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.

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.

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.

Top Dem: Cures bill funding cut to $4B

Originally published in The Hill:

A top Democratic negotiator said on September 28th that new funding in a major medical cures bill has been cut significantly as lawmakers look for a path for passage.

Rep. Gene Green, the top Democrat on the House Energy and Commerce health subcommittee, told The Hill that a new version of the 21st Century Cures bill will allocate about $4 billion over five years for research at the National Institutes of Health (NIH), down from the original $8.75 billion.

He also said funding for the Food and Drug Administration (FDA) is down to $300 million, from about $500 million in the original bill.

However, as negotiations have continued, the final number could end up higher.

“We’re working to finalize the Cures package, so any numbers would be preliminary to share,” a committee spokesperson said.

Lawmakers are looking for a bipartisan deal to move this slimmed down version of the bill when Congress returns for a lame-duck session after the elections. The measure could be a way to fund medical research priorities such as Vice President Joe Biden’s cancer “moonshot.”

The original version of the bill, which seeks to accelerate the FDA’s approval process for new drugs and invest in medical research, passed the House on a bipartisan vote last year.

But it has been mired in the Senate amid months of negotiations over a bipartisan way to pay for the new spending.

Sensing that the clock is ticking, House Energy and Commerce Chairman Fred Upton (R-Mich.), who has made the bill his signature issue, is looking to jumpstart the process by passing a new, slimmed down bill through the House in consultation with the Senate. The upper chamber could then take up that new measure.

“We’re only here a week in November, so that will be the week we need to deal with it, so that’s what Chairman Upton said,” Green said.

He said the House would pass the new bill first, after consultations with the Senate to make sure it can pass in that chamber as well.

“Basically we’re going to try to make sure that we do what the Senate said they can do,” Green said.

Top negotiators in both parties in the House and Senate on Wednesday released statements pledging to work to pass the bill after the election.

Green noted that while the new research funding is less than he hoped, he views it as a starting point, and noted that the regular appropriations process could also increase some funding for the NIH.

“To me, it’s like a down payment,” he said. “We’re not going to get everything we started with.”

From EveryLife Foundation: Take Action to Support Lifesaving Newborn Screening Legislation in California

The EveryLife Foundation is working to expand the lifesaving potential of newborn screening in California. Last month, SB 1095 was introduced by pediatrician and California State Senator Dr. Richard Pan. This bill would help eliminate the legislative delay in newborn screening and ultimately ensure that patients are treated as early as possible. Early screening plays a vital role in ensuring that rare diseases are detected and can be better treated. Read more

Sign-On to Ask Senator Warren to Include OPEN in the Upcoming Senate Bill on Innovation

The EveryLife Foundation for Rare Diseases has asked that advocacy organizations sign-on to this letter urging Senator Elizabeth Warren (D-MA) to support the OPEN ACT (Orphan Product Extensions Now, Accelerating Cures & Treatments).  A link to the letter can be found below, and the letter’s text can be read after the break.

MA Sign-On Letter to Senator Warren: Support the OPEN ACT!

Read more

I Am (Still) Essential

The “I Am (Still) Essential” campaign, representing hundreds of patient groups and their allies, was successful in advocating for the U.S. Department of Health and Human Services (HHS) to make critical changes in Essential Health Benefits for 2016 and beyond.

Now, in order to realize these important patient protections and improve access to care, HHS must enforce these new requirements. These include new transparency provisions and exceptions processes along with adequate drug coverage and standards with which to guard against discriminatory practices such as high patient cost-sharing and excessive use of prior authorization and other medical management techniques. Federal review of the 2016 plans begins May 18th.

In order to urge HHS Secretary Sylvia Mathews Burwell and her staff to conduct a thorough review of the plans and reject those that do not comply with these new patient protections, a coalition of patient groups is circulating d a SIGN-ON LETTER . If you would like your patient organization to sign on to the letter, please fill out this form or go to http://bit.ly/1IH4x1c by COB Thursday, May 14, 2015.

Rare Disease Champion Noah Coughlan, Running Across America to Raise Rare Disease Awareness, Stops in DC to Hold Press Conference

Noah Coughlan is on his way to becoming the only American to run across the United States three times. His goal is to bring awareness, support, and positive impact to the rare disease community and the 30 Million Americans and 350 Million worldwide affected by a Rare Disease. We are so proud of Noah and encourage everyone to support and publicize his mission.  Read more

Action Needed: Draft Guidance on Disclosing Reasonably Foreseeable Risks in Research Evaluating Standards of Care

Rare Disease Patients and their families will be dramatically impacted by the Draft Guidance on Disclosing Reasonably Foreseeable Risks in Research Evaluating Standards of Care recently released for comment by the Office of Human Research Protections. We are writing to ask that you allow us additional time in which to offer comments in response to this draft guidance you announced on October 24, 2014.

 

To be included in this request, please send your name, organization, and city/state to Kyle McEvilly (kmcevilly@geneticalliance.org) by 5 PM on November 17, 2014. Please also indicate whether you are signing on as an organization or individual. You may also sign on online. If you have any questions please contact (sterry@geneticalliance.org).