Alexion Pharmaceuticals (Nasdaq:ALXN) joins the European Organization for Rare Diseases (EURORDIS), the National Organization for Rare Disorders (NORD) and patient organizations worldwide in recognizing Rare Disease Day 2014, a global effort to increase awareness of rare diseases, their profound impact on patients and the need for improved diagnosis and treatment. Alexion supports key objectives of EURORDIS, NORD and other patient organizations through its mission to develop and deliver life-transforming therapies for patients worldwide who suffer from severe and life-threatening rare diseases. Diagnosis and Treatment Challenges for Rare and Ultra-Rare Diseases Many rare and ultra-rare diseases are chronic, progressive and marked by continuing pain, severe disability and high mortality. Few physicians are familiar with diagnosing and treating these illnesses, which frequently leads to missed, delayed or inaccurate diagnoses even when an approved, effective therapy is available. 1 According to global survey results published in the current issue of The Journal of Rare Disorders (JRD), conducted on behalf of Global Genes, patients living with rare diseases visited an average of 7.3 physicians before receiving an accurate diagnosis. 2 The survey also reveals that 44 percent of patients surveyed believed that a slow diagnosis resulted in delayed treatment with a negative impact on their condition. 2 “Patients with rare and ultra-rare life-threatening disorders often face long delays in receiving an accurate diagnosis. Without an accurate diagnosis, or access to effective treatment options, patients too often are at risk for catastrophic medical consequences,” said Leonard Bell, M.D., Chief Executive Officer of Alexion. “On this Rare Disease Day and every day, through our continued commitment to breakthrough medical research, each of us at Alexion are focused on improving the knowledge of rare diseases and developing and delivering therapies to transform the lives of these patients worldwide.” Delivering Life-Transforming Therapies Across the Globe Alexion’s research and development programs are focused on highly innovative therapies with the potential to transform the lives of patients with severe and life-threatening ultra-rare disorders and for which there are no effective treatment options. The company’s development programs are solely focused on:
- Severe disorders with devastating and life-threatening medical consequences
- Disorders with ineffective, or no treatment options
- Disorders that are ultra-rare and affect very small numbers of patients
About AlexionAlexion is a biopharmaceutical company focused on serving patients with severe and rare disorders through the innovation, development and commercialization of life-transforming therapeutic products. Alexion is the global leader in complement inhibition and has developed and markets a treatment for patients with PNH and aHUS, two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation. This press release and further information about Alexion can be found at: www.alexionpharma.com. [ALXN-G] References 1. EURORDIS. The Voice of 12,000 Patients: Experiences and Expectations of Rare Disease Patients on Diagnosis and Care in Europe. 2009. http://www.eurordis.org/IMG/pdf/voice_12000_patients/EURORDISCARE_FULLBOOKr.pdf 2. Engel PA, Gabal S, Broback M, Boice N. Physician and patient perceptions regarding physician training in rare diseases: the need for stronger educational initiatives for physicians. J Rare Dis. 2013; 1(2):1-15. 3. U.S. Food and Drug Administration. Definition of Disease Prevalence for Therapies Qualifying Under Orphan Drug Act: http://www.fda.gov/ForConsumers/ConsumerUpdates/ucm135130.htm 4. Definition from REGULATION (EC) No 141/2000 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 16 December 1999 on orphan medicinal products and from DIRECTIVE 2011/24/EU OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 9 March 2011 on the application of patients’ rights in cross-border healthcare. 5. UK National Institute for Clinical Effectiveness (NICE). Citizen Council Report on Ultra-Orphan Drugs. 2004; 27-28. Available at http://www.nice.org.uk/niceMedia/pdf/Citizens_Council_Ultraorphan.pdf.