War on cancer, take two

With the signing of the National Cancer Act and the requested appropriation of $100 million for research, President Richard Nixon launched the first “war on cancer” in 1971. It was a campaign “to find a cure.” To give hope to cancer patients, Nixon asserted during a televised address that everything possible “will now be done.”

The past 40 years have brought a greater understanding of the disease's genomics and the related mortality rate has dropped by 23% since 1991, translating to more than 1.7 million deaths averted through 2012. Yet optimism about finding cures has tempered, dampened by high drug costs, lack of collaboration and unfulfilled promises.

“Let's make America the country that cures cancer once and for all,” said President Barack Obama, receiving a round of applause during his final State of the Union address. Obama tasked Vice President Joe Biden with spearheading a new “moonshot” approach to fighting cancer. Biden said he would break down silos that prevent data-sharing and ultimately, “make a decade worth of advances in five years.”

Last week, some of that work began. The National Cancer Institute said it would launch a database this summer that contains information about genetic mutations and cancer treatments. Data will come from as many as 50,000 patients and clinical-trial participants in the Therapeutically Applicable Research to Generate Effective Treatments program, as well as the Cancer Genome Atlas.

“There's never going to be (just) one cure,” said Dr. Julie Vose, president of the American Society of Clinical Oncology. Cancer is thousands of different diseases. Presentations and treatment can vary from patient to patient. Vose warned that it's “impossible to generalize treatments to all patients with cancer.”

To that end, ASCO issued a timely policy statement last week noting how the personalized nature of cancer treatment has resulted in a flood of clinical pathways—protocols that guide which treatment should be chosen for a patient's specific diagnosis. The administrative burden of managing those pathways “is at a breaking point,” experts say.

Also at a breaking point are the overwhelming costs of conducting clinical research, comparing existing therapies and the steep prices that preclude some patients from affording life-saving treatment.

The number of Americans taking at least $100,000 worth of prescription drugs annually from 2013 to 2014 tripled, according to Express Scripts. High-cost cancer medications were partly to blame.

The most promising recent advancements—such as immunotherapy, which uses the body's natural defenses to fight cancer—could cost as much as $130,000 for a 12-week course of treatment. Oncologist Dr. Peter Eisenberg at Marin General Hospital in Greenbrae, Calif., said he is fed up with prescribing “fabulously expensive and marginally effective” drugs.

And profiteering from drugs is a trend that “shows no sign of slowing,” added Dr. Lee Newcomer, senior vice president of oncology, genetics and women's health at UnitedHealthcare. To help control costs, the insurer—the largest in the country—in 2009 launched a collaboration with five oncology groups. They use a value-based payment model based on best practices and patient outcomes.

The national focus on quality and value has also bolstered interest in comparative effectiveness studies that assess the merits of new drugs. But even that research faces challenges.

A 2014 NCI analysis found that some cancer drugs are so expensive they hinder post-market trials that compare which ones work best. It's not in the best interests of the manufacturer of a more expensive drug to provide free medication for those trials, making it necessary for a third party to finance the research. A study comparing the brand and generic versions of two prostate cancer drugs could rack up to $70 million in drug costs alone, the authors wrote. Oncologists have urged changes in federal law to allow Medicare to negotiate drug prices.

Dr. Vincent DeVita, former director of the NCI and a pioneer in oncology research, argues that the U.S. Food and Drug Administration could help by speeding up the approval process, which now can take more than two years. “The ability to innovate is gone,” DeVita said. “The FDA has become the oncologist.”

Also, the nation's investment in cancer research has been dropping since 2003, partly because the NCI has lost nearly 25% of its budget, the agency said. Its budget was increased by $260 million in the recent 2016 budget, but the agency had requested more. Additional funding could help cover expenses for transportation and lost work time, issues that often prevent participation in clinical trials.

Less than 5% of adult patients participate in studies, according to ASCO. And as physicians see more patients in the changing healthcare climate, they may be too busy to discuss the option.

Most patients don't even know to ask about studies, said Dr. Michael Seiden, chief medical officer of the US Oncology Network and McKesson Specialty Health.

In light of the challenges seen over the past 40-plus years, experts say any new approach to curing cancer, moonshot or otherwise, must get back to the basics.

Historically, clinical breakthroughs, including drugs and other therapies, have been the shiny new thing. Most recently, the pending 21st Century Cures Act aims to fast-track the most promising treatments. But the “gee whiz stuff” isn't all there is to it, said Dr. Len Lichtenfeld, deputy chief medical officer for the American Cancer Society.

Efforts to reduce obesity and smoking and boost access to primary and preventive care could significantly cut the cancer mortality rate. “We could do much better in coordinating our efforts in that regard,” Lichtenfeld said.

Eisenberg added that specialty cancer drugs have diverted attention from research into the chromosomal abnormalities that result in cancer variations among patients. “Basic research is not very sexy,” he said. But “if we better understood the science, we could better design the drugs.”