Orphan drugs presentation

Orphan Drugs
Presenter:- Dr Atul Rajpara
Post Graduate Resident,
Dept. of Pharmacology,
Baroda Medical College.
26-Aug-15Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.

Outline
 Definition
 Rare Diseases
 Essential Vs Orphan
 Orphan Drug Act’1983
 Examples of drugs and Manufacturers
 Orphan drug designation process
 Indian Perspective
 References
26-Aug-15
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Venue: Department of Clinical Pharmacology, KEM Hospial, Mumbai.

Orphan Drug Definition
 “The Orphan Drug Designation program provides
orphan status to drugs and biologics which are
defined as those intended for the safe and effective
treatment, diagnosis or prevention of rare
diseases/disorders.”
 The so-called 'orphan drugs' are intended to treat
diseases so rare that sponsors are reluctant to develop
them under usual marketing condition.
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Rare Diseases
 A rare disease occurs infrequently in a population, but
there is no universal definition.
 3 elements to the definition as used in various
countries are as follows:
1. The total number of people having the disease
2. Its prevalence
3. Non-availability of treatment for the disorder
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Country Total population
affected, less than
1 per
Prevalence per
10000 of
population
USA 200,000 7.5
JAPAN 50,000 4
SOUTH KOREA 20,000 4
AUSTRALIA 2,000 1.1
TAIWAN 10,000 1
EUROPE - 5
China 500,000 -
India  
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 India, like many developing countries, currently has
no standard definition. Considering the large
population of India, ORDI (Organisation for Rare
Diseases in India) suggests a disease to be defined as
rare if it affects 1 in 5,000 people or less.
 The World Health Organization (W.H.O.) has suggested
that a rare disease should be defined as one with
frequency less than 6.5 – 10 per 10,000 people.
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Rare Disease Facts
 There are more than 7000+ Rare Diseases
known/reported world-wide.
 Over 350 Million people world-wide are affected ~30
Million in USA, ~30 Million in EU, ~70 Million in India.
 About 80% of RDs are genetic in origin many of them
being monogenic.
 50% of RDs are onset at birth and the rest are late
onset.
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 Only ~500 approved orphan drugs exist in the market
and over one hundred in clinical trials.
 Majority of the diseases have no treatment and when
they exist, are mostly unaffordable
 Early diagnosis is a critical challenge in RD
management (Avg. time: 5-7 years)
 Affordability
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Rare Diseases are...
 Inherited cancers,
 Autoimmune disorders,
 Congenital malformations,
 Infectious diseases and
 Others like,
Haemangiomas, Hirsch sprung Disease, Gaucher disease,
Cystic Fibrosis, Muscular Dystrophies and Lysosomal
Storage Disorders.
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Systemic Prevalence
Represents orphan drug designations through 2006
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Essential Vs Orphan drugs
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Orphan Drug Act’1983
 USA became the first country to enact Orphan Drug
Act on January 4th,1983.
 The law is designed to facilitate the development and
commercialization of drugs to treat rare diseases,
The salient features of the act are as follows:
i. Incentives to Pharma companies investing in
Orphan Drugs R&D
ii. Faster/Priority clearance of Orphan Drugs
Application through FDA
iii. Market exclusivity for 7 years for Pharma
companies to recover costs
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Impact Of The Acts
 Marketed Orphan Designated Drugs: 281
 Orphan Designated Drugs in Clinical Phase: 400
 Highest Number of Drugs in Phase-2 Trial: 231
 US Dominates Clinical Trial Process: 350 in Pipeline
(Research till Registration)
 Key Market: US (Sales > US$ 40 Billion)
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Companies involved in the
manufacture of orphan drugs
 Pfizer
 GlaxoSmithKline
 Novartis
 Sanofi Aventis
 Johnson and Johnson
 Bayer
 Orphan drug specialists
- Genzyme
- Actelion
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Few examples of Orphan
drugs and it’s market players
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Orphan Drug Designation
Process
 Sponsors have to first send request to the Office of
Orphan Products Development (OOPD) to grant
orphan designation to their drug or biological product
to take advantage of financial incentives available for
further product development.
 And then send NDA (New Drug Application) to the
Centre for Drug Evaluation and Research (CDER) or
the Centre for Biologics Evaluation and Research
(CBER) to market their orphan drug or biological
product.
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 No IND is required
SUBMISSION
OF NDA
Pre-Clinical Development Clinical Development
CAN SUBMIT DESIGNATION
REQUEST
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After Designation Request Is
Submitted…
 Typical review cycle ~ 90 days (often less)
 Will either receive:
-Designation Letter OR
-Deficiency Letter
 Once designated, sponsor is required to submit annual
reports until drug is approved
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Review of a Designation
Request involves…
1. What is the disease/condition?
2. Is the disease rare (prevalence)?
3. Is there sufficient scientific rationale that demonstrates
“promise” that the drug/biologic will treat, diagnose or
prevent the disease/condition at issue?
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Indian Perspective
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Rare disease treatments
currently available in India
 In India, about 6000-8000 rare diseases, mostly genetic
in nature.
 In theory, close to 400 US FDA approved orphan drugs
and about 80 EMA approved orphan drugs are
available in India and world-wide.
 However, in practice, most of them are either not
accessible to most patients in India or are
unaffordable.
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Financial incentives available to
orphan drug developers in India
 Currently, drug developers in India are receiving
no formal incentives from the Government and hence
they are more focused on
o developing affordable drugs for more common
diseases such as oral insulin,
o statins for preventing or slowing the progression of
cardiovascular disease,
o vaccines and antibiotics for a number of preventable
infectious diseases, etc.
 Hence, patients with rare diseases in India have to rely
on imported drugs from western countries which
makes these treatments (even when available)
unaffordable.
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Venue: Department of Clinical Pharmacology, KEM Hospital, Mumbai.

Financial assistance to rare
disease patients in India
 The healthcare system in India is mostly self-funded by
the patients.
 This restricts the affordability threshold for drugs and
diagnostic tests to around 25 thousand rupees (~400-
500 USD) per instance or about a lac rupee (~1600-
2000 USD) per year on recurring expenses for most
patients.
 The costs of most available orphan drugs are
significantly higher than this affordability range
requiring the need for financial assistance.
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 Patients look for patient foundations, other non-
governmental organizations, and charitable access
programs offered by certain Pharma companies to
cover these costs.
 Examples of such initiations are:
o Birth Defects Registry of India(BDRI)
o Lysosomal Storage Disorders Support Society
o Metabolic Errors and Rare Diseases organization
o Association for Social and Health Advancement
o Haemophilia Foundation
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o Sjogren’s India
o Pompe Foundation
o Muscular Dystrophy Foundation
o Alzheimer and Related Disorders Society Of India
o Rett Syndrome Foundation
o Association of Persons with Rare Eye Diseases
o Amrithavarshini
o ABLE (Association of Biotechnology Led Enterprises)
o Mumbai Marathon
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Biggest hurdles to improving
patient access to orphan drugs
in India
 Awareness among doctors, patients and other
stakeholders of rare diseases, relevant clinical trials
and orphan drugs.
 Affordability and Accessibility (easy import and
distribution) of orphan drugs in India.
 Enactment of the Orphan Drug Act by the
Government of India. Without assurance of marketing
exclusivity, IP rights and other financial incentives, the
orphan drugs industry is unlikely to succeed in India.
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Conclusion
 The new approved orphan drugs should be easily
accessible and affordable to patients with rare
diseases in India.
 Government of India and the other
Pharma/Biotech/Diagnostic industry should work
together to enact an Orphan Drugs Act (ODA) and
that will create incentives for orphan drug developers.
 Government needs to create a framework that is
conducive to enabling the manufacturing of orphan
drugs in India
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References:
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 Launched on Feb. 18, 2014.
 Vision:
ORDI aim to empower rare disease patients and their families
in India with access to national and international resources
to improve their quality of life.
 Mission:
ORDI strive to catalyse the rapid development and delivery
of affordable diagnostics and treatments for rare diseases
through innovative collaborations and partnerships among
stakeholders to benefit rare disease patients in India.
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 Orphanet is the reference portal for information on
rare diseases and orphan drugs, for all audiences.
 Orphanet’s aim is to help improve the diagnosis, care
and treatment of patients with rare diseases.
Rare diseases are rare, but
rare disease patients are numerous
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Orphanet services
 An inventory of rare diseases and a classification of
diseases elaborated using existing published expert
classifications.
 An encyclopaedia of rare diseases in English and French,
progressively translated into the other languages of the
website.
 An inventory of orphan drugs at all stages of
development.
 A directory of expert resources, providing information
on expert clinics, medical laboratories, ongoing research
projects, clinical trials, registries, networks, technological
platforms and patient organisations, in the field of rare
diseases, in each of the countries in Orphanet’s
consortium.
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 An assistance-to-diagnosis tool allowing users to
search by signs and symptoms.
 An encyclopaedia of recommendations
and guidelines for emergency medical care and
anaesthesia.
 A fortnightly newsletter, OrphaNews, which gives an
overview of scientific and political current affairs in the
field of rare diseases and orphan drugs, in English and
French.
 A collection of thematic reports, the Orphanet Reports
Series, focusing on overarching themes, directly
downloadable from the website.
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Thank You…
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Orphan drugs presentation

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