BIO Comments on FDA’s Public Meeting Announcement: Best Practices for Development and Application of Disease Progression Models
March 26, 2021
On Friday, March 26th, BIO submitted comments on the U.S. Food and Drug Administration (FDA)’s public meeting announcement titled, “Best Practices for Development and Application of Disease Progression Models.” BIO appreciates that FDA is seeking input from stakeholders on three key areas, including: the development and application of different types of disease progression models; modeling natural history of disease, specifically methodological considerations and challenges in characterizing the natural relationship between pharmacodynamic markers and clinical outcomes; and clinical trial simulations based on disease progression/natural history models to support drug development and regulatory decisions. Within each topic area, BIO proposed a list of specific issues that would highlight the opportunities and limitations in the development and application of disease progression models. In its comments, BIO also encouraged FDA to include case study examples from the public domain that would be illustrative of disease progression models, and to invite sponsors to present case examples that may not yet be in the public domain, including those that highlight both positive outcomes, lessons learned from negative outcome use cases, and use cases from the Model Informed Drug Development (MIDD) pilot program. To support this discussion, BIO members are willing to offer case study examples for inclusion in the agenda topics. BIO further encourages FDA to develop hypothetical case examples based on Agency experience that illustrate key issues and serve to generate discussion on how those challenges may be overcome.
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BIO Comments on FDA’s Public Meeting Announcement: Best Practices for Development and Application of Disease Progression Models
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On Friday, March 26th, BIO submitted comments on the U.S. Food and Drug Administration (FDA)’s public meeting announcement titled, “Best Practices for Development and Application of Disease Progression Models.” BIO appreciates that FDA is seeking input from stakeholders on three key areas, including: the development and application of different types of disease progression models; modeling natural history of disease, specifically methodological considerations and challenges in characterizing the natural relationship between pharmacodynamic markers and clinical outcomes; and clinical trial simulations based on disease progression/natural history models to support drug development and regulatory decisions. Within each topic area, BIO proposed a list of specific issues that would highlight the opportunities and limitations in the development and application of disease progression models. In its comments, BIO also encouraged FDA to include case study examples from the public domain that would be illustrative of disease progression models, and to invite sponsors to present case examples that may not yet be in the public domain, including those that highlight both positive outcomes, lessons learned from negative outcome use cases, and use cases from the Model Informed Drug Development (MIDD) pilot program. To support this discussion, BIO members are willing to offer case study examples for inclusion in the agenda topics. BIO further encourages FDA to develop hypothetical case examples based on Agency experience that illustrate key issues and serve to generate discussion on how those challenges may be overcome.