Holly Lumgair, patient advocacy manager at Clinigen speaks to RARE Revolution Magazine about Clinigen's new innovative fellowship scheme which will support patient advocates to help improve early access.
An increase in patient resources, regulatory structures like the 21st Century Cures Act in the United States, and societal pressure are all playing their part in increasing awareness of, and transparency around early access.
The COVID-19 pandemic has brought with it a striking development in societal awareness of what it is like to live with a life-changing, sometimes life-limiting medical condition with no approved treatment options. Consequently, more of us have grappled with the tension between the need to generate essential evidence about new treatments, and the need to treat, with all of the ethical and practical questions that this raises.
For those people with rare and chronic conditions, including cancer, living with this unmet need is a stark reality - with only 5% of the 7000+ rare diseases having a licensed treatment. Early Access Programmes (EAPs) are important because they have the potential to provide ethical access to treatment for those most in need, when no licensed, suitable alternatives exist.
Patient group leaders can play a pivotal role both during the design and subsequent roll out of EAPs: they can ensure the voices and needs of their community are taken into account by industry and help explain the complexities of access to investigational treatments to families who are desperate for treatments. These are complex and heavily regulated areas requiring an understanding of the process, the drivers and the delicate balancing act that EAPs can be to create and deliver.
We asked the question - are patient communities equally able to be engaged and have a voice when it comes to early access? Although community-facing education around early access is improving, awareness amongst patient groups is not established across the board. It is our understanding that although there are some very well-informed groups, well versed in the intricacies of EAPs, there are still many barriers to entry at a patient group and patient community level. With this in mind, Clinigen has created the Patient Advocate Fellowship in early access to create a network of advocates who understand early access and can help each other.
We explored this with several patient advocates from patient groups who experienced a steep learning curve when EAPs appeared and there was a need to engage and respond appropriately, on behalf of their patient community. We spoke to these patient advocacy experts about the challenges they faced and how best to tackle the educational gaps that exist, for the benefit of all stakeholders.
A major challenge for patient groups is low resource levels. Time, staff and budget are stretched across multiple priorities and urgent needs, leaving little resource to think about early access in advance. Bettina Ryll from Melanoma Patient Network Europe (and member of EU Cancer Mission Board), explains:
Those who have been through the journey of navigating early access in their community are valuable assets with insights that can benefit other organisations. However, having the right mechanism for knowledge-sharing that is efficient and not a drain on already stretched resources is paramount to ensure peer-to-peer learning and support. Nicole Gusset from SMA Europe put it like this:
Our discussion group all felt it is very much in the interests of patient advocates to be well equipped in the area of early access education since it impacts access to treatment in such a fundamental way. Jane Lyons from Cancer52 explains why engaging in early access education should be a consideration for certain patient groups.
The Patient Advocate Fellowship in early access: closing the education gap
There are a growing number of resources appearing in the early access arena, coming from industry, academic institutions and patient-organisation-led programmes that exist to provide training and upskilling to advocates in many areas of healthcare. It has been great to see these resources developing.
With the Patient Advocate Fellowship in early access, we have worked with patient advocates who have walked the path before to create a scheme which can foster efficient learning about early access. Using live discussion, real-life examples, case histories, networking and ongoing peer support, we aim to bring something dynamic, bite-sized and intimate which can be incorporated into busy advocate roles.
Such a scheme will aim to complement other efforts in this area and will be delivered through:
Alongside a good grounding in early access best practices and ethics, there were two important aspects of such a scheme which came across from the patient group expert inputs we received. The first of these would be creating the forum for sharing experiences, to gain a clear view of the reality rather than just the theory within early access. Lindsay Weaver from Metabolic Support UK voiced this point very clearly.
The second key area of key learning and development identified by the group was the importance of understanding the different perspectives that impact early access from different angles – the pushes, pulls and vested interests. Again, Lindsay had a clear view on the requirements here:
Goals of the Patient Advocate Fellowship in Early Access
Impacting the future
Our hope is that those advocates going through the interactive and engaging fellowship scheme are not only better placed to understand early access, but are empowered to be part of the conversation in their community, now and in the future. In doing so, our aspiration is that EAPs might better achieve what they set out to do, from all perspectives.
Clinigen is committed to delivering meaningful, compliant and ethical Early Access Programmes (EAPs) for patient communities with unmet needs. To this aim, we are working on various initiatives which support the inclusion of the right inputs and perspectives to foster successful EAP development. The patient voice is absolutely central to this. We are therefore thrilled to provide a platform for an initiative such as this. As a service provider with over three decades of experience in running more than 300 Early Access Programmes, we are uniquely placed to bring real-life answers to some of the burning questions that patient advocates have about early access.
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Rare Revolution Editor