StemCell Therapy

This article discusses how to estimate the probability of regulatory/registration success for pharmaceutical products in development. The authors define the factors and methods used to assess regulatory/registration success and offer objective and data-driven methods to define probabilities for the purposes of documenting specific factors and/or risks relative to a likelihood of success. They provide a case study demonstrating how to use data and make estimate adjustments based on a variety of factors specific to individual programs. While this example applies to a drug and the US Food and Drug Administration (FDA), the same strategic approach can be applied to biologics, medical devices and combination products under review with any global regulatory Health Authority (HA).The article is based on a presentation by the authors at RAPS Regulatory Convergence in September 2019.IntroductionDetermining the Probability of Regulatory Success (PRS) for a product approval is a key driver for ensuring business/market readiness, including prioritization for the purposes of budgeting and resource planning. When considering PRS, a decision sciences approach focusing on facts is critical for avoiding biases. To do this consistently requires an ability to evaluate a program and create an objective PRS in a concise, well-documented, transparent manner along with objective, analytically driven decision-making. While a gut feeling about regulatory success may be based on experience and regulatory acumen, it cannot be objectively revised for changes in the regulatory environment and does not allow for a documented, well-vetted strategic and holistic analysis of the risks (downward adjustment) or strengths (upward adjustment) of regulatory considerations. Furthermore, gut-feeling does not support development of actionable mitigation strategies based on facts. Thus, this approach provides an objective framework for forming comparable assessments across a variety of projects.Technical vs. Regulatory SuccessIt is important to note that technical and regulatory success are different objectives and need to be assessed separately. Probability of Technical Success (PTS) applies to the probability a given clinical trial/study will be successful based on pre-defined endpoints, feasibility and other factors. Probability of Regulatory Success (PRS) is about whether FDA or another regulatory authority will grant approval for a product and based on factors within the scope of regulatory affairs, which often includes evaluation of the HAs perspective regarding the clinical relevance for a particular endpoint as it applies to suitability for defining efficacy in the context of an approval application for registration. The overall probability of success will need to consider both the PRS and the PTS and is calculated as PTRS = PRSxPTS.Six Elements of Good Decision MakingThere are six elements for good decision-making.Figure 1. Six Elements1

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Estimating the Probability of Regulatory Registration Success - Regulatory Focus

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