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
Scientists at Alexion are working relentlessly to understand the
underlying causes of these diseases and to discover and develop
breakthrough medicines to treat them. To learn more about Alexion’s
Research & Development programs, visit www.alexionpharma.com/pipeline.
To learn more about Rare Disease Day, visit www.rarediseaseday.us
for U.S. activities and www.rarediseaseday.org
for global activities.
About Rare and Ultra-Rare Disorders
In the United States, a disease is defined as rare if it affects fewer
than 650 patients per million of population.3 The European
Union definition of a rare disease is one that affects fewer than five
patients per 10,000 of population.4 In contrast, a disease is
generally considered to be ultra-rare if it affects fewer than 20
patients per million of population5 (one patient per 50,000)
– and most ultra-rare diseases affect far fewer people than this.
Despite the very small numbers of patients they affect, the impact of
these rare and ultra-rare diseases on patients, their families, and
society is profound, as many of these conditions are severe, chronic and
progressive, with significant premature mortality. Patients with severe
and life-threatening ultra-rare diseases often live without hope, have
no effective treatment options and may face premature death.
About Alexion
Alexion 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.
Copyright Business Wire 2014