Free 21st Century Cures Tracker Available from FasterCures

FasterCures, a non-profit think tank with the goal of speeding and improving the medical research system, recently debuted a new resource to track implementation of the 21st Century Cures Act.

The free 21st Century Cures tracker focuses on the more than 100 sections in Division A, which include the key provisions on biomedical research:

“A few examples of the issues we will be tracking include the impact of changes to the National Institutes of Health’s administrative requirements for grantees, how the Food and Drug Administration implements new changes designed to enhance patient engagement and how the Department of Health and Human Services is using its new authorities to promote health information technology interoperability.

Many external factors will affect the progress of these initiatives, including policies related to hiring freezes and uncertain funding levels.”

FasterCures welcomes your feedback and suggested updates for the tracker, which can be submitted here.

Congress Begins Process of Reauthorizing Prescription Drug User Fee Act

Timely reauthorization of the Prescription Drug User Fee Act (PDUFA) was one of the hot issues discussed during the Legislative Conference during Rare Disease Week on Capitol Hill.

First enacted in 1992, PDUFA enables the Food and Drug Administration (FDA) to collect user fees from biopharmaceutical companies in order to enable the Agency to review the safety and efficacy of new medicines more quickly. According to PhRMA, it took FDA more than two years to review new medicines and more than 70% of medicines were approved outside of the U.S. before PDUFA.

Every five years, FDA and the biopharmaceutical industry negotiate a new user fee agreement, which Congress must enact in legislation. PDUFA was reauthorized in 2012 in the Food and Drug Administration Safety and Innovation Act, which also included provisions creating the Breakthrough Therapy designation as well as the Rare Pediatric Disease Priority Voucher program. PDUFA is due to be reauthorized this year, as the current user fee agreement expires on September 30th.

Released last year, the draft PDUFA VI agreement includes specific performance goals for drug review, proposed plans for enhanced use of biomarkers, expanded patient engagement, and improved specialization of reviewers for rare diseases.

The Senate Health, Education, Labor and Pensions Committee will convene a hearing on PDUFA reauthorization on March 21st at 10am ET with testimony from senior FDA leadership, and will be available by livestream. The House Energy and Commerce Subcommittee on Health will hold a hearing on PDUFA on March 22nd at 10:15am.

As discussed on the March webinar, it is important for PDUFA to be reauthorized by the end of July or FDA will need to send furlough notices to staff who review new medicines.

Learn More about 21st Century Cures Act Provisions on Cell and Gene Therapies in Free Webinar on February 2nd  

The Alliance for Regenerative Medicine (ARM) will host a free webinar on February 2nd at 1-2pm ET to provide an overview of the provisions of the 21st Century Cures Act concerning cell and gene therapies. The webinar will feature Michael Werner, Executive Director for ARM, as well as a representative from the U.S. Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research. Questions will be welcome.

According to ARM, the 21st Century Cures created a Regenerative Medicine/Advanced Therapy product designation, as well as regenerative medicine-specific language intended to optimize the FDA’s approval pathways for regenerative medicine products.
More information and a link to register is available HERE.

Learn More about 21st Century Cures Act in Two Free Webinars

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.

You can learn more and register at http://www.fdalawblog.net/fda_law_blog_hyman_phelps/2016/12/21st-century-cures-act-hpm-to-offer-two-complimentary-webinars-on-topics-of-interest.html.

A Deep Dive on 21st Century Cures

We previously highlighted the provisions in the 21st Century Cures Act of greatest interest to the rare disease community, but there were many more in the 312 page legislation.

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.

President Obama Signs Landmark 21st Century Cures Bill into Law

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.

Delivering #CuresNow: Speaker Ryan Signs Bipartisan Game-Changing Medical Innovation Bill

Dec 8, 2016
Press Release
Bill Officially Heads to The White House to be Signed into Law

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.


With Speaker Ryan’s signature, the bill now heads to the president’s desk.

“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!”

For more information on the 21st Century Cures Act, click HERE.

Visit C-SPAN to view the signing ceremony.

’Cures’ Research Package Draws Strong Bipartisan Vote

Originally published in Roll Call:

The House Wednesday night approved, 392-26, a sweeping biomedical research package that also aims to overhaul the mental health system and make targeted changes to Medicare.

Representatives passed an earlier version of the legislation, known as 21st Century Cures, last year, only to see it get delayed in the Senate over disagreements on mandatory funding for the National Institutes of Health and the Food and Drug Administration, among other things.

The revised measure is expected to have an easier path in the Senate this time, according to lobbyists and aides. The White House on Tuesday said it “strongly supports” the bill. Senate Health, Education, Labor and Pensions Chairman Lamar Alexander of Tennessee said the chamber would vote on the package early next week.

Sen. Patty Murray of Washington, the top Democrat on the HELP committee, said her colleagues are “getting very excited” about the bill.

“I think there’s been a lot of good changes made over the last 24 hours that makes me feel a lot more confident,” she said Wednesday.

One of the changes sought by Democrats and Republicans alike was to strike a provision related to federal disclosure requirements for physicians. The language in the updated bill would have exempted doctors from reporting certain compensation they received from pharmaceutical and medical device companies. That provision was dropped after opposition from Senate Judiciary Chairman Charles E. Grassley of Iowa, incoming Senate Minority Leader Charles E. Schumer of New York, and others.

Democrats were also able to add language that would direct money to combat opioid abuse to the states with the highest need.

Unlike the earlier bill, the House measure has no language protecting drugmakers’ patents for longer periods. And while the previous bill would have provided $8.75 billion in funding for the NIH over five years, updated language released last week would provide $4.8 billion over a decade for specified projects within the agency, including President Barack Obama’s Precision Medicine Initiative and cancer “moonshot” program.

The new legislation would also provide $500 million over nine years for the FDA. It would also provide $1 billion to the states to help fight the opioid epidemic.

Offsets for the bill would come mainly from the federal Strategic Petroleum Reserve and a fund created in the 2010 health care overhaul to promote disease prevention and public health.

To accommodate the concerns of Republicans in both chambers, sponsors revised a funding mechanism so that dollars would be set aside in what are referred to as “innovation” funds. Appropriators would then need to approve withdrawals from those accounts each year.

The change was met with some backlash from Democrats in both chambers.

“This bill authorizes the NIH for a quarter of the funding that was in the original bill that was passed in the House last year,” Massachusetts Rep. Jim McGovern said during a Rules Committee hearing on Tuesday.

Sen. Elizabeth Warren, on Monday, also blasted the Cures package as a giveaway to the pharmaceutical industry.

“When American voters say Congress is owned by big companies, this bill is exactly what they are talking about,” the Massachusetts Democrat said in a speech on the Senate floor. Senate Republicans have “let Big Pharma hijack the Cures bill. This final deal has only a tiny fig leaf of funding, for NIH and for the opioid crisis,” she said.

Patient-Advocacy Community Urges Congress to Move Forward with 21st Century Cures Legislation

Originally published on the National Health Council website:

Washington, DC (November 16, 2016) – More than 200 patient and research associations representing individuals affected by a broad range of diseases and disabilities sent a letter to Congressional leadership today, calling on them to pass the 21st Century Cures Act during the lame duck session.

The legislation, which passed the House in July 2015 with broad bipartisan support, is based on recommendations from the entire health community and will help ensure access to essential treatments.

“This is a patient-focused bill that will advance the discovery and development of treatments, strengthen the patient voice in the research and regulatory environment, increase funding for the National Institutes of Health and Food and Drug Administration, and greatly improve our innovation ecosystem,” the letter said.

Millions of Americans are awaiting effective treatments and cures for chronic diseases or disabilities, and delaying passage of the legislation only makes the wait longer.

Click here to read the letter and see the list of organizations that signed on.

How should Congress use its lame-duck session? First, it can save lives.

Originally published in the Washington Post:

REPUBLICANS ARE signaling that they will pursue an ambitious conservative agenda when they take the reins of government next year. But before that happens, the current Congress will convene in its lame-duck session, valuable legislative time that Senate Majority Leader Mitch McConnell (R-Ky.) said this week he hopes to put to good use.

The session may be consumed by arguments over federal budgeting. But if there is time for anything else, Mr. McConnell may push the 21st Century Cures Act, a bipartisan effort that has taken years to get close to passing. Congress should nudge it across the finish line — taking care to repair a few problems along the way.

The act, a version of which passed in the House last year, proposes a one-time, multibillion-dollar increase in funding for the National Institutes of Health. The money could provide a sharp boost to the Obama administration’s cancer initiative, or to research into precision medicine, which tailors treatments to people’s genomes. Rapid progress in both is possible and could save many lives; new cancer drugs have emerged targeting specific mutations in tumor cells, and they have shown encouraging initial results in treating even some of the most complex cancers. The new funding could also go into competitive grants for scientists with particularly innovative projects that are nevertheless underfunded. Given that so many lifesaving pharmaceuticals have their origins in government-sponsored scientific research, the funding boost would be a good investment.

The new money alone, however, would struggle to attract strong bipartisan backing. So lawmakers linked it to various reforms of the Food and Drug Administration’s approval process, arguing that the agency has been hamstrung in getting new drugs to market. One reform on the table would adjust hiring standards at the FDA, which is perpetually short-staffed. The agency has improved on the time it takes to approve new medications, but a more flexible hiring policy could help further. There are also worthwhile provisions that would give gravely sick patients with few options easier access to experimental medication.

Critics have raised some valid concerns. For example, lawmakers should ensure that a proposed adjustment to rules on the approval and use of new antibiotics does not have the unintended side effect of encouraging antibiotic overuse and resistance — the very problem the provision is supposed to combat.

The bill’s backers insist that the FDA’s bottom-line legal mandate would continue to ensure that drugs were safe and effective, even as the agency was granted more flexibility in meeting that standard. This means, then, that it would be up to the FDA to use its new powers wisely.

Despite the caveats, though, the act is worth supporting. If the lame-duck Senate can pass its version and merge it with the House’s, addressing some of the concerns in the process, it would be a valuable use of Congress’s time.