This week Science Life is reporting from the 20th BIO International Convention, an annual gathering of biotech industry leaders, from pharmaceutical companies and insurance providers to technology firms and university researchers.
The Affordable Care Act is Here to Stay
Today’s first session focused on the elephant in the room at this conference, the Affordable Care Act and how it will affect the kind of research and development driving many of the companies and agencies in attendance. A constant refrain from many of the sessions at the conference has been that pharmaceutical and biotech companies have to find new ways to measure value in addition to revenue, especially in terms of quality of life for patients.
Each of the panelists had a different perspective on this issue, while mostly coming to the same conclusion: There is still a great deal of uncertainty about how the ACA will be implemented. Jonathan Blum, director of the Center for Medicare, said that the goal for his agency—arguably the biggest driver of change—is to shift payment systems from focusing on the cost of services to what happens to a patient over an “episode of care,” or a time period to deal with a particular health issue. There is tremendous variation across the country in how Medicare services are used, the associated costs and total quality delivered, he said, and a key focus of the ACA is to understand why these differences exist and standardize usage toward the common goal of better care.
The tricky part of this, however, is defining those measures of quality, a concern that has come up again and again during this conference. Marc Boutin, executive vice president of the National Health Council, a nonprofit patient advocacy group, said that this is an area where nonprofit organizations and disease advocacy groups can help, because they are accustomed to defining their success by outcomes—fewer deaths, fewer new diagnoses—rather than revenues. Developing more sophisticated qualitative measures will give companies new metrics on which they can compete, he said.
The underlying concern at this panel, and others, is that even now after parts of the ACA have been implemented, there is still a lot we don’t know about how it will work. Kristine Peterson, CEO of Valeritas, a small device manufacturer, said that the market to raise funds for new development is inherently skittish about uncertainty, and it’s difficult to build the five to ten years it may take to fully implement the ACA into an R&D plan. While everyone was certain that the ACA would ultimately improve care, the actual work to get there still seems very much up in the air.
Looking to the Future: Where Do We Go from Here in Addressing Alzheimer’s
The afternoon’s sessions took a turn back toward the kind of research on diseases left to be done in this environment, one of which—Alzheimer’s—may pose one of the greatest challenges. In a panel focused on the future of Alzheimer’s treatment and research, George Vradenburg, co-founder of advocacy group USAgainst Alzheimer’s, said, “We’re going the wrong way” on research for the disease. With death rates going up 60 percent each year, academics cannot publish new studies and complete clinical trials fast enough, he said, and pharmaceutical companies aren’t sharing enough data about their work to avoid wasted effort. The key, he said, is developing strategies for early intervention in patients with very mild or even no symptoms of cognitive decline, because by the time someone does demonstrate more serious symptoms, it’s too late.
Richard Mohs, a neuroscience research fellow at Eli Lilly, said that he is encouraged by recent studies on disease mutations and biomarkers that have been linked to Alzheimer’s, and echoed Vradenburg’s sentiment that the science is pushing us toward earlier intervention. The problem, he said, is that developing accurate measures of subtle, early cognitive decline is difficult. Sandra Weintraub from the Alzheimer’s center at Northwestern University spoke about some of the strategies her colleagues are developing to intervene at earlier stages, including a partnership with Chicago’s Lookingglass theater to host improv classes to help early onset patients preserve memory function. It’s part of a strategy she called “cope with hope,” or helping patients and caretakers dealing with Alzheimer’s cope with the disease until better treatments are developed.
Rare Disease Research at Comer Children’s Hospital
The last session of the day featured members of our own faculty from Comer Children’s Hospital talking about the kind of innovative research we’re doing to tackle rare pediatric diseases. First, Dr. Erika Claud, associate professor of pediatrics, spoke about her work on neonatal necrotizing enterocolitis, an inflammatory bowel disease that affects premature infants. A key to her research is understanding the gut microbiome in these infants to learn why some are able to thrive and others get sick. By utilizing advanced DNA sequencing through our partnership with Argonne National Laboratory, she and her colleagues are looking to formulate a therapy that can intervene by inducing a healthy microbiome in premature infants.
Finally, Dr. Elizabeth McNally, director of the cardiovascular genetics clinic, spoke about her research on the genetic defects that cause muscular dystrophy. Again, through our partnership with Argonne National Laboratory, she and her colleagues have identified strategies to treat MS and induce its milder forms through gene correction and protein replacement therapies. It’s the kind of exciting research that demonstrates what can be done through collaborations of the best minds in medicine and biotechnology, ending a day that started off with some gloom and doom on a high note.
More BIO International 2013 coverage from Science Life: