emphasis on real-time sharing of scientific discoveries among experts; and partnerships with industry. Another often-cited group, FasterCures, seeks to encourage innovation and efficiency in medical research generally and to promote the diffusion of successful strategies (http://www.fastercures.org/index.cfm/OurPrograms/Overview). Among rare diseases organizations, the Cystic Fibrosis Foundation has, in many respects, led the way in developing and implementing a systematic research strategy that is tailored to evolving research progress and scientific and technological opportunities.
Two umbrella organizations, the National Organization for Rare Disorders and the Genetic Alliance, provide assistance to organizations trying to develop and implement research strategies. Advocacy groups with a focus on rare diseases research can also benefit from various initiatives of the Office of Rare Diseases Research at the National Institutes of Health (NIH) and the Office of Orphan Product Development at the Food and Drug Administration.
Rare diseases advocacy organizations that support research and development vary in their approaches and emphases. Their research objectives and the strategies for attaining them may be influenced by a number of factors, including their financial resources, the existence of effective treatments, and the experiences, priorities, and expertise of the group’s founders. Other factors that may shape a group’s research directions include whether or not a disease’s cause is known; how well the disease process is understood; what research, if any, is being undertaken by other public, nonprofit, or commercial entities and what niche the advocacy organization is best equipped to fill; whether it is critical to recruit new scientists into the research area; and how challenging it is to recruit patients and families to participate in research.
Organizational strategies may be highly focused on one segment along the spectrum from basic to clinical research or they may span the spectrum. As research progresses, strategies may shift from an emphasis on identifying the cause and genetic and molecular basis of a disease to identifying and testing promising therapies and securing FDA approval.
Groups vary significantly in their resources. For the organizations used as illustrative examples in this appendix, Table F-1 shows major differences. The years and definitions may not be completely consistent for the figures cited in the table, but they give a sense of the substantial range in organizational resources and the concentration of organizations in the lower end of the range. (Two groups with spending higher than that of most of the groups in the table are the Multiple Myeloma Research Foundation at around $13 million and MDA, formerly the Muscular Dystrophy Association, at around $39 million for 2009 according to their 2009 annual reports.)
The examples below illustrate different components of research strate-