Abstract
High-volume, big-population drugs have fueled the industry' s growth over the past 15 years. But with increasing uncertainty about the ROI of primary care drug development, big pharma is looking for additional avenues of growth. Specialty Pharmaceuticals: Driving Industry Growth into the Next Decade is a window into the complex world of specialty medicine: its stakeholders, its evolving regulatory environment, and its economics. Based on extensive thought-leader interviews, this report is both a manual and a guidebook to competing and prospering in the world of specialty pharmaceuticals. Specifically, it provides:
Strategies for . . .
- Sustaining a specialty franchise by building relationship equity with specialists
- Identifying and communicating with hard-to-reach patient populations
- Generating referrals, raising awareness, working with disease associations and patient groups
Data that capture . . .
- Cost, time, and approval rate comparisons between specialty and primary care R&D
- Trends in specialty drug sales, pricing, and health care expenditures
- Specifics about the large- and small-molecule specialty pipelines in big pharma
Insight into . . .
- Pricing and uptake prospects for biogenerics and orally delivered proteins
- The impact on practice economics of Medicare CAP and the shift away from buy-and-bill
- Positioning specialty products for managed care
Specialty Pharmaceuticals: Driving Industry Growth into the Next Decade delivers the case studies, the recommendations, and the insider insights to help you navigate the shifting terrain of specialty markets: the gathering cost pressures, rising competitive heat, disruptive technologies, changing regulatory guidelines, new reimbursement paradigms, and more. The report also drills down into two specialty disease commercial opportunities -- pulmonary arterial hypertension and scleroderma -- detailing the epidemiology, the drugs in development, and the opportunities for improving on the current standard of treatment.
Table of Contents
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)
