Reauthorization of the pediatric voucher program
On December 27, 2020 Congress passed the Creating Hope Reauthorization Act to extend the pediatric voucher program. Under to this new reauthorization, after September 30, 2024 FDA may only award vouchers for approved rare pediatric disease product applications if a company has a rare pediatric disease designation for the drug granted by the FDA by September 30, 2024.
Pursuant to the Creating Hope Act, a company that develops a drug for a pediatric rare disease – and receives FDA approval – also receives a voucher. The voucher comes with rights to faster FDA review of any future drug, enabling the voucher holder to receive an FDA “priority review” instead of a “standard review.” The voucher is transferable.
Pediatric vouchers have been sold for as much as $350 million and are now trading at $100 million. Over $2 billion of vouchers have been traded. Since the enactment of the Creating Hope Act, the FDA has approved 34 drugs expressly for pediatric rare diseases, two of which are pediatric cancer drugs.
Nancy Goodman, CEO of Kids v Cancer, stated: “I thank Representative G.K. Butterfield and Representative Michael McCaul, Senator Michael Bennet and Senator Susan Collins for their dedication to children with cancer and their commitment to shepherding the Creating Hope Reauthorization Act through Congress.
How a company uses the pediatric voucher program
The FDA guidance on vouchers is here:
— Applicant company obtains an orphan designation for an indication (often phase 1 or even preclinical)
— With the orphan designation, applicant company applies for a rare pediatric disease designation (phase 1 or preclinical).
—- The definition of “pediatric” is usually the sticking point. A rare pediatric disease is one that is serious or life-threatening in which the serious or life-threatening manifestations primarily affect patients from birth to 18 years, including neonates, infants, children, and adolescents.
—- A designation from the FDA is conditional and signals that at the time of the issuance of the designation, the indication for which the drug is being developed meets all of the 507 eligibility criteria set forth in section 529(a)(4). The final answer to a conditional 508 designation of an application comes in the form of a voucher award or non-award at 509 the time of marketing approval, if the applicant company requests such a voucher in the NDA/BLA.
— FDA grants applicant company a rare pediatric disease designation.
— If the applicant company (now named the sponsor) receives an FDA approval for a drug or biologic under this program, they can redeem the voucher for priority review of a future marketing application, or they can sell the voucher to another sponsor.