President Barack Obama Dec. 13 signed the 21st Century Cures Act, a bill that changes regulatory standards at FDA, slates additional research funds for NIH, and authorizes $1.8 million over seven years for Vice President Joe Biden’s National Cancer Moonshot Initiative.
The legislation, a $6.3 billion health care reform measure designed to accelerate drug development and modernize clinical trials, was signed into law during an unusual legislative period.
The federal government is running on its second FY 2017 continuing resolution, which was approved by a lame-duck, GOP-led Congress determined to align funding priorities with President-Elect Donald Trump’s administration (The Cancer Letter, Nov. 18).
“I started the 2016 State of the Union address by saying we might be able to surprise some cynics and deliver bipartisan action on the opioid epidemic,” Obama said at the signing ceremony Dec. 13. “And in that same speech, I put Joe in charge of mission control on a new Cancer Moonshot.
“And today, with the 21st Century Cures Act, we are making good on both of those efforts. We are bringing to reality the possibility of new breakthroughs to some of the greatest health challenges of our time.”
Biden praised lawmakers for their bipartisan work on the bill. The moonshot initiative, renamed “Beau Biden Cancer Moonshot,” will be implemented through targeted, multi-year funding authorized through the Cures Act (The Cancer Letter, Dec. 9).
“When the president asked me last year at the State of the Union to head the Cancer Moonshot, we said we were going to ask you all for significant funding increases at the NIH and the NCI,” Biden said at the signing ceremony. “And you all stepped up again, Republicans and Democrats.
“As part of the moonshot, we set up what’s called a Blue Ribbon Panel to review what should be the scientific priorities as we tackle this to try to end cancer as we know it. We’ll try to do in the next five years what ordinarily would take ten years.”
Of the $4.3 billion authorized for NIH in the Cures Act, the moonshot immediately received $300 million under the current CR, the full amount available for cancer research in fiscal 2017. The Precision Medicine and BRAIN Initiatives respectively received $40 million and $10 million in FY 2017.
“This past week, we witnessed President Obama sign the 21st Century Cures Act into law,” Ellen Sigal, chair and founder of Friends of Cancer Research, said to The Cancer Letter. “This never would have been accomplished without the hard work of Reps. Fred Upton (R-Mich.) and Diana DeGette (D-Colo.) who have spent many years developing the bill, as well as Senators Lamar Alexander (R-Tenn.) and Patty Murray (D-Wash.) whose tireless dedication helped make sure the bill would be passed. For that, I, and the millions of patients across the country who will benefit from this bill, thank them.
“I see the signing of this bill as a monumental victory for not only patients, but Congress who put partisan politics aside for the sake of what’s right for patients. It was with Vice President Biden’s heroic leadership that FDA will now get essential resources for implementing innovative new programs, such as the FDA Oncology Center of Excellence, and patients will now be put first by operationalizing their input into research. Without the Vice President’s determination and fortitude, critical initiatives, such as the Cancer Moonshot, the President’s Personalized Medicine Initiative, and the BRAIN Initiative at NIH would not be funded.”
FDA: We Will Have an Implementation Plan
FDA received $20 million in FY 2017, out of the $500 million slated for FDA over the next nine years to enable competitive recruiting and to fund legislative changes to FDA’s regulatory processes.
The latter sparked vocal opposition to the Cures Act by a number of Democrats and consumer advocacy groups (The Cancer Letter, Dec. 2).
“Cures will greatly improve FDA’s ability to hire and retain scientific experts,” wrote FDA Commissioner Robert Califf in a blog post Dec. 13. “One of our ongoing challenges has been recruiting and retaining the experts we need in specialized areas to allow us to get our work done and meet our growing responsibilities.
“Cures will also support our efforts to modernize and improve efficiency in clinical trial design. This has been an important FDA priority for decades, but exciting new approaches are now available, and we need to develop a common understanding of which designs should be used for which clinical issues.
“In cancer, for example, we’re already weighing the use of common control trials, which share a control arm, involve multiple different drugs for the same indication, and may even involve different companies. One of the benefits of using a common control arm is that the overall number of patients who need to be recruited and enrolled decreases, thereby optimizing clinical trial resources and potentially shortening the time it takes to get a new study off the ground.”
The Cures Act, with provisions defining novel clinical trial designs and the use of real world evidence in the regulatory setting, has “significant implications” for the agency, said Janet Woodcock, director of the Center for Drug Evaluation and Research.
“The 21st Century Cures Act increases funding for NIH, including the ‘Precision Medicine’ initiative, and provides a billion dollars over two years to the states to supplement opioid abuse prevention and treatment activities,” Woodcock wrote in an email to CDER staff Dec. 15. “It also contains a number of provisions that impact the medical product Centers at FDA. The Act provides $500 million over 10 years to FDA to carry out activities in Title III of the Act, which is focused on medical product development.
“Title III has provisions on patient-focused drug development, qualification of drug development tools, continuous manufacturing of pharmaceuticals, novel clinical trial designs, use of real world evidence in the regulatory setting, and antimicrobial drug development, among others. It also has a section on expansion of the Senior Biomedical Research Service, and additional hiring authorities for FDA.
“CDER leadership has been working with the administration and Congress in providing technical assistance for many of these provisions, so they are familiar to us. A number of them reflect initiatives we have been working on for many years, such as the qualification processes. By instantiating these in statutory provisions, Congress is effectively making these part of our mission.”
The agency will share its implementation plan for relevant provisions in the Cure Act, Woodcock said.
“The agency, and CDER, are currently conducting a detailed analysis of the provisions of this Act. Once we have them analyzed and have an implementation plan, we will share this with everyone,” Woodcock wrote in the email. “I believe that the patient-focused drug development and drug development tools provisions will have the most significant impact on our work, in the sense that they will require additional processes and procedures to be put into place. We have begun considering how to do this.
“I want to thank Associate Director for Legislative Affairs Bob Guidos for spearheading this complex effort. Bob played a key role in our interactions with Congress, and was particularly instrumental in stressing our need to hire and retain high-quality scientific and professional staff. I believe he made a real difference.”
ASCO: Cures Addresses Data Interoperability
The Cures Act reduces some of the major barriers to advancing cancer research, said Clifford Hudis, CEO of the American Society of Clinical Oncology.
“This historic legislation brings new hope to millions of Americans facing life-threatening diseases and to their families,” Hudis said in a statement. “Enacting 21st Century Cures is a momentous achievement and, potentially, an important pivot point for cancer research progress.”
The Cures Act contains priorities that ASCO has advocated for, including:
- Addressing the interoperability of electronic health records and restricting intentional information blocking to make it easier to coordinate patient care and advance big data and precision medicine efforts,
- Requiring drug companies to make available public information about their expanded access plans after Phase II and Phase III clinical trials so that patients and providers can more easy get access to promising new treatments,
- Improving the way research is conducted by requiring the use of centralized Institutional Review Boards when appropriate, encouraging data standardization, and bringing the patient voice into the drug development process, and
- Additional provisions of interest to ASCO members, including providing a site neutrality exemption for certain cancer center outpatient departments, increasing transparency around the Local Coverage Determination Process, advancing guidance on incorporating novel clinical trial design into new drug applications, and authorizing funding for states to address the opioid crisis, among other measures.
“As the vice president has highlighted throughout 2016, we are at an inflection point in cancer research, which is the result of decades of dedicated efforts to increase our knowledge and understanding of the more than 200 diseases called cancer,” said Nancy Davidson, president of the American Association for Cancer Research and executive director of the Fred Hutch/University of Washington Cancer Consortium. “There are 15.5 million cancer survivors who are alive today because of cancer research.
“Today’s action by President Obama provides us with a down payment for the resources necessary to save more lives from cancer.”