Prize Competitions to Spur Medical Innovation and Patient Voice in FDA Approvals May Benefit under 21st Century Cures Act

In late 2016, Congress passed and President Barack Obama signed into law the 21st Century Cures Act, a sweeping medical innovation bill authorizing more National Institutes of Health (NIH) funding and supporting patient perspectives in U.S. Food and Drug Administration (FDA) approvals.

ASN advocated for these and other provisions in the new law, including calling on the NIH to support prize competitions to improve patients’ lives in fields where there is a significant disease burden, or where current investment is disproportionately small relative to federal costs. With 20 million Americans with kidney disease and over 600,000 with end stage renal disease (ESRD), Medicare spends over $80 billion annually providing care for kidney patients. The NIH investment in kidney research of $585 million is less than 1% of Medicare kidney care expenditures.

At the White House Organ Summit in June 2016, ASN pledged the first $7 million toward a prize competition to develop a novel wearable or implantable device that replaces kidney function and improves patient quality of life. The new law may facilitate collaboration with NIH as ASN seeks to launch the prize competition in 2017.

“21st Century Cures’ call for NIH to support prize competitions for the development of novel therapies could help revolutionize kidney patient care,” said ASN President Eleanor D. Lederer, MD, FASN.

The $6.3 billion bipartisan law is also designed to help find cures for cancer, provide $1 billion to fight opioid addiction, treat mental illness, and better understand the brain to prevent diseases like Alzheimer’s. Specifically, the Cures Act provides multiyear funding for three highly innovative scientific initiatives: the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative, the Precision Medicine Initiative (PMI), and the Beau Biden Cancer Moonshot. It also includes a promising new research initiative focused on regenerative medicine.

In a New England Journal of Medicine article titled “The 21st Century Cures Act—A View from NIH,” NIH Director Francis S. Collins, MD, PhD, and NIH Deputy Director for Science, Outreach, and Policy Kathy L. Hudson, PhD, write about measures in the Act that reduce red tape and nurture data sharing while protecting privacy.

“Sharing data is essential for progress in biomedical research,” Collins and Hudson write. “Rapid data sharing was key to the success of the Human Genome Project, and that same commitment has been spreading across biomedicine in the past two decades, as advances in technology and ‘big data’ have enabled an entirely new level of data sharing and inquiry. Despite the clear value of sharing data, the NIH has been constrained from requiring in a straightforward way that NIH-funded investigators share their data. The Cures Act solves this problem by allowing the NIH director to require that data from NIH-supported research be shared, giving all scientists the opportunity to use these data as quickly as possible to advance biomedical research.”

The Cures Act’s provision of support for patient perspectives in the FDA approval process is in line with an important aim of the Kidney Health Initiative (KHI). The KHI is a public-private partnership between the ASN and the FDA whose membership includes patient groups, health professional organizations, dialysis organizations, pharmaceutical and device companies, and government agencies.

“A pivotal aspect to be realized through the Act is the capacity to collaborate and promote appropriate innovations based on the experiences and perspectives of patients,” said James A. Sloand, MD, FASN, senior medical director at Baxter Healthcare and member of the board of directors for the KHI. “The mission of the FDA is to advance therapies and promote public health while ensuring the safety, efficacy, and high quality of novel drugs and medical devices. Given the chronicity and severity of kidney disease, patients’ knowledge, experience, and desire for self-determination are recognized as important factors in FDA deliberations. FDA has previously sought earlier and greater input from patients in the discernment process for drug and device approval. The 21st Century Cures Act supports this valuable exchange so that the patient’s voice is now fully heard and carefully considered in the product approval process.”

Sen. Lamar Alexander (R-TN), chair of the Health, Education, Labor and Pensions (HELP) Committee, called the bill a “Christmas miracle… that will help virtually every American family.” The Cures Act passed the Senate by a vote of 94–5 and the House by a vote of 392–26.

To accomplish such large bipartisan votes in both chambers, the chairs of each committee in the Senate and House had to work closely with the ranking Democrats on their committees—and they did. ASN publicly thanked Senators Alexander and Patty Murray (D-WA) of the Senate HELP Committee and House Energy and Commerce Committee Chairman Fred Upton (R-MI) and Democrats Frank Pallone (D-NJ) and Diana DeGette (D-CO).