April 14, 1999

Dear Colleague:

Your letter is one of many received recently by the Food and Drug Administration (FDA) requesting approval of the drug temozolomide for the treatment of adult patients with malignant glioma at first relapse. Additionally, issues regarding advisory committees, the drug review process, reimbursement for pharmaceuticals, and information to physicians were raised in many of the letters. I hope that you will understand that I am unable to respond to each letter individually. Instead I will try to address in this letter all of the concerns that have been raised.

On January 12, 1999, the Oncologic Drugs Advisory Committee (ODAC) reviewed the safety and effectiveness data on temozolomide and recommended, by a vote of 11-0 with one abstention, that FDA not approve this drug for use in the treatment of glioblastoma multiforme at first relapse. At the same meeting, the committee recommended accelerated approval of temozolomide for treatment of relapsed anaplastic astrocytoma.

The primary role of an advisory committee is to provide independent expert advice to FDA in evaluating drugs. These outside experts include physicians, scientists, consumers and patient representatives from across the nation. The committees broaden FDA's perspective on important decisions, so that the agency's final decisions reflect a balanced evaluation. Hearing from the public during the public comment session of the meeting enhances the advisory committee process.

FDA is not required to follow a committee's recommendations, but FDA regulatory decisions are generally consistent with advisory committee recommendations. The FDA's decision whether or not to approve a new drug for marketing under the Federal Food, Drug and Cosmetic Act is based upon consideration of two questions:

Do the results from testing of the new drug provide substantial evidence of effectiveness for the condition being studied?

Do the results show that the product is safe under the conditions of use proposed in the labeling for the product?

Members of FDA staff involved in the process are currently evaluating available data to answer these two questions with respect to the use of temozolomide.

Enclosed are two articles that explain FDA's drug review process, and the underlying drug development process:

"The FDA, What Does It do For Cancer Patients?"   and

"Understanding Clinical Trials from The Patient Perspective"

Also included is a copy of the Cancer Letter's coverage of the January 12, and 13, 1999 ODAC proceedings. This article is provided with the permission of the Cancer Letter. I hope the articles will help clarify issues for you.

As a matter of record, FDA cannot approve a drug on the basis of reimbursement-related considerations. The decision must be made on whether the drug has been shown to be safe and effective for its proposed use. Once a drug is approved, the Food and Drug Administration Modernization Act of 1997 provides a mechanism for manufacturers to disseminate reports from medical journals to physicians regarding other uses of a marketed medical product while the manufacturer pursues further development of those uses.

We appreciate the time you took to express your concerns and encourage active participation in this very important process. If you have any questions, you may contact Patty Delaney or JoAnn Minor with the FDA Office of Special Health Issues, Cancer Liaison Program at 301-827-4460.

Sincerely,
/s/
Robert L. Justice, M.D.
Acting Director
Division of Oncology Drug Products
Office of Drug Evaluation I
Center for Drug Evaluation and Research

Posted on the Web by:
Cancer Liaison Program
Office of Special Health Issues
Office of International and Constituent Relations
April 21, 1999