Orphan Drug Strategies
Datamonitor
October 2, 2009 75 Pages - SKU: DFMN2470156
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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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- 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
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