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.

’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.

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.

Trump, GOP In Congress Could Use “Must-Pass” Bills To Bring Health Changes

Originally published in Kaiser Health News:

Throughout the campaign, President-Elect Donald Trump’s entire health message consisted of promising to repeal the Affordable Care Act.

That remains difficult with Democrats still commanding enough power in the Senate to block the 60 votes needed for a full repeal. Republicans could use fast-track budget authority to make some major changes to the law, although that could take some time. In the short term, however, Trump could use executive power to make some major changes on his own.

Beyond the health law, Trump also could push for some Republican perennials, such as giving states block grants to handle Medicaid, allowing insurers to sell across state linesand establishing a federal high-risk insurance pool for people who are ill and unable to get private insurance.

But those options, too, would likely meet Democratic resistance, and it’s unclear where health will land on what could be a jam-packed White House agenda.

Still, there are several health issues the next Congress and the new administration will be required to address in 2017, if only because some key laws are set to expire.

And those could provide a vehicle for other sorts of health changes that might not be able to clear political or procedural hurdles on their own.

Here are some of the major health issues that are certain to come up in 2017: 

The Affordable Care Act

If the GOP could not repeal the law and Trump were to turn to Congress to address some of the issues associated with it, it’s not clear if the executive and legislative branches could work together to respond to rising insurance premiums, declining insurance company participation or other unintended impacts of the health law. Nonetheless, some aspects of the law are unavoidable next year. For example, Congress in 2015 temporarily suspended or delayed three controversial taxes that were created to help pay for the law.

One of those taxes, a fee levied on health insurers, is suspended for 2017, while a 2.3 percent tax on medical devices was suspended for 2016 and 2017. Both industries lobbied heavily for the changes — arguing that the taxes boosted the prices of their products — and would like to permanently kill the taxes.

Also on hold is the most controversial health law tax of all, the so-called “Cadillac Tax” that levies a 40 percent penalty on very generous health insurance plans. The idea is to prevent consumers who pay little out of pocket because of their coverage from overusing health care services and driving up overall health costs.

The tax was technically put off from 2018 to 2020, but experts say pressure will begin to mount next year for reconsideration because employers will need a long lead time if they are to change benefits to avoid paying it. While economists are virtually unanimous in their support for the tax on high-end health plans, business and labor both strongly oppose it.

Children’s Health Insurance Program

The Children’s Health Insurance Program, a federal-state partnership that Hillary Clinton helped set up in negotiations with Congress during her husband’s administration, is up again for renewal in 2017. CHIP covers more than 8 million children from low- and moderate-income households and has made a huge dent in the number of uninsured children. According to the Census Bureau, nearly 95 percent of children had insurance coverage in 2015.

When the federal health law passed in 2010, many policymakers thought CHIP would quietly go away because most of the families whose children are eligible for the program became eligible for tax credits to help them purchase plans for the entire family in the health law’s marketplaces. But it turned out that CHIP in most states remained more popular because it provided better benefits at lower costs than did plans through the ACA.

In 2015, Congress compromised between those arguing to extend CHIP and those who wanted to end it, by renewing it for only two years. That ends Oct. 1, 2017. In practice, if Congress wants to extend CHIP, it needs to act early in 2017 because many states have fiscal years that begin in July and need lead time to plan their budgets.

Prescription Drug And Medical Device User Fees

Also expiring in 2017 is the authority for the Food and Drug Administration to collect “user fees” from makers of prescription drugs and medical devices.

The Prescription Drug User Fee Act, known as PDUFA (pronounced pah-doof-uh), was originally passed in 1990 in an effort to speed the review of new drug applications by enabling the agency to use the extra money to hire more personnel. The user fees were later expanded to speed the review of medical devices (2002), generic copies of brand-name drugs (2012) and generic biologic medicines (2012).

PDUFA gets reviewed and renewed every five years, and its “must-pass” status makes it a magnet for other changes to drug policy. For example, in 2012 the renewal also created a program aimed at addressing critical shortages of some prescription drugs. Earlier renewals also included separate programs that gave pharmaceutical firms incentives to study the effect of drugs in children.

Some policy-watchers think this year the bill could serve as a vehicle for provisions to help bring down drug prices, although it is not clear how well many of the ideas currently being floated would work.

“I think [Congress] will talk a lot about it and do very little,” said Robert Reischauer of the Urban Institute, who called the drug price issue “incredibly complex.”

Medicare’s Independent Payment Advisory Board

One more issue that might come up is a controversial cost-saving provision of the federal health law called the Independent Payment Advisory Board, or IPAB. The board is supposed to make recommendations for reducing Medicare spending if the program’s costs rise significantly faster than overall inflation. Congress can override those recommendations, but only with a two-thirds vote in each of the House and Senate.

So far the trigger hasn’t been reached. That’s lucky because the board has turned out to be so unpopular with both Democratic and Republican lawmakers, who say it will lead to rationing, that no one has even been appointed to serve.

The lack of an actual board, however, does not mean that nothing will happen if the requirement for Medicare savings is triggered. In that case, the responsibility for recommending savings will fall to the secretary of Health and Human Services. Medicare’s trustees predicted in their 2016 report that the targets will be exceeded for the first time in 2017.

That would likely touch off a furious round of legislating that could, in turn, lead to other Medicare changes.

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.”

Apply Today for a Travel Stipend for Rare Disease Week on Capitol Hill 2017

Please mark your calendar and plan to join us for Rare Disease Week on Capitol Hill, to be held February 27 through March 2, 2017 in Washington, DC.  The week of events brings together rare disease community members from across the country to be educated on federal legislative issues, meet other advocates, and share their unique stories with legislators. There will be new Members of the House and Senate next year, and it is critical for them to meet members of their communities affected by rare disease.

All events are free for patient advocates, and registration will open in early January.

Applications for Travel Stipends Now Open

The EveryLife Foundation is now accepting applications for travel stipends!  We awarded more than $55k in stipends this year, and hope to enable even more advocates to join us in Washington, DC next year. Patients, caregivers, and others in the rare disease community can apply online. The deadline to apply is December 18th, and all applicants will be notified in early January. Please note that stipends are limited to one per family, and attendance at the Legislative Conference and Lobby Day is required.

Not Able to Attend?

We want every Member of Congress to hear from constituents affected by rare disease, and you can help even if you can’t join us in person. Please share your unique perspective and let your legislators know what issues matter most to you by filling out our online form by February 12th so that we can hand-deliver it on the Lobby Day.  And please share this opportunity with your network to make sure the voice of your rare disease community is heard!

Please sign up for our email list or check the Rare Disease Week on Capitol Hill webpage for more information and updates.

RareVoice Awards Gala Honors Rare Disease Advocates and Congressional Leaders

More than 300 patient advocates and industry leaders as well as staff from Capitol Hill and federal agencies joined us last week for the fourth annual RareVoice Awards Gala at Arena Stage in Washington, DC.

Dr. Steve Groft received a Lifetime Achievement award for his dedication to stimulating research and advancing development of therapies during his tenure at the Food and Drug Administration (FDA) and National Institutes of Health (NIH), where he served as Director of the Office of Rare Disease Research from 1993 until his retirement in 2014.

The judges had difficulty selecting the winners among the passionate patient advocates making a difference in states across the country and on Capitol Hill.  Patricia Weltin, President and Founder of the Rare Disease United Foundation, received an Abbey for patient advocacy at the state level. Lisa and Max Schill with RASopathies Network received an Abbey for patient advocacy at the federal level for their efforts in support of the 21st Century Cures Act.  Ronald Bartek, President and Founder of the Friedreich’s Ataxia Research Alliance, received a Lifetime Achievement Award for his tireless advocacy before Congress as well as the FDA and Social Security Administration.

The Chairman and members of the House Energy and Commerce Committee were recognized for their leadership on the 21st Century Cures Act which has several provisions critical to the rare disease community, including incentives to spur development of new therapies. Representative Fred Upton (R-MI) received a Lifetime Achievement Award. Representative Gus Bilirakis (R-FL), G.K. Butterfield (D-NC) and Diana DeGette (D-CO) were honored with Congressional Leadership Awards. Saul Hernandez, Deputy Chief of Staff and Legislative Director for Representative G.K. Butterfield, and Clay Alspach, Chief Health Counsel of the Energy and Commerce Committee, received Abbeys for their work on behalf of the rare disease community.

Dr. Kakkis, President and Founder of the EveryLife Foundation for Rare Diseases, ended the awards ceremony with a call to action, urging everyone in attendance to contact their Senators to ask that the many provisions of the 21st Century Cures Act supported by the rare disease community be included in Innovation for Healthier Americans, the Senate companion bill.

Photos of the awards ceremony and reception will be posted on the Rare Disease Legislative Advocates Facebook page and the RareVoice Awards website, so be sure to visit both.

Thank you to Shire, the Presidential Sponsor, and other sponsors including AbbVie, Alexion, Amgen, Amicus, Genzyme, Novartis, PhRMA, Raptor, and Vertex. Proceeds from the Gala fund Rare Disease Week on Capitol Hill, to be held February 29 through March 3 next year.

Rare Disease Congressional Caucus Briefing: Urgent Healthcare Policy Needs of the Rare Disease Community

Rare Disease Legislative Advocates & the National MPS Society in coordination with Rare Disease Congressional Caucus Co-Chairs:
Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), held a briefing,

Urgent Healthcare Policy Needs of the Rare Disease Community

Thursday, February 26th, 2015

Russell Caucus Room

Lunch Provided by event sponsors AbbVie, Alexion, Raptor, & Shire
Moderator:  Anthony J. Castaldo, President, US Hereditary Angiodema Association (HAEA)
  • Limited Access to Specialists in Private Insurance Networks, Stephanie Bozarth, National MPS Society
  • Coverage Gaps for Medically Necessary Foods, Nicole Dreyer-Gavin, PKU Parent Advocate
  • Medicare Coverage Challenges for Cystinosis Patients, Kristina Broadbelt, Patient Advocate
  • Step-wise Therapies: Negative Implications for the Health of Rare Disease Patients, Anthony J. Castaldo, President, US Heriditary Angiodema Association

 VIEW THE BRIEFING HERE

Briefing Hosted by:

 

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).