Why the 21st Century Cures Act is a Good Thing

A Q&A with Mary Woolley, President and CEO of Research!America

Originally published Feb. 8, 2017

Q: You attended the December signing by President Obama of the 21st Century Cures Act and are recognized to be a strong supporter. Yet harsh criticism of it has quickly appeared in JAMA, BMJ, a variety of other venues, as well as on these pages. Please tell our readers why this is good legislation and how the public health will be protected from exploitation in this very different regulatory world.

A: The bi-partisan 21st Century Cures Act is grounded in a commitment to assuring that our nation’s research ecosystem has the capacity to accelerate the pace at which safe and effective medical advances reach patients. The Act will expand the efficiency, reach and impact of medical discovery in a manner that sustains crucial safeguards against unsafe or ineffective products. The law finances more research, helps to reduce the administrative cost surrounding basic research, and takes additional steps to overcome challenges the Food and Drug Administration (FDA) faces. Patient groups, health care professionals, academic leaders, industry leaders and the FDA and the National Institutes of Health (NIH) were frequently consulted regarding provisions of this bipartisan bill, and their insights were incorporated. We at Research!America were closely involved throughout development of the bill, and are pleased that it crossed the finish line last December.

After years of automatic spending cuts and flat-funding, researchers have been stressed as they work to find solutions to deadly and complex diseases. The 21st Century Cures provides some relief in that regard with an initial $352 million in FY17 to support the NIH Precision MedicineBRAIN, and Cancer Moonshot initiatives. Congress recognizes that these dollars are targeted and temporary; they do not supplant the need to grow NIH’s annual budget. As reflected in surveys that Research!America commissions regularly, Americans recognize the importance of federally-funded research and support streamlining the pursuit of medical research and innovation.

The FDA, which has for years been underfunded, will also receive new funding with an initial $20 million in FY17 to improve efficiencies in the R&D pipeline. This new funding, in combination with other provisions of the law, is particularly meaningful as it will give the FDA more flexibility to recruit additional experts needed to assure that our regulatory system can properly evaluate rapidly evolving science in areas such as immunology and regenerative medicine.

One important example of rapidly evolving science is the potential to diversify the evidence base used to evaluate the safety and efficacy of medical advances by leveraging “real world evidence” (RWE). The Cures Act defines real world evidence as “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than randomized clinical trials.” While concerns have been raised that the RWE provisions would force the FDA to relax critical safety and efficacy standards, these provisions were developed with agency input. This section of the law is designed to empower, not require, the FDA to capitalize on real world data. Real world data will be used when — and only when — it is appropriate to do so.

Faster medical progress saves lives. The 21st Century Cures Act will fuel faster progress. It’s incumbent upon research advocates to engage elected officials to build on the Cures Act, and ensure that adequate funding is provided to make the promise of science and innovation a reality in our lifetime.

Copyright: This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.


ASCO Calls for Cancer Trials to Include More Seniors

“In a position statement published online July 20 in the Journal of Clinical Oncology, the American Society of Clinical Oncology has called on the U.S. government and the cancer research community to broaden clinical trials to include older adults.

” ‘Older people living with cancer often have different experiences and outcomes in their treatment than younger cancer patients,’ Julie Vose, M.D., M.B.A., society president, said in a news release from the group. ‘As we age, for example, the risk of adverse reactions from treatment significantly increases. Older adults must be involved in clinical trials so we can learn the best way to treat older cancer patients, resulting in improved outcomes and manageable toxicity,’ she explained.

“More than 60 percent of cancers in the United States occur in people aged 65 and older, the statement authors say, noting the number of seniors will increase in coming years. However, there is a lack of evidence about cancer treatments for the elderly because too few are included in clinical trials, and clinical trials designed specifically for seniors are rare.”


GeneGame: An App That Lets You Be the Scientist

Researchers all over the world have collected vast amounts of data on cancer patients, from genetic information to drug treatment records to cell biopsies, and this information can be very valuable when working to develop new treatments. But the data pool is so vast that finding useful information can take an incredibly long time. Cancer Research UK has now developed a website, called CellSlider, to let the public click through actual microscope slides to identify cancerous cells; it took the public 3 months to analyze as much material as the organization’s scientists can cover in 18 months. Building on that success, Cancer Research UK is preparing to launch an app, GeneGame, which will enable patients to analyze data on their smartphones in a fun, interactive setting. The data will then be sent back to scientists to use in their research at a much faster rate, perhaps hastening the development of a cure.


GeneGame: An App That Lets You Be the Scientist

Researchers all over the world have collected vast amounts of data on cancer patients, from genetic information to drug treatment records to cell biopsies, and this information can be very valuable when working to develop new treatments. But the data pool is so vast that finding useful information can take an incredibly long time. Cancer Research UK has now developed a website, called CellSlider, to let the public click through actual microscope slides to identify cancerous cells; it took the public 3 months to analyze as much material as the organization’s scientists can cover in 18 months. Building on that success, Cancer Research UK is preparing to launch an app, GeneGame, which will enable patients to analyze data on their smartphones in a fun, interactive setting. The data will then be sent back to scientists to use in their research at a much faster rate, perhaps hastening the development of a cure.


GeneGame: An App That Lets You Be the Scientist

Researchers all over the world have collected vast amounts of data on cancer patients, from genetic information to drug treatment records to cell biopsies, and this information can be very valuable when working to develop new treatments. But the data pool is so vast that finding useful information can take an incredibly long time. Cancer Research UK has now developed a website, called CellSlider, to let the public click through actual microscope slides to identify cancerous cells; it took the public 3 months to analyze as much material as the organization’s scientists can cover in 18 months. Building on that success, Cancer Research UK is preparing to launch an app, GeneGame, which will enable patients to analyze data on their smartphones in a fun, interactive setting. The data will then be sent back to scientists to use in their research at a much faster rate, perhaps hastening the development of a cure.


Moffitt Cancer Center Researchers Design Small Molecule to Disrupt Cancer-Causing Protein

“Researchers at Moffitt Cancer Center and colleagues at the University of South Florida have developed a small molecule that inhibits STAT3, a protein that causes cancer. This development could impact the treatment of several tumor types, including breast, lung, prostate and others that depend on STAT3 for survival. The study appeared in the Jan. 15 online issue of Cancer Researcha publication of the American Association for Cancer Research.”


Moffitt Cancer Center Researchers Design Small Molecule to Disrupt Cancer-Causing Protein

“Researchers at Moffitt Cancer Center and colleagues at the University of South Florida have developed a small molecule that inhibits STAT3, a protein that causes cancer. This development could impact the treatment of several tumor types, including breast, lung, prostate and others that depend on STAT3 for survival. The study appeared in the Jan. 15 online issue of Cancer Researcha publication of the American Association for Cancer Research.”


Moffitt Cancer Center Researchers Design Small Molecule to Disrupt Cancer-Causing Protein

“Researchers at Moffitt Cancer Center and colleagues at the University of South Florida have developed a small molecule that inhibits STAT3, a protein that causes cancer. This development could impact the treatment of several tumor types, including breast, lung, prostate and others that depend on STAT3 for survival. The study appeared in the Jan. 15 online issue of Cancer Researcha publication of the American Association for Cancer Research.”