Global Orphan Drug Pipeline & Regulatory Insight 2015

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Global Orphan Drug Pipeline Regulatory Insight
2015
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Summary
Rare diseases are also known as Orphan diseases.
These diseases are generally categorized as
chronic, degenerative, life threatening in nature
and normally include certain cancers, metabolic
conditions, diseases of the nervous system and
musculoskeletal disorders. As per the official
definition, rare diseases are classified as those
that affect fewer than 200,000 in the United
States and less than 5 in 10,000 in the EU. These
two regions form a dominant portion of the global
market for orphan drugs. In Japan, any disease
with less than 50,000 prevalent cases is defined
as a rare disease. In Australia, the perspective
is different. The Therapeutic Substances
Regulations in Australia defines rare drugs as
those which should not be intended for use in
more than 2000 patients annually. In Taiwan, the
official definition of rare diseases is that
these diseases would be termed rare if the
prevalence rate is 110,000 people.
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Owing to the pharmaceutical companies
inadequate interest in this category of drugs,
the term orphan was coined for drugs targeted
to treat rare diseases. Almost 222 orphan
designated drugs in US and 132 in Europe are
marketed till date. However, in spite of the
increased efforts in this market, there is still
a shortage in the number of treatments for most
rare disease indications. Thus, the market for
rare diseases in the United States, Europe and
other regions continue to offer significant
potential for growth owing to enormous unmet
medical need.Globally, there was a desire to
address the unmet treatment needs of orphan
diseases, which transformed into the 1983 U.S.
Orphan Drug Act, as well as similar Acts in 1991
in Singapore, 1993 in Japan, 1997 in Australia
and in 2000 by the European Union. The
introduction of these acts, along with
high-profile philanthropic funding, has made
orphan diseases an attractive segment and
encouraged investment in RD for a number of
these destructive conditions.
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For drugs to get an orphan designation in Korea,
less than 20,000 people in Korea should suffer
from the disease/condition, or no other
alternative treatment should be available for the
disease in the country. Historically, the
development of 70-75 of orphan drugs was mostly
in the realm of smaller biotechnology and
specialty pharmaceutical companies, and the
remaining 25-30 of the orphan drugs were
developed by the pharma giants. However, in the
past decade, the share of pharma giants in the
orphan drug approvals has increased to close to
40-45. The US and European region are the major
markets for orphan drugs. It is in these two
regions that the popularity of these drugs and
diseases is increasing rapidly. The US alone
accounts for a dominating share of 55-60 in the
global pie, followed by Europe. As the Asian
pharmaceutical markets are still in nascent
stage, there opportunities for orphan drugs in
Asia are also immense and getting increasingly
popular. It is expected that by 2017-18, the
Asian orphan drugs market would witness steady
growth.
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Global Orphan Drug Pipeline Regulatory Insight
2015 Report Highlights Findings Global
Orphan Drug Market Overview In-depth Insight on
Regulatory Framework for Orphan Drugs by
Region Orphan Drug Designation Criteria
Reimbursement Policy by Region Comprehensive
Insight on Global Orphan Drug Clinical Pipeline
Global Orphan Drug Clinical Pipeline 697 Drugs
Majority Orphan Designated Drugs in Phase-II
249 Globally Marketed Orphan Drugs 274
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Table of Contents
1. What Are Orphan Drugs? 1.1 Introduction To
Orphan Drug. 1.2 Favorable Parameters For Orphan
Drugs Acceptance. 2. Global Orphan Drug Market
Outlook. 2.1 Market Overview. 2.1.1 By Biological
Non-Biological Orphan Drugs. 3. Orphan Drug
Designation Criteria. 3.1 US Orphan Drug
Designation Criteria. 3.2 Europe Orphan Drug
Designation Criteria. 4. Reimbursement Policy
For Orphan Drug. 4.1 US Orphan Drug Reimbursement
Policy. 4.2 Europe Orphan Drug Reimbursement
Policy. 5. US (FDA) Regulatory Framework For
Orphan Drugs. 5.1 Content Format Of A Request
For Written Recommendations. 5.2 Provision For
Granting Refusing Written Recommendations.
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To view the full Executive Summary and Table of
Contents, please visit Global Orphan Drug
Pipeline Regulatory Insight 2015 Contact
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