development research on a specific rare disease (see listings at

Competing companies may also combine insights and work together to solve a particular regulatory problem. A recent example is from the Critical Path Institute (C-Path), an independent, nonprofit organization that brings together FDA, pharmaceutical companies, and others to focus on drug development issues and to support FDA’s Critical Path Initiative (see Chapter 3). C-Path’s Predictive Safety Testing Consortium involves 16 companies. Recently, as a result of joint efforts across these companies, FDA and its European counterpart, the European Medicines Agency, have both agreed to a new standard for preclinical testing of drugs entering development to predict renal toxicity (C-Path, 2009b). The consortium is now working to qualify and validate new biomarkers in other areas. C-Path was also instrumental in developing the Coalition Against Major Diseases.

Advocacy Groups

Given the challenges and expense that beset the traditional model of pharmaceutical research and development in bringing new drugs to market, developing treatments for rare diseases represents an opportunity to test new paradigms. Led by patients and families, disease-specific foundations have begun to do just that (IOM, 2008). As described in Chapter 4, the strategy includes “de-risking” early-stage research and development for promising products by providing philanthropic capital as well as research tools and access to patients.

Although advocacy groups have traditionally provided support for basic discovery research, many of them have recently assumed a more active role in shepherding the drug development process in their areas of focus. Again, one objective is to minimize the risks associated with the early phases of therapeutic development. For example, building on the promising results of its basic research program, the Muscular Dystrophy Association now supports the preclinical work necessary for an IND application, as well as funding a national patient database, early clinical trials, and associated research infrastructure costs (see information at Appendix F includes other examples. These novel approaches have begun to bear fruit, increasing the number of promising therapies that proceed to later-stage clinical trials (IOM, 2008).

Another Model: Public-Private Partnerships for Neglected Diseases

Public-private partnerships have played an important role in advancing therapeutics for neglected diseases of the developing world. For example, as mentioned in Chapter 4, the Medicines for Malaria Venture is working with

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