Good intentions could have damaging results

As the president of the National Breast Cancer Coalition, I have had the privilege to work alongside thousands of volunteers to help end breast cancer. Our efforts are aimed at saving as many lives as possible.

As a breast cancer survivor and an advocate, I have come to understand that cancer is an incredibly complex disease and the research needed to address it is difficult; answers take a long time and true patient benefit is rare. We exist in a cancer world of hype, false hope, devastating cost and incremental improvement. Talk of “cures” or “breakthroughs” continue to be unfulfilled promises. To save lives we must have a system of research and regulation that speeds promising treatments to the public protects individuals from unnecessary harm and provides access to all who need it. The bill sponsored by Senator Brownback (R-KS) will undermine these goals.

The Brownback bill would seriously weaken our necessary system of science-based health care and fundamentally change the system by which the Food and Drug Administration approves many cancer drugs. The existing approval process exists to protect the public from harm and to make certain that drugs given to people actually work. The system was designed in response to the thalidomide scandal of the early 1960’s and to create accountability to the public.

The legislation proposed by Senator Brownback substitutes Congress’ idea of science for high-quality research. The bill will also allow approval based on surrogate endpoints or biomarkers without proof of clinical benefit. This approach will put more drugs on the market, but will not help patients.
In addition to the bill’s attempt to redesign the research itself, it will make it more difficult to get the evidence we need. It wipes away the incentive to participate in clinical trials. Consequently, the entire system of drug trials will falter, making it nearly impossible to evaluate a drug’s safety and efficacy.
Moreover, because of an unfortunate reality of cancer treatment, this legislation carries with it the very real threat of making harmful, investigational drugs broadly available. Under our present system patients can access drugs outside of trials after they are approved as safe and effective. However, once approved, a drug can be prescribed by a physician for any purpose, not limited to the condition that was studied. Because our current system looks carefully at safety, and the drugs have been proven effective for a specific condition, giving these treatments “off-label”, while problematic, has become routine in cancer treatment. The Brownback legislation would result in all cancer patients – not just those described in the bill - having off-label access to unproven, potentially lethal, therapies.

There are many examples of the damage done when the appropriate scientific process is ignored. In the 1990’s, many breast cancer patients were given high-dose chemotherapy with bone marrow transplantation outside of clinical trials with the hope that this highly toxic treatment would offer a desperately needed cure. Consequently, it became difficult to recruit patients for studies. Without that reliable research, doctors prescribed this regimen to some women whose cancer was less advanced and who had other proven treatment options. We eventually learned that this high-risk, grueling and expensive procedure is no more helpful to women with breast cancer than standard care. This supposed “breakthrough” in fact harmed many of the women who received it, resulting in unnecessary deaths.

NBCC activists are frustrated with the lack of answers and pace of progress. We and others are working in a responsible way to address the issues. We have worked with scientists and policymakers to find new sources of research funding and create new and innovative approaches to getting answers, including ways to speed up the clinical trials process while retaining scientific integrity.

Recently, NBCC convened a three-day conference of leading researchers, policymakers and advocates to plan the future of biomarker research. We agreed on a plan of action to ensure that work on biomarkers will expeditiously lead us to effective and safe ways of preventing, diagnosing and treating breast cancer that are relevant to all cancers. We will continue creating projects to bring real help to patients more quickly.

It may seem compassionate to allow access to unproven drugs for seriously ill patients But this approach has every likelihood of harming more people than it could possibly help. We must not let our emotions blind us to the need for careful and methodologically sound practices. I firmly believe that it is the integrity of the research and regulatory process that holds the key to lifesaving breakthroughs. I urge Senator Brownback to support this process, rather than try to undo it.

Fran Visco

Bill would give seriously ill patients access to early stage drugs

While it sometimes seems the Senate can hardly agree on anything, this year 92 senators sent an open letter to the White House making the case for an aggressive strategy to end death and suffering from cancer by 2015.

An important part of the war against cancer is the ACCESS Act, which provides seriously ill patients with cancer or other life-threatening diseases with accelerated access to promising treatments still in the investigation stage.

We need the ACCESS Act because it takes an average of 12 years to approve new drugs. But time is not on the side of late-stage cancer patients who have exhausted every treatment option. The least we can do is to provide these patients, under the supervision and sanction of their doctors, with quicker access to treatments that could improve their condition.

Through this legislation we can supplement the FDA’s regular approval process by creating a parallel system for terminally ill patients who are currently blocked from clinical studies and compassionate access programs because of bureaucratic hurdles.

The ACCESS Act would create a new three-tiered approval system specifically for products that treat serious or life-threatening diseases. In essence, if a drug shows promise in one or both of the first two phases of the FDA’s current approval process—meaning that it passes safety tests and holds meaningful promise as an effective treatment—patients with life-threatening diseases could access the drug before the traditional approval process is complete.

Furthermore, before providing early access to a new treatment, the FDA would evaluate the potential benefits of the treatment against the potential risks. If the risk of a patient’s condition outweighs the risk of the treatment, and if the treatment holds promise to benefit the patient, the FDA could approve the product.

Only seriously ill patients who have exhausted all treatment options would have access to drugs approved under this new expedited approach. Given their terminal condition, when a drug meets safety standards and offers treatment promise the least we can do is offer these patients a chance at life.

To prevent companies from sidestepping the regular FDA approval process, the bill gives the FDA authority to withdraw this new kind of approval if a drug sponsor fails to conduct post-marketing studies.

The ACCESS Act also would simplify the cumbersome application procedures for expanded access by shifting the decision process from Washington bureaucrats to patients and doctors.
While some critics fear that creating exceptions to the approval process will compromise the FDA’s ability to safely screen drugs, the ACCESS Act takes a narrow and limited approach to addressing the unique needs of seriously ill patients.

When I faced cancer myself I was fortunate to have several treatment options. I can only imagine what it must be like to feel helpless in the face of a terminal condition where treatments options range from little to none. These patients and their doctors have the right to make the personal decisions that affect their health and quality of life.

Sam Brownback

Posted on August 1, 2006 06:00 AM