Published : Jul-2018
No. of Pages : 95
1 Table of Contents 2
1.1 List of Tables 5
1.2 List of Figures 5
2 Amyotrophic Lateral Sclerosis: Executive Summary 6
2.1 Global ALS Market Expected to Experience Explosive Growth by 2027 7
2.2 Pharmaceutical Companies Target Novel MOAs in a Market with Few Approved Therapies 8
2.3 Several Key Unmet Needs Identified in the ALS Market, Providing Opportunity for New Players 9
2.4 Several Different Classes in the ALS Pipeline Look to Fill the Gaps in the Market 9
2.5 What Do Physicians Think? 10
3 Introduction 13
3.1 Catalyst 13
3.2 Related Reports 14
3.3 Upcoming Related Reports 14
4 Disease Overview 15
4.1 Etiology and Pathophysiology 15
4.1.1 Etiology 15
4.1.2 Pathophysiology 17
4.2 Classification or Staging Systems 18
4.3 Symptoms 19
4.3.1 Complications 19
4.4 Quality of Life 20
5 Epidemiology 21
5.1 Disease Background 21
5.2 Risk Factors and Comorbidities 21
5.3 Global and Historical Trends 22
5.3.1 US 24
5.3.2 5EU 25
5.3.3 Japan 25
5.4 Forecast Methodology 25
5.4.1 Sources 26
5.4.2 Forecast Assumptions and Methods 27
5.5 Epidemiological Forecast for ALS (2017-2027) 33
5.5.1 Diagnosed Prevalent Cases of ALS 33
5.5.2 Age-Specific Diagnosed Prevalent Cases of ALS 34
5.5.3 Sex-Specific Diagnosed Prevalent Cases of ALS 35
5.5.4 Diagnosed Prevalent Cases of ALS by Type 36
5.5.5 Total Prevalent Cases of ALS 37
5.6 Discussion 38
5.6.1 Epidemiological Forecast Insight 38
5.6.2 Limitations of Analysis 40
5.6.3 Strengths of Analysis 41
6 Current Treatment Options 42
6.1 Overview 42
7 Unmet Needs and Opportunity Assessment 46
7.1 Overview 46
7.2 Faster Diagnosis 47
7.3 Curative Agent 49
7.4 Extended Patient Survival 51
7.5 Identification of Biomarkers Associated with ALS 52
8 R&D Strategies 55
8.1 Overview 55
8.1.1 Novel Targets 55
8.1.2 Disease-Modifying Therapies 56
8.1.3 Reformulations 58
8.2 Clinical Trials Design 59
8.2.1 Endpoints 60
8.2.2 Length of Clinical Trials 61
8.2.3 Patient Recruitment 61
9 Pipeline Assessment 63
9.1 Overview 63
9.2 Innovative Early-Stage Approaches 66
10 Pipeline Valuation Analysis 68
10.1 Clinical Benchmark of Key Pipeline Drugs 68
10.2 Commercial Benchmark of Key Pipeline Drugs 70
10.3 Competitive Assessment 72
10.4 Top-Line 10-Year Forecast 73
10.4.1 US 75
10.4.2 5EU 76
10.4.3 Japan 76
11 Appendix 78
11.1 Bibliography 78
11.2 Abbreviations 83
11.3 Methodology 84
11.3.1 Forecasting Methodology 84
11.3.2 Diagnosed Patients 84
11.3.3 Percent Drug-Treated Patients 85
11.3.4 Drugs Included in Each Therapeutic Class 85
11.3.5 Launch and Patent Expiry Dates 85
11.3.6 General Pricing Assumptions 86
11.3.7 Individual Drug Assumptions 87
11.3.8 Generic Erosion 88
11.3.9 Pricing of Pipeline Agents 88
11.4 Primary Research - KOLs Interviewed for This Report 89
11.4.1 KOLs 89
11.4.2 Payers 90
11.5 Primary Research - Prescriber Survey 90
11.6 About the Authors 91
11.6.1 Analyst 91
11.6.2 Therapy Area Director 91
11.6.3 Epidemiologist 91
11.6.4 Managing Epidemiologist 92
11.6.5 Global Director of Therapy Analysis and Epidemiology 93
11.6.6 Global Head and EVP of Healthcare Operations and Strategy 93
11.7 About GlobalData 94
11.8 Contact Us 94
11.9 Disclaimer 95
1.1 List of Tables
Table 1: ALS: Key Metrics in the 7MM 6
Table 2: Gene Involved in the Familial Forms of ALS 16
Table 3: Symptoms of ALS 19
Table 4: Risk Factors and Comorbidities for ALS 22
Table 5: Leading Treatments for ALS, 2018 43
Table 6: Treatment Guidelines for ALS 45
Table 7: Comparison of Therapeutic Classes in Development for ALS, 2017-2027 65
Table 8: Innovative Early-Stage Approaches for ALS, 2018 67
Table 9: Clinical Benchmark of Key Pipeline Drugs - ALS 70
Table 10: Commercial Benchmark of Key Pipeline Drugs - ALS 71
Table 11: Key Events Impacting Sales for ALS, 2017-2027 74
Table 12: ALS Market - Global Drivers and Barriers, 2017-2027 75
Table 13: Key Historical and Projected Launch Dates for ALS 85
Table 14: Key Historical and Projected Patent Expiry Dates for ALS 86
Table 15: High-Prescribing Physicians (non-KOLs) Surveyed, By Country 90
1.2 List of Figures
Figure 1: Global Sales Forecast by Country for ALS in 2017 and 2027 8
Figure 2: Competitive Assessment of Marketed and Pipeline Drugs, Benchmarked Against the SOC, Rilutek 10
Figure 3: Overview of the human body and muscles involved in ALS 15
Figure 4: Pathophysiology of ALS 17
Figure 5: Staging Systems for ALS 18
Figure 6: 7MM, Age-Standardized Diagnosed Prevalence of ALS, Ages ?40 Years, 2017 24
Figure 7:Sources Used for Diagnosed Prevalent Cases of ALS 26
Figure 8:Sources Used for Diagnosed Prevalent Cases of ALS Type 27
Figure 9: 7MM, Diagnosed Prevalent Cases of ALS, Men and Women, Ages ?40 Years, 2017 34
Figure 10: 7MM, Age-Specific Diagnosed Prevalent Cases of ALS, Men and Women, 2017 35
Figure 11: 7MM, Sex-Specific Diagnosed Prevalent Cases of ALS, Men and Women, Ages ?40 Years, 2017 36
Figure 12: 7MM, Diagnosed Prevalent Cases of ALS by Type, Men and Women, Ages ?40 Years, 2017 37
Figure 13: 7MM, Total Prevalent Cases of ALS, Men and Women, Ages ?40 Years, 2017 38
Figure 14: Unmet Needs and Opportunities in ALS 47
Figure 15: Overview of the Development Pipeline in ALS 64
Figure 16: Key Phase II and III Trials for Promising Pipeline Agents 65
Figure 17:Competitive Assessment of the Marketed and Pipeline Drugs Benchmarked Against the Standard of Care (SOC), Rilutek 72
Figure 18: Global (7MM) Sales Forecast by Country for ALS in 2017 and 2027 74
