The last chance: how early access programmes save lives – and where patient engagement can help
Olivia Kersey, Patient Affairs / Clinical Research and Strategy Consultant. Lifelong Sibling Carer
Few things are as heartbreaking as confronting the limits of modern medicine. Especially in rare diseases, patients and families facing serious / life-threatening illness can find themselves out of options, even before any kind of treatment ‘journey’ has begun. This is where early access programmes (EAPs) can come in – often literally meaning the difference between life and death.
Early access programmes provide urgent access to promising treatments which have not yet received regulatory approval (i.e., been officially declared safe and effective by national bodies such as the FDA). Many people consider clinical trials to be “the last chance” for expanding treatment options, but EAPs take possibilities a step further, specifically being available to those who are not eligible to receive treatment through enrolling onto a study.
Because the treatments on EAPs have not yet been approved, they are subject to additional safety processes to ensure patients are protected while respecting the urgency of trying out the experimental treatment.
The importance of early access programmes was highlighted at a recent session at ‘Operationalise: Early Access Programmes Summit (Europe)’. The session featured Meryl Chambers, Chair & Coordinator of Soft Bones UK Hypophosphatasia Foundation, who shared her family’s story through a live discussion with Naomi Litchfield, ISPEP Ambassador, Director of Patient Advocacy at Bionical Emas, and previous clinical research nurse at Great Ormond Street Hospital. Meryl explained how her son Maddox was diagnosed with hypophosphatasia, a rare bone condition, after becoming gravely unwell as a newborn. As no treatment was available, Maddox was expected to be put on palliative care – but then an opportunity to join an EAP arose. The urgency of Maddox’s illness was such that he was rapidly enrolled onto the EAP, becoming the first person in the UK to receive the investigational treatment. This treatment saved his life, and he is now 12 years old and thriving.
This is a powerful example of the importance of EAPs – but there is much to be done. Many patients and families (and even specialist clinicians) are not aware of EAPs as a potential option; this represents a real risk of avoidable death and decline to quality of life. Awareness gaps persist for those who are fortunate enough to access an EAP – Meryl herself was unaware that Maddox’s treatment could potentially be taken away if the medicine was not ultimately approved, only finding out when taking part in the NICE appraisal process as a patient representative. Meryl explained that while some information was provided about the EAP, it was limited, jargon-heavy, and did not make the investigational status of the treatment or the possibility of losing access clear. When survival is dependent on an experimental treatment, this is not a detail that anyone can afford to miss. Meryl noted that short, accessible videos that families could “dip in and out of” would have been really helpful in her situation – adding that when families are going through the emotional and intense period of urgently enrolling onto an EAP, they’re not necessarily able to take in all the relevant information in one go.
Recognising the crucial need for educational resources that address patient/carer needs, back at Bionical Emas, Naomi is spearheading a patient co-creation board; through this collaboration, patients have the opportunity to shape the development of EAP awareness materials.
This initiative is a great example of how patient engagement can fit into the world of EAPs – ensuring that information and support materials are tangibly aligned to the needs of the ‘end users’.
What can patient engagement professionals do to make a difference in Early Access?
Work with cross-functional colleagues to secure budget allowance for patient engagement in EAPs
Co-create or review patient-facing EAP materials with people with relevant lived experience
Develop long-term relationships with patient advocacy groups to raise community awareness of EAPs in general and better understand barriers to access, increasing the chance of those who need access actually getting it
Early Access is a lifeline for patients and families facing unimaginable medical crises, yet relatively little collaboration is done with them to ensure their needs are met. As such, patient engagement professionals have an opportunity to apply transferable expertise from ‘traditional’ research avenues and make a difference in this high-impact area.
What are your thoughts on patient engagement in Early Access? Let us know in the comments!
We will be discussing this and hearing from Naomi Litchfield, Steve Clark, Lindsay Randall and Oliver Bromley in an ISPEP Masterclass on 11th November 2026 at 3pm GMT. Be sure to register for the session.
Further reading: What patient groups need to know about Early Access Programmes (EAPs) – a course from Beacon for Rare Diseases
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