Monthly Archives: February, 2014

International Rare Disease Day 2014: Joining Together for Better Care

The last day in February marks the annual International Rare Disease Day, first celebrated in 2008 by European coordinators the European Organization for Rare Diseases (EURORDIS).  For the past five years, the United States has participated in this effort thanks to the hard work of the National Organization for Rare Disorders (NORD) and other committed alliances and patient advocacy groups.

Rare diseases are classified in the U.S. as illnesses affecting fewer than 200,000 individuals – about 0.005% of Americans.  These patients and their families often struggle to be heard and there are major gaps in research on treatments and cures due to the small percentage of the population impacted by these diseases.  However, advocacy groups across the nation, often coordinated by patients and their families, are leading the way to increase the voice of those with rare diseases and to ensure research on these illnesses receives well-deserved funding.  Three examples include:

  • The Progeria Research Foundation has been fighting to increase awareness and support of Progeria since 1999.  Hutchinson-Gilford Progeria Syndrome (HGPS, Progeria) is a disorder that causes accelerated aging that eventually leads to pre-mature death due to heart disease.  Drs. Leslie Gordon and Scott Berns, along with their son Sam who passed away in January from the syndrome, have been vocal advocates on Capitol Hill and were featured in the recent HBO special “Life According to Sam.”  Funding raised by PRF contributed to the 2012 discovery of the first-ever treatment for children with the lethal disease.  The Progeria Research Foundation will be honored by Research!America with the Paul G. Rogers Distinguished Organization Advocacy Award on March 12, 2014. Continue reading →

A Weekly Advocacy Message from Mary Woolley: Finally, tax policy is on the agenda

Dear Research Advocate:

What will determine the speed and scope of medical progress in the years to come? There is more to it than the essential ingredients of money and brainpower.

Sound tax policy is essential if we are to propel medical progress.

Yesterday, Rep. Dave Camp (R-MI-04), chairman of the House Ways and Means Committee, introduced a comprehensive tax reform bill. While the prospects for passage during this election year are — to put a positive spin on it — uncertain, Congressman Camp laid down the gauntlet for much-needed tax and entitlement reform, and he also proposed making the R&D tax credit permanent. Uncertainty surrounding future access to the R&D tax credit has reduced its power to drive private sector R&D investment. While the Camp bill does not contain the ideal package of changes needed to optimize the usefulness of the credit, and in fact contains some potential setbacks, his decision to support making the R&D tax credit permanent sets the stage for finally achieving this long-standing goal.

Scientists, physicians and patients must all work to increase clinical trial participation.

In a recent Washington Post op-ed, a personal hero of mine, former Surgeon General and CDC Director David Satcher, MD, discusses the importance of African-Americans contributing to medical progress by participating in clinical research. Using Alzheimer’s disease as a lens, he argues that adequate research funding is not the only imperative; individuals must be willing to volunteer for clinical trials. Participation is especially valuable for racial and ethnic groups who have much to gain as health disparities persist, but who understandably remember mistreatment in trials in the past. Polling commissioned by Research!America has affirmed this lack of trust but also, importantly, has revealed that African-Americans in particular say they want to help others by participating in trials. We also learned from our polls that most Americans, across all demographics, look to their physicians to be the touchpoint for learning about clinical trial participation.

Improved scientist engagement with the public and policy makers is essential.

Medical research stands a better chance of becoming a higher national priority if people can connect meaningfully to scientists. As Alan Alda said at the annual AAAS meeting last week, and in an interview with Claudia Dreifus in The New York Times, “How are scientists going to get money from policy makers if our leaders and legislators can’t understand what they do?” He and his colleagues at the Alan Alda Center for Communicating Science at Stony Brook use of the some of the same approaches we do to help the science community connect with non-scientists in ways that can truly move mountains. Alda adds a passion for science with dramatic talent for a skill set we can all learn from.

Media attention — old school and new school — is key.

Both traditional and social media play a role in the fate of U.S. medical progress because of their ability to call public and policy maker attention to possibilities and stumbling blocks. Research!America and the Pancreatic Cancer Action Network hosted a media luncheon today to discuss the challenges involved in turning cancer, in all its insidious forms, into a manageable chronic condition. It was reinforced to us that journalists’ questions are good markers of questions the public in general are raising; it’s important for scientists and advocates to listen and respond. Sometimes we fall into the pattern of just repeating our own messages louder and louder, but we should instead step back and listen to the sometimes-challenging questions being raised by media as they seek to inform the public. All of us who care about the future of research for health should seek out opportunities to engage with journalists. Contact us for suggestions on how to get started!

Sincerely,

Mary Woolley

Research!America and the Pancreatic Cancer Action Network Host Panel Discussion – “A World Free from Cancers: Probable, Possible, or Preposterous?”

  Leading Experts Focus on the Challenges and Opportunities Affecting the Fight Against Cancer

ALEXANDRIA, Va. – February 27, 2014 A panel of leading health, economics and policy experts today discussed the prospects for a future where cancers are rendered manageable or even eradicated and the variables affecting progress toward that goal so that cancer patients are able to lead normal, productive lives – and thus be “free from” their cancers. The forum was hosted by Research!America and the Pancreatic Cancer Action Network. The event, titled, “A World Free from Cancers: Probable, Possible, or Preposterous?” was held at the New York Academy of Sciences.

Medical innovation has contributed to the economic success of the U.S. over the last 50 years and it offers enormous potential to make a meaningful difference in the quality and length of our lives in the next 50 years. Of all the critical trends that will create a prosperous future, the panelists believe that medical innovation will be the most important. In order to achieve a culture of change where science and medicine will be part of the solution, all stakeholders must stand up and advocate for pro-patient and pro-innovation policies and laws. By supporting a positive regulatory and legislative environment and working toward innovative solutions for complex health care challenges, policy makers can help combat devastating diseases like cancers.

“While medical innovation has driven extraordinary progress against cancer in the U.S. and peer nations, we know that globally, cancer cases and death rates are rising. And even in the U.S., the incidence of some cancers, including pancreatic cancer, is rising,” said Mary Woolley, president and CEO, Research!America. “We need to work together to address these alarming trends, and commit to overcoming the barriers to achieving a world free from cancers. Ensuring that U.S. policy makers sustain a policy environment conducive to rapid-pace medical innovation is crucial.”

The panel addressed the role of medical innovation, not only in the fight against cancer, but as a major force in our nation’s economic progress. Among the technological advances of the 21st century, medical innovation has been the biggest factor in improving the lives of patients, benefiting the health care system and improving prosperity. Over the past 50 years, medical innovation has been the source of more than half of all economic growth in the United States.

The panel, moderated by Fox News Channel’s Jim Pinkerton, featured several leading figures in the cancer and health care community, including:

  • Clifton Leaf, journalist and author, “The Truth in Small Doses: Why We’re Losing the War on Cancer-and How to Win It”
  • Julie Fleshman, president and CEO, Pancreatic Cancer Action Network
  • Laurie MacCaskill, seven-year pancreatic cancer survivor and chair, national Board of Directors, Pancreatic Cancer Action Network
  • Amy Abernethy, MD, PhD, director, Duke Center for Learning Health Care
  • Robert J. Hariri, MD, PhD, chairman, founder and chief scientific officer, Celgene Cellular Therapeutics
  • Scott Gottlieb, MD, resident fellow, American Enterprise Institute
  • Frank Lichtenberg, PhD, Courtney C. Brown Professor of Business, Columbia University

“Although medical innovation has played a key role in the fight against cancer and improving the overall cancer survival rate, much work lies ahead especially for deadly cancers such as pancreatic cancer where the five-year survival rate is just 6%,” said Julie Fleshman, president and CEO, Pancreatic Cancer Action Network. “In order to move towards a world free from cancers, the cancer infrastructure has to continue to keep up with the advances in science and our nation needs to make medical research a priority.”

The panel discussed the benefits of past breakthroughs for some types of cancer: there have been an estimated 50 million life-years saved and $4.9 trillion added in economic value due to innovative cancer treatments since 1990. However, further success in reducing the devastating impacts of cancers and accelerating medical innovation is dependent on developing effective collaborative solutions from an “ecosystem of innovation” – bringing together scientists, patients, health care providers, private-sector medical innovators, academia, payers and policy makers – to find solutions that will save lives from all types of cancers.

“We have made great progress since 1971, when President Nixon declared the war on cancer, in terms of understanding the epidemiology of the disease, improving diagnoses, discovering new treatment paradigms and novel therapeutic approaches to better manage cancers,” said Robert Hariri, MD, PhD, chairman, founder and chief scientific officer, Celgene Cellular Therapeutics. “But the progress we’ve made is not enough. We need to continue the momentum we have started and work together to change the course of human health for patients, health care, our economy and future generations.”

About Research!America

Research!America is the nation’s largest nonprofit public education and advocacy alliance working to make research to improve health a higher national priority. Founded in 1989, Research!America is supported by member organizations representing 125 million Americans. Visit www.researchamerica.org.

About the Pancreatic Cancer Action Network

The Pancreatic Cancer Action Network is the national organization creating hope in a comprehensive way through research, patient support, community outreach and advocacy for a cure. The organization is leading the way to change the survival for people diagnosed with this devastating disease through a bold initiative — The Vision of Progress: Double Survival for Pancreatic Cancer Survival by 2020. Together, we can know, fight and end pancreatic cancer by intensifying our efforts to heighten awareness, raise funds for comprehensive private research, and advocate for dedicated federal research to advance early diagnostics, better treatments and increase chances of survival. To learn more, visit www.pancan.org.

More African Americans need to participate in clinical trials

Excerpt of an op-ed by David Satcher, MD, PhD, honorary chairman of the African American Network Against Alzheimer’s, former U.S. surgeon general and Research!America’s 2007 Raymond and Beverly Sackler Award for Sustained National Leadership award winner, published in The Washington Post.

DavidSatcher1Every February our society measures its progress in the march toward equality as part of Black History Month. But seldom do we discuss inequality in health, an injustice that continues to plague African Americans.

A whole host of health disparities remains unaddressed, including Alz­heimer’s — a disease that African Americans are two to three times more likely to develop than non-Hispanic whites. This disparity is rooted as much in our cultural heritage as in our genes.

For years, studies have found that African Americans have a profound mistrust of doctors and scientists. Consequently, we participate in clinical trials at far lower rates than other ethnic groups, which helps to perpetuate the sort of disparities seen with diseases such as Alzheimer’s. This fear of clinical trials dates to a dark chapter in our shared history: the Tuskegee syphilis experiments.

The Tuskegee study was an infamous clinical experiment in which researchers and the U.S. Public Health Service led African American men with syphilis to believe that they were receiving free medical care while, unbeknown to them, they were being left untreated so scientists could study the effects of prolonged syphilis. After the Associated Press exposed the truth, sparking a public outcry, the U.S. government ended the study in 1972, 40 years after it began.

The 1974 National Research Act set new guidelines for the use of humans in clinical studies. In 1997, the Clinton administration worked with higher education institutions to usher in new training requirements and ethical standards for physicians, researchers and medical students as part of an official apology President Bill Clinton issued on behalf of the nation to the victims of the experiments. While these standards go a long way toward helping to prevent future such experiments, much damage was already done among African Americans.

Read the full op-ed here.

A Weekly Advocacy Message from Mary Woolley: Inspiration from Sochi

Dear Research Advocate:

People everywhere are captivated by the world-class athletes competing at the Winter Olympics. The personal commitment, dedication and motivation on display is certainly an essential ingredient for medalling, but it is not sufficient: Each nation fielding a team must commit to supporting sustained excellence. And both the public and private sectors play a role. There are some interesting parallels to science and innovation — we don’t see it in the public eye every day but when it comes to the fore, it’s the kind of success that affirms the human spirit in a compelling way. When lives are saved with a new therapy or new vaccine, we all take heart and we celebrate, perhaps not realizing that it took years of training, teamwork and ‘practice’ to arrive first at the finish line. What it takes to remain internationally competitive in any global arena — very much including science and innovation — is the combination of well-trained and dedicated people at the top of their form, plus a firm national commitment over a many-year period.

In journalistic coverage that we don’t see often enough, a special report in Monday’s Washington Post describes how government-funded basic research has led to new cancer therapies and a potential “cancer vaccine” currently undergoing testing in the private sector. This is a perfect example of the well-honed teamwork that is our public-private sector research enterprise. But without public sector financing, private sector capital and a commitment to STEM education, the pipeline will not only dry up, its infrastructure will crumble. As Congress readies itself to receive and respond to the president’s budget in early March, email your representatives in Washington to let them know that when it comes to medical research and innovation, the U.S. must continue to go for the gold. That means recommitting to global leadership.

With long-standing champions of science retiring, spurring that commitment will undoubtedly be a steeper climb. Congressman Rush Holt, a physicist whose legacy in Congress as a champion for science, research and STEM education is truly superlative, announced his retirement on Tuesday. His is the latest retirement in a string that reminds us how pivotally important one Member of Congress can be in advancing the best interests of our nation, and it underscores the importance of cultivation of new champions.

Tomorrow morning several NIH directors (NINDS, NICHD, NHLBI and NIAMS) will appear on C-SPAN’s Washington Journal. The call-in program airs from 7:30 – 9:30 a.m. Eastern. I hope you take advantage of participating in this nationally broadcast program. Ask the directors what they think it will take to assure gold-medal winning research now and in the years ahead! Here are the Washington Journal’s phone numbers for calling in tomorrow:

  • Democrats: 202-585-3880
  • Republicans: 202-585-3881
  • Independents: 202-585-3882
  • Outside U.S.: 202-585-3883

I hope to hear your voice on the air!

Sincerely,

Mary Woolley

Now Is the Best Time to Take Care of the Hearts That Matter Most to You

Excerpt of an op-ed by American Heart Association CEO Nancy Brown published in the Huffington Post.

Nancy-Brown1Every day, all across the country, the work continues in the fight against heart disease, in ways big and small.

The medical community is on the front lines, of course, treating sick patients, helping others recover and — equally importantly — educating and encouraging others how to avoid the dangerous tentacles of the No. 1 killer of Americans.

Researchers are hard at work seeking answers. They are conceiving and refining tests that can help with diagnosis, and the medicines and equipment that can help with treatment and prevention.

Americans from other fields contribute, too, sometimes in the literal sense. Donations help fund research, and volunteers give their time to help with awareness and education campaigns.

There’s still one more big piece of this puzzle. Our nation’s lawmakers.

From city councils to statehouses, Congress to the Oval Office, our elected leaders set and maintain public health policies that govern the way we live. Their efforts in beating heart disease are evidenced in the strides made fighting tobacco use the last 50 years.

This month, the federally-designated American Heart Month, their support has been quite evident again.

Read the full op-ed here.

Join NORD in Supporting Rare Disease Day!

Guest blog post by the National Organization for Rare Disorders.

NORD_Logo_2012February 28 is Rare Disease Day – a time to show support for the 30 million Americans, and millions more around the world, living with rare diseases.

Only a few hundred of the nearly 7,000 rare diseases have approved therapies. Many are not being studied at all by medical researchers.

Often, patients – or parents of patients – feel that nothing will be done unless they raise the money, recruit the researchers and fuel the search for their own lifesaving treatment. They find themselves in a race against time, since rare diseases tend to be serious and many are life-threatening.

To promote awareness of these and other rare disease challenges, the National Organization for Rare Disorders (NORD) hosts a national website where all patient advocates can find ways to show their support for Rare Disease Day.  Continue reading →

A Weekly Advocacy Message from Mary Woolley: Snow — not the debt limit — shuts down the government

Dear Research Advocate,

Ironically, the government is closed down today. But that’s due to a major snowstorm, not because of failure to agree on increasing the debt limit! Agreeing to increase the debt limit is an encouraging sign that this Congress, weighed down as it is by ideological and political differences, and with record- low approval rankings from the public, can get its job done! Our job is to be sure research is a top priority in this election year — spoken of with conviction by all candidates and by the media and others who influence them.

Standing tall among Members of Congress who champion science are the Chair and Ranking Member of the House Appropriations’ Commerce, Justice, Science and Related Agencies subcommittee, Rep. Frank R. Wolf (R-VA-10) and Rep. Chaka Fattah (D-PA-02). At our upcoming March 12 Advocacy Awards dinner, Research!America will honor Reps. Wolf and Fattah with the Edwin C. Whitehead Award for Medical Research Advocacy, saluting their tireless efforts to champion policies that promote federal and private sector medical research and innovation. Be sure to join us!

Robert Samuelson observes in The Washington Post that Congress, whether by action or inaction, is making too many decisions “on the sly,” without real public awareness or comprehension. Samuelson says that in so doing Congress is compromising priorities like defense and medical research while simultaneously failing to address tax and entitlement reform. I think it is telling that he chose to identify the loss of purchasing power by the NIH as one of three critical problems created as our elected representatives fail to find a clear path through the ideological storm. One of these days they will make those major decisions, and that’s when it will pay off that research has been well-positioned as a top national priority. We must continue to make the case and make it forcefully.

Even as we work to keep our issue in the forefront of big-picture policy change, we must at the same time make our case via the appropriations process, which is proceeding, for the first time in years, according to ‘regular order.’ Right now, in FY14, funding for NIH is lower than in FY12 (and in constant dollars is lower than FY03!) — a shortfall that makes absolutely no sense if the goal is to serve the best interests of America and Americans. Other science agencies are underfunded as well, and the policy environment for private sector research and innovation is not compatible with our nation’s goals of global leadership. As you prepare to pound the pavement and take to social media to make the case to appropriators for research, take inspiration and new data from the following:

And this: According to the National Retail Federation, Americans are expected to spend $17.3 billion in celebration of Valentine’s Day. That amount would fund the National Heart, Lung and Blood Institute for more than five years! We are a wealthy nation; we can well afford to spend more on the future of health than we currently are.

Sincerely,

Mary Woolley

Research!America Honors Congressmen Frank Wolf and Chaka Fattah for Advancing Medical Innovation to Save Lives and Strengthen the Economy

Reps. Wolf and Fattah to Receive the Edwin C. Whitehead Award for Medical Research Advocacy at Research!America’s Advocacy Awards Dinner on March 12

ALEXANDRIA, Va.February 12, 2014-Reps. Frank Wolf (R-VA) and Chaka Fattah (D-PA) will receive the Edwin C. Whitehead Award for Medical Research Advocacy for their leadership and unwavering commitment to supporting policies that promote federal and private sector medical research and innovation. Reps. Wolf and Fattah have spearheaded efforts to create a legislative and regulatory climate conducive to medical innovation.

“Representatives Wolf and Fattah are exceptional champions for research,” said Research!America Chair John Edward Porter. “They have worked vigorously to increase funding for research, support policies that ignite public and private sector innovation, maintain our global competitiveness, and help patients and their families struggling with costly and debilitating diseases.”

Wolf is currently a senior member of the House Appropriations Committee, presides as chairman of the Commerce, Justice, Science Subcommittee, and is a member of the Transportation, Housing and Urban Development and State and Foreign Operations subcommittees. Throughout his distinguished tenure in Congress, Wolf has worked to advance the state of science and R&D, and he recognizes the role innovation plays in our nation’s economy, health and international competitiveness. Notably, he was a founder of the “Rising Above the Gathering Storm” Commission which sparked a national effort to bolster federal science, technology, engineering and mathematics (STEM) education and R&D programs. These efforts culminated in the enactment of the first America COMPETES Act in 2007 to increase public-private partnerships and provide assistance to innovators throughout the country. Wolf also supported the act’s reauthorization in 2010. He is an active member of several caucuses, including research and development, Parkinson’s, Alzheimer’s and multiple sclerosis. Continue reading →

US Biomedical Research: We Must Reverse a Decade of Neglect

Excerpt of an op-ed by the Albert and Mary Lasker Foundation President Claire Pomeroy, MD, published in the Huffington Post.

PomeroyAs an HIV physician, I began my career early in the AIDS epidemic before effective antiviral medications existed. I held my patients’ hands as they cried when receiving their diagnosis and I went to their funerals. I saw hope in their eyes when new antivirals became available. And when protease inhibitors were licensed and “triple therapy” became the norm, I could help patients plan how they would live, rather than how they would die. Scientific breakthroughs happened only because of our nation’s commitment to biomedical research, but this power of research to make lives better is at great risk.

The decline of U.S. prominence in global biomedical research is upon us: The National Institutes of Health budget has been flat for 10 years and lost 25 percent of its purchasing power, sequestration cut $1.7 billion from the 2013 NIH budget and the 2014 budget is $714 million less than the level approved for 2013, the federal government shutdown prevented enrollment of patients into clinical studies and delayed clinical research protocols, and next generation researchers are taking ideas and talents to other countries. The U.S. sits on the sidelines as nations such as China and India increase research investment by nearly 20 percent while the U.S. drops by 5 percent.

The government’s failure to ensure significant ongoing support for biomedical research undermines the future of science and health in our nation and threatens a strategic driver of the economy. The call to action is clear: The research community must increase advocacy, develop novel research partnerships, and create new opportunities for young researchers.

Read the full op-ed here.

Measuring Economic Growth: R&D Investments

Guest blog post by the  American Chemical Society.

ACSHow has the Super Bowl’s economy-driving market impact grown thanks to scientific research?

Can a value be placed on innovation? What is the economic impact of science and technology research? What is the return on investment of research and development?

These questions were addressed at the January 30, 2014, American Chemical Society Science & the Congress briefing, Measuring Economic Growth: R&D Investments, held on Capitol Hill. Moderated by the National Academies’ Stephen Merrill, PhD, panelist Steve Landefeld, PhD, of the Bureau of Economic Analysis spoke on how R&D numbers are now included in gross domestic product reports. Carol Corrado, PhD, of The Conference Board and Georgetown University explained how this captures “intangible” portions of the economy.

Researchers and scientists discover knowledge. Inventors and engineers apply understanding into tangible products like medicines, cars and computer software. Artists use technology from pens and paints to instruments and computers to produce works of entertainment. R&D thus seeds economic impact.

To illustrate R&D’s economic impact, IBM Chief Economist Martin Fleming, PhD, remarked that the Super Bowl attracts more viewers thanks to computer science: The 1st down line appears as “paint” on the field and not moving players. This TV “magic” results from scientific research of light and information. Camera sensors turn images into data, the Internet exchanges big data packages, then computer graphics paint on the TV screen in real time [with credit to Hollywood for development]. That the Super Bowl’s marketing power translates into significant consumer spending is hard to deny.

Andrew Lo, PhD, of MIT’s Sloan School of Management shows how R&D numbers are candy to financial markets to drive investment decisions that provide for economy-growing business. He showed that billions of dollars put into scientific and medical research in the War on Cancer has led to lifesaving drugs. The lives saved by these drugs contribute trillions of dollars to the economy. When considering multi-billion dollar federal spending, Lo states, “You can’t manage what you can’t measure.”

ACS Science & the Congress Project holds briefings in Washington, DC, to educate and inform Members of Congress, their staffs and policy professionals on issues of science and technology. Previous installments are available at https://vimeo.com/channels/acssciconhill and https://vimeo.com/channels/sciencesocietychallenges. For more information on these events open to the public, contact science_congress@acs.org.

A Weekly Advocacy Message from Mary Woolley: Americans say scientists aren’t communicating the impact of science

Dear Research Advocate:

Since President Obama declared 2014 as a “year of action” in his State of the Union address, several people have asked my view on how the president might advance science by executive order. Some options that come to mind: the president can (1) pump up the budget for NIH and other science agencies in his FY15 budget blueprint, scheduled for release in early March; (2) require an assessment of the impact on innovation, access and economic growth before making any administration-initiated cuts to drug, biologic or device reimbursement; and (3) designate a task force to formulate a national science strategy.

As several Members of Congress noted after the president’s address, American progress can’t be achieved solely by executive order. But rather than debating constitutional authority, it’s past time for the administration and Congress to work together to advance the priorities of the Americans who hired them. Congress is reportedly getting a jump-start on the FY15 appropriations process, so this is perfect timing for advocates to make the case for science funding levels that capitalize on the multi-faceted return on that investment. Continue reading →

World Cancer Day

Today is World Cancer Day. Cancer is the second leading cause of death in the U.S., accounting for nearly one of every four deaths. Today, the American Cancer Society, the American Association for Cancer Research  and many others organizations are joining forces to raise awareness and dispel misconceptions about cancer, while encouraging policy makers to make cancer research a national priority.

What can you do?

  • Call and email your representatives.
  • Make some noise. Join the conversation on social media using hashtags #cancerresearch, #WorldCancerDay, #cancer and #curesnotcuts.
  • Take a look at the list of World Cancer Day events for more ways to get involved.

Did you know? Over the past 40 years, mortality rates for childhood cancer have been reduced significantly, dropping 66% during this time period due to early detection techniques and treatment. Learn more, here.

Federal funding for cancer research is in steady decline. Now is the time to tell your representatives that funding for cancer research is not a luxury but a MUST for improving Americans’ health. We need cures not cuts!