The second draft of the proposed 21st Century Cures Act—which aims to accelerate the discovery, development and delivery of next-generation health products—includes a significant increase in NIH funding, expansion of an FDA patient-focused drug development initiative and removal of some drug exclusivity provisions.
The new draft—which at 200 pages is half the size of the 400-page original version released in late January—has been presented and discussed before the House Energy & Commerce Committee. Additional hearings on and changes to the draft are expected in the coming weeks, before the legislation is submitted to Congress to act on it.
The most welcomed portion of the updated draft called for increases in NIH funding to almost $32 billion for fiscal 2016, $33 billion for fiscal 2017 and nearly $35 billion for fiscal 2018. That includes a new $10 billion NIH Innovation Fund ($2 billion per year for five years) beginning in 2016, intended as a mandatory program and not subject to the appropriations process.
However, the updated draft, while it could stabilize NIH funding, leaves out additional dollars for the FDA.
“There is widespread support on additional NIH funding,” said Craig Burton, a director at Avalere Health, a policy advisory firm focused on healthcare business strategy and insights, who has closely followed the iterations of the 21st Century Cures legislation. “We and other observers pointed out—along with the FDA—that resources are an issue. If you are giving new responsibilities to the FDA, you need to ensure that the resources are commensurate with the funding.”
The draft also includes a range of policies to strengthen the development of new treatments and cures, and improves the transition from discovery to development and from development to delivery. They include provisions to incorporate the patient perspective into the drug development process; foster the development, qualification and utilization of biomarkers; and modernize clinical trials.
“We are encouraged that these themes have been addressed in this legislation and look forward to providing additional feedback on specific proposals as we evaluate the details of this draft,” said Janet Woodcock, M.D., the FDA’s director of the Center for Drug Evaluation and Research in joint testimony with Jeffrey Shuren, M.D., FDA director of the Center for Devices and Radiological Health.
The draft’s provisions changing Section 114 of the FDA Modernization Act (FDAMA) to allow greater scientific and economic evidence-based communications between biopharmaceutical companies and payers have drawn praise from industry.
“There is legislative language in this draft that gives a little more clarity over how manufacturers engage with payers and is intended to develop data that are not reflected on the label but speak to the economic value of a product and are shared with formulary committees,” said Burton. “This is not off-label promotion. Let’s say a product shows it can reduce hospital re-admissions. As a manufacturer, this is worth something to payers and formulary committees. Over the years, the challenge since FDAMA was enacted has been a lack of FDA guidance on this issue. That would change under this new draft.”
Another major part of the 21st Century Cures draft is its attempt to attract patients into the drug development and approval process. While the FDA has a Patient-Focused Drug Development initiative, the new draft seeks to expand that program and would require the FDA to provide greater clarity in how it incorporates those patient perspectives as part of the clinical trial review process. The FDA also would explain what it would do with that accumulated information, prompting it to formally issue guidance and create a framework for the use patients’ views in the drug review process.
The draft also raises concern about a lack of increased FDA funding despite increased responsibilities. Burton maintains the Energy and Commerce Committee is sending a signal to appropriators that the NIH should be given more money. The NIH language doesn’t actually hike NIH funding but simply reauthorizes the agency at higher spending levels, contingent on congressional appropriations, he said.
“We are hopeful that the committee speaks to the question of the FDA needing more money for its role in the proposed changes,” said Steven Grossman, deputy executive director of the Alliance for a Stronger FDA, an organization of pharma companies, patient groups and health professionals that supports the FDA’s capabilities through adequate funding.
He cited concerns about where the additional dollars will come from to fund the FDA under the proposed legislation, along with other yet-to-be-resolved issues, such as NIH’s drug repurposing initiative. Those are among a number of placeholders that will be decided in the coming months.The new draft has gained new support from Democratic lawmakers including Reps. Frank Pallone (D-N.J.) and Gene Green (D-Texas).
As for the urgency to finalize the draft and submit it to Congress, Committee Chairman Rep. Fred Upton (R-Mich.) said the bipartisan legislation must be completed this year and include subcommittee and full committee markups His colleague, Rep. Diana DeGette (D-Colo.), said there still are many places in the draft where detailed language issues are not resolved.
Still, there is significant optimism. House Health Subcommittee Chairman Joseph Pitts (R-Pa.) called the legislation a work in progress, saying the 21st Century Cures Act is a “significant step forward in closing the gap between what the initiative calls ‘the science of cures’ and the regulation of therapies in the U.S.”
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