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Disease incidences are rapidly escalating across the globe and few rare diseases have
emerged for which effective medications are not available. Occurrence of such disease is
not unusual and their roots could be traced back to genetic or metabolic anomalies. They
affect limited number of individuals and widely uncommon in nature. Such diseases are
called rare diseases and corresponding drugs are known as orphan drugs. The orphan drug
status to a particular formulation depends upon the public health policy due to which
number of individuals may vary from country to country. This fact reflects the marketing
opportunity in particular region but largely limited market size is observed. Previously,
the market for orphan drugs was not as lucrative as it is today. Due to small patient
population, the pharmaceutical companies generally ignored them and hence these drugs
were given the designation "orphan. However, in 1983, the Orphan Drug Act was passed
in the US in order to encourage pharmaceutical companies to develop orphan drugs
followed by enactment of law for orphan drugs by European Medical Agency in 1999.
Encouragingly, large numbers of orphan drugs have been introduced in global market and
many more are at different stages of clinical trials. Innovative technologies are being
employed for the development of these orphan drugs. For instance, bone marrow stemcells are being explored for treatment of Amyotrophic Lateral Sclerosis (ALS). Targeted
RNAi therapies for Duchenne Muscular Dystrophy (DMD) are under investigation. Market
introduction of orphan drugs based on these innovative therapies may take time but their
future seems optimistic due to increasing activity of pharmaceutical companies in this
segment. Moreover, owing to continuous effort of orphan drug developers for several
decade, many medical breakthroughs have been achieved which are also applicable for
other diseases. For instance, statins drugs developed for homozygous familial
hypercholesterolemia is widely prescribed for cardiac disorders.
Strong clinical pipeline of orphan drugs for different rare diseases using innovative
technologies
makes
them
worthy
candidates
having
high
pharmacological
and
commercialization potential. But, challenges in clinical trials may hamper their growth
and limit their marketing potential. Owing to low patient base it is not always possible to
generate high quality data in clinical trials. Regulatory authorities also understand this
issue due to which some relaxations have been made which is imperative in such
scenarios. Their clinical trials are quite risky and orphan drug developer may run the
chances of having high failure rates. This may lead to loss of valuable time and resources
factors.
Introduction
of
innovative
technologies,
favorable
pricing,
reimbursement, unmet medical necessities and strong clinical pipeline could be attributed
as important factors responsible for their growth.
"Global Orphan Drug Market Future Outlook 2020" report highlights:
Table of Contents
15.6 Celgene
15.7 Eli Lilly
15.8 Genethon
15.9 Genzyme Corporation
15.10 Glaxosmithkline
15.11 Merck
15.12 Novartis Pharmaceuticals
15.13 Orphan Europe
15.14 Pfizer
15.15 Prosensa
15.16 Rare Disease Therapeutics
15.17 Roche
15.18 Sanofi
15.19 Shire
15.20 Teva Pharmaceutical
List of Figures
Figure 2-1: Orphan v/s Non-Orphan Drugs -Phase II to Launch Clinical Development
Time
Figure 2-2: Probability of Regulatory Success of Orphan v/s Non-Orphan Drugs
Figure 3-1: Global Orphan Drugs Market Value (US$ Billion), 2013-2020
Figure 3-2: US Orphan Drugs Market Value (US$ Billion), 2013-2020
Figure 3-3: Europe Orphan Drugs Market Value (US$ Billion), 2013-2020
Figure 3-4: Asia* Orphan Drugs Market Value (US$ Billion), 2013-2020
Figure 4-1: Biological & Non Biological Orphan Drug Segment (%), 2014 & 2020
Figure 4-2: Biological & Non Biological Orphan Drug Market (US$ Billion), 2013-2020
Figure 4-3: Global Orphan Drugs Market by Therapeutic Area, 2012 & 2020
Figure 4-4: Regional Markets for Orphan Drugs, 2014 & 2020
Figure 5-1: Global - Orphan Drug Pipeline by Phase (%), 2015
Figure 5-2: Global - Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 5-3: Global - No Development Reported in Orphan Drug Pipeline by Phase (%),
2015
Figure 5-4: Global - No Development Reported in Orphan Drug Pipeline by Phase
(Numbers), 2015
Figure 5-5: Global - Discontinued Orphan Drug Pipeline by Phase (%), 2015
Figure 5-6: Global - Discontinued Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 5-7: Global - Suspended Orphan Drug Pipeline by Phase (%), 2015
Figure 5-8: Global - Suspended Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 6-1: US Market for Orphan Drugs (US$ Billion), 2012-2020
Figure 6-2: US - Orphan Drug Pipeline by Phase (%), 2015
Figure 6-3: US - Orphan Drug Pipeline by Phase (Number), 2015
Figure 6-4: US - No Development Reported in Orphan Drug Pipeline by Phase (%), 2015
Figure 6-5: US - No Development Reported in Orphan Drug Pipeline by Phase (Number),
2015
Figure 6-6: US - Discontinued Orphan Drug Pipeline by Phase (%), 2015
Figure 6-7: US - Discontinued Orphan Drug Pipeline by Phase (Number), 2015
Figure 6-8: US - Suspended Orphan Drug Pipeline by Phase (%), 2015
Figure 6-9: US - Suspended Orphan Drug Pipeline by Phase (Number), 2015
Figure 7-1: European Market for Orphan Drugs (US$ Billion), 2012-2020
Figure 7-2: Europe - Orphan Drug Pipeline by Phase (%), 2015
Figure 7-3: Europe - Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 7-4: Europe - No Development Reported in Orphan Drug Pipeline by Phase (%),
2015
Figure 7-5: Europe - No Development Reported in Orphan Drug Pipeline by Phase
(Numbers), 2015
Figure 7-6: Europe - Discontinued Orphan Drug Pipeline by Phase (%), 2015
Figure 7-7: Europe - Discontinued Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 7-8: Europe - Suspended Orphan Drug Pipeline by Phase (%), 2015
Figure 7-9: Europe - Suspended Orphan Drug Pipeline by Phase (Numbers), 2015
Figure 8-1: Asia Pipeline by Phase (%), 2015
Figure 8-2: Asia Pipeline by Phase (Number), 2015
Figure 8-3: No Development Reported Asia Pipeline by Phase (%), 2015
Figure 8-4: No Development Reported Asia Pipeline by Phase (Number), 2015
Figure 8-5: Discontinued Asia Pipeline by Phase (%), 2015
Figure 8-6: Discontinued Asia Pipeline by Phase (Number), 2015
Figure 8-7: Asia - Number of Orphan Drug by Clinical Phase
Figure 11-1: Japan Orphan Drug/Medical Device Designation System
Figure 11-2: Japan Orphan Drug/Device Designation Process
Figure 15-1: AOP Orphan
Figure 15-2: Agenus Pipeline
Figure 15-3: Genethon Drug Pipeline
Figure 15-4: Genzyme Drug Pipeline
Figure 15-5: Orphan Europe Pipeline
Figure 15-6: Prosensa Drug Pipeline
Figure 15-7: Shire Drug Pipeline