Meeting

Developing a new drug or biologic product has become exceedingly expensive; the average cost is estimated to be nearly 1.3 billion. While pharmaceutical and biotechnology companies may be willing to foot such a bill to develop potential blockbuster products, these high costs make the development of drugs for rare and neglected diseases far less commercially attractive. Disease-specific foundations and other philanthropic organizations have developed a range of financial and operational strategies to encourage the development of such drugs. One important strategy is to "de-risk" investments in drug candidates for industry and venture capitalists by filling critical funding gaps along the drug development pathway and by pursuing a highly targeted approach to early phase development. In addition to providing financial capital, these organizations also contribute important intellectual capital to the enterprise. These strategies involve a number of different elements, including: intellectual property agreements, data sharing, and strategies for facilitating clinical trials. These elements were discussed in depth during the workshop, and successful strategies were highlighted. The discussion considered the factors that contribute to successful approaches, the feasibility of adopting these approaches by other organizations and in other disease areas, and the potential opportunity for accelerating research that these approaches represent for rare and neglected diseases as a whole. In addition, these discussions provided opportunities for organizations that are interested in successful mechanisms to share ideas and identify potential collaborative activities. As personalized medicine advances, these strategies may offer important insights for the development of individualized therapies, and for the pharmaceutical industry as a whole.

Objectives:

The broad objectives of this one day workshop were to:

• Outline changes in the translational research (discovery through phase II) process that have taken place over the past ten years and why.
• Discuss new models for funding translational research and new technologies and consider their impact on the process.
• Examine regulatory, legislative, and institutional policy tools currently in place to help advance therapeutic development for rare or neglected diseases, and individualized therapies, and discuss whether these tools are adequate or whether new ones are needed in light of these new funding models.