Leukemia Society of America Testifies on FDA Reform

In February 21, I presented testimony on behalf of the Society before the Senate Labor and Human Resources Committee during a hearing on Food and Drug

Administration (FDA) reform. Chaired by Senator Nancy Kassebaum (R-KS), the committee held hearings on Senator Kassebaum's bill to reform the FDA.

The FDA is an important and critical federal agency with responsibility for regulating more than 25% of all consumer products in the United States. Advocates of FDA reform cite a number of issues within the agency, including restrictions on the dissemination of information and delays in getting new products or new indications to market, as evidence that changes will be helpful.

The Leukemia Society of America supports efforts to reform FDA as a means of increasing patient access to new and effective treatments and therapies. People with leukemia and other cancers rely heavily on anticancer drugs that have been approved for the treatment of one particular disease, or symptom. However, often drugs are effective in treating other cancers, but problems in the FDA submission and approval system slow down the process by which new indications are added. In some cases, the lack of FDA approval results in insurance companies denying coverage for these drugs because they are deemed "experimental."

The Leukemia Society of America has identified four fundamental principles of FDA reform that will help the agency to help cancer patients. My testimony focused on these reform principles, and the Society is seeking broad support among the cancer community for their inclusion in any FDA reform legislation enacted by Congress.

Principles of FDA Reform for People With Cancer

Cancer is a life-threatening disease for which, in many cases, there is currently no satisfactory treatment. People with cancer need new treatment options, and reform of Food and Drug Administration (FDA) policies and procedures offers hope for access to potentially lifesaving therapy. Also, because well over 50% of cancer treatment involves "off-label" uses of approved products, cancer patients are heavily reliant upon ready access to, and information about, such uses. Supporting the essential role of FDA in ensuring the safety and effectiveness of anticancer products but also recognizing the distinct needs of people with cancer, we endorse the following fundamental principles of FDA reform:

• As a life-threatening disease, cancer should be recognized as a special category of regulatory review with expedited approval and early access to products under review.
• FDA should not seek to regulate distribution of reliable information whether from texts, peer-reviewed articles or standard medical compendia. In order to be consistent with standard medical practice and with Medicare reimbursement guidelines, FDA should join with other interested groups to ensure that information recognized as reliable by physicians and reimbursement authorities is freely available.
• As a means of addressing the chronic problem of outdated labeling for anticancer products, FDA should be required to accept peer-reviewed literature reports of clinical trials as adequate and well-controlled studies suitable for supporting approval of new uses of approved drugs, biologics, or devices.
• Legislation should clarify that there are situations where one adequate, well-controlled trial may suffice to meet requirements for approval of new drugs, biologics, or devices.

FDA has done much to speed the approval of new drugs to treat life-threatening diseases. Cancer patients have benefited from such activity. However, more can be done, and advances should be made permanent through legislative relief rather than through administrative action that may change over time. The above principles represent modest change that will markedly improve options for people with cancer.