Stakeholder Opinions: Pulmonary Arterial Hypertension clinical community builds order from chaos

Published by: Datamonitor

Published: Mar. 19, 2009 - 134 Pages

Table of Contents

ABOUT DATAMONITOR HEALTHCARE

About the cardiovascular analysis team

CHAPTER 1 EXECUTIVE SUMMARY

Strategic scoping and focus

Datamonitor insight into the disease market

Related current reports

Related future reports

CHAPTER 2 MARKET DEFINITION

Market definition for this report

Geographic coverage

Defining pulmonary arterial hypertension

CHAPTER 3 DISEASE BACKGROUND

The short and animated history of pulmonary arterial hypertension

Aminorex flags a problem

WHO symposium drives hunt for definitions

National Institutes of Health registry sets the standard

Evian creates structure, IPPHS and SNAP offer etiological insight

Venice improves standardization, maps future directions

Dana Point: consensus and contention

Classification - largely unchanged but pre-PAH pondered

Genetics still too nebulous, but prognostic and risk factors at the fore

Exponential rise in commercial interest

Modest beginnings

The drug development surge

PAH-specific treatments

Marketed drug summaries

Endothelin receptor antagonists

Prostacyclin agonists

Phosphodiesterase-5 inhibitors

CHAPTER 4 THE CURRENT CLINICAL PARADIGM

Introduction

Earlier diagnosis:

The clinical community:

The broader patient potential:

A diagnosis of exclusion

Lengthy, costly, risky diagnoses of exclusion: ideal for missing cases?

Few immediate prospects for improved diagnostic capabilities

Experimentation, documentation, standardization

The importance of the clinician power-base

The evolution of consensus

Compare and contrast: European and US guidelines

Expanding patient population or expanding at-risk population?

A miniscule patient group diagnosed too late?

Pre-PAH: increasing the identified at-risk population?

CHAPTER 5 DEMANDS OF THE FUTURE PARADIGM

Improved outcomes - with data or without it

EU and US guideline convergence?

Clear guidance on product positioning essential

Balancing pressure on trial design against reasonable expectation

New approaches and treatments

Triage and selectivity are the watchwords

The commercial expectation

Pipeline overview

BIBLIOGRAPHY

Journal papers

Presentations

Website information

Books

Datamonitor reports

APPENDIX A - EPIDEMIOLOGICAL ASSUMPTIONS

Total population data

Incidence and prevalence

Incidence

Prevalence

Functional class at time of diagnosis

Etiology and functional class/etiology

Gender and gender/etiology

Patient potential

Note on data limitations

APPENDIX B

Contributing experts

Report methodology

About Datamonitor

About Datamonitor Healthcare

About the Cardiovascular Disease analysis team

Disclaimer

List of Tables

Table 1: Clinical classifications of pulmonary arterial hypertension - Venice, 2003

Table 2: New York Heart Association/World Health Organisation classification of functional status of patients with pulmonary arterial hypertension

Table 3: Overview of marketed products for pulmonary arterial hypertension, 2009

Table 4: Drug development activity and active approvals per year - pulmonary arterial hypertension versus select breakthrough indications, in the seven major markets

Table 5: Currently approved therapies for pulmonary arterial hypertension - class, formulation, dosing, reimbursement and primary and secondary indications, 2009

Table 6: Letairis (ambrisentan) overview

Table 7: Tracleer (bosentan) overview

Table 8: Thelin (sitaxsentan) overview

Table 9: Flolan (epoprostenol) overview

Table 10: Careload LA (beraprost) overview

Table 11: Ventavis (iloprost)

Table 12: Remodulin (treprostinil) overview

Table 13: Revatio (sildenafil) overview

Table 14: Comparative American College of Chest Physicians' (ACCP) and European Society of Cardiology (ESC) clinical guidance coverage for pulmonary arterial hypertension

Table 15: UK interim national commissioning policy for pulmonary arterial hypertension management, 2008

Table 16: Pulmonary arterial hypertension therapy involvement in combination trials - as backbone, add-on or combination component

Table 17: Overview of pulmonary arterial hypertension combination and add-on trial

Table 18: Phase III pipeline overview for pulmonary arterial hypertension, 2009

Table 19: Phase II pipeline overview for pulmonary arterial hypertension, 2009

Table 20: Phase I and selected preclinical pipeline overview for pulmonary arterial hypertension, 2009

Table 21: Seven major market population data, 2009

Table 22: Estimated incidence and prevalence rates of pulmonary arterial hypertension in the 5EU, US and Japan, 2008

Table 23: Estimated incidence and prevalence of pulmonary arterial hypertension, in the 5EU, US and Japan, 2008

Table 24: New York Heart Association classification of pulmonary arterial hypertension patients at time of presentation by functional class in the seven major markets, 2009

Table 25: Functional class at presentation - high and low pulmonary arterial hypertension incidence and prevalence rates for the seven major markets

Table 26: Etiology at the time of diagnosis of pulmonary arterial hypertension for the seven major markets

Table 27: Etiology at the time of diagnosis of pulmonary arterial hypertension - incidence and prevalence ranges in the seven major market

Table 28: Etiology and group functional class at the time of diagnosis of pulmonary arterial hypertension in the seven major markets

Table 29: Estimated incidence range by etiology and group functional class at the time of diagnosis of pulmonary arterial hypertension in the seven major markets

Table 30: Estimated prevalence range by etiology and group functional class at the time of diagnosis of pulmonary arterial hypertension in the seven major markets

Table 31: Proportional gender representation by etiology in pulmonary arterial hypertension in the seven major markets

Table 32: Estimated proportional gender representation across total incident and prevalent pulmonary arterial hypertension populations in the seven major markets

Table 33: Estimates of seven major market incidence of pulmonary arterial hypertension in the seven major markets by etiology and gender, 2008

Table 34: Estimates of seven major market prevalence of pulmonary arterial hypertension in the seven major markets by etiology and gender, 2008

Table 35: Estimated seven major market epidemiology for conditions associated with pulmonary arterial hypertension

List of Figures

Figure 1: Pathophysiology of right ventricular dysfunction in pulmonary hypertension

Figure 2: Timeline of key events impacting pulmonary arterial hypertension management, 1950s 2009

Figure 3: Determinants of risk in pulmonary arterial hypertension

Figure 4: Drug development activity over time - pulmonary arterial hypertension versus select breakthrough indications in the seven major markets

Figure 5: Average approvals per year - pulmonary arterial hypertension versus select breakthrough indications in the seven major markets

Figure 6: Targets for current therapies in pulmonary arterial hypertension, 2009

Figure 7: European Society of Cardiology diagnostic approach for pulmonary arterial hypertension

Figure 8: American College of Chest Physicians updated evidence-based clinical practice guidelines for the medical treatment of pulmonary arterial hypertension (2007)

Figure 9: Incidence and prevalence ranges for pulmonary arterial hypertension in the US, Japan, and five major EU markets, 2009

Figure 10: Seven major market (7MM) incidence and prevalence of pulmonary arterial hypertension by functional class at diagnosis and etiology, 2009

Figure 11: New York Heart Association functional class I and II versus III and IV - proportion of patients at diagnosis by etiology

Figure 12: Pulmonary arterial hypertension 5-year survival by etiology

Figure 13: Estimated incidence and prevalence of populations at risk of pulmonary arterial hypertension in the seven major markets, 2009

Figure 14: Improvements in six minute walk test distances, comparison of trial data

Figure 15: Relative frequency of product positioning in add-on and combination trials

Figure 16: Pipeline overview for pulmonary arterial hypertension, 2009TypeFigTitleHere

Figure 17: Pulmonary arterial hypertension epidemiology data integrity and methodology

Abstract

• Defining the key clinical characteristics of the pulmonary arterial sector that present a unique commercial challenge
• Translation of critical clinical issues into pertinent practical considerations for both incumbent players and new market entrants
• Insight into the challenge of determining a patient potential in the context of a miniscule diagnosed population and a phenomenal at-risk population
• Assessment of future directions in management and the changing roles of therapies

• Understand the challenges in aligning product positioning to the demands of the future clinical paradigm
• Identify development strategies that allow optimal buy-in from an increasingly important clinical community
• Qualify market expectations against the practical realities of targeting this fragmented, niche patient population

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