This presentation will gives you a brief idea about Orphan Diseases, Orphan Drugs, the Orphan Drug Act (ODA), and Incentives and Impact of ODA

1. Ancy Jose
Biotechnology Engineering
2011-2015 Batch
SCET,Kodakara

2.  Neglected Diseases: Rare diseases and tropical diseases.
 Any disease that affects a small percentage of the population.
 About 250 new rare diseases are discovered each year.
 Medical literatures describe about 7000 rare diseases ; in that 650 have
received official designation as rare diseases (Europe)
 The known rare diseases include :
Autoimmune Diseases, Cancer Types, Communication Disorders,
Cutaneous Conditions, Endocrine Diseases, Eye Diseases And Disorders,
Genetic Disorders(80% ), Infectious Diseases, Intestinal Diseases,
Neurological Disorders.

3.  Diseases that manifest in patient populations representing at the maximum
6–8% of the world population are defined as “rare diseases” or “orphan
diseases”.

4.  Any drug is developed to treat an ‘orphan or a rare disease’ condition is
called an ‘Orphan Drug’.
 The lack of economic feasibility leads to a lack of even preliminary research
into these disorders.
 These are not developed by the pharmaceutical industry for economic
reasons but which respond to public health need.
 Developing a orphan drug is extremely challenging for any pharmaceutical
company because of :
High cost intensive R&D initiatives
Availability of a low return on investments.

6.  Determination of the human genome sequence by the
International Human Genome Project,
 Advances in genomics and proteomics, and
 A detailed understanding of cellular biochemical networks

8.  The Orphan Drug Act (ODA) was passed on January 28, 1983.
 USA was the first nation to propose a legal frame work to encourage
development and availability of orphan drugs.
 Following the same policy as the US, Singapore (1991), Japan (1993) and
Europe (2000) passed laws with aim to promote research and development in
the field of rare diseases .
 A group of pharmacologists requested the Indian government to institute
ODA at the conference held by the Indian Drug Manufacturing Association
in November 2001, but nothing concrete has materialized so far.

9. .

10.  Funding towards investigation for "orphan disease" treatment
 Tax credit for clinical research
 Waiver of fees for new drug application
 Offering more lucrative incentive than product patent (product patent
requires the drug to be novel), as the orphan designation of the
product by the US Food and Drug Administration (FDA) and
product approval by them are the only requirements for 7 year
market exclusivity of an "orphan drug" for the specified indication
 Market exclusivity of "orphan drugs" become effective from the date
of regulatory approval, unlike product patent, product development
time remains outside this period.

11.  The drugs, which are not eligible for product patent, may be eligible for market
exclusivity as an "orphan drug" by the US-FDA.
 Recommendations from FDA staff to sponsors about nonclinical and clinical
studies that would support approval of a drug for a rare disease.
 Other special assistance, such as accelerated approval or fast track or priority
review, may also be available for sponsors of orphan drugs.
 Tax-incentives accelerate and support R & D for rare disease for the discovery and
development of medicines, diagnostics, and vaccines for rare diseases, with a
suitable level of pricing for the patients, families and health insurances

12.  Since the introduction of ODA, nearly 1100 drugs and biological
products have been designated as orphan products.
 The FDA has approved over 231 of these for marketing, thereby
facilitating treatment for an estimated 11 million patients in the
USA.
 A decade after in 1993, Japan took similar initiative followed by
Australia in 1999. Currently, Singapore, South Korea, Canada, and
New Zealand are also having their country specific ODA.
 But India has still not inacted the Orphan Drug ACT

13.  Hyderabad based NATCO Pharma’s novel anti-cancer drug (NRC-AN-019)
has received “Orphan Drug Designation” from the USFDA for three
indications-
Glioma (brain tumor)
Pancreatic cancer
Chronic Myelogenous Leukemia.

14.  Troikaa pharmaceuticals Ahmedabad manufactures following
orphan drug preparations:
Tachyban (adenosine injection)
Hemaprot (aprotinin injection)
Neopam (pralidoxime chloride injection)
Narcotan(naloxone hydrochloride injection)
Cyan sos

15.  The Cipla’s Mumbai-headquartered firm is manufacturing the fixed dose
combination drug of Artesunate and Mefloquine (ASMQ FDC) under a
technology transfer agreement with Neglected Diseases initiative (DNDi).
 The combination of AS and MQ is one of five Artemisinin Combination
Therapies (ACTs)currently recommended by WHO for the treatment of
uncomplicated P.falciparum malaria, and is the first-line treatment in a
number of South East Asian countries.

16.  In India, to increase awareness for the rare diseases, Rare Diseases Day was
observed in New Delhi on February 28, 2010.
 Subsequently 2nd and the 3rd‘Rare Disease Days’ were observed in Chennai
and Mumbai in 2011 and 2012, respectively.
 In 2014, Lysosomal Storage Disorders Support Society observed Rare
Diseases Day in Bangalore, New Delhi, Mumbai, Chennai, Jaipur, & Kolkata

17.  400 US FDA approved orphan drugs and about 80 EMA approved orphan
drugs are available in India and world-wide.
 Most of them are either not accessible to most patients in India or are
unaffordable.
 Still effective research is not widespread in india.
 ORDI aims to work between the Government of India and the
Pharma/Biotech/Diagnostic industry to enact an Orphan Drugs Act.
 The National Institute of Pharmaceutical Education and Research is getting
into research on ‘orphan drugs’ to solve the rare diseases problem in India

20.  High prices of “orphan drugs”
 Small patient population
 Delay in diagnosis & Deficient diagnostic systems
 Limited treatment availability
 Lack of knowledge & training
 Disease is poorly understood
 Lack of adequate expertise and review by authorities
 Limited public awareness