Clinigen educates on early access to rare disease therapies

access to rare disease therapies

On Rare Disease Day, Clinigen has launched a campaign to try to encourage doctors and patients to explore how they can get early access to investigational therapies.

The ‘What is Possible?’ campaign revolves around a new web page that explains how early access programmes (EAPs) can provide a route for some patients with rare and life-threatening diseases to get treated with investigational therapies before they are approved for marketing.

Clinigen has taken the initiative to launch the campaign after its own studies found that only around a third of patients are aware that early access is possible.

The UK company is currently running more than 300 EAPs around the world, covering many rare conditions, including rare cancers, and has drawn on that experience to produce resources that can help “support and educate” patients and clinicians about potential routes to early treatment.

All told, there are over 7,000 rare diseases impacting over 300 million people globally, and yet only 5% have a licensed treatment. Getting enrolled in a clinical trial is one way for patients to access therapies still in testing, but there is no guarantee that a suitable study will be available and places on it may be limited.

That means EAPs – which are known in some countries by other terms, such as managed access or compassionate use programmes – are often the only option available, providing early, but managed and controlled, access for patients who have seriously debilitating or life-threatening diseases and no other approved treatment options.

Explaining the rationale for the campaign, Dr Lorna Pender, global patient engagement lead at Clinigen, said: “Shockingly, it can take five to thirty years to get a diagnosis of a rare disease and, when they finally receive one, patients often struggle to cut through the complex healthcare industry to find a way forward.”

She added: “It shouldn’t depend on where you live and the doctor you happen to see if you can get on an EAP. We want to democratise the process and make EAPs available for more people in need.”

'What is Possible?' has grown out of another Clinigen initiative, called NaviGATE, that was set up to help the rare disease community and patient advocacy groups (PAGs) engage with the pharma industry, clinicians, and other stakeholders in order to participate in R&D for new medicines and support their members in accessing them once they become available.

Patient-representing organisations often serve as the only source of medical information for rare disease patients and their families, but it can be challenging for PAGs to understand and communicate the intricacies and rules of early access to patients, according to the company.

“Empowering people with chronic illnesses and rare diseases to navigate their healthcare journey requires a collaborative effort among diverse stakeholders,” said Carole Scrafton, director of Flutters & Strutters, a PAG representing patients with rare diseases and chronic illnesses that has collaborated with Clinigen in setting up the campaign.

“By engaging patient advocacy groups, healthcare organisations, and professionals in education and training, this is a step towards accessible and personalised care,” she added.

Image adapted from Clker-Free-Vector-Images from Pixabay