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Reforming U.S. Regulation to Achieve Better Science

May 21, 2014
A bipartisan initiative known as “21st Century Cures,” spearheaded by the House Energy and Commerce Committee, is aiming to keep the U.S. at the forefront of biomedical innovation.
The U.S. bio-phrama industry’s distinction as No. 1 hasn’t gone unnoticed in the rest of the world, says “A Path to 21st Century Cures,” a white paper that describes the initiatives main objectives. “[O]ther nations are now actively working to gain a competitive edge in various elements, whether through a focus on basic research or a streamlined approval process to bring new treatments to market more quickly,” according to the report.

Committee Chairman Rep. Fred Upton, (R-Mich.), and Committee member Rep. Diana DeGette (D-CO) said there is a “common goal” of “accelerating the pace of cures” in the U.S. and to accomplish that mission will mean “working together and taking a comprehensive look at the full arc of accelerating cures, from discovery to development to delivery and back again.”

“Health research is moving quickly, but the federal drug and device approval apparatus is in many ways the relic of another era,” DeGette said in a video introducing the initiative. “We have dedicated scientists and bold leaders at agencies like the NIH and the FDA, but when our laws don’t keep pace with innovation, we all lose.”

Upton’s own remarks in the video noted the importance of reforming the current system “to make sure there’s not a major gap between the science of cures and the way we regulate these therapies.”

Figuring out how to close that gap is an on-going conversation, the white paper says.  To help foster discussion and uncover the most innovative solutions the committee will issue a series of white papers seeking input and soliciting ideas on how Congress can help quicken the pace of the innovation cycle that breaks down into three major areas: discovery, development and delivery of new treatments to patients.


Discovery is the first part of the innovation triad.  While the U.S. led the Human Gnome project a decade ago, today that leadership “is being threatened as other countries contribute more to basic research from both public and private sources,” according to the white paper. For example, the Beijing Genomics Institute is now the world’s largest genetic research center and produces 25 percent of the world’s genomic data.

How can the U.S. “collectively ‘connect the dots’ more quickly and start developing potential therapies and cures?” the paper asks.

This phase of innovation may be the most vital. At its core, development entails translating ideas into reality, “with the goal of bringing treatments to patients and being rewarded for the pursuit,” according to the paper.

Other countries are taking steps to attract innovative companies. The paper notes that some years ago an average of 40 biotech companies a year were spawned in Cambridge, Mass. alone. Today that number has dropped to 15. In 2010, China created more biotech companies than the U.S. “How are other countries attracting companies and investment? Should we adopt some of those policies, too? What else can we do to lead the way?” the paper asks.

Also in the cross hairs: the arduous clinical trial process required by the Food and Drug Administration. The time, size, failure rates, and costs of trials are at an all-time high, the paper notes, asking what can be done to reverse the trends.

And with costs of developing a new drug now sitting at $1 billion, the paper asks if the current economic incentives and policies are in place to sufficiently encourage future investment and innovation.

FDA approval of a new drug for market isn’t the end. In many ways it’s just the beginning of a drug’s life, the paper says. Different uses for the drug are often discovered after it has been approved by the FDA. The paper asks whether social media and modern data analysis can be better harnessed to play a role in finding new uses for drugs.

“There are reports that diagnostic testing breakthroughs sit unrealized due to regulatory uncertainty and other market forces that deter translating such innovation into patient-centered solutions,” the paper says. “Overall, the policies we have in place must allow for delivery to serve as a platform for new discovery and development. Any legal or regulatory framework that slows this cycle is not only ignoring reality, but also discouraging hope.”

The Committee hopes to deliver reform legislation next year addressing all these issues, having been informed by the answers to the questions floated in the series of initiative white papers.

Reforming U.S. Regulation to Achieve Better Science was originally published on Ideas Lab