As the costs and uncertainty of drug development go up, so do the challenges of making prescription drugs affordable and available to the Americans who need them most, says Mark B. McClellan, Commissioner of the Food and Drug Administration.
"There is a growing chasm between the discovery of new biomedical research insights that you hear about in the news, and the creation of a new treatment that patients can be confident is safe and effective," said McClellan, who is both an economist and physician. Public health relies on the FDA and Congress working toward the twin goals of encouraging medical innovation and ensuring that patients have access to safe, effective, and affordable medical care.
Discussing "the politics of innovation" at the Harvard Business School Alumni Healthcare Conference on November 7, McClellan said government plays a critical role in improving the pathway between scientific breakthroughs and bringing affordable drugs to patients.
"I'm optimistic that we're making progress on these issues," he said, while acknowledging that many people disagree on how to bridge the gap between high science and low prices.
"We are living in a very special and uncertain time when it comes to health policy, our healthcare system, and the future of the health of the nation," he said.
The mission of the FDA is to advance and protect the health of the public, according to McClellan. Fulfilling that mission is more challenging than ever before, yet various advances in science have also meant that there are more opportunities for solving problems. Entire fields of promising new biomedical science have sprung up in the last ten years, such as nanotechnology, genomics, and next-generation information technology systems. Even so, for the first time in two decades, the FDA received the lowest number of applications last year and approved the smallest number of new drugs, he said. The number of new biologics and medical devices has also gone down.
Analysts say it is not surprising that there are fewer new solutions are coming forward at this point in time, he explained. The cost and uncertainty of developing new medical products has increased greatly. A lot of "priming the pump" in terms of grappling with new information needs to take place before treatments and devices relying on innovations in nanotechnology, for instance, can take shape.
Mail-order snake oil
In his talk at HBS, McClellan outlined new policies the FDA is pursuing. At the top of the list is finding solutions to the high cost of developing, using, and ensuring the safety of prescription drugs and medical products. These pressures will only increase due to recent pricing trends and the aging of the population, he said. Many people cannot afford expensive new treatments and feel they have no alternative but to buy outside the U.S. People in the U.S. buying drugs in American pharmacies often pay the highest prices in the world, he said.
"People are very angry about this situation—and they should be. Many Americans don't understand why Canadians can get many drugs that are similar to those available here, safely and at a lower price, than we can in the United States. The United States hasn't found a way to provide drugs that are affordable and safe within our own country. Until we find solutions that don't force Americans to make choices between safety and affordability, all of us involved in policy cannot rest," he said. A solution needs to come from Congress in tandem with the FDA "right now," he added.
For fifty-five years, the system of drug safety in the U.S. has been based on scientific studies, and when people go outside the FDA's regulatory system they may be buying the equivalent of snake oil, he warned. The FDA has legitimate safety concerns about the drugs imported from elsewhere, according to McClellan.
While the FDA tries to tackle price issues, it also needs to ensure that approved drugs here are as safe as possible. The FDA is taking specific new steps to improve the quality review process, he said. Once new medical treatments have been developed, the FDA also needs to do more to ensure that it is using the products effectively and deriving more value from them—by monitoring approved treatments for safety problems and differences in effectiveness among patients.
The FDA is also paying close attention to the potential of pharmacogenomics and the study of differences in how people respond to drugs. Better analysis of pharmacogenomic data will help mitigate risk, he said. Improved reporting systems and electronic medical records are also essential, according to McClellan.
"True healthcare reform requires better information. Modern information technology has been slow in coming to healthcare," he said. Electronic prescribing and electronic drug labels are the wave of the future, he predicted.
He also wants to see increased availability of low-cost generic drugs after patents have expired. Even though a generic may have been around for years, the FDA needs to continue to play a role in monitoring its safety, he said. "Make no mistake, generic biologics are coming worldwide. The FDA has to be ahead of the curve" on scientific standards to make sure they are acceptable, he said.
As for direct-to-consumer (DTC) advertising, McClellan acknowledged that such ads sometimes do play a role in educating people about illnesses and treatments. Some people may read about a medicine or watch an ad on television and use it as an impetus to see their doctor when they wouldn't have otherwise, which is probably for the good. At the same time, however, the FDA needs to carefully weigh the risks of such forms of advertising.
Still, this is a good time to be in the healthcare field. "It's never been more important for public and private sector leaders to come together as they have the opportunity to do at a conference like this one, to exchange ideas and contribute directly to the advancement of the public health," McClellan said.
McClellan was appointed to his post one year ago by unanimous consent of the U.S. Senate. Prior to his appointment to the FDA, he taught both economics and medicine at Stanford, was a practicing internist, and was director of the Program on Health Outcomes Research at Stanford University. In 2001 and 2002, he served in the White House as a member of the President's Council of Economic Advisers and was a senior policy director for healthcare and other economic issues for the White House.