What are rare illnesses
Rare Illness:
- It is characterised as a low-prevalence illness that affects fewer individuals than other common diseases in the broader population.
- Numerous uncommon disease instances have the potential to be grave, persistent, and even fatal.
- Of the more than 7,000 identified diseases globally, just 5% are curable.
Dermatomyositis:
- This kind of autoimmune illness is uncommon.
- The sickness impacts the skin as well as the muscles.
Symptoms of it:
- Swelling of the muscles
- The skin has red rashes.
- Weakness and weariness in the body
- It is more common in the following two age groups:
- In kids, those who are between the ages of five and fifteen
- In individuals between the ages of 40 and 60
- More women than males are affected by this illness.
- uncommon illnesses in India
- With over 450 diseases identified, India accounts for one-third of the occurrence of rare diseases worldwide.
- They include Whipple’s disease, mucopolysaccharidosis type 1, and spinal muscular atrophy as well as Gaucher’s disease.
- Rough calculations indicate that 8–10 crore Indians are afflicted with a rare disease;
- More than 75% of them are kids.
Problems related:
- These illnesses are generally disregarded.
Resource limitations:
- When it comes to rare disease awareness, diagnosis, and drug development, India lags well behind.
- In order to address uncommon diseases, the Ministry of Health and Family Welfare developed a national strategy in 2017, but it was withdrawn in 2018 due to
“Implementation challenges” and uncertainty around disease coverage:
- Patient authorization
- spending-sharing.
- In 2021, the National Policy for Rare Diseases (NPRD), a revamped policy, was unveiled.
- The policy blames a lack of data, as though routine data gathering and epidemiological analyses are outside the purview of the government.
- An accurate and timely diagnosis is essential to the effective therapy of any illness.
- Patients with uncommon diseases may have to wait seven years for a diagnosis—if one is made at all.
- Most doctors don’t know how to analyse the symptoms and indicators.
- For healthcare personnel to become more accurate in their diagnoses, they must receive training.
- It is required that expectant mothers who have a family history of uncommon disorders receive prenatal screening as well as postnatal diagnosis and management.
- Of the roughly 450 uncommon diseases found in India, less than half can be cured.
- Only a small number of rare diseases have treatments approved by the Drugs Controller General of India, and these treatments are only available through Centres of Excellence (CoEs).
- CoEs are few (12), dispersed erratically, and lack coordination.
- delayed diagnosis
- insufficient treatments
- It’s common to be unavailable on time.
Problems:
- Despite growing each time, the budget’s allotment for uncommon diseases—which is only ₹93 crore for 2023–2024—remains modest.
- As per the NPRD standards, each patient is eligible to receive up to ₹50 lakh, which will be paid to the relevant CoE.
- This sum is woefully insufficient, given chronic uncommon diseases typically require lifelong management and therapy.
- The CoEs are hesitant to start any treatment that they might have to stop later, as this puts them at risk of legal action from patients and their families.
- Fund utilisation confusion: Of the ₹71 crore in financial support allotted to the 11 CoEs for the current year, almost ₹47 crore is still unutilized.
- There isn’t any equality among CoEs.
- Mumbai used up all of its resources while only treating 20 out of 107 patients.
- Delhi uses fewer than twenty percent.
- The CoEs are being asked by NPRD to crowdfund patients’ treatment for rare diseases.
- In three years, less than ₹3 lakh has been received from a portal with over 1,400 registered patients.
The Way Ahead:
- The Central government must define rare diseases uniformly, allocate more funds for research and development, enhance the number of CoEs, and boost budgetary expenditures.
- Make sure there is improved collaboration and prudent financial management.
- Under the CoEs, state governments are required to establish satellite centres and implement social assistance activities.
- financial for public and private enterprises might be pooled; partnerships and CSR initiatives could be used to make up for financial gaps.
- It is necessary to address the problem of excessive drug pricing and availability.
- The government eliminated customs and GST duties on medications for uncommon illnesses last year.
- Only medications that are to be “imported for personal use” are exempt from this law; medications that are sold commercially in India are not.
- The government should consider alternatives including bulk imports and repurposed medications, as well as lowering clinical trial requirements when appropriate and providing incentives to domestic manufacturers through the Production-Linked Incentive Scheme.
- The GST on life-saving medications must be removed by the government.
