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

US

FY 2024 Biosimilar User Fees Show Sharp Decrease, Other Fees Increase

While prescription drug user fees increased a hefty 19.9 percent and medical device fees a modest 8.7, fees for biosimilar applications dropped up to an eye-popping 373 percent.

CDER Finalizes Voluntary Consensus Standards for Pharmaceutical Quality Program

The FDA has issued a final guidance outlining a program that allows stakeholders to propose pharmaceutical quality standards for recognition by CDER, providing industry with additional resources for pharmaceutical development and manufacturing.

DOJ and FTC Draft Merger Guidelines Focus on Competition in a Modern Economy

The Department of Justice (DOJ) and the FTC have issued draft guidelines the government will use to determine whether a merger may violate anti-trust laws by risking lessened competition now and in the future.

Foreign Drug Inspections the Focus of House E&C Committee Letter to Califf

Republican members of the House Energy and Commerce (E&C) Committee are pressing FDA Commissioner Robert Califf on the effectiveness of the agency’s foreign drug inspection program given that numerous Indian and Chinese firms have repeatedly flouted FDA regulations. In a letter dated July 18, Chair Cathy McMorris Rodgers (R-Wash.), Subcommittee on Health Chair Brett Guthrie (R-Ky.) and Subcommittee on Oversight and Investigations Chair Morgan Griffith (R-Va.) outlined a series of ongoing concerns regarding the FDA’s foreign inspections, the latest in a back-and-forth between the committee and the agency.

FDA Guidance Harmonizes Assessment and Control of DNA Reactive Impurities

The FDA has issued final guidance on assessment and control of DNA reactive impurities in harmony with guidance prepared by the International Council for Harmonization (ICH).

A Three-Pronged Strategy for Recalls

Recalls can be messy, complicated matters, especially for manufacturers that don’t have a plan in place for analyzing a problem, determining a course of action and carrying out all recall-related tasks in compliance with FDA requirements.

FDA Clarifies When It’s OK to Miss Post marketing Requirement Milestones

In an effort to improve adherence with post marketing requirements (PMR), the FDA has laid out the circumstances under which noncompliance with PMRs is justified in a new draft guidance.

Managing Manufacturing Changes of Gene Therapy Products is Focus of Draft Guidance

Acknowledging the complexity of human cellular and gene therapy (CGT) products, the FDA has written a draft guidance intended to help manufacturers manage and report changes in products, based on a lifecycle approach, and how to assess the effect of manufacturing changes on product quality.

Dietary Management Critical in IEM Drug Trials, FDA Guidance Says

Optimizing dietary management during drug trials is critical to accurately assessing drug efficacy in clinical trials for inborn errors of metabolism (IEM), the FDA says in a new draft guidance. Dietary management is the core avenue of treatment for a number of IEMs in which specific enzyme mutations prevent the breakdown of dietary components and subsequently lead to toxic metabolites and organ damage. The new eight-page draft guidance says trials that don’t adequately consider and brace for the impact of dietary changes may jeopardize the interpretability of their findings.

FDA Offers Direction on Dietary Management in IEM Drug Trials

Optimizing dietary management during drug trials is critical to accurately assessing drug efficacy in clinical trials for inborn errors of metabolism (IEM), the FDA says in a new draft guidance.

CMS’s Updates to IRA Guidance Don’t Change Big-Picture Impact for Manufacturers

In the face of multiple lawsuits and 7,500 public comments, CMS adjusted some aspects of the Inflation Reduction Act’s drug price negotiation provisions, but the changes don’t lessen the law’s overall power to require pharmaceutical companies to sell medications to the government at what they fear will reflect below-market rates.

Expansion, Clarification Needed for Guidance on Incorporating COAs Into Endpoints

Feedback on the FDA’s draft guidance for incorporating clinical outcome assessments (COA) into endpoints has trickled in from a number of stakeholders, including BIO and the International Society for Cell & Gene Therapy, who feel the guidance would be well served with expansion and clarification in certain areas.

EU

EMA Revises Guidance for Its “PRIME Scheme” Drug Development Assistance Program

EMA has issued revised guidance for its PRIority MEdicines (PRIME) scheme that provides early consultation and scientific advice between applicants and regulators for medicines under development and not currently authorized in the EU.

EMA Opens Comment on ICH-Endorsed Draft Paper on RWD/RWE

Use of real-world data (RWD) and real world evidence (RWE) continues to gain traction in countries around the world, as seen in a draft reflection paper endorsed by the International Council on Harmonization and issued for comment by the European Medicines Authority (EMA).

Obtaining an EU marketing authorization, step-by-step

The European Medicines Agency (EMA) is responsible for the scientific evaluation of applications for centralized marketing authorizations in the European Union (EU). This authorization procedure allows pharmaceutical companies to submit a single marketing authorization application to EMA and to market the medicine and make it available to patients and healthcare professionals throughout the European Economic Area on the basis of a single marketing authorization. Applicants may apply in parallel for an EU marketing authorization under the centralized procedure and an opinion for their medicine to be used outside the EU.

Changing the labelling and package leaflet (Article 61(3) notifications)

The page lists questions that marketing-authorization holders (MAHs) may have on Article-61(3) notifications. It provides an overview of the European Medicines Agency’s position on issues that are typically addressed in discussions or meetings with MAHs in the post-authorization phase. When can I submit my 61(3) Notification, What are Article 61(3) Notifications are updated.

Letter of intent for the submission of a consultation to the European Medicines Agency by a notified body on a companion diagnostic in accordance with Regulation (EU) 2017/746

Template of Letter of intent for the submission of a consultation to the European Medicines Agency by a notified body on a companion diagnostic is updated. A single letter of intent should be used if the companion diagnostic concerns several medicinal products falling under the EMA consultation. Applications details, description of companion diagnostic should be filled.

Exemptions from Icelandic package labelling requirements

The Icelandic Medicines Agency has updated guidelines on applications for exemptions from package labelling requirements. In most cases an exemption is only granted on a temporary basis with the aim of protecting human or animal health.