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市場調查報告書
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1024810

全球囊性纖維化市場:到 2030 年的藥物分析和預測

Cystic Fibrosis - Global Drug Forecast and Market Analysis to 2030

出版日期: | 出版商: GlobalData | 英文 122 Pages | 訂單完成後即時交付

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  • 簡介
  • 目錄
簡介

七大國家(美國、法國、德國、意大利、西班牙、英國、加拿大)的醫藥市場囊性纖維化 (CF) 市場在預測期內以 4.5% 的複合年增長率增長,收入很高預計將從2020年的82億美元增至2030年的127億美元。市場的主要增長因素包括七個主要國家的人口增長、CF 患者預期壽命延長導致 CF 患者數量持續增長以及高價藥物的推出和普及。

但是,歐盟部分國家和加拿大新CFTR調節劑的贖回延遲,以及粘液溶解劑和吸入劑領域進入CF市場的新產品稀缺,阻礙了市場的增長,存在風險。

本報告調查了七個主要國家(美國、法國、德國、意大利、西班牙、英國和卡)的囊性纖維化市場,重點是到 2030 年的藥物分析和預測。我們提供有關疾病概述、流行病、競爭、未滿足需求和機會評估、研發戰略、管道分析、區域市場分析、市場前景等。

目錄

第 1 章囊性纖維化:執行摘要

  • CF 跨膜電導調節劑調節劑顯著促進 2020 年至 2030 年囊性纖維化市場的增長
  • 主要疾病調節劑對 CF 市場的壟斷
  • 未滿足的需求仍然存在,而非處方 CFTR 調節劑為大多數 CF 患者提供了足夠的治療
  • 管道代理機會
  • 醫生的觀點

第 2 章介紹

  • 催化劑
  • 相關報導
  • 未來報告

第 3 章疾病概述

  • 發病機制和病理生理學
    • 發病機制
    • 病理生理學
    • 生物標誌物/受影響的目標
  • 分類
  • 症狀
  • 預後
  • 生活質量

第 4 章流行病學

  • 疾病背景
  • 風險因素和合併症
  • 世界和過去的趨勢
    • 基於註冊表的 CF 患病率
    • 對低估進行調整的基於註冊表的診斷患病率
  • 預測性調查方法
  • CF 流行病學預測(2020-2030)
  • 討論

第 5 章疾病管理

  • 診療概述
    • 診斷
    • 治療指南和主要處方藥
  • KOL 對疾病管理的見解
    • 與早期診斷和更多治療選擇的輕症 CF 患者建立聯繫
    • 疾病嚴重程度評估
    • 治療方法
    • 從兒童到成人的過渡

第六章競爭評估

  • 概述

第 7 章未滿足需求和機會的評估

  • 概述
  • 開發更有效或更治癒的治療方法
  • 慢性肺部感染治療的發展
  • 粘液溶解療法的發展
  • 提高治療依從性

第八章研發戰略

  • 概述
    • 重新處方策略
    • CFTR 調節劑和疾病修飾療法
    • 抗炎藥的開發
  • 臨床試驗設計
    • 目前的臨床試驗
    • CFTR 調節劑臨床試驗

第 9 章管道評估

  • 概述
  • 臨床開發中的有前途的藥物

第10章管道評估分析

  • 概述
  • 競爭評估
    • CFTR 調製器
    • 抗炎劑

第11章當前和未來的企業

  • 概述
  • 交易趨勢

第 12 章市場展望

  • 世界市場
    • 預測
    • 增長因素和障礙-國際問題
  • 美國
    • 預測
    • 主要活動
  • 歐盟五國
  • 加拿大

第 13 章附錄

目錄
Product Code: GDHC225PIDR

Cystic fibrosis (CF) is an autosomal recessive disease characterized by pancreatic insufficiency and chronic endobronchial airway infection. The disease is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. The most common mutation is the deletion of phenylalanine at codon 508 (known as F508del). Recent progress in the understanding of the disease, increased newborn screening, and discovery of disease-modifying drugs have revolutionized the treatment approach and improved the quality of life and life expectancy of CF patients.

Marketed drugs currently available for CF target multiple aspects of the disease. Inhaled antibiotics are used for the treatment of chronic lung infections, mucolytic agents are used for reducing the viscosity of the mucus in the lungs, pancreatic enzyme replacement therapies (PERTs) are used for the treatment of CF-related Exocrine pancreatic insufficiency (EPI), and CFTR modulators enhance CFTR function, targeting the underlying cause of disease. The disease-modifying nature of CFTR modulators has transformed the treatment paradigm for CF over the last decade, a trend that is likely to persist.

Over the past decade, there have been significant changes in the CF market due to the development of CFTR modulator therapies that target the underlying cause of CF. The most recently launched CFTR modulator, Vertex Therapeutics' Trikafta/Kaftrio, is the first triple drug combination treatment and quickly became the gold standard soon after approval. However, GlobalData is expecting the emergence of a next-generation triple-combination CFTR modulator, Vertex's VX-121 + tezacaftor + VX-561 (deutivacaftor), to take over the market by the end of the forecast period.

GlobalData projects that the global CF market, which comprises the seven major pharmaceutical markets (7MM) (US, France, Germany, Italy, Spain, UK, and Canada), will experience growth during the forecast period, at a Compound Annual Growth Rate (CAGR) of 4.5%, with sales growing from $8.2B in 2020 to $12.7B in 2030.

The greatest drivers of growth in the global CF market include -

  • The continued uptake of high-priced Trikafta/Kaftrio and label expansion to a growing population of CF patients
  • The launch of VX-121 + tezacaftor + VX-561 (deutivacaftor) in 2025 in the US and in 2026 in the 5EU, a high-priced next-generation triple combination.
  • Continued growth of the CF patient population due to overall population growth across the 7MM and increasing life expectancy of CF patients.

The main barrier to growth in the CF market include -

  • Slow reimbursement for novel CFTR modulators in some 5EU countries and Canada.
  • Decreased usage of symptomatic drug classes such antibiotics, mucolytics, and anti-inflammatory drugs due to overall improvements to lung health following use of Trikafta/Kaftrio.
  • Lack of novel products entering the CF market in the mucolytic or inhaled drug class.
  • Key unmet needs include the lack of curative therapies, the need for better antibiotic regimens to fight lung infections, limited choice in mucolytic products, and low patient adherence to treatment.

KEY QUESTIONS ANSWERED

  • Which unmet needs are limiting the treatment of CF in the 7MM?
  • What strategies can the pharmaceutical industry employ to increase treatment rates for CF? How should these strategies differ across different geographical markets?
  • What effect will the launch of generics have on the sales of branded agents?
  • What are the main R&D trends in the CF market and which companies are leading the way?
  • Are there major differences in the mechanisms of action used by therapies in late-stage versus early-stage clinical development?

Scope

  • Overview of CF including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
  • Topline CF market revenue, annual cost of therapy, and major pipeline product sales in the forecast period.
  • Key topics covered include current treatment and pipeline therapies, unmet needs and opportunities, and the drivers and barriers affecting CF therapeutics sales in the 7MM.
  • Pipeline analysis: Comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.
  • Analysis of the current and future market competition in the global CF therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Reasons to Buy

The report will enable you to -

  • Develop and design your in-licensing and out-licensing strategies, using a detailed overview of current pipeline products and technologies to identify companies with the most robust pipelines.
  • Develop business strategies by understanding the trends shaping and driving the global CF therapeutics market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global CF market in the future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
  • Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
  • Track drug sales in the global CF therapeutics market from 2020-2030.
  • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

Table of Contents

Table of Contents

1 Cystic Fibrosis: Executive Summary

  • 1.1 CF Transmembrane Conductance Regulator Modulators Will Drive Substantial Growth in the Cystic Fibrosis Market from 2020-2030
  • 1.2 Vertex's Disease-Modifying Drugs Monopolize the CF Market
  • 1.3 Marketed CFTR Modulators Will Provide Appropriate Treatment for the Majority of the CF Population, but the High Unmet Needs Will Remain
  • 1.4 Opportunity for Pipeline Agents
  • 1.5 What Do Physicians Think?

2 Introduction

  • 2.1 Catalyst
  • 2.2 Related Reports
  • 2.3 Upcoming Reports

3 Disease Overview

  • 3.1 Etiology and Pathophysiology
    • 3.1.1 Etiology
    • 3.1.2 Pathophysiology
    • 3.1.3 Biomarkers/Targets of Interest
  • 3.2 Classification
  • 3.3 Symptoms
  • 3.4 Prognosis
  • 3.5 Quality of Life

4 Epidemiology

  • 4.1 Disease Background
  • 4.2 Risk Factors and Comorbidities
  • 4.3 Global and Historical Trends
    • 4.3.1 Registry-Based Diagnosed Prevalence of CF
    • 4.3.2 Registry-Based Diagnosed Prevalent Cases Adjusted for Underestimation
  • 4.4 Forecast Methodology
    • 4.4.1 Sources
    • 4.4.2 Sources Not Used
    • 4.4.3 Forecast Assumptions and Methods
    • 4.4.4 Registry-Based Diagnosed Prevalent Cases of CF
    • 4.4.5 Registry-Based Diagnosed Prevalent Cases of CF by Specific Mutation
    • 4.4.6 Registry-Based Diagnosed Prevalent Cases of CF with Chronic MRSA Infection
    • 4.4.7 Registry-Based Diagnosed Prevalent Cases Adjusted for Underestimation
  • 4.5 Epidemiological Forecast for CF (2020-2030)
    • 4.5.1 Registry-Based Diagnosed Prevalent Cases of CF
    • 4.5.2 Age-Specific Registry-Based Diagnosed Prevalent Cases of CF
    • 4.5.3 Sex-Specific Registry-Based Diagnosed Prevalent Cases of CF
    • 4.5.4 Registry-Based Diagnosed Prevalent Cases of CF with Specific Mutations
    • 4.5.5 Registry-Based Diagnosed Prevalent Cases of CF with Chronic MRSA Infection
    • 4.5.6 Registry-Based Diagnosed Prevalent Cases of CF Adjusted for Underestimation
    • 4.5.7 Age-Specific Registry-Based Diagnosed Prevalent Cases of CF Adjusted for Underestimation
    • 4.5.8 Sex-Specific Registry-Based Diagnosed Prevalent Cases of CF Adjusted for Underestimation
    • 4.5.9 Registry-Based Diagnosed Prevalent Cases of CF with Specific Mutations Adjusted for Underestimation
    • 4.5.10 Registry-Based Diagnosed Prevalent Cases of CF with Chronic MRSA Infection Adjusted for Underestimation
  • 4.6 Discussion
    • 4.6.1 Epidemiological Forecast Insight
    • 4.6.2 COVID-19 Impact
    • 4.6.3 Limitations of Analysis
    • 4.6.4 Strengths of Analysis

5 Disease Management

  • 5.1 Diagnosis and Treatment Overview
    • 5.1.1 Diagnosis
    • 5.1.2 Treatment Guidelines and Leading Prescribed Drugs
  • 5.2 KOL Insights on Disease Management
    • 5.2.1 Earlier Diagnosis and More Treatment Options Have Led to a CF Patient Population with Milder Disease
    • 5.2.2 Evaluation of Disease Severity
    • 5.2.3 Treatment Approach
    • 5.2.4 Transition from Pediatric to Adult Care

6 Competitive Assessment

  • 6.1 Overview

7 Unmet Needs and Opportunity Assessment

  • 7.1 Overview
  • 7.2 Development of More Effective or Curative Therapies
  • 7.3 Development of Chronic Lung Infection Therapies
  • 7.4 Development of Mucolytic Therapies
  • 7.5 Improvement of Treatment Compliance

8 R&D Strategies

  • 8.1 Overview
    • 8.1.1 Reformulation Strategies
    • 8.1.2 CFTR Modulators and Disease-Modifying Therapies
    • 8.1.3 Development of Anti-Inflammatory Drugs
  • 8.2 Clinical Trials Design
    • 8.2.1 Current Clinical Trials
    • 8.2.2 CFTR Modulator Clinical Trials

9 Pipeline Assessment

  • 9.1 Overview
  • 9.2 Promising Drugs in Clinical Development

10 Pipeline Valuation Analysis

  • 10.1 Overview
  • 10.2 Competitive Assessment
    • 10.2.1 CFTR Modulators
    • 10.2.2 Anti-inflammatory Agents

11 Current and Future Players

  • 11.1 Overview
  • 11.2 Deal-Making Trends

12 Market Outlook

  • 12.1 Global Markets
    • 12.1.1 Forecast
    • 12.1.2 Drivers and Barriers - Global Issues
  • 12.2 US
    • 12.2.1 Forecast
    • 12.2.2 Key Events
  • 12.3 5EU
    • 12.3.1 Forecast
    • 12.3.2 Key Events
  • 12.4 Canada
    • 12.4.1 Forecast
    • 12.4.2 Key Events

13 Appendix

List of Tables

List of Tables

  • Table 1: CF: Key Metrics in the 7MM
  • Table 2: Onset of CF by Approximate Age
  • Table 3: Classification and Criteria of the CFTR Gene Mutations in CF
  • Table 4: Symptoms of CF
  • Table 5: Risk Factors and Comorbidities for CF
  • Table 6: Treatment Guidelines for CF
  • Table 7: Leading Treatments for CF, 2021
  • Table 8: CF Market - Global Drivers and Barriers, 2020-2030
  • Table 9: Key Events Impacting Sales for CF in the US, 2020-2030
  • Table 10: Key Events Impacting Sales for CF in the US, 2020-2030
  • Table 11: Key Events Impacting Sales for CF in Canada, 2020-2030
  • Table 12: High-Prescribing Physicians (non-KOLs) Surveyed, By Country

List of Figures

List of Figures

  • Figure 1: Global (7MM) Sales Forecast by Country for CF in 2020 and 2030
  • Figure 2: Analysis of the Company Portfolio Gap in CF During the Forecast Period
  • Figure 3: Competitive Assessment of the Late-Stage Pipeline Agents that GlobalData Expects to Be Licensed for the Treatment of CF During the Forecast Period
  • Figure 4: CFTR Mutation Classes in CF
  • Figure 5: 7MM, Registry-Based Diagnosed Prevalence of CF, Men and Women, All Ages, 2010-2030 (%)
  • Figure 6: 7MM, Registry-Based Diagnosed Prevalence of CF Adjusted for Underestimation, Men and Women, All Ages, 2010-2030 (%)
  • Figure 7: Sources Used for Registry-Based Diagnosed Prevalent Cases of CF
  • Figure 8: Sources Used for Registry-Based Diagnosed Prevalent Cases of CF by Specific Mutation
  • Figure 9: Sources Used for Registry-Based Diagnosed Prevalent Cases of CF with Chronic MRSA Infection
  • Figure 10: Sources Used for Registry-Based Diagnosed Prevalent Cases of CF Adjusted for Underestimation
  • Figure 11: Registry-Based Diagnosed Prevalent Cases of CF, 7MM, Men and Women, All Ages, 2020
  • Figure 12: 7MM, Age-Specific Registry-Based Diagnosed Prevalent Cases of CF, Men and Women, 2020 (N)
  • Figure 13: 7MM, Sex-Specific Registry-Based Diagnosed Prevalent Cases of CF, All Ages, 2020 (N)
  • Figure 14: 7MM, Registry-Based Diagnosed Prevalent Cases of CF with Specific Mutations, Men and Women, All Ages, 2020 (N)
  • Figure 15: 7MM, Registry-Based Diagnosed Prevalent Cases of CF with Chronic MRSA Infection, Men and Women, All Ages, 2020 (N)
  • Figure 16: Registry-Based Diagnosed Prevalent Cases of CF Adjusted for Underestimation, 7MM, Men and Women, All Ages, 2020
  • Figure 17: 7MM, Age-Specific Registry-Based Diagnosed Prevalent Cases of CF Adjusted for Underestimation, Men and Women, 2020 (N)
  • Figure 18: 7MM, Sex-Specific Registry-Based Diagnosed Prevalent Cases of CF Adjusted for Underestimation, All Ages, 2020 (N)
  • Figure 19: 7MM, Registry-Based Diagnosed Prevalent Cases of CF with Mutations Adjusted for Underestimation, Men and Women, All Ages, 2020 (N)
  • Figure 20: 7MM, Registry-Based Diagnosed Prevalent Cases of CF with Chronic MRSA Infection Adjusted for Underestimation, Men and Women, All Ages, 2020 (N)
  • Figure 21: Genetics of CF
  • Figure 22: Unmet Needs and Opportunities in CF
  • Figure 23: Overview of the Development Pipeline (Phase I and II) in CF
  • Figure 24: Key Late-Stage Trials for the Promising Pipeline Agents that GlobalData Expects to Be Licensed for CF in the 7MM During the Forecast Period
  • Figure 25: Competitive Assessment of Pipeline CFTR Modulators Benchmarked Against the SOCs, Kalydeco and Trikafta/Kaftrio
  • Figure 26: Competitive Assessment of the Pipeline Anti-inflammatory Drug, LAU-7b, Benchmarked Against the SOC, Azithromycin
  • Figure 27: Analysis of the Company Portfolio Gap in CF During the Forecast Period
  • Figure 28: Global (7MM) Sales Forecast by Country for CF in 2020 and 2030
  • Figure 29: Sales Forecast by Class for CF in the US in 2020 and 2030
  • Figure 30: Sales Forecast by Class for CF in the 5EU in 2020 and 2030
  • Figure 31: Sales Forecast by Class for CF in Canada in 2020 and 2030