Tag Archives: Amicus Therapeutics

Highlights of the 2013 National Health Research Forum

Research!America’s National Health Research Forum — held September 12 at the Newseum’s Knight Conference Center in Washington, DC — examined the current and future state of research to improve health. This year’s theme was “Straight Talk about the Future of Medical and Health Research.” Three expert panels delved into different aspects of the research ecosystem.

_DSC5052Reseach Amercia NatHealth Research Forum 9.12.13 BarrettResearch!America’s president and CEO, Mary Woolley, and chair, The Honorable John Edward Porter, opened the program. Porter introduced Bart Peterson, JD, senior vice president of corporate affairs and communications at Lilly who delivered a brief keynote speech.

“We developed an innovation ecosystem, and that ecosystem requires sound public policy. From the private sector perspective, that includes solid intellectual property protection; a fair, rigorous, transparent regulatory system; a market system of health care delivery and pricing that offers choice for patients and health care providers,” Peterson said. “But the public sector has a role far beyond just producing sound public policy … Public funding for research, which is so threatened today, is absolutely critical to the future and we care about that as much from the private sector perspective as anybody else does.”

R!A 2013 Forum

The first panel, focusing on biomedical research and development, was moderated by journalist Eleanor Clift of Newsweek and the Daily Beast and featured John Crowley, president and CEO of Amicus Therapeutics and a patient advocate; William Hait, MD, PhD, global head of R&D at Janssen Pharmaceuticals; Margaret Hamburg, MD, commissioner of the Food and Drug Administration (FDA); and Peterson. The discussion centered on innovation within the pharmaceutical industry and the relationship between companies and regulators. Continue reading →

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A Weekly Advocacy Message from Mary Woolley: Don’t Settle for the “New Normal”

Dear Research Advocate,

Yesterday, the House passed a budget for the remainder of the fiscal year that includes this year’s cuts from sequestration along with an additional one percent across-the-board cut.  The bill now heads to the Senate for consideration, where we are likely to see higher funding levels than the House version, but with sequestration still in place. Congress seems anxious to avoid the brinksmanship and the government shutdown threats that have characterized past debates. While the less rancorous environment surrounding the CR is a welcome change, the complacency around sequestration is not.  As research advocates, we cannot let these cuts stand.

Sequestration isn’t a one-year cut, it is ten years worth of cuts, none of which are evidence-based.  We may be looking at the early stages of an elusive “grand bargain” as the president meets with Republican senators to discuss tax and entitlement reform – two key pieces for solving the deficit puzzle. Eliminating sequestration must be part of that bargain. In addition, we must ensure that funding for biomedical and health research, including the resources FDA needs to do its job, are assigned a high priority in fiscal year 2014. That should be reflected in the budget resolution and obviously in the FY 2014 funding bill.

None of this will be easy. Working together, advocates have raised the profile of medical research with policymakers and the media. We need to turn the volume up louder yet on it, while cultivating more champions in Congress.  Continuing to engage the media is part of that equation.  Some of the largest news outlets in the country including Fox News, NBC, and CBS, and a number that are new to our issue including Al Jazeera quoted Research!America when writing about sequestration’s impact on science. The Economist published a thoughtful piece about how cutting American health research will harm the world. Industry is adding its voice with an op-ed in Forbes coauthored by three legendary executives, including Research!America board member and former NIH director, Dr. Elias Zerhouni, Marc Tessier-Lavigne of The Rockefeller University and P. Roy Vagelos, Chairman of Regeneron Pharmaceuticals. I also want to highlight a letter that Dr. Herb Pardes, Executive Vice Chairman at New York Presbyterian and Research!America board member, sent to the President.  He captures the very themes that will anchor our advocacy going forward.

At the same time as policymakers were cutting federally funded research dollars, researchers were delivering another astonishing breakthrough – the real possibility of a functional cure for HIV. This remarkable achievement, bringing us a step closer to a world free from the scourge of HIV/AIDS, would not have been possible were it not for NIH funding that supported the research and development of anti-retroviral drugs. The CDC is also in the news, with troubling warnings about the spread of “nightmare bacteria” – germs that can be deadly because they are resistant to traditional medicines.  As CDC works to track and halt the spread of these germs and fulfill the numerous other public health functions for which they are responsible, the agency is not only contending with sequestration. Over the past several years, CDC has been subject to some of the deepest cuts of any health agency. Our Nation is fast approaching a tipping point.  Are public health and safety and the progress borne of medical innovation priorities, or not?

As many of you already know our annual events are coming up next week! Please join us for the Annual Meeting (free of charge to members) to hear remarks from Rep. Joe Kennedy (D-MA), a freshman member of Congress who is already championing research, and also John Crowley, CEO of Amicus Therapeutics. I hope to see you all at our Annual Advocacy Awards Dinner later that evening – seats are selling fast, but still available.

Sincerely,

Mary Woolley

Research!America Honors Senators Richard Burr and Bob Casey for Strengthening our Nation’s Commitment to Research and Development

Burr and Casey to Receive the Edwin C. Whitehead Award for Medical Research Advocacy at Research!America’s Advocacy Awards Dinner on March 13

Alexandria, Va.February 6, 2013–Sens. Richard Burr (R-NC) and Bob Casey (D-PA) will receive the Edwin C. Whitehead Award for Medical Research Advocacy for their leadership and strong support of federal and private sector medical research and innovation. Sens. Burr and Casey have worked individually and in a bipartisan manner to promote a robust medical research pipeline in the U.S. and ensure patients receive access to new, safe and effective treatments and technologies on a timely basis.

“Senators Burr and Casey exemplify what it means to be a research champion,” said Research!America Chair John Edward Porter. “They have each devoted their energies to ensuring that federal funding and policies are aligned with the goal of accelerating medical progress, from basic research to private sector discovery to timely patient access.  Congressional support for the public and private sector research pipeline is critical to capitalize on recent breakthroughs, maintain our global competitiveness as other nations dramatically ramp up their investments in medical innovation, and fight back against costly, devastating illnesses such as Alzheimer’s, Parkinson’s, heart disease and cancer.”

During their combined 14 years of experience as members of the Senate Health, Education, Labor and Pensions (HELP) Committee, Sens. Burr and Casey have championed legislation to catalyze and improve the research pipeline, and they jointly sponsored a bipartisan letter in support of research conducted by the National Institutes of Health.

Sen. Burr introduced and achieved passage of legislation to establish the National Institute of Biomedical Imaging and Bioengineering (NIBIB), which uses pioneering research to develop new technologies aimed at diagnosing, preventing and treating, among other conditions, breast cancer and spinal cord injuries. He also introduced the Promoting Accountability, Transparency, Innovation, Efficiency and Timeliness at FDA (Patient’s FDA) Act with Sen. Tom Coburn, MD (R-OK) to ensure timely patient access to new drugs and medical devices. Sen. Burr’s Biodefense and Pandemic Vaccine and Drug Development Act of 2005 allows rapid development of certain drugs and vaccines in case of a pandemic or natural disease outbreak.

“I am honored to receive this award, but the people who really deserve it are the hard-working, dedicated and brilliant researchers and scientists in North Carolina and across the country who are making breakthroughs every day that enhance the quality of life and, in many cases, save lives,” said Sen. Burr. “They are the ones we should all be honoring.”

Sen. Casey led a bipartisan group of lawmakers in introducing the Life Sciences Jobs and Investment Act of 2011, legislation that would double the R&D tax credit for life science firms. He also introduced the Creating Hope Act of 2011, a bill to foster the development of research breakthroughs for rare and neglected diseases, such as pediatric cancers and malaria. Last year, he sent a letter to Senate appropriators to inform them of a breakthrough in genetics research and emphasize the importance of maintaining NIH funding.

“I am honored to receive the Edwin C. Whitehead Award for Medical Research Advocacy from Research!America,” said Sen. Casey. ”Pennsylvania is a leader in the area of medical research which is critical to preventing, treating and curing diseases. Medical research is also a field that employs thousands across the state and plays an important role in the Commonwealth’s economy. I believe that it is essential to continue support for medical research because of the potential health benefits for all Americans and the importance of ensuring that our nation remains at the forefront of medical innovation.”

The Whitehead Award will be presented to Sen. Burr and Sen. Casey at Research!America’s 2013 Advocacy Awards dinner on Wednesday, March 13, in Washington, DC. The program honors outstanding individuals and organizations in advocacy for medical, health and scientific research. The Whitehead Award, named in honor of Research!America’s founder, Edwin “Jack” Whitehead, recognizes exemplary leaders, particularly those in public office, who have demonstrated a deep commitment to advancing biomedical and health research as a national priority and who galvanize others in support of science.

Other Advocacy Award winners include Diane Rehm, author and host of WAMU 88.5 and NPR’s “The Diane Rehm Show”; John F. Crowley, patient advocate and inspirational entrepreneur, chairman and CEO of Amicus Therapeutics, Inc.; John Mendelsohn, MD, director, Khalifa Institute for Personalized Cancer Therapy and former president at The University of Texas MD Anderson Cancer Center; Mark Rosenberg, MD, president and CEO, The Task Force for Global Health; and the California Institute for Regenerative Medicine (CIRM).

About Research!America’s Advocacy Awards Dinner

The annual Research!America Advocacy Awards program was established in 1996 by the Board of Directors to honor outstanding advocates for medical, health and scientific research. Recognized individuals and organizations are those whose leadership efforts have been notably effective in advancing our nation’s commitment to research. This year the awards event will take place on March 13, 2013, at the Andrew W. Mellon Auditorium in Washington, DC. For more information, visit www.researchamerica.org/advocacy_awards.

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.

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Research!America Honors Trailblazers In Health Research Advocacy

Diane Rehm; John F. Crowley; Dr. John Mendelsohn; Dr. Mark Rosenberg;and California Institute for Regenerative Medicine to Receive 2013 Research!America Advocacy Awards

WASHINGTON-October 2, 2012Research!America’s 17th annual Advocacy Awards will convene leaders from government, academia, industry and health advocacy organizations to honor leading medical and health research advocates of our time. The event will take place on the evening of Wednesday, March 13, 2013, at the Andrew W. Mellon Auditorium in Washington, DC.

The 2013 Advocacy Award winners are Diane Rehm, author and host of WAMU 88.5 and NPR’s “The Diane Rehm Show;” John F. Crowley, patient advocate and inspirational entrepreneur, chairman and CEO of Amicus Therapeutics, Inc.; John Mendelsohn, M.D., director, Khalifa Institute for Personalized Cancer Therapy and former president at The University of Texas MD Anderson Cancer Center; Mark Rosenberg, M.D., president and CEO, The Task Force for Global Health; and California Institute for Regenerative Medicine (CIRM). A sixth Advocacy Award winner will be named by Research!America’s Board of Directors in December 2012.

“This year’s awardees embody the spirit of pioneers in advocacy, setting high standards for others to follow in achieving a greater awareness and appreciation for research to improve health,” said Mary Woolley, president and CEO of Research!America.”We applaud their leadership and tenacious dedication to informing and engaging the public.”

“The tireless efforts of these extraordinary individuals have brought hope to people affected by devastating diseases worldwide,” said Harry Johns, Research!America Board member, co-chair of the Advocacy Awards selection committee, and president and CEO of the Alzheimer’s Association  “Their collective achievements have greatly contributed to medical progress and innovation in the U.S. and abroad.”

Diane Rehm, author and host of WAMU 88.5 and NPR’s “The Diane Rehm Show,” will be honored with Research!America’s 2013 Isadore Rosenfeld Award for Impact on Public Opinion for emphasizing the value of research and increasing the level of awareness among policy makers and the public of medical conditions such as traumatic brain injuries, Parkinson’s disease, multiple sclerosis and Alzheimer’s disease. Her show reaches millions of listeners around the world, contributing significantly to public understanding of science and health.

Patient advocate and inspirational entrepreneur John F. Crowley, chairman and CEO of Amicus Therapeutics, Inc., will receive Research!America’s 2013 Gordon and Llura Gund Leadership Award for his outstanding efforts to accelerate new treatments for Pompe disease and other genetic diseases. His dedication led to the discovery of a treatment for Pompe disease, saving the lives of his children, Megan and Patrick. Crowley’s remarkable story and perseverance inspired the movie Extraordinary Measures starring Harrison Ford and Brendan Fraser.

John Mendelsohn, M.D., director of the Khalifa Institute for Personalized Cancer Therapy at The University of Texas MD Anderson Cancer Center, will receive Research!America’s 2013 Geoffrey Beene Builders of Science Award for pioneering translational research that has provided the foundation for targeted cancer therapies and for his advocacy for increasing our nation’s support for biomedical research. His leadership of MD Anderson during an expansive period of growth, as well as his own groundbreaking discoveries, have transformed cancer treatment, benefiting thousands of patients worldwide.

Mark Rosenberg, M.D., president and CEO of The Task Force for Global Health, will be honored with Research!America’s 2013 Raymond and Beverly Sackler Award for Sustained National Leadership for advancing injury prevention and road safety, reframing the concept that road traffic crashes are not accidents. His advocacy has increased funding for research and programmatic interventions for injury control and improved traffic safety not only in the U.S. but also in many developing countries.

The California Institute for Regenerative Medicine (CIRM) will receive Research!America’s 2013 Paul G. Rogers Distinguished Organization Advocacy Award for its role in accelerating stem cell research and the potential development of a new generation of promising therapies for previously untreatable human disorders. CIRM-funded discoveries have laid the foundation for a new industry in California and attracted top-level stem cell researchers to the state. Jonathan Thomas, PhD, JD, and chairman of CIRM’s Independent Citizens Oversight Committee, will accept the award on behalf of the organization.

The annual Research!America Advocacy Awards program was established by the Board of Directors in 1996 to honor outstanding advocates for medical, health and scientific research. Recognized individuals and organizations are those whose leadership efforts have been notably effective in advancing our nation’s commitment to research.

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 that represent the voices of 125 million Americans. Visit www.researchamerica.org.

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