Each month, Research!America features a member in its award-winning newsletter The Research Advocate. Among those recently spotlighted in Members Take Action:
- Founded: 1992
- Location: San Francisco, Calif.
- Mission: To discover and develop cancer therapeutics.
At Onyx Pharmaceuticals, Inc., an Amgen subsidiary, our imperative is to build a leading cancer therapeutics company like no other, working toward a unified goal that drives us forward ... to deliver breakthrough cancer treatments to patients with the urgency they deserve. By working in collaboration with patient advocacy groups, healthcare providers and business partners, we push the boundaries of science and cancer therapeutic development.
Onyx's beginning can be credited to taking significant risks on behalf of patients, which was made possible, in part, by NIH funding to develop our understanding of the cellular pathways that some of our therapies target.
Through public-private partnerships, both Onyx and Amgen are working to develop better approaches to addressing serious diseases. In the case of multiple myeloma, a rare blood cancer, Onyx Pharmaceuticals has partnered with academia and patient advocacy organizations, such as the International Myeloma Foundation and the Multiple Myeloma Research Foundation, to develop new diagnostic approaches to better assess the level of disease in patients with multiple myeloma, also known as minimal residual disease (MRD). Understanding the level of MRD will support continuing development of therapies that can completely eradicate all evidence of disease from the patient. In addition, by developing new tools to monitor MRD in myeloma patients, we will be able to incorporate this measurement as a predictor of clinical outcome.
Since 2012, Onyx has been focused on advancing the understanding of the health disparities associated with multiple myeloma and liver disease to drive appropriate and unencumbered patient access to the best possible treatments. Recognizing the racial and socioeconomic disparities in incidence rates and health outcomes for multiple myeloma and liver cancer patients, Onyx began investigating opportunities to advance health equity for patients living with these life-threatening diseases. Through education and engagement of key policy stakeholders about the scientific and social determinants contributing to the health disparities associated with HCC and multiple myeloma, our goal is to develop and disseminate tools necessary to improve access to care for traditionally underserved populations.
In 2013, Onyx and the D.C. Cancer Consortium sponsored a strategic policy roundtable to advance health equity under the Affordable Care Act (ACA) and addressed strategies and models for engaging traditionally underserved populations living with cancer. In October 2014, Onyx will host its second annual roundtable focused on ACA's provisions aimed at reducing health disparities, efforts to increase minority enrollment into clinical trials, and improving access to high quality treatment.
At Amgen and Onyx, our shared mission is to serve patients. The ultimate way that we, as the collective innovation ecosystem - industry, government and nonprofits - can serve patients is through continued and increased funding of basic medical research, which, in turn, supports Research!America's mission of making medical research a national priority.
Research!America is one of the few organizations that advocates for biomedical innovation and supports continued investment in research, which is a core area of focus for Onyx. Research!America's efforts to empower the public to voice their opinions on biomedical research so that all participants in the research community can continue finding ways to meet the needs of patients also aligns with our goals for more engagement in the research process.
Pablo J. Cagnoni, M.D., is the president of Onyx Pharmaceuticals, Inc., an Amgen subsidiary.
- Founded: April 2005
- Location: Northbrook, Ill.
- Mission: To provide patients, customers, community and employees with a bright future by changing tomorrow.
Astellas Pharma U.S., Inc., is a U.S. affiliate of Tokyo-based Astellas Pharma Inc. Located in Northbrook, Illinois, the company serves as the headquarters for the Americas and employs nearly 3,000 people. Astellas is a pharmaceutical company dedicated to improving the health of people around the world through the provision of innovative and reliable pharmaceutical products.
Our commitment to changing tomorrow is measured not only by our focus on bringing innovative and effective new medicines to patients and physicians, but also by the importance we place on the impact we can make within the communities where we work and live. Astellas supports many causes and has created two initiatives to demonstrate our commitment: Changing Tomorrow Day, an annual, worldwide volunteer event, and Science WoRx, a nationwide mentoring program and online resource network for science teachers and their students.
Launched in April 2005, Astellas was formed through the historical merger of Japan's third and fifth largest pharmaceutical companies - Yamanouchi, founded in 1923, and Fujisawa, founded in 1894. Both companies possessed similarities where it matters most - philosophy and vision. But the companies also differed where it matters most - areas of therapeutic concentration and expertise.
Yamanouchi brought a record of developing blockbuster drugs, a pipeline full of promising new compounds and a sales and marketing culture of deeply grounded, data-driven expertise. Fujisawa brought dominance in transplantation, a soaring reputation for in-depth understanding of the disease states and treatments within its market niches, and a track record for developing high-profile, market - leading products that become new standards of care.
With an unrelenting commitment to R&D as a value-creating core of its business, Astellas is consolidated around complementary strengths from Yamanouchi and Fujisawa. Astellas stands on a much stronger, more advanced business platform than either of the former companies could have mounted individually.
At Astellas, we build on our proud specialty heritage to develop innovative products that improve the lives of patients. To achieve this goal, our strategy is to focus on select therapeutic areas in order to develop a deep understanding of specific health conditions and the unique needs of our patients and customers. Our U.S. product portfolio and rich clinical pipeline are grounded within these therapeutic areas.
We share the same values with Research!America to make research to improve health a higher national priority. For more information about Astellas Pharma U.S., Inc., please visit our website at www.astellas.us.
Jeffrey D. Bloss, M.D., is senior vice president of Astellas Scientific and Medical Affairs and oversees medical affairs in the Americas. The mission of Astellas Scientific and Medical Affairs is to "improve patients' lives through trusted scientific and medical expertise that bridges drug discovery, development and commercialization to establish the value of and ensure the safe and effective use of Astellas products."
- Founded: February 25, 1847
- Location: Iowa City, IA
- Mission: The conduct of research, scholarship and creative activities is at the core of The University of Iowa's multiple missions of education, research, service and outreach to the state, region, nation and beyond.
The University of Iowa (UI) has long been recognized as a leader in fields as diverse as health care, the sciences, humanities and the arts, in interdisciplinary studies and in the recognition of creative activities in awarding advanced degrees. The research enterprise is an essential element of the university's identity as one of the most distinguished public research universities in the United States.
The Office of the Vice President for Research and Economic Development is committed to forging new frontiers of discovery by providing resources and support to researchers and innovators at the university, to promote a culture of creativity that enriches the campus, the state and the world. This encouraging environment produces research breakthroughs with local, national and international impacts, particularly in the area of vaccine development.
A UI professor of microbiology discovered the cytomegalovirus (CMV) promoter, a tool to "promote" the expression of proteins that transformed the production of numerous protein-based drugs now on the market. Today, a vaccine is currently under development to protect against lethal pneumonia caused by Staphylococcus aureus (staph) bacteria, including drug-resistant strains such as methicillin-resistant Staphylococcus aureus (MRSA).
"We bring together people with complementary skills to attack problems as a team that no one of them would be able to attack singly," said Daniel A. Reed, Ph.D., vice president for research and economic development. "Our big focus is on how we catalyze more multidisciplinary research." The state's rural aging population presents challenges that require a multifaceted approach.
"The College of Public Health, the College of Nursing, the (Carver) College of Medicine and even our Public Policy Center are intersecting to work on rural health, safety and related issues," Reed said.
A challenge to the work of advocating to advance research is that research funding in inflation-adjusted dollars is at a multi-year low. This places undue stress on the national research infrastructure.
"Advocacy for research funding is critical, especially to explain the lag from research discoveries to impact, which can be a decade or more," Reed said. "Today's discoveries feed the pipeline for future impacts, and discoveries from decades ago are having impacts today."
Explaining the research enterprise is important, as there's a lack of understanding among some stakeholders.
"Many do not understand what happens in ‘those buildings with the glass walls,' and that applies to legislators too," Reed said. "We have to explain that research is a human endeavor and remind people of the passion and curiosity they felt as children."
Research!America's work is important to the university, particularly the organization's messaging regarding the consequences of the failure to invest in research, and the economic and political realities of why it's important for researchers to engage in debate about investment, Reed added.
"If you're not engaged in the debate it doesn't mean the debate won't happen," Reed said. "The decisions will happen, but you just won't be a part of them."
Learn more at http://research.uiowa.edu.
- Founded: 2004, building opened 2008
- Location: Huntsville, AL
- Mission: To translate the promise of genomics into all practical realms-medical, educational, agricultural and commercial-in order to improve our lives.
HudsonAlpha began with biotech entrepreneur Jim Hudson and the philanthropic Alpha Foundation, set up by Lonnie McMillian. With other leaders and more than $80 million in private donations, Hudson and McMillian built a nonprofit research organization that not only advances the life sciences but fosters the collaborations necessary to speed the movement of discoveries out of research labs into patient care. An additional $50 million came from then-Alabama Gov. Bob Riley in August 2005. Five months later, ground was broken for the HudsonAlpha Institute for Biotechnology, located in Cummings Research Park, the U.S.'s second largest research park.
Recognizing the huge potential of genomics to revolutionize medical care, Hudson and McMillian invested in an impressive concentration of genomics research infrastructure and expertise, including Richard M. Myers, PhD, who became HudsonAlpha's president and science director.
Genomics - the study of a living being's entire set of DNA - helps us understand at the molecular level the basis for the development and progression of disease. The genomics field, born from the U.S. government's initial $3 billion public investment in the Human Genome Project between 1990-2003, has the potential to impact every person.
A unique aspect of HudsonAlpha's approach is its co-location of nonprofit scientists with entrepreneurs and corporate leaders who can incorporate innovations into products destined for the medical marketplace.
One success story originated during the 2009 H1N1 flu pandemic. HudsonAlpha faculty investigator Jian Han, MD, PhD, studied the virus' genome, developed an assay and secured a sample of the virus from the Centers for Disease Control and Prevention to validate that his test is indeed an excellent diagnostic tool.
"Dr. Han literally walked across the hall to a company that quickly incorporated the assay into its testing service and secured (Food and Drug Administration) emergency use approval," Elizabeth K. Newton, PhD, vice president for research affairs, said. "All told it took about 13 days to move from discovery into a company's offering."
There are 26 for-profit companies located in HudsonAlpha buildings on 153 acres. These companies' employees regularly cross paths with their nonprofit counterparts at HudsonAlpha.
"The idea is to put under one roof and on one campus both the nonprofit scientists and the for-profit entrepreneurs and companies," Newton said. "Our programs and the design of our physical spaces foster a collaborative atmosphere."
This fall, HudsonAlpha and Science will host a conference in Huntsville on immunogenomics, a field that involves the application of genomic technology to the study of the immune system. Multiple HudsonAlpha scientists contribute to the field, including Han, who developed many technologies for immune repertoire sequencing.
Advocacy for research-and the positive effect that research has on human health-is an important reason for HudsonAlpha's membership with Research!America.
"We're working toward the same end," Newton said. "We want to have a significant impact on the future of society and on human health. We have to join our voices to accomplish that. And public funding of science is absolutely crucial."
Learn more at http://hudsonalpha.org.
- Founded: 1954
- Location: Rosemont, IL
- Mission: To advance the global orthopaedic research agenda through excellence in research, education, collaboration, communication and advocacy.
The Orthopaedic Research Society recently held its 60th annual meeting in New Orleans; the four-day event welcomed more than 3,000 attendees. (The original annual meeting was one day, and eight were in attendance.) ORS is comprised of around 2,900 members; slightly more than 2,000 are based in the U.S. Most are bioengineers, orthopaedic surgeons, biologists and veterinarians.
The musculoskeletal system gives us shape and allows us to move—and is the primary concern of those involved in orthopaedic research. Typically, some associate "bone" to orthopaedics; broken bones, but it is so much more. The musculoskeletal system also includes cartilage, ligaments, tendons, joints and muscle. The areas of research conducted by ORS' membership is broad, as it includes the entire musculoskeletal system and our moving parts; hips, knees, shoulders, elbows, hands, ankles, feet, wrists and spine. As ORS Executive Director Brenda Frederick points out, all of us know someone who suffers from, or has suffered from, a musculoskeletal disease or injury that may be more commonly known as a torn ACL, or rotator cuff, bad hip or knees, or suffer from back pain or arthritis. In fact, "musculoskeletal disorders, injuries and disease are the leading cause of disability in the United States, with an estimated $950 billion annually in direct health care costs and lost wages," Frederick said.
But like so many other areas of research, orthopaedic research has been affected by sequestration and other budget cuts. The National Institutes of Health, through the National Institute of Arthritis and Musculoskeletal and Skin Diseases, is the primary funder of research for ORS members, but other funding sources include the Department of Defense, private foundations and grants.
"Across the board, funding for musculoskeletal research has decreased, leaving many unanswered questions in basic, translational and clinical orthopaedic/musculoskeletal research," Frederick said.
ORS' advocacy is a key tenet in changing this. Its board of directors has a committee dedicated to advocacy, currently led by Lynne Jones, PhD, of John Hopkins Medicine. ORS, along with other orthopaedic groups, also supports a Capitol Hill day—an annual event for more than two decades—that brings together researchers, practitioners and patients for outreach to elected officials.
Other ORS programs include the New Investigator Workshop, held jointly with the American Academy of Orthopaedic Surgeons and the Orthopaedic Research and Education Foundation in May. The workshop aims to help young researchers secure funding for their work. In the fall, ORS will partner with the Icahn School of Medicine at Mount Sinai for a meeting called New Frontiers in Tendon Research.
Research!America's work in advocacy complements ORS' efforts, and that is an important reason for the organization's membership.
"The resources provided by Research!America have proven to be very valuable and will provide us with a basis from which to develop our advocacy agenda," Frederick said. "The Research!America website also provides valuable resources and information that can be used to improve our advocacy efforts. In addition, the ties to Capitol Hill are invaluable. We have also appreciated their level of professionalism and expertise."
Learn more at www.ors.org.
- Founded: 1998
- Location: Metro Atlanta
- Mission: To make kids better today and healthier tomorrow.
Children's Healthcare of Atlanta grew out of the merger of Egleston Children's Health Care System and Scottish Rite Medical Center in 1998. In the years since, it has grown in scope-in 2012, Children's treated patients from all 159 counties in Georgia-and in scale: The addition of Hughes Spalding Children's Hospital in downtown Atlanta gives Children's three hospitals, along with 20 neighborhood locations and access to more than 1,700 pediatric physicians, representing more than 60 pediatric specialties and programs.
Patrick Frias, MD, oversees all physician practices employed or managed by Children's, as well as growing the relationships Children's has with the private practice community. In addition to that, he is responsible for the many research relationships that Children's has with regional academic partners, Emory University, Georgia Tech, the Morehouse School of Medicine and the University of Georgia among them.
Frias' role, as it relates to research, is-in a short version-to develop and execute Children's academic mission.
"We've chosen a strategy at Children's where we leverage the strengths of our partners and they leverage the strengths of Children's," Frias said. "Rather than erecting research buildings that are purely owned by Children's, for instance, we partner with Emory University to leverage all of our resources to promote child health research."
For example, their newest building on Emory's campus also houses a joint biomedical engineering department between Emory and Georgia Tech.
While the expanse of Children's is great, so is the need for pediatric health care services. Frias said that Children's size is justified by the number of patients it treats, nearly 350,000 unique patients in 2013. Moreover, given Atlanta's size and notorious traffic congestion, Frias said that it made sense to utilize several locations.
Children's is also dedicated to research to improve the lives of children. Frias said that Children's recently updated clinical strategy highlights the importance of health services and high impact, bench-to-bedside research.
The steady erosion of federal research funding is a key component to Children's membership with Research!America.
"We all know federal funding is at risk. An organization like Research!America-with a focus to help maintain the funding for our research, emphasize importance of research, and be a voice for research-is key for us," Frias said. "It's very important to be part of an organization that is really keeping research at the forefront day in and day out."
Learn more at www.choa.org.
- Founded: 2005
- Location: Alexandria, VA
- Fight Colorectal Cancer demands a cure for colon and rectal cancer. We educate and support patients, push for changes in policy that will increase and improve research and empower survivors and those touched by cancer to raise their voices against the status quo.
Since its beginning Fight Colorectal Cancer-formally The Colorectal Cancer Coalition (C3)-in 2005, advocacy has been the key component of its mission. When founder and chair Nancy Roach established the organization, it was because she saw an unfilled need for advocacy for colorectal cancer. And that advocacy is certainly needed; according to the American Cancer Society, colorectal cancer is the third most common form of cancer.
An early focus-and success-of Fight Colorectal Cancer, among other groups, was the push to have colorectal cancer included in the Department of Defense's Congressionally Directed Medical Research Programs. And since 2009, CDMRP's Peer Reviewed Cancer program has funded more than $6 million in colorectal cancer research. That was an important component in Fight Colorectal Cancer's larger mission: that a cancer of such prevalence was in need of greater awareness from funders and the public.
"[It was important] bringing a spotlight that research dollars weren't coming to colorectal cancer," said Anjee Davis, the organization's interim executive director.
While colorectal cancer has become more of a priority, Davis said more money is necessary for a disease that was expected to kill more than 50,000 people in 2013. And conquering colorectal cancer isn't a mission solely for researchers; preventive measures, including programs from the Centers for Disease Control and Prevention, are just as critical. Fight Colorectal Cancer advocates for them as well.
The CDC's colorectal cancer screening program has helped prevent deaths from the disease in more than half of the states as well as four Native American tribes. Moreover, it helps narrow a disparity between ethnicities and groups.
"There is a higher mortality rate among African-Americans," Davis said, "and there are lower screening rates within the tribal nations and within the African-American community. As far as an outreach and awareness, we have a campaign called One Million Strong. The campaign highlights the 1 million survivors living in the U.S. today. We're launching that in March in New York City, but we're also incorporating outreach to the African-American community ... There's a definite disparity there and [it's] an issue that needs to be addressed."
The One Million Strong program is entering its second year; its public unveiling came a year ago in Times Square with the help of survivors, advocates and some star power, among them singers Sheryl Crow and Charles Kelley (of the country group Lady Antebellum) and five-time Major League Baseball All-Star Frank White. March also marks Fight Colorectal Cancer's 8th annual Call-on Congress, in which advocates descend on Capitol Hill to increase awareness among policy makers.
Research!America's eye on Congress has been an important reason why Fight Colorectal Cancer joined the alliance.
"I specifically, when I started two years ago, wanted to join Research!America, because of the work [it does] in monitoring research dollars and where the Members of Congress are voting," Davis said. "The thing that I value the most is probably seeing the impact of those research dollars at the state level. So, for us, it was like having kind of an extension of our staff-we couldn't have the studies done, so it was very helpful for our advocacy efforts to be armed with insightful reports and data that we can us in the community."
- Founded: 1889
- Location: Albuquerque, NM
- The mission of the University of New Mexico is to serve as New Mexico's flagship institution of higher learning through demonstrated and growing excellence in teaching, research, patient care, and community service.
By almost 23 years, the University of New Mexico (UNM) predates statehood: The school was founded in 1889 by an act of the Territory of New Mexico. By the mid-20th century, UNM was well established in the medical sciences; in 1994, the school opened its Health Sciences Center (HSC). Today, the HSC features a designated cancer center and 15 academic programs, including medicine, nursing and pharmacy. Research is a critical component of UNM, whether at the HSC or-given the school's proximity to Los Alamos National Laboratory and Sandia National Laboratories-in aerospace and physics.
UNM's provost and executive vice president for academic affairs, Chaouki Abdallah, PhD, has a background in electrical engineering; though his purview is the non-medical parts of the university (including the hard sciences), there are several areas where the hard sciences meet the medical sciences.
For instance, there is UNM's Center on Alcoholism, Substance Abuse and Addictions, which counts health economics among its many areas of expertise. There's also the Robert Wood Johnson Foundation Center for Health Policy as well as Project ECHO (Extension for Community Healthcare Outcomes) at the HSC, which seeks to treat disease in rural and underserved areas. But the biggest joint effort is between the HSC's Cancer Center and material scientists and chemical and nuclear engineers within the university.
"There is a lot of high-quality research that is leading into both companies and patents," Abdallah said.
UNM also has plans to start a school of public health; the school's president, Robert Frank, PhD, has a background in public health and started a similar school while he was the provost at Kent State University. But even before the ceremonial first shoveling of dirt, Abdallah explained the incredible amount of work that goes into opening a new school.
"For things like this, you need to have very specific requirements for accreditation," he said. "I was thinking we could just pull people from different areas to start it, then grow it. But you actually need to have five specific areas, you need to have at least five faculty members whose primary appointment is in public health. The accreditation requirements are also very specific on the name of the school as well as the academic programs within the school."
Abdallah said concern about funding from the National Institutes of Health is a major reason why UNM is a Research!America member.
"We are concerned like everybody else about the drop in federal funds. We have a lot of NIH funds on both sides of campus," Abdallah said. "We have many R01 [grants], and we really need to make sure that they're maintained and increased because many of our researchers do depend on them."
In 1997, a year after the gene that causes Friedreich's ataxia was identified, Ron and Raychel Bartek received the diagnosis of their son, Keith, and discovered that there was no treatment for the disease and no group dedicated to finding one. So they started FARA. Since then, FARA alone has funded more than $20 million in research on the disease and helped secure a similar amount from co-funding organizations. That has helped lead to multiple clinical trials encompassing a range of treatment approaches.
FARA President Ron Bartek remembers when he and his wife decided to start a nonprofit dedicated to finding a treatment for Friedreich's ataxia.
"On the night we received Keith's diagnosis, we saw online there was no treatment, very little research into the disease and no organization devoted entirely to supporting such research. We were living in the DC suburbs, we were 30 minutes from the [National Institutes of Health], and we both worked on Capitol Hill," he recalled. "Maybe we're supposed to do this."
Things moved quickly after that: On the day FARA was approved for incorporation, Bartek drove to the NIH to apply for a workshop grant. A friend helped them quickly attain 501(c)(3) status; with that secured, the NIH approved the workshop grant. Two months later, FARA co-hosted with the National Institute of Neurological Disorders and Stroke the world's first Friedreich's ataxia workshop. Sixty-five scientists from around the world joined 15 NINDS researchers for that first conference.
Though a treatment remains elusive, that's about the only thing that hasn't changed. The last conference on Friedreich's ataxia was held in Strasbourg, France; 200 people attended, and many others were turned away due to space limitations. Pharmaceutical companies and advocacy organizations were well-represented. The Barteks' initial goal was being accomplished.
"That was our first objective: to grow the field," Bartek said.
Most importantly, an approved treatment may be near at hand—with others following along and providing a "cocktail therapy" to treat the disease effectively and eliminate it in subsequent generations.
Research!America's obvious effectiveness at advocacy is a critical reason why FARA is a member.
"Research!America is a key advocacy organization in support of medical and health research across the front. They're so effective at the highest levels and in collaborating with other advocates," Bartek said. "With all of us pulling together, we're such a powerful voice."
When the Pancreatic Cancer Action Network was founded in 1999, the 5-year relative survival rate was 5.3%, according to National Cancer Institute statistics. In the intervening years, the survival rate has remained stubbornly low. What is different is awareness: Today, more research is being conducted by more scientists, and the pancreatic cancer advocacy community has never been bigger. So too for Manhattan Beach, CA-based Pancreatic Cancer Action Network, which hired its first employee in 2000 and will soon hire its 100th.
President and CEO Julie Fleshman, JD—who was that first employee—can explain just how far the science around pancreatic cancer has come. Tumors tend to be surrounded by, in Fleshman's words, a dense microenvironment that makes drug delivery difficult; it's one of many reasons why treating pancreatic cancer is so challenging.
"That finding ... is something we've only started to appreciate the significance of in the last five years," Fleshman, who lost her father to pancreatic cancer in 1999, said. "It shows you that when you finally dedicate time and resources and people to a topic, you start to understand what the challenges are, which then allows people to focus their efforts in the right way to make scientific advancements."
And the Pancreatic Cancer Action Network sees those advances are coming. Early in 2011, the organization announced an initiative to double the survival by 2020; now, seven years out, Fleshman said reaching the goal will be difficult but far from impossible. Even so, the future holds promise for patients. There are 142 active clinical trials, according to the organization's comprehensive pancreatic cancer clinical trials database. Moreover, earlier-stage research is ongoing and being taken up by an ever greater number of scientists.
"That's probably the most significant change," Fleshman said. "We're learning more about the disease, we're understanding better the challenges about why it is so difficult to treat, and that is going to get us to changing patient outcomes in the future."
And because research is such a critical part of the puzzle, Fleshman said it's important for the organization to be a Research!America member.
"Biomedical research is a key priority for our organization," she said. "With pancreatic cancer, there is no detection tool, and we need better treatment options. We're not going to make those scientific advances unless there is more federal funding for the disease. We're very proud to partner with Research!America to make medical research a national priority."
Founded in 2002, the Association of Clinical Research Organizations-ACRO-began with five institutional members and currently has eight, all of which are in the private sector; those eight companies are far and away the giants of the industry. Though academic and nonprofit organizations are not prohibited from becoming ACRO members, ACRO is focused on the private sector.
ACRO began as a trade organization designed to promote and represent clinical research organizations; little more than a decade later, ACRO is much more involved in advocacy and legislative and regulatory affairs. And it's not just American regulatory affairs; the group keeps abreast of developments in Europe and emerging markets, like India, wherever its members do business.
"We're being a little more proactive," said John Lewis, ACRO's vice president for public affairs, "having started out playing defense primarily."
Though ACRO's website lists a number of key issue areas, the most pressing domestic issue over the past several years has been taxation. One particular concern was tax repatriation legislation, since ACRO's members are mostly U.S.-based but roughly half of their revenues come from outside the U.S., Lewis said. ACRO's members are also engaged with certain tenets of the R&D tax credit.
"In other countries-the U.K., France, Canada, Austria, a number of other countries-CROs can claim an R&D tax credit," Lewis said. "... And there are many factors, but we think that's one of the factors why more research is being done outside the United States."
According to clinicaltrials.gov, as recently as 2008, about 53% of industry-sponsored clinical trials were conducted in the United States. That percentage dropped to about 47% in 2012.
Being a member of a respected organization like Research!America enables ACRO to leverage its public policy messaging around the promotion of medical research. Membership also opens up opportunities to participate in Research!America's polls, such as the recent one on public attitudes toward clinical trials.
"By being able to partner with Research!America on the clinical trials poll, we could draw a broader audience for our core issue. We subsequently shot videos with Mary Woolley, which added great content for our YouTube channel, and we are now discussing a Hill briefing on clinical trial participation," Lewis said. "So, from ACRO's perspective, we were able to co-sponsor important research, boost our communications activities and take our message to Congress. This is a great ‘return on investment' from our membership."
From the beginning, the American Chemical Society has had a tie to medicine: It was founded in 1876 by 35 chemists at the College of Pharmacy of the City of New York. Chemistry in the pursuit of health remains a focus for ACS and its membership-now more than 163,000. The organization employs nearly 2,000 people; most work to produce the organization's more than 40 peer-reviewed journals and multiple databases that are indispensable to scientists worldwide.
On August 25, 1937, President Franklin D. Roosevelt signed the law that gave ACS its congressional charter. Its mandates included the promotion of research and advancement of the science; maintenance of standards of ethics and education; increase and diffusion of chemical knowledge; and promotion of the science in the interest of public happiness and welfare, education and economic development.
This language, from a 76-year old public law, may seem dusty. Instead, it is dynamic: Madeleine Jacobs, ACS executive director and CEO, refers to the charter each time she meets a new employee.
"It guides us in everything we do," Jacobs said. "We're very proud to be one of the few organizations to have it and, believe me, I cite this all the time."
Another guiding principle is ACS' tagline, "Chemistry for life," which intentionally incorporates the facet of medical research. Chemistry is the basis for pharmaceuticals, after all, and Jacobs said ACS has 40,000 industrial and academic members involved in the life sciences in some way. She added that many ACS members receive funding from the National Institutes of Health. "It's a big source of funding for a large number of our academic members," she said.
Jacobs explained that ACS advocates for a "vital, robust and sustained" scientific enterprise. Other prongs of its advocacy include a favorable environment for industry and entrepreneurs as well as a workforce that is well-educated in the STEM fields.
Those are also tenets of Research!America's advocacy, and that alignment is a key reason why ACS is a member of Research!America's alliance.
"I don't think that we can be successful working in a vacuum, working individually. I think we can be much more successful when we work together," Jacobs said. "[Research!America is] the kind of coalition we want to be involved in ... Whatever we can bring to the table in helping you achieve, I think it also means we will be achieving things for our members as well."
Since 1974, AdvaMed-short for Advanced Medical Technology Association-has represented the medical technology sector. Today, the organization has more than 300 members of all sizes, representing medical devices, diagnostic products and health information systems.
The idea of a medical device may conjure images of a cutting-edge machine using breakthrough technology or perhaps the latest diagnostics or genetics test; indeed, that would certainly qualify. But items such as tongue depressors and hospital gowns also fall under the umbrella of AdvaMed's membership.
"In a sort of broad-brush way, it's everything that's used in medicine that's not a drug," explains David Nexon, AdvaMed's senior executive vice president.
The medical device tax that was enacted with the Affordable Care Act is a key policy concern for AdvaMed at the moment. "It's such a drain on our U.S. competitiveness and our companies' ability to fund research and development," Nexon said.
Other policy concerns include keeping America's taxes competitive globally as part of any tax reform movement; changing the incentives in the reimbursement system to focus on sustaining medical progress while reducing costs; helping AdvaMed's members navigate the regulatory and payment structures outside the U.S.; and continually working with the Food and Drug Administration to improve the agency's efficiency at reviewing new technology. Like pharmaceutical companies, medical device companies pay user fees to FDA in order to speed up the approval process; sequestration has affected FDA's ability to use those fees for their intended purposes.
"The sequester, with the way it's set up, it keeps you paying the user fees but then doesn't let FDA use a portion of the money," Nexon said, "and that's clearly wrong."
Nexon said that Research!America's focus on advocating for all research, with the eventual end goal of better treatments for patients, is a key reason why AdvaMed is a member.
"I think Research!America is a very powerful voice for the importance of both public investment in research, but also creating a climate in the United States that's friendly to medical progress and developing innovation, whether that comes out of the private sector or the public sector," Nexon said.
To learn more, visit http://advamed.org.
The genesis of the Parkinson's Action Network (PAN) goes back to 1987, four years before the organization's founding. That year, Joan Samuelson left a career in law after being diagnosed with Parkinson's disease; she threw her might into advocating for people living with Parkinson's. Four years later, PAN was born, and its advocacy continues today.
PAN is a unique organization in the patient advocacy world; it represents the entire Parkinson's community on funding and quality of life policy priorities for those living with the disease. PAN works with other national Parkinson's organizations and is the only organization addressing government programs and policies that impact the community.
"We've worked on a vision that if our community came together on policy issues, with one, singular voice, we would be much more effective," said PAN CEO Amy Comstock Rick, JD, who became a Research!America Board member in March. "Really in the last 10 years, we've been able to effectively achieve that mission so that PAN is the only organization in the Parkinson's community that works on policy issues, even though we have a number of national organizations."
Augmenting its policy expertise is PAN's robust grassroots network, with directors and assistant directors at the state level and volunteers in key congressional districts. But, Rick points out, PAN does not maintain its own chapters; rather, consistent with its goal of unification, it relies on the chapter structures of the other national organizations to reach the community.
"PAN does advocacy work through our grassroots leaders," Rick said. "... Without these advocates, the Parkinson's community couldn't accomplish all of the work we do each year. These advocates are the core of PAN."
The chance to network beyond the community and beyond Washington are key reasons for PAN's membership with Research!America.
"Our structure is so unique ... the goals of Research!America are really the same goals in terms of a unified voice. Research!America gives us not only the opportunity to work with other disease organizations," Rick said, "it also is a great way for us to work with other members of the community who have the same ultimate goal, which is getting therapies out there ... You have to get to know people and see things from their perspective, and having an organization like Research!America that brings us together really adds value to that."
In 1976, Ken Stuart, PhD, opened the Issaquah Group for Health and Environmental Research, which was set up to foster the best and brightest researchers who were working to combat trypanosomes and related parasitic diseases. Today, the organization is known as Seattle BioMed, and it employs 15 faculty members and more than 330 workers in all.
Despite its expansion over the past 37 years, current President Alan Aderem, PhD, explains that Seattle BioMed (officially known as Seattle Biomedical Research Institute) has stayed true to its roots. The organization's focus lies in the basic research that leads to better understanding of, and interventions for, tuberculosis, malaria, HIV and parasites such as those causing African sleeping sickness-diseases that, at a fundamental level, remain a mystery.
Aderem was one of three co-founders of the Institute for Systems Biology, also a Research!America member. Aderem believes that systems biology, when integrated with immunology and infectious disease research, can accelerate progress in the development of vaccines, drugs and diagnostics, and he is implementing that approach at Seattle BioMed. There's already one success story.
"We have a program in South Africa with 6,000 adolescents all latently infected with TB. We took blood from them every six months over the course of two years. In that time, a small number of them became ill with active TB," Aderem explained. "By comparing the networks within the wide-circulating white blood cells, we were able to come up with a predictive network that allows us to tell very early on whether or not a kid with a latent infection is likely to get sick."
Aderem said that combating public health threats such as TB is critically important to the U.S.
"Totally drug resistant TB is a very real danger. This is especially true because TB is so easy to spread worldwide," Aderem said. "We need to make lawmakers aware of this issue because it's a major public health threat-not only in resource-poor countries, but also in the United States. Despite the fact that the bacterium that causes tuberculosis was discovered 130 years ago, we still don't have an effective vaccine against it. We need to understand the basic mechanisms by which the bacterium subverts the immune system. Continued investment in scientific research is critical, and the advocacy and public polling carried out by Research!America helps bring these issues to lawmakers' attention for the collective of medical research institutes that includes Seattle BioMed."
Learn more at www.seattlebiomed.org.
From its original 121 member institutions when it was founded in 1969, AACN has grown to more than 700 member institutions in all 50 states, DC and Puerto Rico. Besides advocacy, AACN is a resource to its members in a number of other ways: sponsoring an autonomous accreditation unit, developing curriculum standards and warehousing statistics about nursing schools and students across the country.
AACN's CEO, Geraldine "Polly" Bednash, PhD, RN, came to AACN in 1986; she explains the organization's growth like this: "When I first started at AACN, we had seven staff members, and this organization was basically a networking group for deans of nursing programs," she said. "Now, however, AACN has nearly 50 employees and strives to benefit everyone involved in nursing academics-deans, faculty, students, grad students, researchers and business staff."
As a profession, nursing has expanded similarly. Besides the traditional roles of nurses as frontline caregivers, nurses can now be found as CEOs and high-level government appointees. (Bednash points out that Marilyn Tavenner, acting administrator of the Centers for Medicare & Medicaid Services, began her career as a nurse.)
"We've become a much more expansive career option, with lots of places where nursing knowledge and experience and science is important to the decisions shaping both the delivery and the business of health care," Bednash said.
Advocacy is another key facet of AACN's work. Nurses can play a key role in explaining the implications of research to legislators and the need to follow the evidence when making health care policy. To that end, AACN has extensive resources for nurse scientists and nursing researchers to understand and explain why its work is important.
Research!America's own work in that area is a key reason why AACN is a member.
"AACN applauds Research!America for delivering the message that science is an important part of this country's investment for the future, and that the National Institutes of Health is a vital resource for improving the health care of our nation's population," Bednash said. "Research!America is making clear that research is not some esoteric, distant activity created by people in obscure places, but in fact is critically important to addressing this country's major health care concerns."
Learn more at www.aacn.nche.edu.
The Georgia Research Alliance doesn't conduct research itself; instead, as its name implies, it facilitates research collaboration among six universities in the State of Georgia: Clark Atlanta University, Emory University, the Georgia Institute of Technology, Georgia State University, Georgia Health Sciences University and the University of Georgia. Since 1990, GRA has leveraged $525 million of state funding into $2.6 billion in additional public and private financing; moreover, it has played a role in the creation of more than 150 companies.
As President and CEO C. Michael Cassidy explains it, GRA at its heart is an economic development organization. It achieves its goal of growing the state's economy by first recruiting world-class scientists and building R&D capacity and, later, shaping and seeding companies around the most promising discoveries. GRA is planning to scale up the latter in the near future, but not at the expense of the former.
"We have an overflowing pipeline of really interesting research activity that's coming out of the universities," Cassidy said. "We're going to put some more tools in place to assist in translation and commercialization of those discoveries."
GRA is broadly focused; Cassidy identified Internet security and energy management products as ones in the pipeline that have him excited. But biomedical research plays a critical role in the state's economic development plans, and more than half of GRA's portfolio involves biomedical research.
Looking back, Cassidy says that the Emory Vaccine Center has been a shining example of how GRA proves the power of business, government and academia working together. In the mid-1990s, GRA and Emory University lured to the state noted vaccine researcher Rafi Ahmed, PhD, as part of GRA's Eminent Scholars recruitment program. Ahmed launched the Emory Vaccine Center in 1996, which has since secured more than $260 million in research funding. One of the discoveries at the Emory Vaccine Center led to the creation of GeoVax, which employs 14 people in suburban Atlanta and reported nearly $5 million in revenues in 2011.
Policy perspective from Washington is a key factor for GRA's membership with Research!America.
"We derive tremendous value through our membership in Research!America. Insight into what's going on at the federal level is giving us a better idea of how to shape our own strategies and policies, and Research!America's opinion polls have been particularly informative," Cassidy said. "GRA was very active as one of the sites within the global health initiative and really enjoyed working with the Research!America team. We are much more tuned in to Washington in ways that we probably wouldn't have been on our own."
To learn more, visit www.gra.org.
The Children's Hospital of Philadelphia was founded in 1855, but it took nearly seven decades for research to become part of the institution's mission. Growing from a one-room laboratory in 1922, Children's Hospital has made incredible progress since: the basis of the foundation of the Society for Pediatric Research, the country's first pediatric research department, and numerous scientific breakthroughs and vaccine discoveries.
Children's Hospital's 10 Centers of Emphasis encompass seemingly disparate research areas, but all contribute to the greater strategic goals of the hospital's research efforts.
"Our interests are embedded in those Centers of Emphasis," said Philip R. Johnson, MD, director of the hospital's research institute as well as its chief scientific officer and an executive vice president. The centers that Johnson oversees are dedicated to applied genomics, autism, biomedical informatics, cellular and molecular therapies, pediatric cancer, developmental biology and pediatric disorders, injury research and prevention, mitochondrial and epigenomic medicine, pediatric clinical effectiveness, and even health care policy.
The hospital also supports Research Affinity Groups, which Johnson likened to a "grassroots" effort among researchers to explore topics of interest. The groups, designed to encourage interdisciplinary approaches to child health and development, cover areas different from the Centers of Emphasis.
Johnson's own research centers on finding a vaccine for HIV, but not in the traditional way a vaccine works. Instead, viruses are used to carry genes that code for HIV antibodies. When injected into muscle, the muscle takes over production of those antibodies which can then defeat HIV. Johnson said this concept is nearing a clinical trial in humans. The hope, then, is that such an approach might be useful in developing similar vaccines for other diseases and conditions as well.
Research!America's efforts to educate the public and policy makers are a big reason why Children's Hospital is a member, Johnson said.
"It's amazing the amount of misunderstanding about research that exists in the general population," Johnson said. "... They understand it's important, but they don't understand the mechanics of it, the politics and the support that's required.
"Research!America does a better job of that than anybody else, and that's why we're very supportive of the activities."
For more, visit www.research.chop.edu.