FDA releases draft guidance on children's oncology drug research

Today, the FDA released a draft guidance on the research of children's cancer drugs The purpose of the draft is to provide information about the submission of the preliminary research plan for children's cancer drugs (IPSP) to enterprise research institutions, etc., which provides guidance in the form of questions and answers to solve the most common problems The FDA said there is no legally enforceable responsibility, but described the FDA's current thinking on children's cancer drug research This draft will take August 18, 2020 as an important time node According to the latest FD & C act, the application for original drugs will be submitted on or after August 18, 2020 According to the molecular mechanism of action, the research on the application of some targeted cancer drugs with new active ingredients in children will be carried out The fdara has set up a mechanism that requires the evaluation of some new drugs that may address the current medical needs of children In order to accelerate the evaluation of these products and ultimately promote the development of appropriate new therapies for children patients, we need to study the anti-tumor activities of potential effective targeted drugs in adults in time, and consider the drug toxicity of these drugs relative to the unique growth and development of children patients It is also mentioned that any original drug that submits a new drug application (NDA) or a biological product license application (BLA) for a new active ingredient on or after August 18, 2020 must include a molecular targeted cancer study report for children, unless 56 requirements are approved for extension or waiver The draft guidance refers to, but is not limited to, the following points: 1 The marketing application (or supplementary application) of new active ingredients, new indications, new dosage forms, new drug delivery plans or new drug delivery routes planned to be submitted before August 18 needs to be submitted to IPSP In the case of planning to submit the listing application of adult cancer drug after August 18, whether the drug is an adult cancer indication or a designated indication drug for a rare disease, IPSP shall be submitted; 2 If the application of the research plan is a new active ingredient, but it is not sure whether its molecular target is considered to have a substantial relationship with children's cancer, then we should refer to the relevant list of children's molecular targets, scientific literature, seek the opinions of children's cancer experts, and consider the pre clinical evaluation of its products in the children's tumor model system; 3 If a product containing a new active ingredient is awarded the title of orphan drug for the sought indication, but is aimed at a molecular target that is considered to be substantially related to childhood cancer, if the product is used to treat adult cancer, it can be exempted from preea only if it is submitted before August 18, 2020 Because the development time may be unpredictable, FDA recommends that all companies developing such drugs submit IPSP anyway If the application is submitted on or after August 18, 2020, an applied study of the child is required unless waived Early discussion with FDA before application submission helps to plan the time of application submission and promote the development of research if necessary to ensure that the requirements of pre a are met The above listed common issues mentioned in several draft guidelines are designed to consider the unique growth and development of children patients and promote the development of appropriate new therapies for children patients Reference source: pediatric study plans for oncology drugs: transitional information until full implementation of fdara Section 504 questions and answers.