Max Neeman shares India's potential impact on clinical studies for orphan drugs

1. Orphan Disease
Scenario in India

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India Orphan Drug Scenario
Rich & Naive Patient Population
India’s population has a significant prevalence of orphan diseases estimated to be around 72,611,605 (more than the US and EU orphan disease populations combined). This makes India a highly desirable region for research.
While premier institutions support the naïve population by establishing diagnosis techniques and providing screening tools to needy people, further involvement of state government is needed to address unmet need of this.
Key opinion leaders in this area were approached for discussion and to identify challenges of bringing awareness to the untapped patient pool of orphan conditions in India.

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Orphan Disease Definition
An orphan disease occurs infrequently in a population and there is no universal definition. Three elements to the definition as used in various countries
the total number of persons having the disease
its prevalence
non-availability of treatment for the disorder
India, like many developing countries, currently has no standard definition. Considering the large population of India a disease to be defined as rare if it affects 1 in 5,000 people or less.
India Orphan Drug Scenario

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Orphan Disease Facts
There are >7000+ Rare Diseases known/reported world-wide
The RARE GENES project maintains a comprehensive directory of all RDs
Over 350 Million people world-wide are affected [~30 Million in USA, ~30 Million in EU, ~70 Million in India]
1 in 20 Indians is affected by one of these RDs
80% of RDs are genetic in origin, many of them are monogenic
50% of RDs are onset at birth and the other half are late onset
RDs include inherited cancers, autoimmune diseases, congenital malformations, and infectious diseases + others
Hemangiomas, Hirschsprung disease, Gaucher disease, cystic fibrosis, muscular dystrophies and Lysosomal Storage Disorders
Being highly populated and having a significant prevalence of orphan diseases, India is focusing more on research and development in this area.
India Orphan Drug Scenario

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QHow is the availability of diagnostic centers / facilities for orphan diseases in India?
AMany premier medical Institutions have a genetics department supported by state-of-the-art equipment and offer diagnostic facilities for most of the orphan disorders for more than a decade now.
KOL Orphan Drug Q&A

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QWhat is the opinion of international fraternity of research publications by Indian physicians?
AThe impression many publishers make in global conferences is that Indian physicians particularly pediatricians have extensive knowledge and expertise on diagnosis of orphan diseases. Institutional approach supported by states health and multi-specialty approach is required to reduce morbidity and improve quality of life of patients with orphan disease. There is a lot can be done to this group of subjects in partnership with patients groups and medical experts.
KOL Orphan Drug Q&A

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QWho do the patients & families approach for management of orphan diseases?
AThe associations on orphan diseases backed by academic Institutions are working on educating and encouragement. These associations have done significant work in bringing attention of media and state health department on need of fund allotment and developing expertise.
KOL Orphan Drug Q&A

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To Learn More
Contact Information: Ann Vawter Director, Business & Marketing Max Neeman International 117 Edinburgh South Dr., Ste 105 Cary, NC 27511 P: 919.424.3332 / F: 919.852.5574 E: ann.vawter@neeman-medical.com www.neeman-medical.com