Orphan Drugs and Repurposed Orphan Drugs in India

  Dec 29, 2023

Orphan Drugs and Repurposed Orphan Drugs in India

What is an Orphan Drug (OD)?

An Orphan Drug is a medicine considered crucial for treating, preventing, or diagnosing life-threatening or chronically debilitating diseases but not commercially viable due to a small patient population. In India, ODs are defined as drugs intended to treat conditions affecting no more than 0.5 million people.

What are Rare Diseases (RDs) in India?

Rare Diseases are severe, often life-debilitating conditions that are infrequent in the population. Despite their individual rarity, cumulatively, they affect a substantial number of people. In India, about 96 million people live with RDs.

What challenges are faced in treating RDs in India?

Despite the high prevalence of RDs, there has been limited attention from the government and pharmaceutical industry. The lack of domestic manufacturers and the high cost of ODs make access to treatment difficult.

What is the National Policy for Rare Disease (NPRD)?

Released in 2021, the NPRD in India acknowledges the challenges in accessing ODs for RDs, stating these drugs are expensive and not widely available or accessible.

What are Repurposed Orphan Drugs (RODs)?

RODs are medicines originally developed for common conditions, now repurposed for RDs. They offer a cost-effective and quicker alternative due to existing data on their safety and efficacy.

Why is India a key player in the use of RODs?

India, being the largest global manufacturer of generic medicines, has significant potential in the production and use of RODs. Many such drugs, no longer under exclusivity, can be manufactured and made available in India.

How does India’s patent regime support the use of RODs?

India's patent regime prevents the evergreening of drugs, allowing for the generic production of many RODs. This policy supports the availability of RODs by enabling the manufacture of generics once the exclusivity period of a drug expires.

National Policy for Rare Diseases (NPRD), 2021 Highlights: National Policy for Rare Diseases (NPRD), 2021

Introduction of the NPRD:

The Union Health Ministry introduced the National Policy for Rare Diseases (NPRD) in 2017 to comprehensively tackle rare diseases in India.

Policy Update and Financial Support:

The policy was updated in 2021, proposing one-time financial assistance of up to Rs. 50 lakhs for the treatment of patients with rare diseases, irrespective of their economic background.

Establishment of Centers of Excellence (CoEs):

As part of the NPRD, the central government established 11 Centers of Excellence across the country. These CoEs are tasked with diagnosing, preventing, and treating rare diseases.

Approach for Financial Assistance:

Patients seeking treatment for rare diseases can approach the nearest CoE for assessment and to avail of the financial support offered by the policy.

Financial Support Framework:

The financial assistance provided under the NPRD is outside the purview of the Rashtriya Arogaya Nidhi scheme, emphasizing its independent and dedicated approach to rare diseases.

Dynamic Update of Disease List:

The NPRD includes a continually updated list of rare diseases to ensure that the policy remains inclusive and relevant.

Promotion of Research and Drug Development:

The NPRD, 2021, also focuses on promoting research and development for the diagnosis and treatment of rare diseases.

Indigenous Drug Manufacturing:

A significant aspect of the policy is the encouragement of local development and manufacturing of drugs for rare diseases, aiming at making them affordable and accessible.


The structured approach of the NPRD, from its introduction to the detailed implementation strategies, reflects a comprehensive effort by the Indian government to address the multifaceted challenges posed by rare diseases.

Definition and Categorization:

The NPRD, 2021, defines rare diseases in three distinct groups, categorized based on clinical expertise and treatment approach.

Group 1: Disorders Amenable to One-Time Curative Treatment:

Includes conditions treatable with Hematopoietic Stem Cell Transplantation (HSCT) and those amenable to organ transplantation.

Group 2: Diseases Requiring Long-Term/Lifelong Treatment:

Encompasses disorders manageable with special dietary formulae or Food for Special Medical Purposes (FSMP) and those treatable with hormones or specific drugs. These require less costly treatment and documented surveillance.

Group 3: Diseases with Available Treatment but High Cost and Selection Challenges:

Diseases where effective treatment exists, but patient selection, very high cost, and the need for lifelong therapy pose challenges. Includes conditions with documented evidence for long-term outcomes but limited or awaiting further research.

Government Initiatives under NPRD:

Establishment of eleven Centers of Excellence (CoEs) for diagnosis, prevention, and treatment of rare diseases.

Increased financial support for rare disease patients, from up to Rs. 20 lakhs to up to Rs. 50 lakhs per patient, to facilitate access to necessary treatments.

The NPRD, 2021, represents a comprehensive approach by the Indian government to address the complexities and challenges associated with rare diseases, focusing on improved diagnosis, treatment, and financial aid to patients.



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