAGs), particularly in the rare disease space. As part of our mission to support access to unlicensed medicines, we believe it’s essential to raise awareness about early access, compassionate use, and post-trial access programs – and to better understand the patient journey from those who live it. Throughout this article, we’ll use the term ‘Early Access Programs’ (EAPs) to refer collectively to Managed Access, Compassionate Use, and Post-Trial Access pathways – programs designed to provide patients with access to potentially life-changing treatments outside of clinical trials and before full commercial availability Our initial outreach was simple: to connect with PAGs open to collaboration, to provide education for communities that may not be aware that EAPs could be a life-changing option, and to listen deeply to the challenges patients face so we can improve our services accordingly. Rare Beacon, a UK-based advocacy group with a global network of patient communities committed to advocating for the needs of rare disease patients, responded with enthusiasm – and a shared interest in addressing this gap.
What We Created
Together, we developed a new educational resource: “Navigating Access: A Guide to Early/Managed Access Programs” This guide was created specifically for rare disease patient groups and individuals to help them better understand what EAPs are, how they work, and how to begin exploring these options with their physician. It includes a downloadable patient checklist designed to support more informed, confident conversations between patients and their healthcare providers. But it’s more than just a guide. It’s a starting point for patients who are navigating uncertainty, fear, and a sense of urgency – and who need information that feels understandable and actionable. Our shared goal was to help return agency to patients -placing them in the driver’s seat of their treatment journey. While EAPs can seem complex and tightly regulated, accessing education is key to helping patients make informed decisions, ask the right questions, and feel empowered to take the next step in their care.
Why This Matters
Patients are at the core of every EAP but for many, the path to accessing these programs is unclear, information is hard to find, full of unfamiliar terminology, and often not tailored to patient needs. At BAP Pharma, we believe that knowledge should never be a barrier to access. While we understand the complexities and sensitivities surrounding unlicensed medicines, we also see an opportunity to provide clear, balanced information that empowers patients to engage confidently in conversations about their care. This collaboration with Rare Beacon was rooted in that principle—providing patient communities the tools they need to better understand what EAPs really mean, how to navigate them, and where to start. It’s about putting patients in control, supporting advocacy efforts, and ensuring their voices shape the future of access. We also believe in learning from – not just about – patients. Their lived experience helps shape better-designed programs, reduce unnecessary burdens, and ensure that access truly meets their needs – not just regulatory or commercial requirements. Too often, the conversation around access focuses on systems, approvals, and compliance – but behind every program is a patient who is out of options, fighting for time, clarity, or even just hope. That’s what makes this work urgent. We’ve heard stories from patients who spent weeks trying to even find out that these programs exist, or who didn’t know how to ask their doctor the right questions. These moments shouldn’t happen in isolation. They should be met with support, information, and reassurance.
The Impact
The collaboration with Rare Beacon was open, creative, and deeply aligned from the start. Both teams brought ideas to the table, and the final guide reflects a shared commitment to clarity, accessibility, and real-world usefulness. The potential impact of this resource is far-reaching. For patients navigating uncertainty, understanding that EAPs may be an option can be life-changing. For advocacy groups, it’s a tool to help inform and guide their communities. And for the industry, it underlines the importance of integrating patient voices early in the development of any resource or initiative. As Blayne from Rare Beacon put it: “This was the collaboration of the year for me and so much fun to be a part of, so thank YOU for everything you did to bring this valuable resource to life.” We’re proud of what we’ve created together, and excited about the potential for this guide to support and inform more rare disease patients as they consider their treatment options. Explore the live resource here: Navigating Access: A Guide to Early/Managed Access Programs
What’s Next
This project is part of our wider goal to build more partnerships with patient advocacy organisations -so we can continue to centre patient needs in the work we do. By working directly with PAGs, we gain critical insights that help us deliver services that aren’t just compliant and efficient, but genuinely patient-centric. If you’re part of a patient organisation or community and would like to share your experience, collaborate on resources or simply have a conversation – we’re here and we’re listening.
Let’s continue making access clearer, earlier, and more patient-driven.
This was the collaboration of the year for me and so much fun to be a part of, so thank YOU for everything you did to bring this valuable resource to life.
