Opinion: The rare pediatric disease voucher program creates new treatments. I have new data to prove it
The Food and Drug Administration’s rare pediatric disease priority review voucher program, which has been providing incentives for lifesaving innovations since 2012, is doomed to disappear unless Congress reauthorizes it before the end of September. Some lawmakers have downplayed its impressive track record based on unfounded allegations. I have data showing it works.
Tracy Dixon-Salazar — whose two-year-old daughter, Savannah, started having hundreds of seizures a day in 1995 before being diagnosed with Lennox-Gastaut syndrome at age 5 — has seen the impact of these vouchers. With no FDA-approved treatment effective for Savannah’s condition, the family tried nearly 30 off-label medications in their search for relief. None worked.
