Early Access Patient Advocacy Insights 2019: What did melanoma patient advocates care most about having a say on?

Engaging with patient groups about Early Access is one of the biggest privileges of working within patient advocacy at the leading and most experienced provider in this space. The year of 2019 has presented plenty of opportunity to do just that.

As a patient-focussed organisation, we welcome unique moments like this, in order to hear what really matters to patient groups and understand what the most pressing challenges seem to be when it comes to gaining early access to medicines.

Clinigen’s patient advocacy team were invited in March 2019 to speak with 130+ patients and advocates in melanoma and mid-2019 to engage with a pan-EU group of Oncology Patient Advocates as part of the WECAN Masterclass. Despite cancer being a disease area that has seen some very significant Early Access programs over the past 5 years, it seems that for a large number of patient advocates and patients attending, this was still a first exposure to the fact that regulated routes do exist to allow access to an unlicensed medicine at the request of a physician, for a patient with an unmet medical need – an approach collectively known as “Early Access”. This highlights that even as a patient advocate, until it has become your issue, you are not always aware that this is an issue to be aware of. And by the time the topic has come right into your corner, it is already urgent and it may be too late to up-skill and develop the requisite knowledge which could open the door to being part of the conversation about Early Access with the company in question.

Much credit is due to MPNE, the Melanoma Patient Network Europe, for identifying this factor as of importance and seeking to educate its members on the topic. Clinigen’s Patient Advocacy team was asked to be part of a more in-depth educational session about Early Access as part of the annual MPNEHubs event in Krusenberg, Sweden. We ventured into an exposition of the necessary decision-making on the part of industry regarding Early Access Program design, and the ethical considerations associated with those decisions. The discussion also took a philosophical direction in looking at the wider place of Early Access/Compassionate Use within our society, and the ethical considerations associated with Early Access Program design.

This latest meeting also led to noteworthy insights, generated through a spontaneous survey with some of the gathered attendees. Clearly, there are multiple areas where patient groups would want to input, but when asked which one area of Early Access decision-making they were most keen to be involved with, 50% of advocates within melanoma stated that development of the eligibility criteria was the number one area where they wanted to see the patient view incorporated, with increased engagement and wider inputs into Early Access program planning.

This raises a few immediate questions. If you are working within industry, how do you go about designing the eligibility criteria for your Early Access Program? If it’s less than straight forward and threatens to become a stumbling block, have you considered engaging with an external ethics advisory group on this topic? Critically, how are patient groups and patient inputs being considered in those discussions? How can a company make the best decision that is available to them, when the stakes are so high on all sides? It seems that patient advocates would often agree it’s better to have a “program with limitations” than no program at all. With that in mind, if the relevant medical, patient group and ethical inputs have been sought, it follows that the company may be more able to settle on where the inevitable boundaries should lie for their program, and thereby keep it viable.

As part of its Unlicensed Medicines division, this is an area where Clinigen has taken significant strides forward during 2019. To hear more about how Clinigen is helping its partners to gain an independent external view on their access program, including the relevant inputs, please sign up to attend Clinigen’s Insights meeting in 2020.