Tag Archives: FDA

Americans Say Congress Should Take Swift Action to Assure Patients Benefit from Treatments and Cures for Diseases

New Poll Data Summary booklet reveals concerns among Americans about the pace of medical progress

AS15Majorities across the political spectrum say it is important that the new 114th Congress takes action on assuring the discovery, development and delivery of treatments and cures for diseases in the first 100 days of the legislative session (75% Democrats, 64% Republicans and 60% Independents), according to America Speaks, Volume 15, a compilation of public opinion polls commissioned by Research!America. As Congress considers numerous proposals in support of research, including the 21st Century Cures draft legislation aimed at speeding the delivery of lifesaving treatments to patients, it is notable to see public support in favor of accelerating medical progress.

“The new Congress has the opportunity to reinvigorate our research ecosystem and enact policies that will enable the private sector to expand innovation,” said Research!America Chair John Edward Porter. “Congress must work in a bipartisan fashion to realize the potential of promising studies to prevent and treat disease.”

An increasing percentage of Americans say the U.S. Food and Drug Administration (FDA) should move more quickly in order to get new treatments to patients, even if it means there may be risks. In 2015, 38% favor faster regulatory review, compared to 30% in 2013. Meanwhile, 25% say the FDA should act more slowly in order to reduce risk, even if it means patients may wait longer for treatments.  Another 19% are undecided on this question and 18% do not agree with either position.   Continue reading →

A Weekly Advocacy Message from Mary Woolley: Tsunami of attention to research in Washington!

Dear Research Advocate:

I warn you that today’s letter is long. There is a lot going on; suddenly, lots of people in Congress are staking out leadership roles as champions for research! On Tuesday, the first draft of the much anticipated 21st Century Cures legislation was released. Chairman Fred Upton (R-MI-06) and Congresswoman Diana DeGette (D-CO-01), who will jointly receive Research!America’s 2015 Edwin C. Whitehead Award for Medical Research Advocacy at our Advocacy Awards dinner on Wednesday, March 11 (click here to join us for the event), have been partnering on this effort since last spring. Congresswoman DeGette has not endorsed the current draft, but she has made it clear that she continues to be committed to the process. We are, too. Over the coming weeks, we will be meeting with the 21st Century Cures team and participating in a variety of discussions on the intricacies of the bill.

Among our priorities will be to ensure that basic discovery is not neglected (a house built on sand…), and to make sure that, for the U.S. Food and Drug Administration (FDA) and other agencies – already struggling to fulfill their current responsibilities – if given new “to do” lists, must also be given new resources. We will continue to push for final bipartisan language that effectively boosts the return on medical progress by accelerating discovery, development and delivery. See our statement on the 21st Century Cures release here. And click here to see the Committee’s documents. They have invited suggestions on the draft, and ask that you send your comments to cures@mail.house.gov. Continue reading →

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 →

Research!America urges the 114th Congress to Advance Top 5 Science Priorities in First 100 Days

Today, Research!America urged the 114th Congress to take action on five science priorities in the first 100 days of the legislative session in order to elevate research and innovation on the nation’s agenda:

  • United States Capitol BuildingAdvance the 21st Century Cures Initiative. Spearheaded by Representatives Fred Upton (R-Mich.) and Diana DeGette (D-Colo.),the initiative is a promising step in the right direction, focusing on speeding medical progress from bench-to-bedside by integrating patient perspectives into the regulatory process, modernizing clinical trials, and reducing red tape, among other things.
  • Repeal the medical device tax. A provision in the Affordable Care Act, efforts to repeal the medical device tax have garnered bipartisan support as policymakers and industry leaders raise concerns about the tax’s impact on jobs and innovation.
  • Enhance and make the R&D tax permanent. The credit, established in 1981, allows companies to deduct certain research expenses, but the short-term extensions have created uncertainty for businesses that rely on long-term planning for research investments.
  • Eliminate Sequestration. As part of the 2011 Budget Control Act, sequestration has taken a significant toll on the research ecosystem, forcing institutions to scale back or eliminate important studies and cut jobs.  A two-year bipartisan budget deal for FY14 and FY15 reduced the cuts for those years, but the full sequester returns in FY16.

Continue reading →

A Weekly Advocacy Message from Mary Woolley: 2015: Pragmatism over politics

Dear Research Advocate:

As America rings in the New Year, many of us will be reflecting on the past and making resolutions for the future. To get a feel for the numerous ways in which NIH, CDC, AHRQ, NSF and FDA contributed to the well-being of Americans and others throughout the world in 2014, click here. I hope lawmakers are taking time now to establish New Year’s resolutions and set priorities for the new Congress, which convenes one week from today. My biggest wish for the new Congress?  Pragmatism over politics. If pragmatism rules, the next Congress will shake off the stultifying complacency that is weighing our nation down and act to reignite U.S. innovation. More here.

One reason pragmatism is so crucial is that it accommodates complexity. It would be terrific if the benefits of medical research and innovation could be catalogued like books in the library, but as Norm Augustine explains in The Journal of Clinical Investigation, no can do. The quantifiable benefits of research can stretch so far into the future and be so wide-ranging that it is nearly impossible to fully capture them. And not all the benefits are quantifiable. Policymakers are understandably interested in hard data to help predict the return on federal investment, but that doesn’t mean the value of science can’t be meaningfully conveyed to them. Norm’s commentary is an important reminder that as advocates, we should be prepared to defend science against inadequate estimates of its impact. It also speaks to our role in bridging the distance between scientific progress and such human values as compassion, empathy and curiosity. Pragmatic means logical and reasonable, not “monetizable.”  Continue reading →

A Weekly Advocacy Message from Mary Woolley: “Congress: Strong letter follows… “

Dear Research Advocate:

So much is troubling our nation – evidenced in protests of recent grand jury decisions and the controversy over release of the Senate’s report on the CIA – that most people probably haven’t noticed or cared that the Congress is delaying and may even abort action on the long overdue funding of the federal fiscal year that began on Oct. 1.  People have grown tired of Congress missing self-imposed deadlines, only to say they can only act in the face of those deadlines, and now they are talking of doing it again.  And thus we are lulled into thinking it doesn’t matter what the Congress does.  But that would be wrong: priority-setting by the Congress plays a major role in determining the economic security and health status of the nation and everyone in it.

Right now, Congress is keeping the nation in limbo, and not just when it comes to funding deadlines. “How low can we go” does seem to be the theme of the appropriations process. If the currently negotiated plan is adopted and signed into law – and that is a big if – the good news is that one-time supplemental funding will be allocated to NIH, CDC and other agencies to work on advancing Ebola-related research and clinical trials. That aside, NIH and CDC would receive razor thin increases compared to FY14, as noted in our statement about the “Cromnibus.” NSF and FDA fare slightly better with increases reaching the level of full percentage points, 2.4 percent and 1.4 percent, respectively. AHRQ is slated to receive a decrease of .08 percent, but, importantly, the agency will at long last be given budget authority, i.e., will not have to rely on passing the hat, so to speak, to other agencies to help fund it. Now Congress must take AHRQ to a higher level of support if we are ever to get our arms around inefficiencies in health care delivery. Continue reading →

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: No recess for advocates!

Dear Research Advocate:

As I write, most members of Congress are on the way home for August recess. As anticipated, no further action has been taken on the appropriations front – or much else, for that matter. In terms of issues we care about: no movement on tax reform, which means no much-needed enhancement of the research and development tax credit; no repeal of the medical device tax; and no final passage of Fiscal Year 2015 appropriations bills.  In upcoming letters I will talk in more detail about Capitol Hill-focused advocacy strategies through the election and beyond.

In the absence of legislative action, some attention – in a bipartisan manner – is being given to research for health. In previous letters, I’ve talked about an effort spearheaded by House Energy and Commerce Chairman Fred Upton (R-MI-06) and Ranking Member Diana Degette (D-CO-01) called the 21st Century Cures Initiative that will remain active over the recess. Public input is being sought as central to this initiative. The truly engaged and whip-smart congressional staff coordinating this initiative have indicated that they would welcome your thoughts at any time. They are particularly interested in the National Institutes of Health, the Food and Drug Administration and public-private partnerships. If you believe the key to faster medical progress is increased funding, tell them. If you feel that bottlenecks in the clinical trials process are the priority concern, tell them. This is not only an opportunity to seed positive change; it is an opportunity to elevate the priority of medical progress going forward. When you think about it, the volume of comments is nearly as important as their content. Issues with an army behind them get attention. To submit comments, e-mail cures@mail.house.gov. 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 Mary Woolley: The role of advocates in the appropriations process

Dear Research Advocate:

There is still time — if you act quickly — to urge your representative to sign on to the House letter authored by Representatives McKinley (R-WV-01), Davis (D-CA-53), Carson (D-IN-07) and King (R-NY-02) urging more support for NIH — it will be finalized by close of business today. A similar Senate letter, authored by Senators Casey (D-PA) and Burr (R-NC), will be finalized Tuesday, April 1; ask your senators to sign on today!

An appropriations mechanism known as a “tap” made the news Tuesday when, during a hearing on NIH, Members of Congress asked advocates questions about the use of a tap by the Department of Health and Human Services (HHS) to move money from the NIH appropriation to fund the Agency for Healthcare Research and Quality (AHRQ) and for other uses. While it can sound as though HHS makes this allocation on its own initiative, actually it is the Appropriations Committee that has determined to fund AHRQ in this way, rather than funding it as an independent agency or otherwise. Bottom line, the funding mechanism isn’t what’s at issue here — the real question is whether AHRQ serves the interests of Americans. And it certainly does. As noted in our testimony submitted for the hearing at which the tap issue was raised, AHRQ supports lifesaving, quality and efficiency-enhancing health care research. Like NIH, AHRQ meets our nation’s need for basic non-commercial knowledge, while the private sector finances the critical, commercial R&D that brings final products to the market.

Continue reading →