Progress on the action plan for liver disease research
Authors: Jay H. Hoofnagle, Megan E. Miller
Journal: Hepatology (2006)

The Trans-NIH Action Plan for Liver Disease Research was completed and made available in December 2004, first on the NIH website and as a monograph shortly thereafter. The Action Plan was developed by a broad consortium of basic and clinical investigators involved in liver disease research, with input from staff of the NIH, other Federal agencies, industry, health care providers, and concerned lay persons. The purpose of the Action Plan is to advance research on liver and biliary diseases, with the ultimate aim of decreasing the burden of these diseases in the United States. The Action Plan provides a brief introduction to liver disease morbidity and mortality and current NIH funding. The core of the Action Plan is a list of 214 specific research goals for the next decade, including their background and justification. The research goals are organized into 16 topic areas, and each research goal is categorized by degree of difficulty (low, medium or high risk) and estimated time for completion (short, medium or long-term). The Action Plan is an ambitious and optimistic document that provides a structure and focus for research on liver and biliary diseases, which together rank among the top 10 causes of death in the United States. The Action Plan also includes plans for implementation. Attainment of its research goals will be promoted through: (1) broad distribution of the document, (2) encouragement of its use in grant applications and in peer review, (3) promotion of collaborations between research funding entities (including industry), and (4) specific initiatives from the NIH and other Federal agencies concerned with liver disease research, such as the Food and Drug Administration, Centers for Disease Control and Prevention, and Department of Veterans Affairs. As part of implementation, the Liver Disease Research Branch of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), NIH led the preparation of a Progress Review for 2005, the first year after release of the Action Plan. The Progress Review was prepared with help from members of the 16 Working Groups who initially provided input into the research goals, as well as members of the Liver Disease Subcommittee of the Digestive Diseases Interagency Coordinating Committee that oversees the Action Plan. The 2005 Progress Review is now available on the NIH website (http://liverplan.niddk.nih.gov). The Progress Review provides a concise analysis of progress made toward each of the 214 goals and, when appropriate, a brief description of initiatives focusing on that goal. The Progress Review is not comprehensive, but does provide specific examples of important advances made in 2005 that are apropos to research goals. Finally, the degree of progress made toward each specific research goal is estimated on a scale of 0 percent (little or no progress) to 100 percent (full attainment of the goal) in increments of 10 percent. These estimates are based on general agreement among members of the 16 Working Groups. The Action Plan also features a section on Summary Recommendations, including a series of 10 overarching "benchmark" research goals. These 10 research goals are important, cross-cutting goals that were mentioned in several of the 16 topic areas and would constitute clear advances in the management, control, and prevention of liver diseases. These benchmarks are to be used to assess the overall success of the Action Plan. A discussion of progress toward these benchmark goals concludes the Progress Review. The 2005 Progress Review reports that material advances have been made toward 116 of the 214 research goals. None of the goals was achieved entirely during this first year, but several are now approaching completion. Notable examples of progress in 2005 include: (1) development of a fully permissive cell culture system for hepatitis C virus replication, (2) further definition of pathways and regulation of hepatic cholesterol synthesis and secretion, (3) further clarification of normal molecular pathways of iron metabolism in humans, and (4) development of an animal model for primary sclerosing cholangitis. Among the 10 benchmark goals, perhaps the most progress has been made in developing more effective means of long-term management of chronic hepatitis B. The implementation of the Action Plan calls for a yearly review of progress toward research goals and a major assessment after five and ten years. Comments about the 2005 Progress Review are encouraged and can be submitted through the website. Input on progress made so far in 2006 is also welcome. The NIH joins with the research community in its commitment to attaining all of the research goals of the Trans-NIH Action Plan for Liver Disease Research.