In a moderately consolidated global orphan drugs market, players are primarily focused on strategic acquisitions and are strategizing to gain maximum returns on investments made for R&D of orphan drugs and to leverage the incentives offered by governments for research on orphan drugs, according to recent market intelligence by Transparency Market Research. In this scenario, some of the companies to hold a commanding position in the global orphan drugs market are Novartis AG, Celgene Corporation, Pfizer Inc., Alexion Pharmaceuticals Inc., GlaxoSmithKline plc, and Johnson & Johnson.

The global orphan drugs market is projected to display a healthy 12.1% CAGR from 2017 to 2025, for the market to reach a valuation of US$318.5 bn by the end of 2025.

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Vis-à-vis revenue, in 2016, oncology stood as the dominant segment based on disease type, and is expected to hold on to its dominant position over the forecast period. The increasing genetic mutations and the increasing burden of rare cancers are attributed to the growth of this segment.

In terms of product type, biologic products led the market in 2016 on the basis of revenue. The segment is expected to display a significant CAGR over the forecast period backed by factors such as high efficacy, fewer side effects, and difficulty to develop generic versions of these drugs. Hospital pharmacies led the global orphan drugs market in terms of revenue in 2016 amongst all distribution channel segments.

North America held the leading share of the orphan drugs market in 2016. Within North America, the U.S. is a leading domestic market contributing substantial revenue to this market.

As per an analyst at TMR, the global orphan drugs market is anticipated to expand at a significant growth rate on account of a number of factors. Amongst all, high return on investments for the development of orphan drugs is a key one. The development of phase- III orphan drugs costs over 50% more than the cost of development of non-orphan drugs. The return on investments is estimated to be over 1.2 times greater for phase- III orphan drugs than non-orphan drugs.

In developed countries, strong focus on research and development of orphan drugs, which receives government aid, tax credits, and fee waivers is contributing to the growth of this market. As per records of the Research Office Legislative Council Secretariat, there are more than 30 million individuals suffering from rare diseases in the U.S. As a result, 4,171 drugs have received orphan drug status in the U.S., which is attracting drug manufacturers to invest in this market.

Furthermore, increasing number of rare diseases that are related to genetic reasons leading to investments for drug development of these diseases is benefitting the orphan drugs market. As per statistics of Global Genes, a non-profit organization, more than 7,000 types of rare diseases have been identified globally. At present, more than 300 million people worldwide are estimated to be affected by rare diseases that calls for considerable production of orphan drugs.