Regulatory

81. FDA Releases Draft Guidance On Clinical Recommendations For “N of 1” Gene Therapies 3/15/2022

    To eliminate ineffectiveness due to genetic differences, clinical trials of individual patients, described as an “N of 1” (single-subject) trial, can provide crucial insight. The regulatory path for conducting such gene therapy trials has been difficult to ascertain until now. This article shares the key takeaways of the FDA's draft guidance and provides an analysis for moving forward.

82. FDA Releases Guidance On Digital Health Data Acquisition In Clinical Investigations 3/8/2022

    Increasingly, digital health technologies are becoming part of the conduct of clinical trials. They cover a broad range of applications, including ingestible and implantable sensors, wearables, electronic signatures on consent forms, and more. This article summarizes the key takeaways of the FDA's new draft guidance, Digital Health Technologies for Remote Data Acquisition in Clinical Investigations. The public comment period ends March 22, 2022.

83. 3 Ways The FDA Is Taking Action On Lack Of Diversity In Cancer Clinical Trials 3/3/2022

    Beyond issuing guidance documents, what actions has the FDA taken to ensure inclusion of relevant patient populations in cancer clinical trials? And what has been the impact? This article outlines three observations regarding the agency's recent efforts.

84. The Clinical Trial Sponsor's Roadmap To Avoid EMA (Cyber) Perdition 3/1/2022

    Clinical trials are one of the sectors most vulnerable to cyberattacks. In the European Medicines Agency (EMA)'s Guideline on computerized systems and electronic data in clinical trials, the EMA goes beyond the traditional software validation and data integrity expectations. It sets requirements and expectations pertaining to user management and ongoing security measures.

85. 6 Tips To Ensure Your NDA/BLA Is Ready For FDA Review 2/25/2022

    When preparing a marketing authorization application for an NDA or a BLA, one aspect that is easily overlooked by small start-ups and mature multi-product drug sponsors alike is verification that all commitments made to the FDA have been completed and that evidence of this has been submitted to the agency.

86. FDA Issues Proposal To Amend The Medical Device Quality System Regulation 2/24/2022

    The FDA is proposing to amend the current good manufacturing practice (cGMP) requirements of 21 CFR Part 820: Quality System Regulation (QSR) to align more closely with the requirements of the internationally recognized ISO 13485:2016. The proposed amendment will also affect 21 CFR Part 4: Regulation of Combination Products.

87. FDA: Postapproval CMC Changes To Be Documented In Annual Reports 2/18/2022

    New CDER guidance represents the FDA's thinking regarding the changes that BLA holders for specified biological products must document in an annual report. The products include therapeutic DNA plasmid products, therapeutic synthetic peptide products of 40 or fewer amino acids, mAb products for in vivo use, and therapeutic recombinant DNA-derived products.

88. Pharmaceutical Quality Compliance In 2022: Data Is Paramount 2/11/2022

    Pharma quality and compliance leaders must anticipate potential manufacturing issues before they happen by leveraging risk-based methodologies, continuously monitoring their systems, and tracking the profusion of data generated during the process. This article outlines the potential of data and analytics to improve quality performance.

89. Clinical Trials: FDA Proposes Benefit-Risk Assessment For New Biologics 2/11/2022

    The FDA's draft guidance illustrates how a drug’s benefits, risks, and risk management options factor into certain premarket and postmarket regulatory decisions. Communication between the sponsor and the FDA regarding the benefit-risk assessment is typically conducted at the end of the Phase 2 study, which can help influence the design of Phase 3 studies.

90. Variables Impacting Cell And Gene Therapy Development Success 2/3/2022

    Explore four key interconnected variables exerting pressure on therapeutics developers to improve their processes earlier in the product’s development.