The Power of Research Networks
Research involving people as subjects is often both paradigm-shifting and logistically very difficult. You can’t control for genetic and environmental variables with people the way you can with cells in a dish. You can’t ensure that an adequate number of people will agree to participate in your study. And sometimes you can’t ethically include the perfect control condition in your study the way you can at the laboratory bench. Research involving children is among the most difficult kinds of human subjects research. This is especially true for research on medical conditions that are rare or infrequent in the general population, but life-threatening for the children afflicted with them.
One way physician-scientists manage to conduct such research is to form national networks that allow them to recruit patients from all around the country and implement the same research techniques and procedure in “matching” populations everywhere the studies are being done. Often, these networks include a central office to which data collected in a standardized way are sent. The data is analyzed by someone who is not involved in the day-to-day examination and management of the patients enrolled or in the collection of the data from them. That way, the information gets an unbiased review before it is reported in the scientific and medical literature. And, with many, many medical centers participating, it becomes possible to enroll a sufficiently large number of children in the study that, even for rare conditions, one stands a chance of arriving at generalizable, useful conclusions.
Golisano Children’s Hospital participates in many of these research networks. Here are just a few.
PALISI stands for Pediatric Acute Lung Injury & Sepsis Investigators. This national network of pediatric critical care medicine physicians at 78 pediatric intensive care units across North America hopes to identify optimal supportive, preventive and therapeutic strategies for acute lung injury, sepsis, multi-organ failure, and other acute, life-threatening pulmonary or systemic inflammatory syndromes that affect infants and children. To put it as it is stated on their website (www.palisi.org), “PALISI network members have the ultimate goal of improving the care and health outcomes of critically ill infants and children.” Because children with many different kinds of illnesses can get sick enough to need the intensive care unit, PALISI studies investigate critical illness aspects of conditions as varied as Type 1 diabetes, acute lymphoblastic leukemia, influenza, and deep vein thrombosis. The network has published their studies in some of the highest-impact journals in their field, including JAMA, New England Journal of Medicine, Critical Care Medicine, and Annals of Surgery.
COG is the Children’s Oncology Group – a research network devoted to the optimization of diagnosis and treatment strategies for childhood cancer. COG is an essential research network because, thankfully, cancer is an uncommon disease in childhood. Furthermore, there are so many kinds of childhood cancer that for any one kind, a single hospital or medical center may not see enough patients in a year or two to do a valid study. But the strength that comes from working and planning together (www.childrensoncologygroup.org) is evident in the great strides that have been made in the treatment of children with leukemia, lymphoma, and some kinds of brain tumors, just to name a few. In fact, COG and its predecessors, Children’s Cancer Group (CCG) and Pediatric Oncology Group (POG) have been so successful that they now have the privilege of helping define the best practices for long-term survivors of childhood cancer – a concept that seemed like an untenable dream just two or three decades ago.
COG studies range from biology studies that seek to understand the origins and subtypes of childhood cancer to treatment studies that seek to optimize surgery, chemotherapy, radiation therapy, and bone marrow and stem cell transplantation for children with cancer, to outcomes and psychosocial studies that seek to enhance quality of life for children and families dealing with and surviving childhood cancer. This network receives funding from the National Institutes of Health for its important work.
The CF Foundation Therapeutics Development Network (TDN) is a nationally-coordinated collaboration of Cystic Fibrosis (CF) Centers that brings together experts from across the U.S. to evaluate the safety and effectiveness of new CF therapies through clinical studies. The participants in this network work continually to improve research methods and pursue new areas of scientific inquiry. Seventy-four CF Foundation-accredited care centers with demonstrated expertise in clinical research recruit study participants and conduct clinical trials. A group of laboratories and interpretation centers specialize in developing and measuring CF clinical trial outcomes in a uniform way across the whole network. Finally, a central Coordinating Center supports new clinical trial development, trial management, and data analysis.
Since its founding in 1998 as a subset of the CF Foundation’s care center network, the TDN has conducted more than 100 clinical studies for CF in a wide range of therapeutic areas, including modulators of the function of the mutated protein responsible for the disease, anti-infectives, anti-inflammatories, nutritional therapies, and airway surface liquid hydrators. These studies have resulted in more than 175 publications.
The success of the TDN (www.cff.org/research/TDN/) has not only helped make new therapies available to people with CF; it also has allowed the TDN to serve as a role model for many other clinical research networks.
The Vermont Oxford Network (VON) is a non-profit voluntary collaboration of health care professionals dedicated to improving the quality and safety of medical care for newborn infants and their families. Established in 1988, VON is today comprised of over 900 Neonatal Intensive Care Units around the world.
In support of its mission, VON maintains a database including information about the care and outcomes of high-risk newborn infants. The database provides unique, reliable and confidential data to participating units for use in quality management, process improvement, internal audit, and peer review.
Health care professionals from member institutions participate actively in clinical trials, long-term follow-up studies, and epidemiologic and outcomes research. Members also have the option of participating in VON’s Quality Improvement Collaboratives for Neonatology consisting of multidisciplinary improvement teams working together to identify and implement better practices aimed at achieving measurable improvements in quality and safety.
VON disseminates the results of its work in publications, in scientific articles in peer reviewed medical journals, and on its web site (www.vtoxford.org). The Network also sponsors an Annual Meeting for individuals from member institutions and an Annual Quality Congress for Neonatology open by registration to all interested health professionals, facilitating communication both within VON and between VON and other interested professionals and groups.