Abstract
Summary
The successful introduction of orphan products in Europe remains problematic
for the developers, although they can seek to alleviate some of the issues by
carefully developing a launch strategy.
The first marketing authorisations for orphan drugs in Europe took place a
decade ago. Since then there have been over 60 designated orphan products.
These products now account for annual sales of more than $6 billion.
Traditionally the development and marketing of orphan drugs has not been an
area of interest for major pharmaceutical companies, reflecting the view that
orphan drugs were specialty products with limited market potential thus
offering a poor fit for companies based upon the blockbuster model. However, a
number of major companies have reassessed their strategy with respect to
orphan drugs and five of the top ten have made explicit commitments to the
development of such drugs. This has seen a number of changes in the orphan
drug landscape and further developments are likely, especially from some of
those not yet active in the field.
The 302 drugs that are approved for the treatment of 373 orphan indications
only over a fraction of the approximately 7,000 rare diseases categorised as
rare by the National Institute for Health (US).
The products Glivec and Tracleer show, it is possible for orphan drugs to
generate substantial returns in Europe clearly demonstrating the commercial
viability of Europe as a market for orphan drugs. Orphan Drug Markets in
Europe - Key developments and the strategies of major pharmaceutical
companies is a unique report from URCH Publishing that offers the reader a
comprehensive overview of the state of the orphan medicines market in Europe.
The carefully authored study provides expert insight into the market to date,
regulatory challenges and future opportunities concerning orphan drugs.
Particular attention is paid to the differences in regulation and market
between Europe Japan and the US.
Some key findings from the report include:
- Within Europe sales of approved orphan products account for approximately
$6bilion.
- Novartis' Glivec (imatinib mesylate) sales are over $1.5 billion.
- Since January 2000 there has been a steady increase in applications for
orphan designation with the Committee for Orphan Medicinal Products (COMP)
averaging ten positive recommendations per month.
- Five companies generated major revenues from the marketing of orphan drug
products in 2010, four primarily by supplying biological products while just
Actelion supplied small molecule therapeutics.
- Big pharma has started to take orphan drugs seriously and a number have
formed dedicated business units to explore opportunities.
- Alexion's Soliris shows that small patient populations are not
incompatible with commercial success.
- Although central approval covers 30 European countries, it does not
necessarily provide for national availability as each national authority has
to agree labelling and reimbursement.
- The increasing collaboration between the FDA and EMA has led to the
adoption of a common application form should facilitate developers' efforts to
exploit orphan drugs on a more international basis.
Reasons to Buy
- Obtain a complete understanding of the orphan drug regulations in Europe.
- Gain insight into the key companies operating in this sector.
- Understand the differences between the Europe and US markets.
- Evaluate which rare diseases offer the greatest commercial opportunity.
- Assess the performance of specialist orphan drug companies.
- Review how big pharma is starting to enter this market segment.
- Find the new areas of pharmaceutical market growth and key opportunities
for delivering successful sales growth over the next five years.
- Support internal planning and decision-making with an external perspective
founded on detailed analysis and transparent market forecasts.
Who should read this unique report
This report is designed for anyone who needs a comprehensive overview of
orphan medicines in Europe, including:
- Strategy managers and directors in large pharmaceutical companies
- Biotechnology companies with niche product pipelines
- Investors looking for opportunities
- US-based companies with an interest in expanding to Europe.
Companies & Organisations Mentioned in the Report Include
Actelion, Alexion, Alnara Pharmaceuticals, Amgen, Amicus Therapeutics,
AmpliPhi Biosciences, Aventis, Bayer, BiogenIdec, Biovitrum, bluebird bio,
Bristol-Myers Squibb, Celgene, Cephalon (Teva), COMP, Del Mar Pharmaceuticals,
Edimer Pharmaceuticals, Eli Lilly, EURODIS, EUSA Pharma, FDA, Genethon,
GlaxoSmithKline, Hyperion Therapeutics, Intermune, Isis Pharmaceuticals, JCR
Pharmaceutical, Lotus Tissue Repair, Merck, National Institute for Health,
NORD, Novartis, Novo Nordisk, Orfagen, Orphan Europe (Recordati), Orphan
Therapeutics, Pfizer, Pharmamar, Prosena, Protalix Biotherapeutics, Roche,
Sanofi, Santhera Pharmaceuticals, Shire, Sigma-Tau, SpePharm, Swedish Orphan
Biovitrum, Sygnis Bioscience, Synageva BioPharma, Takeda, Teva, Trophos,
United Therapeutics, Vivendy Therapeutics, Voisin Consulting, Zacharon
Pharmaceuticals, Zymenex
Orphan medicines mentioned in this report include
Adcirca - Aldurazyme - Avonex - Campath - Cerezyme - ClolarCopaxone -
Elaprase - Epogen - Esbriet - Evoltra - ExJade - Fabrazyme - Gleevec / Glivec
- Kogenate - Mozobil - Myozyme (and Lumizyme) - Neupogen - Nexavar - Novoseven
- Provigil - Pulmozyme - Rebif - Revatio - Revlimid - Rituxan - Sensipar -
Soliris - Somavert - Sprycel - Temodar - Thyrogen - Tracleer - Velcade -
Veletri - Ventavis - Xagrid - Yondelis - Zavesca
Table of Contents
Executive Summary
Chapter 1: An introduction to orphan drugs in Europe
Chapter 2: Orphan Drug Legislation
- Global progress
- The United States
- Europe
- Japan
- Other markets
- Singapore
- Australia
- Taiwan
- Switzerland
- South Korea
- Brazil
- A comparison
Chapter 3: Rare Diseases and Orphan Drugs
- Overview
- Rare Diseases
- Genetic disorders
- Cystic fibrosis
- Muscular dystrophy
- Thalassaemia
- Lysosomal storage disorders
- Cancer
- Very Rare Diseases
- Repurposing
- Patient registries
Chapter 4: Progress of orphan drugs in Europe
- Introduction
- Revenues from orphan drugs in Europe
- What is similar?
- Designated orphan drugs
- Success stories
- Soliris (Alexion)
- Enzyme replacement therapies
- Tracleer
- Glivec (Novartis)
- Fragmented market issues
Chapter 5: Orphan drugs in the US
- Overview
- 25 years of approvals
- Major successes
Chapter 6: Specialist Companies
- Introduction
- Major players
- Genzyme
- Actelion
- Shire
- BioMarin
- Alexion
- Established specialist players
- Orphan Europe (Recordati)
- Sigma-Tau SpA
- Swedish Orphan Biovitrum
- PharmaMar
- EUSA Pharma
- Orfagen
- Emerging speciality companies
- Amicus Therapeutics
- AmpliPhi Biosciences
- bluebird bio
- Del Mar Pharmaceuticals
- Edimer Pharmaceuticals
- Genethon
- Hyperion Therapeutics
- Lotus Tissue Repair
- Orphan Therapeutics
- Prosena
- Santhera Pharmaceuticals
- SpePharm
- Sygnis Bioscience
- Synageva BioPharma
- Trophos
- Vivendy Therapeutics
- Zacharon
- Zymenex
Chapter 7: Major pharmaceutical companies' involvement in orphan drugs
- Introduction
- Eli Lilly
- GlaxoSmithKline
- Novartis
- Pfizer
- Sanofi
- Other companies
- Outlook
Chapter 8: Outlook for orphan drugs
- Targeting rare diseases more popular
- High profile
- Commercial opportunities
- Potential pitfalls
- Competition issues
- European specific issues
- Conclusions
- Bibliography
- Glossary
List of Figures
- Figure 2.1 Global Time Line of Orphan Drug Legislation
- Figure 2.2 FDA definitions of similarity
- Figure 2.1 EMA key relationships relating to the designation and approval
of orphan drugs
- Figure 2.2 EU regulations pertaining to the orphan drug regulation
(Regulation 141/2000)
- Figure 2.3 The process of orphan drug designations by the COMP
- Figure 2.4 The number of applications to the COMP for orphan drug
designation, and their outcomes, to July 2011
- Figure 3.1 Relative prevalence of 27 lysosomal storage disorders in
Australia
- Figure 4.1 Orphan drug authorizations in Europe by year (to July 2011)
- Figure 4.2 Orphan drug approvals by marketing company
- Figure 4.5 Approved orphan drugs by therapeutic area
- Figure 6.4 Potential return from an orphan therapeutic in Europe for a
prevalent indication
- Figure 4.7 Applications for Orphan Drug Designation in Europe
- Figure 4.8 Unsuccessful Applications for Orphan Drug Designation in Europe
- Figure 4.9 COMP opinions by patient population
- Figure 4.10 Geographic availability of 21 orphan drugs in Europe in 2007
- Figure 4.11 Confirmed availability, and unavailability, of 60 orphan drugs
in selected EU countries in 2007
- Figure 4.12 Average price and reimbursement delays for generic products by
country
- Figure 4.13 Intermune's proposed EU launch strategy for Esbriet
- Figure 5.14 European orphan designations by disease prevalence to 2010
- Figure 5.15 New Drug Approvals by the FDA 2004-2010
- Figure 7.1 The changing orphan drug landscape and major pharma
- Figure 8.1 Competitive developments for treating Gaucher disease
- Figure 8.2 Availability of 21 approved orphan drugs in Europe in 2007
- Figure 8.3 Intermune's European launch strategy for Esbriet
List of Tables
- Table 2.1 Comparison of Orphan Drug Criteria in the US, the EU and Japan
- Table 4.1 European status of therapies for pulmonary hypertension
- Table 5.2 Selected commercially successful biological orphan products in
the US
- Table 5.3 Selected commercially successful small molecule orphan products
in the US
- Table 6.4 Genzyme's orphan drug portfolio and 2010 sales
- Table 6.5 Genzyme's orphan drug pipeline
- Table 6.6 Actelion's orphan drug portfolio and 2010 sales
- Table 6.7 Actelion's orphan drug pipeline
- Table 6.8 Shire's orphan drug portfolio and 2010 sales
- Table 9.6 Shire's orphan drug pipeline
- Table 6.10 BioMarin's orphan drug portfolio and 2010 sales
- Table 6.11 BioMarin's orphan drug pipeline
- Table 6.12 Recordati's orphan drug portfolio
- Table 6.13 Recordati's orphan drug pipeline
- Table 6.11 Sigma-Tau's orphan drug pipeline
- Table 6.12 Swedish Orphan Biovitrum's orphan drug portfolio and 2010
revenues
- Table 6.13 PharmaMar's orphan drug pipeline
- Table 6.14 Orfagen's orphan drug pipeline
- Table 6.15 Amicus' orphan drug pipeline
- Table 6.16 Prosensa's orphan drug pipeline
- Table 6.17 Santhera's orphan drug pipeline
- Table 6.18 Synagis' orphan drug pipeline
- Table 6.14 Synageva's orphan drug pipeline
預計歐洲孤兒藥(孤兒藥 drug)的數量將擴大，大製藥公司表示關心
2011年12月28日
Global Information, Inc.已開始銷售Urch Publishing, Ltd.所發行的報告書「Orphan Drug Markets in Europe (歐洲的孤兒藥物市場)」
本報告書預測在歐洲獲得認可、銷售的孤兒藥數量穩定增加。
過去10年許多孤兒藥在商業上獲得成功，提高大製藥公司的關心。大製藥公司的暢銷藥存在專利期限到期等問題，今後亦無法持續增加高收益。
許多大製藥公司已經再度評估有關孤兒藥的策略，前10大公司中的5家公司明確表示要開發孤兒藥。
本報告書的執筆者、亦是分析家的Dr Peter Norman敘述：「開發稀少疾病治療法，對製藥公司來說，是提高最近惡化中的企業形象之機會，亦帶來維持企業收益的商機。」。
「最近，GSK、Pfizer和Sanofi透過收購和合作，專心致力於強化有關稀少疾病的研究。」
然本報告書指出，對開發者來說，與比歐洲規模小的美國市場相較，歐洲的孤兒藥市場課題更多。
EU依據中央認可審查方式，對加盟30個國家同時進行醫藥品的認可，然並非獲得認可後，醫藥品就自動在各國上市。
各國當局針對各自標籤和償還須獲得認可，結果導致醫藥品延遲上市，孤兒藥亦在很後面才能讓許多加盟國利用。
依據EURODIS實施的2個調查，獲得認可完畢的孤兒藥全部（或幾乎全部）都可利用的，只包括法國和德國數個國家。
因此，歐洲孤兒藥的投入市場策略，對EU區域內的償還問題、潛在患者數及患者分佈的慎重計劃就變成是必要的。
然關於一般較稀少的疾病，主要5個國家早期使孤兒藥上市的策略，與孤兒藥以外的藥劑一樣，成為令人喜歡選擇的方案。
