|
|
|
FDA Home Page | Search FDA Site | FDA A-Z Index | Contact FDA
|
FOR IMMEDIATE RELEASE |
Media Inquiries: 301-827-6242 |
The Food and Drug Administration (FDA) today proposed new steps to assure the safety and efficacy of certain unapproved, but widely marketed medicines. The proposal aims to make sure that patients can be confident that the products will work as labeled, without creating circumstances in which valuable treatments would not be available for a health problem.
These unapproved drugs include medicines that were developed and marketed before modern standards for drug approval were established. The Food, Drug, and Cosmetic Act generally requires that drugs marketed in the United States be shown to be both safe and effective. Although the unapproved drugs are required by law to demonstrate their effectiveness, these drugs have continued to be prescribed by some health professionals because they are believed by some people to be safe and effective for certain uses. Much of this belief is based on longstanding use or anecdotal reports, not actual scientific evidence.
In some cases, these beliefs may be right: actual scientific evaluation of these unapproved drugs may clearly establish their safety and efficacy. In other cases, when scientific studies are performed, some unapproved drugs may be shown to be ineffective and even dangerous. The quality of these unapproved drugs - especially with regard to formulation -- varies widely. Indeed, FDA has received reports of injuries associated with the use of unapproved products.
For these reasons, FDA has developed a clear policy, in the form of a draft guidance for public comment, to assure that adequate scientific safety and efficacy data are developed for all of these drugs in a manner that does not unnecessarily restrict patient access to useful medicines.
"It’s essential that we learn how to use these drugs safely and effectively, but we must balance that goal against continued patient access to treatments," said FDA Commissioner, Mark B. McClellan, M.D., Ph.D. "Today’s action provides a roadmap for reaching this goal while providing patients and healthcare providers with vital medical information."
The keystone to this effort is a draft Compliance Policy Guide (CPG) the agency is issuing outlining FDA policies to encourage companies to sponsor these drugs through the approval process. The draft guidance also describes for public comment the agency's enforcement approach, giving the highest priority to those products which pose the most likely risk to public health, either because of inherent safety concerns about the drugs, or because there are alternative treatments available that have been shown to be safe and effective in accordance with the law.
The draft guidance reflects the agency's desire to address this issue with policies that are predictable, reasonable, and supportive of the public health. The agency's approach encourages companies to comply with the drug approval process, but it also seeks to safeguard consumer access to important medicines. The draft guidance explains that FDA will continue to give priority to enforcement actions involving unapproved drugs (1) with potential safety risks, (2) that lack evidence of effectiveness, and (3) that constitute health fraud. It also explains how the agency intends to address those situations in which a firm obtains FDA approval to sell a drug that other firms have long been selling without FDA approval.
FDA is emphasizing to the sponsors that many of the potentially beneficial drugs in this category could be approved based on straightforward scientific data that would not involve conducting new clinical studies of safety and effectiveness. By providing adequate scientific evidence of safety and efficacy through other means (e.g., peer-reviewed medical literature, or other existing data) these drugs could be approved with relatively little time and expense.
To deter the continued marketing of unapproved products likely to pose a risk to public health, FDA is proposing to prioritize its enforcement actions on these products. High-priority targets will include:
Extended-release guaifenesin, a drug used to clear mucus in patients with upper respiratory conditions, is an unapproved drug that illustrates how FDA’s policy works. Extended-release drugs are labeled as releasing their active ingredient at a controlled rate. Many contain much more drug than their immediate-release counterparts because they are designed to be taken less frequently. Thus, if it is not designed, formulated, and manufactured properly, it could cause a "dose dump" - releasing too much drug too quickly, potentially compromising patient safety and then failing to provide extended relief of symptoms. Thus, as a general matter, FDA requires extended release products to get approval before marketing to assure safety and efficacy.
On July 12, 2002, FDA approved a new drug application for Mucinex, a single-ingredient guaifenesin 600 milligram extended release drug product, sponsored by Adams Laboratories. Following this approval, the Agency reviewed the marketing status of all strengths of single-ingredient guaifenesin extended release drug products and determined that all such products should no longer be marketed without approved applications. In this case, not only was an extended-release product available that had demonstrated safety and effectiveness; in addition, immediate release guaifenesin will continue to be widely available over-the-counter, as well as other FDA approved treatments such as acetylcysteine, an inhaled product available by prescription used to thin airway phlegm using a nebulizer. Finally, the Agency was aware of evidence that certain extended-release guaiafenesin products were not released in the body at the appropriate rate for an extended-release product, indicating that consumers who purchased the products may not be receiving the benefits claimed.
Consequently, on October 11, 2002, the Agency sent warning letters to 18 manufacturers and 48 repackers and distributors of unapproved single-ingredient guaifenesin extended release products. The warning letters stated that the FD&C Act requires single-ingredient guaifenesin extended release drug products to have an approved application before they can be sold and marketed in the United States.
On February 25, 2003, FDA issued a follow-up letter to the warning letter recipients, again asking the companies to provide the evidence necessary to confirm that their products worked as they claimed, and providing a grace period to allow distribution to continue until October 23, 2003 and sales to continue until the end of November, 2003. As noted above, a straightforward process is available for companies that wish to continue marketing their products beyond this time. As soon as any of these companies submits a drug application containing the limited evidence required, and the application is reviewed and approved by FDA, then that company will be able to resume marketing its product as an approved drug.
FDA is seeking public comment on the draft Compliance Policy Guide over the next 60 days. The draft Compliance Policy Guide is posted at http://www.fda.gov/cder/guidance/5704dft.pdf. Written comments on the draft document may be sent to: Dockets Management Branch, Food and Drug Administration, 5630 Fishers Lane, Room 1061, HFA-305, Rockville, MD 20852.
####