A disease that has not been adopted by the pharmaceutical industry because it provides little financial incentive for the private sector to make and market new medications to treat or prevent it.  An orphan disease may be a rare disease or a common disease that has been ignored because it is far more prevalent in developing countries than in the developed world. A rare disease is defined by the European Union as one that affects less than 5 in 10,000 of the general population.

There are thousands of rare diseases. To date, six to seven thousand rare diseases have been discovered and new diseases are regularly described in medical literature. 7% of the population are estimated to be affected by a rare disease at some point in their lives. 75% of rare diseases affect children, and 30% of rare disease patients die before their fifth birthday. Rare diseases include rare cancers such as childhood cancers, sarcoma and some other well known conditions such as cystic fibrosis, Huntington’s disease, Crohn disease, etc.

Drug development strategies

The growth of Pharma industries has slowed in recent years because of various reasons such as patent expiries, generic competition, drying pipelines, and increasingly stringent regulatory guidelines. Many blockbuster drugs will lose their exclusivity in next 5 years. Therefore, the current economic situation plus the huge generic competition shifted the focus of pharmaceutical companies from the essential medicines to the new business model such as orphan drugs. The new business model of orphan drugs could offer an integrated healthcare solution that enables Pharma companies to develop newer areas of therapeutics, diagnosis, treatment, monitoring, and patient support. Incentives for drug development provided by governments, as well as support from the FDA and EU Commission in special protocols are a further boost for the companies developing orphan drugs.

An orphan drug can be defined as one that is used to treat an orphan disease. For example, haem arginate, used to treat acute intermittent porphyria, variegate porphyria, and hereditary coproporphyria, is an orphan drug. However, it comes as a surprise that ibuprofen can also be categorized as an orphan drug, because it has been used to treat an orphan disease, namely patent ductus arteriosus in neonates. This observation stresses that barriers to the development of orphan drugs do not occur only at the premarketing stage; in some cases it may not be commercially worth mounting an efficacy trial, even for a drug whose efficacy elsewhere is well established.

Orphan drug status

Products intended to treat rare diseases are developed to treat patients suffering from very serious diseases for which no treatment, or at least a satisfactory one, has so far been available. These diseases affect only a small proportion of the population most often at birth or in infancy. The number of rare diseases for which no treatment is currently available is estimated to be between 4,000 and 5,000 worldwide. Few drugs which are withdrawn from the market for some reasons, e.g., thalidomide widely used as a hypnotic drug some years ago for its high teratogenic risk may show a very interesting therapeutic application, i.e., analgesic properties in rare diseases such as leprosy and lupus erythematosus. These are diseases for which no satisfactory treatment is available.

According to market report 10 top selling orphan drugs
Drug Indication
Rituxmab Oncology
Ranibizumab Ophthalmology
Somatropin EPR, Metabolism
Lenalidomide Oncology
Imatinib mesylate Oncology
Filgrastim Hematology
Glatiramer Acetate MSP
Recombinant Factor VIII Hematology
Bosentan Monohydrate Cardiovascular
Bortezomib Oncology

Orphan drugs market level

According to market estimate the global orphan drugs market has reached around $112 billion by 2014 growing from $84 billion in 2009. The market is expected to grow at a compound annual growth rate (CAGR) of nearly 7% to reach $125 billion by 2018.

The future of orphan drug

As the Asian pharmaceutical markets grow, so too will opportunities for orphan drugs in Asia. Asian governments are becoming more aware of the importance of orphan drugs, and reimbursements for these products will increase in the future. This encouragement can bring in a revolution among the pharmaceutical and biotechnology companies for developing and marketing orphan drugs.

The issue of orphan drugs becomes more important for third world countries like India, which are affected the most. Why no new drug is coming up or nobody is investing in research and development in malaria, leishmania, etc. Usually we criticize the pharmaceutical industry or manufacturers for this. It is not easy to produce and market orphan drugs. The manufacturers of drugs have to amortize their operational expenses, their research investment, and they have to make reasonable profit so that they can finance new ventures in the future.

DelveInsight Orphan Indications reports

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