Sick Patients Need Cutting-Edge Drugs

Anna Tomalis was a bright, pretty, 13-year-old girl who liked horseback riding and soccer. During the last few years, she rarely had a chance to think about those things. Since September 2005, Anna battled cancer. And, instead of wringing all she could out of childhood, this courageous teenager tried to get members of Congress to act like adults. Anna had embryonal sarcoma, a rare form of liver cancer. Surgery and chemotherapy seemed to work at first, but the tumors came back. In March of this year, doctors told her there was nothing more they could do.

She and her parents didn't give up, though. With a little research, they discovered a number of experimental drugs that show promise in treating soft-tissue sarcomas like Anna's, including one called Deforolimus, developed jointly by the drug companies ARIAD and Merck. Unfortunately, Anna was too young and too sick to be admitted to the clinical trials in which she might have gained access to one of those drugs. And the Food and Drug Administration (FDA) very rarely grants so-called compassionate-use exemptions to administer unapproved medicines outside the clinical testing process.

Just a few months before Anna Tomalis received her fateful diagnosis, this editorial page introduced readers to an Indiana woman name Kianna Karnes. Mrs. Karnes was waiting for the FDA to consider her own petition to use an experimental drug, and editors proposed a "Kianna's Law" to reform the clinical testing process for drugs to treat life-threatening illnesses. Tragically, no such law was enacted; Mrs. Karnes died the very day FDA approved her exemption.

Anna Tomalis was only barely more fortunate. It took months for her exemption to come through and for Anna to get access to one of the experimental drugs her family thought might help her. Anna's mother Liz says she "came into this process so naive, thinking that those of us who seek compassionate use of drugs actually get them. It was a shock to find out I had been seriously misled."

Her treatment regimen began at the end of July, but for Anna it was too little, too late. She passed away on Aug. 15. Of course, there is no way to know whether the experimental drug would have stopped the tumors from spreading, even if Anna was able to begin her treatment as soon as she ran out of alternatives.

Why don't we know that? Why do terminally ill patients have to wait so long to get access to the only treatments that hold any promise of saving their lives? And why is it not their right to decide? The problem has been magnified in recent years as the number of new drug approvals has fallen dramatically. The FDA approved just 16 new drugs last year, and is on pace to approve only 18 this year. That's down from a high of 53 in 1996 and 39 in 1997.

After a few high-profile drug scares, such as the 2004 withdrawal of Vioxx from the market, FDA officials have become gun-shy about approving new products. After all, the agency receives scathing criticism from Congress and the press when an approved drug turns out to be more risky than expected -- but rarely for keeping beneficial ones off the market.

Last year alone, the FDA rejected five new cancer drugs, including a breakthrough treatment for prostate cancer called Provenge. A panel of cancer experts that advises the FDA on new drug approvals unanimously agreed that Provenge was safe, and voted 13-4 that it was effective enough. But the FDA demanded still more testing that may delay approval for three years.

Despite fading health, Anna Tomalis worked closely with two patient advocacy organizations, the Abigail Alliance and A Right to Live, to change the system. Together, they have lobbied for legislation to improve the compassionate-use process for terminally ill patients, and expand access to promising treatments. In May, Sen. Sam Brownback (R., Kan.) and Rep. Diane Watson (D., Calif.) introduced a bill to do just that. The Access, Compassion, Care, and Ethics for Seriously Ill Patients Act (ACCESS) would make promising new medicines available as soon as they are shown to be safe in the first of the FDA's three-phase testing process, and once preliminary evidence of their effectiveness is established.

Anna realized that ACCESS would not be passed soon enough to help her. But she continued to fight for it. She knew that it could help save the lives of countless others. Does the U.S. Congress know this also? And will it do the right thing?

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America Needs a National Market for Health Insurance

Much to our surprise, the Census Bureau reported yesterday that the number of people in the U.S. with health insurance actually increased by 3.6 million last year. That's the good news. The bad news is that nearly three million of them got their coverage through government programs. The slide toward a government-dominated, taxpayer-supported health sector will continue unless the 45.7 million Americans who don't have insurance now are given more opportunities to buy private coverage.

States could help by lightening their regulatory burdens to encourage greater competition for more attractive and affordable coverage. The federal government needs to do its part by updating today's tax policies to better fit a mobile, 21st-century economy.

A new study by University of Minnesota researchers Stephen Parente and Roger Feldman shows that Congress could boost by more than 12 million the number of people who have health insurance without spending taxpayer dollars. The change required is to allow people to buy health insurance across state lines, so they can shop for less expensive policies.

The cost of health insurance varies widely, but it is closely tied to state regulations and legislative mandates dictating what services and providers must be covered. More regulation and less competition generally mean less affordable coverage, and vice versa. For example, a typical health-insurance policy in heavily regulated New York costs more than three times as much as in less regulated Iowa ($388 a month versus $98 a month for the same coverage).

Every state requires health-insurance policies to cover certain services, ranging from maternity care to bone marrow transplants and hair prostheses. Nationwide there are more than 1,900 coverage mandates in all. The Council for Affordable Health Insurance estimates that these mandates add 20% to 50% to the cost of health-insurance policies.

Each mandate can be defended in its own right. But as the burden increases, fewer people can afford to buy insurance. Usually, individuals and small businesses are the first to be priced out of the market.

Yet laws designed to make health insurance more affordable often backfire. Many states tell insurance companies they must charge similar rates to everyone; they also force insurers to sell policies to people who wait until they are sick to buy coverage. It's a little like allowing a person to wait until his house is on fire, and then requiring an insurance company to sell him a homeowner's policy at the same rate as those who paid the premiums all along.

States should be giving residents more options to buy policies that suit their budgets, not the priorities of politicians. Rep. John Shadegg, a Republican from Arizona, has proposed federal legislation that would allow people to buy health insurance across state lines.

But Congress could do more than simply knocking down the barriers to interstate health insurance. For starters, it could make health insurance more portable. One way to do that would be to change the tax subsidies already going to those who get health insurance at work and turn them into refundable tax credits. This would make the subsidies available to everyone, and help millions of people buy coverage who can't afford it now. It would also help people keep their health insurance when they lose their jobs or move.

Freeing Americans to buy health insurance across state lines would give people more choices in health care. And giving individuals a direct tax break for purchasing coverage would put armies of consumers to work to find affordable policies. That would force states to lighten their regulations or lose out to other, less regulated states. The complex problems in our health sector are best cured by a bigger dose of market competition, not more government intervention.

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