Dawn Vasquez has a rare disease that requires medication for only a small population of patients.?
Which act has allowed her drug to be produce even though it is not profitable to the pharmaceutical industry?
- MagsLv 78 years agoFavorite Answer
Well, the Orphan Drug Act of 2002 provides for research into any condition affecting fewer than 200,000 people in the USA. Actually it did not constitute that much of a change from the Orphan Drug Act of 1983 which was designed to do the same thing: encourage research which was too expensive (not profit-worthy) for pharmaceutical companies to otherwise undertake.
"The United States' Orphan Drug Act includes both rare diseases and any non-rare diseases "for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will [be] recovered from sales in the United States of such drug" as orphan diseases"
Once a drug or treatment has been developed, many pharmaceutical companies have Patient Assistance programs in place. I must admit that they do not seem to provide for long term care for those who cannot afford the treatments otherwise, but they are a step in the right direction.
Once the research has progressed to a point where clinical trials are the next step for FDA approval, the expense really escalates. And here is where FDA fast tracking is needed.
And this is where I am confused by the role of the Orphan Drug Act.
So perhaps the next thing to read is from the Institute of Medicine - 2010 - which pretty much tells you that without policy and guidelines, the 2002 Act was primarily lip service or perhaps functioned as a doorman.:
Where more promise lies is in the National Institute of Health: Office of Rare Diseases Research.
There is no act which "allowed" her medication to be produced although there are acts which would have encouraged it or made the research possible as well as policies and programs which would have expedited making it available to the public.
- JoanLv 44 years ago
If God exists then God gives us a certain element of self determination. By that I mean we have to take responsability for our own actions - as human beings. Therefore when an illness comes about and cause alot of pain, suffering and death it is all to easy to blame God and say why has he done this to us. The reality is we have done this to ourselves. Now don't think I am saying that there is no sympathy for people with illnesses, that is not my intention at all. However there comes a point in our lives where we have to stop blaming rxternal sources and look to ourselves and even our own personal God for the solutions. This is not to degrade or usurp God as omnipotent (if that is your belief) but it is to understand that we have a certain element of self determination!