Chapter 1. INTRODUCTION: THE ASCENDANCY OF THE PRIMARY CARE MODEL
Definition of a Specialty Pharmaceutical
1.1. Background: Evolution of the Industry’s Focus on Primary Care Markets
1.2. The Impact of the Blockbuster Model on the Current Structure and Economics of the Pharmaceutical Industry
R&D
Sales and Marketing
1.3. The Uninsurable Risk of High-Volume Drugs
1.4. The Stirrings of a Shift
Increased Spending on Specialty Drugs
A Surge in New Product Development of Biologicals

Chapter 2. THE EMERGING PORTFOLIO BALANCE
2.1. Drivers behind Big Pharma’s Interest in Specialty Markets
Cost Reduction
Clinical Development
Comparative Approval Rates: Large vs. Small Molecules
Sales and Marketing
Pharmacovigilance
Incremental Revenue
Risk Management
R&D
Post Market
Public Image
2.2. Hoffman-La Roche Case Study: The Big Pharma Specialist
Background
Evolution of Roche’s Specialty Care Strategy
Roche’s Financial Performance
2.3. Other Examples from Industry
Pfizer: Buy It and Sell It
Bayer: Hunker Down and License
Eli Lilly: Build It Up from Scratch

Chapter 3. THE WORLD OF THE SPECIALIST
3.1. The Specialist Emerges as a Key Player in the Health Care Economy
3.2. Specialist Practice Economics
Changes in Reimbursement and Utilization Management Impacting Specialists
Medicare CAP and “Buy and Bill”
Special Feature: The Medicare Competitive Acquisition Program (CAP)
Movement of Injectables to the Pharmacy Benefit
Practice Structure and the Service Component
Conditions Requiring “High-Touch” Service
Referral Generation
PCPs
“Suspect and Refer” Physicians
Patient Self Referrals
Impact of Medicare Part D on Referrals
Specialists and Clinical Research
Upside for Physicians
Upside for Sponsoring Company
3.3. Detailing to Specialists
The Specialist vs. the PCP
Ease of Access
PCPs and Primary Care Reps
Specialists and Specialty Care Reps
Opportunities for Providing Value-Added Support Services to Specialist Practices
The Specialist Rep
3.4. The Specialist-Focused Strategy
Relationship Equity with KOLs
The Cycle of Mutual Support
KOLs and Clinical Research

Chapter 4. THE WORLD OF THE PATIENT
4.1. Profile of the Specialty Care Patient
An Engaged and Proactive Mindset
Patient Activism
Specialist Marketers Discover the Patient
4.2. Reaching the Specialty Care Patient
The Channels
The Specialist
Specialty Pharmacies
Patient Advocacy Groups
DTC Advertising of Specialty Drugs
4.3. A 3-Step Method for Reaching New Patients: A Case Study of Pegasys for Hepatitis C
Know Your Patient Population
Know the Disease
Know Your Physician Population

Chapter 5. REGULATORY AND REIMBURSEMENT ISSUES
5.1. FDA Regulatory Pathways for Specialty Care Products
The Orphan Drug Act
The Accelerated Approval Program
5.2. Managed Care and Specialty Drugs
Specialty Products Move from the Medical Benefit to the Pharmacy Benefit
A Bewildering Variety of Benefit Plans and Copay Structures
Specialty Injectables and the Formulary
Tactics for Gaining Formulary Placement
Clinical Data will be the Cornerstone of Negotiations
How Managed Care is Controlling Utilization of High-Cost Specialty Products
Prior Authorization
Managed Injectable Programs
Cost Shifting
5.3. Biogen Idec’s Amevive: A Case Study in Positioning a Specialty Product for Managed Care

Chapter 6. ANALYSIS OF TWO SPECIALTY DISEASE MARKETS: PULMONARY ARTERIAL HYPERTENSION AND SCLERODERMA
6.1 Pulmonary Arterial Hypertension
Pathophysiology, Taxonomy, and Symptoms
Pathophysiology
Taxonomy and Symptoms
Epidemiology
Diagnosis
Treatments
Calcium Channel Blockers
Prostanoids
Endothelin Receptor Antagonists (ETRAs)
Phosphodiesterase 5 (PDE-5) Inhibitors
Market Dynamics
6.2. Scleroderma
Pathophysiology, Taxonomy, and Symptoms
Pathophysiology
Taxonomy and Symptoms
Epidemiology
Diagnosis
Treatments
Fibrosis Inhibitors: CTFG Antagonists and TGF-beta
Immune Inflammation: CCR2 Antagonists, Cyclophosphamide, Interferon, and Leukocyte Elastase Inhibitors
Vascular: Prostacyclin and Alpha-2-Adrenoreceptor Agonists
Market Dynamics

Chapter 7. CONCLUSIONS: IMPLICATIONS AND CONSIDERATIONS
7.1. Factors Impacting the Future Commercial Vitality of Specialty Disease Markets
Price and Utilization Controls
High-Cost Specialty Drugs Come under Fire
The Industry Reconsiders the Rationale for High Drug Prices
What does It Mean for Specialty Products?
Biogenerics
R&D Considerations
A Finite Number of Targets for Protein Drugs?
The Oral Deliverability of Biologicals
Q&A with Manuel Vega, PhD, of Nautilus Biotech
Small Molecules that Target Protein-protein Interactions
7.2. Some Strategic Implications of Big Pharma’s Embrace of Specialty Products
The Organization
GSK’s Biopharmaceutical CEDD
The Rise of the Specialist
The Ongoing Morphing of Disease States
Conclusion

EXPERTS INTERVIEWED FOR THIS REPORT

Thomas Bumol, PhD (Vice President of Biotechnology Discovery Research at Eli Lilly)

David Caponera (former Vice President of Strategic Initiatives at TheraCom, a division of Caremark)

Dan Hawkins (founding Partner of Clarion Healthcare Consulting) 

Kenneth Kaitin, PhD (Director of the Tufts Center for the Study of Drug Development)

Sandy Lauterbach (Senior Director of Global Marketing at Genzyme) 

Jesus Leal (Vice President North America Infectious Diseases, Transplantation & Immunology at Novartis; former Vice President of Interferons at Roche)  

Paul C. Nagel (Partner at Biomedical Insights) 

James F. Resch, PhD (Director of Strategic Intelligence at AstraZeneca) 

Ramana Sonty, PhD (Senior Director of Project Management and Medical Operations at Pfizer)

Debra Stern (Vice President of RxPerts) 

Manuel Vega, PhD (CEO of Nautilus Biotech)

Eugene Williams (CEO of Cambridge Healthtech Associates; former Senior Vice President of Immune-Mediated Diseases at Genzyme)

References

Glossary

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