QSAR - Orphan Drugs: Future Directions for a High Growth Market 2004-2009

Orphan Drugs: Future Directions for a High Growth Market 2004-2009
new visiongain report

Do you know...

• what the incentives are to entering the orphan drug market?
• how to enter and stay in the orphan drug market?
• how to profit from the market?
• what the features of the best-selling drugs on the market are?
• what the most successful therapeutic categories and indications on the
market are?
• what important orphan drugs are in the pipeline?
• what drug, indication and company types will dominate the market to 2009?
• if thethe new Medicare reimbursement rules will affect the market in
future?

If you answered 'no' to any of these questions, you need to be updated - and
quickly! This brand new report will be the answer to your questions.

* In 1983 there were just 10 orphan drugs available to treat rare diseases,
but in the space of 20 years there are now 210 different drugs resulting in
a market that is worth more than $27 billion today. The high cost of orphan
drugs and lack of competition in the market means that the industry is
extremely profitable for many drugs. Which companies are dominating this
market? Is your company maximising its potential in this market sector? Is
this the future direction for your product portfolio?

* In 2003, there were 9 orphan drugs that had reached Blockbuster status,
but these highly successful products typically have additional indications
for non-orphan diseases as well. The introduction of the Orphan Drug Act in
the US has provided financial incentives for companies to develop and market
drugs to target these rare diseases that affect less than 200,000 sufferers.
Could your company qualify for such incentives?

* In terms of revenue, in 2003, this market was dominated by blood
disorder, cancer and hormonal treatments, with the blood disorder treatment
category alone worth more than $5.6 billion. But which products, treatments
and therapy areas will experience growth in the medium term future?

* The visiongain report “Orphan Drugs: Future Directions for a High Growth
Market 2004-2009” provides analysis on the current industry, the top ten
best selling drugs in this market sector and the performance potential of
newly released products. The report also analyses the profile of the key
factors for success in this market area. By 2009, visiongain believes that
this industry opportunity will grow to be worth almost $65 billion from the
$27 billion it was worth in 2003. Make sure that you do not miss a share of
this sector.

Why You Must Buy This Report
With 50 tables, figures and graphs, an insight into how to enter and stay in
the orphan drug market, and in-depth analysis of the current and future
market, this report is key to understanding the orphan drug market and its
prospects for the future.

Order visiongain's Orphan Drugs Report today. Only a set number of these
reports have been produced, so order your copy today to ensure you do not
miss out.

Below is the short then full table of contents:

Orphan Drugs: Future Directions for a High Growth Market 2004-2009 - Table
of Contents

Chapter 1. Executive Summary
Chapter 2. Introduction
Chapter 3. The Orphan Drug Act
Chapter 4. A Review of the Orphan Drug Market, 2003-2004
Chapter 5. Profiles of the Top Ten Best-Selling Orphan Drugs
Chapter 6. Leading Indications of Orphan Drugs
Chapter 7. The Performance and Potential of Newly Approved Orphan Drugs,
2003-2004
Chapter 8. Do the Best-Selling Orphan Drugs Have a Particular Profile?
Chapter 9. The Future Orphan Drug Market 2005-2009
Chapter 10. Medicare Reimbursement for Orphan Drugs
Chapter 11. Conclusion: The Orphan Drugs Market Will More Than Double in
Value by 2009

Orphan Drugs - Full Table of Contents:

Chapter 1. Executive Summary

Chapter 2. Introduction
2.1 Why Are Orphan Drugs of Little Interest to the Pharmaceutical Industry?
2.2 Rare Diseases are Usually Chronic and Life-Threatening
2.3 The Orphan Drug Act Provides Incentives for Companies to Develop Drugs
for Rare Diseases
2.4 Aims, Scope and Format of the Report

Chapter 3. The Orphan Drug Act
3.1 Life Before the Orphan Drug Act
Graph 3.1 Number of Orphan Drugs with Marketing Approval, Pre-1983 to
Mid-2004
3.2 Implementing the Orphan Drug Act
3.2.1 Definitions in the Orphan Drug Act
Table 3.1 Amendments to the Orphan Drug Act Subsequent to Implementation
3.2.2 Incentives to Orphan Drugs Providers in Terms of R&D, Intellectual
Property and Marketing
3.3 The Effect of the Orphan Drug Act on the Pharmaceutical Industry
3.3.1 FDA Orphan Drug Grants
3.3.2 The Orphan Drug Tax Credit
Graph 3.2 Orphan Drug Tax Credits Uptake
3.3.3 Marketing Exclusivity
3.3.3.1 Exclusivity Can be Applied to Both New and Old Drugs
3.3.3.2 Multiple Indications
3.3.3.3 Marketing Exclusivity Can Be More Valuable than a Patent
3.3.3.4 The Cost of Exclusivity to Patients Can be High
3.3.3.5 Manufacturers Can Reap High Rewards from Exclusivity
3.3.4 Fast-Tracking
3.3.4.1 How Do You Get Fast-Track Product Designation?
3.3.4.2 Several Orphan Drugs with Active Marketing Approval Were
Fast-Tracked
Table 3.2 CDER Fast Track Designated Orphan Products Approved Since 1998
3.3.5 Treatment Investigational New Drug (T-IND) Status
3.4 The Downside of the Orphan Drug Act
3.4.1 High Prices Prohibit Access to Drugs
3.4.2 Highly Profitable Drugs are Being Protected from Price Competition
3.4.2.1 Designation Can Not be Rescinded if a Drug Proves to be Profitable
3.4.2.2 Profitability of AIDS Treatments
3.4.2.3 Monopoly Pricing: A Necessary Evil
3.4.3 The Race to Market Exclusivity
3.4.3.1 Companies Do Not Realise They are in the Race
3.4.3.2 The Pros and Cons of a Race to Exclusivity
3.4.4 Is the Spirit Behind the Act Being Violated?
3.5 The Act Has Been a General Success

Chapter 4. A Review of the Orphan Drug Market, 2003-2004
4.1 The Orphan Drug Market was Worth in Excess of $27 Billion in 2003
Table 4.1 Orphan Drugs' Revenue, 2002-2003
4.2 Less Than Half of the Best-Selling Orphan Drugs Have Patent Protection
or Market Exclusivity
Table 4.2 Orphan Drugs' Patent and Orphan Drug Exclusivity (ODE) Status
4.3 Blockbuster Orphan Drugs Control 50% of The Total Orphan Drug Market
Graph 4.1 Top Ten Orphan Drugs' Revenue, 2003
Figure 4.1 Top Ten Orphan Drugs' Market Share, 2003
Table 4.3 Top Ten Orphan Drugs' Market Share
4.4 Several Orphan Drugs Grew by More Than 50% in 2003
Table 4.4 Indications of Blockbuster Orphan Drugs
4.4.1 Lidoderm: The Largest Growth Rate
4.4.2 Gleevec: The Second Largest Growth Rate
4.4.3 Zometa: The Third Largest Growth Rate
4.4.4 Ellence: The Greatest Loser in the Orphan Drugs Market
4.4.5 The Intron A Family is Rapidly Losing Ground
4.4.6 Genotropin: The Rapidly Shrinking Growth Hormone
Graph 4.2 Top 10 Winners and Losers in the Orphan Drug Market, 2003
4.5 Cancer Treatments and Hormones are the Leading Therapeutic Categories in
Terms of Orphan Indications
Graph 4.3 Top Five Therapeutic Categories for Orphan Drugs by Number of
Orphan Indications, 2003
Figure 4.2 Top Five Therapeutic Categories’ (By Number of Indications)
Percentage Share of All Orphan Drugs’ Indications
4.5.1 Lupron is the Best-Selling Orphan Hormonal Treatment
4.5.2 Rituxan is The Best-Selling Orphan Cancer Treatment
4.6 Blood Modifiers and Cancer Treatments are the Leading Therapeutic
Categories in Terms of Revenue
Figure 4.3 Orphan Drugs’ Market Share by Therapeutic Category, 2003
4.7 Anaesthetics, Bone Metabolism Regulators and Anti-Convulsants are the
Most Successful Therapeutic Categories in Terms of Growth
4.8 Several Orphan Drugs Received FDA Approval in the First Seven Months of
2004
4.9 Over A Thousand Orphan Designations Have Yet to Achieve Marketing
Approval
4.10 Amgen Leads the Way in Sales of Orphan Drugs
Table 4.5 Top Ten Companies/Partnerships in the Orphan Drugs Market, 2003
Figure 4.4 Top Ten Companies’/Partnerships’ Share of the Orphan Drugs
Market, 2003
4.11 The Market Will More Than Double in Value by 2009
Graph 4.4 Total Value of the Orphan Drug Market, 2003-2009
4.12 What Drives and Restricts Sales of Orphan Drugs?
4.12.1 Drivers of Orphan Drug Sales
4.12.2 Restraints of Orphan Drug Sales

Chapter 5. Profiles of the Top Ten Best-Selling Orphan Drugs
Table 5.1 Top Ten Orphan Drugs
5.1 Epogen
5.1.1 HIV-Associated Anaemia
5.1.2 End Stage Renal Disease (ESRD)-Associated Anaemia
5.1.3 Epogen in 2004
5.2 Intron A and Rebetol
5.2.1 Intron A for Kaposi's Sarcoma
5.2.2 Intron A Has Nine Additional Orphan Designations
Table 5.2 Active Orphan Designations for Intron A
5.2.3 Rebetol and Paediatric Hepatitis C
5.2.4 Intron A and Rebetol's Patent and Exclusivity Status
5.2.5 What is Paediatric Exclusivity?
5.2.6 The Intron A Family in 2004
5.3 Remicade
5.3.1 Crohn's Disease: Prevalence and Causes
5.3.2 New Anti-Inflammatory Drugs Increase TB Risk?
5.3.3 Remicade Has Five Additional Orphan Designations
Table 5.3 Active Orphan Designations for Remicade
5.3.4 Remicade Lacks Marketing Exclusivity but Continues to Grow
5.4 Enbrel
5.4.1 Why Remicade is Exceeding Enbrel's Success
5.4.2 Enbrel in 2004
5.5 Rituxan
5.6 Neupogen
5.6.1 Neutropenia Epidemiology
5.6.2 Amgen Has Released a New-Improved Form of Neupogen: Neulasta
5.6.3 Little Competition for Amgen in the Neutropenia Market
5.7 Avonex
5.7.1 Multiple Sclerosis: Incidence and Prevalence
5.7.2 Avonex Has Additional Orphan Designations
5.7.3 Avonex in 2004
5.8 Gleevec
5.9 Topamax
5.9.1 Lennox-Gastaut Syndrome: Prevalence
5.9.2 Generic Competition for Topamax
5.9.3 New Indications May Alleviate the Threat to Topamax from Generic
Competition
5.9.3.1 Migraine Prophylaxis
5.9.3.2 Obesity and Diabetes
5.9.3.3 Alcoholism
5.9.4 The Future for Topamax
5.10 Taxol
Graph 5.1 Number of New HIV Cases Per Year, USA
5.10.1 Why the Turnaround for Taxol?
Graph 5.2 Taxol Revenue, 1999-2004
5.11 Sales Projections for the Top 10 Orphan Drugs to 2009
Graph 5.3 Sales Projections for the Top 5 Orphan Drugs to 2009
Table 5.4 Sales Projections ($m) for the Top 10 Orphan Drugs to 2009
Graph 5.4 Overall CAGR Projections for the Top Ten Orphan Drugs, 2003-2009

Chapter 6. Leading Indications of Orphan Drugs
Graph 6.1 Sales of Orphan Drugs by Therapeutic Category, 2003
6.1 Blood Disorder Treatments: Dominating the Orphan Drug Market
Table 6.1. Orphan Blood Modifying Drugs
Figure 6.1 Market Share for Drugs in the Orphan Blood Modifier Market
6.1.1 Epogen is the Best-Selling Orphan Blood Modifying Treatment
6.1.2 Neupogen is the Second-Best-Selling Orphan Blood Modifying Treatment
6.1.3 Haemophilia Medications Command More than a Quarter of the Orphan
Blood Disorder Treatments (OBDT) Market
6.1.3.1 Haemophilia Prevalence
6.1.3.2 NovoSeven: The Top Selling Haemophilia Drug
6.1.3.3 Kogenate: The Runner-Up to NovoSeven
6.1.3.4 Kogenate Will Not be Sold With Bayer's Blood Products Division
6.1.3.5 Kogenate in 2004
6.1.3.6 Benefix: Wyeth's Breakthrough Haemophilia B Treatment
6.1.3.7 ReFacto: Wyeth's Breakthrough Haemophilia A Treatment
6.1.4 Trasylol: Bayer's Drug for Blood Loss Prevention
6.1.5 Agrylin: Shire's Thrombocythemia Treatment
6.2 Cancer Therapies: A Five Billion Dollar Market for Orphans
Table 6.2 Orphan Drug Cancer Treatments, 2003
Figure 6.2 Percentage Market Shares for Orphan Cancer Treatments, 2003
6.2.1 Four Out of Thirteen Orphan Cancer Drugs Have Indications for
Leukaemia
6.2.2 Fludara is the Second Best-Selling Leukaemia Treatment After Gleevec
6.2.3 Rituxan is the Best-Selling Orphan Cancer Treatment But Campath is the
Fastest Growing
Table 6.3. Clinical Trials Involving Campath
6.2.4 Why Do Drugs Indicated for Kaposi's Sarcoma Generate Billions of
Dollars?
6.2.4.1 Intron A Has Multiple Indications, Some for Diseases Affecting
Millions
6.2.4.2 Taxol Has Indications for Two of the Most Common Cancers in Women
6.2.5 Temodar: A New and Highly Protected Orphan Drug
6.2.6 Ellence: Experiencing a Reversal of Fortunes in Its First Full Year
with Pfizer
6.2.6.1 Ellence Experienced Losses in 2003
6.2.6.2 Ellence is Back on Course in 2004
6.2.7 Proleukin: All Change After Stagnant Growth in 2003
6.2.8 Ethyol: An Orphan Drug with Rare Extensive Patent Protection
6.3 Hormones: The Third Biggest Market for Orphan Drugs But Stagnant
6.3.1 Causes of Physiological Growth Failure
Table 6.4 Orphan Hormonal Treatments in the Top 400, 2003
6.3.2 The Three Top-Selling Growth Hormone Drugs
6.3.2.1 Genotropin: Pfizer Under Investigation
6.3.2.2 Humatrope: Eli Lilly's Growth Hormone
6.3.2.3 Nutropin, Nutropin Depot and Protropin
6.3.3 Lupron: The Best-Selling Hormonal Treatment
6.3.4 Sandostatin is Set to Overtake Lupron in 2004
6.3.4.1 VIPomas
6.3.4.2 Acromegaly
6.3.4.3 The Outlook for Sandostatin is Good
6.3.5 Serostim: An Uncertain Future for an Important Drug
6.3.5.1 HIV-Associated Wasting Affects Over a Third of HIV Sufferers
6.3.5.2 Short Bowel Syndrome: A New Treatment is Available
6.3.5.3 Serostim in Clinical Trials for HIV-Associated Adipose
Redistribution Syndrome
6.3.5.4 Serostim in 2004

Chapter 7. The Performance and Potential of Newly Approved Orphan Drugs,
2003-2004
7.1 Cancer Treatments Make Up 31% of Newly Approved Orphans
7.1.1 Alimta: Hoping to Follow in Gemzar's Footsteps
7.1.2 Bexxar: Not Selling as Well as Hoped
Graph 7.1 Cumulative Bexxar Sales, 2004
7.1.3 Velcade: Millennium's Growth-Driver
7.1.3.1 Velcade for Multiple Myeloma
7.1.3.2 Velcade is in Clinical Trials for Several Additional Indications
7.1.3.3 Velcade is the U.S. Market Leader
7.1.3.4 Velcade Approved in the EC
7.1.3.5 One of the Most Successful Oncology Drug Launches Ever
7.1.4 Vidaza: The Orphan Drug Treatment for MDS
7.2 Rebetol Oral: A New Anti-Infective for the Paediatric Market
7.3 An Additional Treatment for Acromegaly
7.4 A New Treatment for Parkinson's Disease
7.4.1 Parkinson’s Disease
7.4.2 Apokyn is One of the First Orphan Drugs for Parkinson's Disease
7.4.3 Apokyn is Currently Only a Rescue Therapy
Table 7.1 Drugs for Parkinson's Disease with Active Orphan Indications
7.5 Two Products Were Fast-Tracked to Market
7.5.1 Fabrazyme: The First Treatment for Fabry Disease
7.5.1.1 Fabry Disease
7.5.1.2 How Did Fabrazyme Generate so Much Revenue in Just 8 Months?

Chapter 8. Do the Best-Selling Orphan Drugs Have a Particular Profile?
8.1 Number of Indications and Patient Population Influences Orphan Drug
Sales
Graph 8.1 Total Potential Patient Population of the Top Ten Orphan Drugs
8.2 The High Price of Orphan Drugs
Graph 8.2 Cost Per Patient Per Year of the Top Ten Orphan Drugs
8.3 Type of Disease Treated
8.4 Experienced Marketers Drive the Orphan Drugs Market
8.5 The General Profile of a Successful Orphan Drug
Table 8.1 Summary of Features of the Top 10 Orphan Drugs

Chapter 9. The Future Orphan Drug Market 2005-2009
9.1 Smaller Companies Will Continue to Flourish Due to the Orphan Drug Act
9.2 Orphan Drug Status Will Be Used as A Stepping Stone to More Prevalent
Indications
9.3 Specialist Orphan Drug Companies May Diversify in the Future
9.4 Genzyme Will Gain a Greater Share of the Orphan Drug Market
9.5 Orphan Drugs Will Always Find a Path to Market, Despite Being Largely
Ignored by Big Pharma
9.6 The Number of Orphan Drugs with Marketing Approval Will Increase in the
Five Years to 2009
Graph 9.1 Predictions for Number of FDA Orphan Drug Approvals, 2004-2009
9.7 The OOPD Orphan Clinical Trials Budget Will More Than Double in the Five
Years to 2009
Graph 9.2 OOPD Orphan Drug Clinical Trials Budget, 2004-2009
9.8 Uptake of the Orphan Drug Tax Credit Will Increase Substantially in the
Five Years to 2009
Graph 9.3 Orphan Drug Tax Credits Uptake, 2004-2009
9.9 Pipeline Orphan Drugs
9.10 Pipeline Orphan Drugs for Cancer
Table 9.1 Selected Pipeline Orphan Drugs (Recently Designated, 2004)
9.10.1 Avastin
9.10.2 Tarceva
9.10.3 Thalomid as an Orphan Drug
Graph 9.4 Thalomid Sales, 2003-2009
9.11 Orphan Drugs with Blockbuster Potential
9.11.1 Alimta
9.11.2 Avastin
9.11.3 Cerezyme
9.11.4 Lamictal
9.11.5 Tarceva
9.11.6 Taxol
9.11.7 Thalomid
9.11.8 Zometa
Table 9.2 Selected Orphan Drugs with Blockbuster Potential, 2004-2009
9.12 Leading Therapeutic Categories of the Future Orphan Drugs Market
Graph 9.5 Top Ten Categories in the Orphan Drugs Market, 2009
9.12.1 The Outlook for the Cancer Market
Table 9.3 Growth Rates for the Leading Orphan Drug Therapeutic Categories,
2003-2009
9.13 New Legislation Will Secure the Future of the Orphan Drugs Market

Chapter 10. Medicare Reimbursement for Orphan Drugs
10.1 Devastating Cuts to Medicare Reimbursement Rates in 2003
10.2 Twenty Five Million Patients Affected
10.3 True Orphan Drugs?
10.4 Reimbursement For Single Indication Orphan Drugs Only
10.5 The Reimbursement for Orphan Drugs Will Decrease as More Drugs Receive
Multiple Indications
Graph 10.1 Medical Reimbursement for Orphan Drugs, 2001-2009

Chapter 11. Conclusion: The Orphan Drugs Market Will More Than Double in
Value by 2009
Graph 11.1 Total Number of Orphan Drugs on the Market, 2004-2009
Graph 11.2 Total Value ($bn) of the Orphan Drugs Market, 2003- 2009

Appendix. How To Apply for Orphan Drug Designation

1. Content and Format of a Request for Orphan Drug Designation
2. Tips on Applying for Orphan Drug Status

Companies mentioned in this report:
Actelion Pharma Ltd
Allergan
Almerall
Amgen
Avax
Aventis Pasteur
Aventis Pharmaceuticals
Barr Laboratories
Bayer
Berlex Labs
Bertex Pharma Inc
Biogen Idec
Biogen
BioMarin Pharma Inc
Bio-Technology General
Bristol-Myers Squibb
Bryan Corp
Celgene
Centocor
Chiron
Corixa Corp
Cumberland Pharma Inc
Diacrin
Eli Lilly
Endo Pharmaceuticals
Genentech
Genetics Institute
Genzyme LLC
Gilead Sciences
GlaxoSmithKline
Heyl Chemisch-Pharmzeutische Fabrik
Hoffman-La Roche Inc.
ILEX Oncology
ILEX Pharmaceuticals
Immunex
Janssen-Cilag
Johnson and Johnson
Ligand Pharmaceuticals
Medicaid
Medicare
Medimmune
Millennium and ILEX Partners
Millennium Pharmaceuticals
Mylan Laboratories
Nexstar Pharma
Nouval Pharma Inc.
Novartis
Novo Nordisk
Orphan Medical
Ortho Biotech Products
Ortho-McNeil
Pentech Pharmaceuticals
Pfizer
Pharmacia
Pharmion Corp
Priority Healthcare Corporation
Savient Pharmaceuticals
Scherer DDS
Schering-AG
Schering-Plough
Sensus Corp
Serono
Serono Laboratories Inc
Shire Pharmaceuticals
Somerset Pharmaceuticals Inc.
Tap Pharma
Teva Pharmaceuticals
Titan Pharmaceuticals Inc
Wyeth Pharmaceuticals

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Received on 2001-09-13 - 03:03 GMT