Context: Recently, World Sickle Cell Day 2020 was observed in India to increase awareness about the Sickle Cell Disease (SCD) at the national level.
Key Points

• World Sickle Cell Day — recognised by the United Nations and celebrated every year on June 19.
• The United Nations General Assembly adopted a resolution recognising sickle cell disease as a public health problem on December 22, 2008.

About Sickle Cell Disease

• Sickle cell disease is a group of disorders that affects haemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body.
• People with this disorder have typical haemoglobin molecules called haemoglobin S, which can distort red blood cells into a sickle, or crescent shape.
• Signs and symptoms of sickle cell disease usually begin in early childhood. Characteristic features of this disorder include a low number of red blood cells (anaemia), repeated infections, and periodic episodes of pain. The severity of symptoms varies from person to person. Some people have mild symptoms, while others are frequently hospitalised for more serious complications.

Sign and Symptoms of ‘Sickle Cell disease’

• The signs and symptoms of sickle cell disease are caused by the sickling of red blood cells. When red blood cells sickle, they break down prematurely, which can lead to anaemia.
• Anaemia can cause shortness of breath, fatigue, and delayed growth and development in children.
• The rapid breakdown of red blood cells may also cause yellowing of the eyes and skin, which are signs of jaundice.
• Painful episodes can occur when sickled red blood cells, which are stiff and inflexible, get stuck in small blood vessels. These episodes deprive tissues and organs of oxygen-rich blood and can lead to organ damage, especially in the lungs, kidneys, spleen, and brain.
• A particularly serious complication of sickle cell disease is high blood pressure in the blood vessels that supply the lungs (pulmonary hypertension). Pulmonary hypertension occurs in about one-third of adults with sickle cell disease and can lead to heart failure.

India's Policy for treatment of rare Disease

• In India, Haemophilia, Thalassemia, Sickle cell anaemia and Primary Immuno Deficiency in children, auto-immune diseases, Lysosomal storage disorders such as Pompe disease and Gaucher disease are in the rare diseases list. 
• According to Health Ministry, about 95 per cent rare diseases have no approved treatment. "Less than one in 10 patients receives disease-specific treatment," the policy states. 
• The Centre first prepared such a policy in 2017, but appointed a committee in 2018 to review it. 
• Presently, very few pharmaceutical companies manufacture drugs to treat rare diseases and India has no domestic manufacturer:

About the National Policy

• The latest policy creates three categories of rare diseases requiring one-time curative treatment, diseases which need long-term treatment but the cost is low, and diseases that require life-long treatment and the cost is high.
• The policy states that the Centre will provide assistance of Rs I5 lakh to patients suffering from rare diseases that require one-time curative treatment under the Rashtriya Arogya Nidhi scheme. The treatment is limited to beneficiaries of Pradhan Mantri Jan Arogya Yojana. Some diseases in this category are osteopetrosis, immune deficiency disorders and Lysosomal Storage Disorders. 
• The policy has recommended that state governments support patients belonging to the second category of diseases that include Phenylketonuria and Galactosemia, among others.
• It also recommends crowdfunding as a source to fund treatment of rare diseases and advises hospitals to report such cases on digital platforms to gather funds.
• The policy also intends to kick-start a registry of rare diseases that the Indian Council of Medical Research will maintain.