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.

1. 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 anomali 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 stem-cells 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
in clinical trials resulting in dominance of big pharmaceutical companies in this
segment. However, few small/mid-sized orphan drug developers could also be
found in this segment which is developing autoimmune and cancer
therapeutics.
In recent years, several new rare diseases are being included in clinical trials
for the development of orphan drug candidate. Oncology, Genetic diseases,
Autoimmune, pulmonary disease, Central nervous system and others are main
focus of orphan drug developers. All of these diseases have large patient base
but their rare forms doesn’t have suitable therapeutics which reflects the
marketing potential of orphan drugs in these segments. Out of these, oncology

2. segment has got maximum attention followed by rare genetic disorders,
neurological disorder and autoimmune disorders. In a bigger view, orphan drug
segment is expected to grow several folds in coming years due to several
favorable 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:
• Global Orphan Drug Market Overview
• Global Orphan Drug Market Segment Analysis
• FDA & EMA Regulation for Clinical Trials Orphan Designated Drugs
• Orphan Drug Designation Criteria & Reimbursement Policy by Region
• Comprehensive Insight on Global Orphan Drugs Clinical Pipeline & Patent
Analysis by Company, Country, Indication & Phase
• Global Orphan Drugs Clinical Pipeline: 697 Drugs
• Majority Orphan Drugs in Phase-II Trials: 249 Drugs
• Marketed Orphan Drugs: 274 Drugs