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By Balaram Gajra and Shraddha Limbachiya

Balaram GajraShraddha Limbachiya-finalAn orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition. India currently has no regulations for orphan drug manufacturing or selling. Treating rare or orphan diseases is important to India but very costly, which increases the patient burden. In India, 72,611,605 people are suffering from rare diseases, and 6,000–8,000 rare diseases can be found, including Leishmaniasis, Norrie Disease, Arthrogryposis, Cystic Fibrosis, Wilson Disease, etc., many of which still do not have any cure and are mostly genetic in nature.

Disease Per 100,000 In India
Acatalasmia 3 36,000
Acromegly 5 60,000
Alkaptornuria .3 3,600
Alpa-1 antritrypsin 25 300,000
Grave disease 50 600,000
Parkinson disease 15 180,000

Adapted from the Rare Diseases List provided by the Foundation for Research on Rare Diseases and Disorders (accessed May 7, 2015)

Many countries have different definitions and regulations of orphan drugs, outlined below.

Country Definition
United States The Orphan Drugs Act (ODA) is a federal law concerning rare diseases that affect fewer than 200,000 people or are of low prevalence (less than 5 per 10,000 in the community).
Europe According to the Orphan Drug Regulation in Europe, an orphan disease is a disease or disorder that affects fewer than 5 in 10,000 citizens.
Japan In Japan, any disease with fewer than 50,000 prevalent cases (0.4%) classifies as a rare disease. In Australia, orphan drugs are drugs used to treat diseases or conditions affecting fewer than 2,000 patients annually (0.2%).
Canada Canada has no official “orphan disease” status. Based on international standards it could be defined as diseases with a potential patient population between 3,300 to 22,500.

The lack of regulation in India increases the burden on the patients but also negatively impacts the economic success of India’s pharmaceutical industry. Orphan drugs may help pharmaceutical companies reduce the impact of revenue loss by patent expiration of blockbuster drugs. Although there may still be challenges ahead for the industry, orphan drugs seem to offer the key to recovery and stability within the market. Governments of various countries have proactively implemented special incentives for the manufacturers of orphan drugs. For example, regulations include accelerated marketing procedures, marketing exclusivity, tax credit grants for research, reconsideration of applications for orphan designation, and technical assistance for elaboration of the application file.


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In India, several nongovernment organizations (NGOs) are working in the area of rare diseases like the Foundation for Research on Rare Diseases and Disorders, Alzheimer’s and Related Disorders Society of India – ARDSI, Down Syndrome Federation of India, Hemophilia Federation India, International Parkinson and Movement Disorder Society, etc. However, there are no federal regulations or incentives.

The government of India should address the situation immediately and issue an appropriate Official Development Assistance combating the challenges. This would allow the domestic pharmaceutical and biopharmaceutical industries of India to rapidly emerge as a force to reckon with in this important global space. Such legislation could also bring relief to the unlisted and potentially large groups of rare diseases sufferers in India.

In order to lower the cost and availability of orphan drugs, we suggest:

  1. Government of India (GoI) should come up with a clear, separate Orphan Drugs Act or an addendum to Drugs and Cosmetics Act specifying the definition of orphan diseases and other related things.
  2. GoI should provide special incentives to industries for the research and manufacturing of orphan drugs.
  3. GoI should motivate and support NGOs to import the available drugs for rare diseases at affordable rates.
  4. GoI should have a special program on rare diseases to be run by government organizations like primary health centers and community health centers to provide special care to the patients of rare diseases.
  5. GoI should run awareness campaigns regarding rare diseases.

Patients with rare diseases in India should not be left to their own tragic circumstances without any compassion and care, and the GoI should develop an appropriate Official Development Assistance combating these challenges.

Balaram Gajra, Ph.D., is an associate professor at the Department of Pharmaceutics and Pharmaceutical Technology, Ramanbhai Patel College of Pharmacy (RPCP), Charotar University of Science and Technology (CHARUSAT), Changa, India. He played a key role in starting the AAPS RPCP-CHARUSAT student chapter and served as the faculty advisor for 2013–2014.
Shraddha Limbachiya is acquiring her Master of Pharmacy in pharmaceutics (drug regulatory affairs) at Ramanbhai Patel College of Pharmacy (RPCP), Charotar University of Science and Technology (CHARUSAT) in Changa, India. She is member of the AAPS RPCP-CHARUSAT student chapter.