Drug Development in Rare Diseases: Focus on Clinical Trials and...

Drug Development in Rare Diseases: Focus on Clinical Trials and the Regulatory Landscape

Report Code: PHM243A

Publish Date: Feb 2021

Publisher: BCC Publishing

Category: Pharmaceuticals

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Report Highlights

The orphan drug industry is gaining importance as a significant and rewarding market in the pharmaceutical and biotech sectors. Technological and scientific innovations in specific drug discovery pathways as well as major developments in genetics are cultivating growth in this industry. BCC Research’s objective in conducting this report is to provide detailed information on drug development in rare diseases. This report provides a comprehensive analysis and examines the future direction of these drugs as an important means for the treatment of orphan and rare diseases.

Report Includes

  • An overview of the drug development in rare diseases with special focus on clinical trial and regulatory landscape
  • Detailed description of different kinds of orphan drugs and a comparative assessment on biological and chemical or non-biological orphan drugs
  • Discussion on Orphan Drug Act; insights into general framework for the regulation of drugs and biologics, and regulations for clinical trials of orphan designated drugs
  • Highlights of technological advances and innovations in genetic manufacturing technologies and information on terms such as generic competition, patent expirations, premium pricing and information on orphan drug designation applications

Report Scope

This report analyzes the regulatory framework for the clinical trials of orphan drugs in rare and orphan diseases. The report also analyzes the trends and characteristics of clinical trials conducted on rare diseases from 1999-2018, using information from various sources. This report covers an overview of the global orphan drugs market and its competitive landscape.

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BCC Research Team possesses expertise and experience in life and physical science domains. They specialize in offering valuable business insights, including industry analysis, competitor intelligence, strategic and financial analysis, and opportunity assessment. The team has in-depth knowledge of various sectors, including healthcare, biotechnology, pharmaceuticals, IT, automation, advanced materials, and energy. They are proficient in qualitative and quantitative market intelligence providing clients with actionable insights. With a vast understanding of the competitive landscape, the team can support clients in making data-driven decisions to help them achieve a competitive edge in their respective markets.

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Table of Contents

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Full Report: Drug Development in Rare Diseases: Focus on Clinical Trials and the Regulatory Landscape107Free

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This is an Excel-only version of the report.

Report Includes

    Total Number of Tables in this Report: 16

  • Table 1: Rare Diseases and their Prevalence
  • Table 2: List of Documents Included in the Orphan Designation Application
  • Table 3: Summary of Orphan Designation in Europe
  • Table 4: Overview of Organizations and Responsibilities in Japan
  • Table 5: Comparison of the Regulation of Rare Diseases and Orphan Drugs Worldwide
  • Table 6: Characteristics of the NCT, EUCTR and JPRN Trials
  • Table 7: Number of Trials in NCT, EUCTR and JPRN Trials, by Recruitment Status
  • Table 8: Number of Trials in NCT, EUCTR and JPRN Trials, by Gender
  • Table 9: Number of Trials in NCT, EUCTR and JPRN Trials, by Phase
  • Table 10: Number of Trials in NCT, EUCTR and JPRN Trials, by Country
  • Table 11: Top 20 Most Studied Rare Diseases, by Number of Trials
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Drug Development in Rare Diseases: Focus on Clinical Trials and the Regulatory Landscape
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