画期的治療法：市場力学と投資機会

Report Highlights

The global market for breakthrough therapy drugs was valued at $133.3 billion in 2023. This market is expected to grow from $150.6 billion in 2024 to $287.5 billion by 2029, at a compound annual growth rate (CAGR) of 13.8% from 2024 to 2029.

Summary:

BTD is a procedure intended to expedite the development and evaluation of medications and biologics intended for treating severe illnesses, provided that primary clinical evidence indicates that the drug significantly improves upon current therapy on a clinically meaningful endpoint. By providing additional FDA support and assistance and accelerating the release of medications to the public, BTD enables pharmaceutical companies to expedite the development process. Fast-track designation, accelerated approval, and priority review are significant tools that have been used for many years in addition to BTD. These all lean toward authorizing medications used to treat severe illnesses. The development process,

consisting of three stages of extensive and controlled trials, requires standard clinical testing even though these procedures can shorten a drug’s time to market.

The market for breakthrough therapy drugs is driven by the need for pipeline drugs to be developed quickly and the rising incidence of life-threatening illnesses. The main driver is the need for efficient treatments for severe conditions. Because of the faster market access and greater returns on investment, pharmaceutical companies pay special attention to medications classified as breakthrough therapies. A direct result of their market designation is that breakthrough therapy medications frequently go through less-thorough clinical trials. The market for breakthrough therapy medications is further boosted by the FDA’s improved support for small-scale industries in R&D, including more funding

and accelerated drug approval procedures.

Report Scope

This report examines the potential risks and benefits of receiving breakthrough therapy designation (BTD), compares and contrasts fast-track drug development approaches (logistics, criteria and implications), and highlights the opportunities and challenges of creating breakthrough therapies. Cardiovascular, central nervous systems and neurology, rare diseases, oncology and other diseases are among the therapy areas in which this report analyzes BTD. It also examines leading companies, approved and pending products, and product revenues. It examines BTD’s achievements and shortcomings and discusses the tactics organizations use to optimize BTD’s competitive edge.

To provide an in-depth understanding of the market, this report also includes profiles of market participants, the competitive landscape, key competitors and respective market share.

The market in this report is divided into four regions: North America, Europe, Asia-Pacific and Rest of the World (RoW). For market estimates, data has been provided for 2023 as the base year, with forecasts for 2024 through 2029.

Report Synopsis

Report Includes

– 26 data tables and 49 additional tables

– An overview of the global market dynamics and investment opportunities for breakthrough therapy designation (BTD) drugs

– Analyses of the global market trends, with sales data for 2021-2023, estimates for 2024, and projections of compound annual growth rates (CAGRs) through 2029

– Evaluation and forecast the market size for breakthrough therapies based on type, and geographical region

– Discussion of emerging technologies and developments and various macroeconomic factors affecting the breakthrough therapies market

– Review of patent data and new developments regarding various categories and sub-categories of breakthrough therapies

– A discussion on ESG challenges and practices of the industry

– Assessment of the vendor landscape, including the market shares of leading companies, their product portfolios and financial overviews

– Information on recent mergers, acquisitions, expansions, collaborations, investments, divestments, product launches, and other strategic developments

– Profiles of the leading market vendors, including F. Hoffmann-La Roche Ltd., Merck & Co. Inc., Bristol-Myers Squibb Co., Novartis AG and AstraZeneca

Table of Contents

Chapter 1 Executive Summary

Market Outlook

Scope of Report

Market Summary

Chapter 2 Market Overview

Overview

Porter’s Five Forces Analysis

Potential for New Entrants: High

Bargaining Power of Suppliers: Medium

Bargaining Power of Buyers: Medium

Threat of Substitute Products: High

Industry Competition: High

PESTEL Analysis of the Pharmaceutical Industry

Political Factors

Economic Factors

Social Factors

Technological Factors

Environmental Factors

Legal Factors

Impact of Breakthrough Therapy Drugs on Business Growth

Chapter 3 Market Dynamics

Market Dynamics

Market Drivers

More Drugs Gaining Breakthrough Status

Expedited Approval Process in the EU

Increasing Prevalence of Life-Threatening Conditions

Market Restraints

Increasing Preference for Generics

Market Opportunities

Rising Use of Technologies in Clinical Trials

Chapter 4 Emerging Trends

Overview

Nanotechnology: Enhancing Drug Discovery and Delivery

Merging Technologies in Drug Discovery

Conclusion

Chapter 5 Regulatory Landscape

Overview

Policy

Procedures

CDER-Sponsor Meetings and Other Communications

CDER Internal Meetings and Communications

CDER Review of Breakthrough Therapy Drug Development Programs

Rescinding a Breakthrough Therapy Designation

Chapter 6 Market Segmentation Analysis

Segmentation Breakdown

Market Breakdown by Therapy

Cardiovascular

CNS and Neurology

Infectious Diseases

Oncology

Rare Diseases

Other Diseases

Geographic Breakdown

Market Breakdown by Region

North America

Europe

Asia-Pacific

Rest of the World

Chapter 7 Competitive Intelligence

Overview

PRIME Designation Lists

Global Ranks of Leading Companies

Chapter 8 Sustainability in the BDT Market: An ESG Perspective

Role of ESG in the Pharma Industry

Environmental

Social

Governance

ESG Risk Ratings

Conclusion

Chapter 9 Appendix

Methodology

Abbreviations

Sources

Company Profiles

ABBVIE INC.

ASTRAZENECA

BRISTOL-MYERS SQUIBB CO.

HOFFMANN-LA ROCHE LTD.

JOHNSON & JOHNSON SERVICES INC.

MERCK & CO. INC.

NOVARTIS AG

PFIZER INC.

REGENERON PHARMACEUTICALS INC.

VERTEX PHARMACEUTICALS INC.

List of Tables

Summary Table : Global Market for Breakthrough Therapies, by Therapy, Through 2029

Table 1 : CBER Breakthrough Therapy Designation Requests Received, by Fiscal Year, 2012–2024

Table 2 : CDER Breakthrough Therapy Designation Requests Received, by Fiscal Year, 2012–2024

Table 3 : IND Submissions with Regulatory-Mandated or FDA-Established Timelines

Table 4 : Global Market for Breakthrough Therapies, by Therapy, Through 2029

Table 5 : Breakthrough Therapy Drugs for Cardiovascular Disease

Table 6 : Global Market for Breakthrough Therapies for CVD, by Region, Through 2029

Table 7 : Breakthrough Therapy Drugs for CNS and Neurology

Table 8 : Global Market for Breakthrough Therapies for CNS and Neurology, by Region, Through 2029

Table 9 : Selected Breakthrough Therapy Drugs for Infectious Diseases

Table 10 : Global Market for Breakthrough Therapies for Infectious Diseases, by Region, Through 2029

Table 11 : Selected Breakthrough Therapy Drugs for Oncology

Table 12 : Global Market for Breakthrough Therapies for Oncology, by Region, Through 2029

Table 13 : Selected Breakthrough Therapy Drugs for Rare Diseases

Table 14 : Global Market for Breakthrough Therapies for Rare Diseases, by Region, Through 2029

Table 15 : Selected Breakthrough Therapy Drugs for Other Diseases

Table 16 : Global Market for Breakthrough Therapies for Other Diseases, by Region, Through 2029

Table 17 : Global Market for Breakthrough Therapies, by Region, Through 2029

Table 18 : North American Market for Breakthrough Therapies, by Therapy, Through 2029

Table 19 : North American Market for Breakthrough Therapies, by Country, Through 2029

Table 20 : European Market for Breakthrough Therapies, by Therapy, Through 2029

Table 21 : European Market for Breakthrough Therapies, by Country, Through 2029

Table 22 : Asia-Pacific Market for Breakthrough Therapies, by Therapy, Through 2029

Table 23 : Asia-Pacific Market for Breakthrough Therapies, by Country, Through 2029

Table 24 : RoW Market for Breakthrough Therapies, by Therapy, Through 2029

Table 25 : CDER Breakthrough Therapy Approvals, 2024

Table 26 : CBER Breakthrough Therapy Approvals, 2024

Table 27 : CDER Breakthrough Therapy Approvals, 2023

Table 28 : CBER Breakthrough Therapy Approvals, 2023

Table 29 : Medicines Granted PRIME Eligibility in 2024

Table 30 : Medicines Granted PRIME Eligibility in 2023

Table 31 : Ranks of Leading Pharmaceutical Manufacturers

Table 32 : ESG Risk Ratings and Rankings for Pharmaceutical Companies, 2024*

Table 33 : Abbreviations Used in this Report

Table 34 : Information Sources in this Report

Table 35 : AbbVie Inc.: Company Snapshot

Table 36 : AbbVie Inc.: Financial Performance, FY 2022 and 2023

Table 37 : AbbVie Inc.: Product Portfolio

Table 38 : AbbVie Inc: News/Key Developments, 2021–2023

Table 39 : AstraZeneca: Company Snapshot

Table 40 : AstraZeneca: Financial Performance, FY 2022 and 2023

Table 41 : AstraZeneca: Product Portfolio

Table 42 : AstraZeneca: News/Key Developments, 2022–2024

Table 43 : Bristol-Myers Squibb Co.: Company Snapshot

Table 44 : Bristol-Myers Squibb Co.: Financial Performance, FY 2022 and 2023

Table 45 : Bristol-Myers Squibb Co.: Product Portfolio

Table 46 : Bristol-Myers Squibb Co.: News/Key Developments, 2023

Table 47 : F. Hoffmann-La Roche Ltd.: Company Snapshot

Table 48 : F. Hoffmann-La Roche Ltd.: Financial Performance, FY 2022 and 2023

Table 49 : F. Hoffmann-La Roche Ltd.: Product Portfolio

Table 50 : F. Hoffmann-La Roche Ltd.: News/Key Developments, 2022–2024

Table 51 : Johnson & Johnson Services Inc.: Company Snapshot

Table 52 : Johnson & Johnson Services Inc.: Financial Performance, FY 2022 and 2023

Table 53 : Johnson & Johnson Services Inc.: Product Portfolio

Table 54 : Johnson & Johnson Services Inc.: News/Recent Developments, 2022–2024

Table 55 : Merck & Co. Inc.: Company Snapshot

Table 56 : Merck & Co. Inc.: Financial Performance, FY 2022 and 2023

Table 57 : Merck & Co. Inc.: Product Portfolio

Table 58 : Merck & Co. Inc.: News/Key Developments, 2021–2024

Table 59 : Novartis AG: Company Snapshot

Table 60 : Novartis AG: Financial Performance, FY 2022 and 2023

Table 61 : Novartis AG: Product Portfolio

Table 62 : Novartis AG: News/Key Developments, 2023 and 2024

Table 63 : Pfizer Inc.: Company Snapshot

Table 64 : Pfizer Inc.: Financial Performance, FY 2022 and 2023

Table 65 : Pfizer Inc.: Product Portfolio

Table 66 : Pfizer Inc.: News/Key Developments, 2023 and 2024

Table 67 : Regeneron Pharmaceuticals Inc.: Company Snapshot

Table 68 : Regeneron Pharmaceuticals Inc.: Financial Performance, FY 2022 and 2023

Table 69 : Regeneron Pharmaceuticals Inc.: Product Portfolio

Table 70 : Regeneron Pharmaceuticals Inc.: News/Key Developments, 2022–2023

Table 71 : Vertex Pharmaceuticals Inc.: Company Snapshot

Table 72 : Vertex Pharmaceuticals Inc.: Financial Performance, FY 2022 and 2023

Table 73 : Vertex Pharmaceuticals Inc.: Product Portfolio

Table 74 : Vertex Pharmaceuticals Inc.: News/Key Developments, 2022–2024

List of Figures

Summary Figure : Global Market for Breakthrough Therapies, by Therapy, 2021–2029

Figure 1 : Market Dynamics of Breakthrough Therapies

Figure 2 : CBER Breakthrough Therapy Designation Requests Received, by Fiscal Year, 2013-2024

Figure 3 : CDER Breakthrough Therapy Designation Requests Received, by Fiscal Year, 2012–2024

Figure 4 : Global Market Shares of Breakthrough Therapies, by Therapy, 2023

Figure 5 : Worldwide Deaths from Cardiovascular Diseases, by Region, 2000 and 2021

Figure 6 : Worldwide Deaths from Cardiovascular Diseases, by Type, 2021

Figure 7 : Global Market Shares of Breakthrough Therapies for CVD, by Region, 2023

Figure 8 : Global Burden of Conditions Affecting the Nervous System, 2021

Figure 9 : Global Market Shares of Breakthrough Therapies for CNS and Neurology, by Region, 2023

Figure 10 : Global Market Shares of Breakthrough Therapies for Infectious Diseases, by Region, 2023

Figure 11 : Share of Global Incidence Cases of All Cancer Types, by WHO Region, 2022

Figure 12 : Cancer Incidence in the U.S., 2000–2021

Figure 13 : Global Market Shares of Breakthrough Therapies for Oncology, by Region, 2023

Figure 14 : Global Market Shares of Breakthrough Therapies for Rare Diseases, by Region, 2023

Figure 15 : Global Market Shares of Breakthrough Therapies for Other Diseases, by Region, 2023

Figure 16 : Global Market Shares of Breakthrough Therapies, by Region, 2023

Figure 17 : North American Market for Breakthrough Therapies, by Therapy, 2021–2029

Figure 18 : North American Market Shares of Breakthrough Therapies, by Country, 2023

Figure 19 : European Market for Breakthrough Therapies, by Therapy, 2021–2029

Figure 20 : European Market Shares of Breakthrough Therapies, by Country, 2023

Figure 21 : Asia-Pacific Market for Breakthrough Therapies, by Therapy, 2021–2029

Figure 22 : Asia-Pacific Market Shares of Breakthrough Therapies, by Country, 2023

Figure 23 : RoW Market for Breakthrough Therapies, by Therapy, 2021–2029

Figure 24 : PRIME Recommendations Granted, by Therapeutic Area, Till 2024

Figure 25 : AbbVie Inc.: Revenue Share, by Business Unit, FY 2023

Figure 26 : AbbVie Inc.: Revenue Share, by Country/Region, FY 2023

Figure 27 : AstraZeneca: Revenue Share, by Business Unit, FY 2023

Figure 28 : AstraZeneca: Revenue Share, by Region/Country, FY 2023

Figure 29 : Bristol-Myers Squibb Co.: Revenue Share, by Business Unit, FY 2023

Figure 30 : Bristol-Myers Squibb Co.: Revenue Share, by Country/Region, FY 2023

Figure 31 : F. Hoffmann-La Roche Ltd.: Revenue Share, by Business Unit, FY 2023

Figure 32 : F. Hoffmann-La Roche Ltd.: Revenue Share, by Country/Region, FY 2023

Figure 33 : Johnson & Johnson Services Inc.: Revenue Share, by Business Unit, FY 2023

Figure 34 : Johnson & Johnson Services Inc.: Revenue Share, by Region/Country, FY 2023

Figure 35 : Merck & Co. Inc.: Revenue Share, by Business Unit, FY 2023

Figure 36 : Merck & Co. Inc.: Revenue Share, by Country/Region, FY 2023

Figure 37 : Novartis AG: Revenue Share, by Business Unit, FY 2023

Figure 38 : Novartis AG: Revenue Share, by Country/Region, FY 2023

Figure 39 : Pfizer Inc.: Revenue Share, by Business Unit, FY 2023

Figure 40 : Pfizer Inc.: Revenue Share, by Country/Region, FY 2023

Figure 41 : Regeneron Pharmaceuticals Inc.: Revenue Share, by Business Unit, FY 2023

Figure 42 : Regeneron Pharmaceuticals Inc.: Revenue Share, by Country/Region, FY 2023

Figure 43 : Vertex Pharmaceuticals Inc.: Revenue Share, by Business Unit, FY 2023

Figure 44 : Vertex Pharmaceuticals Inc.: Revenue Share, by Country/Region, FY 2023

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