Source: nytimes.com
Author: editorial

The nation’s most important system for judging the clinical effectiveness of cancer treatments is approaching “a state of crisis.” That is the disturbing verdict of experts assembled by the National Academy of Sciences to review the performance of clinical trials sponsored by the National Cancer Institute.

Unless the shortcomings are remedied, some of President Obama’s ambitious health care reforms will be jeopardized and his audacious goal of finding “a cure for cancer in our time” will have almost no chance at all.

The most shocking deficiency highlighted by the report, issued by the academy’s Institute of Medicine, is that about 40 percent of all advanced clinical trials sponsored by the Cancer Institute are never completed. That is an incredible waste of effort and money, and a huge obstacle at a time when researchers are developing promising new therapies that must be rigorously tested.

These large, government-sponsored studies are supposed to be the gold standard — and very different from the narrow, occasionally biased studies sponsored by manufacturers seeking approval of a new drug.

The government-sponsored trials can be invaluable in comparing one therapy against another (manufacturers rarely want to put their products up against a competitor’s), combinations of therapies, or therapies for rare diseases with little commercial potential.

So it is especially worrying to hear the experts say that the system — run by the Cancer Institute at the National Institutes of Health — is so mired in cumbersome procedures that it needs to be completely overhauled.

The Cancer Institute funds clinical trials primarily through 10 “cooperative groups” of experts. They generate ideas for testing new therapies and conduct the trials through networks of cancer centers and community oncology practices. More than 25,000 patients, 3,100 institutions and 14,000 patients participate each year.

Yet a series of reviews in recent years found that the testing operation is mired in bureaucracy and poorly coordinated. A typical trial must navigate past dozens of overlapping reviews by different boards and agencies that must approve the original concept for the trial and then the protocol that will govern how it is conducted before the investigators can start enrolling any patients.

The average time between developing the concept for a study and getting it started is about 2.5 years. The longer a study takes to get started, the more likely it is to become scientifically out of date, and the less likely it is that doctors or patients will want to participate.

Other factors, including failure to pay investigators and their institutions the full costs of a trial, can also impede enrollment. And if not enough patients are enrolled, the study lacks the statistical power to generate meaningful results and cannot be completed.

The Institute of Medicine panel, headed by John Mendelsohn, president of the M.D. Anderson Cancer Center in Houston, offered a range of suggestions for improving the prospects for success.

It called for reducing and consolidating the number of cooperative groups, committees and reviews; increasing the money to support the trials; increasing the academic rewards to encourage researchers to run clinical trials; setting strict deadlines for each step in the process; and prioritizing the studies most likely to be successful. All that should be done even if money has to be transferred from other research activities.

The need for improvement looms especially large now that the Obama administration is pouring substantial sums into “comparative effectiveness research.” That is essential to helping doctors determine which treatments work well and which do not — and holding down the cost of medical care.

More than 11 million Americans are living with cancer or the prospect that cancer may return, and 1.5 million more may get new diagnoses of the disease this year, driving total cancer care costs above $100 billion a year. Nearly one in four Americans are projected to die from cancer. It is vitally important to find the best treatments for them. Repairing the clinical trials system is critical not only for health care reform but for the health of millions of Americans.