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(Relevance: Health and government policies regarding it form an important component of the UPSC syllabus. In 2020, UPSC asked questions on geriatric and maternal healthcare. Similarly, in 2018, a question was asked on local community-level healthcare intervention to achieve ‘Health for all.’)

On October 4, the Delhi High Court directed that the Centre establish a National Fund for Rare Diseases and allocate Rs 974 crore for 2024-25 and 2025-26. This was to be approved and transferred in 30 days. It also said “treatment for all eligible… patients, as per an AIIMS report dated July 21, who are before the court in these batches of petitions shall commence in 45 days as per the NRDC (National Rare Diseases Committee) recommendations.”

1. The World Health Organisation (WHO) has defined rare diseases as a debilitating, lifelong condition that affects 1 or fewer people in 1,000. Around 6% to 8% of the population is estimated to have a rare disease, meaning 8.4 crore to 10 crore Indians are living with these conditions for which treatments either do not exist or therapies are extremely expensive. Some common rare diseases are Haemophilia, Pompe disease, Thalassemia, Sickle-cell Anaemia, and Gaucher’s disease.

2. The Delhi High Court said that the National Rare Diseases Committee (NRDC), which was constituted on May 15, 2023, shall continue to function for a further period of five years with the ICMR director general as chairperson of the committee. The Court also directed the government to expand the existing number of centres of excellence (COEs) “considering patient density”. As of 2023, there were 11 COEs across the country.

3. Currently, 63 rare diseases are included under the National Policy for Rare Diseases, which was recommended by the Central Technical Committee for Rare Diseases (CTCRD).

4. Therapies are available for less than 5% of rare diseases, leading to less than 1 in 10 patients receiving disease-specific care. Existing treatments are often very expensive. While the Centre provides financial assistance to various CoEs for treatment, stakeholders have gone to court to highlight challenges in accessing funds.

5. In India, rare diseases are categorised into three groups based on the nature and complexity of available treatment options.

→ Group 1 includes diseases that can be treated with a one-time curative procedure.

→ Group 2 diseases require long-term or lifelong treatment which are relatively less costly and have shown documented benefits, but patients need regular check-ups.

→ Group 3 diseases are those for which effective treatments are available, but they are expensive and must often continue lifelong. There are challenges in selecting the right beneficiaries for these treatments.

6. Many medicines and therapies for rare diseases are patented, which makes them very expensive. The market for these drugs is small and the development costs are high, so pharmaceutical companies often don’t find it profitable to produce them, pushing up prices. Therefore, these drugs are called ‘orphan drugs’

1. In 2021, the National Policy for Rare Diseases (NPRD) was launched. Under this policy, financial assistance up to Rs 50 lakh is provided to patients receiving treatment at an identified CoE. The CoEs include AIIMS in Delhi, PGIMER in Chandigarh, and the Institute of Postgraduate Medical Education and Research at Kolkata’s SSKM Hospital.

2. The Health Ministry has opened a Digital Portal for Crowdfunding & Voluntary Donations with information about patients and their rare diseases, the estimated cost of treatment, and bank details of the CoEs. Donors can choose the CoE and patient treatments they wish to support. Each CoE also has its own Rare Disease Fund, which is used with approval from its governing authority.

3. In August 2024, the government told Parliament that financial assistance of Rs 24 crore had been released to CoEs for treating rare disease patients until August in the current financial year.

4. The import of rare disease medicines by patients does not attract customs duty, but companies that bring these drugs to India still pay 11% customs duty and 12% GST.

5. On January 3, 2019, the Department of Pharmaceuticals under the Union Ministry of Chemicals and Fertilisers issued an order freeing orphan drugs from price controls. The High Court expressed concern over this exemption, saying “the position cannot continue in this manner”.

Rare Disease Day

(Source: ‘Don’t stop or interrupt therapy’, Issues in the treatment of ‘rare diseases, Delhi HC directs Centre)

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