On Capitol Hill, New Initiatives for Medical Research Advance at Brisk Pace

New Capitol Hill and White House initiatives could bolster the budget of the National Institutes of Health (NIH), streamline drug approvals, and hasten the day when techniques of precision medicine are used more widely to treat cancer and other diseases, an expert panel said at the 40^th annual AAAS Forum on Science & Technology Policy.

The budgeting efforts, if successful, would help reverse a decade of near decline in funding from all sources for U.S. medical research.  E. Ray Dorsey, director of the Center for Human Experimental Therapeutics at the University of Rochester Medical Center, said that funding for medical research in the United States from all sources slipped to a compound annual growth rate of just 0.8 percent from 2004 to 2012, compared to an annual growth rate of 6.3% from 1994 to 2004. If only federal funding is considered, the picture is even bleaker. NIH sustained significant declines in purchasing power from 2004 onward, according to a AAAS analysis.

"U.S. funding for research has slowed for all sources, especially the NIH," said Dorsey, co-author of a January article in the Journal of the American Medical Association (JAMA) of U.S. and international spending patterns on medical research.

But the outlook could be about to change. Katie Novaria, a professional staff member for the House Committee on Energy and Commerce, said the panel — chaired by Rep. Fred Upton (R-Michigan) — is expected to mark up a "21^st Century Cures" bill in the next several weeks. The legislation, which could reach the House floor for a vote in June, would invest an additional $2 billion a year in NIH over a five-year period starting with the 2016 fiscal year. One aim would be to give the agency a "robust and steady stream of funding," Novaria said.

Elizabeth Farrar, a committee staff member and aide to Rep. Diana DeGette (D-Colo.), was optimistic that the legislation, which Upton and DeGette initiated, will pass the House. A discussion draft, released on 29 April, also includes provisions to better incorporate patient voices into decision making by the Food and Drug Administration (FDA) regarding the benefits and risks of new medical products.

Robert Cook-Deegan, research professor of public policy at Duke University, noted that the Senate Committee on Health, Education, Labor, and Pensions (HELP) also is considering legislation, based on an "Innovation for Healthier Americans" report released in January by Senators Lamar Alexander (R-Tenn.) and Richard Burr (R-N.C.). The report discussed ways the FDA may be able to get safe treatments and medical devices to patients more quickly. Alexander, chairman of the HELP committee, and ranking member Patty Murray (D-Wash.) have been holding hearings and have convened a bipartisan working group to examine the time and cost of FDA's drug and medical device approval process.

At the same time, the White House has been moving ahead on a Precision Medicine Initiative announced by President Obama in his State of the Union message in January. The administration has proposed spending $215 million on the initiative in the 2016 fiscal year. According to Carrie Wolinetz, associate director for science policy at NIH, the short-term focus of the initiative is to apply research advances in understanding genes, proteins, and human metabolism toward more targeted treatment of specific cancers.

Over the longer term, the initiative — if fully funded — would generate data needed to move precision medicine into virtually all areas of health and disease, Wolinetz said. The effort would include creation of a research cohort of more than a million volunteers who would agree to share their genetic data, lifestyle information, and other biomedical data that would be linked to their electronic health records. Among the goals of the study would be answering the question: Why do some people show resilience to illness even when their medical and family history suggests they should be developing a disease?

"There's a heck of a lot of stuff going on," said Cook-Deegan, who co-moderated the 30 April session at the policy forum on new initiatives in biomedical research along with Erin Heath of the AAAS Office of Government Relations. He noted that versions of the House, Senate, and White House initiatives were all announced during a single week in January, and all are moving forward at a fairly brisk pace in a town where partisan gridlock has become a fact of life.

While applauding the new initiatives, Cook-Deegan also said they tend to focus on providing more resources for R&D efforts that already work pretty well: development of new drugs and medical devices that can improve health outcomes. But he called for more attention to another part of the health care system that he termed "wildly inefficient" — how people get access to health care practitioners and health care services, how much care costs, and what happens to patients as a result of that care.

Such health services research, as it is called, accounts for less than 30 cents of every $100 spent on health care, according to Dorsey.

Health care providers and insurers, who stand to benefit perhaps the most from research on more efficient and effective ways to deliver care, invest the least on R&D among major industrial sectors, Dorsey said. The JAMA study he co-authored found that insurance carriers, as a group, spent only 0.04% of revenue on R&D in 2011. The pharmaceutical and biotechnology sectors spent closer to 15%.

Investment in new approaches to health care delivery is coming from non-traditional sources rather than established players, Dorsey said. "Last year, $4 billion in venture capital money went into digital health companies," he said. There has been rapid growth in mobile technologies to enhance health care, such as devices to record and transmit measures of heart function.

Dorsey noted that the pending "21st Century Cures" legislation includes a section on telemedicine which will exploit developments in mobile technology. He predicted that novel methods of health care delivery will continue to emerge from sources other than traditional providers.

Wolinetz said the Precision Medicine Initiative would provide opportunities for experimentation not just in biomedical science but also on ways to use new technologies and make the system work better.  With an increase in the use of electronic health records, she said, there "is a lot of thought going into interoperability of access to those health records."  She called it "a very exciting time" to see what will come out of experiments on mobile health technology and other innovations in health care delivery.

As for increasing the amount of spending on medical research, Cook-Deegan mentioned one idea incorporated into a Senate bill introduced by Sen. Elizabeth Warren (D-Mass.) in January. Under the proposed legislation, a drug company that engages in illegal sales tactics would be required to pay a fee amounting to 1% of its net income multiplied by the number of its "blockbuster" drugs that were developed in part with federal funding.

The collected fees would go to FDA for advancing regulatory science for medical products and to the NIH for research on diseases for which there are unmet medical needs and for other purposes. Warren estimates her "swear jar" proposal would have added roughly $6 billion a year to NIH's budget if it had been in effect for the past five years. But prospects for the bill are uncertain. The Pharmaceutical Research & Manufacturers of America, which speaks for the drug industry, has said it would siphon money from pharmaceutical research budgets and result in fewer medicines for patients.

Cook-Deegan said there is bipartisan support for another idea to help NIH. He noted that Newt Gingrich, a Republican who was Speaker of the House from 1995 to 1999, urged in a recent New York Times op-ed that Congress once again undertake a doubling of the NIH budget as it did in a five-year effort that started under President Bill Clinton and Republican majorities in Congress following the 1994 elections and finished under President George W. Bush.

"We are in a funny space here where the partisan labels don't necessarily map to the politics very cleanly when it comes to biomedical research," Cook-Deegan said.

[Credit for associated teaser image: Flickr/USCapitol]