FDA Clinical Trials - Advancing Regulatory Science at the FDA

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There is a very interesting new FDA strategic plan that has just been issued in August 2011 entitled "Advancing Regulatory Science at the FDA."

This is a well-crafted and well-marketed document. It covers a wide range of FDA clinical trials concepts with the focus on using science to improve drug development. All too often, the FDA has been known to rest on historic norms, but this pushes the envelope and provides a focus as to areas where the FDA would like to see new developments. Therefore, this far reaching and in-depth FDA strategic plan has the potential to affect all of us involved in clinical trials. For those of you not wishing to read all 35 pages, of particular interest are the key items (at least as they relate to BioClinica and the area with which I am involved):

1. Identify and evaluate biomarkers and endpoints that can be used in non-clinical and clinical evaluations
• Evaluate the accuracy (specificity and sensitivity) with which animal models and cell-based assays correctly predict potential human risk.
• Assess concordance between animal and human biomarkers of toxicity and determine how the performance of these markers and their interpretation may vary across different organ systems and human populations.
• Evaluate quantitative imaging (e.g., positron emission tomography, magnetic resonance imaging, computed tomography) and other advanced approaches (e.g., metabolomics) for identifying new biomarkers and predictors of efficacy and safety.
2. Companion diagnostics
3. Develop and refine clinical trial designs, endpoints and analysis methods
• Continue to refine clinical trial design and statistical methods of analysis to address issues such as missing data, multiple endpoints, patient enrichment, and adaptive designs.
• Identify and evaluate improved clinical endpoints and related biomarkers for clinical trials in areas where optimal endpoints are lacking (e.g., efficacy and safety endpoints for osteoarthritis in humans and animals, for gene therapy, for ophthalmic indications, for tumor vaccines, and for stem cell-derived therapies).
• Develop novel clinical trial designs and endpoints for special needs (e.g., small trials for orphan indications, designs and endpoints for pediatric trials including neonatal trials).
• Continue to refine the use of modeling and simulation in clinical trial design to enhance the effectiveness of clinical studies.
• Work with a broad coalition of partners to identify key opportunities for improving the conduct and efficiency of clinical trials.
• Continue development and refinement of tools and approaches for assessing benefit/risk ratios.

There are many other topics that are covered in this FDA strategic plan on regulating clinical trials, including computer modeling, etc. So, this hardly covers the in-depth discussion of 35 pages that are published although, unusually for the FDA it also includes pictures and a nice flow! It certainly points to the future of the FDA thinking in clinical trials and adds to the steps that were taken with the Critical Path Initiative a few years ago.

What are your thoughts on this FDA strategic plan as it relates new regulations in clinical trials? Share your thoughts and experiences on FDA clinical trial regulations in the comments area below.