July 29, 2019 | By Zachary Brennan | Regulatory Focus

The US Food and Drug Administration (FDA) on Monday released revised draft guidance on rare pediatric disease priority review vouchers (PRVs), which are awarded to companies developing certain rare pediatric disease treatments and can then be sold.

From 2014 to today, FDA has issued 20 rare pediatric disease PRVs, with one selling for $350 million to AbbVie in 2015 and more recently selling for between $80 million and $130 million.

The revisions to FDA’s guidance include a new definition for rare pediatric disease, as created by the Advancing Hope Act of 2016, which amends section 529 of the FD&C Act to define the pediatric population as from birth through 18 years. FDA previously considered the pediatric population as from birth to 16 years.

The agency also spells out how it interprets the definition of “rare pediatric disease,” noting that a manifestation of the disease or condition should be serious or life-threatening in children under the age of 18 and it must be a disease or condition that affects less than 200,000 persons in the US.

“For example, if a disease or condition’s onset typically begins in childhood, but manifestations of the disease or condition do not become serious or life-threatening until adulthood, the disease or condition is not a rare pediatric disease. Similarly, if treatment for the disease or condition begins in childhood, but under current standard of care the manifestations of the disease or condition are not serious or life-threatening in children, the disease or condition is not a rare pediatric disease,” the revised draft says.

FDA also explains how the rare pediatric disease PRV program sunsets. After 30 September 2020, FDA may only award a voucher if the drug has rare pediatric disease designation and that designation was granted by 30 September 2020, the draft adds.

FDA also includes revisions in the draft based on FDA’s experience with implementing the rare pediatric disease PRV program, including voucher request procedures.

The revisions also include a further explanation of the rare pediatric disease PRV eligibility requirements, the rare pediatric disease designation process and examples to illustrate the agency’s current thinking on these review determinations.

Source: Rare Pediatric Disease Priority Review Vouchers: Guidance for Industry (FDA)