An orphan disease is one which is very rare, defined by the FDA as a condition that affects fewer than 200,000 people in the United States, and therefore there is minimal funding dedicated toward finding a cure. Sufferers may often feel that they have been left alone in the depths of hell. Most of these diseases are caused by genetic mutations, and are thus identifiable from a young age. However, getting an accurate diagnosis can sometimes take years, because of the rarity of the condition. In addition, sufferers may be isolated from one another, and never know that someone else shares their disease.
The importance of helping this group of patients cannot be overstated. What is to be done when someone you know or love suffers from a disease so rare that many people don't even understand what it is? Here's what you can do-- try and create awareness about it, so that combined efforts can by gathered to combat and counter it. This is not as difficult as it may sound; today's world is thoroughly inter-connected through the internet, where numerous social networks, support groups and relevant associations can be found. It is wise, however, to rely solely on social networks for medical advice; unproven remedies and miracle procedures are constantly being promoted as the ultimate cure. Most important of all, one person's positive experience does not necessarily promise the same result for others. The internet and social networks should be used to complement the exchange of information between doctor and patient, not to replace it.
In the United States and the European Union, the term "orphan diseases" has a distinct legal meaning. The European Organization for Rare Diseases (EURORDIS) has put much effort into studying and estimating these distinct rare diseases. Organizations such as the Rare Genomics Institute and the Global Genes Project research prevailing therapies and bring together medical, scientific and business professionals from various fields all over the world to promote awareness and patient advocacy. In light of the work being carried out by various organizations in different parts of the world, each region must make an effort on its own to raise awareness of rare orphan diseases and further research toward finding appropriate treatment options.
In the face of rare diseases, the pharmaceutical industry also has a major role to play. It must provide a financial incentive for the private sector to encourage research aimed at preventing and countering these diseases, ultimately resulting in new medications on the market. . According to the NIH, as many as 25 million people suffer from these orphan diseases in the US alone. In 1983, the US congress passed the Orphan Drug Act (ODA), which created financial incentives for drug manufacturers to work toward this end. Since then, the ODA has overseen the development of more than 250 orphan drugs, which are now available to treat the orphan disease population. Governments can also provide the infrastructure necessary for the research facilities or subsidies needed to promote the cause
In a world which is evolving every day, constantly bringing new challenges in the form of rare illnesses, efforts must be made to support sustained research. It is the duty of all of us, as members of the human race, to battle issues which could be faced by anyone. There is evidence that the approximately 6,000 known orphan diseases (as well as new ones which are discovered every year) are receiving increased attention from governments, patient groups and the pharmaceutical industry. Identifying and finding treatments for them has finally been recognized as a serious public health concern.