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Nov 3, 2018

This guidance provides recommendations to sponsors of drugs or biologics for the treatment of cancer regarding the design and conduct of clinical trials intended to simultaneously evaluate more than one investigational drug and/or more than one cancer type within the same overall trial structure (master protocols) in adult and pediatric cancers. In general, the recommended phase 2 dose (RP2D) has been established for an investigational drug or drugs evaluated in a master protocol.

This guidance is intended to assist applicants in preparing abbreviated new drug applications (ANDAs) for submission to FDA under section 505(j) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 355(j)). This guidance details the information that should be provided in each section of the common technical document (CTD) format for human pharmaceutical product applications and identifies supporting guidance documents and recommendations issued by FDA to assist applicants in preparing their ANDA submission. This guidance identifies the information that an applicant should include to ensure that a complete, high-quality application is submitted to FDA. FDA has previously published guidance documents on the filing process, including the guidance for industry about refuse-to-receive standards, and common, recurring deficiencies which should be reviewed thoroughly prior to submission of an ANDA.

The purpose of this document is to recommend international standards for, and promote harmonization of, the nonclinical safety studies recommended to support the development of pediatric medicines. Harmonisation of the guidance for nonclinical safety studies will define the current recommendations and reduce the likelihood that substantial differences will exist among regions. It should facilitate the timely conduct of pediatric clinical trials and reduce the use of animals in accordance with the 3Rs (replace/reduce/refine) principles.

The European Medicines Agency (EMA) and the European Commission’s Directorate-General for Health and Food Safety (DG SANTE) have published today a joint action plan to support the development of medicines for children in Europe

The action plan addresses challenges identified by the European Commission’s ten-year report on the implementation of the Paediatric Legislation. It also takes into account the ideas on how to better apply the Paediatric Regulation to boost the development of medicines for children collected during the multi-stakeholder workshop organised by EMA and the European Commission in March 2018.

The actions in the plan are clustered around five key areas:

• identifying paediatric medical needs;
• strengthening cooperation between decision makers;
• ensuring timely completion of paediatric investigation plans (PIPs);
• improving the handling of PIP applications;
• increasing transparency around paediatric medicines.

Read the pdf