Global Orphan Cancer Drug Market & Clinical Pipeline Insight

The drug development strategies of the pharmaceutical companies is undergoing a paradigm

shift from manufacturing traditional essential medicines to orphan drugs due to significant
competition from generics and the associated economic situation. The returns on investment

from orphan drugs are expected to help the pharma companies to overcome the impact of
revenue loss due to expiry of patents of blockbuster drugs. The major drivers for companies
to increasingly invest in orphan drugs include government incentives for drug development

and strong support from the FDA and EU Commission in special protocols. Inspite of pharma
companies facing many obstacles in this field, this segment of orphan drugs is likely to have
the potential to provide the key to recovery and stability within the market.

Oncology has emerged as the most promising and revenue generating segment of global
orphan drug market in recent years. A major reason for such a high level of concentration of
oncology particularly is that the underlying genetic aberration of oncology generally helps in

the process of identification of the disease. Oncology, as a therapeutic area has also

transformed from evolution to customized or precision medicine in the pharmaceutical

industry. Utilizing biomarkers to develop targeted treatments and therapies is an approach
which is being increasingly used in recent years, which also further explains the reasons
behind this therapeutic areas majority share of orphan drug approvals.

Owing to high level of demand for better treatments for multiple types of cancer, there has

been a shift in focus of the pharmaceutical and biotechnology companies towards the
Oncology sector. The global oncology market is experiencing a paradigm shift in terms of

targeted therapy methods, which would be used for patients in the coming decade.
Specifically, breast cancer and non-small cell lung cancer (NSCLC) would continue to drive
high levels of R&D due to the high incidence and subsequent potential for market success.

With the oncology pipeline for orphan drugs being very promising, it is expected that over the

next few years, many drugs are likely to be developed over a range of indications including
the commonly diagnosed lung and colorectal cancer types. With the introduction of these
products in the market, it is likely that there would be a significant improvement in the
survival rates of the patients.

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Global Orphan Cancer Drug Market & Clinical Pipeline Insight

Global Orphan Cancer Drug Market & Clinical Pipeline Insight Report
Highlights:

Orphan Cancer Drug Market Outlook

Orphan Drug Designation Criteria: US, Europe & Asia

Reimbursement Policy: US, Europe & Asia

Regulatory Framework: FDA & EMA Guidelines

Orphan Cancer Drugs Clinical Pipeline by Phase, Country & Target Indications

Orphan Cancer Drug Patent Analysis

Orphan Cancer Drug in Development Phase: 235

Majority of Orphan Cancer Drugs in Phase-II: 81

Marketed Orphan Cancer Drugs: 70

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Global Orphan Cancer Drug Market & Clinical Pipeline Insight

Table of Contents
1. Introduction to Orphan Drugs
1.1 What are Orphan Drugs?
1.2 Why Need for Orphan Drugs?
2. Orphan Drug Designation Criteria: US, Europe & Asia
3. Orphan Drug Patent Protection & Market Exclusivity
4. Orphan Drug Regulatory Framework
4.1 FDA Guidelines
4.2 EMA Guidelines
5. Orphan Drug Reimbursement Policy: US, Europe & Asia
6. Global Orphan Cancer Drug Market Outlook
6.1 Market Overview
6.2 Orphan Cancer Drug Clinical Pipeline Analysis
7. Orphan Cancer Drugs Clinical Pipeline by Phase, Country & Target Indications
7.1 Phase Unknown
7.2 Preclinical
7.3 Clinical
7.4 Phase I
7.5 Phase I/II
7.6 Phase II
7.7 Phase II/III

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Global Orphan Cancer Drug Market & Clinical Pipeline Insight

7.8 Phase III
7.9 Preregistration
7.10 Registered
8. Marketed Orphan Cancer Drugs
9. Suspended & Discontinued Orphan Cancer Drugs in Clinical Pipeline
9.1 No Development Reported
9.2 Discontinued
10. Competitive Landscape
10.1 Bristol Myers Squibb
10.2 Celgene
10.3 Eli Lilly
10.4 Enzon-Pharmaceuticals
10.5 Endocyte
10.6 Glaxosmithkline
10.7 Merck
10.8 Novartis Pharmaceuticals
10.9 Nerviano Medical Sciences
10.10 Pfizer
10.11 Roche
10.12 Sanofi

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Global Orphan Cancer Drug Market & Clinical Pipeline Insight

List of Figures
Figure 1-1: Orphan v/s Non-Orphan Drugs -Phase II to Launch Clinical Development Time
Figure 1-2: Cancer Drug Approval Time (Years)
Figure 1-3: Percentage of Cancer Drug Approved
Figure 1-4: Probability of Regulatory Success of Orphan v/s Non-Orphan Drugs
Figure 1-5: Time Approval for Medicine With Special & Non Special Status
Figure 1-6: Number of Orphan Cancer & Non-Cancer Drug Approval (2002, 2007-2011)
Figure 1-7: Orphan Drug Designation V/s Orphan Drug Approval
Figure 3-1: Orphan Drug Market Exclusivity by Country
Figure 6-1: Global Orphan Drugs Market Value (US$ Billion), 2012-2018
Figure 6-2: Global Orphan Drugs Market by Therapeutic Area, 2012 & 2018
Figure 6-3: Orphan Cancer Drug Market (US$ Billion), 2012-2018
Figure 6-4: Orphan Cancer Drug Pipeline by Clinical Phase (%), 2014
Figure 6-5: Orphan Cancer Drug Pipeline by Clinical Phase (Number), 2014
Figure 6-6: Discontinued & No Development Reported in Orphan Cancer Drug Pipeline, 2014

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