Orphan Drug Strategies

Published by: Datamonitor

Published: Oct. 2, 2009 - 75 Pages

Table of Contents

ABOUT DATAMONITOR HEALTHCARE

About the Strategic Pharmaceutical Analysis Team

Executive Summary

Strategic scoping and focus

Datamonitor insight into the disease market

Related reports

Table of Contents

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

The rate of uptake of orphan drugs is similar to that of non-orphan drugs

Orphan drug status offers no protection against generic erosion

Drivers and incentives for orphan drug development and commercialization

Market-specific orphan drug incentives

US incentives

EU initiatives

Japanese initiatives

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

2009 decline in orphan drug designations linked to biotech funding crisis

Continued forecast growth for European orphan drug designations and approvals

Factors contributing to EMEA refusal of orphan drug designations

Orphan drug approval trends in Japan

Orphan drug research and development trends

Clinical trial and approval strategies for orphan drugs

Adaptive clinical trials ideally suited to orphan drug assessment

Orphan drug pipeline trends

Orphan drug reimbursement issues

Orphan drug access in the US

Orphan drug access in Europe

Orphan drug risk-sharing in the UK set to become more frequent driven by new drug launches and disease stratification

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

Segmentation through pediatric orphan indications and disease specialization

The growing use of biomarkers in disease stratification

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

About Datamonitor

About Datamonitor Healthcare

Datamonitor consulting

Disclaimer

List of Tables

Table 1: Comparison of orphan product policies across the US, EU and Japan, 2009

Table 2: Non-orphan, orphan and ultra-orphan drug designations in the US, 2009

Table 3: Comparison of orphan and non-orphan drug growth rates after launch, 2005-08

Table 4: Comparison of access to orphan drugs in selected EU countries, 2009

Table 5: Summary of US and EU orphan drug designations, approvals and approval rates, 1980s-2000s

Table 6: Top 20 most frequently targeted orphan indications by manufacturers in the EU, 2000-08

Table 7: FDA approved orphan drugs with multiple indications, 2009

Table 8: Drugs with at least one orphan drug status and associated orphan biomarkers

Table 9: Most frequently targeted orphan indications by manufacturers in the EU, 2000-08

Table 10: Exchange rates, 2008

List of Figures

Figure 1: Orphan drug policies by country

Figure 2: Uptake of orphan and non-orphan drugs during the first 3 years post launch, 2005-08

Figure 3: Erosion of orphan drugs versus average US brand erosion following patent expiry, 2005-08

Figure 4: The sliding scale of blockbuster sales: primary care drugs to ultra-orphan drugs

Figure 5: Factors driving the shift away from the primary care blockbuster model towards niche indications

Figure 6: Molecule type of approved orphan drugs, 2007

Figure 7: Number of designated and approved orphan drugs in the US, 1993-Q2 2009

Figure 8: Number of designated, approved, withdrawn/suspended and refused orphan drugs in the EU, 2000-Q2 2009

Figure 9: Number of orphan drug designations across a selection of European countries, 2000-07

Figure 10: Factors responsible for refusal of EMEA market authorization

Figure 11: Factors responsible for refusal of EMEA orphan drug designations

Figure 12: Number of orphan drugs receiving approval from the Japanese Pharmaceuticals and Medical Devices Agency (PMDA), 2004-07

Figure 13: US orphan drug Phase I-III clinical trial designs, 2009

Figure 14: Orphan drug comparator designs by phase of clinical trial

Figure 15: Reasons for adopting adaptive clinical trials for orphan drugs

Figure 16: Factors to be considered when designing an adaptive clinical trial

Figure 17: US and European pipeline orphan designated drugs by clinical trial phase, 2009

Figure 18: US pipeline orphan designated drugs by therapy area, 2009

Figure 19: EU orphan drug market authorizations by therapy area, 2008

Figure 20: Japanese orphan drug approvals by therapy area, 2004-07

Figure 21: Factors to consider when developing risk-sharing agreements for orphan drugs

Figure 22: End goals of orphan drug strategies for biotech and Big Pharma

Figure 23: Orphan drug developers split by company type, 1990-2008

Figure 24: Orphan drug strategies to increase revenue potential through sales expansion or protection

Figure 25: Criteria to be considered when stratifying a disease into smaller niche and potentially orphan indications

Figure 26: Forecast global sales of Genzyme's orphan and non-orphan drug portfolios, 2008-2014

Figure 27: Forecast global sales of Actelion's orphan and non-orphan drug portfolios, 2008-2014

Figure 28: 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

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

• 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

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.

Reasons to Purchase

• 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.

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