Mifepristone Compassionate Use Program
MIFEPRISTONE: A Medical Breakthrough
Mifepristone's success as a method of early abortion is well known. Less well-known is that because mifepristone blocks the action of progesterone, it also may be able to inhibit the growth of progesterone-dependent tumors. Mifepristone shows tremendous promise as a treatment for a number of serious diseases and conditions that primarily affect women
Anti-Abortion Politics Jeopardize Clinical Trials
Despite mifepristone's very exciting medical promise, anti-abortion politics has interfered with the development of the drug's full potential. In the 1990s mifepristone research came to a virtual standstill because the controversy anti-abortion forces created around its abortion use prevented most U.S. researchers from obtaining supplies of the medication. FDA approval of mifepristone on September 28, 2000 means that not only is the drug now available for early abortion, but also that U.S. mifepristone supplies will enable trials on the medication's other non-abortion uses to move forward at last.
Millions of women have used mifepristone for a safe, effective and early medical abortion. Nonetheless, the Bush administration, in perhaps an attempt to restrict mifepristone or take it off the market, has indicated it may review mifepristone's already proven safety record. Unless Mifepristone remains on the market, there will be no supplies available for U.S. clinical trials.
History of the Compassionate Use Program
Despite the dearth of clinical trials, a few patients have been able to receive mifepristone treatment. Until late 1998, the French firms Roussel Uclaf and then Exelgyn provided mifepristone to seriously ill patients to treat meningioma and other life-threatening illnesses for what is called "compassionate use." Patients could obtain the medication after their physicians received single-patient Investigational New Drug (IND) approval from the FDA when other methods of treatment were not working and mifepristone has shown some clinical efficacy in ameliorating the disease or condition. In late1998, Exelgyn decided that they could no longer administer the compassionate use program for U.S. patients. Neither the Population Council, which holds the U.S. patent rights to mifepristone and the Danco Group, the American company that has been licensed by the Population Council to produce mifepristone for the U.S., also were unable to take on the program. In January 1999, the Feminist Majority Foundation, with two respected physicians serving as co-medical directors, assumed sole responsibility for administering the mifepristone compassionate use program for seriously ill patients in the U.S. Without our intervention, these patients would have no other avenue for obtaining the drug.
FDA Approval and Compassionate Use
The Feminist Majority Foundation expected that after FDA approval of mifepristone physicians would be able to obtain the medication directly through Danco's distribution system and to use it off-label to treat non-abortion indications. However, the conditions of final FDA approval require that physicians intending to administer mifepristone for any use other than early abortion must obtain an IND, even for single-patient treatment. As a result of this unusual requirement, our compassionate use program remains in place as the only way patients can obtain the medication.
How the Compassionate Use Program Works
Physicians apply to the Feminist Majority Foundation for supplies of mifepristone for each patient. They also file an IND application to obtain permission for single patient use of mifepristone from the FDA. Our medical directors review each case and secure physician and patient agreements. The FDA issues an IND number in those cases where patients have exhausted other treatment options and suffer from serious diseases for which mifepristone shows some research promise as a treatment. The Feminist Majority Foundation purchases mifepristone at cost. We then distribute mifepristone at cost to physicians.
How Mifepristone Is Affecting Patient's Lives
Over the past two years, the Feminist Majority Foundation has provided mifepristone to a total of 71patients - 53 women and 18 men. These patients have received mifepristone treatment for meningioma, leiomysarcoma, endometrial cancer, breast cancer, and Cushing's Syndrome. Many of the patients have told us that mifepristone is literally saving their lives and improving their capacity to maintain a good quality of life. Said a college professor whose meningioma has been treated with mifepristone for eight years, "I see this as a matter of life and death. Mifepristone is saving my life and sight. Discontinuing its use can ultimately result in death or blindness. It is enabling me to continue my career as a professor."
In a recent study of 34 ovarian cancer patients whose tumors were resistant to other treatments, 26.5% responded to mifepristone treatment - 3 patients had a complete response and 6 had a partial response.
Studies have found that mifepristone can cause a 50% reduction in the size of uterine fibroid tumors, which are one of the leading causes of the some 600,000 hysterectomies that are performed each year and the most frequent cause of hysterectomies for women between the ages of 35-54.
Other studies have found that mifepristone may be effective in treating endometriosis, endometrial cancer, meningioma (a type of brain tumor), and some types of breast cancer, and in assisting labor induction. New research shows that mifepristone in very low doses is an effective form of emergency contraceptive.
And mifepristone's anti-glucocorticoid action may have profound implications for the treatment of depression, HIV virus, Alzheimer's disease, and other conditions related to elevated levels of cortisol. It is an effective treatment for Cushing's Syndrome, an adrenal disorder.
Please Contact us at 703-522-2214 if you are interested in learning more about the Compassionate Use Program.