Having shared my feeling about the 21st Century Cures effort making its way through the U.S. Congress (see my editorial for the December issue, of which this article here is a part), I thought I'd share the several comments from other organizations that have passed through my email inbox. The latest version of the bill easily made its way through the House of Representatives and, as of the time the December issue of the magazine went to printer, the Senate had just approved it as well.
What does the potential of that passage mean to some of the key life-sciences players out there?
Just prior to the passage of the Cures bill in the House, Avalere Health had some thoughts. Avalere is a strategic advisory company whose core purpose is to create innovative solutions to complex healthcare problems, delivering actionable insights, business intelligence tools and custom analytics for leaders in healthcare business and policy.
Breaking down the legislation, Avalere noted that key provision changes include the following:
- Increased funding levels for both the FDA and the NIH have been trimmed since earlier versions. New funding for the FDA is now at $500 million (previously $550 million) and NIH is now at $4.8 billion (previously $9.3 billion). Despite these reductions, funding for precision medicine and the BRAIN initiative remain in place.
- Provisions in this bill are now organized into more therapeutic areas, as well as class-specific. The legislation provides $1 billion in grants to states to help them combat opioid abuse. It also funds mental health and family services. Provisions related to regenerative medicine, antimicrobials and medical countermeasures were also added.
- Funding for the bill mainly comes from a drawdown of the Strategic Petroleum Reserve and other appropriations previously allocated to the ACA’s Prevention and Public Health Fund and territory funding. Other offsets include Medicare and Medicaid payment changes.
“Cures has generally had broad bipartisan support because many of its provisions are intended to help get medical products to patients faster without sacrificing FDA’s central safety and effectiveness mantra,” said Jay Jackson, manager at Avalere Health. “Provisions that were debated and not included in the current iteration of the bill, such as FDA guidance on off-label communication and laboratory-developed test regulation, may well reappear through further amendments, the upcoming user fee re-authorization and other regulatory or legislative actions.”
Kids v Cancer
Nancy Goodman, executive director of Kids v Cancer, was among those giving a thumbs-up to the passage of 21st Century Cures in the House and urging Senate approval as well. Of note to her was that the legislation includes wording on the Creating Hope Act Pediatric Priority Review Voucher Program. Specifically, pursuant to 21st Century Cures, the Creating Hope Act Pediatric PRV program will be extended to Sept. 20, 2020. Moreover, rare pediatric disease drugs that receive designations by that date will have until Sept. 20, 2023 to earn a voucher.
“This is a landmark step for our kids,” said Goodman. “Going forward, drug companies will continue to have an opportunity to develop drugs for children with cancer and other life-threatening illnesses. The Creating Hope Act works. It has generated almost $1 billion in industry-funded incentives already.
“21st Century Cures also includes support for other programs that benefit kids, including authorization for new funding for NIH, FDA, the Cancer Moonshot program (which includes provisions for pediatric research), and new mental health and drug abuse programs.”
Biotechnology Innovation Organization
The Washington, D.C.-based Biotechnology Innovation Organization (BIO) was also praising the progress of the bill. President and CEO James C. Greenwood noted, “The 21st Century Cures Act passed by the House of Representatives today is an important victory for patients and for the next generation of medical innovation. The legislation advances important patient-centered policies that can speed the pace of drug development, while authorizing essential funding for scientific discovery and the promotion of biomedical advancements that can help transform healthcare for patients with the promise of next-generation modern medicines.”
BIO is reportedly the world’s largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations.
American Society of Clinical Oncology
In a statement about 21st Century Cures, Dr. Daniel F. Hayes—president of the American Society of Clinical Oncology (ASCO)—wrote: “Today, by passing the 21st Century Cures Act, the U.S. House of Representatives took a significant step forward to accelerate the pace of bringing promising new treatments to patients, including the 1.6 million Americans diagnosed with cancer each year … The 21st Century Cures Act will provide a much-needed boost in supplemental funding to the National Institutes of Health (NIH) and the Food and Drug Administration (FDA) to support critical cancer research and accelerate the momentum of the Cancer Moonshot Initiative. The $4.8 billion for NIH and $500 million for FDA will help spur advances in cancer prevention, detection, diagnosis, treatment, and quality of life of patients.”
ASCO is also particularly pleased that the bill takes a step forward in addressing the interoperability of electronic health records (EHRs) and that the legislation puts restrictions on intentional information blocking.
“These much-needed improvements will make it easier to coordinate patient care across a variety of medical providers—and advance important efforts on Big Data and precision medicine. Big Data initiatives, such as ASCO’s own CancerLinQ, rely on the ability to share massive amounts of clinical data from large groups of cancer patients that provide treatment insights and improve decision-making in a clinical setting,” according to Hayes. “Increased interoperability of EHRs will make it possible for physicians to better coordinate patient care, reduce unnecessary and redundant cost and allow these big data initiatives to be more successful.”
Advocacy group Public Citizen has pretty much been against 21st Century Cures from the start, and its view was expressed the day of the House vote by Dr. Michael Carome, the group’s director of health research, as:
“The U.S. House of Representatives, after weeks of secret deliberations, today passed the dangerous 21st Century Cures Act. The bill has been sold erroneously as a commonsense, bipartisan compromise that enables scientific innovation and medical breakthroughs for America. But in reality, the legislation includes a grab bag of goodies for Big Pharma and medical device companies that would undermine requirements for ensuring safe and effective drugs and medical devices.
“Moreover, many House members were persuaded to support the bill, despite the many harmful provisions, in exchange for promises of increased funding for the National Institutes of Health (NIH). But there is no guarantee that this funding will be appropriated by Congress in future years. Permanently weakening the U.S. Food and Drug Administration in exchange for tenuous promises of increased NIH funding is a bad deal for patients.”