INSIGHTS ON CELL & GENE REGULATORY ISSUES

  • These Are The Top Priorities & Challenges For New CDER Director Patrizia Cavazzoni, M.D.

    Recently appointed to the permanent CDER director role, Dr. Cavazzoni’s appointment was not unexpected, as she was recruited to join the FDA in 2018 from a senior industry role and served as acting CDER director at the start of the agency’s pandemic response. Her experience suggests she will continue to evolve – rather than revolutionize – FDA’s drug review policies.

  • ANDA Or 505(B)(2): Choosing The Right Abbreviated Approval Pathway For Your Drug

    For developers seeking to obtain approval for previously approved drug products in the United States, the U.S. Food and Drug Administration (FDA) offers two abbreviated approval pathways — an abbreviated new drug application (ANDA) and a 505(b)(2) application. Read about these in the available blog.

  • New FDA Draft Guidance Provides Insights On Use Of Pros In Oncology Trials

    Incorporating patient-reported outcomes (PROs) into clinical trials can help sponsors better understand patients’ symptoms and how a therapy will affect their quality of life. Read how that’s changed with the recent publication of a new draft guidance from the U.S Food and Drug Administration’s (FDA) Oncology Center of Excellence (OCE), Core Patient-Reported Outcomes in Cancer Clinical Trials.

  • Planning The Way Forward In Neurodegenerative Disease Studies

    The Food and Drug Administration (FDA) recently released specific draft guidance titled, Human Gene Therapy for Neurodegenerative Diseases (ND). This guidance provides recommendations for Sponsors who are developing human gene therapy (GT) products for neurodegenerative diseases, which affect adult and pediatric patients. It focuses on considerations for product development, preclinical testing, clinical trial design and marketing approval pathways. This article outlines several areas to consider when preparing for regulatory agency interactions.

  • FDA Releases Guidance On CMC Changes To An Approved Application: Certain Biological Products

    At the end of June, the FDA released a new guidance to assist applicants and manufacturers of certain licensed biological products in determining which reporting category is appropriate for a change in chemistry, manufacturing, and controls (CMC) information to an approved biologics license application. This article summarizes the guidance.

  • Navigating DSCSA Implementation: Key Requirements & 4 New FDA Guidances

    In the U.S., you're grappling with how to set up a fully interoperable electronic system for securing and tracing products across industry sectors per the Drug Supply Chain Security Act (DSCSA) by Nov. 2023. Here's how to navigate the key requirements, as well as the FDA's four new guidances on the subject released in June.

  • The Importance Of Feasibility Assessments In The Standards Development Process

    Accelerating the adoption of standards relating to best practices in scientific protocols, product testing, product quality and performance specifications will help accelerate innovation, encourage precompetitive collaboration, knowledge sharing, and help facilitate the regulatory review process.

  • PIC/S Annex Update: What Is Your ATMP Control Strategy?

    Recently, the Pharmaceutical Inspection Co-operation Scheme (PIC/S) revised Annex 2 of its GMP guide addressing the manufacturing of advanced therapy medicinal products (ATMPs). While this guide is not an FDA or EMA guidance, it represents the consensus thoughts of a group that those regulators participate in. 

  • Choose The Right FDA Program To Expedite Your Rare Oncology Trial

    With their poorly understood natural histories, phenotypic heterogeneity, and diverse clinical manifestations, rare cancers pose challenges to drug development and represent a significant unmet need in oncology. Faced with limited treatment options, researchers, clinicians, and patients may be seeking approaches to accelerate the development and approval of novel therapies. In this blog post, we review the regulatory programs available to expedite treatments for rare disorders and serious diseases.

  • Data Integrity And IQOQ In A MHRA Audit

    If you’re using or planning to use your Advanced Instruments osmometer in a GMP environment then its level of compliance will be part of the discussion during an inspection by regulators such as MHRA, FDA or others. In this session, Ian Ramsay of Ramsay Pharma talks about the deficiencies typically encountered during inspection, in particular, relating to data integrity.