In this article we will discuss about: Understanding Orphan drugs: what are they, How are they classified and what are their challenges in the Indian context?
Orphan drugs refers to the medicines which are developed to help treat, prevent or diagnose rare , life-threatening, chronic condition or disorder. It is often called as Orphan drug because of its limited market as it is used to treat “orphan” (rare) diseases, which are conditions that each affect fewer than 200,000 people. In India about 450 such diseases are recorded so far. So they are unlikely to be adopted by pharmaceutical companies as developing these drugs are intending to treat a rare disease which does not allow the recovery of the capital invested for it’s research.
Some Orphan diseases are:
- Genetic diseases: cystic fibrosis and duchenne muscular distrophy
- Rare cancers: neuroblastoma and gliomas
- Metabolic disorders: Gaicher’s disease and fabry disease
- Autoimmune diseases: systematic sclerosis
Criteria to designate a drug as Orphan drug
- Must have a low prevalence, and disease must lack approved treatments.
- Must have scientific evidence that the drug has the potential to treat the condition.
- Evidence can be presented at any stage of drug development from preclinical research to late phase clinical trials.
Once designated, orphan drugs receive several incentives to encourage their development, including market exclusivity, tax credits for research and development (R&D) expenses and fee waivers for regulatory applications.
Law for Orphan drugs was first enacted in USA in 1983, Japan in 1985, Australia in 1997 and European Union in 2000.
About USA Orphan Drug Act, 1983
The Orphan Drug Act of 1983 (ODA) is a United States federal law. This law encourages pharmaceutical companies to develop drugs for rare diseases by providing incentives and subsidies for clinical trials and market exclusivity to such drugs maker institutions. 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.
- 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
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 – This drug was initially developed for the blood disorder paroxysmal nocturnal hemoglobinuria, but now it is used as treatment for atypical hemolytic uremic syndrome.
- Revlimid – Originally it was developed for blood cancer (myeloma), but now it is 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 drug treatment for serious smallpox vaccination complications.
- Kalydeco – It is used to treat a rare genetic disorder cystic fibrosis which primarily affects the lungs but can also affect the pancreas, liver, intestine, kidneys, and liver.
- Ivacaftor: It is a therapy to treat cystic fibrosis, a genetic disorder that causes problems with breathing and digestion.
- Alglucerase: It is used to treat Gaucher disease, which causes pain and damage to tissue in the liver, spleen, lungs and bone marrow.
- Coagulation factor IX: This drug helps to prevent bleeding in people with hemophilia B.
- Imatinib: It is an oncology therapy used to treat certain types of leukemia.
- Rucaparib: It is a treatment for ovarian cancer.
Orphan Drugs in India
In India, about 450 such rare diseases have been identified so far. 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.
While orphan drugs are crucial to treat rare, sometimes debilitating conditions the high cost of their research and development ia a major barrier for their development in India. Even now India faces significant challenges in ensuring the development, affordability and accessibility of such drugs especially compared to countries like United States of America and European Union. 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. And 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.
- So, 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.
- 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.
Challenges for Orphan drugs development in India
- High R&D costs
- Clinical trials: limited number of patients prolongs development timeline.
- Prices and accessibility: High cost of development makes it inaccessible to needy.
- Limited financial incentives
National policy for treatment of rare diseases, 2017
- An inter-ministerial consultative committee should be constituted to coordinate the initiative of different ministeries and departments on rare diseases.
- Creation of Corpus fund at Central and State level for treatment of rare diseases.
- Create a patient registry for disease in Indian council of medical research.
- Create awareness among health professionals, families of patients and public.
National Policy for Rare Diseases (NPRD), 2021
Although, the central government had laid out the National Policy for Treatment of Rare Diseases in 2017. But, somehow they 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.
Importance of Orphan Drugs
- Orphan drugs are crucial for public health needs but they may not be developed by pharmaceutical companies for economic reasons.
- These drugs can help alleviate the revenue loss when blockbuster drug patents expire.
- Orphan drugs should be developed so as to make them more affordable.
- New Orphan drugs should be developed to make them more effective against the rare diseases.
- Development of these drugs in India can make them easily accessible to the patients who need them, generally, patients need to import most of the orphan drugs.
Why in the NEWS?
- The Delhi High Court has established the National Rare Diseases Committee, a five-member panel, to implement the Centre’s rare diseases policy on May 15, 2023.
- On 4 October 2024, the Delhi High Court directed that the Centre establish a National Fund for Rare Diseases and allocate Rs 974 crore for 2024-25 and 2025-26. This was to be approved and transferred in 30 days. It also said “treatment for all eligible… patients, as per an AIIMS report dated July 21, who are before the court in these batches of petitions shall commence in 45 days as per the NRDC (National Rare Diseases Committee) recommendations.”
Conclusion
India is still in the developing stage, there are many concerns regarding regulation and development in orphan disease research. However, the Government of India has introduced a new drug policy “The National Policy on Rare Diseases (NPRD),” to strengthen government and private institutions for the development of orphan medicine in the year 2021. Delhi’s high court has also directed the center to setup National Fund for Rare Diseases (NFRD) to facilitate the functioning of The National Policy on Rare Diseases (NPRD).
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