孤兒藥品策略 是由出版商Datamonitor在2009年10月所出版的。
這份英文市場調查報告書包含75 pages 價格從美金3800起跳。
在藥品產業針對基礎照護所需的暢銷藥品模式矚目程度下降當中，大型製藥企業正努力開發符合利基市場需求，具有高度價值之小眾患者治療用孤兒藥品，並積極促進其商品化。
本報告書內容包括：孤兒藥品概要及開發、許可動向、大型製藥企業的未來預測、該市場未來的規模及成長性等。內容綱要摘記如下：
實施概要
第2章 孤兒藥品概要
- 主要調查結果
- 孤兒藥品定義
- 美國
- EU
- 日本
- 美國、EU、日本的孤兒藥品指定比較
- 超級孤兒藥品及疾病標準
- 孤兒藥品開發及商品化的促進因素及阻礙因素
第3章 孤兒藥品開發及許可動向
- 主要調查結果
- 孤兒藥品指定及許可動向
- 孤兒藥品研究開發動向
- 孤兒藥品補貼動向
第4章 孤兒藥品策略及未來預測
- 主要調查結果
- 對大型製藥企業而言孤兒藥品的魅力
- 3大策略
- 孤兒藥品的專業組織
- 孤兒藥品銷售金額預測
第5章 參考文獻
附錄
Abstract
Introduction
With the pharmaceutical industry reducing its focus on the primary care
blockbuster model, Big Pharma is now looking towards the development and
commercialization of more niche, high value, often biologic therapies, for the
treatment of smaller patient populations.
Scope of this research
- Provides an overview of orphan drugs, including drivers and resistors of
investing in this niche market
- Examines orphan drug developmental and approval trends
- Analyzes Big Pharma' s attraction towards orphan drugs, supplemented with
future forecast analysis
- Assesses the future size and growth of the orphan drugs market
Research and analysis highlights
Few markets have implemented robust orphan drug policies with the exception of
the US, Europe, Japan, Australia and Singapore. Nevertheless, a number of key
emerging markets have instigated partial orphan drug policies which are
anticipated to be strengthened in the near future.
With increasing cost pressures facing payers, combined with the growing number
of orphan drugs now on the market and in development, measures aimed at
reducing costs can be expected by shifting payment onto the patient or even
the pharma companies themselves through risk-sharing agreements.
Oncology is the most frequently investigated therapy area, with nearly half of
all pipeline orphan drugs designated for such indications. However, with the
availability of marketed products for a number of the most frequently targeted
orphan indications, competition in these areas is set to grow.
Key reasons to purchase this research
- Gain insight into the challenges companies face when developing and
commercializing orphan drugs.
- Examine which are the most popular orphan indications, and the most
dominant orphan drug players in the market.
- Identify strategies to maximize the commercial opportunities of orphan
drugs through both revenue expansion and protection.
Table of Contents
1. EXECUTIVE SUMMARY
- Strategic scoping and focus
- Datamonitor insight into the disease market
- Related reports
2. ORPHAN DRUG OVERVIEW
- Key findings
- Orphan drug definitions
- US orphan drug criteria
- EU orphan drug criteria
- Japanese orphan drug criteria
- Orphan drug designation comparisons across the US, EU, and Japan
- Ultra-orphan drug and disease criteria
- Drivers and resistors to orphan drug development and commercialization
- Developmental and commercial barriers to orphan drugs
- Drivers and incentives for orphan drug development and commercialization
- Market-specific orphan drug incentives
3. ORPHAN DRUG DEVELOPMENT AND APPROVAL TRENDS
- Key findings
- Orphan drug designation and approval trends
- Decline in recent number of US orphan drug designations linked to
Biotech funding crisis
- Continued forecast growth for European orphan drug designations and
approvals
- Orphan drug approval trends in Japan
- Orphan drug research and development trends
- Clinical trial and approval strategies for orphan drugs
- Orphan drug pipeline trends
- Orphan drug reimbursement issues
- Orphan drug access in the US
- Orphan drug access in Europe
4. ORPHAN DRUG STRATEGIES AND FUTURE OUTLOOK
- Key findings
- Big Pharma' s growing attraction for orphan drugs
- Big Pharma' s dominance in the orphan drug market
- Strategies employed to grow Big Pharma' s orphan drug presence
- Three key orphan drug strategies
- Market access and subsequent expansion into non-orphan diseases offers
significant opportunity for revenue generation
- Multiple indications shore up a drug' s exclusivity in a niche
therapeutic area
- Stratification of indications into niche patient populations
- Orphan drug specialists
- Genzyme' s success in ultra-orphan markets
- Actelion' s orphan drug portfolio driven by pulmonary arterial
hypertension
- Orphan drug sales forecast
5. BIBLIOGRAPHY
- Publications and online articles
- Datamonitor reports
APPENDIX
TABLES
- Table: Comparison of orphan product policies across the US, EU and Japan,
2009
- Table: Non-orphan, orphan and ultra-orphan drug designations in the US,
2009
- Table: Comparison of orphan and non-orphan drug growth rates after launch,
2005 - 08
- Table: Comparison of access to orphan drugs in selected EU countries, 2009
- Table: Summary of US and EU orphan drug designations, approvals and
approval rates, 1980s - 2000s
- Table: Top 20 most frequently targeted orphan indications by manufacturers
in the EU, 2000 - 08
- Table: FDA approved orphan drugs with multiple indications, 2009
- Table: Drugs with at least one orphan drug status and associated orphan
biomarkers
- Table: Most frequently targeted orphan indications by manufacturers in the
EU, 2000 - 08
- Table: Exchange rates, 2008
FIGURES
- Figure: Orphan drug policies by country
- Figure: Uptake of orphan and non-orphan drugs during the first 3 years
post launch, 2005 - 08
- Figure: Erosion of orphan drugs versus average US brand erosion following
patent expiry, 2005 - 08
- Figure: The sliding scale of blockbuster sales: primary care drugs to
ultra-orphan drugs
- Figure: Factors driving the shift away from the primary care blockbuster
model towards niche indications
- Figure: Molecule type of approved orphan drugs, 2007
- Figure: Number of designated and approved orphan drugs in the US, 1993 -
Q2 2009
- Figure: Number of designated, approved, withdrawn/suspended and refused
orphan drugs in the EU, 2000 - Q2 2009
- Figure: Number of orphan drug designations across a selection of European
countries, 2000 - 07
- Figure: Factors responsible for refusal of EMEA market authorization
- Figure: Factors responsible for refusal of EMEA orphan drug designations
- Figure: Number of orphan drugs receiving approval from the Japanese
Pharmaceuticals and Medical Devices Agency (PMDA), 2004 - 07
- Figure: US orphan drug Phase I - III clinical trial designs, 2009
- Figure: Orphan drug comparator designs by phase of clinical trial
- Figure: Reasons for adopting adaptive clinical trials for orphan drugs
- Figure: Factors to be considered when designing an adaptive clinical trial
- Figure: US and European pipeline orphan designated drugs by clinical trial
phase, 2009
- Figure: US pipeline orphan designated drugs by therapy area, 2009
- Figure: EU orphan drug market authorizations by therapy area, 2008
- Figure: Japanese orphan drug approvals by therapy area, 2004 - 07
- Figure: Factors to consider when developing risk-sharing agreements for
orphan drugs
- Figure: End goals of orphan drug strategies for biotech and Big Pharma
- Figure: Orphan drug developers split by company type, 1990 - 2008
- Figure: Orphan drug strategies to increase revenue potential through sales
expansion or protection
- Figure: Criteria to be considered when stratifying a disease into smaller
niche and potentially orphan indications
- Figure: Forecast global sales of Genzyme' s orphan and non-orphan drug
portfolios, 2008 - 2014
- Figure: Forecast global sales of Actelion' s orphan and non-orphan drug
portfolios, 2008 - 2014
- Figure: Historical and forecast sales of drugs with orphan status for at
least one indication, marketed by the top 50 global pharmaceutical companies
in the US and five major EU market, 2001 - 2014
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