Recently it was reported that the applications of most patients suffering from a rare disease called Lysosomal Storage Disorder have been pending with the Union Ministry of Health and Family Welfare for the last several months awaiting financial support for treatment.
Rare disease main point:
- More than 2,000 children are infected with rare diseases across the country. Many of them require Enzyme Replacement Therapy-ERT.
- ERT is a medical treatment. Through this, the enzymes present in the body are displaced in small quantities.
- There has also been a demand for reform in the National Policy for Treatment of Rare Diseases, 2017 for the treatment of these rare diseases.
Lysosomal storage disorder:
- Lysosomal storage disorder is a genetic metabolic disease. The lack of enzymes in it results in abnormal buildup of various toxins in the body cells.
- It can affect various parts of the body including the skeleton, brain, skin, heart, and central nervous system.
- There are currently no approved treatments available for many lysosomal storage diseases but they can be controlled to some extent by ERT.
- Rare disease is a health condition that is less common in people or very few people are affected by these diseases than normal diseases.
- There is no universally accepted definition of rare diseases and it has different definitions in different countries.
- Although rare diseases occur in fewer people, they collectively affect a much larger proportion of the population.
- 80 percent of rare diseases are basically genetic, so it has the opposite effect on children.
- 56–72 million people in India are affected by rare diseases.
National Policy for the Treatment of Rare Diseases, 2017:
- This policy highlights the efforts made by the government to deal with rare diseases.
Under this, an idea has been set to constitute an inter-ministerial consultation committee to coordinate and control the initiatives of various ministries and departments for rare diseases.
- It also mentions the creation of a financial fund at the center and state level for the treatment of rare diseases.
- Under this policy, the Patient Registry will be arranged in the Indian Council of Medical Research-ICMR to promote research related to rare diseases.
- Because the treatment of rare diseases requires high cost. Therefore, with the access to treatment, an attempt has been made to make the health system affordable through this policy.
- Its purpose is to make doctors, family members of the patient and common people aware of rare diseases.
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