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Opinion: STAT+: BridgeBio CEO: Congress must renew a popular, bipartisan FDA program benefiting sick kids
In Washington, even widely hailed programs with bipartisan support — like the rare pediatric disease priority review voucher program — are falling by the wayside, writes BridgeBio’s Neil Kumar.
Thousands of children’s lives have been saved by a cost-free government initiative that expired last December. Congress needs to renew this program quickly because too many babies are dying needlessly.
Housed at the Food and Drug Administration and fully funded by pharmaceutical companies at no cost to taxpayers, the rare pediatric disease priority review voucher program offers a carrot to companies developing treatments for children with rare and life-threatening diseases. Since so few kids suffer from any single illness, these drugs rarely make enough money to fund the incredibly expensive research underpinning them. So instead of expensive taxpayer-funded grants or subsidies, the government has another way to make up the shortfall. Companies that successfully bring a treatment for a rare pediatric disease to market get a voucher for a speedy FDA review that can be used for a different, unrelated drug. These vouchers can then be sold to other companies.
Since some medicines earn billions of dollars annually, these certificates can be enormously valuable to the giant pharmaceutical companies that develop blockbuster drugs to treat adult-onset illnesses. A priority review voucher recently sold for $150 million — enough to fund years of research. The vouchers are a kind of Robin Hood program in which huge conglomerates give money to tiny, cash-strapped biotechnology firms to save the world’s most vulnerable patients.
