OCE Regulatory Programs 2024

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Project Orbis
Pediatric Oncology Program
Rare Cancers Program
Oncology Labeling Program
Oncology Safety
Project Renewal
Project Confirm

Project Orbis

In its fifth year, Project Orbis collaborated with international regulatory partners on 23 product approvals that included 5 new products and 18 indication extensions. OCE strengthened its partnerships with key agencies, particularly the European Medicines Agency (EMA) and Japan's Pharmaceuticals and Medical Devices Agency (PMDA), advancing programs to support oncology drug development.

Our international engagement expanded through hosting and traveling for strategic meetings. At the White Oak campus, we welcomed various international delegations, including a multi-stakeholder group from Sweden and representatives from Israel's Ministry of Health (IMoH), Japan's PMDA, and Norway's Norwegian Medical Products Agency (NOMA).

An OCE delegation conducted collaboration visits in late 2024, first traveling to Health Canada (HC) in Ottawa, Ontario, in September. This was followed in November by meetings with European regulators including Belgium's Federal Agency for Medicines and Health Products (FAMHP), the UK's Medicines and Healthcare products Regulatory Agency (MHRA), and Switzerland's Swissmedic. These engagements focused on maintaining current initiatives and exploring new opportunities for regulatory cooperation in oncology.

OCE remains committed to fostering these regulatory partnerships, recognizing their crucial role in improving outcomes for cancer patients worldwide.

Download the full list of Project Orbis Approvals (as of March 2025). Visit Oncology (Cancer)/Hematologic Malignancies Approval Notifications for further information on these approvals.

Pediatric Oncology Program

In 2024, the OCE Pediatric Oncology Program continued its mission to advance development of new safe and effective drugs and biologics to treat cancers in infants, children and adolescents. By leveraging the authority under the Pediatric Research Equity Act (PREA) to require pediatric investigations for certain new targeted cancer therapies, and the Best Pharmaceuticals for Children Act (BPCA) to encourage voluntary conduct of pediatric clinical trials, this program promotes timely evaluation and clinical development of drugs and biological products that have the potential to improve outcomes of pediatric patients with cancer.

In 2024, there were 10 approvals of 8 different drugs and biologics to treat pediatric patients with cancer. Notable approvals include:

Tovorafenib for patients 6 months of age and older with relapsed/refractory pediatric low-grade glioma harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.

Blinatumomab for adult and pediatric patients one month of age and older with CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia in the consolidation phase of multiphase chemotherapy.

Vorasidenib for adult and pediatric patients 12 years of age and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible dehydrogenase-1 (IDH1) or isocitrate dehydrogenase-2 (IDH2) mutation following surgery.

Revumenib for adult and pediatric patients 1 year of age and older with relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation.

Visit OCE Pediatric Oncology Drug Approvals for more information.

Initial Pediatric Study Plans (iPSPs) and Written Requests 2024
Type F Meetings to discuss iPSPs prior to submission	5
iPSPs reviewed	124
Agreed iPSPs issued	93
Amended Agreed iPSPs issued	9
Proposed Pediatric Study Requests reviewed	16
Written Requests issued	2

In addition, the program continues to engage and collaborate with scientists, patient advocates, and international regulatory authorities to promote timely and efficient international pediatric cancer drug development. A few notable meetings convened in 2024 include:

May 22, 2024: Meeting of the Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee (ODAC) to discuss perspectives relating to implementation of amendments made by the FDA Reauthorization Act of 2017 (FDARA) to section 505B of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (also referred to as the Pediatric Research Equity Act, or PREA) and its impact on pediatric cancer drug development to date.
October 15, 2024: FDA Oncology Center of Excellence Pediatric Advocacy Forum to promote informal dialogue between FDA staff involved in overseeing development of new products to treat pediatric cancers and the patient advocacy community in order to foster understanding, awareness, and future opportunities for collaboration.
November 19, 2024: In conjunction with the European Medicines Agency (EMA), Conversations on Cancer - Pediatric Cancers: Navigating the Challenges Together, to examine an array of challenging decisions faced by members of the pediatric oncology community.

Rare Cancers Program

Launched in 2021, the Oncology Center of Excellence (OCE) Rare Cancers Program’s mission is to leverage OCE’s ongoing initiatives to promote development of safe and effective new drugs and biologics to treat patients with rare cancers.

Outreach activities in 2024 included participation in FDA Rare Disease Day, European Medicines Agency (EMA)/European Organisation of Research and Treatment of Cancer (EORTC) Soft Tissue and Bone Sarcoma workshops, a meeting related to rare cancer product development held by the National Academies of Sciences, Engineering and Medicine (NASEM), and the National Cancer Institute (NCI) Rare Cancers Prevention and Interception Virtual Workshop.

Alongside OCE’s Project Catalyst, the OCE Rare Cancers Program continues to work with the Foundation for the National Institutes of Health (FNIH), the National Cancer Institute (NCI) and the National Center for Advancing Translational Sciences (NCATS) on a public-private consortium to accelerate development of new safe and effective treatments for patients with ultra rare cancers. The program also supports OCE-Funded Extramural Research on rare cancers and the CURE ID project focus area aimed at capturing the real-world experience of patients with rare cancers.

Some notable 2024 approvals of drugs or biologics for rare cancers include:

Tovorafenib for patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.
Vorasidenib for adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, following surgery including biopsy, sub-total resection, or gross total resection.
Zanidatamab-hrii for adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test.
Zenocutuzumab-zbco for adults with advanced, unresectable, or metastatic pancreatic adenocarcinoma harboring a NRG1 gene fusion with disease progression on or after prior systemic therapy.
Afamitresgene autoleucel for adults with unresectable or metastatic synovial sarcoma who have received prior therapy, are HLA-A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive and whose tumor expresses the MAGE-A4 antigen as determined by FDA-approved or cleared companion diagnostic devices.

OCE Labeling Program

Regulatory Guidance Development

Led finalization of FDA draft guidance on QTc information in prescription drug and biologic labeling, following the public comment period.
Contributed to FDA Draft Guidance on Drug Interaction Information for Human Prescription Drug and Biological Products, as part of cross-center team.
Advanced development of three priority FDA draft labeling guidance documents for 2025 publication covering clinical pharmacogenomics, organ impairment, and biosimilar product labeling.

Clinical and Safety Initiatives

Successfully negotiated updates to NCI CTCAE v6.0 for diabetes and hyperglycemia adverse event grading to enhance clinical trial and labeling accuracy.
Coordinated cross-FDA Center initiatives to standardize labeling policies across multiple areas, including generic drugs with diagnostic devices, anti-PD1/L1 updates, and adverse reaction reporting.

Stakeholder Engagement

Enhanced oncology labeling awareness through presentations at the Global Labeling and Regulatory Symposium, Oncology Nursing Society podcast, and University of Maryland Pharmacy School.

Oncology Safety Program/Project Protect

Drug Safety Review

Responsible for the issuing, negotiation, and consistency of 164 new PMRS/PMCs in 2024.
Supported the regulatory review and approval of 55 NDAs and BLAs including supplements providing postmarket safety and PMR/PMC support in the Office of Oncologic Diseases.
Provided data scientist support for regulatory review of 31 applications and 16 research projects in OCE.
Developed and launched the Oncology Safety Evaluation (OncoSET )Tool based on an R programming package that allows for efficient safety analyses in oncology applications.

Outreach/Stakeholder Engagement

Engaged with Project Data Sphere and academic collaborators in the immune mediated adverse reactions space to further research on this important topic to enhance our understanding of the patterns of organ involvement of immune mediated adverse reactions.
Engaged with the CTCAE 6.0 revision working group to provide oncology specific FDA input on revisions to the CTCAE grading system for toxicities of oncology drugs.

Safety Regulatory Guidance Development

Co-led the development of the draft guidance, “Cancer Clinical Trial Eligibility Criteria: Laboratory Values.”
Contributed to development of final guidance “Sponsor Responsibilities - Safety Reporting Requirements and Safety Assessment for IND and Bioavailability/Bioequivalence Studies.”
Contributed to the published draft guidance, “Assessment of Ovarian Toxicity in Premenopausal Adults During Drug Development for Oncologic Products.”
Contributed to the final guidance, “Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases

Project Renewal

Project Renewal is an OCE initiative to evaluate publicly available information to update drug labeling for long-standing cancer drugs with decades of use. The project provides a unique opportunity for FDA to externally engage with the oncology community to obtain feedback on contemporary use of cancer drugs and promote awareness of FDA’s regulatory standards and product labeling. In its sixth year, Project Renewal continues to refine repeatable evidence evaluation processes that engage multi-disciplinary teams of oncologists, clinical fellows, and other scientific experts to efficiently review and update older oncology drug labeling and streamline the FDA independent review process.

In 2024, Project Renewal:

Facilitated review for the FDA approval of updated prescribing information for Fludarabine Phosphate Injection on November 19, 2024. With this action, Project Renewal has facilitated reviews for the approval of 15 NDA supplements for new indications for use and/or dosage regimens.
Collaborated with FDA Office of Generic Drugs to update the prescribing information for generic fluorouracil drugs (ANDAs) and align advice and information for DPD deficiency previously approved in the 2022 Xeloda (capecitabine) labeling.
Completed FDA independent reviews of two additional oncology products.
Streamlined the standalone PLR/PLLR conversion process for products under Project Renewal.
Initiated review of four additional oncology products, including evaluating potential new or updated uses.
Engaged with nine oncologists and four clinical fellows from 13 institutions across the U.S. to evaluate labeling as part of our on-going effort to improve awareness and education of FDA product labeling.
Through the end of 2024, a total of 34 oncologists and 26 clinical fellows from 42 institutions across the U.S. have participated in Project Renewal evaluations.
Expanded Project Renewal outside of oncology through collaboration with FDA’s Division of Cardiology and Nephrology on the standalone PLR/PLLR conversion of two cardiology drugs. One cardiologist and two labeling experts from two institutions participated in Project Renewal.

Project Confirm

Begun in 2021, OCE’s Project Confirm enhances transparency around oncology-related Accelerated Approvals through a comprehensive public database. This database, which tracks all oncology Accelerated Approvals since 1992, categorizes approvals as ongoing, converted to traditional approval, or withdrawn. The platform has become an essential resource supporting OCE publications and Oncologic Drugs Advisory Committee meetings.

In 2024, the project expanded its scope to support new requirements under the 2023 Consolidated Appropriations Act, particularly monitoring 180-day confirmatory trial progress reports. Project Confirm also assisted with several initiatives, including a draft industry guidance clarifying FDA's criteria for determining whether confirmatory trials are actively underway during Accelerated Approval.