The theme of Rare Disease Day 2016, “Patient Voice — Join us in making the voice of rare diseases heard,” was a major cause for Switzerland-based Actelion, a leader in the field of pulmonary arterial hypertension (PAH). It is also an opportunity for the company to highlight its long-term commitment to support the needs of patients.
On Rare Disease Day 2016, which was Feb. 29, patients, families and caregivers, patient associations, health professionals, politicians and researchers joined forces to raise awareness for what it means to live with a rare disease.
For Actelion, listening to patients’ voices and working closely with the rare disease community is crucial to the company’s mission to discover groundbreaking drugs for rare diseases and to help more patients. At the end of 2015, more than 65,000 patients were using Actelion’s drugs for the treatment of a rare disease. The company is continually working with governments and other stakeholders to expand the accessibility to their drugs worldwide.
On Feb. 23, the European Organization for Rare Diseases (EURORDIS) publicly recognized Actelion’s commitment to rare diseases and honored the company with the EURORDIS Company Award 2016. The EURORDIS awards acknowledge the exceptional contributions that patient advocacy groups, volunteers, companies, and policy-makers make to reduce the impact of rare diseases on people’s lives.
This year’s award has acknowledged Actelion’s collaborative work with patient groups to support patients and healthcare professionals.
Actelion is an active partner in raising awareness and promoting activities for those living with rare diseases. The company has developed educational resources such as the innovative PAHuman eBook, dedicated to PAH, and the “Think Again. Think NP-C,” a campaign focused on encouraging earlier diagnosis of Niemann-Pick type C disease among the medical community.
“Actelion has provided invaluable support to our activities and, as a result, our collaboration has led to many great achievements over the years,” Gerald Fischer, CEO of the European Pulmonary Hypertension Association, said in a press release. “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.”
Actelion is currently conducting an ongoing clinical trial in patients with the rare Eisenmenger syndrome. The company has been working with ethics committees, support groups and families, and has extended the trial on Eisenmenger syndrome to patients with Down syndrome to address the unmet medical need for targeted PAH therapies in this vulnerable population.
“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,” Martine Clozel, M.D., chief scientific officer at Actelion, said.
“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,” Clozel said.
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