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Who pays for new specialized drugs debated

– When the Food and Drug Administration recently approved a promising new lung cancer drug named Zykadia four months ahead of schedule, it heralded the medication as a breakthrough therapy.

The drug isn’t meant for everyone with the devastating disease, which kills an estimated 160,000 Americans each year. Or even for the majority of patients with its most common form, non-small-cell lung cancer.

Rather, Zykadia is designed for a sliver of patients – about 5 percent – who have advanced non-small-cell lung cancer and have a specific gene mutation that causes tumors to become resistant to existing treatment. For them, and only them, the drug has the proven potential to shrink tumors and extend lives.

The FDA’s speedy approval of Zykadia offered the latest evidence that the age of “personalized medicine,” while long predicted, is increasingly becoming reality. For reasons scientific and economic, one-size-fits-all blockbuster drugs are giving way to treatments tailored to individuals’ genetic makeups and aimed at narrow subsets of broader diseases.

“It’s a new world,” said Wendy Selig, president of the Melanoma Research Alliance, the largest private funder of research on the disease, which this year is expected to kill nearly 10,000 Americans. “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 patients with certain types of genetic mutations. The agency also has given the green light to many drugs for other specific cancers, and to a revolutionary treatment for a small proportion of people with cystic fibrosis. Companion diagnostic tests often help identify which patients might benefit from the targeted treatments.

Pharmaceutical companies have ramped up investment in personalized medicine in recent years, and the number of targeted therapies in the development pipeline reflects that evolution. For example, the FDA said about 80 percent of the nearly 50 drugs it has designated as potential “breakthrough” drugs involve targeted therapies.

For patients who benefit, the advantages are striking: 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?

Many of the current breakthroughs stem from successful mapping of the human genome, completed in 2003. Armed with an intimate understanding of the human genetic code, scientists have spent the past decade deciphering the underlying causes of certain diseases.

As a result, applications for drugs aimed at specific populations of patients – whether for rare diseases or subsets of more common diseases – keep arriving at the FDA. One factor: a lack of blockbuster drugs that once sustained big pharmaceutical companies. Another reason: financial incentives such as accelerated approval and longer exclusivity for drugs deemed especially necessary.