Navigate: shaping the future of early access

Helping the rare disease community navigate the world of early access

It is our mission to accelerate access to medicines for people around the world. We work with many of the pharmaceutical and biotech companies who are developing treatment options for rare conditions and help them get these innovative medicines to the patients who need them the most.

Due to the nature of rare conditions, with often no existing treatment options, strict clinical trial criteria, distant location of the trial site, financial pressures, or difficulty finding a trial investigator; people may miss out on participating in a clinical trial, and hence potentially lose out on access to treatment.

That’s where Clinigen comes in. For 35 years we’ve helped our pharma and biotech clients to open up access to medicines via an Early Access to Medicines Program (EAP). We currently have over 300 EAPs running globally, and this track record puts Clinigen in a unique position to address a large unmet educational need for patient groups to advocate on behalf of themselves and others more effectively. We want to empower them to ask – “What is Possible.”

WHAT IS AN EAP?

Early Access Programs (EAPs), also known as Managed Access Programs, offer ethical, compliant, and controlled mechanisms for patients to access investigational medicinal products (IMPs) outside of the clinical trial space and before they are officially marketed. These programs are designed for patients with life-threatening diseases who meet specific criteria:

1. No Treatment Options: Patients who have no available treatment options due to the severity of their condition.
   
   
2. Clinical Trial Ineligibility: Individuals who do not match the entry criteria for clinical trials.
   
   
3. Completed Clinical Trials: Patients who have completed a clinical trial and would benefit from continued access to the investigational product before its formal authorisation.

However, these programs have different terminology in different countries, and it may be that the people in your family, your community, or your patient organization may not be aware of what they are, or how to gain access to them.

That’s why we’ve launched Navigate, a new initiative for 2024 that aims to provide navigational support and tools to help the rare disease community engage with healthcare providers, industry and regulatory bodies. Co-created by Clinigen with the involvement of representatives of the rare community and patient advocacy groups, the overall goal is to support greater participation in rare disease research and development, and to enable increased access to innovative medicines for more patients.

We want to empower patients and patient advocates to support their communities to improve access to much-needed medicines, and give them the confidence to ask “What is Possible.” 

A THIRD OF RARE DISEASE PATIENTS ARE NOT AWARE THAT THEY CAN ACCESS UNLICENSED MEDICINES

The first phase of NaviGATE saw the distribution of a UK-wide survey to identify what patient advocacy groups need from an educational program. The results showed that a third of rare disease patients are not aware that they can access unlicensed medicines, so there’s a clear knowledge gap that we will work to address through the creation of an inclusive, accessible, multi-format training program for the rare community. You can read our white paper and survey results in full here.

We are working with several partners on the program and here's what Carole Scrafton, Director of Flutters & Strutters Patient Advocacy Organisation had to say: “Education and training across multiple stakeholders, including patient advocacy groups, healthcare organisations, and healthcare professionals, is key to ensuring that people living with chronic illness and rare disease are able to navigate to receive the care that they need. I am so pleased to have this opportunity to be involved in co-creating and designing a training program that meets the needs of the rare community."

WITH MANY THANKS TO OUR CO-CREATORS:

Rebecca Stewart, RARE Revolution Magazine
Carole Scrafton, Flutters & Strutters
Michelle Conway, CRD Consulting Ltd