carl wiens for the boston globe

Every week, it seems, biopharmaceutical companies announce new breakthroughs in “personalized medicine” — customized health care where the goal is to tailor drug therapies to individuals. Competition between gene sequencing businesses to catapult us into the age of the $1,000 genome drives much of the hoopla. Gene-based companion tests, for cancer drugs especially, promise to help doctors discern which patients are likeliest to benefit from which treatments, curtailing the need for mix-and-match, trial-and-error, one-size-fits-all chemotherapies.

But what if every new drug — however much more effective than current treatments — works only for smaller and smaller numbers of patients? And what happens to those minimally beneficial but mega-profitable blockbusters that now dominate cancer treatment when it becomes clearer that only small percentages of the people taking them will truly benefit, people who now can be identified beforehand? When this is the way standard medicine operates — optimists, led by National Institutes of Health Director Dr. Francis Collins, predict huge strides in the next decade — how are insurers and strapped governments going to pay for it?


Advances in genomic medicine will no doubt mean better, more targeted treatments. That’s good news for patients with the “right” mutations and for their loved ones. The rest of us? Maybe not so much. The industry-wide narrowing down on targeted therapies is already yielding a host of unintended consequences.

Try a thought experiment: You’re a pharmaceutical executive faced with a new reality — that the surest, quickest route to profits is developing a high-value treatment for a few thousand people. The blockbuster era of marketing drugs that offer incremental improvements for common illnesses is over. Wall Street is breathing down your neck. What diseases do you go after? Where do you put your resources? The vogue for specialized and rare diseases means less talent and money will be devoted to widespread scourges like diabetes, not to mention long-neglected killers like tuberculosis.

Or consider that you’re a public official, insurer, or hospital administrator trying to determine whether a new drug that works at least initially in just a few thousand people is “worth” the cost. Or, you’re trying to put a value on an old drug that up till now was the standard of care for everyone with one of the most common forms of cancer but really is effective only in the 10 percent of patients known to have a specific defect. In medicine, patients are the coin of the realm. Who gets to treat them, what protocols are prescribed: These drive the medical economy. Who gets effective health care and who doesn’t determine overall how much we pay as a society.


So far the United States has blithely skirted these questions. The reason? Because the numbers of patients receiving true “personalized” medicines remains small enough not to have a significant impact on the financial arrangements that govern the health care economy. In America, selling new drugs is unlike selling anything else. The consumer (the patient) is not the customer (the prescribing physician) and the customer is not the payer (a managed care company or insurer or government program). As a result, drugmakers set prices much higher than consumers can afford to pay. Customers, especially oncologists and the hospitals that own their practices, then mark up those prices as much as several times over, charging payers whatever the market will bear. As long as the numbers of patients remains in the thousands, no one has a material interest in changing the system.

To glimpse the messy, uncharted future of health care, I recommend the case of ivacaftor, also known as Kalydeco, the revolutionary treatment for Cystic Fibrosis approved both in the US and Europe in 2012. Lauded by Collins and Food and Drug Administrator Dr. Margaret Hamburg as a sterling example of the promise of personalized medicine, the drug was discovered and developed by Vertex Pharmaceuticals, which last month opened its gleaming twin towers on the Boston Waterfront with cheers — and subsidies — from state and local leaders. Ivacaftor transforms the lives of patients with a particular group of genetic mutations affecting around 10 percent of people with the disease, by correcting the underlying defect. At more than $300,000 a year, insurers and government payers in the United States across the board enthusiastically pay the cost for fewer than 2,000 patients, while the company provides it free to anyone who can’t afford it. Everyone, it seems, wins; nobody, presumably, is hurt.


But what about when such drugs are the rule rather than the exception, when the numbers of new ultra-high-priced medicines and the many more patients who may benefit from them reach the point where something has to give? Ivacaftor has been vastly more controversial in Canada and England, where very real and sobering opportunity costs come into play. Government health officials and politicians have had to ask themselves: How can we justify an eye-wateringly high-priced specialty drug for a few hundred patients that will cost as much as all other medications for everyone else with the disease combined? The British National Health Service negotiated to buy ivacaftor at a substantially lower price, and after some heart-wrenching stories in the media all appropriate patients are now getting the drug, no matter how poor. But personalized medicine doesn’t get more personal than when your family’s ability to get a life-saving drug for your child takes away from mine. In Canada, Ontario Premier Kathleen Wynne recently pledged, after 14 months of negotiations with Vertex, greater effort in getting ivacaftor for a 12-year-old girl whose family now relies on charitable fundraising to pay for it.


High-value medicines like ivacaftor should be the solution, not the problem. For patients who get them, they are. But it’s not hard to anticipate the pileup that will occur as more and more very expensive drugs for relatively small numbers of patients gain approval just as pressure to spend less on health care inevitably forces a more value-based pricing model to take hold in the United States. Then we will find out, like Britain, Canada, and other nations, that access to medicine is not just a matter of matching medical breakthroughs with patients who have the correct genetic profile, but a social choice that affects us all.

Barry Werth is the author of “The Antidote: Inside the World of New Pharma” and “The Billion Dollar Molecule: The Quest for the Perfect Drug.”