By Alexander J. Varond –
Late last week, the Senate voted unanimously to extend the rare pediatric disease voucher program until December 31, 2016. In doing so, it also voted to amend the definition of “rare pediatric disease” set forth in Section 529 of the Federal Food, Drug, and Cosmetic Act. As discussed in greater detail below, the new definition of “rare pediatric disease” would likely expand eligibility for the program.
The version of the Advancing Hope Act (S. 1878) passed by the Senate last week closely tracks earlier versions of the bill. However, instead of reauthorizing the program until at least September 30, 2022, the current version only extends the pediatric voucher program by three months. It is clearly a stopgap measure.
In keeping with earlier versions of the bill, the Advancing Hope Act would amend the current definition of “rare pediatric disease,” which requires that:
(A) The disease primarily affects individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents. (Emphasis added.)
S. 1878 amends the definition to require that:
(A) The disease is a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents. (Emphasis added.)
We discussed an earlier version of the Advancing Hope Act and several other reauthorization bills last week.
The newly proposed definition of “rare pediatric disease” places greater emphasis on disease burden, rather than on disease prevalence. Indeed, many stakeholders had expressed concern that the current definition’s emphasis on prevalence was misguided. These concerns are easily understood when considering, for example, diseases with varying forms of severity. If patients with the most severe forms of the disease die while they are young but patients with less severe forms live long lives, the disease might not be eligible under the current definition.
The Advancing Hope Act has been referred to the House and efforts are underway to seek a vote on the measure this week. If the Advancing Hope Act passes the House and is signed into law, it would likely expand eligibility for pediatric vouchers. For example, diseases with varying degrees of severity (i.e., diseases with very serious forms with very short life expectancy and less serious forms with much longer life expectancy) would be eligible. In addition, diseases that are extremely severe in childhood but tend to be less severe in adulthood may qualify.
FDA’s interpretation the revised definition of “rare pediatric disease” will be critical. One of FDA’s first tasks would be to provide clarity around what constitutes “serious or life-threatening manifestations.” Focusing on “manifestations” of a disease is somewhat novel, compared to FDA’s typical paradigm, which focuses on whether the disease itself is “serious or life-threatening.”