Legislators tee up renewed effort to pass rare pediatric drug bills
Life Sciences
| By Alexander Gaffney, MS, RAC, Laura DiAngelo, MPH
Several reforms that nearly passed at the end of last year have been re-introduced as new legislation in the House of Representatives. The bills would impose new pediatric cancer testing requirements on combination therapies, penalize sponsors failing to complete required pediatric studies, renew a priority review voucher program for rare pediatric diseases, close an exclusivity loophole for rare pediatric disease drugs, require FDA to open an office in the Middle East, and more.
Background and context
- Toward the end of last year, a bipartisan group of legislators attached a package of health care bills to an appropriations bill intended to fund the government through March 14, 2025. Though in Washington, it’s common to add legislative “riders” to must-pass legislation, the move attracted scrutiny from legislators and some observers.
- Among other provisions, the 1,600-page bill included four pieces of legislation addressing pediatric conditions and diseases, with a particular focus on pediatric cancer.
- One bill was called the “Give Kids a Chance Act.” As AgencyIQ discussed back in September 2024, the long-debated Give Kids a Chance Act (GKAC) was recently modified to overhaul requirements to test certain combination oncology products in pediatric populations and impose new pediatric compliance requirements. Among other requirements, the bill would require sponsors of molecularly targeted drugs that work in combination with other drugs to assess whether those combinations might also work in pediatric patients. The primary updates to the latest version of the bill in December relate to a provision focused on already completed preclinical studies. The version in September simply stated that sponsors could be required to provide “the results of all preclinical studies on which the decision to conduct such investigation was based.” However, the newer version in the CR is more granular, stating that “the Secretary may require the results of any completed preclinical studies relevant to the initial pediatric study plan be submitted to the Secretary at the same time that the initial pediatric study plan required… is submitted.”
- A second bill, the Innovation in Pediatric Drugs Act, focused on the process for noncompliance letters under 21 U.S.C. 355c(d). For sponsors failing to conduct pediatric studies as required and/or to submit a request for approval of a pediatric formation before additional enforcement actions can occur, this provision offers a buffer. Under the bill, the noncompliance letter provisions would be stricken, meaning sponsors who fail to meet pediatric study requirements could also be subject to the penalties listed in 21 U.S.C. 333, including civil monetary fines as soon as 180 days of the bill’s passage. However, the language newly clarifies that such enforcement actions shall only be taken if the FDA—upon reviewing the written response to its noncompliance letter—determines that the sponsor has “demonstrated a lack of due diligence in satisfying the applicable requirement.” In addition to enforcement action against sponsors that fail to fulfill PREA study requirements, the bill called for the FDA to inform Congress of these transgressions by including a “listing of penalties, settlements, or payments” in the reports required to be submitted by the FDA to Congress every five years. Furthermore, each entry within the listing should include the “name of the drug, the sponsor thereof, and the amount of the penalty, settlement, or payment imposed.”
- A third bill was called the Creating Hope Reauthorization Act and would have extended FDA’s authority to issue priority review vouchers to encourage treatments for rare pediatric diseases. The bill would have re-authorized the Rare Pediatric Disease Priority Review Voucher (PRV) program until Sept. 30, 2029. The bill also clarified that “the priority review user fee required by this subsection shall be due upon the submission [of the application] … for which the priority review voucher is used. All other user fees associated with the human drug application shall be due as required by the Secretary or under applicable law.” In addition, the bill directed the Government Accountability Office to develop a report on “the effectiveness” of the program, including the types of products on which vouchers are granted, their scope and impact on unmet need, the “value” of the voucher “if transferred” and the size of the companies that use the vouchers. This report is due in five years.
- A fourth bill, the Retaining Access and Restoring Exclusivity (RARE) Act, focused on marketing exclusivity periods provided to approved drugs that had been designated under FDA’s Orphan Drug Designation program. The bill clarified that products awarded orphan drug designation by the FDA are granted seven years of marketing exclusivity only for the specific indication they are approved to treat. This provision is meant to address an issue that was exposed after the FDA’s 2021 loss in a high-profile court case, Catalyst v. Becerra, which interpreted the Orphan Drug statute differently than FDA’s long-held position. The bill would have reinstated FDA’s interpretation of the statute, meaning that FDA could approve other products that treat the same rare disease or condition as an orphan-designated drug product as long as the approvals for the subsequent products are for different indications than the original product.
- A fifth bill sought to establish an office “known as the Abraham Accords Office” at the FDA within two years of the bill’s passage. As AgencyIQ has previously explained, the Abraham Accords are a series of U.S.-brokered bilateral agreements between Israel and Bahrain, Morocco, Sudan, and the United Arab Emirates. The office, based in an Abraham Accords signatory country, would work to “strengthen the international oversight of regulated commodities, provide technical assistance to regulatory partners in Abraham Accords countries on strengthening regulatory oversight and converging regulatory requirements for the oversight of regulated products… and facilitate interactions between the [FDA] and interested parties in Abraham Accords countries.”
- Ultimately, the appropriations bill – also known as a Continuing Resolution – was streamlined to ensure it was able to pass Congress in time. Because the appropriations legislation, introduced on Dec. 17, 2024, needed to be passed by midnight on Dec. 20, 2024 to avoid a government shutdown, unanimous consent was needed in the Senate to end debate on the measure and proceed to a vote. Sen. RAND PAUL (R-Ky.) refused to provide that consent unless the bill was stripped of most non-budgetary provisions. As a result, the four bills mentioned above were removed.
Now the bills have been reintroduced in a new, combined piece of legislation known as the Give Kids a Chance Act of 2025
- Unlike prior versions of the bill, this version includes all of the previous legislation under a combined header. It also includes a provision authorizing another $25 million to fund pediatric testing in fiscal years 2025, 2026 and 2027, as well as another provision reforming the Organ Procurement and Transplantation Network. The combinations may be intended to make the bill easier to pass, since opposing a bill called the “Give Kids a Chance Act” doesn’t make for especially good headlines.
- Like the original version of GKAC, the new bill has plenty of bipartisan cosponsors already, including its sponsor Rep. MICHAEL MCCAUL (R-Texas) and Representatives GUS BILIRAKIS (R-Fla.), DEBBIE DINGELL (D-Mich.), KIM SCHRIER (D-Wash.), DIANA HARSHBARGER (R-Tenn.), KATHY CASTOR (D-Fla.), MIKE KELLY (R-Pa.), DORIS MATSUI (D-Calif.), DAN CRENSHAW (R-Texas), LORI TRAHAN (D-Mass.) and RANDY WEBER (R-Texas). In this congress, a bill attracting this many bipartisan sponsors is an exceptionally rare thing.
- The next steps for this legislation remain uncertain for now. Legislators have a few different options they could pursue, including trying to get the legislation attached to a larger health care package of health care, getting it passed as standalone legislation on a bipartisan basis, or trying to ride along with the upcoming budget reconciliation legislation slated for passage in March 2025. However, the bill’s authorization for the National Institutes of Health to fund pediatric research ($25 million for each of three fiscal years) might make it complicated to pass as part of the budget reconciliation bill, which is targeting substantial spending cuts. It might similarly be difficult to ask legislators to cut the FDA’s funding as part of the reconciliation bill while also asking the FDA to establish a new foreign office and make it operational with no additional funding provided.
Featuring prior research from Laura DiAngelo and Rachel Coe.
To contact the author of this analysis, please email Alexander Gaffney ( agaffney@agencyiq.com)
To contact the editor of this analysis, please email Kari Oakes ( koakes@agencyiq.com)