“Patient Voice – Join us in making the voice of rare diseases heard.” This is the theme of Rare Disease Day 2016 – and a cause for Actelion to emphasize its long-term commitment to support the needs of the patient community.
ALLSCHWIL, SWITZERLAND – 29 February 2016 – Today, on Rare Disease Day 2016, patients, their families and caregivers, patient organizations, medical professionals, politicians and researchers are coming together to raise awareness for what living with a rare disease means for those affected and for their families.
Patients play a crucial role in bringing both research and the standard of care in rare diseases forward, which is recognized in the theme of this year’s Rare Disease Day, ‘Patient Voice’. For Actelion, listening to patients’ voices and working closely with the rare disease community is key to delivering on the company’s mission to discover new innovative drugs for rare diseases and to help more patients with groundbreaking therapies. At the end of 2015, over 65,000 patients were benefiting from Actelion’s rare disease drugs, and the company continues to work with governments and other stakeholders to widen accessibility around the world.
On February 23 in Brussels, our commitment to rare diseases was publicly recognized by the European Organization for Rare Diseases (EURORDIS) as Actelion was honored with the EURORDIS Company Award 2016. Presented to pioneering companies for the development of treatments for rare diseases, the award in particular acknowledges Actelion’s work with patient groups to support the patient and carer community.
Through the company’s collaborative work with patient groups and healthcare professionals, Actelion has become a trusted partner in supporting activities that raise awareness of the challenges facing those living with rare diseases and help bring about positive change. Activities include the development of educational resources such as the innovative PAHuman® eBook to better explain the disease and its impact, and the “Think Again. Think NP-C” campaign to encourage earlier diagnosis of Niemann-Pick type C disease among healthcare professionals unfamiliar with this rare condition.
Gerald Fischer, CEO of the European Pulmonary Hypertension Association (PHA Europe), said: “Actelion has provided invaluable support to our activities and, as a result, our collaboration has led to many great achievements over the years. It’s clear that Actelion’s commitment extends beyond the discovery and development of new medicines and includes initiatives that increase knowledge and understanding of PAH and other rare diseases, which is crucial to organizations like ours.”
An example of Actelion’s pioneering work in rare diseases is an ongoing clinical study involving patients with Eisenmenger syndrome. Working with ethics committees, patient advocacy, support groups and patients’ families, Actelion has extended the study on Eisenmenger syndrome to Down syndrome patients, which will help to address the high unmet medical need for effective, targeted PAH therapies in this vulnerable and too rarely considered population.
Martine Clozel, MD, Chief Scientific Officer at Actelion, commented: “I am very proud that, together with the PAH community, Actelion has brought about not one, but several paradigm shifts in PAH care. The three established pathways in PAH are now well served, but our discovery efforts continue. We are turning to new pathways in PAH, helped by our strong connections with academia, to further improve the prognosis for patients living with this devastating disease. Actelion also plays a leadership role in Niemann-Pick type C disease, where the lives of many patients have been positively impacted. I am convinced that with our strong and innovative research, Actelion will continue to advance care in many rare diseases.”