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

Healthcare

Publication Date	March 2009
Publisher	Datamonitor
Product Type	Report
Pages	134
ISBN Number	not applicable
Product Code	DAT14955

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Summary

Introduction

Despite an explosion in the number of drugs and treatment strategies available, pulmonary arterial hypertension is still a rapidly fatal condition for a small, hard to identify patient group. A small clinical elite is currently leading the way in advancing treatment outcomes, and is central to determining which new treatment strategies are used.

Scope

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

Highlights

The estimated 8,11516,186 diagnosed pulmonary arterial hypertension patients across the US, Japan, France, Germany, Italy, Spain and the UK represent a fraction of the 28.0135.6 million group known to be at elevated risk. Identifying the true patient potential within known high risk groups remains highly problematic.

A small, but relatively powerful clinical community has brought structure, order and an increasing influence to bear on management through a few specialist centers. This internationally cohesive group has been responsible for laying the foundations for substantial volumes of data pertaining to patients managed in increasingly standardized ways.

A high number of approved and late stage pipeline products, and the increasing exploration of combination therapeutics places a higher emphasis than normal on the perceptions of specialist clinicians, who will increasingly need to triage their attentions to the most promising avenues for improving outcomes.

Reasons to Purchase

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

Content

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