Good morning. My name is Carolina Hinestrosa. I am a two-time breast cancer survivor and the Executive Vice President of the National Breast Cancer Coalition. I am pleased to have the opportunity to testify before ODAC about the need for rigorous evaluation of supportive therapies in breast cancer: first, to make sure patients are not harmed; and second, to ensure they don't receive costly treatments they don't need.
The National Breast Cancer Coalition has been fighting for improvements in breast cancer research and care since its inception in 1991. NBCC is a coalition of hundreds of organizations and tens of thousands of individuals nationwide who represent the diversity of breast cancer. Our mission is to end breast cancer. We educate and empower those affected by this disease to influence research, quality of care, access and public policy agendas. NBCC embraces the philosophy of evidence-based healthcare. We believe that as a community (scientists, clinicians, regulators, manufacturers, patients and society), we need to learn what really works for women with and at risk for breast cancer, and make sure that knowledge is incorporated into clinical practice. We must also figure out what price we pay, health-wise and financially, to make a determination of what's acceptable and appropriate care.
There is an underlying trust patients and consumers have in scientists, doctors and FDA regulators to look after their best interests as YOU evaluate the benefits and harms of the drugs, tests and all interventions that become available to fight cancer. Consumers trust the system to offer interventions that are based on high-quality evidence, and appropriately designed randomized clinical trials are the gold standard to obtain evidence of benefits and harms for all interventions: therapeutic or supportive. The National Breast Cancer Coalition believes strongly that for the system to be truly responsive to consumers' needs, WE the consumers need to be involved at all levels where important decisions about our lives are made. We are pleased to see true consumer representation at this ODAC meeting.
The news last March that the FDA and the manufacturers of Erythropoiesis-Stimulating Agents had agreed to add a boxed label warning on harms to patients due to off-label use of ESAs was in part reassuring, but mostly sobering. FDA action was clearly needed to inform consumers and educate physicians about the very serious dangers of off-label use of these agents. It also was a case in point of a distorted system where care is allowed to be based on poor or no evidence and the interests of secondary stakeholders take precedence over those of the primary stakeholder: the patient.
Over the decades we have witnessed a philosophy for cancer care where "more must be better". We have used (before we had evidence and harming women in the process) high-dose chemotherapy with autologous bone marrow transplantation; we have piled up toxic treatment on top of toxic treatment in order to obtain marginally better, and often short-lived outcomes; and have used and overused supportive therapies to help us sustain that approach. A cancer patient trusts that all that care is needed to save her life.
I don't know how many of you have been diagnosed with cancer. I can tell you that it is hell. Every day women go through the hell of diagnosis and the hell of treatment with the hope that in the end THEY will live and will be OK. THEY are the primary stakeholder. All others must focus on the best way to help these women live, and minimize harms to them. It is not about selling more drugs or getting them approved faster, or about supporting start-up companies or the growth of existing ones. It is about saving people's lives?truly.
ESAs were first approved for cancer to meet a medical need: to lower the risk for blood transfusions due to anemia caused by chemotherapy. At that time, the FDA raised concerns about the potential for these agents to promote tumor growth. There was also limited information about harmful effects of these agents. The medical community went ahead and incorporated them into clinical use on- and off-label. Whole new approaches to breast cancer treatment such as dose-dense chemotherapy are supported by the use of G-CSF therapy to repopulate white blood cells, and ESAs to stimulate production of red blood cells. This for marginal improvement in outcomes, without a thorough assessment of the true benefits and harms of supportive treatments, and at a great cost to patients and the healthcare system.
Now we know that off-label use of ESAs can speed disease progression and death, and have other effects.
It is well known that there are perverse financial incentives in our system that motivate physicians to prescribe these and other costly drugs, imaging and other procedures regardless of whether they are indicated, or despite their questionable value to patients. I realize it is not up to the FDA to fix this part of the problem, but it must do its part to demand quality data from high-quality studies to inform patients and practitioners, and to assure the public that it is looking after its best interests.
The questions FDA asks of ODAC today are important:
I am left with a sense that we really don't know what we are doing - we know much more about the effects of ESAs at unapproved doses than we know about their effects at approved doses. As a committee you must assess and rely on the quality of the evidence before you in order to make recommendations to the FDA. Your role is critically important to patients who trust these decisions are made keeping their best interests at heart.