The Delhi High Court on January 28, 2021 directed the Centre to finalise the National Health Policy for Rare Diseases of 2020 by March 31 and make operational provision of crowdfunding envisaged under the law for treatment of high-cost rare diseases.
About Rare Disease?
- Rare diseases are serious chronic diseases, and may be life-threatening.
- In the EU, a disease is considered to be rare when the number of people affected is less than 5 per 10 000.
- There are between 5 000 and 8 000 rare diseases, most of them with a
- genetic basis.
- A very rough estimate would be that one out of 15 persons worldwide could be affected by a rare (“orphan”) disease – 400 million people worldwide, of whom 30 million are in Europe and 25 million in the United States.
- In the United States, a rare disease is defined as a condition that affects fewer than 200,000 people in the US. This definition was created by Congress in the Orphan Drug Act of 1983.
- Rare diseases became known as orphan diseases because drug companies were not interested in adopting them to develop treatments.
- The Orphan Drug Act created financial incentives to encourage companies to develop new drugs for rare diseases.
- The rare disease definition was needed to establish which conditions would qualify for the new incentive programs.
- There are many different causes of rare diseases. The majority are thought to be genetic, directly caused by changes in genes or chromosomes.
- Indian Council of Medical Research has launched ‘Indian Rare Disease Registry’ for certain rare diseases, including Thalassemia and Sickle Cell Anemia.
- An estimated number of 1,20,000 to 1,50,000 patients are affected by Sickle Cell Disease in India with 20-35% carrier rate in tribals and other backward communities.
- The estimated number of beta Thalassemia cases is 1,00,000 to 1,20,000 with a carrier rate of 3-4% in overall population.
(Sources: The Hindu, WHO, US NIH)
