Breakthrough drugs for targeted diseases work wonders, but are very expensive; some see a trend to worry about

Drugs focusing on narrow subsets of broader diseases are now being developed for individuals with specific genetic makeups. This trend prompts questions about who covers the cost, who gets the drugs and which diseases get the investment dollars, Brady Dennis reports for The Washington Post.

The newest “breakthrough” pharmaceutical is a new lung cancer drug named Zykadia, Dennis reports. The Food and Drug Administration released it four months ahead of schedule.

This drug is not designed to treat everyone who has lung cancer, which kills an estimated 160,000 Americans each year, including 3,500 Kentuckians. Kentucky’s lung cancer death rate is almost 50 percent higher than the U.S. rate, placing Kentucky first in the nation for new cases and deaths from lung cancer.

Zykadia is designed to treat only about 5 percent of patients who fit into a specific subset of the disease: those who have advanced, non-small-cell lung cancer, and a specific gene mutation that causes tumors to resist other treatment, Dennis writes. For them—and only them—the drug has the proven potential to shrink tumors and extend lives.

“It’s a new world,” Wendy Selig, president of the Melanoma Research Alliance, told Dennis. “We’re segmenting what we thought of as large diseases into smaller populations of patients that are defined by genetic distinctions. . . . The goal is to match the right therapy to the right patient and to do it with minimal collateral damage.”

Since 2011, the FDA has approved numerous new treatments for melanoma, or skin-cancer patients with certain types of genetic mutations, specific cancers and a small proportion of people with cystic fibrosis, Dennis notes.

The FDA told Dennis that almost 40 of the nearly 50 drugs it has designated as potential “breakthroughs” involve targeted therapies. These targeted therapies often offer recipients earlier and better diagnoses, more effective treatments and even possible cures—or at least more time—for people who previously had little hope.

“But the trend toward targeted medicine also is forcing hard questions on regulators, drug makers, insurers and patients alike: Who should pay for the growing number of specialized drugs, which can cost hundreds of thousands of dollars a year? How does society ensure that everyone who might benefit—rich or poor—can receive treatment? What about patients whose specific conditions or mutations have not attracted investment dollars or the attention of researchers, leaving them to watch and hope from the sidelines?” Dennis writes.

Private insurers and Medicare generally cover the high drug costs, which currently account for only a fraction of overall health-care spending, Dennis reports.

But Barry Werth, an author and journalist who has spent decades writing about the pharmaceutical industry, asks, “What happens when targeted drugs become the rule rather than the exception? Will insurers refuse to cover some of them? Will the government ponder rationing them? Will only the wealthy be able to afford the best and newest treatments?”

This scenario could be happening with Sovaldi, a “groundbreaking new treatment” for the estimated 3 million Americans infected with hepatitis C, Dennis reports. Insurers are publicly rebelling against the drug’s price of $1,000 per pill, or $84,000 for a 12-week treatment.

“The worry is that Sovaldi is the poster child for a “tsunami of expensive medicines” that collectively threaten to bankrupt the health-care system,” John Rother, president of the National Coalition on Health Care, which represents insurers, employers, consumer and religious groups, health-care providers and some drug manufacturers, told Dennis.

“We’ve been able to absorb it so far because there are very few of them and because they are consumed by very small numbers of people,” Rother said of specialty drugs. “[But] if they’re all priced at the same level Sovaldi is, we really won’t be able to absorb it in the current system. It’s not sustainable.”

Edward Abrahams, president of the Personalized Medicine Coalition, an advocacy organization that includes drug makers, medical providers and patient and research groups, told Dennis that the cost of creating targeted drugs cost billions of dollars and often takes a decade or more to develop. Without the ability to recoup those costs, he said, “research will wither.”

“As a society, we’re schizophrenic about this,” Abrahams told Dennis, adding that everyone wants to cure cancer and other diseases. “But at the other end, you have to be willing to pay for these products. . . . When you develop a drug for a segmented population, the cost of that drug is likely to be higher than if it worked for everybody.”

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