Just as an aside, I wouldn't have even known about the Creating Hope Act if it weren't for someone posting about it on the Wegener's forums I watch. A big thanks to the original poster! I hope I did a decent job summarizing the article.
The Orphan Drug Act was signed into law by President Ronald Reagan on January 4, 1983. This original law completed its purpose of helping R & D which in turn helped create treatments whose only focus was on rare diseases, but much more needs to be done. In the United States alone over 25 million Americans suffer from one of the 7,000 + "orphan diseases". An orphan disease is a disease that has not been a focus of pharmaceutical companies because there is a lack of financial pressure to do so. In other words, the pharmaceutical companies would not receive as much profit from these research into these diseases because of their rarity or they are diseases more prevalent in developing nations. This makes it very hard for a person with a rare disease to get a diagnosis, let alone treatment. When you consider that many of these diseases, when they are active, can cause death or cripple a person in a matter of months and it takes YEARS for some of them to get diagnosed (I was lucky in that it only took me about a year to get a firm diagnosis, for many people with Wegner's they can go decades if they don't have a fatal flair before someone figures out what is going on.) something certainly needs to be done. The Orphan Drug Act offered these companies tax incentives for for doing clinical trials and 7 years of exclusive marketing. More than 25 years later, only 2,400 drugs have been given the orphan designation and out of those only about 370 have actually been approved for use by the FDA. Recently, though, new laws are being proposed by NIH (the National Institute of Health which is also trying to bring about an Undiagnosed Disease Program) and the FDA that would help unite patient advocates, researchers and companies that want to develop new therapies. One such law is the Creating Hope Act, Senate Bill 606, which would enable drug developers to receive a "priority review voucher" which a company could sell or transfer multiple times if they submit an orphan drug for review. One problem here is the only action that has taken place on this bill sponsored by three democrats and two republicans was on May 31, 2011 where the bill was sent on for review to the Committee on Health, Education, Labor, and Pensions. No one is sure about where prospects for this bill currently stand due to lack of support. This is not the only bill that is hoping to improve the futures for people with rare diseases and there has been some connections made with European counterparts to the FDA and patient advocacy groups in the hopes that we can begin to trade information and decrease some of the processes that are duplicated between each country when it comes to bringing drugs to the market (look how long Rituximab/Rituxan has been available for treatment of vasculitis in Europe verses it only being approved for us in the United States a few weeks ago).
The Creating Hope Act and the re-establishment of the Congressional Rare Disease Caucus shows a lot more activity from advocates groups, but they can only go so far. It is up to the patients with rare diseases, their families and friends to write to their representatives asking them to join the coalition in support of these bills. Finding your representative can be done through this link: http://whoismyrepresentative.com/
Further Readings:
Honestly, I've tried to fix the formatting at least six times now and it just won't do it. Rest assured, I am capable of inserting paragraph breaks, just not in this post I guess.
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