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.

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.

National Health Council Urges Patient Organizations to Show Support for 21st Century Cures

The National Health Council is circulating a sign-on letter to Congressional leadership to urge passage of the 21st Century Cures Act by the end of 2016. It is imperative that the tireless work by Congress and the patient community does not go to waste by delaying action until the next Congress in 2017.

For to view the letter and sign, please click here. The goal is to present the letter to House and Senate leaders before Congress comes back on Monday, November 14th, so signatures are requested no later than Thursday, November 10th.

Only the name of organizations will be listed on the letter, not the individual signers.  Please note that you must be authorized on behalf of your organization to commit to signing publicly.

If you have any questions about the letter, please contact Eric Gascho, Vice President of Government Affairs at the National Health Council, at egascho@nhcouncil.org or 202-973-0545.

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.

Pediatric Priority Review Vouchers Saved in the Eleventh Hour

From FDA Law Blog:

One day before the program’s sunset, President Obama signed a bill to temporarily reauthorize the rare pediatric disease priority review voucher program for 3 months.  The program has had significant interest from industry, and the program’s extension on September 30 was the product of substantial efforts on the part of stakeholders, including rare disease advocates. 

This is the second short-term reauthorization of the program, which was originally slated to end in March 2016. Unlike the first reauthorization (which we discussed here), the Advancing Hope Act (S. 1878) was a more ambitious undertaking.  As such, it amended the definition of “pediatric rare disease,” likely expanding eligibility for the program.  We discussed the law’s impact on the program’s scope in a post last week.

The law makes several other key changes. First, the Advancing Hope Act now requires Sponsors to notify FDA of the Sponsor’s intent to request a pediatric voucher upon the submission of a rare pediatric disease product application.  This requirement comes into effect 90 days after the enactment of the Advancing Hope Act, which incidentally is just a few days prior to the program’s December 31, 2016 sunset.  Second, Sponsors are now expressly prohibited from receiving more than one priority review voucher per drug.

Reading the law makes it abundantly clear that the September 30, 2016 reauthorization is a stop-gap measure. Congress’s effort to quickly pass the bill and avoid creating a gap in the program, meant making a last-minute amendment to the program’s end date of at least September 2022 to December 31, 2016.  As a result of the last-minute amendment, some parts of the bill are fairly nonsensical.  For example, by 2022, GAO will be required to issue another report on the program, similar to the one it issued in March 2016.  It would be odd if the pediatric voucher program was not reauthorized past 2016 and yet the GAO was required to report on the program in 2022 (more than 5 years after the end of the program).

Members of Congress have expressed a strong desire to address FDA-related legislation (including the 21st Century Cures Act, which we discussed here) during the lame duck session of Congress after the November elections.  Part of this attention will go towards discussing the pediatric voucher program and its long term or permanent reauthorization.

Editorial: “Why I’m Stepping Out of My Comfort Zone and Into My Congressman’s Office for Rare Disease

TheMighty published an editorial by Sharon Rose on the need for the rare disease community to advocate in support of the 21st Century Cures Act:

Rare disease has changed my life in many ways, but the most unexpected recent change has been my interest in laws and political issues regarding health care and treatment options. You see, I am a right brainer. I enjoy and flourish in the arts. I’ve never jumped for joy while taking a math or science class. I can recall looking up medical terminology after diagnoses and subsequent MRI or test reports years ago, and how strange it was for me to learn about medicine. I was in a new arena and out of my comfort zone.

However, I realized that in order to advocate for my care, it was a necessary step, and I was finding it interesting! I was being forced to expand in ways I had never imagined. Being creative had always been my focus whether in study, or for fun. But more recently, as a patient and advocate, I’ve experienced another surprising shift in interests. Why, you ask?  Because for the past 6+ years, I’ve been relentlessly and urgently seeking treatments as a patient for Klippel-Feil syndrome, cervical dystonia, Ehlers-Danlos syndrome, and vasculitis – all rare diseases. Time and time again, I have found little understanding or relief. I’ve traveled to five states seeking care. Yes, there are a few great doctors out there. Great doctors who have limited time, limited accurate information, and limited treatment and medication options to offer me as a patient dealing with such an odd laundry list of rare conditions.

To read more from this article, click here.

Little News is Good News

An editorial published by the Alliance for a Stronger FDA staff:

What do the opioid legislation, the Zika legislation and Cures/Innovations bills have in common, other than that they are “hot button” issues? In each case, Congress has done its best to seriously consider healthcare problems facing our nation and managed to find compromises in order to reach consensus. Also — significantly — the three pieces of legislation are stalled by funding issues.

Some Members of Congress (mostly Democrats) think that action on these three topics is so pressing that the legislation should contain enough mandatory funding so that they can be implemented as soon as possible. Other Members of Congress (mostly Republicans) agree on the importance of the three issues, but feel strongly that funding should be left to the appropriations committees. For the moment, the three bills are deadlocked, but it is money, not substance, that is holding them back.

One or more of the three bills may move next week or in September or (possibly) during a lame duck session. But then again, there is no guarantee that they aren’t deadlocked for the year because of the funding issues.

Meantime, the appropriations process seems to be facing a similar fate. There is some compromise and consensus baked into the current set of bills, but also lots of “poison pill” amendments and games-playing. Analysts are still predicting either a continuing resolution or an omnibus bill.

Heading into a presidential election and a difficult transition (regardless of who wins), it is already clear that the most important issue next year, as this year, will be money to fund programs. Under the existing budget agreements, there is not enough domestic discretionary spending to meet high priority needs, no less to handle emergency situations (like Zika and opioids). Under the circumstances, it is hard to say where the money will come from to pay for the new administration’s initiatives. The primacy of funding concerns will occur regardless of which party controls the House and the Senate.

None of this is good news for FDA or any of the public health service agencies. As the foremost advocate for FDA funding, the Alliance will continue to tell Congress about the broad mission and ever-increasing responsibilities on FDA. It will take all of us to make a dent against so much downward funding pressure.

Note: This week’s Analysis and Commentary was written by Steven Grossman, the deputy executive director of the Alliance for a Stronger FDA.

Join Us to Ensure the Rare Disease Community is Heard This Summer

During the summer recess, Members of Congress return to their home states to hear from constituents and attend community events.  Meeting with them during this time is a critical opportunity for rare disease advocates to build relationships, educate them on key legislative needs and follow-up on requests made during Rare Disease Week on Capitol Hill or phone calls or emails about legislative issues since then.

As a FREE service to the rare disease community, Rare Disease Legislative Advocates (RDLA) will set-up a meeting for you and fellow advocates at your convenience. Any rare disease advocate is encouraged to join us for In-District Lobby Days, held from July 18th to September 5th.  Registration is free and available online through Friday, July 15thYou can specify when you are available during the summer recess and how far you are willing to travel for a meeting.

This is a critical juncture for our community.  Although the 21st Century Cures Act passed the House of Representatives with broad bipartisan support last July, the Senate has yet to pass the companion legislation.  In the current partisan environment in Washington, it is important for Members of Congress to hear first-hand how united the American public is on the subject of medical innovation.  A recent poll found that 78% of Americans think Congress should prioritize fostering medical innovations, with an emphasis on finding cures for diseases that still lack treatments.  By joining us for In-District Lobby Days, you can urge your Senators and Representatives to support 21st Century Cures and advocate for other legislation benefiting our community.

To register for In-District Lobby Days, please click HERE.  RDLA will use the information you provided to schedule meetings for you, and help you prepare by providing key background materials and hosting a preparatory webinar to be held on July 13th.

It is up to each of us to ensure the voices of rare disease patients and caregivers are heard in Washington.  Please help us tell Congress loud and clear:  WE NEED CURES NOW!