President Obama, in announcing an ambitious plan on Friday to invest hundreds of millions of dollars in cutting-edge biomedical research for treatments tailored to the genetic makeup of individual patients, said the effort would “lay the foundation for a new generation of lifesaving discoveries.”
In recent years, targeted therapies have led to more effective treatments for various cancers and other diseases, such as cystic fibrosis. The Food and Drug Administration has approved a growing number of specialized drugs for patients with specific genetic mutations, and the biotech industry has many more drugs in development.
The trend has benefited from sharp declines in the cost of genome sequencing. Decoding one person’s genome, which used to cost hundreds of millions of dollars, costs about $1,000. Storing massive amounts of data is cheaper than ever, and the world is increasingly connected by mobile technologies and electronic medical records.
Lawmakers from both parties have embraced the broad idea of speeding up medical innovation, even if the details about how best to do that remain unresolved.
“The really good news — this is how you know that the moment is right — is there’s bipartisan support for the idea here in Washington,” Obama said in the East Room on Friday morning, drawing a laugh from the crowd of renowned researchers, rare disease patients and officials from the biotech industry. “Which makes me very happy.”
Obama said he hopes to tap that bipartisan support and ask Congress for a $215 million for research on precision medicine. The request will be part of his annual budget scheduled for release Monday.
Still, for all its promise, the potentially staggering cost of some of the targeted drugs that have this kind of research has raised concerns.
Kalydeco, a drug approved in 2012 and highlighted by Obama on Friday, has done wonders for about 4 percent of cystic fibrosis patients with a specific genetic mutation. But it carries a price tag of about $300,000 a year.
“We want to be sure that when these drugs come to market, they aren’t priced at a level people can’t afford,” said John Rother, president of the National Coalition on Health Care, which represents insurers, employers and consumer and religious groups, among others. “That’s not the kind of world we want to live in.”
Drug makers and researchers note out that the cost of developing them is high, while the number of patients who benefit from them can be small. In addition, they say, creating treatments that profoundly change the course of a disease can save money by avoiding other long-term medical costs.
The centerpiece of Obama’s plan involves $130 million for NIH to develop a national database, containing the genetic profiles, medical histories and other data of a million or more willing Americans. That effort, which would likely combine patients from existing studies, is aimed at helping doctors and researchers better understand the complicated mechanisms underlying any number of illnesses.
“This research will dramatically advance our knowledge of diseases, how they originated and how we may prevent or treat them,” said Francis Collins, director of the National Institutes of Health.
Another $70 million would go to the National Cancer Institute to expand research on the genetic factors that can lead to cancer and to develop better treatments. Cancer, among the most pervasive and deadly diseases, is also where researchers have made some of the greatest strides in developing effective therapies for certain subsets of patients.
Under Obama’s plan, the FDA and the Office of the National Coordinator for Health Information Technology would receive $10 million and $5 million, respectively, to evaluate new innovations and put them on a fast track, and to build the technological infrastructure to share data while protecting patients’ privacy.