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Year of Cures yielding excitement, hope, innovation

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By Mari Serebrov
Regulatory Editor

The FDA and NIH are easily passing their first annual checkup of their implementation of the 21st Century Cures Act, but funding will be key to future progress.

"[A] law is not worth the paper it's printed on if it is not implemented properly," Sen. Lamar Alexander (R-Tenn.) said Thursday in opening the Senate Health, Education, Labor and Pensions (HELP) Committee's second oversight hearing on the Cures Act, which was signed into law a year ago to boost biomedical research at a time of what Alexander described as "limitless opportunity."

Thursday's hearing focused on the progress the FDA and NIH are making to unleash biomedical innovation. HELP's first oversight hearing, in October, looked at the implementation of the electronic health care record provisions included in Cures. The committee plans to look at the mental health care provisions of the law at a third hearing next week.

The excitement and potential of Cures are as great today as they were when the Senate voted 94-5 to pass the bill. "When the Senate passed Cures one year ago today, we hoped to unleash medical innovation and give Americans more access to life-changing treatments and cures, so more Americans could experience medical miracles," Alexander said. "It is not an overstatement to say that the 21st Century Cures Act has the potential to affect virtually every American family by taking advantage of breathtaking advances in biomedical research." (See BioWorld Today, Dec. 8, 2016.)

With its focus on accelerating the time from birthing an idea to getting a new therapy – be it a drug, gene therapy, diagnostic, device or combination product – to the right patient, Cures handed the FDA and NIH several new tools and authorities.

Echoing some of their testimony before a House committee last week, FDA Commissioner Scott Gottlieb and NIH Director Francis Collins regaled the HELP members with examples of how their agencies are using those authorities and making advances under Cures, ranging from the FDA's first approval last week of a breakthrough device to the forthcoming recommendations of the NIH's Task Force on Research Specific to Pregnant Women and Lactating Women and the full launch in a few months of the All of Us research program that aims to capture data from at least 1 million Americans as part of the Precision Medicine Initiative. (See BioWorld, Dec. 1, 2017.)

Dollars for research

While Cures is filled with hope and promise, it fell short on funding, Sen. Elizabeth Warren (D-Mass.) said at the hearing. She claimed the bipartisan legislation didn't send a single new dollar to either the FDA or the NIH. It merely opened the door for future Congresses to provide the funding to carry out the program.

To fill that gap, Warren said the Democratic members of the HELP Committee are reintroducing legislation to provide $50 billion to the two agencies to support new biomedical research.

Alexander, the HELP chairman, countered Warren's claim, noting that Cures provided $4.8 billion in a type of "hybrid funding" for the Cancer Moonshot, the Brain Initiative, the Precision Medicine Initiative and regenerative medicine research. He also reminded the committee that its job is policy; discretionary funding is up to the Appropriations Committee.

"For us to appropriate say $50 billion in new funding for the National Institutes of Health is a wonderful aspiration, but it's not what we do," Alexander said. "We decide, for example, whether Dr. Gottlieb should have a new breakthrough path for medical devices."

Alexander emphasized that the Senate, for the third year in a row, is pushing for a $2 billion bump in the NIH appropriations base. When a bump like that is approved, lawmakers anticipate it will continue at least for the next 10 years. Thus, three consecutive bumps of $2 billion would be expected to yield a $60 billion increase over a little more than a decade.

Alexander said he wanted researchers across the country "to know that this Congress, in a bipartisan way, for the last three years has been extraordinarily committed to significant increases in funding for the National Institutes of Health, and I think it's made a difference."

Financial reality

Despite the increase, the NIH is able to fund only 19 percent of the research applications it receives with its current $34 billion budget, Collins told the committee. In 2001, the agency was funding 30 percent to 35 percent of the applications.

No one asked whether the agency is receiving more applications today than it did 16 years ago, but Warren did ask how many of the applications are worthy of funding. The top third of the applications are all good science, Collins responded.

The grant numbers are a bit more dismal for new researchers, who are critical to the future of biomedical research. While there has been improvement in the number of first-time awards, thanks to Cures and other efforts, the agency is funding about 16 percent of applications from new researchers. Its goal is to fund 25 percent of those applications, Collins said.

Given the competition for NIH funding, there is a lot of anxiety in the research community and among science students. "I'm trying to reassure people that things are looking much better than they think they are. The sort of sense out there of trouble is probably beyond what it ought to be considering the path that we're on," Collins said.

There's a "great deal of reason to be excited and optimistic about a career in biomedical research – first of all because the science now is so incredibly promising and exciting," he said, adding that this is the golden era for science because of the opportunities being opened by technology and big data.

The second reason for excitement is the upward trajectory of government support for biomedical research, Collins said.