Orphan Drugs Legislation- comparison of Indian and global scenario

Orphan Drugs Legislation- comparison of Indian and global scenario


What are Orphan Drugs?

Drugs used to treat rare medical conditions are called Orphan Drugs like narcolepsy, multiple sclerosis, and fibrosis. These drugs derived the name as Orphan Drugs because pharmaceutical companies do not put enough efforts to research and develop medicines which are not profitable. The sale of medication would not recover the amount of money invested in developing these drugs.

The rare diseases which are treated by Orphan drugs can be referred to as Orphan Diseases. The government of a nation decides whether to grant a disease an Orphan disease and work for the development of a drug to treat it. 

It is only due to the government’s efforts of different nations; several breakthroughs have been achieved, which would not have been possible otherwise due to economic profit factors.



Law related to Orphan Drugs around the world

In 1980s Orphan Diseases in the USA gained attention for the first time. The case of a young boy with Tourette’s Syndrome was able to gain considerable attention to orphan disease patients’ condition. On January 28, 1983, the USA passed a set of laws devoted to orphan diseases and orphan drugs in the form of the Orphan Drugs Act (ODA). The key goal of the act was to support orphan disease development and research of orphan drugs. Following this, several other countries took inspiration and drafted their own laws as per requirements.


Legislation in the European Union

In 1990s European Union showed interest in the development of laws related to orphan diseases. And in the year 2000, the European Union enacted similar legislation, Regulation (EC) No 141/2000(Orphan Drug Regulation’ ODR’). The act has undergone only one amendment in 2009, which says that an orphan drugs will be stripped of its orphan status in 5 years if it is proved to be extraordinarily profitable to the pharmaceutical companies. 

The definition of Orphan Drugs used by the European Union is more comprehensive than the USA as it includes Tropical diseases generally found in the developing countries. Orphan Status is granted to a drug if it is invented to treat a disease which affects not more than 5 in 10,000 people in the European Community. Committee on Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) regulates the legislation related to orphan drugs and diseases.


Legislation in Japan

On October 1, 1993, Japan framed its own set of orphan drug regulations adopting a few unique provisions to encourage Research and Development of Orphan Drugs. 

The new rules say that the status of orphan drugs can be given only to those that meet the 2 requirements mentioned below:

(1) Number of patients The number of patients who may use the drug or medical device should be less than 50000 in Japan. 

(2) Medical needs The drugs or medical devices should be indicated for the treatment of serious diseases, including difficult-to-treat diseases. In addition, they must be drugs or medical devices for which there are high medical needs satisfying one of the following criteria.


Legislation in Singapore and Australia

Singapore in 1991 gave the definition to Orphan Drugs and had set up a legal framework which regulates the import of orphan drugs in Singapore. Australia adopted the orphan drug legislation in 1997 with the assistance of the FDA and USA. The legislative framework is mostly based on on the US model and legislations.


Legislation in India

Unfortunately, the knowledge of Orphan and treatment has not flowed into the Indian population’s consciousness. So far, there have been more than 450 rare diseases that have been found in India. According to the census of 2011, there were about 72,611,605 people in India affected by rare diseases. India has a very high population of people affected by rare diseases compared to the world average, and there is no initiative by the government. 

The large population suffering from orphan diseases in India poses a great opportunity for pharmaceutical companies to grow their businesses. However, the absence of legislation and the mindset of concerned authority deters the pharmaceutical industry from showing any interest.