Leukemia Society of America Testifies on FDA Reform

In February 21, I presented testimony on behalf of the Societybefore the Senate Labor and Human Resources Committee during ahearing on Food and Drug

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

The FDA is an important and critical federal agency with responsibilityfor regulating more than 25% of all consumer products in the UnitedStates. Advocates of FDA reform cite a number of issues withinthe agency, including restrictions on the dissemination of informationand 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 FDAas a means of increasing patient access to new and effective treatmentsand therapies. People with leukemia and other cancers rely heavilyon anticancer drugs that have been approved for the treatmentof one particular disease, or symptom. However, often drugs areeffective in treating other cancers, but problems in the FDA submissionand approval system slow down the process by which new indicationsare added. In some cases, the lack of FDA approval results ininsurance companies denying coverage for these drugs because theyare deemed "experimental."

The Leukemia Society of America has identified four fundamentalprinciples of FDA reform that will help the agency to help cancerpatients. My testimony focused on these reform principles, andthe Society is seeking broad support among the cancer communityfor 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 cancerneed new treatment options, and reform of Food and Drug Administration(FDA) policies and procedures offers hope for access to potentiallylifesaving therapy. Also, because well over 50% of cancer treatmentinvolves "off-label" uses of approved products, cancerpatients are heavily reliant upon ready access to, and informationabout, such uses. Supporting the essential role of FDA in ensuringthe safety and effectiveness of anticancer products but also recognizingthe distinct needs of people with cancer, we endorse the followingfundamental principles of FDA reform:

• As a life-threatening disease, cancer should be recognizedas a special category of regulatory review with expedited approvaland early access to products under review.
• FDA should not seek to regulate distribution of reliable informationwhether from texts, peer-reviewed articles or standard medicalcompendia. In order to be consistent with standard medical practiceand with Medicare reimbursement guidelines, FDA should join withother interested groups to ensure that information recognizedas reliable by physicians and reimbursement authorities is freelyavailable.
• As a means of addressing the chronic problem of outdated labelingfor anticancer products, FDA should be required to accept peer-reviewedliterature reports of clinical trials as adequate and well-controlledstudies suitable for supporting approval of new uses of approveddrugs, biologics, or devices.
• Legislation should clarify that there are situations whereone adequate, well-controlled trial may suffice to meet requirementsfor approval of new drugs, biologics, or devices.

FDA has done much to speed the approval of new drugs to treatlife-threatening diseases. Cancer patients have benefited fromsuch activity. However, more can be done, and advances shouldbe made permanent through legislative relief rather than throughadministrative action that may change over time. The above principlesrepresent modest change that will markedly improve options forpeople with cancer.