- Prelims: Current events of national importance, Rare diseases, dermatomyositis, Gaucher’s disease, WHO, National Policy for Rare Diseases (NPRD) etc
- Mains GS Paper I & II: Development and management of social sectors/services related to Health and education etc
ARTICLE HIGHLIGHTS
- The tragic death of 19-year-old child actress Suhani Bhatnagar from dermatomyositis a rare disease.
- According to the World Health Organization, rare diseases afflict 1 or less per 1,000
INSIGHTS ON THE ISSUE
Context
Rare Diseases:
- It is defined as a health condition of low prevalence that affects a small number of people when compared with other prevalent diseases in the general population.
- Many cases of rare diseases may be serious, chronic and life-threatening.
- Barely 5% of the over 7,000 known diseases worldwide are treatable.
Dermatomyositis
● This is a rare type of autoimmune disease.
● This disease affects both muscles and skin.
● Its symptoms:
○ Muscle swelling
○ Red rashes on the skin
○ Physical weakness and fatigue
● It is seen more in two main age groups-
○ In children, whose age is between 5 to 15 years
○ In adults, aged between 40 and 60 years
● This disease is seen more in women than in men.
Rare diseases in India
● India accounts for one-third of the global rare disease incidence, with over 450 identified diseases.
● These range from Spinal Muscular Atrophy and Gaucher’s disease to Mucopolysaccharidosis type 1 and Whipple’s disease.
● A rough estimate suggests that about 8 crore-10 crore Indians suffer from one rare disease or another;
○ over 75% are children.
Issues associated:
● These diseases are largely overlooked.
● Resource constraints
● India languishes near the bottom on awareness, diagnosis, and drug development for rare diseases.
● The Ministry of Health and Family Welfare formulated a national policy to treat rare diseases in 2017 but withdrew it in 2018 owing to
○ “implementation challenges” and confusion regarding disease coverage
○ patient eligibility
○ cost-sharing.
○ A revised policy, the National Policy for Rare Diseases (NPRD), was announced in 2021.
● The policy attributes to a lack of sufficient data, as if regular data collection and epidemiological assessments are not the government’s job.
- Timely and accurate diagnosis is indispensable for the robust management of any disease
- For rare disease patients, it takes an average of seven years for their conditions to be diagnosed (if at all).
- Physicians are generally unaware of how to interpret the signs and symptoms
- healthcare professionals must be trained to improve their diagnostic accuracy.
- Expectant mothers with a history of rare diseases in their family must undergo mandatory prenatal screening and postnatal diagnosis and care.
- Less than 50% of the 450-odd rare diseases identified in India are treatable.
- Treatments approved by the Drugs Controller General of India are available for just about 20 rare diseases and can be availed only from Centres of Excellence (CoEs).
- CoEs are few (12), unevenly distributed, and uncoordinated
- late diagnosis
- inadequate therapies
- Lack of timely availability are the norm.
Challenges:
- The Budget’s allocation for rare diseases, although increasing over the years, remains low at ₹93 crore for 2023-2024.
- Under the NPRD guidelines, up to ₹50 lakh is allowed per patient, which will be disbursed to the concerned CoE.
- As chronic rare diseases usually require lifelong management and therapy, this amount is woefully inadequate.
- The CoEs are wary of beginning any treatment that they may need to suspend later, leaving them vulnerable to judicial action from patients and their kin.
- Confusion in the fund utilization: More than ₹47 crore of the ₹71 crore financial assistance allocated to the 11 CoEs for the current year remains unused.
- There is no parity between CoEs
- Mumbai exhausted all its funds (while treating only 20 of 107 patients
- Delhi utilizing less than 20%.
- NPRD has urged the CoEs to crowdfund to treat rare disease patients.
- A portal with over 1,400 registered patients has collected less than ₹3 lakh in three years.
Way Forward
- It is imperative for the Central government to frame a standard definition of rare diseases, increase budgetary outlays, dedicate funding for drug development and therapy, and increase the number of CoEs
- Ensure better coordination and responsible utilization of funds.
- State governments must introduce social assistance programmes and develop satellite centers under the CoEs.
- Public and private companies could be co-opted for funding; CSR initiatives and partnerships can be leveraged to meet shortfalls.
- The issue of exorbitant drug prices and availability must be addressed.
- Last year, the government waived off GST and customs duty on medicines for rare diseases.
- This exemption applies only to drugs which are to be “imported for personal use” and not to the ones commercially available in India.
- The government must incentivise domestic manufacturers under the Production-Linked Incentive Scheme, reduce clinical trial requirements in appropriate cases, and look into options such as repurposed drugs and bulk-import.
- Government must withdraw the GST on life-saving drugs.
QUESTION FOR PRACTICE
Besides being a moral imperative of the Welfare State, primary health structure is a necessary precondition for sustainable development.” Analyze.(UPSC 2021) (200 WORDS, 10 MARKS)
