Clinigen launches ‘What is Possible?’ campaign to accelerate access to medicines on Rare Disease Day

Clinigen launches ‘What is Possible?’ campaign to accelerate access to medicines on  

Rare Disease Day 

With over 7,000 rare diseases that impact over 300 million people globally, just 5% of rare diseases have a licensed treatment* 

LONDON, UK, 29 February 2024 – Clinigen, the global pharmaceutical services company, today launched a new campaign called ‘What is Possible?’ to help empower patients and medical professionals to ask what more is possible in the treatment of rare diseases. There are over 7,000 rare diseases impacting over 300 million people globally, and yet only 5% have a licensed treatment. The campaign, complete with a newly launched webpage, aims to support and educate both patients and clinicians about potential routes and solutions for rare disease treatment, through its Early Access Programs. 

Early Access Programs** (EAPs) can give people with life threatening or debilitating conditions early controlled access to unlicensed medicines that are still in development, giving fresh hope to thousands of patients. It is an area of medicine that is not widely known or understood; in fact, according to a Clinigen study, just over a third (32%) of patients are aware that they can access these EAPs. The ‘What is Possible?’ microsite and campaign have been developed to highlight to both healthcare practitioners and patients that they/their patient could be eligible for an EAP and gain access to an unlicensed medication or a clinical trial.  

Clinigen is a world leader in this space; with 38% of its programs focused on rare diseases, the company is currently running approximately 300+ active Early Access Programs globally. To date, Clinigen has supplied over 17,000 patients in the UK with an unlicensed medicine through an access program; and with 365 UK hospitals/healthcare institutes and over 2,000 UK physicians already registered, many patients are receiving access to these EAPs. But many are not. There is inequity in access and knowledge across the UK, and Clinigen wants to change this with the launch of this new initiative. 

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. Patients and doctors need to be backed by knowledge and resources, and knowing how to navigate their way to early access to medicines. That’s why we have launched the ‘What is Possible?’ campaign. 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.” 

Carole Scrafton, Director of Flutters & Strutters Patient Advocacy Organisation said: “Empowering people with chronic illnesses and rare diseases to navigate their healthcare journey requires a collaborative effort among diverse stakeholders. By engaging patient advocacy groups, healthcare organisations, and professionals in education and training, this is a step towards accessible and personalised care. I’m looking forward to further collaborating with Clinigen to co-create and design a training program that meets the needs of the rare disease community.” 

*Source Rare Revolution article https://www.clinigengroup.com/media/2655/rare-revolution-and-clinigen-early-access-spotlight.pdf 

**Different countries have different definitions for Early Access Programmes, for example Expanded Access, Compassionate Use. For more information on Clinigen’s ‘What is Possible?’ campaign visit:  clinigengroup.com/what-is-possible/patients/ 

Notes to Editors 

Why are medicines unlicensed? 

Medicines are unlicensed when they are either still under investigation in research, or they are in the period between the trial ending, and they haven’t been given regulatory approval yet. Early access to medicines programmes involving unlicensed medicines offer a route for access to treatment for those with critical unmet medical needs. These medicines can be vital for patients with rare diseases. 

Contact details 

C: Michelle Shearly, Director of Communications, Clinigen  

E: michelle.shearly@clinigengroup.com 

C: Tali Kramer, Senior Director, Fight or Flight     

E: Tali@fightorflight.com 

About Clinigen 

Clinigen is a global, specialist pharmaceutical services company focused on providing ethical access to medicines. Its mission is to accelerate access to medicines for patients​ in every corner of the globe. The Group supports pharmaceutical and biotech companies across the medical product lifecycle, from clinical through to commercial and operates from sites in North America, Europe, Africa and the Asia Pacific. Clinigen has more than 1,000 employees across five continents in 15 countries and provides access in more than 130 countries every year. 

For more information on Clinigen, please visit http://www.clinigengroup.com.