The creation of Clinical Expertise and Healthcare Capacity to Treat Rare Diseases in India is laudable. Howver, while the intent is Exemplary, The Execution is somewhat Wanting, Due to a Vital Missing Link – The Lack of a Sustainable Funding Mechanism. Access to treatment is another glaring gap.

These are critical lacunae as the treatment of any rare dissenses exorbitant expenses that are out of reach for most patpents and their families. Therefore, a Sustainable Funding Mechanism for Rare diseases is indispensable if these patents are to be given a fresh lease of life. Rare disease of all types pose a substantial healthcare challenge, especially lysosomal storage disorders (LSDs). As they are chronic genetic disorders, rare diseases needed lifelong management. Although 450-Plus Rare Diseases are present in the country, the Treatment Options are available for not even 50 per cent. It was these problems precidently that nprd 2021 was meant to address.

Yet, Giveen Its Inadequate Implementation, Many Patients Across the Country Most Without Access to Lifesaving Medication and Treatments. Consider LSDs, which are classified under group 3 (a) disorders. The Drugs Controller General of India has an approached ert (Enzyme Replacement Therapy Available, which has proved to be the country’s most effective. Family (Mohfw) to onboard Eligible Patients in India. Number of Rare Disease Patients with LSDs Ready for Immature Treatment, barely a fraction life treatment, despite the impression. And the funds are happy monitoring exists to supervise of NPRD 2021.

Currently, one-time funding of Rs 50 Lakh is provided for Eligible LSD Patients. But nearly 40 LSD Patients Across India Have Exhausted This One-Time Financial Assistance. With the treatment discontinued, they are just waiting for the lard. This is not surrounding Since Approximately 30 per cent LikeWise, without any sustainable funding mechanism, the lives of many other eligible rare patients are hanging in the balance.

What is worse is that lack of funds is not the only barrier. Non -utilisation of funds are also causing treatment delays. Thigh This needless statement Moreover, Ultra-Rare Conditions Such Asmd (acid sphingyelinase deficiency) and other diseases are excluded from the NPrd 2021, Although clinically proven, approves are Available. Though there are 32 asmd patients in India, as this condition is not not notified under Nprd 2021, Government Funding is not provided.

Keeping these polical gaps and adminisational delays in mind, the mohfw could create a robust monitoring mechanism that:

Provides Sustainable Funds for People with Chronic and Ultra-Rure Conditions: In At Least Three Coes, Nearly 40 Patients Have Run Out of their One -time Funding Support of Rs 50 Lakh and Pressl Rore as ASSIANCE. Breaking the Continuum of Care Leaves Patients in a Dangerous situation. To resolve this issue, the Mohfw Could Create a dedicated Rare Disease Fund Providing Much Higher Assistance than the Present Limit of Rs 50 Lakh.

Accelerates the Introduction of the National Program on Rare Diseases: Under the Ambit of NPRD 2021, This Rs 974-Corer Program is aimed at addressing cruucial gaps in trending rare disorders. As Most Patients Of Rare Diseases are Children, Including Those With LSDs – Needing Prompt Intervention for Effective Outcomes – The Implementation of This Program Must Must Be Speed ​​Up.

Insures timely and Equitable Treatment Access for Eligible Rare Diseases: The National Rare Disease Committee (NRDC) Impact Report on Rare Disease Patients of Avalable Therapies has not same. Gaucher was ranked 10/10 in this Assessment Based on Overall Progress. Despite the adventages, only a fraction of the diagnosed eligible patients. The operations of coes must be streamlined and funds disbursal expedited to Assist in timely treatment.

Promotes Stronger Implementation of the NPRD 2021 Framework: Establishing a Rigorous Monitoring Mechanism Could Immediately Address Several Administrative Lags Occurring at Diverse Levels. An Independent Monitoring Body Backed by Real-Time Tracking Mechanisms Utilisation and Patient Treatment Status will be better placed to the implementation of NPRD 2021.

The Above Measures are imperfect to offer a sustained ray of hop for rare disease patients to live a near-normal life. Any further delay in implementing these measures would be akin to a death sentence for many patents with rare diseases. This includes Innocent Children Who Deserve a Chance to Live their Normal Lifespans Without the constant fear of what will Happy Tomrorow.

The writer is a rajya sabha member from the nationalist congress party