Orphan Drug Strategies

Table of Contents

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