advocates are included in all steps of the design so that processes such as informed consent, cohort accrual, and participant retention are simplified and improved.

The primary interest of Genetic Alliance constituents is to ensure that the experimental treatments used in the clinical trials are effective. Constituents are willing to assume additional safety risks if a treatment is effective—an approach to drug development that reflects the unique needs of people living with a rare disease. Ultimately, a new, flexible paradigm is needed that:

  • is forward looking and takes into account the rapid changes in research and industry;

  • is well coordinated across federal agencies and companies;

  • has safe harbors for high risk, encourages publishing of negative results, and allows companies to take risks without fear of being penalized as they might be under the current regulatory regime;

  • is transparent and open; and

  • is characterized by more common registries and more shared data.

In addition, intellectual property issues must be addressed to enable win–win situations. And sharing failures is critical to save others from wasting resources. In conclusion, Terry stressed the need for bold leadership to dissolve old boundaries and accelerate the adoption of this new paradigm.



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