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Home / IAS Preparation / Orphan Drugs

Orphan Drugs - Definition, Examples, List of Orphan Drugs, Legislation on Development & Importance

Last Updated on Oct 25, 2023
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An Orphan Drug is a medicine or other medical product used to diagnose, prevent, and treat a life-threatening chronic condition or disorder. It is called an ‘orphan drug’ because of its limited market (which is rare, with about 450 rare diseases recorded in India so far), and is unlikely to be adopted by a pharmaceutical company. By selling only to a small group of patients, a drug manufacturer will likely not be able to recover the development and marketing costs, so the Government of India has implemented a new ‘National Policy for Rare Diseases (NPRD)’ in the year 2021 to encourage the production of these Orphan drugs.

This topic of Orphan Drugs is important from the perspective of the UPSC IAS Examination, which falls under General Studies Paper 3 (mains) and General Studies Paper 1 (Preliminary), particularly in the Science and Technology section (Human Health and Disease). This article will discuss the Orphan Drug and its definition, importance, examples, regulation, orphan drug policy 2021 & more about orphan drugs to boost your UPSC Exam preparation.

Recent News

  • In 2019 – India’s Central Drugs Standard Control Organization (CDSCO) implemented its new Drugs and Clinical Trials Rules.
  • The new rules define orphan drugs – for the first time – as drugs to treat conditions affecting less than 500,000 people in India.
  • As per the new rules, Indian regulators have now been empowered to exempt orphan drugs from Phase III and IV clinical trials.
  • On 13 January 2021, the Government of India introduced a Comprehensive ‘National Policy For Rare Diseases 2021’ (which included – Research & development to manufacture orphan drugs, treatment of rare diseases, etc.), replacing its ‘National Policy for Treatment of Rare Diseases 2017’.

Know more about the Drug controller general of India!

What are Orphan Drugs?

Orphan drugs are drugs that are used to diagnose, prevent, and treat life-threatening diseases or disorders that are rare. The field of rare diseases is complex, heterogeneous, constantly evolving, and needs more medical and scientific knowledge. About 450 rare diseases have been registered in India so far.

Orphan Drugs Examples

An orphan medicine can be described as a medicine used to treat an orphan disease. For example, some of the major medicines that are used in the treatment of orphan diseases are:

  • Alexion - Originally developed for the blood disorder paroxysmal nocturnal hemoglobinuria, but now used as treatment for atypical hemolytic uremic syndrome.
  • Revlimid - Originally developed for blood cancer (myeloma), but now used for late-stage non-Hodgkin’s lymphoma and chronic lymphocytic leukaemia.
  • Kogenate - It is used to treat or prevent bleeding episodes in adults and children with haemophilia A.
  • Vaccinia Immune Globulin IV - It is a first-line drug treatment for serious smallpox vaccination complications.
  • Kalydeco - It is used to treat cystic fibrosis (It is a rare genetic disorder that primarily affects the lungs but can also affect the pancreas, liver, intestine, kidneys, and liver).

List of Orphan Drugs in India

If we talk about the Indian scenario, 450 rare diseases have been identified in India. In India, as per the published data of the 2011 National Population Census, it was estimated that the population of rare diseases and disorders was 72,611,605. List of Orphan drugs in India:

  • India still needs orphan drug laws because these are still in the research and development stage.
  • The Central Drugs Standard Control Organisation(CDSCO) defined orphan drugs as those intended to treat a condition affecting fewer than 200,000.

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About Orphan Drug Act, 1983

The Orphan Drug Act of 1983 (ODA) is a United States federal law. It provides incentives to pharmaceutical companies to develop drugs for rare diseases. The law defines a rare disease as one that affects fewer than 200,000 people in the United States.

The ODA provides the following incentives to pharmaceutical companies:

  • Pharmaceutical companies that develop orphan drugs are granted seven years of market exclusivity. This means that no other company can market or sell the drug in the United States during that time.
  • Pharmaceutical companies that develop orphan drugs are eligible for a tax credit of 50% of the cost of clinical trials.
  • The National Institutes of Health (NIH) provides research grants to pharmaceutical companies that are developing orphan drugs.

Know more about Drug Abuse!

Legislation on the Development of Orphan Drugs in India

India does not have specific legislation on the development of orphan drugs. However, there are many policies and initiatives supporting the development of orphan drugs in India.

  • One of the most important policies is the National Policy for Treatment of Rare Diseases, 2021. This policy aims to improve the access of patients with rare diseases to affordable and effective treatment. The policy provides incentives to pharmaceutical companies to develop and market orphan drugs in India.
  • Another important initiative is the Rare Diseases Cell. This was established by the Ministry of Health and Family Welfare in 2017. The Rare Diseases Cell provides a platform for patients with rare diseases to share information. The Cell also works to raise awareness of rare diseases. It also promotes research and development of orphan drugs.

Know more about the International Day Against Drug Abuse and Illicit Trafficking!

Designating an Orphan Drug

The designation is given to certain orphan drugs, which offer promise in treating, preventing, or diagnosing orphan diseases.

An orphan disease is a rare disease or condition—according to the US FDA—that affects fewer than 200,000 people in the United States. When a drug is designated as an orphan, sponsors may be eligible for incentives like

  • Pharmaceutical companies receive tax credits for qualified clinical trials.
  • Exemption from user fees
  • Potential market exclusivity for seven years after approval.

Orphan Drug & Covid-19

  • On March 23, 2020, the FDA granted orphan drug status to Gilead Sciences for the antiviral drug ‘Remdesivir’ (originally developed to treat Ebola), which was then tested for COVID-19.
  • However, on March 25, Gilead announced that it submitted a request to the FDA to remove its orphan drug designation for remdesivir, and the FDA subsequently removed the drug from its orphan drug list.
  • Previously, Gilead had repeatedly urged the FDA to grant orphan status to the drug remdesivir to expedite drug approval.
  • In recent years, drug companies have been accused of profiting from sales by taking advantage of the law.

Know more about the United Nations Office on Drugs and Crime UNODC!

Importance of Orphan Drugs
  • Orphan drugs address public health needs but may not be developed by pharmaceutical companies for economic reasons.
  • These drugs can help alleviate the revenue loss when blockbuster drug patents expire.
  • Academic physician-scientists have taken the initiative to develop orphan drugs to make them more affordable and effective, and they have successfully treated rare diseases.
  • Sometimes, a drug may be considered an "orphan" because it is initially developed for a common condition but proves challenging to develop for another rare indication.

Know more about Bulk Drug Parks!

National Policy for Rare Diseases (NPRD), 2021

The central government laid out the National Policy for Treatment of Rare Diseases in 2017. However, the central government put the policy on hold, citing implementation challenges.

As per the states, some issues with the policy regarding cost-sharing, disease coverage, and patient eligibility for rare disease treatment under this policy were a need for more clarity. To address these questions, a review committee was constituted in 2018, which submitted its recommendations on 13 January 2021.

The National Policy for Rare Diseases (NPRD) 2021 has the following objectives:

  • The National Policy on Rare Diseases (NPRD), 2021 includes provisions for the promotion of research and development for diagnosing and treating rare diseases.
  • Improving the public health system to examine and detect rare diseases early to prevent and treat them.
  • Making a conducive environment for the indigenous production of drugs for rare diseases at affordable prices.
  • Improving India’s tertiary health care sectors and providing better diagnostic and treatment options.
  • In addition, issues such as a greater emphasis on R&D and local production of orphan drugs have been included.

Learn more about neglected tropical diseases!

Conclusion

India is still in the developing stage, a setback concerning regulation and development in orphan disease research. However, the Government of India has introduced a new drug policy to strengthen government and private institutions for the development of orphan medicine in the year 2021 to assess the spectrum and burden of orphan diseases and for public awareness programs about orphan diseases.

Check out the test series for the UPSC IAS Exam here.

Previous Year UPSC Mains Questions

Q1. What do you understand about Fixed-Dose drug Combinations (FDCs)? Discuss their merits and demerits. (UPSC Mains GS Paper 3 – 2013)

Q2. How is the Government of India protecting traditional knowledge of medicine from patenting by pharmaceutical companies? (UPSC Mains GS Paper 3 – 2019)

We hope all your doubts regarding “Orphan Drugs” are addressed after going through this article. Testbook provides comprehensive notes and online classes for UPSC, including different competitive examinations. It has always assured the quality of its products, like content pages, live tests, GK and current affairs, mocks, and so on. Ace your preparation with the Testbook App!

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Orphan Drugs - FAQs

Alexion and Revlimid Originally developed for blood disorder paroxysmal nocturnal hemoglobinuria and myeloma, but now used in treatment for atypical hemolytic uremic syndrome and chronic lymphocytic leukemia respectively.

An orphan medicine is a drug designed for a rare disease that is used to diagnose, prevent, or treat orphan diseases.

New regulations from India's Central Drugs Standard Control Organization (CDSCO) define orphan drugs—for the first time—as drugs to treat conditions affecting less than 500,000 people in India.

Developing drugs to be used to treat other common medical conditions proves to be a profitable business for pharmaceutical companies seeking use for the treatment of orphan diseases.

Pharma companies get incentives including orphan drug designation to qualify sponsors, tax credits for clinical trials, exemption from user fees and a potential seven-year market exclusivity exemption after approval.

An orphan drug is designated in the US according to the 1984 amendments to the Orphan Drug Act (ODA) intended to treat a condition affecting less than 200,000 people in the United States.
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