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

USA

FDA Releases Guidance on ISO Data Standards for Medicinal Products

To advance alignment with global standards for medicinal product data exchange, the FDA has finalized guidance on implementing five ISO Identification of Medicinal Products (IDMP) standards.

ICH Finalizes S12 Guideline on Gene Therapy Products

The International Council for Harmonization (ICH) has completed a new guideline outlining nonclinical considerations for gene therapy development, paving the way for member nations to adopt their respective versions.

FDA Recommends RCTs for Oncology Drugs Seeking Accelerated Approval

In new draft guidance, the FDA advises that sponsors pursuing Accelerated Approval (AA) for oncology drugs and biologics should generally rely on randomized controlled trials (RCTs) instead of single-arm studies.

Guidance on Review Process for Pediatric Research Not Approvable by IRBs

The FDA has issued draft guidance describing how sponsors and IRBs should manage studies involving children when the research is not typically approvable. IRBs may refer such studies to FDA or HHS’s OHRP. Given the sensitivity of pediatric research, higher scrutiny is required to ensure patient protection.

FDA Final Guidance Defines ‘Suspect’ and ‘Illegitimate’ under DSCSA

The FDA released final guidance clarifying how it interprets the terms “suspect” and “illegitimate” as used in the Drug Supply Chain Security Act (DSCSA).

FDA Shares Updated Thinking on Electronic Systems, Records, and Signatures

New draft guidance details the FDA’s recommendations on implementing electronic systems, electronic signatures, and electronic records in clinical trials, including expectations for validation.

FDORA Enables Records-Based Inspections

A provision in the Food and Drug Omnibus Reform Act of 2022 (FDORA) allows FDA to rely on manufacturer-submitted records and information in place of certain on-site inspections, signaling a potentially significant shift in inspection practices.

FDA Guidance on Potency Assays for mAbs Targeting Viral Proteins

The FDA has released draft recommendations on developing potency assays throughout the lifecycle of monoclonal antibodies (mAbs) that directly target viral proteins.

FDA Encourages RCTs for Accelerated Approval in Oncology

The FDA’s latest draft guidance emphasizes RCTs as the preferred approach for oncology drugs seeking accelerated approval. Sponsors may use a single RCT to both support accelerated approval and confirm clinical benefit, or run separate trials for early and long-term endpoints.

FDA Outlines Strategy for Digital Health Technologies in Trials

The FDA plans public meetings and new guidances on digital health technologies (DHTs) used in drug clinical trials, addressing concerns about the accuracy and reliability of DHTs for data collection.

FDA Draft Guidance on Pharmacogenomic Data Submissions

The FDA has issued draft guidance clarifying what pharmacogenomic findings should be included in INDs, NDAs, and BLAs. It also outlines recommended formats and detail levels. Once finalized, it will replace 2005 guidance.

Industry Seeks More Clarity on Dosage and Administration Labeling

Despite expanded guidance on dosage and administration labeling, industry representatives are requesting more specific direction on handling unique or complex cases. The draft aims to improve consistency in critical prescribing information such as dose ranges, titration, and duration.

FDA Issues Guidance on Long-Acting Local Anesthetics

In line with efforts to reduce opioid use, the FDA has published draft guidance for developing long-acting local anesthetics designed to deliver pain relief for days instead of hours.

FDA Offers Recommendations for Macular Degeneration Drug Trials

The FDA’s 8-page draft guidance provides recommendations on eligibility criteria, endpoints, and trial design for macular degeneration products. Sponsors may consider superiority or noninferiority designs using approved comparators.

EUROPE

EMA Highlights Innovations in Trials and Real-World Data

In its mid-term assessment of the “Regulatory Science Strategy to 2025,” the EMA notes significant progress, including the launch of the EU Clinical Trials Information System and efforts to strengthen the EU’s position in innovative research.

Updated Rules for EMA Fees under Council Regulation (EC) No 297/95

Effective 1 April 2023, the EMA has revised implementing rules for marketing authorization fees, variation fees, annual fees, and inspection fees.

Guideline on Computerized Systems and Electronic Data in Clinical Trials

The EMA has released final guidance on electronic data capture in clinical trials, replacing the earlier reflection paper from 2010 and describing expectations for electronic source data.

Explanatory Note on EMA Fee Structure

The latest explanatory note outlines revisions reflecting a 10.4% inflation adjustment, with rounding changes applied to administrative and non‑administrative fees.

EMA Q&A on Raw Data Pilot for MAAs

The EMA issued a Q&A document to address common sponsor questions related to the raw clinical data review pilot for marketing authorization applications and post-authorization submissions. The pilot aims to evaluate scientific, operational, and technological value of raw data access.

Regulatory and Industry Perspectives on EU GMP Annex 1 Implementation

As the revised Annex 1 for sterile product manufacturing takes effect, EU regulators expect firms to adopt robust contamination control strategies and implement PUPSIT filter testing. FDA inspectors, while not enforcing Annex 1, are aligning their inspection focus with related quality expectations.