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By Jeannie Baumann
Jan. 27 — A House panel Jan. 27 offered a first look at its legislation to accelerate the discovery, development and delivery of promising new treatments and cures.
The legislation has proposals for new advisory bodies and approval pathways but doesn't mention funding increases, according to the discussion document from the House Energy and Commerce Committee.
The discussion document and accompanying summary and white paper culminate a year of hearings, roundtables and white papers under the 21st Century Cures initiative that is designed to transform the clinical trials process and ultimately speed the pace of new medical cures and treatments. Energy and Commerce Committee Chairman Rep. Fred Upton (R-Mich.), who launched the initiative last April with Rep. Diana DeGette (D-Colo.), called the draft a critical first step in the legislation process.
“Our solutions to boost cures and jobs are starting to take shape as we move from broad principles to legislative language. However, this document is far from the final product. Some things may be dropped, some items may be added, but everything is on the table as we hope to trigger a thoughtful discussion toward a more polished product,” Upton said.
DeGette said in a Jan. 27 statement she appreciates Upton's efforts but stopped short of endorsing the legislative draft: “While I don’t endorse the draft document, I know that with continued engagement, we can reach a bipartisan consensus to help advance biomedical research and cures.”
House Energy and Commerce Chairman Fred Upton said that “this document is far from the final product. Some things may be dropped, some items may be added, but everything is on the table as we hope to trigger a thoughtful discussion toward a more polished product.”
Last year, Upton, DeGette and other members of the committee said they wanted to introduce legislation by the end of January. In releasing the draft document a few days before that deadline, the Energy and Commerce Committee said it will “continue on an aggressive schedule to introduce 21st Century Cures legislation and ultimately send a bill to President Obama’s desk for signature by the end of the year.”
The discussion document covers a wide range of topics from the regulation of drugs and devices and modernizing the clinical trials process to data sharing, Medicare coverage of new technologies and health information technology interoperability.
All provisions of the legislation, according to the discussion document, would fall under five titles:
• putting patients first by incorporating their perspectives into the regulatory process and addressing unmet needs;
• building the foundation for 21st century medicine, including helping young scientists;
• modernizing clinical trials;
• accelerating the discovery, development and delivery cycle and continuing 21st century innovation at the National Institutes of Health, the Food and Drug Administration , Centers for Disease Control and Prevention and the Centers for Medicare & Medicaid Services; and
• modernizing medical product regulation.
Rep. Frank Pallone Jr. (D-N.J.), the top Democrat on the Energy and Commerce Committee, said he was disappointed because the discussion document didn't reflect true bipartisanship.
“In its current form, I am concerned that the nearly 400 page draft could create more problems for our health care system than it solves. Further, the draft does not include any real dollars to fund additional basic research at the National Institutes of Health. Increased funding was a common theme during last year’s public engagement, from both sides of the aisle, and is fundamental to truly advancing 21st century cures,” Pallone said.
“Moving forward, I stand ready to work with Chairman Upton, and all Members of the Energy and Commerce Committee, to find bipartisan consensus legislation that would pass the House and the Senate and ultimately be signed by the President,” Pallone said.
The draft would require the FDA to establish a structured framework “for the meaningful incorporation of patient experience data into the regulatory decision-making process, including the assessment of desired benefits and tolerable risks associated with new treatments.”
There also is a proposal to create an advisory body similar to the Medicare Payment Advisory Commission that would advise Congress on issues related to the discovery-development-delivery cycle. The bill also would establish an accelerated approval pathway for medical devices similar to the breakthrough pathway that already is in place for drugs at the FDA, and allow the FDA to accept and review data summaries rather than full data packages.
The committee said in the discussion draft that it is working on language to clarify what information can be shared about experimental drugs and devices to physicians, insurers and researchers.
“The FDA’s current rules and policies governing what drug and device developers may say about their own products were designed decades ago. Since then, the way that medicine is practiced and delivered and the way that information is communicated have fundamentally changed,” the discussion document said.
The bill also would require FDA to issue a guidance document within a year of enacting the legislation on the review process for combination products, which have components of both drugs and devices. The proposal in the discussion document states that the agency center with primary jurisdiction for reviewing a combination product be the sole point of contact for the sponsor.
The discussion document language also includes several health information technology-related provisions, including a section to provide “regulatory certainty” for those who are developing apps and other health information technologies, along with another section to work toward national interoperable health information infrastructure.
While the discussion draft doesn't address funding at the NIH—which has experienced a nearly 25 percent decrease in its purchasing power over the past decade, a point that has been voiced by both research advocates and NIH Director Francis S. Collins—the discussion document includes language to require the medical research agency to issue a NIH research strategic investment plan, in which Congress would require the NIH to ensure at least 55 percent of the agency's budget supports extramural research, which occurs at universities and other research institutions. It is currently about 83 percent, according to figures from the NIH Office of Extramural Research . There also is a proposal to require data sharing by any NIH grantees, issue policies that help promote the careers of young scientists and foster high-risk, high-reward science, which typically doesn't get funded amid budget constraints.
There also is a proposal to require a single institutional review board to review multisite studies, and another proposal to allow clinical data registries to comply with Health Insurance Portability and Accountability Act “privacy and security law” in lieu of complying with the privacy and security provisions of the Common Rule governing human subject protection.
Attorney James Czaban, with Wiley Rein LLP, told Bloomberg BNA that the legislative draft has two key proposals that would change exclusivity rights under the Hatch-Waxman Act. First, he pointed to Section 1241, which the summary from the committee said would extend exclusivity for two years for significant improvements to “existing molecules” or drugs. The summary said, “These improvements could include developing new delivery systems, new drug combinations , and new formulations that lead to less adverse events and increase patient benefits and adherence.”
He also noted that Section 5001 would grant an extension of the 180-day exclusivity period for generic drugs manufactured in the U.S.
Czaban said, “These provisions would be controversial under any circumstances given the highly competitive landscape in the pharmaceutical industry, but they promise to be especially challenging for many companies to address given that more and more companies now have mixed branded/generic business models, and many generic companies manufacture products both in the U.S. and abroad. Thus, both of these provisions may be simultaneously beneficial and detrimental to the same company.”
Stephen J. Ubl, president and chief executive officer of the Advanced Medical Technology Association (AdvaMed), a devices industry group, applauded Upton and DeGette's leadership.
“The medical technology industry is central to the development of technologies and diagnostics that will provide the life-saving and life-enhancing treatments of the future. But the innovation ecosystem that supports our industry is severely stressed. Policy improvements are essential if America is to retain its world leadership and the potential for medical progress in this century of the life sciences is to be fulfilled,” Ubl said. “We look forward to reviewing the discussion draft in detail and continuing to work with Chairman Upton, Congresswoman DeGette, and other policy makers towards this important goal.”
Carrie Wolinetz, president of United for Medical Research, a coalition of nearly 30 patient groups, universities and private industry companies, said in a Jan. 27 statement that the draft represents a promising opportunity for passing meaningful legislation on National Institutes of Health funding and policy.
The 21st Century Cures initiative will help the NIH “restart its engine and ensure the U.S. keeps its title of world leader in medical innovation,” she said.
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The discussion draft is available at http://energycommerce.house.gov/sites/republicans.energycommerce.house.gov/files/114/Analysis/Cures/20150127-Cures-Discussion-Document.pdf. A summary is available at http://energycommerce.house.gov/sites/republicans.energycommerce.house.gov/files/114/Analysis/Cures/20150127-Cures-Discussion-Document-Section-by-Section.pdf.
The white paper is at http://1.usa.gov/1tmepsb.
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