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
- 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.