News

BREAKING NEWS: 21st Century Cures Act – H.R. 6*

* Endorsed by 256 patient organizations! (06-18-15 National Health Counsel letter)
230 co-sponsors in the US House of Representatives as of June 25, 2015
109 Republicans and 121 Democrats

This 309 page bill will significantly increase funding for the National Institutes of Health (NIH), increasing its budget over the next five fiscal years by $14.5 billion … from the current $30.3 billion to $31.8 billion in FY2016; $33.3 billion in FY’17 and $34.8 billion in FY’18. The bill also establishes a new “NIH INNOVATION FUND,” which would be funded with an additional $2,000,000 each year from 2016 through 2020. If your Representative is on the list, please thank them for supporting medical research! IF YOUR REPRESENTATIVE IS NOT ON THE LIST, PLEASE CALL AND/O R WRITE THEM IMMEDIATELY ASKING THEM TO CO-SPONSOR THE BILL!

Click this link to view the complete list of sponsors a state-by-state sponsor listing: HR 6 ~ Cosponsor List 6-25-15

 

Newt Gingrich: Double the N.I.H. Budget

From: The New York Times
By: Newt Gringrich
April 22, 2015

MCLEAN, Va. — NO one who lived through the 1990s would have suspected that one day people would look back on the period as a golden age of bipartisan cooperation. But in some important ways, it was. Amid the policy fights that followed the Republican victories of 1994, President Bill Clinton and the new majorities in Congress reached one particularly good deal: doubling the budget for the National Institutes of Health.

The decision was bipartisan, because health is both a moral and financial issue. Government spends more on health care than any other area. Taxpayers spend more than $1 trillion a year for Medicare and Medicaid alone, and even more when you add in programs like Veterans Affairs, the Children’s Health Insurance Program and the Indian Health Service.

Unfortunately, since the end of the five-year effort that roughly doubled the N.I.H. budget by 2003, funding for the institutes has been flat. The N.I.H. budget (about $30 billion last year) has effectively been reduced by more than 20 percent since then. As 92 percent of the N.I.H. budget goes directly to research, one result is that the institutes awarded 12.5 percent fewer grants last year than in 2003. Grant applications, over the same period, increased by almost 50 percent.

Even as we’ve let financing for basic scientific and medical research stagnate, government spending on health care has grown significantly. That should trouble every fiscal conservative. As a conservative myself, I’m often skeptical of government “investments.” But when it comes to breakthroughs that could cure — not just treat — the most expensive diseases, government is unique. It alone can bring the necessary resources to bear. (The federal government funds roughly a third of all medical research in the United States.) And it is ultimately on the hook for the costs of illness. It’s irresponsible and shortsighted, not prudent, to let financing for basic research dwindle.

For example, the total cost of care for Alzheimer’s and other dementia is expected to exceed $20 trillion over the next four decades — including a 420 percent increase in costs to Medicare and a 330 percent increase in costs to Medicaid. Even without a cure, the premium on breakthrough research is high: Delaying the average onset of the disease by just five years would reduce the number of Americans with Alzheimer’s in 2050 by 42 percent, and cut costs by a third. And that’s not even counting the human toll on both patients and caregivers (often family members), whose own health may deteriorate because of stress and depression.

Yet the N.I.H. is spending just $1.3 billion a year on Alzheimer’s and dementia research — or roughly 0.8 percent of the $154 billion these conditions will cost Medicare and Medicaid this year, more than all federal education spending.

Alzheimer’s isn’t unique: Diabetes, kidney disease, heart disease, cancer, stroke and arthritis all cost enormous sums and cause incredible suffering. But the promise of breakthrough cures and treatments for this disease is amazing. The N.I.H. is funding a clinical study that represents a potential paradigm shift in treatment. Rather than try to eliminate the buildup of plaques in the brain after the onset of dementia, researchers are studying interventions in families with a genetic predisposition to early onset Alzheimer’s to prevent the disease before symptoms even develop.

The N.I.H. is also pioneering the development of immunotherapies, which are already allowing doctors to spur patients’ immune systems to attack cancer and other diseases rather than relying solely on surgery, radiation and chemotherapy. The N.I.H. recently discovered a vaccine that appears to cure an AIDS-like virus in monkeys. The insights from genetics, personalized medicine and regenerative therapies could potentially lead to substantially longer and healthier lives for many. But to achieve that promise will require a greater budget.

Representatives Fred Upton, Republican of Michigan and the chairman of the House Energy and Commerce Committee, and Diana DeGette, Democrat of Colorado, have put forward an initiative, 21st Century Cures, to explore ways to promote the discovery of medical breakthroughs as well as access to new technologies. Ms. DeGette and another committee member, Michael C. Burgess, Republican of Texas and a physician, have called for requiring the Congressional Budget Office to factor in the savings from preventive health measures when “scoring” the financial impact of proposed legislation, at the request of Congress. In the Senate, Lamar Alexander of Tennessee, Ron Johnson of Wisconsin and Jerry Moran of Kansas, all Republicans, have championed increasing funding for basic research.

House and Senate negotiators are at work on a budget resolution for the fiscal year that starts on Oct. 1, and the N.I.H. should be a priority. Doubling the institutes’ budget once again would be a change on the right scale, although that increase should be accompanied by reforms to make the N.I.H. less bureaucratic, to give the director more flexibility to focus resources on the most common and expensive health problems, and to place a stronger emphasis on truly breakthrough research.

We are in a time of unimaginable scientific and technological progress. By funding basic medical research, Congress can transform our fiscal health, and our personal health, too.

______

Newt Gingrich, a Republican, was speaker of the United States House of Representatives from 1995 to 1999.

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A version of this op-ed appears in print on April 22, 2015, on page A23 of the New York edition with the headline: Double the N.I.H. Budget. Order Reprints| Today’s Paper|Subscrib

Testifying for PF on Capitol Hill

On February 25, 2015, Paul Fogelberg testified at a Congressional Briefing on Capitol Hill on Pulmonary Fibrosis, hosted by US Representatives Erik Paulsen (R, MN-3) and Steve Cohen (D, TN-9) and Senator Christopher Murphy (D, CT). The event was excellent with nearly 100 people attending. Please the attached to read the agenda and the remarks. We are making progress!

Congressional Briefing Agenda

Statement Cong. Brief. PF 02-25-2015

Email Congress…Make It Count

How to Email Congress and Make It Count

By Chris Nehls

Reports that email is dead as an advocacy tool have been greatly exaggerated—and nowhere is this notion more true than on Capitol Hill.

Even with the growing sophistication of social media platforms, stodgy old email remains the most important form of communication for constituents and office holders. Though mass emailings have made the tool a blessing and a curse for congressional offices, email still remains the most cost-effective way to communicate. And with the election adding a major cast of new faces— both lawmakers and their staff—those who want to influence Congress in coming weeks will need to know how to use it to best effect.

Click here to read the entire article.

FDA Approves Esbriet for Treatment of IPF

Reproduced from FDA.gov

The U.S. Food and Drug Administration today approved Esbriet (pirfenidone) for the treatment of idiopathic pulmonary fibrosis (IPF).

Idiopathic pulmonary fibrosis is a condition in which the lungs become progressively scarred over time. As a result, patients with IPF experience shortness of breath, cough, and have difficulty participating in everyday physical activities. Current treatments for IPF include oxygen therapy, pulmonary rehabilitation, and lung transplant.

“Esbriet provides a new treatment option for patients with idiopathic pulmonary fibrosis, a serious, chronic lung disease,” said Curtis J. Rosebraugh, M.D., M.P.H., director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research. “We continue to help advance medication therapies by approving products that treat conditions that impact public health.”

The FDA granted Esbriet fast track, priority review, orphan product, and breakthrough designations. Esbriet is being approved ahead of the product’s prescription drug user fee goal date of Nov. 23, 2014, the date the agency was scheduled to complete the review of the drug application.

Esbriet acts on multiple pathways that may be involved in the scarring of lung tissue. Its safety and effectiveness were established in three clinical trials of 1,247 patients with IPF. The decline in forced vital capacity – the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible – was significantly reduced in patients receiving Esbriet compared to patients receiving placebo.

Esbriet is not recommended for patients who have severe liver problems, end-stage kidney disease, or who require dialysis. Esbriet should be taken with food to minimize the potential for nausea and dizziness. Patients should avoid or minimize exposure to sunlight and sunlamps and wear sunscreen and protective clothing, as Esbriet may cause patients to sunburn more easily.

The most common side effects of Esbriet are nausea, rash, abdominal pain, upper respiratory tract infection, diarrhea, fatigue, headache, dyspepsia, dizziness, vomiting, decreased/loss of appetite, gastro-esophageal reflux disease, sinusitis, insomnia, decreased weight, and arthralgia.

The FDA also today approved Ofev (nintedanib) for the treatment of IPF.

Esbriet is manufactured for InterMune, Inc., Brisbane, California.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

IPF Meeting Information

A Message from United States Food and Drug Administration:

Dear idiopathic pulmonary fibrosis meeting attendees,

Thank you for attending the public meeting on idiopathic pulmonary fibrosis patient-focused drug development! FDA collected valuable information on the disease, the impact it has on patients’ lives, and patients’ perspectives on treatment options. We truly appreciate the courage, effort and time invested from everyone who was able to attend the meeting in person or on the web.

We know that not everyone who wanted to come to this meeting was able to attend. For anyone who missed the meeting or is interested in what was discussed, we have posted a full recording of the meeting on our website: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm395774.htm

In addition to the input we gathered at the September 26 meeting, we encourage patients, caregivers, and other stakeholders to submit written comments to the online public docket. The comment period closes on November 26, 2014. Submit your comments through this website: http://www.regulations.gov/#!documentDetail;D=FDA-2014-N-0865-0001

FDA is particularly interested in hearing patients’ perspectives on the questions outlined in the Federal Register Notice that announced this meeting. These questions are pasted below for your reference, and the Federal Register Notice can be found here: http://www.gpo.gov/fdsys/pkg/FR-2014-07-08/pdf/2014-15871.pdf

Again, thank you to everyone who attended the September 26 meeting. If you have any questions, please email PatientFocused@fda.hhs.gov.

Questions for Discussion

Topic 1: Disease signs, symptoms and daily impacts that matter most to patients

1)    Of all the symptoms that you experience because of the condition, which 1-3 symptoms have the most significant impact on your life? (Examples may include shortness of breath, cough, fatigue, etc.)

2)    Are there specific activities that are important to you but that you cannot do at all or as fully as you would like because of your condition? (Examples of activities may include household chores, walking up the stairs, etc.)

a)    How do your symptoms and their negative impacts affect your daily life on the best days? On the worst days?

3)    How has your condition and its symptoms changed over time?

Topic 2: Patient perspectives on current approaches to treating idiopathic pulmonary fibrosis

1)    What are you currently doing to help treat the condition or its symptoms? (Examples may include prescription medicines, over-the-counter products, and other therapies including non-drug therapies such as diet modification.)

a) How well does your current treatment regimen treat the most significant symptoms of your disease?

2)    What are the most significant downsides to your current treatments and how do they affect your daily life? (Examples of downsides may include bothersome side effects, going to the hospital for treatment, etc.)

3)    Because there is no complete cure for your condition, what specific things would you look for in an ideal treatment for your condition?

Public Meeting on Idiopathic Pulmonary Fibrosis Patient-Focused Drug Development

Hosted by United States Food and Drug Administration

Friday, September 26, 2014 from 1:00 PM to 5:00 PM (EDT)

FDA White Oak Campus, 10903 New Hampshire Ave., Building 31, Room 1503 B and C (Great Room), Silver Spring, MD 20993  |  Directions

Call to Action: European IPF Patient Charter

IPF Patient Organisations have identified concrete recommendations and urge European institutions, national governments, funders/payers and healthcare organisations to take the following actions:
[Patients in the USA are also urged to sign the Patient Charter. Celebrate IPF World Week 2014, September 28 – October 5, by joining European Pulmonary Fibrosis patients in signing the Patient Charter!]

Click here to read the entire Call to Action.

In Case You Missed It

From our friends at govdelivery.com

Committee Members Take 21st Century Cures From Coast to Coast

House Committee on Energy and Commerce sent this bulletin at 07/28/2014 05:33 PM EDT

FOR IMMEDIATE RELEASE
July 28, 2014

CONTACT: Press Office
(202) 226-4972

Committee Members Take 21st Century Cures From Coast to Coast

McMorris Rodgers: “This goal is not political or partisan. It is personal.”

The 21st Century Cures initiative has been a collaborative effort from the start. Through a series of four white papers and a number of hearings and roundtables already, the committee has sought ideas from individuals and groups all across the country. Members look forward to continuing the 21st Century Cures initiative in their districts with roundtables and events over the coming weeks. Committee member and House Republican Conference Chair Cathy McMorris Rodgers (R-WA) writes for Forbes that, “as a Congress, we will ensure – with your ideas, big and small – that we can take medical advancement into the 21st century. This goal is not political or partisan. It is personal. Medical innovation affects everyone…”

Committee member Rep. Leonard Lance (R-NJ) also recently announced his office is asking for input from his New Jersey constituents: “To do this right we need first-hand experience and testimony from people who fight this battle every day.” NJ.com reported, “Lance is asking constituents from New Jersey’s Seventh Congressional District to email in their ideas, suggestions or questions on the initiative to NJ07cures@mail.house.gov. These ideas will then be researched and incorporated into a report Lance will present to the Committee.”

Join the effort by emailing cures@mail.house.gov, follow us on Facebook and Twitter, and join the conversation using #Path2Cures.

forbes

July 28, 2014

The Noblest Cause Of Our Time: Saving Lives

By Rep. Cathy McMorris Rodgers (R-WA)

Seven years ago, just hours after giving birth to our son Cole, I learned how a single diagnosis can change your whole life. How two simple words – Down syndrome – are associated with lifelong complications and heart defects and Leukemia and even early Alzheimer’s. But in that moment, when Cole was taken away for surgery and we reeled from the lifetime of uncertainty that suddenly lay before us, I learned firsthand how scientific advancement saves lives.

While breakthroughs in medicine and technology have given hope to Cole and so many millions like him – whether they have Down syndrome or Autism or cancer – we still have a long way to go to remain the world leader in innovation. …It is one of the greatest and noblest causes of our time: to commit ourselves, as a country and a Congress, to saving lives.

That is why, as part of the House Energy and Commerce Committee, we have launched the 21st Century Cures initiative, whose mission is to expedite the discovery, development, and delivery of new and innovative treatments to patients everywhere. We need to leverage technological advances to rethink how we conduct research and break down outdated administrative and procedural hurdles. We are committed to reducing the time and complexity of clinical trials so Americans have the best, most effective treatments right here at home.…

While America has taken the lead for many decades in the field of biomedical research – especially in early discovery – our leadership role is being threatened by other countries, whose research is sustained by both public and private contributions… Our 21st Century Cures initiative explores how we can best achieve that. It examines how other countries incentivize investors, how public-private partnerships improve the discovery process, and how we can streamline the approval process to bring therapies to market more quickly. And as a Congress, we will ensure – with your ideas, big and small – that we can take medical advancement into the 21st century.

This goal is not political or partisan. It is personal. Medical innovation affects everyone: the man whose Alzheimer’s Disease has robbed him of his memory, the child who gives himself insulin shots before school every morning, or the woman who goes to the doctor because she found a lump on her right breast. We owe it to them to chart this course.

With the right policies and regulations, the opportunities for American medical advancement and scientific innovation are boundless. Every day, in laboratories all across the country, new treatments are being discovered and new life-saving drugs are being developed. Let’s make sure they’re produced and approved expeditiously. Let’s make sure innovative treatments for cancer and asthma and heart disease aren’t hindered by exorbitant costs. And let’s make sure that an ineffective regulatory framework doesn’t stand in the way of saving people’s lives. So that when we accompany our aging parents to the doctor, or help those we love endure chronic diseases, or receive diagnoses we never expected, we will still be filled with hope for all the possibilities that lie ahead.

Read the full article online here.

nj

July 28, 2014

Lance announces new initiative to accelerate the pace of medical breakthroughs

Congressman Leonard Lance today, July 25, announced his office is welcoming constituent testimony and comment on a major initiative launched by the House Energy and Commerce Committee. The 21st Century Cures Initiative seeks to accelerate the pace of cures by exploring advancements in technology, the role of federal programming, regulation and research and how best to contribute to medical breakthroughs.

“Remarkable advancements in technology and innovation are transforming health care in the 21st century and presenting an excellent opportunity to advance new cures and treatments. Many young people will see cures in their lifetime for some of today’s greatest public health challenges. To help get there, the 21st Century Cures Initiative is – for the first time – taking a comprehensive look at the full arc of accelerating cures. To do this right we need first-hand experience and testimony from people who fight this battle every day,” said Lance, a member of the Energy and Commerce Committee. “We need the ideas and contributions from patients, health care professionals, innovators and those advocating for a loved one.”

Lance is asking constituents from New Jersey’s Seventh Congressional District to email in their ideas, suggestions or questions on the initiative to NJ07cures@mail.house.gov. These ideas will then be researched and incorporated into a report Lance will present to the Committee.

“New Jersey has always been on the forefront of scientific advancement and medical innovation. If we want to save lives, tackle these major challenges together and keep New Jersey and the United States as an innovation capital then we need all the good ideas we can get,” concluded Lance.

Read the article online here.

Some Good News & An Actionable Item

Friends ~ last Thursday, the Senate Appropriations Committee passed the Senate version  of the FY2015 defense appropriations bill.  Earlier this year, Senator Christopher Coons (Democrat, Delaware) submitted a member request to include pulmonary fibrosis as one of the topics eligible for research funding through the …
Department of Defense (DoD) Peer-Reviewed Medical Research Program (PRMRP – see: http://cdmrp.army.mil/aboutus.shtml
The Senate bill ultimately appropriated $247,500,000 to PRMRP for FY2015 and included PF as one of the eligible research topics.

IF this bill becomes law, or if the Senate and House ultimately pass an omnibus covering all 12 of the appropriations bills that retains the PF language, then we could celebrate a modest new pool of federal research dollars through DoD. This would be in addition to whatever funding appropriated for PF research for FY’15 from the National Heart Lung & Blood Institute – NHLBI. We will know later this year how Congress intends to handle FY15 appropriations (i.e. a Continuing Resolution, omnibus, etc.) and whether the PF language will become law.

Congress will be in recess from August 1st through September 8th.  BUT, with House and Senate members in their home districts, this would be a REALLY GOOD TIME to make an appointment to see your US Senators + US Representative (or, their home district Healthcare Legislative Assistants) and to specifically point out this hopeful opportunity for PF research funding.
Ø  PLEASE ask them to pledge to support the Senate Appropriations Committee version of the DoD appropriations bill (specifically the report language including PF in the PRMRP program on
Page 252 as reprinted below).

Finally, one instant Action Item for your To Do List…Please take a few minutes and send a THANK YOU NOTE to Senator Coons. We need to keep our supporters on Capitol Hill enthusiastic about working on the wide range of strategies to solve the lethal problem of PF. Senator Coons (and his staff) has been especially helpful to the PF cause since he was elected in 2010. Please help by thanking him personally for his efforts on behalf of the PF community.

Address your personal notes to:
The Honorable Christopher Coons
United States Senate
127A Russell Senate Office Building
Washington DC  20510

Keep the faith – and keep working the e-mail, US Mail and telephone as an active PF advocate. Advocacy doesn’t cost. It pays.

Paul

See:  http://www.gpo.gov/fdsys/pkg/CRPT-113srpt211/pdf/CRPT-113srpt211.pdf  (Page 252 of 319) – language clip below:

Pg252of319