HOW SUCCESSFUL HAVE PEDIATRIC INVESTIGATION PLANS BEEN IN STIMULATING RESEARCH FOR PEDIATRIC CANCERS?
Author(s)
Miller KL
University of North Carolina at Chapel Hill, Chapel Hill, NC, USA
Presentation Documents
OBJECTIVES The European Pediatric Medicine Regulation was created in 2007 to encourage further drug development for pediatric diseases, by requiring pharmaceutical companies to submit pediatric investigation plans (PIPs) when submitting the marketing application for a new drug. The objective of this study was to determine how successful this legislation had been in stimulating research in pediatric cancers. METHODS Current oncology PIPs were manually extracted from the EMA website for the study period 2007-2014. Primary variables included: indication, applicant, decision, decision date, and date of expected completion. Indications for approved PIPs were classified into five categories: brain tumors, diagnostics, leukemias/lymphomas, side effects, and solid tumors. RESULTS A total of 105 PIPs were found; 36 of which were waivers, and 69 of which were approved. The study found that 39% (27) of approved PIPs were indicated for solid tumors, including melanomas and malignant tumors; 30% (21) of approved PIPs were indicated for leukemias or lymphomas; 17% (12) of approved PIPs were indicated for chemotherapy-induced side effects, such as anti-nausea and neuotropenia medications; 12% (8) of approved PIPs were indicated for brain tumors; and one oncology diagnostic PIP was also approved. The ramp-up of the PIP program was significant. PIPs approved in 2013 and the first half of 2014 accounted for 54% (37) of all PIPs approved during the study period. CONCLUSIONS Approved PIPs covered a wide range of pediatric cancers, and the number of approved PIPs increased significantly over time. While the ramp-up of the PIP program indicated that it was successful in promoting research in this area, serious concerns remained regarding the feasibility of the program. For example, there were currently four trials planned for completion between 2015-2020 for extremely rare high grade gliomas. This limitation may compromise the integrity of the PIP program.
Conference/Value in Health Info
2014-11, ISPOR Europe 2014, Amsterdam, The Netherlands
Value in Health, Vol. 17, No. 7 (November 2014)
Code
PCN231
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling, Pricing Policy & Schemes
Disease
Oncology, Pediatrics