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

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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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http://www.orpha.net/consor/cgi-bin/Disease_Search.php?lng=EN

http://www.orpha.net/consor/cgi-bin/Disease_Classif.php?lng=EN

http://www.orpha.net/consor/cgi-bin/Disease_ProEncyclo.php?lng=EN

http://www.orpha.net/consor/cgi-bin/Drugs.php?lng=EN

http://www.orpha.net/consor/cgi-bin/Clinics.php?lng=EN

http://www.orpha.net/consor/cgi-bin/ClinicalLabs.php?lng=EN

http://www.orpha.net/consor/cgi-bin/ResearchTrials.php?lng=EN

http://www.orpha.net/consor/cgi-bin/ResearchTrials_ClinicalTrials.php?lng=EN

http://www.orpha.net/consor/cgi-bin/ResearchTrials_RegistriesMaterials.php?lng=EN

http://www.orpha.net/consor/cgi-bin/SupportGroup.php?lng=EN

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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http://www.orpha.net/consor/cgi-bin/Disease_DiagnosisAssistance.php?lng=EN

http://www.orpha.net/consor/cgi-bin/Disease_Emergency.php?lng=EN

http://www.orpha.net/actor/cgi-bin/latest-newsletter-europe.htm

http://www.orpha.net/consor/cgi-bin/Education_Home.php?lng=EN

Thank You…


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Orphan drugs presentation

Health & Medicine

Transcript of Orphan drugs presentation