Marlene Haffner, MD; MPH

Drug Programs for Rare

Diseases: Is Right now the

time for India?

Marlene E. Haffner MD, MPH, CEO Haffner Associates, LLC Rare Diseases and Orphan Drug Development Initiatives in India September 18, 2014

What are Orphan Drug Programs?

Why Do They Exist?

 Orphan Drugs

 Drugs that are not commercially pursued or adopted due

to lack of patient pool; return on ROI

 They exist to promote drug development in areas where

drug development is not economically feasible without incentives

Where Do They Exist?

My Involvement

 how you were instrumental in assisting other countries

Why an Orphan Drug Act?

 India houses 16% of the world population, 21% of the

global diseases, and the largest burden of communicable diseases in the world

 What about “neglected diseases?”  Malaria,  Tuberculosis  Dengue Fever  Leishmaniasis  Sleeping sickness  Helminths

What is the public health impact

of Rare/Neglected Diseases?

 Are these diseases the greatest public health issue facing

India?

 What are India’s greatest public health issues?

 Access to health care

 Improve public funded health facilities

 Infant death rate  Poor nutrition  Communicable diseases  Malaria  TB  HIV  Dengue Fever  Leishmaniasis

 Many diseases that are common in India are Rare in Western

What is happening in the

Pharmaceutical area in India

 India’s pharma exports stood at US$ 15.04 bn during

2013-14

 Huge growth opportunities ~15-20%/ year  Mainly Generic Drugs

 Industry has heavy utilization (about 55%) by US and

other Western Nations.

 Carrying out clinical trials for western nations  Poised to do greater things for India and beyond

Pharmaceutical Industry in

India

 Product patent protection abolishment – 1972

 Reintroduction of Product patent protection -2005

 Higher prices/TRIP Compliance vs. Growth/Accessibility  Partnerships between Major National Companies (MNC’s)

and Indian pharmaceuticals

 Aurobindo-Pfizer , Dr Reddy,-GSK, etc.

 Indian companies agents for imported formulations  Price Control

Let’s Develop a New

Paradigm

 Rare AND Neglected Diseases

 Incentivize drug companies to develop drugs for

patients with both neglected diseases and orphan diseases

 Most neglected diseases products would be purchased by

governments and government agencies

 USAID, Department of Health of India, WHO

 MUST be affordable

 Economies of scale can be reached via large patient pool  E.g. Tuberculosis

Let’s Develop a New

Paradigm

 For Rare Diseases we need a paradigm which fits India

and Indian Culture

 What is a Rare Disease in India

 Should be a ratio so that it can accommodate increasing and decreasing population

 EU uses 5/10,000. EU population ~ 470 million(1/3 that of India)

 US has finite number - <200,000 in the population (~1:1350)  Concept was for the # to be a surrogate for profitability

 If one uses a ratio of 1 per 2,000 in the population ~ 625,000 (too large a number)

Let’s Develop a New

Paradigm

 Which are the WHO listed Neglected Diseases? Could

India serve these diseases even if not seen in India?

• Buruli Ulcer • Leishmaniasis • Podoconiosis

• Taeniasis • Leprosy • Snakebite

• Dengue • Lymphatic filariasis • Strongyloidiasis • Echinococcosis • Trachoma • Rabies

• Foodborn Trematode Infections • Soil Transmitted helminthiases • *Schistosomiasis

Successful Eradication

 Which One’s

 Dracunculiasis – since 1999  Yaws –since 2006

 a group of chronic bacterial infections that cause skin lesions and

disability

 How

 Dracunculiasis – water treatment and education  Yaws – Penicillin

 Who was involved

 Dracunculiasis – WHO, UNICEF, NGO’s, local communities, state

and local governments

 Yaws - WHO, UNICEF, National Institute of Communicable

Diseases (NICD), Delhi

 Cost

 Low cost as penicillin is cheap and has high availability

The Real Issue With Health

Care in India?

 Much of the issues in India are access and affordability of

therapy of any kind

1. Are there centers of excellence for diagnosis and treatment? 2. 70% Indian population pays out of pocket for health care 3. Health Care expenditure is a VERY small % of GDP

Existing Incentives – in the

US, EU and beyond

 Market Exclusivity

 Tax Credits for Clinical Development

 Grants for development of eligible products  Filing fee waiver for review of new products  Protocol Assistance

 Faster Approvals (Happen Stance) because these

Thoughts on what Incentives

Might work/look like for India?

 Marketing Exclusivity only if IP is strong

 Entrance into new markets

 Tax Relief ?

 Government Subsidies?

 International Support

 Social/Socio-economic Value

 Further development of pharmaceutical Industry  Specific Regulation ?

 Minimum alternate tax /Modified value added tax

 Orphan Drugs should be AS SAFE and AS EFFECTIVE as

Nonetheless- Areas where rare

diseases could be affordable

 Certainly excellent scientists and physicians

 Great need in a country of 1.2 billion to treat patients

with every disease

 numbers are proportionately large

 Costs money to care for individuals with every disease  especially chronic diseases

 India has a burgeoning middle class – the country is

growing very positively

Marlene E. Haffner, MD, MPH

11616 Danville Drive Rockville, Maryland 20852 mhaffner.com mhaffner3@verizon.net 301 984 5729 - office 301 641 4268 - cell 301 984 2272 - FAX

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