The World Market for Orphan Drugs

June 2013 | 400 pages | ID: WE13DBDE939EN
Kalorama Information

Abstracts

In recent years, there has been a substantial increase in the number of drugs available to treat rare ("orphan") diseases. This comprehensive report, The World Market for Orphan Drugs, looks at this trend and provides estimates for the market opportunity in the U.S., Europe, Japan and globally. With over 400 pages of market analysis and over 120 figures and tables, the report provides in-depth coverage of key competitors and important trends and challenges for makers of orphan drug treatments.

Osteoporosis, arthritis, Alzheimer’s, and Parkinson’s are major factors affecting quality of life. Mortality from cancer, diabetes, liver and kidney diseases has been slow to change, offering significant areas for research breakthroughs. Cancer, poorly served by traditional chemotherapies, is a major opportunity for biotech firms because the investment compared to return can be favorable: the field has high priority with regulatory authorities who are willing to give it fast track status on the basis of smaller (and therefore cheaper) clinical trials (a few extra months of survival could be enough to win FDA approval); the clinical community is highly concentrated; and the market size is often larger than the approved indication because of a high off-label use (for other cancers). Infectious diseases, the third most common cause of death in many geographic regions, highlight the pressing need for new therapies with novel mechanisms of action to avoid growing issues affecting this industry including a top area of concern—drug resistance.

This report provides estimates for the world market for orphan drugs, as well as the E.U. and Japan markets. Given the size of the U.S. opportunity, the report offers in-depth market data for the U.S. market. Several segments are discussed, including:

• Oncology
• Hematology
• Musculoskeletal
• Neurology
• Endocrine
• Metabolic
• Others

These segments are the most common areas of development and success for orphan drug developers/marketers which required providing detailed information about these orphan drug market segments.

In the U.S., a drug may be designated an orphan drug if the disease or condition for which it is indicated, affects less than 200,000 Americans. Orphan medicinal products in the E.U. member states are intended for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect not more than 5 in 10,000 persons. In Japan an orphan drug may be designated for a patient population of less than 50,000 and provide for an unmet medical need. Other countries have similar designations. These three regional markets for drugs are examined in separate sections of the report.

Analysis of The “Pure” Orphan Drug Market

Some publicized estimates of orphan drug markets include brand revenue that is not truly related to the orphan status of the drug. In this unique research effort, Kalorama Information separates strictly orphan revenue from total brand revenue where necessary to provide a true picture of the pure orphan drug market opportunity. The world orphan drug market is difficult to determine due to the variances in orphan drug status and requirements by country. Products may be available as an orphan drug on one country and be a traditional marketing approval for another country. In addition, some products gain orphan status, are granted approval and then removed from the orphan drug database at the request of the developer. There are a number of factors involved, including reimbursement by country, regulation by country, disease status by country and general development by country.

For this market research report, market numbers discussed in this report are focused on the global market in brief with in-depth market coverage of the U.S. market. Additionally, there is select coverage for the European Union and Japanese market. All sales are displayed at the manufacturers’ level in U.S. dollars.

This report includes orphan drugs. In cases where sales are generated by indications for both orphan indications and non-orphan indications, we have attempted to extract just the orphan portion of sales. The base year is 2012, with forecasts provided for each year through 2017. The market was generated using estimates of individual product sales and compared with market data from a combination of other methods including disease prevalence trends, population trends, pricing trends, government regulations and reimbursements. Both primary and secondary sources were consulted in developing market estimates.

Demographics, Costs, Regulation and Other Trends Examined

The list of trends and factors affecting the orphan drug market remain lengthy. The orphan drug market is affected by both trends that affect the pharmaceutical industry as a whole and trends that specifically affect the orphan drug market:

• Orphan drug pricing
• Aging of the population
• Increasing life expectancy
• Trends in birth rates
• Cost effectiveness
• Regulatory environments
• Orphan drug incentives
• Population demographics
• Product availability by country/region

Due to the wide variety of products to receive orphan designations, there are numerous developers with products in this area. Some of the developers that have received an orphan designation have followed through to receive a full product approval but others may not gain additional approvals.

Contents

CHAPTER ONE: EXECUTIVE SUMMARY

Introduction
Scope and Methodology
  Market Segments
Issues and Trends
World Market Summary
Competitors

CHAPTER TWO: INTRODUCTION

Overview
History of the U.S. Orphan Drug Market
Pharmaceutical Research & Development Overview
Pharmaceutical Industry Important Regulatory Terms
Diseases and Orphan Drug Impact
Disease Descriptions
  Blood Disorders
  Cancer
  Hormone Disorders
  Infectious Diseases
The Cost Side of Orphan Drugs

CHAPTER THREE: U.S. ORPHAN DRUGS

Overview
Recent Orphan Drug Designations
Orphan Drug Approvals by Company
  Abbott Laboratories
  Acorda Therapeutics
  Actelion Pharmaceuticals
  Aegerion Pharmaceuticals
  Alexion Pharmaceuticals
  Allergan, Inc.
  Amgen
  Ariad Pharmaceuticals
  Astellas
  AstraZeneca
  Auxilium Pharmaceuticals, Inc.
  Bausch & Lomb
  Baxter International
  Bayer AG
  Biogen IDEC
  BioMarin Pharmaceutical, Inc.
  Boehringer Ingelheim
  Bristol-Myers Squibb
  Cangene Corporation
  Celgene Corporation
  CSL Behring
  Eisai Co., Ltd.
  Eli Lilly & Company
  EMD Serono
  Endo Health Solutions, Inc.
  (previously Endo Pharmaceuticals, Inc.)
  Exelixis, Inc.
  Gilead Sciences, Inc.
  GlaxoSmithKline
  Grifols, S.A.
  Hyperion Therapeutics, Inc.
  Incyte Corporation
  Ipsen, Inc.
  Jazz Pharmaceuticals plc
  Johnson & Johnson
  KV Pharmaceutical Company
  Merck & Company
  Novartis
  Novo Nordisk
  NPS Pharmaceuticals
  Octapharma USA, Inc.
  Onyx Pharmaceuticals, Inc.
  Otsuka Pharmaceutical Co., Inc.
  Pacira Pharmaceuticals, Inc.
  Pfizer, Inc.
  Rare Disease Therapeutics, Inc.
  Reckitt Benckiser Group plc
  Regeneron Pharmaceuticals, Inc.
  Roche
  Salix Pharmaceuticals, Inc.
  Sanofi
  Savient Pharmaceuticals, Inc.
  Seattle Genetics, Inc.
  Shire Plc
  Sigma-Tau Pharmaceuticals, Inc.
  Spectrum Pharmaceuticals
  Takeda Pharmaceutical Company Ltd.
  Talon Therapeutics, Inc.
  Teva Pharmaceuticals
  United Therapeutics Corporation
  Valeant Pharmaceuticals International, Inc.
  Vertex Pharmaceuticals, Inc.
  ViroPharma, Inc.

CHAPTER FOUR: E.U. ORPHAN DRUGS

Overview
Recent Orphan Drug Designations
Select Company Orphan Drug Activities
  Actelion Pharmaceuticals
  Adienne Pharma & Biotech
  Alexion Pharmaceuticals
  Amgen
  Bayer AG
  BioMarin Pharmaceutical, Inc.
  Bristol-Myers Squibb
  Celgene Corporation
  Eisai Co., Ltd.
  Gilead Sciences, Inc.
  GlaxoSmithKline
  Novartis
  Orphan Europe S.A.R.L.
  Pfizer, Inc.
  Sanofi
  Shire Plc
  Vertex Pharmaceuticals, Inc.

CHAPTER FIVE: JAPAN ORPHAN DRUGS

Overview
Recent Orphan Drug Designations
Select Company Orphan Drug Activities
  Actelion Pharmaceuticals
  Alexion Pharmaceuticals
  Bristol-Myers Squibb
  Celgene Corporation
  GlaxoSmithKline
  Johnson & Johnson
  Merck Serono
  Mitsubishi Tanabe
  Novartis
  Pfizer, Inc.
  Roche
  Sanofi

CHAPTER SIX: COMPETITOR TRENDS AND PRACTICES

Acquisitions and Agreements
U.S. Competitor Market Analysis
  Amgen
  Roche
  Celgene
  Novartis
  Abbott
  Biogen Idec
  Bayer
  Eli Lilly
  Bristol-Myers Squibb
  Novo Nordisk
  Sanofi
  Allergan
  Other Companies

CHAPTER SEVEN: ORPHAN DRUG INDUSTRY TRENDS

Introduction
U.S. Regulation Shaping the Orphan Drug Segment
  Orphan Drug Act of 1983 Rare Disease Act of 2002
Office of Orphan Products Development
Humanitarian Use Device Program
Positives and Negatives of the Orphan Drug Designation
  Orphan Drug Designation in the U.S.
  Orphan Drug Designation in the E.U.
  Orphan Drug Designation in Japan
  Orphan Drug Designation in Canada
  Orphan Drug Designation in Australia
Factors Affecting the Health Industry
  Aging of the Population
Population Statistics
  Global Population Trends
    Global Population over 65 U.S. Population Trends
    U.S. Population over 65   Global Trends in Life Expectancy
    Global Trends in Birth Rates
Global Healthcare Spending Trends
  General Economic Trends by Country
U.S. Health Industry Trends
  U.S. Healthcare Challenges
  Economic Trends in the U.S.
  U.S. Health Expenditures by Type

CHAPTER EIGHT: U.S. ORPHAN DRUG MARKET SIZE AND OPPORTUNITY

Overview
Market Size and Forecast
Markets by Product Classification
  Oncology
    Suppliers Market Share
  Hematology
    Suppliers Market Share
  Musculoskeletal
    Suppliers Market Share
  Neurology
    Suppliers Market Share
  Endocrinology
    Suppliers Market Share
  Metabolic
    Suppliers Market Share
  Others
    Suppliers Market Share

CHAPTER NINE: WORLD MARKET SUMMARY⎯ ORPHAN DRUGS

Overview
Market Distribution: United States and World Markets

CHAPTER TEN: CORPORATE PROFILES

Introduction
Orphan Drug Designations by Company: A U.S. Analysis
Company Profiles
Abbott Laboratories
  Company Overview
  Performance Review
AbbVie
  Company Overview
  Performance Review
Actelion
  Company Overview
  Performance Review
  Overview
  Performance Review
Astellas
  Company Overview
  Performance Review
AstraZeneca
  Company Overview
  Performance Review
Baxter
  Company Overview
  Performance Review
Bayer
  Company Overview
  Performance Review
Bio Products Laboratory
  Company Overview
  Biogen Idec
  Company Overview
  Performance Review
BioMarin
  Company Overview
  Performance Review
Boehringer Ingelheim
  Company Overview
  Performance Review
Bristol-Myers Squibb
  Company Overview
  Performance Review
Cangene
  Company Overview
  Performance Review
Celgene
  Company Overview
  Performance Review
CSL Behring
  Company Overview
  Performance Review
Depomed
  Company Overview
  Performance Review
Eisai
  Company Overview
  Performance Review
Eli Lilly
  Company Overview
  Performance Review
EMD Serono
  Company Overview
  Performance Review
Exelixis
  Company Overview
  Performance Review
Gilead Sciences
  Company Overview
  Performance Review
GlaxoSmithKline
  Company Overview
  Performance Review
ImmunoGen
  Company Overview
  Performance Review
Insmed
  Company Overview
  Performance Review
Johnson & Johnson
  Company Overview
  Performance Review
LUITPOLD
  Company Overview
  Performance Review
Merck & Co
  Company Overview
  Performance Review
Millennium Pharmaceuticals
  Company Overview
  Performance Review
NBI Pharmaceuticals
  Company Overview
Novartis
  Company Overview
  Performance Review
Novo Nordisk
  Company Overview
  Performance Review
NPS Pharmaceuticals
  Company Overview
  Performance Review
Pfizer
  Company Overview
  Performance Review
Roche
  Company Overview
  Performance Review
Sanofi
  Company Overview
  Performance Review
Seattle Genetics
  Company Overview
  Performance Review
Shire
  Company Overview
  Performance Review
Sigma Tau
  Company Overview
  Performance Review
Soligenix
  Company Overview
  Performance Review
Spectrum
  Company Overview
  Performance Review
Swedish Orphan Biovitrum
  Company Overview
  Performance Review
TEVA
  Company Overview
  Performance Review
United Therapeutics
  Company Overview
  Performance Review
Valeant
  Company Overview
  Performance Review
XenoPort
  Company Overview
  Performance Review

REGULATORY AUTHORITIES AND ORGANIZATIONS

LIST OF COMPANIES

List Of Exhibits

LIST OF EXHIBITS

CHAPTER ONE: EXECUTIVE SUMMARY

Table 1-1 World Orphan Drug Market 2007-2017
Figure 1-1: World Orphan Drug Market

CHAPTER TWO: INTRODUCTION

Table 2-1 Phases of Clinical Drug Development
Table 2-2 Total Cancer Incidence by Sex and Country
Figure 2-1 Total Cancer Incidence by Sex and Country
Table 2-3 Average Annual Drug Costs to Patients, Selected Drugs
Figure 2-2 Average Annual Drug Costs to Patients, Selected Drugs

CHAPTER THREE: U.S. ORPHAN DRUGS

Table 3-1 Orphan Drug Designations Granted and Approvals by the FDA 1983-2013*
Figure 3-1 Orphan Drug Designations Granted and Approvals by the FDA 1983-2013*
Table 3-2 12-Months Orphan Drug Designations Granted by FDA (May 1, 2012 - May 1, 2013)
Figure -3-2 May 2012 - May 2013 FDA Orphan Drug Designations by Target Classification
Table 3-3 Abbott Laboratories' Orphan Drug Approvals Granted by FDA
Table 3-4 Acorda Therapeutics' Orphan Drug Approvals Granted by FDA
Table 3-5 Actelion Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-6 Aegerion Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-7 Alexion Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-8 Allergan's Orphan Drug Approvals Granted by FDA
Table 3-9 Amgen's Orphan Drug Approvals Granted by FDA
Table 3-9 (continued)
  Amgen's Orphan Drug Approvals Granted by FDA
Table 3-10 Ariad Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-11 Astellas' Orphan Drug Approvals Granted by FDA
Table 3-12 AstraZeneca's Orphan Drug Approvals Granted by FDA
Table 3-13 MedImmune's Orphan Drug Approvals Granted by FDA
Table 3-14 Auxilium Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-15 Bausch & Lomb's Orphan Drug Approvals Granted by FDA
Table 3-16 Baxter International's Orphan Drug Approvals Granted by FDA
Table 3-17 Bayer's Orphan Drug Approvals Granted by FDA
Table 3-18 Biogen IDEC's Orphan Drug Approvals Granted by FDA
Table 3-19 BioMarin Pharmaceutical's Orphan Drug Approvals Granted by FDA
Table 3-20 Boehringer Ingelheim's Orphan Drug Approvals Granted by FDA
Table 3-21 Bristol-Myers's Orphan Drug Approvals Granted by FDA
Table 3-22 Cangene's Orphan Drug Approvals Granted by FDA
Table 3-23 Celgene Corporation's Orphan Drug Approvals Granted by FDA
Table 3-24 CSL Behring's Orphan Drug Approvals Granted by FDA
Table 3-25 Eisai's Orphan Drug Approvals Granted by FDA
Table 3-26 Eli Lilly's Orphan Drug Approvals Granted by FDA
Table 3-27 EMD Serono's Orphan Drug Approvals Granted by FDA
Table 3-28 Endo Health Solutions' Orphan Drug Approvals Granted by FDA
Table 3-29 Exelixis' Orphan Drug Approvals Granted by FDA
Table 3-30 Gilead Sciences' Orphan Drug Approvals Granted by FDA
Table 3-31 GlaxoSmithKline's Orphan Drug Approvals Granted by FDA
Table 3-32 Grifols' Orphan Drug Approvals Granted by FDA
Table 3-33 Hyperion Therapeutics' Orphan Drug Approvals Granted by FDA
Table 3-34 Incyte Corporation's Orphan Drug Approvals Granted by FDA
Table 3-35 Ipsen's Orphan Drug Approvals Granted by FDA
Table 3-36 Jazz Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-37 Johnson & Johnson Pharmaceutical's Orphan Drug Approvals Granted by FDA
Table 3-38 Alza's Orphan Drug Approvals Granted by FDA
Table 3-39 Janssen's Orphan Drug Approvals Granted by FDA
Table 3-40 KV Pharmaceutical's Orphan Drug Approvals Granted by FDA
Table 3-41 Merck & Co.'s Orphan Drug Approvals Granted by FDA
Table 3-41 (continued)
  Merck & Co.'s Orphan Drug Approvals Granted by FDA
Table 3-42 Novartis' Orphan Drug Approvals Granted by FDA
Table 3-43 Alcon's Orphan Drug Approvals Granted by FDA
Table 3-44 Novo Nordisk's Orphan Drug Approvals Granted by FDA
Table 3-45 NPS Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-46 Octapharma's Orphan Drug Approvals Granted by FDA
Table 3-47 Onyx Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-48 Otsuka Pharmaceutical's Orphan Drug Approvals Granted by FDA
Table 3-49 Pacira Pharmaceutical's Orphan Drug Approvals Granted by FDA
Table 3-49 (continued) Pfizer's Orphan Drug Approvals Granted by FDA
Table 3-49 (continued) Pfizer's Orphan Drug Approvals Granted by FDA
Table 3-50 Rare Disease Therapeutics' Orphan Drug Approvals Granted by FDA
Table 3-51 Reckitt Benckiser's Orphan Drug Approvals Granted by FDA
Table 3-52 Regeneron Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-53 Roche's Orphan Drug Approvals Granted by FDA
Table 3-54 Genentech's Orphan Drug Approvals Granted by FDA
Table 3-55 Salix Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-56 Sanofi's Orphan Drug Approvals Granted by FDA
Table 3-57 Genzyme's Orphan Drug Approvals Granted by FDA
Table 3-58 Savient Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-59 Seattle Genetics' Orphan Drug Approvals Granted by FDA
Table 3-60 Shire's Orphan Drug Approvals Granted by FDA
Table 3-61 Sigma Tau's Orphan Drug Approvals Granted by FDA
Table 3-62 Spectrum Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-63 Millennium Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-64 Talon Therapeutics' Orphan Drug Approvals Granted by FDA
Table 3-65 Teva's Orphan Drug Approvals Granted by FDA
Table 3-66 Cephalon's Orphan Drug Approvals Granted by FDA
Table 3-67 United Therapeutics' Orphan Drug Approvals Granted by FDA
Table 3-68 Valeant Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-69 Vertex Pharmaceuticals' Orphan Drug Approvals Granted by FDA
Table 3-70 ViroPharma's Orphan Drug Approvals Granted by FDA