Articles

Real World Evidence in FDA Drug Approvals: Examples and Reasons for Exclusion from Prescribing Information

Comprehensive analysis of specific examples where Real World Evidence (RWE) and Real World Data (RWD) were considered or excluded from FDA drug labeling, including detailed FDA rationale for inclusion and exclusion decisions.

Patient-Reported Outcomes in FDA Prescribing Information

Examine CDER's practices regarding the inclusion of patient-reported outcomes in prescribing information documents over the past five years.

In-Process Bioburden Limits for Drug Products

Understand FDA expectations for in-process bioburden limits in drug product manufacturing based on recent regulatory actions.

Ozempic Prescribing Information Changes: How Supplied Section Updates Since 2022

Complete chronological review of all documented changes to Ozempic's approved prescribing information since 2022, including How Supplied section modifications with new pen presentations, strength additions, discontinuations, and updated storage instructions across FDA and EMA approvals.

Primary Endpoints for a Stage 3 Clinical Trial for Huntington's Disease

Comprehensive analysis of primary endpoints used in stage 3 clinical trials for Huntington's disease drugs, including clinical rating scales (UHDRS, cUHDRS, TFC, TMS), cognitive assessments, biomarkers (mHTT, NfL, tau), and imaging measures.

Stage 2 Clinical Trial Primary Endpoints for Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma

Comprehensive analysis of primary endpoints for stage 2 trials in R/M HNSCC, including objective response rate (ORR), progression-free survival (PFS), overall survival (OS), disease control rate (DCR), safety assessments, patient-reported outcomes, and biomarker-based measures.

Common FDA Feedback in Type B Meeting Minutes for Solid Tumor IND Preparation

Analysis of recurring FDA feedback themes from Type B meeting minutes for solid tumor therapies preparing IND submissions, covering preclinical data requirements, clinical trial design, safety assessments, manufacturing considerations, and regulatory alignment strategies.

U.S. Enrollment in Multi-Regional Clinical Trials: FDA Oncology Approval Trends and Requirements

Comprehensive analysis of how U.S. patient representation in oncology MRCTs influences FDA approvability, including recent regulatory trends, expectations for meaningful U.S. enrollment, documented cases where low U.S. participation affected outcomes, and implications for future trial design.

CDER Deficiencies and Information Requests for Injectable Drugs 2024-2025

Comprehensive analysis of CDER's specific requests for information and deficiencies regarding injectable drugs and delivery devices disclosed in review memos from 2024 and 2025, covering drug quality tests and device ISO requirements.

FDA ISO 11040 Requirements for Autoinjectors and Prefilled Pens

Explore FDA requirements for prefilled syringe performance testing per ISO 11040 for autoinjectors and prefilled pens, including leakage and flange break resistance.

CDER Questions in BLA Reviews for Autoinjectors and Prefilled Pens

Explore specific questions CDER has asked in recent BLA reviews for autoinjectors and prefilled pens, focusing on design verification, shipping validation, and more.

Autoinjector Shelf Life Extension Studies

Review FDA expectations for shelf life extension studies specifically for autoinjector combination products.

Autoinjector Patient Handling Studies

Learn about FDA expectations for patient handling studies for autoinjector combination products.

FDA ISO 11608 Requirements for Prefilled Syringes

Analyze FDA review documents to determine explicit requirements for ISO 11608 testing and conformance for prefilled syringes and PFS/NSD products.

Autoinjector and Prefilled Pen Usability Studies in EMA CTIS

Analyze the CTIS database to identify clinical trial protocols that include actual use or patient usability studies of autoinjectors or prefilled pens, specifically focusing on studies where patients themselves perform the injections rather than healthcare professionals.

FDA Requirements for URRA Revisions in BLA and NDA Reviews

Documented instances from recent FDA reviews where revisions to Use-Related Risk Analysis (URRA) submissions were explicitly required, including specific examples and FDA rationale.

FDA Information Requests for Autoinjector Testing and Performance

Comprehensive analysis of FDA information requests to BLA and NDA applicants regarding autoinjector testing and performance since 2022, including examples where the FDA explicitly accepted applicant responses.

FDA Combination Product Types and Classification Framework

Comprehensive overview of FDA combination product types including drug-device, device-biologic, and drug-biologic combinations. This review examines the regulatory classification framework, assignment criteria for lead Centers (CDER, CDRH, CBER), examples of each combination product type, and key considerations for determining primary mode of action in combination product development.

FDA Information Requests in BLA Reviews for Part 4 Compliance

Analyze common FDA information requests during BLA reviews regarding Part 4 compliance, focusing on management responsibility, design controls, CAPA, and purchasing controls.

EMA Non-Clinical Data Requirements for Human Polyvalent Immunoglobulins

Examine what non-clinical toxicity data the EMA has accepted for human polyvalent immunoglobulin products, including study omissions and alternative approaches.

FDA Requirements for Retroviral and Lentiviral Vectors in T-Cell Gene Therapy BLA Approvals

Comprehensive analysis of FDA requirements for BLA approval of retroviral and lentiviral vectors used to genetically modify T-cells, covering integration site analysis, long-term follow-up, manufacturing controls, and risk mitigation strategies for insertional oncogenesis.

Dupixent Prescribing Information Changes: How Supplied Section Updates 2020-2024

Comprehensive chronological analysis of all changes to Dupixent's FDA-approved prescribing information from 2020-2024, with detailed focus on How Supplied section updates including new auto-injector presentations, pre-filled pen additions, and regulatory label modifications.

FDA Preclinical Testing Expectations for Large Molecule Drugs in IND Applications

Comprehensive analysis of FDA preclinical testing requirements for biologics in IND applications, including toxicology studies (rodent and non-rodent), pharmacology evaluations, reproductive toxicity assessments, GLP compliance, species selection considerations, and regulatory flexibility for exploratory INDs.

Preclinical Toxicity Endpoints for Large Molecule Drugs in FDA Approval

Comprehensive analysis of toxicity endpoints used in preclinical testing for large molecule drugs seeking FDA approval, including organ toxicity assessments, clinical pathology parameters, histopathology evaluations, GLP compliance requirements, and ICH S6 guidance alignment for biologics.

Transitioning from Research-Grade Protein to Clinically Compliant cGMP Production

Comprehensive roadmap for transitioning research-grade protein products to clinically compliant cGMP manufacturing, covering quality system implementation, process validation, analytical method qualification, stability studies, and regulatory documentation requirements. This review examines critical steps including establishing manufacturing controls, implementing quality assurance systems, characterizing product attributes, conducting comparability studies, and preparing IND-enabling documentation to ensure successful transition from research to clinical-grade protein therapeutics.

Recent Energy-Based Medical Devices Entering the Market

Comprehensive overview of energy-based medical devices that have recently received FDA approval or clearance, covering radiofrequency, ultrasound, laser, and microwave technologies across therapeutic applications including aesthetics, oncology, and other medical specialties.

Clinical Trials for Point-of-Care Diagnostic Devices: Recent Studies

Comprehensive analysis of clinical trials conducted in recent years evaluating point-of-care diagnostic devices across diverse applications including infectious diseases, cardiometabolic conditions, and respiratory illnesses, highlighting study designs and device performance.

FDA Acceptance of Out-of-US Real World Evidence Data for De Novo Device Classifications

Detailed analysis of documented cases where the FDA has accepted Real World Evidence (RWE) data collected outside the United States to support De Novo device classification requests. This review examines specific examples, FDA rationale for acceptance, data quality considerations, and regulatory precedents for using international RWE in De Novo submissions.

Medical Device Product Code Assignment: Process and Responsibilities

Comprehensive examination of the FDA's medical device product code assignment process, explaining how product codes are determined and allocated during regulatory review. It outlines the role of FDA reviewers in device classification, the criteria used for assigning codes, and the administrative procedures within CDRH that govern product code management and maintenance. Accurate product code assignment ensures consistent regulatory oversight and proper device tracking within the FDA framework.

Sampling Plans and Sample Size Justification for ISO 13485 Design V&V

Comprehensive analysis of standards and guidance documents covering sampling plans and sample size justification for design verification and validation under ISO 13485 clauses 7.3.6 and 7.3.7. This review examines statistical methodologies, acceptance criteria, risk-based approaches, and regulatory expectations for establishing appropriate sample sizes in medical device design control activities.

FDA Formal Changes to PMA and HDE Submissions: Quality Management System Regulation Updates

Comprehensive examination of formal FDA changes to Premarket Approval (PMA) and Humanitarian Device Exemption (HDE) submission content requirements in relation to the Quality Management System Regulation (QMSR). This review identifies draft guidances, final guidances, and regulatory updates that address QMSR compliance expectations for PMA and HDE applications, including documentation requirements, quality system alignment, and transition considerations from 21 CFR Part 820 to the harmonized QMSR framework.

Panel Track PMA Submissions: Requirements for FDA Advisory Panel Review

Detailed analysis of FDA requirements for panel track Premarket Approval (PMA) submissions and circumstances under which advisory panel review may be waived or bypassed. This review examines regulatory precedents, FDA guidance on panel track designation criteria, documented cases of panel review exemptions, and the decision-making process for determining when advisory committee consultation is required versus when it may be omitted for panel track devices.

Essential Considerations Before MDR Stage 1 Audit for Reusable Surgical Instruments

Comprehensive guidance on preparing for an MDR Stage 1 audit for reusable surgical instruments, covering quality management system requirements, technical documentation, risk management, clinical evaluation, and conformity assessment procedures. This review examines critical audit preparation steps including ISO 13485 compliance, essential requirements checklist, notified body expectations, and documentation readiness to ensure successful certification under the European Medical Device Regulation.

FDA 513(g) Request Information Requirements for Early-Stage Dental Devices Without Clear Predicates

Comprehensive guidance on preparing a 513(g) Request for Information submission for early-stage dental devices lacking clear predicate devices. This review examines essential documentation requirements including device description, intended use, technological characteristics, proposed classification rationale, and comparative analysis strategies to obtain FDA feedback on appropriate regulatory classification and market pathway determination for novel dental technologies.