President Clinton Signs Bill Intended to Speed Approval of New Drugs

Posted on November 21, 1997

President Clinton today signed legislation designed to reform the Food and Drug Administration (FDA) drug approval process. In addition to overhauling the drug approval process, the new law also renews the Prescription Drug User Fee Act (PDUFA), a popular law that assesses fees on pharmaceutical companies to hire additional FDA staff in order to more rapidly review their products' safety and efficacy. Since PDUFA first went into effect in 1992, 700 full-time reviewers have been added and approval time for priority drugs has been shortened significantly. Even though PDUFA technically lapsed on October 1, Congress successfully used a continuing resolution appropriations bill to extend the law until the FDA bill is passed.

Key Issues For People With Serious Brain Disorders In The New Law

The legislation signed by President Clinton today impacts all activities of the FDA including approval of medical devices and food safety laws. However, a number of provisions are of particular concern to people with serious brain disorders and their families - especially on the issue of access to new psychiatric medications (e.g., new atypical anti-psychotic and new selective serotonin reupdate inhibitors). With each of the provisions noted below, the newer medications of interest to NAMI members are identified for any special treatment under the FDA review process.

1. Special Access for Consumers: The new law contains provisions expanding the FDA's process of allowing persons with life threatening illnesses to use drugs that the agency has not finished evaluating (cancer patients and persons with AIDS were the main beneficiaries of the old law). Under the new law, anyone with a serious illness would be able to petition a manufacturer for a treatment that is still under FDA consideration. Such a petition will have to be filed by a physician who would certify that the individual has no "comparable or satisfactory alternative" and that the risk from the unapproved drug is not greater than the risk of the disease. The FDA will be able to override the request if it determined that it had insufficient evidence that the treatment was safe or effective or if allowing use of the drug would interfere with clinical investigations. While the new law does not specifically reference drugs treating serious brain disorders such as schizophrenia, bipolar disorder or major depression, such new products are expected to meet this standard. This is especially the case with persons with the most treatment resistant forms of brain disorders such as schizophrenia.

2. Fast Track Approval: The new law would establish a new "fast track" approval system for drugs that "demonstrate the potential to address unmet medical needs" for serious illnesses. Again, new psychiatric medications are not specifically noted in the law as eligible for "fast track" approval. Instead, the initial decision about whether a drug goes through "fast track" is based on an election by the manufacturer. Approval for "fast track" consideration will then made by the Secretary of HHS based on a determination that the drug has an effect on a "surrogate endpoint" that is reasonably likely to predict a clinical benefit. In other words, determinations about which products will go through this expedited approval process will be made on a case-by-case basis once a manufacturer applies for "fast track." The new law also contains a provision designed to streamline clinical research on new drugs.

3. Alternate Uses: The new law allows manufacturers to inform medical professionals, pharmacists, managed care plans and government agencies (e.g. state Medicaid plans) of the uses other than those the FDA has approved for a specific drug. This information could include a peer reviewed article from a medical journal or a reference to a textbook. Before circulating the information, a manufacturer will be required to assure the FDA that it intends to study the effects of the use described in the articles and that within 3 years it will submit a supplemental application for FDA approval to use the drug in that fashion. An exemption from this requirement will be available in cases where an "off-label" use of the drug is within widely recognized "sound medical practice." The FDA will be allowed to stop the manufacturer from disseminating the information if it does not agree with the plans. Currently, companies circulate such literature without prior FDA approval. Information regarding such "off-label" usage is especially relevant in the context of psychiatric medications. For example, the drug depakote was originally prescribed for bipolar disorder through "off-label" use.