Pharma/Biotech – USA, Europe Regulatory News, Oct 2023 Vol. 1

USA

FDA Draft Guidance Pushes for Development of Stimulant Use Disorder Treatments

With no FDA drug approvals to date for stimulant use disorder, the FDA has published draft guidance outlining current recommendations for development programs and trial designs for treatment of moderate-to-severe cocaine use, methamphetamine use and prescription stimulant use disorder.

Meeting Requests, Manufacturer Assessments for NDAs and BLAs Subject of Draft Guidance’s

The FDA has issued two draft guidance’s focused on requirements for drug and biological approvals — one on formal meetings and one on alternative methods the agency may use to assess manufacturing facilities named in an NDA or BLA.

FDA Broadens Pandemic-Era Guidance to Cover Trials During PHEs, Disasters

The FDA has expanded the scope of its guidance on conducting clinical trials during the COVID-19 pandemic to apply to public health emergencies (PHE) and other disasters, issuing final guidance that also includes several clarifications.

Prescription Drug Use-Related Software Output Reviewed in Draft Guidance

The FDA has released a draft guidance on how it intends to determine whether sponsor-provided prescription drug software output should be treated as FDA-required labeling or promotional labeling and how, or if, the corresponding software function should be described in the prescribing information (PI).

Single Trial Can Support FDA Drug Approval, Says Draft Guidance

The FDA has offered greater direction on demonstrating substantial evidence of effectiveness for drugs and biologics, publishing draft guidance on whether a single trial plus confirmatory evidence will suffice for a development program.

FDA Final Guidance Tackles Human Factor Studies for Combination Products

The FDA’s latest final guidance features questions and answers on how to apply human factors (HF) engineering principles when developing combination products, clarifying how the uniqueness of these products affect HF engineering considerations for industry and agency staff. The 14-page document, “Application of Human Factors Engineering Principles for Combination Products: Questions and Answers,” finalizes a draft guidance from February 2016 and should be used alongside the guidance’s “Applying Human Factors and Usability Engineering to Medical Devices” and “Safety Considerations for Product Design to Minimize Medication Errors,” as well as other FDA product development guidance’s, advised the agency.

Human Factor Studies for Combination Products Tackled in FDA Final Guidance

The FDA’s latest final guidance features questions and answers on how to apply human factors (HF) engineering principles when developing combination products, clarifying how the uniqueness of these products affect HF engineering considerations for industry and agency staff.

Biosimilar Draft Guidance’s Tackle Labeling and Interchangeables

The FDA released two draft guidance’s for biosimilar manufacturers — one on labeling that focuses on prescribing information and the other on the FDA’s licensure requirements.

FDA Manual of Policies and Procedures Updates ANDA Suitability Petitions

The FDA has issued a revision to its Manual of Policies and Procedures (MAPP) describing how the Office of Generic Drugs will track and respond to ANDA suitability petitions, consistent with the commitments made in the GDUFA III commitment letter.

Cybersecurity Final Guidance Details System Considerations for Premarket Submissions

The frequent electronic exchange of health data through wireless, internet and networks along with the cybersecurity threats and vulnerabilities is driver for an FDA final guidance making recommendations for cybersecurity information to be submitted with premarket applications to CDRH and CBER.

Labeling of Ready-to-Use Prescription Drug Injectables Subject of FDA Guidance

Recommendations on labeling of injectable prescription drug product information submitted in an NDA or certain BLAs is the subject of an FDA guidance on dose banding.

EUROPE

Explanatory note on pharmacovigilance fees payable to the European Medicines Agency

This explanatory note concerns the fees related to pharmacovigilance activities (and the rules of payments) that apply to medicinal products for human use authorized in the Union under Regulation (EC) No 726/2004 and Directive 2001/83/EC. Increase in the level of fees (other than pharmacovigilance annual fees) to adjust for an inflation rate of 10.4% (related to 2022) and rounding off to the nearest EUR 10.  Increase in the level of pharmacovigilance annual fees to adjust for an inflation rate of 10.4% (related to 2022), with no rounding.

Update on human variations web-based electronic application form implementation on product lifecycle management portal

The web-based electronic Application Forms (eAF) in the new Product Lifecycle Management (PLM) portal will replace the current interactive PDF eAFs used for regulatory submissions. This is a first step towards making the form-filling and submission-handling process more efficient. The web-based Variations eAF for Human medicinal Centrally Authorized Products (CAPs) was released on 4 November 2022.

Qualification of novel methodologies for drug development: guidance to applicants

The EMA qualification process is a new, voluntary, scientific pathway leading to either a CHMP Qualification opinion or a qualification advice on innovative methods or drug development tools. The qualification process addresses innovative drug development methods and tools. It will focus on the use of novel methodologies developed by consortia, networks, public/private partnerships, learned societies and pharmaceutical industry for a specific intended use in pharmaceuticals R&D.

IRIS guide for applicants

This guide has been produced to show applicants how to use the IRIS platform to prepare and submit an application and/or data for a scientific procedure (orphan designation application, scientific advice, ITF briefing meeting requests, marketing status reports, inspections and veterinary signal management) and related activities.

User guide for the electronic application form for a Marketing Authorization

This User guide has been prepared in order to facilitate the work of applicants when completing the Electronic Application Forms (eAF) as part of an application for a marketing authorization of a medicinal product for human use / registration of a traditional herbal medicinal products.

European Medicines Agency pre-authorization procedural advice for users of the centralized procedure

This guidance document addresses a number of questions which users of the centralized procedure may have. It provides an overview of the European Medicines Agency’s position on issues, which are typically addressed during the course of pre-submission meetings.

Good clinical practice (GCP) inspection procedures

ANNEX III and V of procedure for conducting gcp inspections requested by the chmp: computer systems have been updated.