There are only a few hundred known cases of Fibrodysplasia Ossificans Progressiva, or FOP, in the world. On a country-by-country basis, the numbers are miniscule – there are probably only 15 people with it in Canada (I’m personally aware of 11), and I think I read that there are about 285 in the USA. With numbers that small, it’s very difficult for people with FOP and their families to have any kind of impact on governments to get them to pass laws and institute policies to help us.
FOP is definitely a rare, or maybe ultra-rare, disease. There aren’t many people with FOP, but there are many people with rare diseases in general. In fact, 1 in 10 people in Canada (and probably the USA as well) are said to suffer from one of several thousand types of rare diseases. When I think about people I know, a number of examples easily come to mind – I know a boy with neurofibromatosis, another with epidermolysis bullosa, a little girl with Down’s Syndrome, and others. People with rare diseases have many things in common, such as difficulty accessing effective medical treatment (or the complete absence of such treatment), need for assistance with disability related issues, challenges with education and work, and more… If the families of people with rare diseases band together, we have a much better chance to get the help our loved ones need.
For this reason, it’s great that a number of countries around the world will be recognizing February 28, 2009 as “Rare Disease Day”. On that day, or close to it, participating nations will have many various public events to raise awareness of the issues surrounding people with rare diseases. Here in Canada, our Governor General, Ms. Michaelle Jean, has agreed to be the patron of this year’s Rare Disease Day. In recognition of that day, the Canadian Organization for Rare Diseases (CORD) will be hosting some events in Edmonton from March 1 to 3 about the issues faced by people with rare diseases.
In Canada, one very important issue for people with FOP and other rare diseases is our country’s dismal complete lack of an orphan drug policy. An “orphan drug” is a drug which treats a rare disorder, and may or may not have any application to medical issues prevalent in wider society. It’s extremely expensive for drug companies to develop orphan drugs, and without government subsidies, tax breaks or other incentives, such drugs are often not developed. Most western world countries have orphan drug policies. The best such drug policies are those which (a) give the necessary incentives for pharmaceutical companies to develop orphan drugs, and (b) help patients pay for the often exorbitant prices of orphan drugs which do become available. CORD has been working on this issue and trying to persuade our federal government of the urgent need for such a policy. Rare Disease Day 2009 will help in this cause.
People with FOP in Canada need an orphan drug policy! Right now there are no medications specifically targeted at FOP, but maybe with such a policy, researchers in Canada might be more interested in doing the work necessary to design effective drugs. Also, if such a drug should become available, it MUST be affordable for our loved ones – nothing makes me sicker to my stomach than imagining a scenario where there’s a medication developed for FOP, but the cost makes it unaffordable for my daughter Miranda.
Please spread the word about Rare Disease Day 2009, and learn about what’s going on in your own country. For more information, please see www.rarediseaseday.org, as well as www.cord.ca.
