#RegulatoryAffairsUpdate List - November 14th
Valerie VIEILLEDENT, MBA ??Regulatory Affairs Leader
USA
Final guidance document, recommendations for the submission of drug and biologics applications for review under Real-Time Oncology Review (RTOR). November 2023
The purpose of this guidance is to advise applicants on the submission process for selected new drug applications (NDAs) and biologics license applications (BLAs) with oncology indications to be reviewed using the RTOR approach. It's important to note that this guidance does not cover FDA's expedited programs such as Fast Track Designation, Breakthrough Therapy Designation, or Priority Review Designation. Further details on these expedited programs can be found in the guidance for industry titled "Expedited Programs for Serious Conditions – Drugs and Biologics" (May 2014).
It's crucial to understand that RTOR is distinct from the Split Time Application Review (STAR) pilot program established under the Prescription Drug User Fee Act (PDUFA) VII commitments.
As a general principle, FDA guidance documents do not establish legally enforceable responsibilities. Instead, they reflect the Agency's current perspectives on a given topic and should be considered as recommendations unless specific regulatory or statutory requirements are explicitly cited. The use of the term "should" in Agency guidances implies a suggestion or recommendation rather than a mandatory requirement.
CANADA
Consultation: Proposed Amendments to Selected Regulations on Food Additives, Compositional Standards, Microbiological Criteria, and Methods of Analysis for Food, 06-Nov-2023
This proposed document was pre-published in the Canada Gazette, Part I, on November 4, 2023 and will be open for comments until February 2, 2024.
The proposal also involves consequential amendments to the following regulations:
Health Canada and the Canadian Food Inspection Agency invite input on the regulatory proposal aimed at modernizing the regulatory frameworks governing food compositional standards, microbiological criteria, methods of analysis, and food additives. The objectives of this regulatory proposal are to enhance the agility, transparency, and responsiveness of the Canadian food regulatory system to new and emerging science, technology, market innovation, and health risks.
EU
EMA/CHMP/136018/2023: Preliminary Guidelines on Clinical Requirements for Non-Replacement Therapy in Haemophilia A and B, 12-Oct-2023
This document from the European Medicines Agency (EMA) presents the preliminary guidelines outlining the clinical requirements for non-replacement therapy in haemophilia A and B.
The guidelines delineate the essential clinical data necessary to support applications for marketing authorization for non-replacement therapy designed to prevent bleeding in patients with haemophilia A and/or haemophilia B. The primary objective of these guidelines is to establish uniform requirements for marketing authorization applications related to non-replacement therapies for haemophilia A and/or B, offering a standardized framework for both applicants and regulatory authorities.
The EMA has issued this document for the purpose of receiving comments. The deadline for submitting comments is set for April 30, 2024.
To provide feedback, stakeholders are encouraged to utilize the online EUSurvey form accessible at https://ec.europa.eu/eusurvey/runner/2f3b4581-597a-1d30-dfb0-8448388895b6.
UK
MHRA Guidance: Submission of Nitrosamine Risk Evaluation, Risk Assessment, and Confirmatory Testing by Marketing Authorisation Holders, 09-Aug-2023
This document offers guidance regarding the submission by Marketing Authorisation Holders (MAHs) of Nitrosamine risk evaluation, risk assessment, and confirmatory testing. Aligned with the CHMP opinion under Article 5(3) of Regulation (EC) No. 726/2004 addressing the presence of nitrosamine impurities in human medicinal products, MAHs are advised to proactively review their manufacturing processes to identify and, if detected, mitigate the risk of nitrosamine impurities.
To address this concern, MAHs are encouraged to collaborate with the manufacturers of Active Pharmaceutical Ingredients (API) and finished products, assessing their manufacturing processes in relation to measures preventing nitrosamine formation, contamination, or cross-contamination. This evaluation should encompass an understanding of manufacturing processes and potential sources of nitrosamine impurities. Additionally, MAHs are expected to periodically revisit and reassess the outcomes of the risk evaluation, particularly when new information arises. This includes modifications to known root causes that might impact the relevant product and its lifecycle, potentially necessitating a revision of the risk evaluation and altering its outcome.
Australia
Guidance: Oversight of Sport Supplements in Australia: Guidance for Importers and Sellers, 08-Nov-2023
In Australia, certain sport supplements are legally designated as therapeutic goods (medicines) to ensure adherence to stringent quality and safety standards. Consequently, these products fall under the regulatory purview of the Therapeutic Goods Act 1989 and are overseen by the Therapeutic Goods Administration (TGA).
The TGA regularly evaluates the advertising of sport supplement products and conducts laboratory testing on sampled products from the Australian market.
The content of this document outlines:
Guidance: Ensuring Compliance After Removing the Product Information Insert, 01-Nov-2023
This guidance document describes options for the removal of the Product Information (PI) as an insert from a medicine package while still complying with the labelling order
CHINA
领英推荐
CDE Notification: Invitation for Public Feedback on Draft Technical Guidelines for Clinical Trials of Drugs for the Treatment of Chronic Heart Failure, 30-Oct-2023
Chronic heart failure encompasses a set of intricate clinical syndromes arising from abnormal changes in cardiac structure and/or function due to various factors, leading to impairment of ventricular systolic and/or diastolic function. With a chronic progressive course, it results in hospitalizations and fatalities. In recent years, research and development of drugs for chronic heart failure have posed challenges and garnered significant attention within the cardiovascular drug landscape. To provide guidance for enterprises in scientifically developing drugs for chronic heart failure treatment and to elucidate technical standards for clinical trials, the Technical Guidelines for Clinical Trials of Drugs for the Treatment of Chronic Heart Failure has been drafted and is open for public comments.
This guideline aims to offer technical recommendations for clinical trials of drugs targeting chronic heart failure and is applicable to the development of chemical drugs and therapeutic biological products. Specifically, it is geared towards the development of drugs for treating chronic heart failure, encompassing both reduced ejection fraction (HFrEF, LVEF ≤ 40%) and preserved ejection fraction (HFpEF, LVEF > 50%) categories.
Focusing on design aspects and review requirements for clinical trials of chronic heart failure drugs, the guideline delves into key elements such as the study population, definition of heart failure types, and selection of efficacy indicators. It provides insights into the design points for clinical pharmacology research, exploratory clinical trials, and confirmatory clinical trials, offering references for the comprehensive strategy of chronic heart failure drug development and clinical trial design. Additionally, the guideline addresses safety evaluation concerns associated with drugs for chronic heart failure.
CDE Notification: Invitation for Public Input on Draft Technical Guidelines for Sample Size Estimation in Drug Clinical Trials, 30-Oct-2023
Sample size estimation constitutes a crucial aspect of drug clinical trial design, serving as a pivotal method to ensure the rationality, accuracy, reliability, and comprehensiveness of the study. Typically, the sample size in a clinical trial must be sufficiently large to provide reliable answers to the clinical question addressed by the study hypothesis. Given the intricate nature and significance of estimating sample size in clinical trials, the Technical Guidelines for Estimating Sample Size in Drug Clinical Trials has been drafted and is open for public comments.
To provide guidance for sponsors in conducting scientifically and reasonably grounded sample size estimations, these technical guidelines primarily expound on fundamental considerations such as parameter setting and sample size adjustment based on statistical assumptions. This guideline is applicable to confirmatory clinical trials intended for registration purposes and can serve as a valuable reference for other clinical studies.
The draft document encompasses the following:
JAPAN
MHLW Document for Public Comments No. 495230214: Draft Guidelines for Nonclinical Safety Evaluation of Synthetic Peptide Drugs with Non-natural Structures, 30-Oct-2023
This document is open for public commentary regarding the draft of Guidelines for the Nonclinical Safety Evaluation of Synthetic Peptide Drugs with Non-natural Structures.
The primary objective of these Guidelines is to streamline the development process of synthetic peptide drugs with nonnatural structures and ensure their safety in human use. The document proposes a recommended foundational framework for the non-clinical safety assessment of such synthetic peptide drugs. By outlining fundamental principles in the nonclinical safety evaluation of non-natural peptide drugs and offering guidance on the design of each safety study, the Guidelines aim to promote safe and ethical development practices. This includes an emphasis on minimizing the use of test animals and resources in alignment with the 3Rs principles (use of alternative methods, reduction in the number of animals used, and alleviation of animal suffering).
The Guidelines specifically address the following key areas:
SOUTH KOREA
MFDS Guide 1303-01: Guidelines for Approval Review of Quasi-Drug Oral Products, 27-Sep-2023
This guideline aims to support companies in enhancing their quality control processes by offering comprehensive standards and test methods for both raw materials and finished products categorized under quasi-drug oral products. It is designed to aid in the preparation of submission materials required for approval when manufacturing (or importing) quasi-drugs. It is essential to note that the standards and test methods provided in this guideline may not uniformly apply to all items. Instead, they may be subject to individual review, depending on the specific ingredients and characteristics of the product.
The contents of the guideline include:
I. Introduction
II. Scope of Oral Products
III. Efficacy and Effectiveness of Oral Products
IV. Matters Relating to the Preparation of Standards and Test Methods
V. Use of Alternative Animal Testing Methods for Oral Products
VI. Appendix
VII. References
Taiwan
TFDA Announcement: Real World Data - Guidance on Assessing Electronic Health Records and Medical Benefits Data for Drug Regulatory Decisions, 30-Oct-2023
Real-world data/real-world evidence (RWD/RWE) stands at the forefront of the global clinical application landscape, offering opportunities to enhance clinical trial design and serve as supplementary evidence for the efficacy and safety of drugs in both pre and post-market phases. Countries worldwide are actively advancing the utilization of real-world evidence, aligning with international regulatory trends and tailoring strategies to domestic contexts. In accordance with these efforts, the "Real World Data: Guidance on Evaluating Electronic Health Records and Medical Benefits Data to Support Drug Regulatory Decisions" is developed as a reference for domestic drug research and development.
This guidance complements the existing "Guidelines for Conducting Pharmacoepidemiologic Safety Studies using Electronic Healthcare Data ." Beyond its application in post-marketing drug safety studies, the electronic health records (EHRs) and medical payment data outlined in this guideline can also contribute to evaluating drug effectiveness and supporting regulatory decisions, including new indications for post-marketing drugs. The studies within this guideline encompass interventional as well as observational studies. Consequently, this guidance delves into specific aspects related to data source selection and provides additional instructions for assessing the suitability and reliability of using EHRs and health care payment data in clinical research. Moreover, it offers a comprehensive overview of considerations related to the use of EHRs and health care payment data, extending to studies aimed at furnishing regulators with assessments of drug effectiveness.
Intended for applicants, researchers, and other stakeholders, this guidance presents key considerations for regulatory decisions concerning the use of EHRs or health care payment data in clinical studies to substantiate the effectiveness or safety of drug products. Given its inclusion of regulatory decisions on efficacy alongside safety, ensuring the reliability and relevance of the data becomes pivotal in guiding study designs, statistical analysis methods, and data source selections.
ARGENTINA
ANMAT-PME-SOF 001-00 (Draft): Guidelines for the Industry on Software as a Medical Device (SaMD) and Machine Learning-enabled Medical Devices (MLMD), 03-Nov-2023
This document aims to furnish essential technical considerations and address regulatory aspects pertaining to the design and manufacturing processes of AI and ML-based software. It is intended for all stakeholders involved in these processes.
The document is open for public comment from 03-Nov-2023 to 03-Dec-2023, accessible through the provided link on the ANMAT webpage: https://opinionpublica.anmat.gob.ar/frmSugerencia.aspx?pno_id_proyecto=5293.
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1 年Wow! FDA are on a roll with all these new accelerated pilots and initiatives in oncology! RTOR sounds a bit like a rolling review but unique to hot off the press clinical oncology data.
Global Regulatory Affairs Strategy, Processes & Labelling
1 年Thanks Valerie
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1 年What an exhaustive summary Valerie VIEILLEDENT, MBA ??Regulatory Affairs Leader well done and congrats to over 500 subscribers!