My name is James Myers. I am a kidney/dialysis patient from Indiana. I am an Advocate for the National Kidney Foundation and a member of the Kidney Action Committee. This Bill passed the House on July 10, 2015. It now goes to the Senate for their consideration.

21st Century Cures Act Passes House, Now Must Navigate Senate



Alex Lash

July 10th, 2015

Follow @alexlash

National

The U.S. House of Representatives took a big step this morning toward major changes in the federal government’s relationship to biomedical research, funding, and regulation. The chamber passed the 21st Century Cures Act, a bill championed early on by a Democrat and a Republican, by a vote of 344-77, an astonishing percentage in this age of partisan acrimony.

The bill aims to speed the development and approval of drugs and devices; provide more financial carrots for making products for small patient populations; and stabilize federal funding for basic biomedical research.

Passage in the House was due in no small part to the bill’s original sponsors, Reps. Diana DeGette (D-CO) (pictured) and Fred Upton (R-MI), whose work—appearances, town halls, info-gathering sessions—for more than a year leading up to the vote at times resembled a permanent campaign.

But the Cures Act still must get through the Senate, where single opponents can often hold popular legislation hostage. And, despite the strong support in the House, critics are asking if the rush to approve new drugs might tilt away from traditional safeguards too much. Fiscal hawks will no doubt pick through the costs associated with the bill, as reported last month by the Congressional Budget Office.

If it does get to President Obama’s desk, the act will likely look different than it does today. It has already been through several edits, at one point earlier this year getting cut back from 400 to 200 pages long.

As it stands today, these are some of its major goals:

—Reauthorize the National Institutes of Health and including an extra $1.9 billion a year for a new “innovation” fund focused on precision medicine and young scientists. (The White House this week applauded the new funding but raised concerns that it would come at the expense of other items in the federal budget because of the spending caps imposed by sequestration.)

—Requiring FDA to make new tools and concepts—such as biomarkers, new clinical trial designs, and surrogate endpoints—easier for drug makers to incorporate into their product development.

—Speed up the regulatory pathway to approve biomedical products, including antibiotics to treat drug-resistant bacterial infections.

—Extend the patent protection for drugs that treat rare diseases by six months and extend through 2018 the priority review voucher program that grants companies a sped-up drug review as a reward for bringing to market a treatment for a rare pediatric disease. (Xconomy reported on an example of the voucher program here.)

—Establishment of a public-private oversight committee, dubbed the Council for 21st Century Cures, that would bring together the heads of the NIH, FDA, and other government agencies with patient advocates, industry representatives, and health care providers.

http://www.xconomy.com/national/2015/07/10/21st-century-cures-act-passes-house-now-must-navigate-senate/

From the NKF:

Victory with Passage of 21st Century Cures Bill

Posted on July 10, 2015 by nkf _advocacy

"We’re pleased to announce that the 21stCentury Cures Bill has passed in the House of Representatives with a 344-77 vote. The bill guarantees increased NIH funding over five years and will ensure greater investment in research, including research on kidney disease.

Here are some of the key provisions that were included in the 21st Century Cures Bill:

An $8.75 billion increase in research funding over five years at the National Institutes of Health (NIH)

This new money will give NIH the resources it needs to engage in research that can lead to new cures and treatments for the millions of Americans living with chronic conditions.

Patient Focused Drug Development

Allows a pathway for patient’s experiences to be collected and used by the FDA when determining the risks and benefits of a product prior to its approval. Patients know best how their disease affects them and how much risk and what type of risk they would be willing to trade for added benefit. This provision will help ensure the FDA takes patients into account when making drug and device approval and labeling decisions.

Surrogate Endpoint Qualification and Utilization

One of the biggest barriers to the approval of new kidney disease drugs is the length of time it takes to reach the current FDA endpoint. An endpoint refers an outcome that can be measured as a target in clinical trials, when a direct relationship can’t be measured through a real endpoint, a surrogate endpoints may be used in some cases to establish a relationship between the drug and patient outcomes. For kidney disease, the surrogate endpoint has typically been the doubling of serum creatinine to replace the outcome of kidney failure (57% decline in kidney function based on glomerular filtration rate). The trial design required to reach that surrogate endpoint can take well over five years and are excessively expensive to conduct, making studies in CKD drugs less attractive to manufacturers. In December 2012, NKF and the FDA co-hosted a workshop to discuss potential surrogate endpoints for clinical trials. They concluded that in some kidney disease populations, a 30 or 40% decline in kidney function was adequate to show a high risk of mortality and progression to ESRD. Following that meeting, another study showed that the lesser decline in kidney function, such as 30% over two years, is a strong predictor of progression to ESRD or higher mortality. A consistent and transparent process for how FDA considers and approves the use of new surrogate endpoints may help spur more clinical trials in kidney disease.

Continuing Medical Education Sunshine Exemption

Continuing medical education (CME) is an important component of spurring new ideas for research and innovation. This provision will make it easier for organizations like NKF to provide CME opportunities to practitioners and for practitioners to easily access peer-reviewed journals and medical literature they need to stay on top of new research and studies.

The passage of this bill would not have happened without for the dedication of NKF’s thousands of supporters and our Kidney Action Committee members. To all of you who wrote letters and called your representatives, thank you for ensuring that kidney patients have a voice in this new legislation."

https://nkfadvocacy.wordpress.com/2015/07/10/victory-with-passage-of-21st-century-cures-bill/#more-1286