Anavex Life Sciences Corp (AVXL)

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PDUFA VI: Enhancing regulatory science and regulatory decision tools to support drug development and review

With two new and substantive sections, PDUFA VI identifies how regulatory science and regulatory decision support tools can be leveraged to support the drug development and review process. These sections outline specific areas of innovation the agency will undertake.

Under Regulatory Science, we identified several key actions for sponsors:

•The promotion of innovation through enhanced communication between FDA and sponsors during drug development. FDA will maintain “dedicated drug development communication and training staff in CDER and CBER;”
•Ensure sustained success of breakthrough therapy program, drugs for rare diseases, and drug-device combinations;
•The agency will consider an early consultation on the use of new surrogate endpoints;
•Enhancing Use of Real World Evidence (RWE) for use in regulatory decision-making.

The new section on Regulatory Decision Tools focuses on the use of quantitative methods for drug development:

•To facilitate the development and application of exposure-based, biological, and statistical models derived from preclinical and clinical data sources, herein referred to as ‘model-informed drug development’ (MIDD) approaches, specifically:

•Physiologically-based pharmacokinetic modeling (PBPK);
•Design analysis and inferences from dose-exposure-response studies;
•Disease progression model development, including natural history and trial simulation;
•Immunogenicity and correlates of protection for evaluating biological products;

•Enhancing capacity to review complex innovative designs, the agency will develop the staff capacity to facilitate appropriate use of complex adaptive, Bayesian, and other novel clinical trial designs;
•FDA will develop the capacity to review and provide feedback to sponsors on the readiness of submitted analysis data sets for statistical review and for biomarker qualification review;
•To incorporate the patient’s voice in drug development and decision-making, the FDA will systematize the collection and use of patient and caregiver data.

https://www.certara.com/2017/10/06/fda-reauthorization-act-fdara-affirms-commitment-to-innovative-midd-strategies/