Overview
Title
Best Practices for Development and Application of Disease Progression Models; Public Workshop; Establishment of a Public Docket; Request for Comments
Agencies
ELI5 AI
The FDA is having a big meeting to talk about how to make better models to understand how diseases change over time, which helps in making new medicines. They want people to share their ideas, and anyone can send in comments about this until March 26, 2021.
Summary AI
The Food and Drug Administration (FDA) is hosting a workshop titled "Best Practices for Development and Application of Disease Progression Models," which is part of their performance commitment under the Prescription Drug User Fee Act of 2017. The workshop aims to discuss the best practices for creating disease progression models that can aid in drug development, and to explore current challenges and research needs in this area. The FDA is inviting the public to submit comments about areas of interest related to disease progression modeling to ensure a comprehensive discussion during the workshop. Comments must be submitted by March 26, 2021, and can be sent electronically or through written/paper submissions.
Abstract
One of the goals of the Prescription Drug User Fee Act of 2017 (PDUFA VI), part of the FDA Reauthorization Act of 2017 (FDARA), is advancing model-informed drug development (MIDD). The "Best Practices for Development and Application of Disease Progression Models" workshop fulfills FDA's performance commitment under PDUFA VI to hold a workshop. The Food and Drug Administration (FDA or Agency) is opening a docket to solicit public input on topics areas for an upcoming disease progression modeling workshop. The purpose of this public workshop is to discuss the best practices for developing disease progression models and their application to support drug development decisions; share experiences and case studies that highlight the opportunities and limitations in the development and application of disease progression models including models for natural history of disease and clinical trial simulations; and discuss the knowledge gaps and research needed to advance the development and use of disease progression models.
Keywords AI
Sources
AnalysisAI
The Federal Register document outlines an upcoming workshop hosted by the Food and Drug Administration (FDA) titled "Best Practices for Development and Application of Disease Progression Models." This workshop is part of a broader initiative under the Prescription Drug User Fee Act of 2017 (PDUFA VI) to advance model-informed drug development. The FDA is seeking public input to shape the workshop's agenda by soliciting comments on various aspects of disease progression modeling. Comments from the public are encouraged to ensure comprehensive discussions during the workshop.
General Summary
The FDA invites comments from the public to identify areas of interest for discussion at the workshop, with particular attention to the development and application of disease progression models. These models predict how diseases develop over time, and their application is crucial in supporting drug development and regulatory decisions. Participants are encouraged to provide input on aspects such as the types of models, challenges in modeling the natural history of diseases, and simulations used in clinical trials.
Significant Issues or Concerns
A notable concern is the complexity of the submission process for public comments, especially when dealing with confidential information. The document outlines procedures for electronic and written submissions, but the instructions are somewhat scattered and technically dense. This complexity may deter the general public from engaging fully, thus limiting the diversity of input the FDA receives.
Another issue is the use of technical jargon, such as "pharmacodynamic markers" and "mechanistic modeling," without providing adequate explanations. This could hinder understanding for those not familiar with drug development or pharmacological terminology.
Additionally, although the document references regulatory frameworks like the FDA Reauthorization Act of 2017 and PDUFA VI, it does not provide context for readers unfamiliar with these terms. This lack of explanation might obscure the importance and implications of the workshop.
Impact on the Public
By soliciting public input, the FDA aims to create a workshop agenda that reflects diverse perspectives, potentially enhancing the quality and relevance of discussions surrounding disease progression models. Engagement from a broad audience can contribute to more robust and patient-focused drug development processes.
However, the procedural complexity may limit participation, thus skewing the input toward those already familiar with regulatory affairs or drug development. Simplifying the language and process could empower more individuals and organizations to contribute.
Impact on Stakeholders
For stakeholders in the pharmaceutical industry and healthcare sector, this workshop represents an opportunity to influence FDA guidelines and policies related to drug development. Companies and researchers can share their expertise and learn from others about the best practices and challenges inherent in disease progression modeling.
On the other hand, patients and healthcare advocacy groups might find it challenging to engage meaningfully due to the technical nature of the content and submission process. Their involvement, however, is crucial for ensuring that drug development aligns with real-world patient needs and experiences.
In summary, while the FDA's initiative to gather public input is commendable, addressing these issues could enhance participation and deepen the impact of the workshop on all stakeholders involved.
Issues
• The document briefly mentions the opening of a public docket to solicit comments but does not specify any budgetary allocations or potential expenses related to the workshop, which makes it difficult to evaluate any spending concerns.
• There is no mention of specific organizations or individuals who might benefit directly from the workshop, reducing the chance of perceived favoritism; however, the lack of participant disclosure may raise concerns.
• The language used to describe the submission process for comments, especially concerning confidential submissions, is dense and could be simplified to enhance understandability for the general public.
• The document refers to regulatory frameworks and laws (e.g., FDARA, PDUFA VI) without providing sufficient context or explanation, potentially limiting comprehension for readers unfamiliar with these terms.
• Technical terms like 'pharmacodynamic markers' and 'mechanistic modeling' are not explained, which might be challenging for individuals without a background in drug development or pharmacology.
• The requirements for different types of submissions (electronic vs. written/paper) are scattered across multiple sections, which could cause confusion for commenters trying to ensure compliance.