Orphan Drug Strategies

Published by	Datamonitor
Published	October, 2009	Product code	101289
Content info	75 pages
Price	US $ 3800 PDF by E-mail (Single User License) US $ 9500 PDF by E-mail (Global Site License)

Introduction
Related Reports

Orphan Drug Strategies published by Datamonitor in October, 2009. This report consists of 75 pages and the price starts from US $ 3800.

Introduction

Abstract

Introduction

With the pharmaceutical industry reducing its focus on the primary care blockbuster model, Big Pharma is now looking towards the development and commercialization of more niche, high value, often biologic therapies, for the treatment of smaller patient populations.

Scope of this research

Provides an overview of orphan drugs, including drivers and resistors of investing in this niche market
Examines orphan drug developmental and approval trends
Analyzes Big Pharma' s attraction towards orphan drugs, supplemented with future forecast analysis
Assesses the future size and growth of the orphan drugs market

Research and analysis highlights

Few markets have implemented robust orphan drug policies with the exception of the US, Europe, Japan, Australia and Singapore. Nevertheless, a number of key emerging markets have instigated partial orphan drug policies which are anticipated to be strengthened in the near future.

With increasing cost pressures facing payers, combined with the growing number of orphan drugs now on the market and in development, measures aimed at reducing costs can be expected by shifting payment onto the patient or even the pharma companies themselves through risk-sharing agreements.

Oncology is the most frequently investigated therapy area, with nearly half of all pipeline orphan drugs designated for such indications. However, with the availability of marketed products for a number of the most frequently targeted orphan indications, competition in these areas is set to grow.

Key reasons to purchase this research

Gain insight into the challenges companies face when developing and commercializing orphan drugs.
Examine which are the most popular orphan indications, and the most dominant orphan drug players in the market.
Identify strategies to maximize the commercial opportunities of orphan drugs through both revenue expansion and protection.

1. EXECUTIVE SUMMARY

Strategic scoping and focus
Datamonitor insight into the disease market
Related reports

2. ORPHAN DRUG OVERVIEW

Key findings
Orphan drug definitions
- US orphan drug criteria
- EU orphan drug criteria
- Japanese orphan drug criteria
- Orphan drug designation comparisons across the US, EU, and Japan
- Ultra-orphan drug and disease criteria
Drivers and resistors to orphan drug development and commercialization
- Developmental and commercial barriers to orphan drugs
- Drivers and incentives for orphan drug development and commercialization
- Market-specific orphan drug incentives

3. ORPHAN DRUG DEVELOPMENT AND APPROVAL TRENDS

Key findings
Orphan drug designation and approval trends
- Decline in recent number of US orphan drug designations linked to Biotech funding crisis
- Continued forecast growth for European orphan drug designations and approvals
- Orphan drug approval trends in Japan
Orphan drug research and development trends
- Clinical trial and approval strategies for orphan drugs
- Orphan drug pipeline trends
Orphan drug reimbursement issues
- Orphan drug access in the US
- Orphan drug access in Europe

4. ORPHAN DRUG STRATEGIES AND FUTURE OUTLOOK

Key findings
Big Pharma' s growing attraction for orphan drugs
- Big Pharma' s dominance in the orphan drug market
- Strategies employed to grow Big Pharma' s orphan drug presence
Three key orphan drug strategies
- Market access and subsequent expansion into non-orphan diseases offers significant opportunity for revenue generation
- Multiple indications shore up a drug' s exclusivity in a niche therapeutic area
- Stratification of indications into niche patient populations
Orphan drug specialists
- Genzyme' s success in ultra-orphan markets
- Actelion' s orphan drug portfolio driven by pulmonary arterial hypertension
Orphan drug sales forecast

5. BIBLIOGRAPHY

Publications and online articles
Datamonitor reports

APPENDIX

TABLES

Table: Comparison of orphan product policies across the US, EU and Japan, 2009
Table: Non-orphan, orphan and ultra-orphan drug designations in the US, 2009
Table: Comparison of orphan and non-orphan drug growth rates after launch, 2005 - 08
Table: Comparison of access to orphan drugs in selected EU countries, 2009
Table: Summary of US and EU orphan drug designations, approvals and approval rates, 1980s - 2000s
Table: Top 20 most frequently targeted orphan indications by manufacturers in the EU, 2000 - 08
Table: FDA approved orphan drugs with multiple indications, 2009
Table: Drugs with at least one orphan drug status and associated orphan biomarkers
Table: Most frequently targeted orphan indications by manufacturers in the EU, 2000 - 08
Table: Exchange rates, 2008

FIGURES

Figure: Orphan drug policies by country
Figure: Uptake of orphan and non-orphan drugs during the first 3 years post launch, 2005 - 08
Figure: Erosion of orphan drugs versus average US brand erosion following patent expiry, 2005 - 08
Figure: The sliding scale of blockbuster sales: primary care drugs to ultra-orphan drugs
Figure: Factors driving the shift away from the primary care blockbuster model towards niche indications
Figure: Molecule type of approved orphan drugs, 2007
Figure: Number of designated and approved orphan drugs in the US, 1993 - Q2 2009
Figure: Number of designated, approved, withdrawn/suspended and refused orphan drugs in the EU, 2000 - Q2 2009
Figure: Number of orphan drug designations across a selection of European countries, 2000 - 07
Figure: Factors responsible for refusal of EMEA market authorization
Figure: Factors responsible for refusal of EMEA orphan drug designations
Figure: Number of orphan drugs receiving approval from the Japanese Pharmaceuticals and Medical Devices Agency (PMDA), 2004 - 07
Figure: US orphan drug Phase I - III clinical trial designs, 2009
Figure: Orphan drug comparator designs by phase of clinical trial
Figure: Reasons for adopting adaptive clinical trials for orphan drugs
Figure: Factors to be considered when designing an adaptive clinical trial
Figure: US and European pipeline orphan designated drugs by clinical trial phase, 2009
Figure: US pipeline orphan designated drugs by therapy area, 2009
Figure: EU orphan drug market authorizations by therapy area, 2008
Figure: Japanese orphan drug approvals by therapy area, 2004 - 07
Figure: Factors to consider when developing risk-sharing agreements for orphan drugs
Figure: End goals of orphan drug strategies for biotech and Big Pharma
Figure: Orphan drug developers split by company type, 1990 - 2008
Figure: Orphan drug strategies to increase revenue potential through sales expansion or protection
Figure: Criteria to be considered when stratifying a disease into smaller niche and potentially orphan indications
Figure: Forecast global sales of Genzyme' s orphan and non-orphan drug portfolios, 2008 - 2014
Figure: Forecast global sales of Actelion' s orphan and non-orphan drug portfolios, 2008 - 2014
Figure: Historical and forecast sales of drugs with orphan status for at least one indication, marketed by the top 50 global pharmaceutical companies in the US and five major EU market, 2001 - 2014