Chronic conditions – like neurofibromatosis – by definition, last a lifetime. Tracking the natural history of the condition (in other words, how it progresses during life) in as many patients as possible, and comparing findings between patients, provides important information that can be used to figure out which interventions and treatments are working best across patients. This type of information is collected in a patient registry – an electronic log in which doctors can enter information about an individual’s health status. Data entered regularly over a patient’s lifetime adds to a collective goldmine of knowledge that could help shape future clinical approaches.
An article* in today’s New York Times highlights the fact that a patient registry can not only change the landscape of how a disease is understood and managed, but can vastly improve and even prolong individual lives. The Cystic Fibrosis Foundation (CFF) has blazed a trail in maintaining a registry that now collects patient information from 100 cystic fibrosis clinical centers around the country. In 50 years, cystic fibrosis has moved from being a condition of early childhood death, to one of adults living lives of dramatically improved quality. Much of this progress can be attributed to the CFF patient registry via its comparing cystic fibrosis clinical management approaches and sharing these with the cystic fibrosis clinical management community. This registry has also fuelled CFFs efforts in ‘driving the bus’ for cystic fibrosis clinical trials, an area CFF activated alone long before the pharmaceutical and biotechnology became interested in rare diseases. Patient registries are now being developed by many other Foundations, professional societies and even the National Institutes of Health.
Building on our NF Clinic Network established in 2007 and now comprising 40+ clinics across the United States, the Children’s Tumor Foundation is currently planning the implementation of both an NF patient registry and NF tissue biobank which we aim to launch in 2010. In January, the Children’s Tumor Foundation will join with a number of other Foundations in an NIH-led meeting to plan for a collaborative patient registry that includes multiple diseases. This important step could make a patient registry possible even for extremely rare diseases where resources to do so are limited.
* Tool In Cystic Fibrosis Fight: A Registry. New York Times, December 22.
