Drug Development for Rare Diseases

Rare diseases affect 30 million Americans, posing challenges for drug developers. This book provides an overview of common issues, strategies, and data utilization in clinical development.

Format: Hardback
Length: 222 pages
Publication date: 13 February 2023
Publisher: Taylor & Francis Ltd

Rare diseases are those that affect a limited number of people, with a prevalence of fewer than 200,000 in the United States. According to estimates, there are over 7,000 rare diseases, impacting approximately 30 million Americans, or 10% of the US population. This diverse and complex disease area presents significant challenges for patients, caregivers, regulators, drug developers, and other stakeholders.

This book aims to provide an overview of the common issues faced by rare disease drug developers, summarize the unique challenges associated with clinical development in small populations, discuss drug development strategies in the evolving regulatory environment, explain the generation and utilization of different data and evidence within and beyond clinical trials, and showcase recent examples that illustrate these challenges and the development strategies employed to address them.

Key Features:

Rare Disease: This book focuses on rare diseases, which are conditions that affect a small percentage of the population. It provides an in-depth understanding of the unique challenges and considerations associated with developing treatments for these conditions.

Drug Development: The book discusses the drug development process for rare diseases, including the identification of therapeutic targets, preclinical testing, clinical trials, regulatory approval, and post-marketing surveillance. It highlights innovative clinical trial design strategies and the use of real-world evidence to support drug development.

Innovative Clinical Trial Design: The book explores innovative clinical trial design approaches tailored to the needs of rare disease populations. It discusses the challenges of recruiting participants, conducting trials in small populations, and ensuring the validity and reliability of trial results. It also discusses the use of surrogate markers, patient-reported outcomes, and other data sources to evaluate the efficacy and safety of treatments.

Regulatory Approval: The book discusses the regulatory framework for rare disease drug development, including the approval process, the criteria for approval, and the challenges faced by drug developers in obtaining regulatory approval. It highlights the importance of engaging with regulatory agencies early in the drug development process and the use of regulatory strategies to optimize the development and approval of treatments.

Real-World Evidence: The book emphasizes the importance of real-world evidence in supporting drug development for rare diseases. It discusses the collection, analysis, and utilization of data from patients outside of clinical trials to inform drug development decisions and improve patient outcomes. It also highlights the challenges of collecting and analyzing real-world data and the need for collaboration between stakeholders to facilitate the generation and utilization of real-world evidence.

Recent Examples: The book provides examples of successful drug development strategies for rare diseases, including targeted therapies, gene therapies, and precision medicine approaches. It showcases recent clinical trials and regulatory approvals that have led to the development and approval of new treatments for rare diseases.

By providing a comprehensive overview of the challenges and opportunities in rare disease drug development, this book aims to assist stakeholders in the field in developing effective treatments and improving the lives of patients affected by these conditions.

Weight: 590g
Dimension: 234 x 156 (mm)
ISBN-13: 9780367518349