Breakthrough Business Models Drug Development for Rare and Neglected Diseases and Individualized Therapies: Workshop Summary (2009) / Chapter Skim
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3 The Food and Drug Administration's Orphan Drug Program
3 The Food and Drug Administration's Orphan Drug Program
Pages 12-18
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From page 12... ...
Academic laboratories would occasionally discover promising new therapies, but without the capital required to conduct the clinical trials necessary for FDA approval and without the interest of a pharmaceutical company in bringing these compounds to market, these potential new products remained undeveloped. While the science of drug discovery progressed rapidly during this period, yielding powerful insights into human biology and the pathologic processes of diseases, rarely were these insights applied to research on rare diseases.
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From page 13... ...
The establishment of the National Institutes of Health's Office of Rare Diseases is a prime example of this. Congress also established the Orphan Products Grant Program at FDA, administered by OOPD, which Coté believes "has become the single most tangibly productive grants program in the entire U.S.
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From page 14... ...
FIGURE 3-1 The number of products that received orphan designation and the number of new drugs approved from 1983 to 2005. Note that the numbers on the charts do not match the numbers in the text because the charts show data only through 2005, while Dr.
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From page 15... ...
The Orphan Drug Act is working well through the core activities of OOPD -- making orphan designations, awarding grants, providing advocacy, and shepherding products through the FDA approval process. OOPD connects sponsors with the relevant review
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From page 16... ...
As noted earlier, 41 drug approvals came out of OOPD's grants program, a clear demonstration of its significant success. Yet the total budget for the program has remained essentially flat when inflation and the increasing costs of conducting clinical trials are taken into account, and the program is currently funded at only $14 million.
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From page 17... ...
This entity would be housed within an existing nongovernmental organization and created fairly rapidly, perhaps through a workshop involving no more than 12 professionals who would generate 40–50 orphan designation applications over the course of 1 week. Drug candidates entered into the orphanage would already be eligible for the priority review voucher and have both FDA and European Medicines Agency (EMEA)
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From page 18... ...
The processes are still independent, but the agencies hold monthly teleconferences and are reviewing many of the same applications. There is also an exchange program whereby people from the European orphan drug office and OOPD meet to learn how each operates.
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