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News Article

Sep 27, 2007

Wall Street Journal: 'Choice' in Health Care

By Richard Miller

Wall Street Journal 

As the 2008 presidential candidates debate possible changes to America's health-care system, and states like California move forward with their own initiatives, one thing is clear -- there will be greater emphasis on patient choice.

From Sen. Hillary Clinton's "Health Choices" plan to Mitt Romney's federalist reform plan, the proposals all seek to spur greater patient choice of insurers, doctors and potential treatments. This is a welcome debate, as Americans currently are prevented from making -- even in consultation with their physicians -- the health-care decisions that would best serve them.

Yet our choices are not only constrained by limited insurance options. Patient choice is also severely compromised by a lack of comparative performance data on individual physicians, hospitals, clinics and treatments. And in many life or death situations, where treatment options exist but carry some risk, the FDA and courts are denying patients the right to choose altogether.

With respect to these critical constraints on patient choice, there is good news and bad news.

The good news is on access to vital information. In August, the district court for the District of Columbia ruled in favor of a consumer group, Consumers' Checkbook, which sued the Department of Health and Human Services to allow disclosures of specific data about physicians from the Medicare claims data base. Judge Emmet G. Sullivan concluded that "a significant public benefit" could be served by releasing the data and ordered the Center for Medicare and Medicaid Services to turn it over by Sept. 21.

With data on more than 40 million patients and 700,000 doctors, the Medicare database is a gold mine for patients, providing them with a key asset -- information. People will now be empowered to make choices based on critical information that has been grossly lacking in the medical marketplace -- including data on outcomes, cost, performance and quality of physicians and associated providers. For example, patients will be able to review the data on everything from hernia repair to open-heart surgery to treatments for diabetes and cancer, helping them to determine which centers and physicians have the most experience, best outcomes, least complications and lowest cost.

Driven by patient choice, the availability of the Medicare database could be the beginning of an important movement in medicine that contributes to higher quality care and lower costs. This will likely affect everything from elective procedures for non-serious conditions to complex and dangerous treatments such as open-heart surgery and cancer therapy.

Patients will benefit from a deeper understanding of the risks and benefits, and will be able to view the information in the context of their individual needs and goals. They will be further empowered to make the best treatment choices, based on consideration of all the relevant factors, and not just the bias of their provider.

The bad news is on access to new drug treatments for those who need them most. In its recent Abigail Alliance decision, the U.S. Court of Appeals for the District of Columbia ruled that dying patients do not have a constitutional right to experimental drugs until they have been proven to be safe and effective, as judged by the FDA. Apparently, the decision of this court, in contrast to the Consumers' Checkbook case, is that terminally ill patients should not be given the choice of how they will defend themselves against a deadly disease.

The great irony is that terminally ill patients, especially those with cancer, are treated with experimental drugs all the time. Cancer drugs are usually approved by FDA for very narrowly defined diseases. For example, a cancer drug is usually approved for a specific type of cancer and clinical situation or indication. However, physicians frequently prescribe cancer drugs for unapproved or "off-label" uses outside of the narrowly defined indication.

More than 60% of cancer-drug usage is for unapproved indications, and often this is done with scant information and with limited evidence of safety and efficacy for the specific disease. Typically, physicians discuss the treatment options with their patients based on the available information, and together they decide whether the risks versus benefits are sufficient, or not, to embark on the treatment.

The off-label use of cancer drugs should not be discouraged. It is vital to patient care and makes important new drugs available to more patients with a range of different types of cancer more quickly than could occur by requiring formal clinical testing in each kind of cancer. The typical paradigm in cancer-drug development has been to obtain approval for a limited clinical setting, and then to let off-label use lead to the discovery of new applications in a broader range of cancers.

What should be discouraged, particularly in light of the Abigail Alliance decision, is the overly restrictive stance of the FDA toward drug approvals for patients with life-threatening diseases. In recent years, the FDA has made it increasingly difficult for terminally ill patients with cancer and other deadly diseases to gain access to new but promising drug treatments even after testing in large clinical trials. The FDA has rejected five promising cancer drugs this year alone. Why should the FDA block approval of new lifesaving drugs based on different standards than are currently employed by physicians when prescribing drugs for "off-label" uses?

It defies logic that physicians and patients have the choice of using an FDA-approved lung-cancer drug off-label to fight brain tumors, while being denied the choice of using a promising but not-yet-approved drug that has demonstrated substantially more evidence of effectiveness in fighting this indication.

Terminally ill patients may not have a constitutional right to choose experimental drugs, but the FDA does have a legislative mandate to grant accelerated drug approval based on limited but promising clinical data. We need the FDA to utilize the regulations promulgated as a result of the AIDS epidemic to give patients the right to choose these treatments while more lengthy definitive trials are undertaken. This in turn will spark greater private-sector investment in breakthrough drugs that will further improve the quality of health care.

The increasingly paternalistic attitude of the FDA is running counter to the welcome trend toward greater patient choice. If we are indeed going to move toward giving patients a more active role in making choices about their health and wellness -- and providing them with more information so that they can make informed choices -- we should not make an exception for new drugs to treat deadly diseases. The FDA plays a key role in our health-care system, and the presidential candidates should rethink FDA drug-approval policies as part of their plans for health-care reform.

Dr. Miller is president and CEO of Pharmacyclics, and adjunct professor of oncology at Stanford University Medical Center.
© 2007 CAHR