The North American Mitochondrial Disease Consortium: A Developing Knowledge Commons
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Description
Rare diseases number between 5000 and 8000. Each affects fewer than 200,000 individuals, but in the aggregate, they affect millions. As summarized in a National Academies Report, Rare Diseases and Orphan Products: Accelerating Research and Development: “Because the number of people affected with any particular rare disease is relatively small and the number of rare diseases is so large, a host of challenges complicates the development of safe and effective drugs, biologics, and medical devices to prevent, diagnose, treat, or cure these conditions. These challenges include difficulties in attracting public and private funding for research and development, recruiting sufficient numbers of research participants for clinical studies, appropriately using clinical research designs for small populations, and securing adequate expertise at the government agencies that review rare diseases research applications or authorize the marketing of products for rare conditions.” Information sharing, collaboration, and community building among researchers, doctors, and patients are critical to rare disease research. The Rare Disease Clinical Research Network (RDCRN) is an NIH program aimed at developing infrastructure and methodologies for rare disease clinical research by creating a network of research consortia. Each RDCRN consortium (RDCRC) involves researchers, other health care professionals, and patients at a group of geographically dispersed clinical sites. Medical knowledge is a nonrivalrous resource, which can be used to treat any number of patients without diminishing its value to others. RDCRCs nonetheless face resource governance challenges, including (1) managing rivalrous inputs, such as research funding and researcher time; (2) managing rivalrous incentives and rewards, such as authorship credit; (3) overcoming incentives to hoard scarce access to patients and their data; (4) reducing the transaction costs of cooperation between widely dispersed researchers; and (5) managing interactions with outsiders, such as pharmaceutical companies. All scientific research confronts tensions between the need to apportion scarce, rivalrous resources and the value of sharing nonrivalrous research results and certain infrastructural data and tools broadly. Mechanisms for managing this tension include public funding, reputation-based systems of peer review and publication, and scientific community norms. In clinical research, additional tensions between the value of the research and potential risks to research subjects are addressed by informed consent regulation, professional ethics prioritizing duty to patients, and institutional review boards (IRBs). These form part of the backdrop for the RDCRN and its associated consortia. We previously studied the Urea Cycle Disorder Consortium (UCDC). Here we focus on the North American Mitochondrial Disease Consortium (NAMDC). The next chapter in this book studies the Consortium for Eosinophilic Gastrointestinal Disease Research (CEGIR). While there are many similarities between these consortia, which face common rare disease research problems and are structured by the RDCRN, there are also significant differences in the underlying challenges the groups face and the approaches they take to those challenges. The UCDC is much better established than the NAMDC and emerged from a history of greater previous cooperation. CEGIR is very new, but, like the UCDC, emerged from a close-knit group of researchers. Mitochondrial disorders are complex, varied, and difficult to diagnose, much less treat. The diseases studied by UCDC and CEGIR are comparatively well understood, with relatively well-accepted diagnostic criteria and targets for treatments. The consortia also have leaders with different styles and personalities and different governance structures.
Source Publication
Governing Medical Knowledge Commons
Source Editors/Authors
Brett M. Frischmann, Michael J. Madison, Katherine J. Strandburg
Publication Date
2017
Recommended Citation
Strandburg, Katherine J. and Frischmann, Brett M., "The North American Mitochondrial Disease Consortium: A Developing Knowledge Commons" (2017). Faculty Chapters. 1671.
https://gretchen.law.nyu.edu/fac-chapt/1671
