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Drugs to Treat Mesothelioma and Other Rare Diseases May Not Be Tested as Rigorously as Non-Orphan Drugs

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Wednesday, June 15, 2011

Mesothelioma is a unique and painful form of cancer, typically affecting the lining of the lungs, caused by exposure to asbestos fibers.

Also called “asbestos cancer,” mesothelioma is diagnosed in just 3,000 Americans each year. Due to the small number of cases, the Food and Drug Administration (FDA) has designated the disease as an “orphan disease.”

In the United States an orphan disease status is assigned to a disease or disorder if it affects fewer than 200,000 Americans at any given time. Like mesothelioma, many of these rare illnesses including fibromyalgia and Hepatitis B and C, afflict so few people that researchers and pharmaceutical companies do not find it beneficial to expend the time, effort or money to find treatments and cures. However, the FDA offers millions of dollars in grants each year per the U.S. Orphan Drug Act providing financial incentives to bring biological products for rare diseases to market.

Now researchers have found that some of the clinical trials that are conducted to determine the safety and efficacy of drugs for the treatment of rare diseases may be of “lower quality” than non-orphan drugs.

In a recent study published in the Journal of the American Medical Association, researchers at Brigham and Women’s Hospital and Harvard Medical School reviewed FDA data from 2004 to 2010 to assess the supporting evidence behind new cancer medications with an orphan drug designation. 15 orphan and 12 non-orphan drugs approved during the time period were analyzed. The researchers found that clinical trials for recently approved orphan drugs for cancer “were more likely to be smaller and to use nonrandomized, unblinded trial designs and surrogate end points to assess efficacy” than their non-orphan counterparts.

They also found that trials of orphan drugs had smaller participant numbers and more patients had serious adverse events than non-orphan trials. However, according to Fox News, Dr. Aaron S. Kesselheim, one of the researchers, said the findings do not necessarily indicate that orphan drugs are unsafe or should not have been approved. Although, he added, that “amending the law might be necessary to achieve more credible data and save ourselves unpleasant surprises.”

Last year Pfizer voluntarily withdrew the drug Mylotarg used for treating patients with acute myeloid leukemia (AML), a bone marrow cancer. The drug was approved in May 2000 under the FDA’s accelerated approval program, but subsequent trials raised concerns about the product’s safety, and the drug failed to demonstrate clinical benefit to patients enrolled in trials. In addition, a greater number of deaths occurred in the group of patients who received Mylotarg compared with those receiving chemotherapy alone.

For the thousands of patients diagnosed in the United States each year with mesothelioma and other rare cancers, clinical trials and ongoing research is the key to finding the breakthrough that can mean the difference between life and death. Alimta, distributed by Eli Lilly and Company, received a priority review from the FDA in 2004 and is also designated as an orphan drug. It is the first, and only, drug approved for mesothelioma treatment.


Copyright 2011

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