Between 2010 and 2018, 33.8 percent of the 402 orphan indications approved by the FDA were approved for children only or targeted pediatric diseases.[1] The rare pediatric disease priority review voucher program is one incentive that’s helped to spur research and development in this area with more than 20 vouchers granted to date, but it…
Expertise: Pediatrics 20 results
Due to the challenges associated with studying investigational medical products in neonates, healthcare professionals must often resort to administering drugs in neonatal intensive care units (NICUs) off-label.
Despite the passage of various legislative initiatives aimed at increasing the frequency and quality of pediatric clinical trials, neonates continue to be treated with medicines that have not been studied or approved in this population.1 Historically, sponsors may have felt that the ethical and logistical challenges limited the opportunities for neonatal research. Our experience has…
Oncology & Hematology
iPSP Considerations for Molecularly Targeted Cancer Drugs
With the passage of the Pediatric Research Equity Act (PREA) of 2003, drug manufacturers were required to submit an initial pediatric study plan (iPSP) prior to commencement of Phase 3 studies (or new drug application [NDA]/biologics license application [BLA] submission in the absence of a Phase 3 study) for studies involving a new active ingredient,…
Oncology & Hematology
5 Things to Know About the FDA RACE for Children Act
The Research to Accelerate Cures and Equity (RACE) for Children Act aims to improve and expand treatment options for pediatric cancer patients by mandating that all new adult oncology drugs also be tested in children when the molecular targets are relevant to a particular childhood cancer. Enacted August 18, 2017, as part of the Food…
If you are planning to submit a New Drug Application (NDA) for an oncology drug product in 2020, revised pediatric study plan requirements could have an impact on the submission. FDA recently released draft guidance on changes to pediatric study requirements put in place by Section 504 of FDARA: FDARA Implementation Guidance for Pediatric Studies of…
Oncology & Hematology
Considerations for Operationalizing Pediatric Oncology Studies
Remarkable progress has been made in our understanding of the genomics of pediatric cancers, and these advancements have led to the recognition that products being studied for use in adult cancer indications may have health benefits for pediatric patients. By closing the orphan drug exemption loophole and enabling earlier discussions with the FDA, the Research…
Oncology & Hematology
Understanding Recent Regulatory Changes for Pediatric Oncology Trials
Of the 1.7 million new cases of cancer in the U.S. in 2018, 10,590 involved children aged 14 or younger.[1] While children typically have a better cure rate for cancer, biopharma companies have traditionally shied away from pediatric oncology trials, resulting in fewer treatment options. However, the regulatory landscape is evolving, and the demand for…