Rare diseases are defined as medical conditions that impact fewer than 1 in 1,000 individuals. Examples of such conditions include Gaucher’s disease and various forms of muscular dystrophy. These diseases often require chronic management and long-term care, which can significantly affect the quality of life of those diagnosed.
Orphan drugs refer to medications that are specifically developed to treat rare diseases. Due to the limited patient population, these drugs are often not commercially viable for pharmaceutical companies, leading to their high costs and restricted availability without government support.
The NPRD 2021 is an initiative launched by the Government of India aimed at addressing the treatment and management needs of patients with rare diseases. This policy provides substantial financial support, offering up to ₹50 lakh per patient for treatment at designated Centres of Excellence (CoEs). The NPRD also focuses on enhancing awareness, promoting early diagnosis, and fostering local research for orphan drug development.
The NPRD 2021 categorizes rare diseases into three distinct groups:
The NPRD offers financial assistance of up to ₹50 lakh per patient for diseases classified under Group 1, primarily through the CoEs. This funding aims to cover the costs associated with expensive, one-time therapies that are often required for rare diseases.
CoEs are specialized medical institutions recognized by the government for their capability to treat rare diseases. Institutions such as AIIMS, PGIMER in Chandigarh, and SSKM Hospital in Kolkata have been designated as CoEs. These centers are responsible for diagnosing, treating, and managing patients suffering from rare diseases under the NPRD framework.
In 2021, the NPRD was allocated ₹25 crore to fund treatments for patients. Alongside this, a Rare Disease Fund was established, supported by voluntary donations and corporate social responsibility (CSR) initiatives. Recently, ₹4 crore was allocated to the CoEs for rare disease treatment in August 2024.
Despite the efforts made under the NPRD, several challenges continue to exist:
The NPRD promotes the local development and manufacturing of orphan drugs to lower treatment costs. The government is also exploring tax incentives for pharmaceutical companies to produce these drugs domestically, thereby making treatments more affordable. Furthermore, efforts are underway to streamline the importation process for these medicines by reducing customs duties and approval times.
The NPRD places significant emphasis on early detection of rare diseases through initiatives such as newborn screening programs and public awareness campaigns. The policy also encourages collaborative research and development efforts with international organizations to enhance diagnostic and treatment capabilities.
The National Policy for Rare Diseases (NPRD), 2021, aims to significantly improve the treatment landscape for patients suffering from rare diseases through financial assistance, research encouragement, and local drug development promotion. While the policy has made commendable progress in enhancing treatment access, challenges such as high drug costs and slow approval processes still need addressing. By enhancing domestic manufacturing of orphan drugs and increasing awareness, the NPRD aspires to secure better health outcomes for rare disease patients across India.
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