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

USA

FDA Requests Feedback on Proposed Consumer Prescription Advertising Survey

The FDA’s Office of Prescription Drug Promotion (OPDP) is inviting public comments on its plan to study how consumers interpret quantitative information included in direct‑to‑consumer (DTC) prescription drug advertisements.

FDA Issues Draft Guidance to Support Drug Development for Radiation Exposure

On 19 April, the FDA released draft guidance encouraging early and continuous communication with the agency during development of treatments intended to prevent or address acute radiation syndrome (ARS). ARS encompasses a range of clinical effects caused by high‑dose radiation exposure over a short period — typically within 45 minutes, according to the CDC.

FDA Shares CMC Insights for Gene Therapy Developers During OTP Town Hall

At a virtual town hall held on 25 April, FDA officials from the Office of Therapeutic Products (OTP) advised gene therapy developers to avoid making manufacturing process changes during later‑stage pivotal studies to prevent out‑of‑specification product issues. The guidance was provided by Graeme Price, team lead for Gene Therapy Branch 1, in response to stakeholder questions regarding CMC expectations for INDs and licensure applications.

FDA Seeks Input on Quantitative Claims in Consumer Advertising

The OPDP has announced plans to conduct research evaluating consumer understanding of quantitative claims used in DTC advertisements. While some quantitative formats have been studied, many questions remain regarding the most effective ways to communicate such information.

FDA Prepares for Transition to Quality Management System Regulation (QMSR)

The agency is advancing efforts to finalize the transition from the existing Quality System Regulation (QSR) to the ISO 13485:2016‑aligned Quality Management System Regulation (QMSR). Internal preparations include system updates, staff training and replacement of the current Quality System Inspection Technique (QSIT).

FDA Releases Draft Guidance on OMOR Formatting and Content

A new draft guidance outlines the required format and content for over‑the‑counter (OTC) monograph order requests (OMORs). OMORs allow marketing of OTC drugs without a formal drug application, provided they are generally recognized as safe and effective (GRASE). Electronic submissions are mandatory and must include five modules: administrative information, summaries, quality, nonclinical study reports, and clinical study reports.

Final Guidance Expands FDA Recommendations on Risk‑Based Monitoring

The FDA has issued updated final guidance expanding recommendations for sponsors on implementing risk‑based monitoring approaches in clinical trials, modernizing the agency’s earlier 2013 guidance.

FDA Requests Additional Information on Manufacturing Disruptions

To reduce or prevent shortages, the FDA is urging manufacturers to provide more detailed information regarding production interruptions that may affect finished drugs, biological products and some APIs.

FDA Requires Mail‑Back Envelopes for Opioid Disposal

In an effort to address the opioid overdose crisis, the agency now requires opioid manufacturers to supply prepaid mail‑back envelopes to outpatient pharmacies and other dispensers, making it easier for patients to safely dispose of unused medications.

EUROPE

EMA Becomes First Regulator to Adopt ICH S12 Guideline

The EMA has formally adopted the ICH S12 guideline outlining best practices for conducting nonclinical biodistribution (BD) studies during development of gene therapy products.

EU Unveils Major Drug Development Legislative Reform

The European Union has released a sweeping proposal to overhaul pharmaceutical legislation, introducing changes affecting nearly all stages of drug development and authorization. The proposal has generated significant opposition from the pharmaceutical industry.

EMA to Restart Publication of Clinical Trial Data

Beginning in September, the EMA will gradually resume public release of clinical trial data for approved medical products.

EMA Expands PRIME Pathway with New Review Features

Updates to the EMA’s Priority Medicines (PRIME) program introduce new mechanisms for monitoring product progress and improving the timeliness of scientific advice.

EU Proposes Reforms on Compulsory Licensing and Supplementary Protection Certificates (SPCs)

The European Commission has introduced regulations to create an EU‑wide compulsory licensing system for emergency use and to establish community‑wide SPCs that may extend patent protection for medicines by up to five years. Currently, SPCs are granted only at the national level.

EU Publishes Draft Overhaul of Pharmaceutical Regulation

Following several delays, the European Commission has released the draft legislation intended to reshape the pharmaceutical regulatory landscape. The proposal follows years of debate, with critics expressing concerns about potential impacts on innovation, investment and access to treatment.

EMA Issues Reflection Paper on Single‑Arm Trials

A new reflection paper offers considerations for designing single‑arm trials (SATs) and strategies to reduce bias when such trials are used to support marketing authorization applications. While the document encourages scientific discussion on SAT design, it does not establish general conditions for their acceptance as pivotal evidence.