Registration Now Open for Rare Disease Week on Capitol Hill

Join fellow rare disease patients, caregivers and other advocates from across the country at Rare Disease Week on Capitol Hill from February 27th through March 2nd, 2017. This is an opportunity for YOU to help educate the new Congress and shape healthcare policy to better meet the needs of the rare disease community.

All of the events are free for rare disease advocates, but advance registration is required for each.

Monday, February 27th, 8:30am-4pm: Rare Disease Day at the National Institutes of Health (NIH)
You can attend in-person or watch via livecast, and registration is available on the NIH website.

Monday, February 27th, 5:30-9:30pm: Cocktail Reception and Rare Disease Documentary Screening presented by Vertex featuring Up for Air at the Naval Heritage Center
Registration is available HERE.

Tuesday, February 28th, 9am-5pm: Legislative Conference presented by Horizon Pharma at FHI 360. Registration closed on February 12th.

Wednesday, March 1st, 7:30-8:30am: Lobby Day Breakfast at Hyatt Regency on Capitol Hill. Registration is now closed.

Wednesday, March 1st, 9am-5pm: Lobby Day Meetings with Members of Congress on Capitol Hill. Registration closed on February 12th.

Wednesday, March 1st, 5-7pm: Rare Artist Reception at the Kennedy Caucus Room of the Russell Senate Office Building
Registration is available HERE.

Thursday, March 2nd, 12-1:30pm: Rare Disease Congressional Caucus Briefing in Room 902 of the Hart Senate Office Building
Registration is available HERE.

For more information on Rare Disease Week on Capitol Hill including the introductory webinar held last month and a link to register for the next webinar on February 8th, please visit http://rareadvocates.org/rdw/.

Sign up to receive news from Rare Disease Legislative Advocates to ensure that you don’t miss any updates on Rare Disease Week on Capitol Hill! You can also follow us on Twitter and Facebook.

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.

Video from Rare Disease Congressional Caucus Briefing on PDUFA Now Available

The final Rare Disease Congressional Caucus briefing of 2016 focused on the Prescription Drug User Fee Act (PDUFA), the agreement between the biopharmaceutical industry and Food and Drug Administration (FDA) regarding user fees paid by industry to supplement federal funding for human drug review.

Congress will need to reauthorize PDUFA before the current agreement expires at the end of September 2017, which will be an opportunity to include provisions to encourage the development and streamline the review of rare disease treatments.

Watch the videos to learn more from representatives from debra of America, National Health Council, Biotechnology Innovation Organization, Genentech and office of U.S. Representative Leonard Lance (NJ).

http://rareadvocates.org/driving-innovation-for-lifesaving-therapies-through-pdufa-reauthorization-in-2017/

Webinar Provides Overview of Rare Disease Week on Capitol Hill 2017

 

Rare Disease Legislative Advocates recently hosted a webinar which provided an overview of Rare Disease Week on Capitol Hill, to be held February 27th through March 2nd, 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 of the events are free for patient advocates, and registration will open on January 4th.

Not Able to Attend? Submit Your Unique Perspective to be Hand-Delivered to Congress

We want every Member of Congress to hear from constituents affected by rare disease, and you can make your voice heard 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 Lobby Day.

Check the Rare Disease Week on Capitol Hill webpage for more information and updates.

Travel Stipends for Rare Disease Week on Capitol Hill Increased!

The EveryLife Foundation for Rare Diseases will provide travel stipends to enable advocates from across the country to participate in Rare Disease Week on Capitol Hill, to be held February 27th through March 2nd, 2017.

Thanks to a generous donation to our Rare Giving program by Horizon Pharma, we are able to increase the amount offered to recipients! Advocates in Maryland and Virginia are eligible to receive $400 stipends, while advocates in other states in the continental U.S. are eligible to receive $800. Advocates in Alaska, Hawaii and Puerto Rico are eligible to receive $1,000 stipends.

In order to receive a stipend, advocates must attend the Legislative Conference and Lobby Day. Applications are available online through December 18th. All applicants will be notified whether they will receive a stipend or are on the waitlist on January 3rd.

We hosted a webinar earlier this month to provide an overview of the events during Rare Disease Week on Capitol Hill, highlight travel options to Washington, DC and answer questions.  You can find the webinar recording and schedule of events online.

We hope you will plan to join us to educate the new Congress and help shape healthcare policy to better meet the needs of the rare disease community. You can sign up to receive news from Rare Disease Legislative Advocates to ensure that you don’t miss any updates on Rare Disease Week on Capitol Hill. You can also follow us on Twitter and Facebook.

Thank you to

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.

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.