SPI Infoamtion

Table of Contents

Executive summary

Orphan drug legislation
The orphan drug pipeline
Orphan drug development trends and strategies
Orphan drug strategies

Chapter 1 Orphan drug legislation

Summary
Introduction
Orphan drug legislation in the US

Orphan drugs act 1983

Orphan drug designation
Orphan grant program

Orphan drug legislation in the EU

The Committee for Orphan Medicinal Products
Orphan designation process
EU incentives for orphan medicinal product developers

Orphan drug legislation in Japan

Incentives for orphan drug developers in Japan

Orphan drug legislation in Australia

Incentives for orphan drug developers in Australia

Chapter 2 The orphan drug pipeline

Summary
Introduction
Oncology

Istodax - romidepsin
Yondelis - trabectedin
Omapro - omacetaxine
Clolar - clofarabine
Onrigin - laromustine
TM601
EGEN - 001

Central nervous system

Zenas - amifampridine
H P Acthar Gel
ITI111 - midazolam

Respiratory and pulmonary systems

Surfaxin

Anti-infectives

Cayston - aztreonam lysine
ABthrax - raxibacumab

Autoimmune and inflammation

EN 101

Genetic diseases and dysmorphic syndromes

Uplyso - taliglucerase alfa

Chapter 3 Orphan drug development trends and issues

Summary
Introduction
Clinical trial and approval strategies for orphan drugs

Adaptive clinical trials in orphan drug development
The role of patient advocacy groups

Patient group/industry R&D partnerships

Orphan drug reimbursement issues

Orphan drug access in the US
Orphan drug access in the EU
Pharmaceutical risk-sharing agreements

Chapter 4 Orphan drug strategies

Summary
Introduction
The move towards niche indications
Opportunities in orphan drugs

Small pharma and biotech
Big pharma and established biotech

Pfizer
GlaxoSmithKline
Novartis
Sanofi-Aventis
Roche
Johnson & Johnson
Merck & Co
Eli Lilly
Bayer

Orphan drug specialists

Genzyme
Actelion

Strategies for sales growth for orphan drugs

Expansion into non-orphan indications
Expansion into further orphan indications

Using biomarkers to develop orphan drugs

Conclusion

Glossary
Index

List of Figures
Figure 1.1: Number of products receiving orphan designation in the US, 1984-December 2009
Figure 1.2: Number of Orphan drug approvals in the US, 1990-2008
Figure 1.3: Average NDA approval time, (months), 1995-2008
Figure 2.4: Therapeutic focus of products given orphan designation in the EU, 2009
Figure 2.5: Therapeutic focus of products given orphan designation in the US, 2009
Figure 2.6: Yondelis deals and alliances structure
Figure 2.7: EGEN-001 mode of action
Figure 2.8: Licensing deals for H P Acthar Gel
Figure 3.9: Blinding in US orphan drug trials, 2009
Figure 3.10: Randomization in US orphan drug trials, 2009
Figure 3.11: Comparators in US orphan drug trials, 2009
Figure 3.12: Reasons for adopting adaptive clinical trials
Figure 3.13: Overview of strategic considerations when designing adaptive clinical trials
Figure 3.14: Overview of operational considerations when designing adaptive trials
Figure 3.15: Relationship between annual cost of treatment (?) per patient and prevalence
Figure 3.16: The relationship between cost-effectiveness and societal value
Figure 4.17: Blockbuster to orphan drugs
Figure 4.18: Required conditions for stratified medicine

List of Tables
Table 1.1: Comparison of orphan drug systems
Table 2.2: Yondelis orphan designations
Table 2.3: Surfaxin's US orphan designations by date
Table 3.4: Recent deals involving CFF and pharma companies
Table 4.5: Pfizer's orphan designations since 2003
Table 4.6: Pfizer's active orphan programs
Table 4.7: GlaxoSmithKline's marketed US orphan drug portfolio
Table 4.8: GlaxoSmithKline's active orphan programs
Table 4.9: Novartis' marketed US orphan drug portfolio
Table 4.10: Novartis's active orphan programs
Table 4.11: Sanofi-Aventis's active orphan programs
Table 4.12: Roche's marketed US orphan drug portfolio
Table 4.13: Roche's active orphan programs
Table 4.14: J&J's marketed US orphan drug portfolio
Table 4.15: J&J's active orphan programs
Table 4.16: Merck & Co's active orphan programs
Table 4.17: Eli Lilly's marketed US orphan drug portfolio
Table 4.18: Eli Lilly's active orphan programs
Table 4.19: Bayer's marketed US orphan drug portfolio
Table 4.20: Genzyme's global marketed orphan product portfolio ($m), 2008
Table 4.21: Genzyme's lysosomal storage disorder product history
Table 4.22: Actelion's global marketed portfolio ($m), 2008
Table 4.23: Actelion's product pipeline
Table 4.24: Gleevec's US orphan indications
Table 4.25: Nutropin's US orphan indications
Table 4.26: Orphan designated products with at least one associated clinically validated biomarker