By Congressman Joe Pitts
Five million Americans suffer from the degenerative brain disease known as Alzheimer's. That number is expected to triple by 2050. Despite the millions of sufferers, there is no cure. In fact, much about the disease is a mystery, including why twice as many women suffer from it.
But now, we have the tools to undertake significant studies of diseases like Alzheimer’s and ALS. We can analyze the human genome to understand the code that runs our bodies. The latest in proteomic testing looks at the protein pathways in our cells to see how they may be malfunctioning. Digital technology and cloud computing can build a massive database to look for connections.
There are new possibilities in medical research. There is hope. But there are also obstacles. The Energy and Commerce Committee, and the Health Subcommittee that I chair, have been engaged in a new initiative this year, 21st Century Cures, an effort to help us move from research to cures.
The first phase of this effort has been to promote conversations and listen. We’ve held roundtables and hearings with health leaders in the government, public, and private sectors. Because I believe it is important that we hear from people outside the Beltway, we held one of these roundtables in Lancaster last week.
I was glad to be joined by two of the top government officials involved in the cures process, National Institutes of Health Director Dr. Francis Collins and Food and Drug Administration Commissioner Dr. Margaret Hamburg. We also had the Chairman of the full Energy and Commerce Committee Fred Upton (R-MI), the Ranking Member of the Health Subcommittee Frank Pallone (D-NJ), and the Vice Chair of the subcommittee Dr. Michael Burgess (R-TX).
Joining our discussion were doctors, researchers, an association head and the mother of a patient. By talking with the people who are on the ground, doing the work to bring us cures, we hope to identify the major barriers that stand in the way.
Dr. Collins and former Rep. Jim Greenwood (R-PA), now the head of the Biotechnology Industry Organization, brought up the possibility of a wide-ranging Alzheimer’s study that could lead to new clues to the development of the disease. If a large number of Alzheimer’s patients could be studied with modern diagnostics, on the order of 100,000 or even a million people, there is no telling what we could find.
That is a big dream, but there are many smaller ways that Congress could help contribute to faster cures and better research. One of the most critical phases of finding a cure is the clinical trial and participants stated their wish for better trials.
Clinical trials test the effectiveness of therapies in the real world. The human body is vastly complex, and something that scientists think might work in the lab may have unintended effects. The trials take significant time and money. The FDA monitors these trials and rules on whether drugs and devices are safe to enter the market. New technologies may make it easier to identify and monitor patients. Better communication and clearer rules from the FDA could make it easier to conduct good, safe trials.
We also heard about the struggles of young researchers. Often young scientists just out of school have great ideas, but have a difficult time getting the financial support to conduct research. They just don’t have the experience in applying for government grants, which end up going disproportionately to larger organizations and those with more experience filling out the paperwork.
Dr. Collins talked about how the Paperwork Reduction Act, which was meant to save federal money, actually makes it very difficult for his researchers to conduct surveys. Asking more than 9 people questions requires approval from the Office of Management and Budget, something that could take months.
There were also concerns about the new medical device tax instituted by the Affordable Care Act. This 2.3 percent excise tax is on total sales, even if a company makes no money they still must pay the tax. Many companies are paying for this by skimping on research, driving innovation to Europe in some cases.
We heard much more in the two hours of discussion. We have a few more informational hearings and roundtables, but this fall we will be working on an agenda to take action. We have to take what we’ve heard and translate it into good, bipartisan bills that can become laws that break down barriers.