Acquired Orphan Blood Diseases Therapeutics Market to 2019 · Acquired Orphan Blood Diseases...

Acquired Orphan Blood Diseases Therapeutics Market to 2019 Search for Disease Modifying Drugs Central to Unlocking Premium Pricing Potential

GBI Research Report Guidance

GBIHC294MR / Published JAN 2013

© GBI Research. This is a licensed product and is not to be photocopied

GBI Research Report Guidance

The report provides an overview of the acquired orphan blood diseases market.

Chapter two gives an introduction to the acquired orphan blood diseases market

Chapter three describes the therapeutic landscape of the acquired orphan blood diseases market. This section includes detailed overview, market sizing, annual cost of treatment, treatment usage patterns, market products, pipeline products and the drivers and barriers for five major indications, namely Paroxysmal Nocturnal Hemoglobinuria (PNH), Idiopathic Thrombocytopenic Purpura (ITP), Myelodysplastic Syndrome (MDS), Myelofibrosis (MF) and Polycythemia Vera (PV). The section also analyses the market analysis of the mentioned indications in the major countries of the US, UK, France, Germany, Italy, Spain and Japan.

Chapter four covers the major deals that have taken place in the acquired orphan blood diseases market in recent years. Coverage includes Mergers and Acquisitions (M&As) and licensing agreements, which are segmented on the basis of indication, geography, year, deal type, and value.



Acquired Orphan Blood Diseases - Executive Summary

Acquired Orphan Blood Diseases - Executive Summary

This report reviews and forecasts the therapeutic markets for five acquired orphan blood diseases to 2019. The indications covered are Paroxysmal Nocturnal Hemoglobinuria (PNH), Idiopathic Thrombocytopenic Purpura (ITP), Myelodysplastic Syndrome (MDS), Myelofibrosis (MF) and Polycythemia Vera (PV). As orphan diseases, they have very low prevalence and incidence rates and in many cases may disproportionately affect individuals of a particular age or sex. Many orphan diseases have high unmet clinical needs and premium pricing potential for successful new market entries; this is the case for all five indications covered.

The MF market will be the fastest growing of the five markets during the forecast period, at a Compound Annual Growth Rate (CAGR) of XX% and reaching a total market size of $XXm in 2019. MDS represents the largest market, expected to reach $XXm by 2019.

These five indications offer significant treatment challenges and many patient populations have a high level of unmet need, in turn offering diverse examples of drug markets.

The PNH market is dominated by Soliris, which is currently the world’s most expensive drug. Due to its market dominance, the PNH pipeline is very weak. The outlook for the MDS market is good as it has the largest pipeline of all XX indications. This is also boosted by improving disease diagnosis and awareness rates. Similarly, MF, a disease with significant unmet needs for treatment, also has a promising pipeline and improving diagnosis rates.



Table of Contents

1 Table of Contents

1 Table of Contents................................................................................................................................. 4 1.1 List of Tables............................................................................................................................. 6 1.2 List of Figures............................................................................................................................ 7

2 Acquired Orphan Blood Diseases - Introduction.................................................................................... 9 2.1 Overview.................................................................................................................................. 9

3 Acquired Orphan Blood Diseases - Therapeutic Landscape...................................................................10 3.1 Paroxysmal Nocturnal Hemoglobinuria.....................................................................................10

3.1.1 Disease Overview.............................................................................................................10 3.1.2 Revenues .........................................................................................................................12 3.1.3 Revenue Analysis by Country ............................................................................................13 3.1.4 Annual Cost of Therapy ....................................................................................................17 3.1.5 Treatment Usage Patterns................................................................................................18 3.1.6 Marketed Products...........................................................................................................19 3.1.7 Market Drivers.................................................................................................................20 3.1.8 Market Restraints ............................................................................................................20

3.2 Paroxysmal Nocturnal Hemoglobinuria: Research and Development Pipeline............................21 3.2.1 Overview .........................................................................................................................21 3.2.2 Trends in Paroxysmal Nocturnal Hemoglobinuria Pipeline .................................................23 3.2.3 Promising Pipeline Molecules ...........................................................................................24 3.2.4 Key Takeaway..................................................................................................................24

3.3 Idiopathic Thrombocytopenic Purpura......................................................................................25 3.3.1 Disease Overview.............................................................................................................25 3.3.2 Revenues .........................................................................................................................27 3.3.3 Revenue Analysis by Country ............................................................................................28 3.3.4 Annual Cost of Therapy ....................................................................................................32 3.3.5 Treatment Usage Patterns................................................................................................33 3.3.6 Marketed Products...........................................................................................................34 3.3.7 Market Drivers.................................................................................................................34 3.3.8 Market Restraints ............................................................................................................34

3.4 Idiopathic Thrombocytopenic Purpura: Research and Development Pipeline.............................35 3.4.1 Overview .........................................................................................................................35 3.4.2 Pipeline by Clinical Phase of Development.........................................................................36 3.4.3 Trends in Idiopathic Thrombocytopenic Purpura Pipeline...................................................40 3.4.4 Promising Pipeline Molecules ...........................................................................................41 3.4.5 Key Takeaway..................................................................................................................43

3.5 Myelodysplastic Syndrome.......................................................................................................44 3.5.1 Disease Overview.............................................................................................................44 3.5.2 Revenues .........................................................................................................................45 3.5.3 Revenue Analysis by Country ............................................................................................46 3.5.4 Annual Cost of Therapy ....................................................................................................50 3.5.5 Treatment Usage Patterns................................................................................................51 3.5.6 Marketed Products...........................................................................................................52 3.5.7 Market Drivers.................................................................................................................53 3.5.8 Market Restraints ............................................................................................................53

3.6 Myelodysplastic Syndrome: Research and Development Pipeline..............................................54 3.6.1 Overview .........................................................................................................................54 3.6.2 Pipeline by Clinical Phase of Development.........................................................................55 3.6.3 Trends in Myelodysplastic Syndrome Pipeline....................................................................80 3.6.4 Promising Pipeline Molecules ...........................................................................................83 3.6.5 Key Takeaway..................................................................................................................83



Table of Contents

3.7 Myelofibrosis...........................................................................................................................84 3.7.1 Disease Overview.............................................................................................................84 3.7.2 Revenues .........................................................................................................................85 3.7.3 Revenue Analysis by Country ............................................................................................86 3.7.4 Annual Cost of Therapy ....................................................................................................90 3.7.5 Treatment Usage Patterns................................................................................................91 3.7.6 Marketed Products...........................................................................................................92 3.7.7 Market Drivers.................................................................................................................92 3.7.8 Market Restraints ............................................................................................................92

3.8 Myelofibrosis: Research and Development Pipeline..................................................................93 3.8.1 Overview .........................................................................................................................93 3.8.2 Pipeline by Clinical Phase of Development.........................................................................94 3.8.3 Trends in Myelofibrosis Pipeline........................................................................................99 3.8.4 Promising Pipeline Molecules .........................................................................................101 3.8.5 Key Takeaway................................................................................................................101

3.9 Polycythemia Vera.................................................................................................................102 3.9.1 Disease Overview...........................................................................................................102 3.9.2 Revenues .......................................................................................................................103 3.9.3 Revenue Analysis by Country ..........................................................................................104 3.9.4 Annual Cost of Therapy ..................................................................................................108 3.9.5 Treatment Usage Patterns..............................................................................................109 3.9.6 Marketed Products.........................................................................................................110 3.9.7 Market Drivers...............................................................................................................111 3.9.8 Market Restraints ..........................................................................................................111

3.10 Polycythemia Vera: Research and Development Pipeline ........................................................112 3.10.1 Overview .......................................................................................................................112 3.10.2 Pipeline by Clinical Phase of Development.......................................................................113 3.10.3 Trends in Polycythemia Vera Pipeline..............................................................................116 3.10.4 Promising Pipeline Molecules .........................................................................................118 3.10.5 Key Takeaway................................................................................................................118

4 Acquired Orphan Blood Diseases - Strategic Consolidations ...............................................................119 4.1 Overview...............................................................................................................................119

4.1.1 Segmentation by Year ....................................................................................................119 4.1.2 Segmentation by Indication............................................................................................119 4.1.3 Segmentation by Geography ..........................................................................................120 4.1.4 Segmentation by Deal Value...........................................................................................120 4.1.5 Segmentation by Deal Type ............................................................................................121

4.2 Major Mergers and Acquisitions.............................................................................................121 4.3 Major Licensing Agreements ..................................................................................................122

5 Acquired Orphan Blood Diseases - Appendix......................................................................................123 5.1 Abbreviations ........................................................................................................................123 5.2 Sources..................................................................................................................................123 5.3 Research Methodology ..........................................................................................................124

5.3.1 Coverage .......................................................................................................................124 5.3.2 Secondary Research .......................................................................................................124 5.3.3 Primary Research ...........................................................................................................125 5.3.4 Therapeutic Landscape...................................................................................................125 5.3.5 Geographical Landscape ................................................................................................128 5.3.6 Pipeline Analysis.............................................................................................................128 5.3.7 Competitive Landscape...................................................................................................128 5.3.8 Expert Panel Validation ..................................................................................................128

5.4 Contact Us.............................................................................................................................128



Table of Contents

5.5 Disclaimer..............................................................................................................................128

1.1 List of Tables

Table 1: Approximate PNH Patient Population, by Country, 2011 ..........................................................10 Table 2: Paroxysmal Nocturnal Hemoglobinuria, Total Market, Revenue Forecast ($m), 2011-2019 .......12 Table 3: Paroxysmal Nocturnal Hemoglobinuria, Total Market, Average Annual Cost of Therapy (%),

2011-2019 ..............................................................................................................................17 Table 4: Paroxysmal Nocturnal Hemoglobinuria, Treatment Usage Pattern (‘000), 2011-2019................18 Table 5: Paroxysmal Nocturnal Hemoglobinuria, Pipeline Molecule Details, 2012 ..................................22 Table 6: Idiopathic Thrombocytopenic Purpura, Approximate Patient Population by Country (‘000), 2011

...............................................................................................................................................25 Table 7: Idiopathic Thrombocytopenic Purpura, Total Market, Revenue Forecast ($m), 2011-2019........27 Table 8: Idiopathic Thrombocytopenic Purpura, Total Market, Average Annual Cost of Therapy (%),

2011-2019 ..............................................................................................................................32 Table 9: Idiopathic Thrombocytopenic Purpura, Treatment Usage Pattern (‘000), 2011-2019 ................33 Table 10: Idiopathic Thrombocytopenic Purpura, Pipeline, Discovery, 2012.............................................36 Table 11: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase I, 2012.................................................36 Table 12: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase II, by Therapeutic Class, 2012...............37 Table 13: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase III, 2012...............................................38 Table 14: Myelodysplastic Syndrome, Patient Population by Country (‘000), 2011...................................44 Table 15: Myelodysplastic Syndrome, Total Market, Revenue Forecast ($m), 2011-2019 .........................45 Table 16: Myelodysplastic Syndrome, Total Market, Average Annual Cost of Therapy (%), 2011-2019......50 Table 17: Myelodysplastic Syndrome, Treatment Usage Pattern (‘000), 2011-2019 .................................51 Table 18: Myelodysplastic Syndrome, Pipeline Overview, Phase I, 2012 ..................................................55 Table 19: Myelodysplastic Syndrome, Pipeline Overview, Phase II...........................................................61 Table 20: Myelodysplastic Syndrome, Pipeline Overview, Phase III..........................................................77 Table 21: Myelodysplastic Syndrome, Pipeline Overview, Other..............................................................80 Table 22: Myelofibrosis, Patient Population by Country (‘000), 2011.......................................................84 Table 23: Myelofibrosis, Total Market, Revenue Forecast ($m), 2011-2019 .............................................85 Table 24: Myelofibrosis, Total Market, Average Annual Cost of Therapy (%), 2011-2019..........................90 Table 25: Myelofibrosis, Treatment Usage Pattern (‘000), 2011-2019......................................................91 Table 26: Myelofibrosis, Pipeline, Phase I, 2012......................................................................................94 Table 27: Myelofibrosis, Pipeline, Phase II, 2012.....................................................................................95 Table 28: Myelofibrosis, Pipeline, Phase III, 2012....................................................................................98 Table 29: Polycythemia Vera, Approximate Patient Population by Country (‘000), 2011.........................102 Table 30: Polycythemia Vera, Total Market, Revenue Forecast ($m), 2011-2019....................................103 Table 31: Polycythemia Vera, Total Market, Average Annual Cost of Therapy (%), 2011-2019................108 Table 32: Polycythemia Vera, Treatment Usage Pattern (‘000), 2011-2019............................................109 Table 33: Polycythemia Vera, Pipeline, Preclinical and Phase I, 2012 .....................................................113 Table 34: Polycythemia Vera, Pipeline, Phase II, 2012 ...........................................................................114 Table 35: Polycythemia Vera, Pipeline, Phase III, 2012 ..........................................................................115



Table of Contents

1.2 List of Figures

Figure 1: Paroxysmal Nocturnal Hemoglobinuria, Total Market, Revenue Forecast ($m), 2011-2019 .......12 Figure 2: Paroxysmal Nocturnal Hemoglobinuria, US, Revenue Forecast ($m), 2011-2019.......................13 Figure 3: Paroxysmal Nocturnal Hemoglobinuria, UK, Revenue Forecast ($m), 2011-2019.......................13 Figure 4: Paroxysmal Nocturnal Hemoglobinuria, France, Revenue Forecast ($m), 2011-2019.................14 Figure 5: Paroxysmal Nocturnal Hemoglobinuria, Germany, Revenue Forecast ($m), 2011-2019.............14 Figure 6: Paroxysmal Nocturnal Hemoglobinuria, Italy, Revenue Forecast ($m), 2011-2019 ....................15 Figure 7: Paroxysmal Nocturnal Hemoglobinuria, Spain, Revenue Forecast ($m), 2011-2019...................15 Figure 8: Paroxysmal Nocturnal Hemoglobinuria, Japan, Revenue Forecast ($m), 2011-2019 ..................16 Figure 9: Paroxysmal Nocturnal Hemoglobinuria, Total Market, Average Annual Cost of Therapy (%),

2011-2019 ..............................................................................................................................17 Figure 10: Paroxysmal Nocturnal Hemoglobinuria, Treatment Usage Pattern (‘000), 2011-2019................18 Figure 11: Paroxysmal Nocturnal Hemoglobinuria, Pipeline Overview ......................................................21 Figure 12: Paroxysmal Nocturnal Hemoglobinuria, Pipeline by Therapeutic Class......................................23 Figure 13: Paroxysmal Nocturnal Hemoglobinuria, Pipeline by Molecule Type ..........................................23 Figure 14: Paroxysmal Nocturnal Hemoglobinuria, Pipeline by Mechanism of Action ................................24 Figure 15: Idiopathic Thrombocytopenic Purpura, Underlying Mechanism................................................25 Figure 16: Idiopathic Thrombocytopenic Purpura, Total Market, Revenue Forecast ($m), 2011-2019........27 Figure 17: Idiopathic Thrombocytopenic Purpura, US, Revenue Forecast ($m), 2011-2019........................28 Figure 18: Idiopathic Thrombocytopenic Purpura, UK, Revenue Forecast ($m), 2011-2019 .......................28 Figure 19: Idiopathic Thrombocytopenic Purpura, France, Revenue Forecast ($m), 2011-2019..................29 Figure 20: Idiopathic Thrombocytopenic Purpura, Germany, Revenue Forecast ($m), 2011-2019..............29 Figure 21: Idiopathic Thrombocytopenic Purpura, Italy, Revenue Forecast ($m), 2011-2019 .....................30 Figure 22: Idiopathic Thrombocytopenic Purpura, Spain, Revenue Forecast ($m), 2011-2019 ...................30 Figure 23: Idiopathic Thrombocytopenic Purpura, Japan, Revenue Forecast ($m), 2011-2019...................31 Figure 24: Idiopathic Thrombocytopenic Purpura, Total Market, Average Annual Cost of Therapy (%),

2011-2019 ..............................................................................................................................32 Figure 25: Idiopathic Thrombocytopenic Purpura, Treatment Usage Pattern (‘000), 2011-2019 ................33 Figure 26: Idiopathic Thrombocytopenic Purpura, Pipeline Overview .......................................................35 Figure 27: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase I by Therapeutic Class..........................36 Figure 28: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase II Molecule Comparison.......................37 Figure 29: Idiopathic Thrombocytopenic Purpura, Pipeline, Phase III Molecule Comparison......................39 Figure 30: Idiopathic Thrombocytopenic Purpura, Pipeline by Therapeutic Class.......................................40 Figure 31: Idiopathic Thrombocytopenic Purpura, Pipeline by Molecule Type...........................................40 Figure 32: Idiopathic Thrombocytopenic Purpura, Pipeline by Mechanism of Action.................................41 Figure 33: Myelodysplastic Syndrome, Total Market, Revenue Forecast ($m), 2011-2019 .........................45 Figure 34: Myelodysplastic Syndrome, US, Revenue Forecast ($m), 2011-2019.........................................46 Figure 35: Myelodysplastic Syndrome, UK, Revenue Forecast ($m), 2011-2019.........................................46 Figure 36: Myelodysplastic Syndrome, France, Revenue Forecast ($m), 2011-2019...................................47 Figure 37: Myelodysplastic Syndrome, Germany, Revenue Forecast ($m), 2011-2019...............................47 Figure 38: Myelodysplastic Syndrome, Italy, Revenue Forecast ($m), 2011-2019 ......................................48 Figure 39: Myelodysplastic Syndrome, Spain, Revenue Forecast ($m), 2011-2019.....................................48 Figure 40: Myelodysplastic Syndrome, Japan, Revenue Forecast ($m), 2011-2019 ....................................49 Figure 41: Myelodysplastic Syndrome, Total Market, Average Annual Cost of Therapy (%), 2011-2019......50 Figure 42: Myelodysplastic Syndrome, Treatment Usage Pattern (‘000), 2011-2019 .................................51 Figure 43: Myelodysplastic Syndrome, Pipeline Overview ........................................................................54 Figure 44: Myelodysplastic Syndrome, Pipeline, Phase I Molecule Comparison.........................................60 Figure 45: Myelodysplastic Syndrome, Pipeline, Phase II Molecule Comparison ........................................76 Figure 46: Myelodysplastic Syndrome, Pipeline, Phase III Molecule Comparison.......................................79 Figure 47: Myelodysplastic Syndrome, Pipeline by Therapeutic Class........................................................80 Figure 48: Myelodysplastic Syndrome, Pipeline by Molecule Type............................................................81 Figure 49: Myelodysplastic Syndrome, Pipeline by Mechanism of Action..................................................82 Figure 50: Myelofibrosis, Total Market, Revenue Forecast ($m), 2011-2019 .............................................85 Figure 51: Myelofibrosis, US, Revenue Forecast ($m), 2011-2019.............................................................86 Figure 52: Myelofibrosis, UK, Revenue Forecast ($m), 2011-2019.............................................................86 Figure 53: Myelofibrosis, France, Revenue Forecast ($m), 2011-2019.......................................................87 Figure 54: Myelofibrosis, Germany, Revenue Forecast ($m), 2011-2019...................................................87 Figure 55: Myelofibrosis, Italy, Revenue Forecast ($m), 2011-2019...........................................................88



Table of Contents

Figure 56: Myelofibrosis, Spain, Revenue Forecast ($m), 2011-2019.........................................................88 Figure 57: Myelofibrosis, Japan, Revenue Forecast ($m), 2011-2019 ........................................................89 Figure 58: Myelofibrosis, Total Market, Average Annual Cost of Therapy (%), 2011-2019..........................90 Figure 59: Myelofibrosis, Treatment Usage Pattern (‘000), 2011-2019......................................................91 Figure 60: Myelofibrosis, Pipeline Overview.............................................................................................93 Figure 61: Myelofibrosis, Pipeline, Phase I Molecule Comparison .............................................................94 Figure 62: Myelofibrosis, Pipeline, Phase II Molecule Comparison ............................................................97 Figure 63: Myelofibrosis, Pipeline, Phase III Molecule Comparison ...........................................................98 Figure 64: Myelofibrosis, Pipeline by Therapeutic Class............................................................................99 Figure 65: Myelofibrosis, Pipeline by Molecule Type ................................................................................99 Figure 66: Myelofibrosis, Pipeline by Mechanism of Action ....................................................................100 Figure 67: Polycythemia Vera, Total Market, Revenue Forecast ($m), 2011-2019....................................103 Figure 68: Polycythemia Vera, US, Revenue Forecast ($m), 2011-2019 ...................................................104 Figure 69: Polycythemia Vera, UK, Revenue Forecast ($m), 2011-2019...................................................104 Figure 70: Polycythemia Vera, France, Revenue Forecast ($m), 2011-2019.............................................105 Figure 71: Polycythemia Vera, Germany, Revenue Forecast ($m), 2011-2019 .........................................105 Figure 72: Polycythemia Vera, Italy, Revenue Forecast ($m), 2011-2019.................................................106 Figure 73: Polycythemia Vera, Spain, Revenue Forecast ($m), 2011-2019...............................................106 Figure 74: Polycythemia Vera, Japan, Revenue Forecast ($m), 2011-2019...............................................107 Figure 75: Polycythemia Vera, Total Market, Average Annual Cost of Therapy (%), 2011-2019................108 Figure 76: Polycythemia Vera, Treatment Usage Pattern (‘000), 2011-2019............................................109 Figure 77: Polycythemia Vera, Pipeline Overview...................................................................................112 Figure 78: Polycythemia Vera, Pipeline, Phase I Molecule Comparison ...................................................113 Figure 79: Polycythemia Vera, Pipeline, Phase II Molecule Comparison ..................................................115 Figure 80: Polycythemia Vera, Pipeline by Therapeutic Class ..................................................................116 Figure 81: Polycythemia Vera, Pipeline by Molecule Type ......................................................................116 Figure 82: Polycythemia Vera, Pipeline by Mechanism of Action ............................................................117 Figure 83: Deal Segmentation by Year, 2004-2012 .................................................................................119 Figure 84: Deal Segmentation by Indication ...........................................................................................119 Figure 85: Deal Segmentation by Geography, Total Market ....................................................................120 Figure 86: Deal Segmentation by Value..................................................................................................120 Figure 87: Deal Segmentation by Deal Type ...........................................................................................121 Figure 88: GBI Research Market Forecasting Model ...............................................................................127



Acquired Orphan Blood Diseases - Introduction

2 Acquired Orphan Blood Diseases - Introduction

2.1 Overview

Orphan diseases are also known as rare diseases and affect only a small minority of a country’s population. In Europe, a disease is considered rare if it affects five or fewer than five people per XX. In the US, a disease is considered rare if it affects fewer than XX (or around XX people per XX people) individuals within the population or a certain subpopulation. In Japan, a disease is considered rare if it affects fewer than XX individuals within the population, or around four people per XX.

The prevalence and therefore status of a disease is likely to vary over time and differ between countries and subpopulations. For example, Acquired Immunodeficiency Syndrome (AIDS) was previously a very rare disease in the US, affecting a disproportionate number of individuals within the homosexual community, intravenous drug users, hemophiliacs and the US Haitian immigrant subpopulation. Today, AIDS is classified as an epidemic by the World Health Organization (WHO) with the highest prevalence among the populations of the countries of Sub-Saharan Africa and South and South East Asia.

Rare diseases are usually serious and often life-threatening. They may present during childhood, but in reality more than XX% only develop during adulthood. Due to their rarity and unfamiliarity within the medical community, sufferers are often misdiagnosed or undiagnosed completely. What treatments do exist are often limited to symptom management, life expectancy extension and improvement of quality of life rather than curative treatments. It is these failings that make the orphan disease drug market such a good opportunity for drug discovery and development.

In this report, the markets for the treatment of five acquired orphan blood disorders will be examined in the US, the top five EU countries (the UK, France, Germany, Italy and Spain), and Japan. The diseases covered are Paroxysmal Nocturnal Hemoglobinuria (PNH), Idiopathic Thrombocytopenic Purpura (ITP), Myelodysplastic Syndrome (MDS), Myelofibrosis (MF) and Polycythemia Vera (PV).

What treatments do exist for rare diseases are often limited to symptom management, life expectancy extension and improvement of quality of life rather than curative treatments


Page 12 © GBI Research. This is a licensed product and is not to be photocopied

Acquired Orphan Blood Diseases - Therapeutic Landscape

3.1.2 Revenues

Below is the cumulative market forecast, for all markets covered in the report, of pharmaceutical treatments indicated for the treatment of PNH. The market is expected to grow at a Compound Annual Growth Rate (CAGR) of XX% over the 2011-2019 period to $XXm by 2019.

Figure 1: Paroxysmal Nocturnal Hemoglobinuria, Total Market, Revenue Forecast ($m), 2011-2019

2011 2012 2013 2014 2015 2016 2017 2018 2019

Rev

enue

($m

)

Source: GBI Research’s Marketed Products database [accessed November 1, 2012]

Table 2: Paroxysmal Nocturnal Hemoglobinuria, Total Market, Revenue Forecast ($m), 2011-2019

Year 2011 2012 2013 2014 2015 2016 2017 2018 2019 CAGR

US

UK

Germany

France

Italy

Spain

Japan


Market growth is forecast to be fairly slow, mostly due to the market dominance of Soliris and the absence of new molecules entering the market during the forecast period.



Acquired Orphan Blood Diseases - Therapeutic Landscape

3.1.5 Treatment Usage Patterns

Diagnosis and treatment rates for PNH are very high because the vast majority of individuals diagnosed with PNH have an existing blood disorder such as aplastic anemia or MDS. These patient populations are often screened annually for the onset of PNH. As they are already receiving treatment they are highly likely to be treated for PNH as well. The following graph shows the treatment usage pattern for all seven markets:

Figure 10: Paroxysmal Nocturnal Hemoglobinuria, Treatment Usage Pattern (‘000), 2011-2019

2011 2012 2013 2014 2015 2016 2017 2018 2019

Diseased Diagnosed Treated


Table 4: Paroxysmal Nocturnal Hemoglobinuria, Treatment Usage Pattern (‘000), 2011-2019

2011 2012 2013 2014 2015 2016 2017 2018 2019 CAGR

Diseased

Diagnosed

Treated


3.1.5.1 Diseased Population

The diseased PNH population of all seven markets covered is expected to grow at a CAGR of XX% to reach XX in 2019.

3.1.5.2 Diagnosed Population

The diagnosed PNH population of all seven markets covered is expected to grow at a CAGR of XX% to reach XX in 2019.

3.1.5.3 Treated Population

The treated PNH population of all seven markets covered is expected to grow at a CAGR of XX% to reach XX in 2019.


3 © GBI Research. This is a licensed product and is not to be photocopied

Acquired Orphan Blood Diseases - Appendix

5 Acquired Orphan Blood Diseases - Appendix

5.1 Abbreviations

ACoT: Annual Cost of Therapy

AIDS: Acquired Immune Deficiency Syndrome

AML: Acute Myelogenous Leukemia

CAGR: Compound Annual Growth Rate

CARES: Connecting to Access, Reimbursement, Education and Support

CCR: Conventional Care Regimens

EMA: European Medicines Agency

FDA: Food and Drug Administration

IND: Investigational New Drug

ITP: Idiopathic Thrombocytopenic Purpura

LDH: Lactate Dehydrogenase

MDS: Myelodysplastic Syndrome

MF: Myelofibrosis

MHLW: Ministry of Health, Labour and Welfare

ITP: Idiopathic Thrombocytopenic Purpura

IVIG: Intravenous Immunoglobulin

PNH: Paroxysmal Nocturnal Hemoglobinuria

PV: Polycythemia Vera

WHO: World Health Organization

5.2 Sources

Herper M (2010). “The World's Most Expensive Drugs”, Forbes, February 22, 2010. Available from: http://www.forbes.com/2010/02/19/expensive-drugs-cost-business-healthcare-rare-diseases.html. [Accessed October 29, 2012].

Hillmen P, et al. (1995). Natural history of paroxysmal nocturnal hemoglobinuria. New England Journal of Medicine; 333(19): 1253-1258.

Imbach P, et al. (1981). High-dose intravenous gammaglobulin for idiopathic thrombocytopenic purpura in childhood. Lancet; 1(8232): 1228-1231.

Ligand Pharmaceuticals Incorporated (2008) Ligand Initiates Phase II Trial with LGD-4665 in Idiopathic Thrombocytopenic Purpura. Available: http://investor.ligand.com/Investors/News-and-Events/Press-Releases/Press-Release-Details/2008/ligand-Initiates-Phase-II-Trial-with-LGD-4665-in-Idiopathic-Thrombocytopenic-Purpura/default.aspx [Accessed 30th November 2012]

Logue, G. L., Gutterman, J. U., et al. (1970) Melphalan therapy of polycythemia vera. Blood; 36(1):70-86

Mazzucconi MG, et al. (2006). Therapy with high-dose dexamethasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura: a GIMEMA experience. Blood; 109(4): 1401-1407.

Patel VL, et al. (2012). Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia. Blood; 119(25): 5989-5995.

Psaila B and Bussel JB (2007). Immune Thrombocytopenic Purpura. Hematology/Oncology Clinics of North America; 21: 743-759.




Saso R, et al. (1999). Bone marrow transplants for paroxysmal nocturnal hemoglobinuria. British Journal of Hematology; 104(2): 392-396.

5.3 Research Methodology

GBI Research’s dedicated research and analysis teams consist of experienced professionals with marketing, market research and consulting backgrounds in the pharmaceutical and medical devices industry as well as advanced statistical expertise.

GBI Research adheres to the codes of practice of the Market Research Society (www.mrs.org.uk) and the Strategic and Competitive Intelligence Professionals (www.scip.org).

All GBI Research databases are continuously updated and revised.

5.3.1 Coverage

The objective of updating GBI Research coverage is to ensure that it represents the most up to date vision of the industry possible.

Changes to the industry taxonomy are built on the basis of extensive research of company, association and competitor sources.

Company coverage is based on three key factors: market capitalization, revenues and media attention/innovation/market potential.

An exhaustive search of 56 member exchanges is conducted and companies are prioritized on the basis of their market capitalization;

The estimated revenues of all major companies, including private and governmental, are gathered and used to prioritize coverage; and

Companies which are making the news, or which are of particular interest due to their innovative approach are prioritized.

GBI Research aims to cover all major news events and deals in the medical industry, updated on a daily basis.

The coverage is further streamlined and strengthened with additional inputs from GBI Research Expert Panel (see below).

5.3.2 Secondary Research

The research process begins with exhaustive secondary research on internal and external sources being carried out to source qualitative and quantitative information relating to each market.

The secondary research sources that are typically referred to include, but are not limited to:

Company websites, annual reports, financial reports, broker reports, investor presentations and SEC filings.

Industry trade journals, scientific journals and other technical literature;

Internal and external proprietary databases;

Relevant patent and regulatory databases;

National government documents, statistical databases and market reports;

Procedure registries; and

News articles, press releases and web-casts specific to the companies operating in the market.




5.3.3 Primary Research

GBI Research conducts hundreds of primary interviews a year with industry participants and commentators in order to validate its data and analysis. A typical research interview fulfills the following functions:

It provides first-hand information on the market size, market trends, growth trends, competitive landscape and future outlook

It helps in validating and strengthening the secondary research findings; and

It further develops the analysis team’s expertise and market understanding.

Primary research involves email interactions and telephone interviews as well as face-to-face interviews for each market, category, segment and sub-segment across geographies.

The participants who typically take part in such a process include, but are not limited to:

Industry participants: CEOs, VPs, marketing/product managers, market intelligence managers and national sales managers;

Hospital stores, laboratories, pharmacies, distributors and paramedics;

Outside experts: Investment bankers, valuation experts, research analysts specializing in specific medical equipment markets;

Key Opinion Leaders: Physicians and surgeons specializing in different therapeutic areas corresponding to different kinds of medical equipment.

5.3.4 Therapeutic Landscape

Revenue for each indication by geography is arrived at by utilizing the GBI Research market forecasting model. The global revenue for each indication is the sum value of revenues of all seven regions.

The annual cost of therapy for each indication is arrived at by considering the cost of the drugs, the dosage of the drugs and the duration of the therapy.

The generic share of the market for each indication is obtained by calculating the prescription share for generic drugs and the respective cost of treatment.

The treatment usage pattern which includes quantitative data on the diseased population, treatment-seeking population, diagnosed population and treated population for an indication, is arrived at by referring to various sources as mentioned below.

The marketed drugs section contains an overview of the drugs, their mechanism of action, efficacy and safety issues related to the drugs. The drugs profiled in this section are chosen based on estimated revenues and their mechanism of action.

GBI Research uses the epidemiology-based treatment flow model to forecast market size for therapeutic indications.




5.3.4.1 Epidemiology-based Forecasting

The forecasting model used at GBI Research makes use of epidemiology data gathered from research publications and primary interviews with physicians to represent the treatment flow patterns for individual diseases and therapies. The market for any disease segment is directly proportional to the volume of units sold and the price per unit.

Sales = Volume of Units Sold Price per Unit

The volume of units sold is calculated on the average dosage regimen for that disease, duration of treatment and number of patients who are prescribed drug treatment (prescription population). Prescription population is calculated as the percentage of population diagnosed with a disease (diagnosis population). Diagnosis population is the population diagnosed with a disease expressed as a percentage of the population that is seeking treatment (treatment-seeking population). Prevalence of a disease (diseased population) is the percentage of the total population who suffer from a disease/condition.

Data on treatment seeking rate, diagnosis rate and prescription rate, if unavailable from research publications, are gathered from interviews with physicians and are used to estimate the patient volumes for the disease under consideration. Therapy uptake and compliance data are fitted in the forecasting model to account for patient switching and compliance behavior.

To account for differences in patient affordability of drugs across various geographies, macroeconomic data such as inflation and GDP; and healthcare indicators such as healthcare spending, insurance coverage and average income per individual are used.

Annual cost of treatment is calculated using product purchase frequency and the average price of the therapy. Product purchase frequency is calculated from the dosage data available for the therapies and drug prices are gathered from public sources.

The epidemiology-based forecasting model uses a bottom-up methodology and it makes use of estimations in the absence of data from research publications. Such estimations may result in a final market value which is different from the actual value. To correct this ‘gap’ the forecasting model uses ‘triangulation’ with the help of base year sales data (from company annual reports, internal and external databases) and sales estimations.

Analogous Forecasting Methodology

Analogous forecasting methodology is used to account for the introduction of new products, patent expiries of branded products and subsequent introduction of generics. Historic data for new product launches and generics penetration are used to arrive at robust forecasts. Increase or decrease of prevalence rates, treatment seeking rate, diagnosis rate and prescription rate are fitted into the forecasting model to estimate market growth rate.

The proprietary model enables GBI Research to account for the impact of individual drivers and restraints in the growth of the market. The year of impact and the extent of impact are quantified in the forecasting model to provide close-to-accurate data sets.

Diseased Population

The diseased population for any indication is the prevalence. The prevalence rates are usually obtained from various journals, online publications, sources such as the World Health Organization (WHO) or associations and foundation websites for that particular disease.

Diagnosis Population

Out of the patients who undergo diagnostic tests to confirm a disease, only a few people get diagnosed with the disease. This number as a percentage of the treatment seeking population is the diagnosis rate. The diagnosis population is primarily driven by the sensitivity of the diagnostic tests, state-of-the-art technology, patient access to these diagnostic tests and cost of the diagnostic tests.




Prescription Population

For any disease, multiple treatment options exist. For example, in cancer treatment various treatment options such as surgery, radiation therapy, and drug therapy are available. Prescription population is defined as the number of patients who are prescribed drug therapy. This is calculated as a percentage of the diagnosis population. The prescription population is primarily driven by the age at which the disease is diagnosed, the disease stage, patient health and cost of drug treatment.

5.3.4.2 Market Size by Geography

The treatment usage pattern and annual cost of treatment in each country has been factored in while deriving the individual country market size.

Forecasting Model for Therapeutic Areas

Figure 88: GBI Research Market Forecasting Model

D is ease P opu la ti onGener al Po pu la tio n 743,535 ,048

Q ualify ing c ond i tion 1 (Age/S ex/O c c upation e tc )Q ualify ing c ond i tion 2 (Age/S ex/O c c upation e tc )

Preva l ence t issu e valve disease 0 .2% 1,784 ,484

Q ualify ing c ond i tion (c om plic at ion, s eve rity)D IS EA SE D P OPU LA T IO N 1,784 ,484

T r eatm en t Flow P a tte rn sT reatm ent S eek i ng R at e (Sy m ptom s/ Dis A wareness ) 89% 1,588 ,191 Diagn os is Rat e (C lin ica l and D iagn ostic Tes ts ) 75% 1,191 ,143

Prescr iption R ate (Ph ysic ian P ercep t io n, Tr ea tm ent E ffec tiv e n ess )Tis s ue V a lve 70% 833 ,800 O the r T reatm ents fo r Va lve (Su r g/M ed/N one ) -

F ulfillm en tA vailab ilit y NAW illingness t o U s e (Pa tient Pe rc eptions) NAR eady to U se (S u rge ry e ligibi lity , R eus e etc ) NA

Affo rd abil it y a t PriceH E as % of G D P s pendA verage Inc om e (per ind iv idual)

P at ien t O ut-o f-poc ke t Budget (A nnua l)Budget al loc a tion to one-t ime s u rgery

Budget al loc a tion to o the r h ealt h needsA verage Pay or C overageP at ien t L iab il ity

Target Pr ic e (@ 20% pat liab)A SP for C os t o f T herapy

T O TA L P AT IEN T V OLU M E SPro du ct P urch ase Fr equ enc y 1

T OT A L U N IT VO LU MES

Pr ic in g per Un it 18 ,000$ In f lat ionP rice D ec reas e due to c om pet it ion

M ar ket Va lu e

G B I R esear ch M ar ke t Siz in g M o del

Source: GBI Research

The above figure represents a typical forecasting model followed in GBI Research. As discussed previously, the model is built on the treatment flow patterns. The model starts with the general population, then diseased population as a percentage of the general population and then follows the treatment seeking population as a percentage of the diseased population and diagnosed population as a percentage of the treatment seeking population. Finally, the total volume of units sold is calculated by multiplying the treated population by the average dosage per year per patient.

Articles from research journals and agency publications such as the British Medical Journal, New England Journal of Medicine, Oxford Journal, National Institute of Health and clinicaltrials.gov are used in the above model. The marketed drugs section is taken from company websites and internal databases




5.3.5 Geographical Landscape

GBI Research analyzes only seven major geographies: the US, the top five countries in Europe (the UK, Germany, France, Spain, and Italy) and Japan. The total market size for each country is provided which is the sum value of the market sizes of all the indications for that particular country.

5.3.6 Pipeline Analysis

This section provides a list of molecules at various stages in the pipeline for various indications. The list is sourced from internal database and validated for the accuracy of Phase and mechanism of action at ClinicalTrials.gov and company websites. The section also includes a list of promising molecules which is narrowed down based on the results of the clinical trials at various stages and the novelty of mechanism of action.

5.3.7 Competitive Landscape

Profiles of leading players are provided along with an overview of key products marketed by the companies for various indications. An analysis of strengths, weaknesses, opportunities and threats of each company with respect to various indications is also listed.

GBI Research aims to cover all major M&A, licensing deals and co-development deals related to the market. This section is sourced from the companies’ websites and internal databases.

5.3.8 Expert Panel Validation

GBI Research uses a panel of experts to cross-verify its databases and forecasts.

GBI Research’s expert panel comprises marketing managers, product specialists, international sales managers from medical device companies, academics from research universities, KOLs from hospitals, consultants from venture capital funds and distributors/suppliers of medical equipment and supplies.

Historic data and forecasts are relayed to GBI Research’s Expert Panel for feedback and adjusted in accordance with their feedback.

5.5 Disclaimer

All Rights Reserved.

No part of this publication may be reproduced, stored in a retrieval system or transmitted in any form by any means, electronic, mechanical, photocopying, recording or otherwise, without the prior permission of the publisher, GBI Research.

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