GBIHC335MR Critical Care Market to 2020 in Major Developed … · Table 4: Critical Care Market,...

Critical Care Therapeutics in Major Developed Markets to 2020 New and Late-Stage Four-Factor PCCs and Recombinant Products to Drive Market

GBI Research Report Guidance

© GBI Research. This is a licensed product and is not to be photocopied GBIHC335MR / Published JUL 2014

GBI Research Report Guidance

Chapter two gives an overview of the epidemiology, pathogenesis, diagnosis, and the therapeutic unmet need of critical care indications. The indications covered in this report are limited to those that are treated with albumin, anti-thrombin concentrates, factor XIII concentrates, fibrinogen concentrates and prothrombin complex concentrates.

Chapter three describes the therapeutic landscape of the critical care market covering marketed products in the eight major developed markets.

Chapter four provides an overview of the drugs in the critical care therapeutic pipeline. This includes in-depth analysis of the promising molecules in late-stage trials, which look set to make an impact on the market.

Chapter five confers revenue forecasts until 2020 for the critical care market in the eight major developed markets – the US, UK, France, Germany, Italy, Spain, Japan and Canada.

Chapter six addresses strategic consolidations and deals that took place in the global critical care market during the 2011–2013 period.


Executive Summary

Executive Summary

Critical Care Market to Reach $XX Billion in the Major Developed Markets by 2020

The critical care market in the eight major developed markets (the US, UK, France, Germany, Italy, Spain, Japan and Canada) was worth an estimated $XX billion in 2013 and is expected to grow to $XX billion by 2020 at a Compound Annual Growth Rate (CAGR) of XX%. The majority of this revenue was due to albumin sales which amounted to $XX billion in 2013 and are expected to grow to $XX billion by 2020. In terms of markets, the US held the largest share with XX% of the total critical care market in 2013 and is projected to grow at the highest rate of any market at XX% during the 2013–2020 period.

Albumin was used to treat the highest share of the critical care treatment population across all eight markets with XX million patients of a total of XX million in 2013. Expectedly, the highest share of these patients was in the US, a total of XX% in 2013. A further XX% of the 2013 treatment population were located in the top five countries of Europe, followed by Japan with XX% and rest from Canada. The treatment population in the US is estimated to grow at a CAGR of XX% until 2020 with highest growth expected in the number of patients treated with Prothrombin Complex Concentrate (PCC) which is expected to grow at a CAGR of XX%. Globally, the treatment population receiving Anti-Thrombin (AT) concentrates and PCCs is expected to be larger than any other category and will grow at a CAGR of XX% during the 2013–2020 period.

Critical Care, Major Developed Markets, Revenue ($bn), 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020

Rev

enue

($bn

)

Low variance Medium variance High variance Projected

Source: GBI Research, Proprietary Marketed Products Database

The projected high growth rate of revenues in the US market of XX% is mainly due the increased uptake of factor concentrates over older alternatives (such as Fresh-Frozen Plasma (FFP)), higher price inflation and the launch of new drugs such as Kcentra (PPC, human) and Tretten (Factor XIII concentrate, recombinant) in 2013. Kcentra is the first four-factor PCC in the US and Tretten is the first recombinant factor XIII concentrate. In the top five European critical care markets there have been no recent significant product launches and none are expected during the forecast period. These markets are therefore anticipated to grow slowly during the forecast period, the UK market will grow at a CAGR of XX%, followed by France at XX%, Germany and Spain will each grow at XX% and Italy at XX%.

US held the largest share with XX% of the total critical care market in 2013.


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Table of Contents

1 Table of Contents

1 Table of Contents ................................................................................................................................. 5 1.1 List of Tables ............................................................................................................................. 8 1.2 List of Figures............................................................................................................................ 9

2 Introduction........................................................................................................................................10 2.1 Albumin in Critical Care ............................................................................................................10

2.1.1 Overview .........................................................................................................................10 2.1.2 Diseases or Conditions Indicated for Albumin ....................................................................10 2.1.3 Epidemiology ...................................................................................................................11 2.1.4 Other Treatment Options .................................................................................................11 2.1.5 Treatment Patterns ..........................................................................................................11

2.2 Anti-Thrombin III Concentrates in Critical Care .........................................................................12 2.2.1 Overview .........................................................................................................................12 2.2.2 Anti-Thrombin Deficiencies ...............................................................................................12 2.2.3 Epidemiology ...................................................................................................................12 2.2.4 Treatment Options ...........................................................................................................13 2.2.5 Treatment Patterns ..........................................................................................................13

2.3 Factor XIII Concentrates in Critical Care ....................................................................................13 2.3.1 Overview .........................................................................................................................13 2.3.2 FXIII Deficiencies ..............................................................................................................14 2.3.3 Epidemiology ...................................................................................................................14 2.3.4 Treatment Options ...........................................................................................................14 2.3.5 Treatment Patterns ..........................................................................................................14

2.4 Fibrinogen Concentrates in Critical Care ...................................................................................15 2.4.1 Overview .........................................................................................................................15 2.4.2 Fibrinogen Deficiencies .....................................................................................................15 2.4.3 Epidemiology ...................................................................................................................16 2.4.4 Treatment Options ...........................................................................................................16 2.4.5 Treatment Patterns ..........................................................................................................16

2.5 Prothrombin Complex Concentrate in Critical Care ...................................................................17 2.5.1 Overview .........................................................................................................................17 2.5.2 Diseases or Conditions Indicated for Prothrombin Complex Concentrate ............................17 2.5.3 Epidemiology ...................................................................................................................18 2.5.4 Treatment Options ...........................................................................................................18 2.5.5 Treatment Patterns ..........................................................................................................19

3 Therapeutic Landscape .......................................................................................................................20 3.1 Albumin Products ....................................................................................................................20

3.1.1 Buminate (albumin, human) – Baxter ...............................................................................20 3.1.2 Zenalb (human albumin) – Bio Products Laboratory ..........................................................20 3.1.3 Human Albumin Biotest (albumin, human) – Biotest..........................................................20 3.1.4 AlbuRx (albumin) – CSL Behring ........................................................................................20 3.1.5 Albuminar (albumin, human) 5% – CSL Behring .................................................................21 3.1.6 Albutein (albumin (human)) – Grifols ................................................................................21 3.1.7 Albuked (human albumin) – Kedrion .................................................................................21 3.1.8 Vialebex (human albumin) – French Fractionation and Biotechnology Laboratory ..............22 3.1.9 Albunorm (human albumin) – Octapharma .......................................................................22 3.1.10 Alburaas (human albumin) – Shanghai RAAS.....................................................................22

3.2 Anti-Thrombin Concentrates ....................................................................................................22 3.2.1 Atryn (antithrombin alfa) – rEVO Biologics ........................................................................22 3.2.2 Thrombate III (AT III human) – Grifols ...............................................................................24 3.2.3 Anbinex (AT III human) – Grifols ........................................................................................24


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Table of Contents

3.2.4 Kybernin P (AT III human) – CSL Behring ............................................................................24 3.2.5 Aclotine (AT III, human) – French Fractionation and Biotechnology Laboratory ..................25 3.2.6 Anti-Thrombin III (AT III human) – Baxter ..........................................................................25 3.2.7 AT III Kedrion (AT III human) – Kedrion ..............................................................................25 3.2.8 Neuart (AT III, human) – Mitsubishi Tanabe ......................................................................25

3.3 Factor XIII Concentrates ...........................................................................................................26 3.3.1 Fibrogammin/Corifact (factor XIII) – CSL Behring ...............................................................26 3.3.2 NovoThirteen/Tretten (catridecacog) – Novo Nordisk ........................................................26

3.4 Fibrinogen Concentrates ..........................................................................................................27 3.4.1 Haemocomplettan P/Riastap (fibrinogen concentrate, human) – CSL Behring ....................27 3.4.2 Clottafact/Clottagen (fibrinogen concentrate, human) – French Fractionation and

Biotechnology Laboratory ................................................................................................28 3.4.3 Other Marketed Fibrinogen Concentrates .........................................................................28

3.5 Prothrombin Complex Concentrates .........................................................................................28 3.5.1 Beriplex P/N/Confidex/Kcentra (Prothrombin Complex Concentrate [Human]) – CSL Behring

........................................................................................................................................28 3.5.2 Bebulin VH (factor IX complex, vapor heated) – Baxter ......................................................28 3.5.3 Cofact (prothrombin complex concentrate) – Sanquin .......................................................29 3.5.4 Octaplex 500 (human prothrombin complex) – Octapharma ..............................................29 3.5.5 Profilnine SD – Grifols .......................................................................................................29 3.5.6 Uman Complex DI – Kedrion .............................................................................................29

4 Pipeline for Critical Care Market ..........................................................................................................30 4.1 Overview of Pipeline ................................................................................................................30 4.2 Promising Pipeline Candidates .................................................................................................30

4.2.1 Octaplex 500 (human prothrombin complex) – Octapharma ..............................................31 4.2.2 KW-3357– Kyowa Hakko Kirin...........................................................................................31 4.2.3 Human AT Concentrate – Grifols .......................................................................................31

5 Critical Care Market Forecast to 2020 ..................................................................................................32 5.1 Geographical Markets ..............................................................................................................32

5.1.1 Major Developed Markets ................................................................................................32 5.1.2 US....................................................................................................................................35 5.1.3 Top Five European Countries ............................................................................................37 5.1.4 Japan ...............................................................................................................................40 5.1.5 Canada ............................................................................................................................42

5.2 Drivers and Barriers .................................................................................................................45 5.2.1 Drivers .............................................................................................................................45 5.2.2 Barriers ............................................................................................................................46

6 Deals and Strategic Consolidations ......................................................................................................48 6.1 Major Deals .............................................................................................................................48

6.1.1 Octapharma Enters Into an Agreement with Pfizer ............................................................48 6.1.2 Grifols to Acquire Blood Transfusion Diagnostics Business from Novartis ...........................48 6.1.3 Shin Poong Pharmaceutical and French Fractionation and Biotechnology Laboratory form a

Korea-based Bio-pharmaceutical Joint Venture .................................................................48 6.1.4 Bain Capital Acquires 80% Stake in Plasma Resources UK ..................................................48 6.1.5 Biomat Acquires Three Plasma Donation Centers from Cangene ........................................49 6.1.6 BioRx Enters into Co-Marketing Agreement with CSL Behring for Corifact ..........................49 6.1.7 Baxter Announces Collaboration with Sanquin to Expand Capacity for Plasma Therapies ...49 6.1.8 Shire Enters into Licensing Agreement with Sangamo BioSciences .....................................49 6.1.9 Baxter Completes Acquisition of Rights to OBI-1 and Related Assets from Ipsen and

Inspiration Biopharma ......................................................................................................49 7 Critical Care Market to 2020 – Appendix ..............................................................................................51

7.1 Abbreviations ..........................................................................................................................51


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Table of Contents

7.2 References ..............................................................................................................................52 7.3 Pipeline Products .....................................................................................................................55 7.4 Market Forecasts to 2020 ........................................................................................................56

7.4.1 Major Developed Markets ................................................................................................56 7.4.2 US....................................................................................................................................57 7.4.3 UK ...................................................................................................................................59 7.4.4 France .............................................................................................................................60 7.4.5 Germany ..........................................................................................................................62 7.4.6 Italy .................................................................................................................................63 7.4.7 Spain ...............................................................................................................................65 7.4.8 Japan ...............................................................................................................................66 7.4.9 Canada ............................................................................................................................68

7.5 Research Methodology ............................................................................................................69 7.5.1 Coverage .........................................................................................................................69 7.5.2 Secondary Research .........................................................................................................70 7.5.3 Primary Research .............................................................................................................70 7.5.4 Therapeutic Landscape .....................................................................................................70 7.5.5 Geographical Landscape ..................................................................................................72 7.5.6 Pipeline Analysis...............................................................................................................73

7.6 Expert Panel Validation ............................................................................................................73 7.7 Contact Us ...............................................................................................................................73 7.8 Disclaimer................................................................................................................................73


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Table of Contents

1.1 List of Tables

Table 1: Critical Care Market, Atryn, Development Timeline, 2007–2013 ...............................................23 Table 2: Critical Care Market, Global, Pharmaceutical Pipeline, 2014 .....................................................55 Table 3: Critical Care Market, Major Developed Markets, Market Forecast, 2013–2020 .........................56 Table 4: Critical Care Market, Major Developed Markets, Albumin, Market Forecast, 2013–2020 ..........56 Table 5: Critical Care Market, Major Developed Markets, Anti-Thrombin Concentrates, Market Forecast,

2013–2020 .............................................................................................................................56 Table 6: Critical Care Market, Major Developed Markets, FXIII Concentrates, Market Forecast, 2013–

2020 .......................................................................................................................................56 Table 7: Critical Care Market, Major Developed Markets, Fibrinogen Concentrates, Market Forecast,

2013–2020 .............................................................................................................................57 Table 8: Critical Care Market, Major Developed Markets, Prothrombin Complex Concentrates, Market

Forecast, 2013–2020 ...............................................................................................................57 Table 9: Critical Care Market, US, Market Forecast, 2013–2020 .............................................................57 Table 10: Critical Care Market, US, Albumin, Market Forecast, 2013–2020 ..............................................57 Table 11: Critical Care Market, US, Anti-Thrombin Concentrates, Market Forecast, 2013–2020 ...............58 Table 12: Critical Care Market, US, FXIII Concentrates, Market Forecast, 2013–2020 ...............................58 Table 13: Critical Care Market, US, Fibrinogen Concentrates, Market Forecast, 2013–2020 .....................58 Table 14: Critical Care Market, US, Prothrombin Complex Concentrates, Market Forecast, 2013–2020 ....58 Table 15: Critical Care Market, UK, Market Forecast, 2013–2020 ............................................................59 Table 16: Critical Care Market, UK, Albumin, Market Forecast, 2013–2020 ..............................................59 Table 17: Critical Care Market, UK, Anti-Thrombin Concentrates, Market Forecast, 2013–2020 ...............59 Table 18: Critical Care Market, UK, FXIII Concentrates, Market Forecast, 2013–2020 ...............................59 Table 19: Critical Care Market, UK, Fibrinogen Concentrates, Market Forecast, 2013–2020 .....................60 Table 20: Critical Care Market, UK, Market Forecast, Prothrombin Complex Concentrates, 2013–2020 ....60 Table 21: Critical Care Market, France, Market Forecast, 2013–2020 .......................................................60 Table 22: Critical Care Market, France, Albumin, Market Forecast, 2013–2020 ........................................60 Table 23: Critical Care Market, France, Anti-Thrombin Concentrates, Market Forecast, 2013–2020 .........61 Table 24: Critical Care Market, France, FXIII Concentrates, Market Forecast, 2013–2020 .........................61 Table 25: Critical Care Market, France, Fibrinogen Concentrates, Market Forecast, 2013–2020 ...............61 Table 26: Critical Care Market, France, Prothrombin Complex Concentrates, Market Forecast, 2013–2020

...............................................................................................................................................61 Table 27: Critical Care Market, Germany, Market Forecast, 2013–2020 ...................................................62 Table 28: Critical Care Market, Germany, Albumin, Market Forecast, 2013–2020 ....................................62 Table 29: Critical Care Market, Germany, Anti-Thrombin Concentrates, Market Forecast, 2013–2020 ......62 Table 30: Critical Care Market, Germany, FXIII Concentrates, Market Forecast, 2013–2020 .....................62 Table 31: Critical Care Market, Germany, Fibrinogen Concentrates, Market Forecast, 2013–2020 ............63 Table 32: Critical Care Market, Germany, Prothrombin Complex Concentrates, Market Forecast, 2013–

2020 .......................................................................................................................................63 Table 33: Critical Care Market, Italy, Market Forecast, 2013–2020 ..........................................................63 Table 34: Critical Care Market, Italy, Albumin, Market Forecast, 2013–2020 ............................................63 Table 35: Critical Care Market, Italy, Anti-Thrombin Concentrates, Market Forecast, 2013–2020 .............64 Table 36: Critical Care Market, Italy, FXIII Concentrates, Market Forecast, 2013–2020 .............................64 Table 37: Critical Care Market, Italy, Fibrinogen Concentrates, Market Forecast, 2013–2020 ...................64 Table 38: Critical Care Market, Italy, Prothrombin Complex Concentrates, Market Forecast, 2013–2020 ..64 Table 39: Critical Care Market, Spain, Market Forecast, 2013–2020.........................................................65 Table 40: Critical Care Market, Spain, Albumin, Market Forecast, 2013–2020 ..........................................65 Table 41: Critical Care Market, Spain, Anti-Thrombin Concentrates, Market Forecast, 2013–2020 ...........65 Table 42: Critical Care Market, Spain, FXIII Concentrates, Market Forecast, 2013–2020 ...........................65 Table 43: Critical Care Market, Spain, Fibrinogen Concentrates, Market Forecast, 2013–2020 .................66 Table 44: Critical Care Market, Spain, Prothrombin Complex Concentrates, Market Forecast, 2013–2020 66 Table 45: Critical Care Market, Japan, Market Forecast, 2013–2020 ........................................................66 Table 46: Critical Care Market, Japan, Albumin, Market Forecast, 2013–2020..........................................66 Table 47: Critical Care Market, Japan, Anti-Thrombin Concentrates, Market Forecast, 2013–2020 ...........67 Table 48: Critical Care Market, Japan, FXIII Concentrates, Market Forecast, 2013–2020 ...........................67 Table 49: Critical Care Market, Japan, Fibrinogen Concentrates, Market Forecast, 2013–2020 .................67 Table 50: Critical Care Market, Japan, Prothrombin Complex Concentrates, Market Forecast, 2013–202067 Table 51: Critical Care Market, Canada, Market Forecast, 2013–2020......................................................68


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Table of Contents

Table 52: Critical Care Market, Canada, Albumin, Market Forecast, 2013–2020 .......................................68 Table 53: Critical Care Market, Canada, Anti-Thrombin Concentrates, Market Forecast, 2013–2020 ........68 Table 54: Critical Care Market, Canada, FXIII Concentrates, Market Forecast, 2013–2020 ........................68 Table 55: Critical Care Market, Canada, Fibrinogen Concentrates, Market Forecast, 2013–2020 ..............69 Table 56: Critical Care Market, Canada, Prothrombin Complex Concentrates, Market Forecast, 2013–2020

...............................................................................................................................................69

1.2 List of Figures

Figure 1: Critical Care Market, Global, Overall Pipeline Analysis (%), 2013 ...............................................30 Figure 2: Critical Care Market, Major Developed Markets, Treatment Use Patterns, 2013–2020..............33 Figure 3: Critical Care Market, Major Developed Markets, Revenue ($bn), 2013–2020 ............................34 Figure 4: Critical Care Market, US, Treatment Use Patterns, 2013–2020 .................................................35 Figure 5: Critical Care Market, US, Annual Cost of Therapy and Revenue, 2013–2020 .............................36 Figure 6: Critical Care Market, Top Five European Countries, Treatment Use Patterns, 2013 and 2020 ....38 Figure 7: Critical Care Market, Top Five European Countries, Annual Cost of Therapy ($), 2013–2020 .....39 Figure 8: Critical Care Market, Top Five European Countries, Revenue ($m), 2013–2020 .........................40 Figure 9: Critical Care Market, Japan, Treatment Use Patterns, 2013–2020 .............................................41 Figure 10: Critical Care Market, Japan, Annual Cost of Therapy and Revenue, 2013–2020 .........................42 Figure 11: Critical Care Market, Canada, Treatment Use Patterns, 2013–2020 ..........................................43 Figure 12: Critical Care Market, Canada, Annual Cost of Therapy and Revenue, 2013–2020 ......................44 Figure 13: GBI Research Market Forecasting Model .................................................................................72


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Introduction

2 Introduction

2.1 Albumin in Critical Care

2.1.1 Overview

Albumin, a low-molecular-weight protein, is produced in the liver and accounts for around XX% of total human plasma mass. It is fundamental to the maintenance of blood volume and the stability of blood circulation and has a serum half-life of approximately XX days. Around XX–XX grams (g) of albumin is present in one liter (l) of blood plasma with the normal range of albumin concentration occurring at XX–XXg per deciliter (dl).

Large blood or plasma loss following traumatic injury or in high-risk clinical settings could lead to hypotension (low blood pressure), necessitating the replacement of plasma volume with albumin. Albumin plays an important role in the management of many clinically significant high-risk conditions such as cardiopulmonary bypass surgery, burn cases, acute liver failure, or in other critically ill patients. It can also be used in the emergency treatment of shock and in other similar conditions where blood volume must be urgently restored.

2.1.2 Diseases or Conditions Indicated for Albumin

2.1.2.1 Albumin in Hypoalbuminemia

Low levels of blood albumin cause albumin deficiency or hypoalbuminemia. Hypoalbuminemia may result in generalized edema (swelling) via a decline in oncotic pressure and usually correlates with increased morbidity and mortality in critically ill patients. Disease conditions such as liver disease (failure to synthesize albumin), excessive loss of albumin (through protein-losing enteropathy or in burn patients), and nephrotic syndrome (increased excretion of albumin in the urine) could result in hypovolemia and/or hypoalbuminemia. A low serum-albumin level may be indicative of liver failure or other liver diseases such as cirrhosis or chronic hepatitis, or possibly an indicator of chronic malnutrition or protein-losing enteropathy.

Hypoalbuminemia is a common problem among patients with acute and chronic conditions. It has been reported that around XX% of all admitted patients with acute or chronic conditions have hypoalbuminemia at the time of hospital admission (Peralta, 2012; Vincent, 2003). Albumin is used in the treatment of hypoalbuminemia. The effect would be temporary if the underlying conditions causing hypoalbuminemia were not reversed, but it would serve as a useful adjunct in cases linked with severe injuries, infections or pancreatitis, which cannot be quickly reversed.

2.1.2.2 Albumin in Hypovolemia

The usefulness of albumin in reversing hypovolemia is primarily dependent upon its ability to draw interstitial fluid into the systemic circulations. Although alternatives to albumin (such as crystalloid solutions and colloid-containing plasma substitutes) can be used in the emergency treatment of shock and other critical conditions, the use of albumin is preferable, by virtue of its efficacy and prolonged intravascular half-life.

Human albumin can be used to treat severe hypovolemia, which usually occurs during the first few months of pregnancy, however, there have been no controlled studies on the volume of replacement required to correct such hypovolemia. Albumin use in such cases should therefore be used with caution. The effectiveness of human albumin for the management of hypovolemia in trauma patients has not been shown to have better results in terms of survival rate in comparison with other alternatives such as crystalloids or any other colloidal volume substitutes (Boldt, 2010).

2.1.2.3 Albumin in Cardiopulmonary Bypass

The use of albumin has been suggested prior to or during cardiopulmonary bypass surgery. Although its advantage over crystalloid solutions has not yet been clearly established, a retrospective analysis has appeared to demonstrate a lower mortality rate in cardiac surgery patients with the use of human albumin (Boldt, 2010).


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Pipeline for Critical Care Market

4 Pipeline for Critical Care Market

4.1 Overview of Pipeline

The critical care pipeline is considerably weak with very few innovative drugs expected to be launched during the forecast period. There are some currently marketed products that are expected to be approved as line extensions for new indications or in new clinical settings. The largest share of pipeline drugs, especially in late-stage trials, are fibrinogen concentrates, indicated for congenital or acquired factor I or fibrinogen deficiency. A proportion of XX% of the critical care pipeline is fibrinogen concentrates followed by albumin at XX%. A total of XX% of the pipeline products is in Phase III of development followed by XX% in discovery Phase.

CSL’s Haemocomplettan P (also marketed as Kcentra or Beriplex), which is a widely used fibrinogen concentrate, is currently under phase three clinical trials for fibrinogen deficiency in complex cardiac surgery in collaboration with Isala Klinieken (CSL Behring, NCT01124981). CSL’s fibrinogen concentrate is also under investigation for surgical or post-operative blood loss (CSL Behring, NCT01475669). Four other leading companies Biotest, Grifols, LFB and Octapharma also have fibrinogen concentrates in late-stage trials.

Octapharma’s Octaplex, which is already marketed in the EU and Australia, is currently in late-stage development in the US and would be the second PCC to be approved in the US after Kcentra. Kyowa Hakko Kirin, the Japanese drug maker has a novel fucose-deficient recombinant AT preparation in late-stage development in Japan, expected to be launched in 2014. The product is in Phase I in EU clinical trials.

Figure 1: Critical Care Market, Global, Overall Pipeline Analysis (%), 2013

Albumin

Fibrinogen concentrates

AT concentrates

Prothrombin complex

concentrates

Pipeline by type of product

n = XX

Discovery

Phase I

Preclinical

Pre-registrationPhase II

Phase III

Pipeline by stage of development

n = XX

Source: GBI Research, Proprietary Pipeline Products Database

Abbreviations: IND: Investigational New Drug; CTA: Clinical Trial Applications


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Critical Care Market Forecast to 2020

5.1.3.2 Annual Cost of Therapy

The ACoT for the critical care market in top five EU is an average of the ACoT for the five blood product categories in these five countries and is calculated based on the total revenues from the five categories divided by the total treatment population. The ACoT for products in critical care markets in the top five EU countries was estimated to be $XX in 2013 and is expected to increase at a CAGR of XX% to reach $XX by 2020. Lower levels of inflation and a lack of significant product launches have contributed towards a slow growth rate. The highest growth in ACoT is expected to occur in FXIII products at a CAGR of XX% due to increased uptake of NovoThirteen, the recombinant FXIII for prophylactic treatment during the forecast period as it has a higher cost than plasma-derived products. Albumin ACoT is also expected to increase at a CAGR of XX% during the forecast period as the demand for albumin is expected to increase in some of the regions. The ACoT for prophylactic treatment with fibrinogen concentrates is highest in Europe with $XX in 2013 followed by prophylactic treatment with rFXIII concentrates at $XX.

Figure 7: Critical Care Market, Top Five European Countries, Annual Cost of Therapy ($), 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020

AC

oT($

)

A) UK

2013 2014 2015 2016 2017 2018 2019 2020

AC

oT($

)

B) Germany

2013 2014 2015 2016 2017 2018 2019 2020

AC

oT($

)

C) France

2013 2014 2015 2016 2017 2018 2019 2020

AC

oT ($

)D) Italy

2013 2014 2015 2016 2017 2018 2019 2020

AC

oT($

)

E) Spain

Low variance

Medium variance

High variance

Projected

Source: GBI Research, Proprietary Marketed Products Database


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Critical Care Market to 2020 – Appendix

7 Critical Care Market to 2020 – Appendix

7.1 Abbreviations

AcoT: Annual Cost of Therapy

AMBAR: Alzheimer Management By Amyloid Removal

ARDS: Adult Respiratory Distress Syndrome

AT: Anti-Thrombin

BCSH: British Committee For Standards In Hematology

BLA: Biologics License Application

CAGR: Compound Annual Growth Rate

CTA: Clinical Trial Agreement

DIC: Disseminated Intravascular Coagulation

dl: deciliter

EU: European Union

EU-5: Top Five Countries in European Union

EMA: European Medicine Agency

F: Factor

FDA: Food and Drug Administration

FEIBA: Factor Eight Inhibitor Bypass Activity

g: grams

HES: Hydroxyethyl Starch

IND: Investigational New Drug

INR: International Normalized Ratio

IU: International Unit

IV: Intravenous

JV: Joint Venture

kg: kilograms

LFB: Laboratoire Français Du Fractionnement Et Des Biotechnologies

LMWH: Low-Molecular-Weight Heparin

mg: milligrams

ml: milliliter

PCC: Prothrombin Complex Concentrate

PPH: Postpartum Hemorrhage Treatment & Management

PT: Prothrombin Time

PRUK: Plasma Resources UK

rFVIIa: Recombinant Activated Factor VII

rFXIII: Recombinant Factor XIII Concentrate

rFXIIIa: Recombinant Factor XIII A-Subunit

SAFE: Saline Versus Albumin Fluid Evaluation


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SD: Solvent Detergent

VTE: Venous Thromboembolism

ZFP: Zinc Finger DNA-binding Protein

7.2 References

Acharya, S.S. and DiMichelle, M., (2008). Rare inherited disorders of fibrinogen. Hemophilia. Vol.: 14 (6), pp: 1151–1158

Aird, W., (2003). The Hematologic System as a Marker of Organ Dysfunction in Sepsis. Mayo Clinical Process; vol.: 78, pp: 869–881

Bershad E, et al. (2009). Prothrombin Complex Concentrates for Oral Anticoagulant Therapy-Related Intracranial Hemorrhage: A Review of the Literature. Neurocritical Care; 12(3): 403–413

Board P.G. et al., (1993). Factor XIII: inherited and acquired deficiency. Blood Review, Vol.: 7(4), pp: 229–42.

Boldt J (2010). Use of albumin: an update. British Journal of Anesthesia; 104(3): 276–284.

Boldt J, Papsdorf M. (2008). Fluid management in burn patients: results from a European survey – more questions than answers. Burns; Vol.: 34(3); pp:328-38

BPRO (2013). Blood Program in Japan. Blood Products Research Organization. Available from http://bpro.or.jp/english/pdf_annual/BPRO2013.pdf

Calizzani, G. et al., (2013). The evolution of the regulatory framework for the plasma and plasma-derived medicinal products system in Italy. Blood Transfusion; vol.: 11(4), pp: 6–12.

CIHI (2013). Highlights of 2011–2012 Emergency Department Visits and Inpatient Hospitalizations. Canadian Institute for Health Information (CIHI). Available from: https://secure.cihi.ca/free_products/DAD-NACRS_Quick%20Stats_Highlights_2011-2012_EN_web.pdf Cochrane Injuries Group Albumin Reviewers (1998). Cochrane Injuries Group Albumin Reviewers. Why albumin may not work. British Medical Journal; pp: 317:235

CSL Behring, Haemocomplettan® P During Elective Complex Cardiac Surgery, NCT01124981. http://http://clinicaltrials.gov/ct2/show/NCT01124981 Accessed on [April 29, 2012]

CSL Behring, Study of Fibrinogen Concentrate (Human) (FCH) to Control Bleeding During Complex Cardiovascular Surgery (REPLACE), NCT01475669. http://clinicaltrials.gov/show/NCT01475669 Accessed on [April 29, 2012]

CSL Behring, Fibrinogen Concentrate In Children After Cardiac Surgery (FiCCS), NCT01187225. http://clinicaltrials.gov/ct2/show/NCT01187225 Accessed on [July 01, 2012]

CSL Behring, The ZEro PLASma Trial (ZEPLAST): Avoidance of Fresh Frozen Plasma in Cardiac Surgery, NCT01471730. http://clinicaltrials.gov/ct2/show/NCT01471730 Accessed on [July 01, 2012]

DeBois, W. et al., (2005). Cardiopulmonary Bypass in Patients With Pre-existing Coagulopathy. The Journal of The American Society of Extra-Corporeal Technology; vol.: 37, pp: 15–22

Farrugia, A and Cassar, J., (2012). Plasma-derived medicines: access and usage issues. Blood Transfusion; vol.: 10(3) pp: 273–278

Franchini, M and Lippi, G (2012). Fibrinogen replacement therapy: a critical review of the literature. Blood Transfusion; 10(1): 23–27

Gando, S. et al., (2013). A randomized, controlled, multicenter trial of the effects of Anti-Thrombin on disseminated intravascular coagulation in patients with sepsis. Critical Care, vol.: 17: R297; doi:10.1186/cc13163

Garcia, D.A., and Crowther, M.A. (2012). Clinician Update: Reversal of Warfarin – Case-Based Practice Recommendations. Circulation; 125: 2944-2947

Grazzini, G., Mannucci, P.M., and Oleari, F. (2013). Plasma-derived medicinal products: demand and clinical use. Blood Transfusion 2013; vol.: 11 (4), pp: s2–5


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7.3 Pipeline Products

Table 2: Critical Care Market, Global, Pharmaceutical Pipeline, 2014

Drug/project name Company Stage of development

Indication Molecule type

Source: GBI Research, Proprietary Pipeline Products Database [Accessed: April 10, 2014]


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7.4 Market Forecasts to 2020

7.4.1 Major Developed Markets

7.4.1.1 Critical Care Market

Table 3: Critical Care Market, Major Developed Markets, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)

Treatment population (million)

Maximum revenue ($bn)

Projected revenue ($bn)

Minimum revenue ($bn)

Source: GBI Research, Proprietary Marketed Products Database [Accessed: April 14, 2014]

7.4.1.2 Albumin in Critical Care

Table 4: Critical Care Market, Major Developed Markets, Albumin, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($bn)


7.4.1.3 Anti-Thrombin Concentrates in Critical Care

Table 5: Critical Care Market, Major Developed Markets, Anti-Thrombin Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR

(%)

Treatment population (‘000)

ACoT ($)

Revenue ($m)


7.4.1.4 FXIII Concentrates in Critical Care

Table 6: Critical Care Market, Major Developed Markets, FXIII Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR

(%)

Treatment population

ACoT ($)

Revenue ($m)



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7.4.1.5 Fibrinogen Concentrates in Critical Care

Table 7: Critical Care Market, Major Developed Markets, Fibrinogen Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Projected revenue ($m)


7.4.1.6 Prothrombin Complex Concentrates in Critical Care

Table 8: Critical Care Market, Major Developed Markets, Prothrombin Complex Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR

(%)


ACoT ($)



7.4.2 US


Table 9: Critical Care Market, US, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Maximum revenue ($m)


Minimum revenue ($m)



Table 10: Critical Care Market, US, Albumin, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



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Table 11: Critical Care Market, US, Anti-Thrombin Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 12: Critical Care Market, US, FXIII Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 13: Critical Care Market, US, Fibrinogen Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)




Table 14: Critical Care Market, US, Prothrombin Complex Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)




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7.4.3 UK


Table 15: Critical Care Market, UK, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)






Table 16: Critical Care Market, UK, Albumin, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 17: Critical Care Market, UK, Anti-Thrombin Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 18: Critical Care Market, UK, FXIII Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



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Table 19: Critical Care Market, UK, Fibrinogen Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR

(%)


ACoT ($)




Table 20: Critical Care Market, UK, Market Forecast, Prothrombin Complex Concentrates, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)



7.4.4 France


Table 21: Critical Care Market, France, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)






Table 22: Critical Care Market, France, Albumin, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



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Table 23: Critical Care Market, France, Anti-Thrombin Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 24: Critical Care Market, France, FXIII Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 25: Critical Care Market, France, Fibrinogen Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR

(%)


ACoT ($)



7.4.4.6 PCCs in Critical Care

Table 26: Critical Care Market, France, Prothrombin Complex Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)




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7.4.5 Germany


Table 27: Critical Care Market, Germany, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)






Table 28: Critical Care Market, Germany, Albumin, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 29: Critical Care Market, Germany, Anti-Thrombin Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 30: Critical Care Market, Germany, FXIII Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR

(%)


ACoT ($)

Revenue ($m)



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Table 31: Critical Care Market, Germany, Fibrinogen Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR

(%)


ACoT ($)




Table 32: Critical Care Market, Germany, Prothrombin Complex Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)



7.4.6 Italy


Table 33: Critical Care Market, Italy, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)






Table 34: Critical Care Market, Italy, Albumin, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



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Table 35: Critical Care Market, Italy, Anti-Thrombin Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 36: Critical Care Market, Italy, FXIII Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 37: Critical Care Market, Italy, Fibrinogen Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR

(%)


ACoT – on demand ($)




Table 38: Critical Care Market, Italy, Prothrombin Complex Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)




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7.4.7 Spain


Table 39: Critical Care Market, Spain, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)






Table 40: Critical Care Market, Spain, Albumin, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 41: Critical Care Market, Spain, Anti-Thrombin Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 42: Critical Care Market, Spain, FXIII Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



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Table 43: Critical Care Market, Spain, Fibrinogen Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)




Table 44: Critical Care Market, Spain, Prothrombin Complex Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)



7.4.8 Japan


Table 45: Critical Care Market, Japan, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)






Table 46: Critical Care Market, Japan, Albumin, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



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Table 47: Critical Care Market, Japan, Anti-Thrombin Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 48: Critical Care Market, Japan, FXIII Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 49: Critical Care Market, Japan, Fibrinogen Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR

(%)


ACoT ($)




Table 50: Critical Care Market, Japan, Prothrombin Complex Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)




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7.4.9 Canada


Table 51: Critical Care Market, Canada, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)






Table 52: Critical Care Market, Canada, Albumin, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 53: Critical Care Market, Canada, Anti-Thrombin Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



Table 54: Critical Care Market, Canada, FXIII Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)

Revenue ($m)



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Table 55: Critical Care Market, Canada, Fibrinogen Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR

(%)


ACoT ($)




Table 56: Critical Care Market, Canada, Prothrombin Complex Concentrates, Market Forecast, 2013–2020

2013 2014 2015 2016 2017 2018 2019 2020 CAGR (%)


ACoT ($)



7.5 Research Methodology

GBI Research’s dedicated research and analysis teams consist of experienced professionals with marketing, market research and consulting backgrounds in the pharmaceutical 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.

7.5.1 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 extensive 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.

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

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

The coverage is further streamlined and strengthened with additional inputs from GBI Research’s expert panel.


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7.5.2 Secondary Research

The research process begins with extensive secondary research using internal and external sources to gather 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

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

7.5.3 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:

Provides first-hand information on market size, market trends, growth trends, competitive landscape and future outlook

Helps to validate and strengthen secondary research findings

Further develops the analysis team’s expertise and market understanding

Primary research involves email correspondence, telephone interviews and face-to-face interviews for each market, category, segment and sub-segment across a range of geographies.

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

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

Distributors, paramedics and representatives from hospital stores, laboratories and pharmacies

Outside experts: investment bankers, valuation experts, research analysts specializing in specific pharmaceutical markets

Key opinion leaders: physicians and surgeons that specialize in the therapeutic areas

7.5.4 Therapeutic Landscape

Revenues for each indication, geographically, are arrived at by utilizing the GBI Research market-forecasting model. The global revenue for each indication is the sum value of revenues of all eight regions.

The annual cost of therapy for each indication is arrived at by considering the cost and 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-use 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.

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


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The forecasting model used by 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 x 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 that are prescribed drug treatment (prescription population).

Prescription population is calculated as the percentage of the 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 that suffers from a disease/condition.

Data on the 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 into the forecasting model to account for patient switching and compliance behavior.

To account for differences in patient affordability of drugs across various geographies, macro-economic 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 therapy 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 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 the subsequent introduction of generics. Historical data for new product launches and generics penetration are used to arrive at robust forecasts. Increase or decrease in prevalence rate, 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 on the growth of the market. The year 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 or associations and foundation websites for that particular disease.

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. The prescription population is defined as the number of patients that 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 the cost of drug treatment.


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7.5.4.1 Market Size by Geography

The treatment-use pattern and annual cost of therapy in each country has been factored in when deriving the individual country market size.

Forecasting Model for Therapeutic Areas

Figure 13: GBI Research Market Forecasting Model

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

Q ualif ying c ondition 1 (Age/S ex/O c c upa tion e tc )Q ualif ying c ondition 2 (Age/S ex/O c c upa tion e tc )

Preva l ence t issu e va lve disease 0 .2% 1,784,484 Q ualif ying c ondition (c om plic at ion , s eve rity)DIS EA SE D P OPU LA T IO N 1,784,484

T r eatm en t Flow P atte rn sT rea tm ent S eeki ng R ate (Sy m ptom s / Dis Awareness ) 89% 1,588,191 Diagn osis Rat e ( C lin ical and D iagn os tic Test s ) 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 alve 70% 833,800 O the r T rea tm en ts fo r Valve (Sur g /M ed/N one ) -

F ulfi llm en tA vai labi lit y NAW illingness to Us e (Pa tient Pe rc ep tions) NAReady to U se (S u rgery e lig ib ility , R eus e e tc ) NA

Affo rd abil it y at PriceHE as % of G DP s pendA verage Inc om e (per individual)P at ien t O u t-o f-poc ke t Budge t (A nnua l)

Budget alloc a tion to one-t im e s u rgeryBudget alloc a tion to o the r h ealth needs

A verage Pay or CoverageP at ien t L iabi lityTarge t Pr ic e ( @20% pa t liab )A SP for Cos t of T herapy

T O TA L P AT IEN T V OLUM E SPro du ct P urch ase Fr equ en c y 1

T OT A L UN IT VO LU M ES

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 Valu e

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

Source: GBI Research

The preceding figure represents a typical forecasting model followed by GBI Research. As discussed previously, the model is built on treatment flow patterns. The model starts with the general population, then the 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.

7.5.5 Geographical Landscape

GBI Research analyzes eight major geographies: the US, the top five countries in Europe (the UK, Germany, France, Spain and Italy),Japan and Canada. 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. The maximum and minimum estimated market sizes are then provided by adjusting all variables expected to impact upon the market during the forecast period to provide the best-case and worst-case scenarios.


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Articles from research journals and agency publications, National Institute of Health and ClinicalTrials.gov are the source of data for the estimation of market size and making forecasts.

7.5.6 Pipeline Analysis

This section provides a list of molecules at various stages in the pipeline for various indications. The list is sourced from an internal database and validated for 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. The latest press releases issued by the companies and news reports are also used to source information for the status of the molecule in the pipeline. This list of pipeline molecules, in conjunction with a list of ongoing and completed clinical trials, is analyzed in this section, and a breakdown of pipeline molecules and clinical trials by Phase and molecule type is provided.

7.6 Expert Panel Validation

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

GBI Research expert panel comprises marketing managers, product specialists, international sales managers from pharmaceutical companies, academics from research universities and key opinion leaders from hospitals.

Historical data and forecasts are relayed to GBI Research’s expert panel for feedback and are adjusted in accordance with their feedback.

7.8 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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