Pharmaceutical Technology - September 2019

Pharmaceutical Technology - Regulatory Sourcebook

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Pharmaceutical Technology REGULATORY SOURCEBOOK SEPTEMBER 2019 7 "This series of guidance documents is intended to facilitate the advancement and use of system- atic approaches to collect and use robust and meaningf ul patient and caregiver input t hat can better inform medical product development and regulatory decision making," FDA stated in an announcement. On July 30, 2019, FDA issued draft guidance that answers questions regarding priorit y re- view vouchers for certain rare pediatric disease treatments that meet criteria of the Food, Drug, & Cosmetic Act (FD&C Act) (2). As part of the FD&C Act, FDA may give special incentives to companies for the development of treatments for rare pediatric diseases. This draft guidance re- vises a previous draft guidance and clarifies the qualifications and process for requesting priority review vouchers. Specifically, the guidance gives detailed an- swers to questions regarding the definition of a rare pediatric disease, eligibility requirements, a sponsor's responsibilities after approval of an ap- plication, designation information requests, the submission process, marketing applications, and use and transfer of a rare pediatric disease prior- ity review voucher. The guidance also discusses drug–drug combinations, previously approved drugs, and orphan drug designation questions. Regulatory submissions Guidance released on July 16, 2019 describes how applicants should organize the content they submit to the agency electronically (3). The guid- ance applies to all submission types under sec- tion 745A(a) of the FD&C Act. The guidance also references technical specif ication documents and t he Electronic Common Technical Docu- ment Conformance (eCTD) Guide. Specifically, the guidance implements the electronic submis- sion of content submitted in new drug applica- tions (NDAs), abbreviated new drug applications (ANDAs), certain biologics license applications (BLAs), and certain investigational new drug ap- plications (INDs) to the Center for Drug Evalua- tion and Research (CDER) or the Center for Bio- logics Evaluation and Research (CBER). A previous version of this guidance, published in May 2015, indicated that NDAs, BLAs, ANDAs, and master files were to be submitted electroni- cally in eCTD format starting on May 5, 2017 (May 5, 2018, for commercial INDs). In response to industry concerns, however, FDA extended the implementation date for drug master files (DMFs) to May 5, 2020. This new revision modifies pre- vious versions by including exemptions for Type III DMFs. The guidance has also been updated to include the criteria identifying those types of submissions that may qualify for a long-term or short-term waiver from the eCTD submission re- quirement and instructions on how to submit a waiver request. FDA published draft guidance on July 11, 2019 for sponsors of NDAs and BLAs on how to apply population pharmacokinetic (population PK) analysis, which is used in therapeutic individu- alization such as tailored dosing. Population PK data collection and analysis may alleviate the need for postmarketing requirements or post- marketing commitments, according to FDA (4). The guidance lists common applications of popu lation PK a na lysis to prov ide sponsors with illustrative examples. FDA provides data

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