Mesothelioma Researchers and Oncology Pharmaceutical Companies Invited to Attend Upcoming FDA Orphan Drug Workshop
Mesothelioma is a unique and rare form of cancer, typically affecting the lining of the lungs, caused by exposure to asbestos fibers. The cancer is highly aggressive and is resistant to many standard cancer treatments making it a difficult disease to treat effectively. Mesothelioma is diagnosed in 3,000 Americans each year, and due to the small number of cases diagnosed, 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. While there is ongoing research to find new treatments or a cure for these orphan diseases, research requires significant amounts of time and money. Since many of these illnesses afflict so few people, researchers and pharmaceutical companies do not find it beneficial to expend the time, effort or money to find treatments and cures.
Through the FDA, however, a pharmaceutical company developing a drug to treat an orphan disease can be granted with an orphan designation. The pharmaceutical company that developed the drug is qualified to receive certain benefits, such as tax credits and marketing incentives, from the federal government in exchange for developing the drug. To qualify for orphan designation the proposed drug must be a treatment for a rare disease and there must be a medical rationale for believing that the proposed drug has “promise” for treating the rare disease or condition. The application process can be intimidating for many in the drug industry.
To help those unfamiliar with the application process and to speed the time of getting treatment options for orphan disease patients to market, the FDA is offering an orphan drug designation workshop. The workshop is for academics and biotechnology companies, and will be held in Claremont, California on Feb. 28 – March 1, 2011, in collaboration with Keck Graduate Institute.
Alimta (pemetrexed), a chemotherapy drug used in combination with cisplatin for treating pleural mesothelioma, distributed by Eli Lilly and Company, received a priority review from the FDA in 2004 and is designated as an orphan drug. It is the first, and only, drug approved for mesothelioma treatment.
According to the FDA’s information for the workshop, participants will propose a specific drug for a specific rare disease and work on an orphan designation application to submit to the FDA at the conclusion of the workshop. FDA staff will provide one-on-one guidance to help participants develop strong applications. Designation requires there be a scientific rationale for expecting the proposed drug to be effective in the treatment, prevention, or diagnosis of that disease or condition.
The FDA estimates that today there are over 6,000 rare diseases affecting more than 25 million people.