Cancer Drugs Allowed to Hit U.S. Market after Subpar Testing

Medications that are used to treat diseases that affect a relatively small number of people and subsequently generate limited revenue for manufacturers are known as orphan drugs. The government has been offering tax benefits, research grants and market rights to pharmaceutical companies as incentives to develop these orphan drugs, in accordance with the Orphan Drug Act of 1983.

However, recent scandals have raised some serious concerns regarding the safety of using these orphan drugs. For example, the drug Mylotag, a product of Pfizer, is an orphan drug that was approved by the FDA in 2000 for use in the treatment of a special type of leukemia. Alarmingly, the drug was ultimately proved to be completely ineffective and even killed patients.

Following mounting concerns over the quality of clinical testing for orphan drugs, researchers conducted a study of FDA data from 2004 – 2010, comparing the testing data of orphan drugs to non-orphan drugs. The study revealed a significant difference in the level of clinical testing between the two groups, noting that less-rigorous testing methods were used to study the orphan drugs.

The issue of allowing orphan drugs to be approved for use in the U.S. market based on low-quality clinical testing is obviously cause for increased concern. However, it does not mean that all orphan drugs are unsafe, dangerous or shouldn’t have been approved. Completing large clinical trials for the treatment of very rare diseases may not be completely feasible. Regardless, it is clear that regulators should revisit and possibly revise the Orphan Drug Act, as well as the incentives offered to pharmaceutical companies for drug development.

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