Reportlinker Adds Orphan Drug Strategies

October 16, 2009

NEW YORK, Oct. 16 /PRNewswire/ — Reportlinker.com announces that a new market research report is available in its catalogue.

Reportlinker Adds Orphan Drug Strategies



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.


*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


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.

Reasons to Purchase

*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.


About the Strategic Pharmaceutical Analysis Team 2

Executive Summary 3

Strategic scoping and focus 3

Datamonitor insight into the disease market 3

Related reports 5

Table of Contents 6

2. Orphan drug overview 7

Key findings 7

Orphan drug definitions 8

US orphan drug criteria 9

EU orphan drug criteria 9

Japanese orphan drug criteria 10

Orphan drug designation comparisons across the US, EU, and Japan 11

Ultra-orphan drug and disease criteria 12

Drivers and resistors to orphan drug development and commercialization 13

Developmental and commercial barriers to orphan drugs 13

The rate of uptake of orphan drugs is similar to that of non-orphan drugs 14

Orphan drug status offers no protection against generic erosion 17

Drivers and incentives for orphan drug development and commercialization 18

Market-specific orphan drug incentives 20

US incentives 20

EU initiatives 22

Japanese initiatives 24

3. Orphan drug development and approval trends 26

Key findings 26

Orphan drug designation and approval trends 27

Decline in recent number of US orphan drug designations linked to Biotech funding crisis 27

2009 decline in orphan drug designations linked to biotech funding crisis 29

Continued forecast growth for European orphan drug designations and approvals 30

Factors contributing to EMEA refusal of orphan drug designations 32

Orphan drug approval trends in Japan 34

Orphan drug research and development trends 35

Clinical trial and approval strategies for orphan drugs 35

Adaptive clinical trials ideally suited to orphan drug assessment 38

Orphan drug pipeline trends 40

Orphan drug reimbursement issues 45

Orphan drug access in the US 45

Orphan drug access in Europe 45

Orphan drug risk-sharing in the UK set to become more frequent driven by new drug launches and disease stratification 46

4. Orphan drug strategies and future outlook 48

Key findings 48

Big Pharma’s growing attraction for orphan drugs 49

Big Pharma’s dominance in the orphan drug market 50

Strategies employed to grow Big Pharma’s orphan drug presence 51

Three key orphan drug strategies 52

Market access and subsequent expansion into non-orphan diseases offers significant opportunity for revenue generation 53

Multiple indications shore up a drug’s exclusivity in a niche therapeutic area 53

Stratification of indications into niche patient populations 55

Segmentation through pediatric orphan indications and disease specialization 56

The growing use of biomarkers in disease stratification 56

Orphan drug specialists 58

Genzyme’s success in ultra-orphan markets 58

Actelion’s orphan drug portfolio driven by pulmonary arterial hypertension 59

Orphan drug sales forecast 61

5. Bibliography 63

Publications and online articles 63

Datamonitor reports 69


About Datamonitor 73

About Datamonitor Healthcare 73

Datamonitor consulting 73

Disclaimer 75

List of Tables

Table 1: Comparison of orphan product policies across the US, EU and Japan, 2009 11

Table 2: Non-orphan, orphan and ultra-orphan drug designations in the US, 2009 12

Table 3: Comparison of orphan and non-orphan drug growth rates after launch, 2005-08 14

Table 4: Comparison of access to orphan drugs in selected EU countries, 2009 23

Table 5: Summary of US and EU orphan drug designations, approvals and approval rates, 1980s-2000s 28

Table 6: Top 20 most frequently targeted orphan indications by manufacturers in the EU, 2000-08 43

Table 7: FDA approved orphan drugs with multiple indications, 2009 54

Table 8: Drugs with at least one orphan drug status and associated orphan biomarkers 57

Table 9: Most frequently targeted orphan indications by manufacturers in the EU, 2000-08 70

Table 10: Exchange rates, 2008 72

List of Figures

Figure 1: Orphan drug policies by country 8

Figure 2: Uptake of orphan and non-orphan drugs during the first 3 years post launch, 2005-08 15

Figure 3: Erosion of orphan drugs versus average US brand erosion following patent expiry, 2005-08 17

Figure 4: The sliding scale of blockbuster sales: primary care drugs to ultra-orphan drugs 18

Figure 5: Factors driving the shift away from the primary care blockbuster model towards niche indications 19

Figure 6: Molecule type of approved orphan drugs, 2007 27

Figure 7: Number of designated and approved orphan drugs in the US, 1993-Q2 2009 29

Figure 8: Number of designated, approved, withdrawn/suspended and refused orphan drugs in the EU, 2000-Q2 2009 30

Figure 9: Number of orphan drug designations across a selection of European countries, 2000-07 31

Figure 10: Factors responsible for refusal of EMEA market authorization 32

Figure 11: Factors responsible for refusal of EMEA orphan drug designations 33

Figure 12: Number of orphan drugs receiving approval from the Japanese Pharmaceuticals and Medical Devices Agency (PMDA), 2004-07 34

Figure 13: US orphan drug Phase I-III clinical trial designs, 2009 36

Figure 14: Orphan drug comparator designs by phase of clinical trial 37

Figure 15: Reasons for adopting adaptive clinical trials for orphan drugs 38

Figure 16: Factors to be considered when designing an adaptive clinical trial 39

Figure 17: US and European pipeline orphan designated drugs by clinical trial phase, 2009 40

Figure 18: US pipeline orphan designated drugs by therapy area, 2009 41

Figure 19: EU orphan drug market authorizations by therapy area, 2008 42

Figure 20: Japanese orphan drug approvals by therapy area, 2004-07 44

Figure 21: Factors to consider when developing risk-sharing agreements for orphan drugs 47

Figure 22: End goals of orphan drug strategies for biotech and Big Pharma 49

Figure 23: Orphan drug developers split by company type, 1990-2008 50

Figure 24: Orphan drug strategies to increase revenue potential through sales expansion or protection 52

Figure 25: Criteria to be considered when stratifying a disease into smaller niche and potentially orphan indications 55

Figure 26: Forecast global sales of Genzyme’s orphan and non-orphan drug portfolios, 2008-2014 59

Figure 27: Forecast global sales of Actelion’s orphan and non-orphan drug portfolios, 2008-2014 60

Figure 28: 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 62

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Reportlinker Adds Orphan Drug Strategies


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CONTACT: Nicolas Bombourg of Reportlinker, Email: nbo@reportlinker.com, US: +1-805-652-2626, Intl: +1 805-652-2626

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