In 2008, on February 29th, a day with a rare calendar occurrence of only once in every four years, EURODIS (European Organization for Rare Diseases) organized and established the first World Rare Disease Day. The European Organization for Rare Diseases, EURODIS, is a patient-driven alliance of patient organizations and individuals active in the field of rare diseases. A rare disease, also referred to as an orphan disease, is any disease that affects a small percentage of the population.
Since 2008, rare disease patient groups and national disease alliances from many different countries have partnered with EURODIS to raise public awareness of rare diseases on World Rare Disease Day. On February 28th of this year, for the first time ever, Dubai joined international rare disease alliances to observe World Rare Disease Day under the slogan "Rare but Equal.” Dubai represents the first country in the Middle East to celebrate World Rare Disease Day.
In the US, SenaCare, an international personalized care organization with company representation also in the Middle East and West Africa, has developed an expert physician panel which is further supported via the company’s medical travel and telemedical services. For patients and families afflicted by rare diseases, SenaCare offers American Board Certified expert panel diagnostic services and recommendations on where the patient can receive the best care for his/her individual ailment. The mission of World Rare Disease Day 2011 was to create greater public awareness about the healthcare inequalities existing in the area of rare diseases and the subsequent dire need for an increase in rare disease medical research, the number of rare disease experts, and the availability of educational resources and emotional support for those afflicted by rare diseases.
As we turn from one day to the next, February 28th, World Rare Disease Day has rolled into another; however for an approximate 250 million people worldwide, suffering from one of the estimated 7000 known rare diseases, the continued focused efforts on issues addressed during World Rare Disease Day is highly important. Overall, approximately 80% of rare diseases are genetic and nearly half of the rare disease patient population is children. According to Orphanet, approximately five new rare diseases are described each week in medical literature.
Over the years, advanced scientific research and clinical trials have succeeded in reducing the number of some common diseases and therefore, led to their transformation into rare diseases. On the flipside, the failure to control the spread of some foreknown rare diseases, such as AIDS, has contributed to their evolution into common diseases. Additionally, a larger employment of global statistical tools has enabled researchers to more accurately track the incidence and prevalence of diseases, resulting in the reclassification of some rare diseases to common, (e.g. Celiac Disease).
Many rare diseases are chronic, life-threatening, and very serious. As stated best by NORD (National Organization for Rare Diseases) “Most of us want a cure for our disease or a disease that may be affecting someone we love. Many of us would be happy just to settle for a safe and effective treatment, such as insulin for diabetes. However, neither cures nor treatments will become available without medical research.”
Although there is still considerable need for medical research and development, through the years, rare disease treatment options and awareness have improved tremendously. Since the US government passed the Orphan Drug Act in 1983, offering tax incentives for clinical trials of orphan products and a 7-year exclusive right to market any product designed to treat a rare disease, nearly 350 drugs have been approved for rare diseases. Recently, in the US Pharmaceutical Research and Manufacturers of America (PhRMA) reported that currently, a record 460 medicines for rare disease are in the late stages of the pipeline, either in clinical trials or awaiting Food and Drug Administration (FDA) approval.
Moreover, major modern scientific and technological developments have enabled researchers and doctors to better understand the complex epidemiology and pathology of rare diseases. On rare disease day in the UAE, parallel to hosting the region’s first Genetic Metabolic Conference, the government launched a series of initiatives to foster dialogue among rare disease patients and communities, provide patients with greater educational resources, and improve healthcare options through increased research. These movements are extremely valuable to those who suffer from genetic rare diseases, as according to Dubai-based Center for Arab Genomic Studies, the Arab region has the highest rate of genetic diseases in the world.
Despite all of the progress, statistics still show 30 percent of children with rare diseases will die before reaching their fifth birthdays (SIOPE). Many rare disease communities continue to depend on one another for support, attempting to create public awareness via online videos and independently fundraising to further research. Unfortunately, the sentiment that reverberates among many rare disease groups is one of the same, “The numbers of people affected by individual categories of rare diseases are not high enough for the medical community to allocate greater resources to the needs of these patients” Nevertheless, although individually rare disease group numbers may be smaller than those of common, it is difficult for one to argue that a six year old child suffering from Niemann Pick Type-C sometimes referred to as "Childhood Alzheimer's” is not entitled to the same quality and access to healthcare as other patients. http://www.cbsnews.com/video/watch/?id=4836925n
Moving forward, the trend toward personalized care is anticipated to greatly improve the healthcare environment for rare diseases. Already through biological analysis, more and more physicians are able to diagnose and target diseases. Additionally, physician care via telemedicine now puts the experts within the reach of patients. As stated by Dr. Wehbe of SenaCare, “Telemedicine empowers patients. For patients suffering from a rare disease, telemedicine is an incredible solution for long-term monitoring, as well as end stage care for a patient. Adding to that, patients with rare diseases will increasingly benefit from the access to expert opinions via telemedicine in order to find the best care for their ailment, tailored to individual medical history, culture, and lifestyle. The landscape of healthcare is changing everyday with the aim to better accommodate the needs of each patient; this is leading to major improvements in the care for each patient suffering from a rare disease”
For more information please contact:
Cassandra Cipoletti, Managing Director of US Operations
Company name: SenaCare
Physical/postal address: 2734 West Country Club Rd, Philadelphia, PA, 19131, USA