Orphan drugs are those that the federal government approves for use in the treatment of rare diseases that affect less than 200,000 people. New medications that treat rare forms of cancer or rare cancer-related symptoms are hitting the market all the time, despite their lack of clinical trials, a study published by Harvard researchers in 2011 in the Journal of the American Medical Association found.
The Orphan Drug Act of 1983
In 1983, the Orphan Drug Act was passed in order to offer incentives to drug manufactures so that new treatments for rare diseases would be developed. The act says that a company that develops a new drug for a rare disease will have exclusive rights to sell it for seven years.
A company has to invest a lot of money in the development of a drug for a tiny target market. The Orphan Drug Act addresses this and attempts to create a win-win situation for both manufactures and consumers.
Approximately 25 million Americans suffer from a rare disease, reports the study authors. There are about 6,000 known rare diseases, some affecting as few as a dozen people. Through 2010, the FDA has given roughly 350 drugs organ drug status.
The Controversy behind the Orphan Drug Act
The Orphan Drug Act has allowed new drugs to be developed for rare diseases that might not have been created had it not been for the these legal incentives.
However, there are a few problems that have arisen because of the act, namely:
- High prices – Because manufactures have a legal loophole to a monopoly, they can essentially charge whatever they want for orphan drugs. For instance the cost of treatment for certain enzyme replacement therapies can cost an upward of $150,000/year, according to a 2010 report in the Journal of Pharmacy and Bioallied Sciences. This makes affordability a huge factor for patients in need.
- Poor product testing – For regular drugs, manufacturers may test thousands of test subjects before bringing the drug to market. This isn’t the case with orphan drugs. Some orphan drug trials for cancer drugs only tested a mere 96 patients, according to the Harvard study, though this is largely attributed to the rarity of the disorders.
Changing the Orphan Drug Act
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Some have recommended changes to the Orphan Drug Act amended to promote fair market trade and safer medications for those with rare diseases. Dr. Aaron Kesselheim, a researcher on the Harvard study, notes that a “clawback provision” requiring follow-up studies after the drug is released to the market may be helpful, according to a Reuters report at the time the study was released.
He also notes that there were certain orphan drugs approved for rare diseases for which there were already therapies approved. He mentions offering the financial incentive only for very rare diseases for which no other treatment is available, according to the Reuters report.
For Dangerous Drug Claims, Contact Chalik & Chalik
If you or a family member was injured by an orphan drug, you might be entitled to compensation. We invite you to call our injury lawyers at Chalik & Chalik to go over your claim. Contact us at 855-529-0269 for a free legal consultation today.
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