Don't start the revolution without me

  • by: |
  • 02/19/2014
From today's edition of the Orange County Register ...

 A health care revolution threatened

“Personalized” medicines represent a health care revolution. Thanks to sophisticated and new diagnostic technologies, medical scientists have created hyper-targeted treatments keyed into the specific physiological traits of individual patients. These drugs are more effective than their conventional counterparts and less likely to cause adverse side effects. And they’ve proven valuable in the fight against diseases previously considered untreatable.

But drug developers need smart laws to flourish. Unfortunately, over just the last year, federal lawmakers have taken several steps that could smother pharmaceutical innovation and compromise private industry’s ability and willingness to pursue more lifesaving breakthroughs.

First, the good news. The market for personalized biopharmaceutical drugs is on track to double in size to $18 billion by 2019.

According to a recent industry survey from Tufts University, 94 percent of American biopharmaceutical companies are now investing in personalized treatments. Half of all drug prescription compounds now under development are personalized.

The sophisticated customization techniques deployed in the development of these treatments have proven particularly effective in the battle against “orphan” diseases – that is, illnesses afflicting fewer than 200,000 people.

There are 7,000 such diseases that affect more than 30 million Americans. In the 1970s, only 10 drugs were approved to treat orphan diseases. Today, there are more than 400, with an additional 450 are under development.

Most promisingly, federal authorities just approved the first drug ever to treat the underlying genetic flaw causing cystic fibrosis. Just a few decades ago, a child with cystic fibrosis was lucky to survive preschool. Today, the average patient lives well into their 40s.

These breakthroughs in personalized drugs are fundamentally changing how health care providers treat disease. But all that progress is now under acute threat from government.

For starters, Uncle Sam recently started up a “comparative effectiveness” research wing. This work is designed to determine if new, more expensive drugs are worth covering in programs like Medicare and Medicaid.

The fundamental flaw of comparative effectiveness research is that there is no such thing as an “average” patient. A treatment that might not make much of a difference for most patients could prove to be a life-saver for others.

If effectiveness research leads to more constrictive public insurance policies, enrollees that could benefit from advanced personalized drugs will be forced to pay for them on their own. Most simply can’t afford to do that. In effect, the government will be denying them access to this revolution in medicine.

Second, the government has created a new “academic detailing” program. It’s now sending its own representatives (usually local nurses) to doctors’ offices to provide an “academic” counterbalance to input from industry. Problem is, how and where these public representatives get their information is largely opaque. And, if a doctor treats publicly insured patients, these detailing officers have a perverse incentive to get her to choose less expensive drugs to save the program money. That’s not academic detailing – it’s government detailing.

Finally, the Federal Trade Commission is trying to micromanage the process by which new personalized medicines get named. This isn’t a minor issue. And approval delays could threaten the financial health of developers. They depend on sales to recoup their investment costs.

Drug developers will continue to make breakthrough, life-saving treatments as long as federal officials don’t needlessly intrude on their work. The year 2014 could be a banner year for drug development. But we need to get the policy right.

Peter J. Pitts is president of the Center for Medicine in the Public Interest and a former associate commissioner of the U.S. Food and Drug Administration.


Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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