Category Archives: Food & Drug Administration

Statement by Research!America President and CEO Mary Woolley on 21st Century Cures Initiative Discussion Draft

The release of the 21st Century Cures Initiative discussion draft is a major bipartisan accomplishment that represents a truly remarkable diversity of innovative ideas to speed the delivery of lifesaving treatments to patients – a testament to the extraordinary commitment of Congressman Fred Upton (R-MI), Congresswoman Diana DeGette (D-CO) and their respective staff members. The Initiative could be a game changer for the medical innovation ecosystem with provisions touching on virtually all phases of the research and development pipeline – from basic and applied research, to FDA review, to coverage and access. Among the many beneficial provisions Research!America fought for is a measure to reduce the administrative burden on researchers.  We look forward to working with the 21st Century Cures team to greatly boost our nation’s commitment to groundbreaking research and drug development.

Statement by Research!America president and CEO Mary Woolley on President Obama’s State of the Union Speech

In his State of the Union address, President Obama highlighted the important role of research and innovation in growing a more prosperous and healthier nation. We’re excited about the launch of the Precision Medicine Initiative, which comes at a time when the challenge of conquering disease – all along the research spectrum, from discovery to translation to innovation and application – has never been more within our grasp. The inspiring story of William Elder, Jr. a medical school student and cystic fibrosis survivor, shows that science can deliver breakthroughs for patients with cystic fibrosis, cancer, Alzheimer’s and other diseases. The remarkable ability of our nation’s researchers to advance precision medicine to hone targeted treatments to improve individual patient outcomes is a compelling example of what can be accomplished with federal support. We’re getting closer and closer to achieving treatments that save time, save money and save lives because they are right the first time.

We can’t afford to ease up on our commitment to research, to assure we can put a whole range of diseases in the history books. A further reason, noted by the President, is that we need robust funding and policies to ensure we’re not behind the eight ball addressing domestic or global outbreaks like Ebola. Current funding levels for federal health agencies put researchers at an extreme disadvantage in pursuing studies that have the potential to cure disease and improve quality of life, and tax policies have stymied the development of new drugs. Policymakers must pivot from short-sighted thinking to formulating a long-term strategy that will bring new treatments across the finish line and spur growth in quality jobs. We think it’s past time to adopt a national strategy that will assure the U.S. retains its world leadership in science and innovation. A new Blue Ribbon Commission established by Congress to explore how science is perceived by the public will help stimulate a meaningful conversation with Americans about the societal and economic benefits of science. Continue reading →

Federal Health Agencies 2014 Year in Review

Photo credit: NIH.gov

Photo credit: NIH.gov

From advances in diabetes research to record approval of drugs to treat rare diseases, taxpayer funded research and the effective employment of regulatory tools played a significant role in improving the health and wellbeing of Americans in 2014. Below is a year-end roundup of research highlights and scientific achievements from the National Institutes of Health, Centers for Disease Control and Prevention, National Science Foundation, Food and Drug Administration and Agency for Healthcare Research and Quality.

To fuel this momentum in scientific discovery and development, policymakers must commit to robust funding for the federal health agencies and policies that support private sector innovation. Take action today and tell your elected officials to make research for health a higher national priority in the 114th Congress.

Statement by Research!America President and CEO Mary Woolley on FY15 Cromnibus Spending Bill

The tiny increases included in the “Cromnibus” bill for the National Institutes of Health (NIH) and our nation’s other health research agencies are just that. The underwhelming support for the NIH, the Centers for Disease Control and Prevention, the National Science Foundation and the Food and Drug Administration following years of stagnant funding and budget cuts begs the question – how low can we go, given health threats the likes of which stand to bankrupt the nation?  And the decision to flat-fund the Agency for Healthcare Research and Quality does not provide what it takes to reduce the much-complained of inefficiencies in our health care system. The pain and economic drain of one disease alone – Alzheimer’s – is not going to be effectively confronted without stronger investments in research. Every American who wants to see our nation overcome health threats, create jobs and shore up our economy for sustained prosperity must make it clear to the next Congress that it can and must do more, making research and innovation a strategic national priority.

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A Weekly Advocacy Message from Mary Woolley: Can we put a dent in the costly toll of suicide?

Dear Research Advocate: 
 
The loss of American Icon Robin Williams has riveted national attention on suicide, one of the 10 most common causes of death in the United States. Today, we are releasing our updated fact sheet on suicide that you can use when meeting with lawmakers and educating others about the impact research can have. Efforts to prevent suicide rightly draw on research findings. But progress has been painfully slow, stymied by serious gaps – partly due to severely limited funding – in the basic research base that precedes private sector development, and stymied by the equivalent of handcuffs placed on social science research.

The notion promulgated by some in the Congress that social sciences research doesn’t add enough value to merit federal funding is not just unfounded, it’s holding us back. Social sciences research saves lives. Case in point: behavioral research guided the development of a suicide intervention that was pilot tested in schools in Georgia and Connecticut and resulted in a 40% reduction in attempted suicides. It has since been implemented in schools across the country. This is just one example of social sciences research at work.

Research moves faster when patient advocates engage. This is the history of the nation’s commitment to defeating polio, to ramping up HIV/AIDS research, to prioritizing breast cancer research and women’s health research overall. Writing in the New Yorker last month, Seth Mnookin described the impact that “dedicated … well-informed families” can have in pushing progress. In his responsive letter to the editor, Peter L. Saltonstall, CEO of the National Organization for Rare Disorders, focused on the use of social media by patient groups to establish global registries, taking full advantage of abilities we didn’t have just a few years ago, and in so doing, saving lives. But there is another message here. The research community must work more closely with patient advocates in order to drive medical innovation. As one of the researchers in the Mnookin article said, “Gone are the days when we could just say, ‘We’re a cloistered community of researchers, and we alone know how to do this.’” Continue reading →

Patient Engagement in Drug Development: Myth or Reality?

HR-researcher_1Is the patient voice adequately represented in the drug development process? The situation has improved but the patient community is not yet recognized as a co-equal in drug development or regulatory review according to Marc Boutin, executive vice president and chief operating officer at the National Health Council, a panelist at a BIO International Convention session on Wednesday. “We scare you,” declares Boutin who says there is an adversarial connection between patients, regulators and industry but the relationship has evolved over the last decade. Patient advocates have developed strategies to amplify their voice and demand access, providing the data and evidence to help transform healthcare delivery.

A section in the 2012 FDA Safety and Innovation Act fosters participation of patient representatives in appropriate FDA meetings with medical product sponsors and investigators, and a new transparent framework to communicate FDA benefit-risk approval decisions to the public. From the patient perspective, “benefit-risk decisions are value judgments, they are not science,” says Boutin. He acknowledges that FDASIA meetings are a critical step but sophisticated patient engagement is still lacking. In-depth interviews, focus groups, crowd sourcing and other strategies should be part of the equation, he adds. Getting the patient perspective in benefit-risk could change the discussion dramatically and improve healthcare delivery, Boutin says. Ensure that clinical trials are designed appropriately for people with chronic conditions, for example, to understand the benefit-risk for a subset of the population. Continue reading →

Global Consortium Protects Drug Supply Chain

RX360logo_noTextMaximizing the return on investment in quality innovation was the topic of a BIO International Convention Super Session on Tuesday which explored challenges and opportunities for pharmaceutical companies in improving efficiencies and ensuring patient safety. Panelist Martin VanTrieste, Senior Vice President, Quality, Amgen described a global initiative formed by industry and regulators to share knowledge and best practices that will help secure the pharmaceutical supply chain and quality of materials. Rx-360, a consortium of pharmaceutical companies, suppliers and regulatory agencies, is tasked with developing voluntary standards for the quality and authenticity of supplies and suppliers, and developing audit standards, training and certification programs. The goal is to prevent counterfeited, adulterated and contaminated materials from entering the supply chain, and jeopardizing patient safety. One tragic example was the introduction of contaminated heparin, a blood thinner, linked to 80 deaths in U.S. patients and allergic reactions in 2008. VanTrieste said the consortium “has been highly effective in bringing industry leaders in the quality and supply chain security space together to come up with common solutions” in addressing the very difficult problems associated with globalization. “By working together we get to the answer faster and it’s more efficient.” Continue reading →

Innovative small businesses get boost from NIH, NSF

Federally-funded research projects that have advanced medical innovation will be on full display at the BIO International Convention Innovation Zone June 23 – 26 in San Diego. Among the new technologies, a device to prevent secondary cataract formation developed with a National Institutes of Health SBIR grant awarded to Sharklet Technologies, Inc. Secondary cataract, a serious complication of cataract surgery, occurs in 25% to 50% of patients. This complication requires a follow-up laser treatment which presents an additional risk to patients and adds more than $300 million in medical costs per year in the U.S. The novel device, a micro-patterned membrane designed to be integrated into a next-generation intraocular lens that has added functionality to prevent secondary cataract formation, could have a significant impact on improving patient care and reducing health care costs.

Improving patient care was also the idea behind a device developed by Actuated Medical. Many patients rely on feeding tubes for medication, nutrition or decompression, however those tubes can sometimes become clogged. A solution was needed to reduce risk and discomfort for patients and lower the expense of tube removal and replacement. SBIR grants from the National Science Foundation (NSF) helped take Actuated Medical’s feeding tube cleaning device from concept to FDA approval. Actuated Medical received a Phase I grant to investigate the technology and prove the feasibility of the device, and then a Phase II grant to develop the device from concept to verification-and-validation testing. Actuated Medical is also exploring various concepts that can be applied to reducing pain and understanding human hormones through the support of SBIR.

Elsewhere, researchers at P2D Bioscience received an NIH SBIR grant to test their lead compound which is an excellent anti-Alzheimer’s disease drug candidate.The research aims to develop an effective drug that can be taken orally to target the underlying neuroinflammation in Alzheimer’s to modify disease progression and improve cognitive function.

The NIH and NSF require robust and sustained funding to support small businesses that are improving the health of Americans. Even if it brings no immediate benefits, a majority of Americans agree that basic scientific research is necessary and should be supported by the federal government, according to public opinion polling commissioned by Research!America.

Sharklet Technologies, Actuated Medical and P2D Bioscience are among the small businesses exhibiting at the BIO International Conference Innovation Zone #BIO2014. For more information about how the Small Business Innovation Research (SBIR) grant program is helping biotech companies across the country, visit: http://www.sbir.gov/

A Weekly Advocacy Message from Research!America: Flat won’t get the job done

Dear Research Advocate:

Today, Chairwoman Barbara Mikulski (D-MD) released the Senate’s 302(b) allocations, which were approved by the Appropriations Committee. As you know from last week, the House 302(b) allocation for the Labor-HHS subcommittee is approximately $1 billion less in fiscal year 2015 than it was in FY 14.The Senate’s allocation for FY 15 is roughly the same as it was in FY 14. The bottom line is that, as expected, we have our work cut out for us to achieve the increases needed for the National Institutes of Health (NIH) and our nation’s other health research agencies. Fortunately, Senator Mikulski and other leaders from both sides of the aisle understand the importance of investing in research to drive U.S. innovation.  That doesn’t reduce advocates’ workload, but it makes success more than a longshot.

Earlier this week, both the House and the Senate Appropriations subcommittees on Agriculture considered bills that would fund the Food and Drug Administration (FDA) in FY 15. The House version calls for a $23 million increase (less than 1%) while the Senate version provides a $36 million increase. While appropriators deserve credit for finding additional dollars for the FDA given overall FY 15 budget constraints, this agency’s responsibility for protecting the very safety of Americans requires more dollars than this.  Continue reading →

A Weekly Advocacy Message from Research!America: The good, the bad, and the ugly

Dear Research Advocate:

Congress continues to pay particular attention to – and make decisions bearing on – the pace of medical progress. To briefly count the ways:

The Senate Labor-HHS Appropriations subcommittee heard testimony yesterday from agency heads within HHS about the significance of health-related spending, including spending on medical and health research. Read our written testimony here.

Congressman Upton (R-MI-06), the Chair of the House Energy and Commerce Committee (which has jurisdiction over authorizing legislation for NIH, CDC, FDA and AHRQ) and Congresswoman Diana DeGette (D-CO-01), a member of the Committee, launched their 21st Century Cures initiative with a roundtable discussion focused on identifying what actions are necessary to maintain our nation’s place as the world’s innovation leader. While Reps. Upton and DeGette are champions of research who should be commended for working to strengthen U.S. medical innovation, there is always the risk that Congress will veer into micromanagement of NIH, stymie FDA’s efforts to ensure that private sector innovators are rewarded for ensuring the safety and efficacy of their medical advances, or “hold off” on providing the funding needed to accelerate medial progress until  longer-term strategies are in place. Your participation can help make this effort a success, and the initiative has established an email address you can use if you wish to give input: cures@mail.house.gov.

So that’s the good. Continue reading →

A Weekly Advocacy Message from Research!America: Are we on the right path to protecting innovation?

Dear Research Advocate:

The budget and appropriations process typically reveals stark differences in funding priorities among the two parties. And this year is no exception. House Budget Committee Ranking Member Chris Van Hollen (D-MD-08) introduced the Democrats’ 10-year budget plan this week, which stands apart from the Republican proposal introduced by Chairman Paul Ryan (R-WI-01) most notably by ending sequestration. The Ryan budget, which won House approval today, is on its way to the Senate but is considered dead on arrival. Note that there’s still time to urge your Members of Congress to support medical and health research as this year’s appropriations process continues!

Teen “whiz kids” profiled in the latest issue of People magazine personify the future of science and medical innovation. Among them, Jack Andraka, who at age 15, created an affordable diagnostic test for pancreatic cancer that provides results in five minutes. He faced tremendous obstacles securing funding for his breakthrough innovation, a problem we see all too often in medical and health research. Such ingenuity propels our best and brightest to take risks but the funding to support their revolutionary ideas is not within their grasp.

Discussing these innovative projects with candidates and elected officials is key to elevating science and technology in the national conversation. In Research!America’s newly released poll data summary booklet, America Speaks, Volume 14, two-thirds of Americans (66%) say it’s important for candidates running for office to assign a high priority to funding medical research. Now is the time to ask future and returning Members of Congress if they believe that medical progress is a high national priority as part of our new national voter education initiative Ask Your Candidates!, which was formally launched this week. More details about America Speaks and the campaign can be found here. As the number of lawmakers with a background in science diminishes, it’s more important than ever to engage with your representatives. Michael S. Lubell writes in Roll Call that if we don’t elect a new scientist in the upcoming elections, it will mark a six-year decline from five to two Members of Congress who have a PhD in a natural science.

National Public Health Week, which wraps up tomorrow, provides another opportunity to engage policy makers about the benefits of health research. Don’t miss our recent blog post celebrating public health — an often underappreciated facet of our research ecosystem.

A new video highlighting backstage interviews with our 2014 Advocacy Award Winners illustrates the passion and drive of these extraordinary leaders who have contributed greatly to medical progress. We encourage you to nominate individuals and organizations whose leadership efforts have been notably effective in advancing our nation’s commitment to research for the 2015 Advocacy Awards.

As you’re aware, members of Research!America’s management team will guest-author this letter in Mary’s absence. This week’s author is Research!America’s vice president of communications, Suzanne Ffolkes.

Sincerely,

Suzanne Ffolkes

Few Americans Know Where Elected Officials and Candidates Stand on Government Support for Research and Innovation, New Polling Booklet Reveals

Research!America and partners launch national voter education initiative to elevate the priority of medical progress

ALEXANDRIA, Va.April 8, 2014—Two-thirds of Americans (66%) say it’s important for candidates running for office to assign a high priority to funding medical research, according to America Speaks, Volume 14, a compilation of key questions from public opinion polls commissioned by Research!America. Polling shows that Americans place a high value on U.S. leadership in medical innovation, yet only 12% say they are very well informed about the positions of their senators and representative when it comes to their support of medical and scientific research. www.researchamerica.org/poll_summary.

To help close this knowledge gap, Research!America and partner organizations are launching a national voter education initiative, Ask Your Candidates! Is Medical Research Progress a Priority? Through online and grassroots activities, social media strategies and on-the-ground events, congressional candidates will be urged to share their views on government policies and support for medical innovation conducted in both the public and private sectors. www.askyourcandidates.org.

“Candidates must do a better job articulating their vision for medical progress, clarifying what level of priority they assign to research as a way to assure improved health, well-being and economic security of all Americans,” said Mary Woolley, president and CEO of Research!America. “Voters need to know whether their candidates view lifesaving medical research as an imperative or an afterthought.”

During election season, Americans want candidates to talk about medical progress. Nearly three-quarters (74%) say it’s important to know whether their candidates for Congress are supportive of medical and scientific research. Notably, more than half of respondents (53%) do not believe elected officials in Washington are paying enough attention to combating the many deadly diseases that afflict Americans. Continue reading →

A Weekly Advocacy Message from Research!America: Talk is cheap; bipartisanship is priceless

Dear Research Advocate:

The doubling of the National Institutes of Health budget between FY99 and FY03 is an example of Congress at its most productive … and it hinged on bipartisanship. A small group of Republicans and Democrats recognized the power of medical progress, and they worked together to increase the budget baseline for NIH by nearly $11.5 billion. Without that doubling, and with the stagnation of virtually all non-defense discretionary funding that followed on its heels, which groundbreaking medical discoveries would still lie dormant? Which of those we hold dear would not be alive today?

Research!America Chair and former Congressman John Porter, who chaired the House Labor-HHS Appropriations Subcommittee, was one of a relatively small group of champions on that bipartisan team. On Monday, March 31, the National Institutes of Health held a dedication ceremony for the John Edward Porter Neuroscience Research Center. NIH Director Dr. Francis Collins, Senators Tom Harkin and Mark Kirk, renowned researchers and NIH alumni Dr. Gerald Fischbach and Dr. Steven Hyman, and other distinguished leaders paid tribute to Congressman Porter, acknowledging his staunch commitment to bipartisanship and his extraordinary contribution to advancing medical research. As Congressman Porter emphasized during his remarks, the two are not unrelated. The severe partisan divide in Congress has served to perpetuate the stagnation of NIH resources, both by compromising the deliberative process that is meant to inform the prioritization of appropriated dollars and by stymying tax and entitlement reform. Scientists must fight back, buoyed by the high esteem in which they are held by the public and armed with unique insights into the societal benefits of investing in research. View photos of the dedication ceremony here and our statement here. Continue reading →

A Weekly Advocacy Message from Mary Woolley: We’re all in this together

Dear Research Advocate:

Fostering research and innovation has long been a multi-pronged effort — government, industry, academia, patients and patient organizations, foundations, and individual philanthropists — all working to advance research. The current interest shown by private philanthropists in advancing science is an echo of a phenomenon witnessed a century ago — and a sign of the opportunity available in some way to all of us to accelerate medical progress and maintain our nation’s competitive edge. As reported in a recent front page New York Times article, private donors are stepping up in a big way at a time when scientific opportunity has never been greater. But it is worth noting that even as philanthropic spending is surging, and while it has historically been an important, often energizing component of U.S. leadership in science, the most robust philanthropic support imaginable would still not be sufficient — nor is it intended to — replace federal support.

In tracking medical R&D spending across all sectors over time, Research!America’s annual investment reports not only support the NYT finding that philanthropic spending is growing, but place that spending in perspective. For example, in 2011, NIH spending dwarfed medical- and health-related philanthropic research spending by nearly $29 billion. That does not mean philanthropic giving isn’t important; rather, it demonstrates that the magnitude of funding needed to drive medical progress is too large to rely on individual or foundation giving. Public and industry dollars are quite simply indispensable to the research pipeline. We call on every sector, every individual (including you billionaires out there!) to step up and increase support. We urge you to fund basic as well as translational research, to identify new approaches and new partnerships, to show us all how to take risks and demand accountability, and to work with and for the overall research enterprise. And — perhaps most important of all — commit to giving confidence to young scientists that their work is valued and will be sustained.

There’s no question about it: We all play a role in achieving better health and quality of life, very much including those who volunteer to participate in clinical trials. We are proud to spread the word about a new campaign initiated by the Pharmaceutical Research and Manufacturers of America (PhRMA) and the National Minority Quality Forum (NMQF). The “I’m In” campaign aims to increase diversity in clinical trials and give patients the opportunity to connect with trials in their communities. Research!America polling shows that while Americans are interested in clinical trials, levels of participation are low, especially among African-American, Asian and Hispanic populations. Advancing medical progress means participating! Our newly released America Speaks, Volume 14 poll data summary booklet includes relevant information on public attitudes about clinical trials.

One time-sensitive way you can exercise your responsibility for advancing medical progress is by asking your representatives in Washington to join the chorus of legislators who support strong, continued funding for research. Members of the House and Senate have the opportunity to share their priorities with the appropriations committees until April 4. Send a note to your representatives urging them to submit appropriations requests that support robust medical research funding in FY15.

Finally, I encourage you to review our just-released 2013 Annual Report, which thanks all our members and supporters — you! — for working with us to inform and engage policy makers, media and the public.

Sincerely,

Mary Woolley

2014 Advocacy Awards Dinner

trofeoOn March 12, Research!America honored extraordinary leaders in medical and health research advocacy during the 2014 Annual Advocacy Awards at the Andrew W. Mellon Auditorium in Washington, DC.

We extend our congratulations to the honorees: Reps. Frank Wolf (R-VA) and Chaka Fattah (D-PA); actress Glenn Close and her family for their work to end the stigmas and misunderstandings surrounding mental illness; Leroy Hood, MD, PhD, president of the Institute for Systems Biology; Kathy Giusti, founder and CEO of the Multiple Myeloma Research Foundation (MMRF); Reed Tuckson, MD, managing director of Tuckson Health Connections; and The Progeria Research Foundation (PRF).

While much has been done to advance research, we have a long way to go.

13130132595_bc0c860475_o“Few out there seem to connect the dots to understand that federal funding is essential to develop the foundation of knowledge which is essential for American enterprise in developing the products and therapies that make our lives longer, healthier, and happier,” said Research!America Chair and former Member of Congress The Hon. John E. Porter in remarks at the Dinner. “There’s nothing more important to our future than investments in science, research, innovation and technology.”

And we agree! Contact your representatives and tell them to make research funding a higher priority.

Distinguished guests included current and former members of Congress and administration officials. Sen. Angus King (I-ME), Rep. David Price (D-NC), Rep. Nita Lowey (D-NY) and Rep. Scott Peters (D-CA), joined the celebration. Research!America board members,  The Hon. Mike Castle, The Hon. Kweisi Mfume and The Hon. Patrick Kennedy also attended the event along with former Congresswoman Mary Bono , former HHS Secretary The Hon. Dr. Louis Sullivan, NIH Director Dr. Francis Collins, NSF Acting Director Dr. Cora Marrett, and PCORI Executive Director Dr. Joe Selby.

Click here to see photos.