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.

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.

’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.

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.

From FDA Law Blog: Priority Review Voucher Updates- Valuation, Eligibility, Reauthorization

From FDA Law Blog by Alexander J. Varond

Hardly a week goes by without news of FDA’s priority review voucher (“PRV”) programs. The last several weeks are no exception. In this blog post, we focus on five key updates related to FDA’s tropical disease and rare pediatric disease (“Pediatric”) PRV programs:

  1. 1. United Therapeutics Corporation (“United Therapeutics”) sold its Pediatric PRV for $350 million;
  2. 2. DA added Chagas disease and neurocysticercosis to its list of eligible tropical diseases;
  3. 3. Sanofi-Adventis (“Sanofi”) successfully redeemed a Pediatric PRV to beat Amgen to market and became the first PCSK9 cholesterol therapy approved in the U.S.;
  4. 4. The Senate released its own version of the Advancing Hope Act; and
  5. 5. Three Duchenne muscular dystrophy sponsors received rare pediatric disease designation.

PRV Prices Continue to Climb

On August 19, United Therapeutics announced the sale of its rare Pediatric PRV for an incredible $350 million to AbbVie Inc. We discussed FDA’s issuance of this Pediatric Voucher to United Therapeutics for Unituxin here. As the graph below shows, the value of PRVs continues to climb dramatically.

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FDA Adds Chagas Diseases and Neurocysticercosis to the List of Qualifying Tropical Diseases

On August 20, 2015, FDA published a final order to add two new diseases, Chagas disease and neurocysticercosis, to the list of qualifying tropical diseases. Recall that, in order for a drug product to be eligible for a PRV, four requirements must be met:

  1. 1. The application must be for a listed tropical disease;
  2. 2. The application must be submitted either as a 505(b)(1) NDA or a 505(b)(2) application;
  3. 3. The drug that is the subject of the application must not contain a previously-approved active moiety; and
  4. 4. The application must qualify for a 6-month priority review under FDA’s policies.

Thus, a sponsor for a drug that treats Chagas disease or neurocysticercosis that meets the requirements above would be eligible for a tropical disease PRV.

Section 524(a)(3)(R) of FD&C Act permits FDA to include “[a]ny other infectious disease for which there is no significant market in developed nations and that disproportionately affects poor and marginalized populations.” (Emphasis added.) In its final order, the Agency also affirmed its commitment to using “a flexible approach” to tropical disease designations based on “scientifically informed, qualitative assessment of disease candidates.” FDA also announced the establishment of a public docket that will remain open to receive future suggestions for tropical disease designations. That docket is here.

The final order explains FDA’s interpretation of the key elements of section 524(a)(3)(R) of FD&C Act:

“Developed nations” – According to the order, “FDA will use a country’s presence on the World Bank’s list of ‘low income economies’ . . . as evidence that the country should not be considered a ‘developed nation’ for purposes of ‘tropical disease’ determination.”

“No significant market” – FDA notes the difficulty in providing a rigid definition of “no significant market” and instead proposes the following factors be considered in determining whether a “significant market” exists in developed countries: (1) occurrence of the disease in developed nations; and (2) the existence of a sizeable indirect market for the tropical disease drug (e.g., government, including the military) that would constitute a financial incentive for drug development.

Disproportionately affects poor and marginalized populations – As with the term “no significant market,” FDA rejected a single definition in favor of four factors that must be weighed. These factors are: (1) the proportion of global disability-adjusted life years for the disease that is attributable to developing countries; (2) the relative burden of the disease in the most impoverished populations within the countries in which it is found; (3) the relative burden of the disease in infants, children, and other marginalized segments of the population; and (4) whether WHO has designated it as a neglected tropical disease.

Applying these standards, FDA exercised the authority granted to it by the Adding Ebola to the FDA Priority Review Voucher Program Act in December 2014 and added Chagas disease and neurocysticercosis to its list of diseases that qualify for the tropical disease voucher program.

Bonus:  On August 24, 2015, Mid-Atlantic BioTherapeutics, Inc. submitted a citizen petition requesting that FDA add the rabies virus to the list of qualifying tropical diseases.  Notably, rabies is on WHO’s list of neglected tropical diseases, as is cysticercosis/taeniasis.

Sanofi Successfully Uses its PRV

On August 19, 2015, FDA announced that Sanofi-Adventis had redeemed a Pediatric PRV for its Praluent (alirocumab) NDA.  With its approval on July 24, 2015, Praluent became the first approved PCSK9 cholesterol therapy in the United States.  The PCSK9 therapeutic class is expected to a blockbuster, with sales projected to be in the billions of dollars per year.

The interesting part of this story is that Amgen’s Repatha beat Sanofi’s Praluent to market in Europe; but, ostensibly, through its redemption of a rare pediatric disease priority review voucher, Sanofi was able to reach the U.S. market before Amgen.  The Praluent approval came a month ahead of Repatha’s August 27, 2015 PDUFA date and approval.

Drugs that are first-to-market often enjoy considerable benefits over second-comers.  U.S. payers, for example, are quick to cover new drugs, and products that are first-to-market often have significant marketing advantages and sales momentum.  Sanofi’s $67.5 million move to accelerate its entry into the U.S. market for its PCSK9 therapy will be closely watched.  After all, it will be a bellwether of whether industry can justify the high prices paid for priority review vouchers.  If things go well for Sanofi, the demand for and price of subsequent PRVs should be high.

An Update to the Advancing Hope Act

In March 2014, Representative G.K. Butterfield (D-NC) introduced the Advancing Hope Act (H.R. 1537) in the House of Representatives.  In our post on the bill, we noted the effort to make the Pediatric Voucher program permanent.  We also noted the bill’s proposal to change the tropical disease voucher program.  At the time, we noted that such changes not only seemed out of place, but they could be counterproductive.

On July 28, 2015, the Advancing Hope Act (S. 1878) was introduced in the Senate.  The Senate version of the bill scrapped the amendments to the tropical disease priority review voucher program.  Instead, the Senate version proposes to make the Pediatric Voucher program permanent and to designate pediatric cancers and sickle cell anemia as rare pediatric diseases.

Rare Pediatric Disease Designations Granted for DMD

On August 19, FDA awarded rare pediatric disease designation to BioMarin’s,Sarepta’s, and Santhera’s investigational products for Duchenne Muscular Dystrophy.  With the rare pediatric disease designation, each company will likely be eligible for a Pediatric Voucher upon approval of its drug product.

BIO Patient & Health Advocacy Summit: Oct 14-15 2014, in DC

 

BIO is throwing their Patient & Health Advocacy Summit, titled “The Power of Partnerships” this October 14-15 in Washington, DC.

The event voluntary brings health organizations and the biotechnology industry together for two days of policy-driven panel discussions, best practice seminars and invaluable networking opportunities. The summit will feature speeches from Janet Woodcock, who serves as Director of the FDA center for Drug Evaluation, Representatives Diana Degette (D-CO) and Joe Pitts (R-PA), and many more.

To register and find out more, clickbit.ly/biosummit

Rare Diseases Congressional Caucus Briefing Sept.17th

Rare Disease Legislative Advocates & Kids v Cancer in coordination with Rare Disease Congressional Caucus Co-Chairs
Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), will host a briefing

“Implementation of the Rare Disease Provisions in the
Food & Drug Administration Safety and Innovation Act of 2012 (FDASIA) ”
Read more