The Senate has passed the Food and Drug Administration Reauthorization Act (FDARA), a legislative package that supplements FDA funding by requiring manufactures to pay user fees when submitting medical products for review in exchange for a timely approval process.
As we noted in a news brief last month, the law firm of Hyman, Phelps & McNamara P.C. has featured several blogposts on different provisions of 21st Century Cures Act. They will host two free webinars this month to further examine the Act, which will be good opportunities for advocates to learn more and ask questions.
The first webinar, to be held on January 12th from 12-1:30pm (EST), will focus on the pharmaceutical and biologics provisions of the Act. The second, to be held on January 18th from 12-1:30 PM (EST), will focus on the device and combination products provisions.
The FDA Law Blog, the official blog of the law firm of Hyman, Phelps & McNamara P.C., recently took a closer look at several sections of the Act. For analysis of the provisions related to medicine, click HERE (part 1) and HERE (part 2). Analysis of the provisions related to medical devices can be found HERE and analysis of drug-device combination products (such as a surgical mesh with an antibiotic coating) can be found HERE.
We will share any analysis of the potential impact of the 21st Century Cures Act as we find it.
NEVER doubt that your voice as a rare disease advocate matters! President Obama just signed the 21st Century Cures Act into law, after it passed both the House and Senate with broad bipartisan support. This would not have been possible without advocates from EveryLife Foundation for Rare Diseases, Global Genes, National Organization for Rare Disorders, Inc. (NORD) and many disease-specific organizations who called, emailed and met with Members of Congress in the past year and a half.
The 21st Century Cures Act includes:
- $4.8 billion in new funding for the National Institutes of Health (NIH)
- $500 million in new funding for the Food and Drug Administration (FDA)
- Formally establishes the Precision Medicine and Cancer Moonshot initiatives
- Reauthorization of the Rare Pediatric Disease Priority Review Voucher program through 2020
- Funding for the establishment of a national neurological disease surveillance system coordinated by the Centers for Disease Control and Prevention (CDC)
- Improved biomarker qualification
- Allowances for the FDA to recruit and retain additional specialized employees
- Strengthened patient engagement at the FDA through the Patient Focused Impact Assessment Act
- A regenerative medicine designation to allow such products to qualify for priority review and accelerated approval
- Provisions to foster programs to improve mental health and deter substance abuse
These provisions and additional funding would boost our nation’s research capacity and help modernize the drug review and approval process at the FDA.
WASHINGTON, DC – Following the Senate’s passage of the 21st Century Cures Act on Wednesday by a vote of 94 to 5, the bill was sent today to Speaker Paul Ryan (R-WI) before moving to the White House for the president’s signature. The House passed the game-changing medical innovation bill on November 30, by a vote of 392 to 26. Both Speaker Ryan and Senate Pro Tempore Orrin Hatch (R-UT) officially signed the bill at this morning’s Enrollment Ceremony.
“This effort has always been about the patients, and I’m so glad that we could have our friend, all-star Cures advocate Max with us today. Not letting rare disease hold him back, this pint-sized dynamo has been with us every step of the way on the #Path2Cures,” said Energy and Commerce Committee Chairman Fred Upton (R-MI). “We look forward to seeing President Obama make #CuresNow law next week. As Max said today, ‘Cures is more than hope, it’s action.’ Next stop, the White House!”
Taylor’s Tale, a Charlotte-based advocacy group in the fight against Batten disease and other rare diseases, spearheaded new legislation in North Carolina to create a new advisory council housed within the UNC-Chapel Hill School of Medicine to work towards finding more treatments for rare diseases.
North Carolina House Bill 823 will establish the council that will give guidance on research, diagnosis, treatment and education related to rare diseases. The council will consist of rare disease advocates including providers, researchers and patients.
In short, the council is directed by the state to, “Advise on coordinating statewide efforts for the study of the incidence of rare diseases within the State and the status of the rare disease community.”
It can be hard to find a specialist who knows how to treat your rare disease. Disease advocacy groups, rare disease organizations and genetics clinics may help you to find one. A partnership between advocacy groups including Taylor’s Tale, academic medical centers, the National Institutes of Health and others keenly interested in the diagnosis and treatment of rare diseases can be a model for the nation as they tackle some of the most challenging diseases affecting our citizens. To learn more visit taylorstale.org
RDLA applauds the House for approving the 21st Century Cures Act by a strong, bipartisan vote of 344 to 77. This landmark legislation will spur scientific discovery and bio-medical innovation, providing hope for patients and their families, while also protecting jobs and America’s leadership role in healthcare innovation.
Attention now turns to the Senate, and we look forward to working with Senators on their ideas and priorities for creating treatments and cures for rare diseases. We encourage the Senate to move quickly in advancing this legislation through the chamber and to the president’s desk.
If you’d like to know more about the contents of the 21st Century Cures Act, click here for our information page on the legislation.
In April, President Obama signed the Gabriella Miller Kids First Research Act authorizing Congress to allocate additional funding for pediatric cancer research through the National Institutes of Health (NIH). Congress, however, has not taken the steps needed to unlock the funding for NIH.
Today, December 10, 2014, Funding for the bill was included in the funding package to be passed by Congress.
Congratulations! This is a great example of what we can accomplish working together as a broad coalition.
If you took action by signing-on to our letter reminding congressional leadership to prioritize funding for rare diseases like childhood cancers THANK YOU!!!
From an Aug. 19 Valley Breeze article , Lincoln, Rhode Island: Legislation requiring insurance coverage for special baby formula and prescribed food is now law in Rhode Island.
The new measure abolishes the mandated cap on coverage for those suffering from rare diseases and inherited disorders requiring prescribed nutrition mandates. The bills sponsored by state Sen. Ryan W. Pearson of District 19 in Cumberland and Lincoln, and state Rep. Raymond A. Hull of District 6 in Providence and North Providence, induced testimony from patients and family members of patients who require prescribed diets and formulas during the hearing process. Read more
State right-to-try laws, which have been signed by a Democratic governor in Colorado and a Republican in Louisiana, are responses to deep dissatisfaction with the pace of drug development and frustration with the ways biopharma companies have responded to pleas for early access to experimental drugs. Read more