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嚢胞性線維症(CF):2030年までの疫学予測

Cystic Fibrosis (CF) - Epidemiology Forecast - 2030

出版日: | 発行: DelveInsight Business Research LLP | ページ情報: 英文 100 Pages | 納期: 即日から翌営業日

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嚢胞性線維症(CF):2030年までの疫学予測
出版日: 2020年08月01日
発行: DelveInsight Business Research LLP
ページ情報: 英文 100 Pages
納期: 即日から翌営業日
  • 全表示
  • 概要
  • 図表
  • 目次
概要

嚢胞性線維症(CF)は、肺と消化器系に損傷を引き起こす常染色体劣性遺伝性疾患で、欧州、 北米、オーストラリアで有病率が高くなっています。CFの原因は、嚢胞性線維症膜コンダクタンス制御因子(CFTR)の遺伝子変異です。 CFTRタンパク質は、ABCトランスポータークラスに属する塩化物伝導膜貫通コンダクタンス制御因子であり、塩化物イオンを運ぶことで電気化学的勾配や通路内の浸透圧と流体のバランスを維持します。遺伝子の常染色体劣性突然変異によるCFTRタンパク質の欠陥は、広範囲にわたる衰弱をもたらす可能性があります。最も一般的な欠陥は、気道を閉塞する高粘度の粘液で、呼吸が困難になるほか、黄色ブドウ球菌、インフルエンザ菌、緑膿菌などさまざまな細菌感染症の繁殖地としても機能します。 CFTRは体の他の部分にも存在しているため、変異の結果として、吸収不良、膵機能障害、不妊症、腸閉塞などを引き起こすことがあります。

当レポートは、主要7ヶ国(米国、ドイツ、フランス、イタリア、スペイン、英国、日本)の2030年までのCFの疫学について展望したもので、疾病の概要、疫学と患者数、国別の疫学、現在行われている治療、未対応のニーズなどの情報を提供しています。

目次

第1章 主な調査結果

第2章 エグゼクティブサマリー

第3章 組織

第4章 疫学と市場調査の手法

第5章 市場の概要

  • 2017年の市場シェア
  • 2030年の市場シェア

第6章 疾病の概要

  • イントロダクション
  • 嚢胞性線維症の分類
  • 病態生理
  • 臨床的兆候と症状
  • 診断

第7章 疫学と患者人口

  • 主な調査結果
  • 主要6ヶ国の症例
  • 米国
    • 仮定と根拠
    • 有病率の合計
    • 性別の症例
    • 年齢別の症例
    • タイプ固有の症例
  • 欧州5ヶ国
  • ドイツ
  • フランス
  • イタリア
  • スペイン
  • 英国

第8章 嚢胞性線維症の治療

  • 医薬品
  • 食事と運動
  • 気道クリアランス技術
  • 遺伝子治療
  • 手術

第9章 主な専門家の見解

第10章 付録

  • 調査手法

章11:DelveInsightのサービス内容

第12章 免責事項

第13章 DelveInsightについて

図表

List of Tables

  • Table 1: Cystic Fibrosis Diagnosis - Clinical care guidelines
  • Table 2: Total cases of Cystic Fibrosis in 6MM (2017-2030)
  • Table 3: Total Prevalent cases of Cystic Fibrosis in the United States (2017-2030)
  • Table 4: Gender-specific cases of Cystic Fibrosis in the US (2017-2030)
  • Table 5: Age-specific cases of Cystic Fibrosis in the US (2017-2030)
  • Table 6: Type-specific cases of Cystic Fibrosis in the US (2017-2030)
  • Table 7: Total Prevalent cases of Cystic Fibrosis in Germany (2017-2030)
  • Table 8: Gender-specific cases of Cystic Fibrosis in Germany (2017-2030)
  • Table 9: Age-specific cases of Cystic Fibrosis in Germany (2017-2030)
  • Table 10: Type-specific cases of Cystic Fibrosis in Germany (2017-2030)
  • Table 11: Total Prevalent cases of Cystic Fibrosis in France (2017-2030)
  • Table 12: Gender-specific cases of Cystic Fibrosis in France (2017-2030)
  • Table 13: Age-specific cases of Cystic Fibrosis in France (2017-2030)
  • Table 14: Type-specific cases of Cystic Fibrosis in France (2017-2030)
  • Table 15: Total Prevalent cases of Cystic Fibrosis in Italy (2017-2030)
  • Table 16: Gender-specific cases of Cystic Fibrosis in Italy (2017-2030)
  • Table 17: Age-specific cases of Cystic Fibrosis in Italy (2017-2030)
  • Table 18: Type-specific cases of Cystic Fibrosis in Italy (2017-2030)
  • Table 19: Total Prevalent cases of Cystic Fibrosis in Spain (2017-2030)
  • Table 20: Gender-specific cases of Cystic Fibrosis in Spain (2017-2030)
  • Table 21: Age-specific cases of Cystic Fibrosis in Spain (2017-2030)
  • Table 22: Type-specific cases of Cystic Fibrosis in Spain (2017-2030)
  • Table 23: Total Prevalent cases of Cystic Fibrosis in the United Kingdom (2017-2030)
  • Table 24: Gender-specific cases of Cystic Fibrosis in the UK (2017-2030)
  • Table 25: Age-specific cases of Cystic Fibrosis in the UK (2017-2030)
  • Table 26: Type-specific cases of Cystic Fibrosis in the UK (2017-2030)

List of Figures

  • Figure 1: Epidemiology and Market Methodology
  • Figure 2: Classification of CFTR mutations according to their clinical consequences
  • Figure 3: ACMG recommended panel of 23 classic CF-causing mutations. These mutations include missense, stop, splicing, and frameshift mutations
  • Figure 4: Classes of CFTR mutation
  • Figure 5: The multitude effects of Cystic Fibrosis on the body
  • Figure 6: Age-related presentations of cystic fibrosis
  • Figure 7: Causes of false-positive test results
  • Figure 8: Nasal potential difference tracking in subjects with cystic fibrosis (left) and in a healthy individual (right)
  • Figure 9: Hierarchy for the diagnosis of cystic fibrosis.
  • Figure 10: Total cases of Cystic Fibrosis in 6MM (2017-2030)
  • Figure 11: Total Prevalent cases of Cystic Fibrosis in the United States (2017-2030)
  • Figure 12: Gender-specific cases of Cystic Fibrosis in the US (2017-2030)
  • Figure 13: Age-specific cases of Cystic Fibrosis in the US (2017-2030)
  • Figure 14: Type-specific cases of Cystic Fibrosis in the US (2017-2030)
  • Figure 15: Total Prevalent cases of Cystic Fibrosis in Germany (2017-2030)
  • Figure 16: Gender-specific cases of Cystic Fibrosis in Germany (2017-2030)
  • Figure 17: Age-specific cases of Cystic Fibrosis in Germany (2017-2030)
  • Figure 18: Type-specific cases of Cystic Fibrosis in Germany (2017-2030)
  • Figure 19: Total Prevalent cases of Cystic Fibrosis in France (2017-2030)
  • Figure 20: Gender-specific cases of Cystic Fibrosis in France (2017-2030)
  • Figure 21: Age-specific cases of Cystic Fibrosis in France (2017-2030)
  • Figure 22: Type-specific cases of Cystic Fibrosis in France (2017-2030)
  • Figure 23: Total Prevalent cases of Cystic Fibrosis in Italy (2017-2030)
  • Figure 24: Gender-specific cases of Cystic Fibrosis in Italy (2017-2030)
  • Figure 25: Age-specific cases of Cystic Fibrosis in Italy (2017-2030)
  • Figure 26: Type-specific cases of Cystic Fibrosis in Italy (2017-2030)
  • Figure 27: Total Prevalent cases of Cystic Fibrosis in Spain (2017-2030)
  • Figure 28: Gender-specific cases of Cystic Fibrosis in Spain (2017-2030)
  • Figure 29: Age-specific cases of Cystic Fibrosis in Spain (2017-2030)
  • Figure 30: Type-specific cases of Cystic Fibrosis in Spain (2017-2030)
  • Figure 31: Total Prevalent cases of Cystic Fibrosis in the United Kingdom (2017-2030)
  • Figure 32: Gender-specific cases of Cystic Fibrosis in the UK (2017-2030)
  • Figure 33: Age-specific cases of Cystic Fibrosis in the UK (2017-2030)
  • Figure 34: Type-specific cases of Cystic Fibrosis in the UK (2017-2030)
目次
Product Code: DIEI0271

DelveInsight's 'Cystic Fibrosis (CF)-Epidemiology Forecast-2030' report delivers an in-depth understanding of the Cystic Fibrosis (CF), historical and forecasted epidemiology in the United States, and EU5 (Germany, Spain, Italy, France, and the United Kingdom).

Cystic Fibrosis (CF) Disease Understanding

First identified nearly 80 years ago, cystic fibrosis (CF) is an autosomal recessive genetic disorder that causes damage to the lungs and the digestive system with the highest prevalence in Europe, North America, and Australia. A genetic mutation causes this disease in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The CFTR protein is a chloride-conducting trans-membrane conductance regulator belonging to the ABC transporter class. It helps in the transportation of chloride ions, thereby maintaining the electrochemical gradient as well as osmotic and fluid balance in the passageways.

A defect in CFTR protein due to an autosomal recessive mutation in the gene can have a wide range of debilitating consequences. The most common defect is highly viscous mucus that obstructs airway tracts which leads to difficulty in breathing. Moreover, it serves as a breeding ground for a huge range of bacterial infections like Staphylococcus aureus, Haemophilus influenza, and Pseudomonas aeruginosa. CFTR is present in other parts of the body as well; therefore, other consequences of such a mutation include malabsorption, pancreatic dysfunction, infertility, bowel obstruction, among many others.

Cystic Fibrosis (CF) Epidemiology

The Cystic Fibrosis (CF) epidemiology division provides insights about the historical and current patient pool along with the forecasted trend for every seven major countries. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings

The total prevalent cases of Cystic Fibrosis (CF) patients are increasing in 6MM during the study period, i.e. 2017-2030.

The disease epidemiology covered in the report provides historical as well as forecasted Cystic Fibrosis (CF) symptoms epidemiology segmented as the Total Prevalent cases of Cystic Fibrosis (CF), Gender-specific cases of Cystic Fibrosis (CF), Age-specific cases of Cystic Fibrosis (CF), Type-specific cases of Cystic Fibrosis (CF). The report includes the prevalent scenario of Cystic Fibrosis (CF) symptoms in 6MM covering the United States and EU5 countries (Germany, France, Italy, Spain, and the United Kingdom) from 2017 to 2030.

Country-wise Cystic Fibrosis (CF) Epidemiology

The epidemiology segment also provides the Cystic Fibrosis (CF) epidemiology data and findings across the United States, and EU5 (Germany, France, Italy, Spain, and the United Kingdom).

The total prevalent cases of Cystic Fibrosis (CF) associated in 6MM countries was 61,306 in 2017.

  • As per the estimates, the United States has the largest prevalent population of Cystic Fibrosis (CF).
  • Among the EU5 countries, the UK had the highest prevalent cases of Cystic Fibrosis (CF), followed by France. On the other hand, Spain had the lowest prevalent cases with 2,075 cases in 2017.

Scope of the Report:

  • The Cystic Fibrosis (CF) report covers a detailed overview explaining its causes, symptoms, classification, pathophysiology, diagnosis and treatment patterns.
  • The Cystic Fibrosis (CF) Epidemiology Report and Model provide an overview of the risk factors and global trends of Cystic Fibrosis (CF) in the six major markets (6MM: US, France, Germany, Italy, Spain, and the UK).
  • The report provides insight about the historical and forecasted patient pool of Cystic Fibrosis (CF) in six major markets covering the United States, and EU5 (Germany, Spain, France, Italy, UK).
  • The report helps witness the growth opportunities in the 6MM with respect to the patient population.
  • The report assesses the disease risk and burden and highlights the unmet needs of Cystic Fibrosis (CF).
  • The report provides the segmentation of the Cystic Fibrosis (CF) epidemiology by Prevalent Cases of Cystic Fibrosis (CF) in 6MM.
  • The report provides the segmentation of the Cystic Fibrosis (CF) epidemiology by Gender-specific Prevalent Cases of Cystic Fibrosis (CF) in 6MM.
  • The report provides the segmentation of the Cystic Fibrosis (CF) epidemiology by Age-specific Prevalent Cases of Cystic Fibrosis (CF) in 6MM.
  • The report provides the segmentation of the Cystic Fibrosis (CF) epidemiology by Type-specific Prevalent Cases of Cystic Fibrosis (CF) in 6MM.

Report Highlights:

  • 11-year Forecast of Cystic Fibrosis (CF) epidemiology
  • 6MM Coverage
  • Total Prevalent Cases of Cystic Fibrosis (CF)
  • Prevalent Cases according to segmentation: Gender-specific cases of Cystic Fibrosis (CF)
  • Prevalent Cases according to segmentation: Age-specific cases of Cystic Fibrosis (CF)
  • Prevalent Cases according to segmentation: Type-specific cases of Cystic Fibrosis (CF)

KOL-Views

We interview, KOLs and SME's opinion through primary research to fill the data gaps and validate our secondary research. The opinion helps understand the total patient population and current treatment pattern. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the indications.

Key Questions Answered

  • What will be the growth opportunities in the 6MM with respect to the patient population pertaining to Cystic Fibrosis (CF)?
  • What are the key findings about the Cystic Fibrosis (CF) epidemiology across 6MM and which country will have the highest number of patients during the forecast period (2017-2030)?
  • What would be the total number of patients of Cystic Fibrosis (CF) across the 6MM during the forecast period (2017-2030)?
  • Among the EU5 countries, which country will have the highest number of patients during the forecast period (2017-2030)?
  • At what CAGR the patient population is expected to grow in 6MM during the forecast period (2017-2030)?
  • What is the disease risk, burden and unmet needs of the Cystic Fibrosis (CF)?
  • What are the currently available treatments of Cystic Fibrosis (CF)?

Reasons to buy:

The Cystic Fibrosis (CF) Epidemiology report will allow the user to -

  • Develop business strategies by understanding the trends shaping and driving the global Cystic Fibrosis (CF) market
  • Quantify patient populations in the global Cystic Fibrosis (CF) market to improve product design, pricing, and launch plans
  • Organize sales and marketing efforts by identifying the age groups and sex that present the best opportunities for Cystic Fibrosis (CF) therapeutics in each of the markets covered
  • Understand the magnitude of Cystic Fibrosis (CF) population by its Prevalence cases
  • Understand the magnitude of Cystic Fibrosis (CF) population by its Gender-specific cases
  • Understand the magnitude of Cystic Fibrosis (CF) population by its Age-specific cases
  • Understand the magnitude of Cystic Fibrosis (CF) population by its Type-specific cases
  • The Cystic Fibrosis (CF) epidemiology report and model were written and developed by Masters and PhD level epidemiologists
  • The Cystic Fibrosis (CF) Epidemiology Model developed by DelveInsight is easy to navigate, interactive with dashboards, and epidemiology based on transparent and consistent methodologies. Moreover, the model supports data presented in the report and showcases disease trends over 11-year forecast period using reputable sources

Key Assessments

  • Patient Segmentation
  • Disease Risk and Burden
  • Risk of disease by the segmentation
  • Factors driving growth in a specific patient population

Geographies Covered

  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Study Period: 2017-2030.

Cloning of CF Transmembrane conductance Regulator (CFTR) gene has provided a breakthrough in the understanding of the molecular basis of this disease. There are over 1,800 mutations of CFTR identified so far, wherein 23 variants account for the majority of CF-causing mutations. The CF-causing mutations of CFTR can be grouped into classes depending on the physiological effect. Some mutations cause little or no protein to be made (Class I and IV), others create a defective protein that does not make it to the cell membrane (Class II), and others cannot be effectively regulated or conduct chloride (Class III and IV).

Classification of the mutations allows the development of specialized drugs to overcome the specific gene mutation. The most common CF mutation, F508del, is primarily considered to be a processing mutation. The F508del mutation removes a single amino acid from the CFTR protein. Without this building block, the CFTR protein cannot stay in the correct 3-D shape. The cell recognizes that the protein is not the right shape and disposes of it.

Approximately 44% of CF patients are homozygous for the F508del mutation (i.e., F508del/F508del), while around 41% are compound heterozygotes (i.e., F508del/other CF causing CFTR mutation), and about 15% have two non-F508del CF-causing CFTR mutations.

Table of Contents

1 Key Insights

2 Executive Summary

3 Organizations

4 Epidemiology and Market Methodology

5 Cystic Fibrosis: Market Overview at a Glance

  • 5.1 Total Market Share (%) Distribution of Cystic Fibrosis in 2017
  • 5.2 Total Market Share (%) Distribution of Cystic Fibrosis in 2030

6 Cystic Fibrosis: Market Overview at a Glance

  • 6.1 Introduction
  • 6.2 Classification of cystic fibrosis
  • 6.3 Pathophysiology
  • 6.4 Clinical signs and symptoms
  • 6.5 Diagnosis

7 Epidemiology and Patient Population

  • 7.1 Key Findings
  • 7.2 Total cases of Cystic Fibrosis in 6MM
  • 7.3 United States
    • 7.3.1 Assumptions and Rationale
    • 7.3.2 Total Prevalent cases of Cystic Fibrosis in the United States
    • 7.3.3 Gender-specific cases of Cystic Fibrosis in the United States
    • 7.3.4 Age-specific cases of Cystic Fibrosis in the United States
    • 7.3.5 Type-specific cases of Cystic Fibrosis in the United States
  • 7.4 EU5 Countries
    • 7.4.1 Assumptions and Rationale
  • 7.5 Germany
    • 7.5.1 Total Prevalent cases of Cystic Fibrosis in Germany
    • 7.5.2 Gender-specific cases of Cystic Fibrosis in Germany
    • 7.5.3 Age-specific cases of Cystic Fibrosis in Germany
    • 7.5.4 Type-specific cases of Cystic Fibrosis in Germany
  • 7.6 France
    • 7.6.1 Total Prevalent cases of Cystic Fibrosis in France
    • 7.6.2 Gender-specific cases of Cystic Fibrosis in France
    • 7.6.3 Age-specific cases of Cystic Fibrosis in France
    • 7.6.4 Type-specific cases of Cystic Fibrosis in France
  • 7.7 Italy
    • 7.7.1 Total Prevalent cases of Cystic Fibrosis in Italy
    • 7.7.2 Gender-specific cases of Cystic Fibrosis in Italy
    • 7.7.3 Age-specific cases of Cystic Fibrosis in Italy
    • 7.7.4 Type-specific cases of Cystic Fibrosis in Italy
  • 7.8 Spain
    • 7.8.1 Total Prevalent cases of Cystic Fibrosis in Spain
    • 7.8.2 Gender-specific cases of Cystic Fibrosis in Spain
    • 7.8.3 Age-specific cases of Cystic Fibrosis in Spain
    • 7.8.4 Type-specific cases of Cystic Fibrosis in Spain
  • 7.9 UK
    • 7.9.1 Total Prevalent cases of Cystic Fibrosis in the United Kingdom
    • 7.9.2 Gender-specific cases of Cystic Fibrosis in the United Kingdom
    • 7.9.3 Age-specific cases of Cystic Fibrosis in the United Kingdom
    • 7.9.4 Type-specific cases of Cystic Fibrosis in the United Kingdom

8 Treatment for Cystic Fibrosis

  • 8.1 Medications
  • 8.2 Diet and Exercise
  • 8.3 Airway clearance techniques
  • 8.4 Gene Therapy
  • 8.5 Surgery

9 KOL Views

10 Appendix

  • 10.1 Report Methodology

11 DelveInsight Capabilities

12 Disclaimer

13 About DelveInsight

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