It is estimated that, approximately 5% of the global population suffer from a rare disease and half of the global population affected by rare diseases are children, which makes rare disease treatment a deliberating concern for children across the globe. There are around 7000 known rare diseases which are one of the most complex healthcare challenge for researchers and health professionals as most of them are difficult to diagnose due heterogeneity in disease epidemiology. Rare diseases that affect 200,000 people in the U.S. (as per FDA definition) or 1 in 2000 population in Europe, are typically genetic in nature and thus present a huge unmet need of potential treatment regimes in the market. As per World Health Organization, 80% of rare diseases are caused due to genetic abnormality and are inherited for generations. Approximately 5 % of rare diseases have treatment and most of the current therapeutic approaches include gene therapy and cell therapy. A significant gap between demand and supply of rare disease dugs is foreseen to create a massive opportunity for manufacturers and researchers in the area of rare disease treatment.
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Rare disease drug development was so far neglected or given less importance due to lower profitability as there are less number of patients deemed eligible for treatment by such drugs. However, incentives offered by Orphan Drug Act (ODA) in the U.S. and EU such as tax reduction and market exclusivity is expected to boost the global market for rare disease treatment. Since, the enactment of ODA, FDA has approved more than 480 orphan drugs and 47% of new drugs approved in 2015 were drugs for rare disease treatment. Moreover, the global market for rare disease treatment is expected to gain traction owing to development of new approaches for rare disease treatment such as regenerative medicine, personalized medicine, drug repurposing and repositioning etc. Unlike other biopharmaceutical drugs low competition in the rare disease treatment market encourages new drug developers to invest in the research and development for rare disease treatment.
High cost of treatment and challenges to reimbursement for rare disease treatment are the factors limiting the growth of global rare disease treatment market. For instance, the average annual cost for treatment with Zavesca – a substrate reduction therapy for treatment of type 1 Gaucher disease is approximately US$ 294,840 per year. Treatment for many rare disease sub-types such as Niemann-Pick Disease, Cystinosis etc. is often very expensive due to lack of coverage for these rare disease treatment by provincial and national health plans.
Increasing R&D expenditures and research grants from government in order to stimulate the development of orphan and rare disease drugs would change the landscape of rare disease treatment over the forecast period of 2016–2024. Orphan drugs are thus becoming one of the prominent candidates in the development pipeline of many of leading biotechnology companies.
Based on treatment type, global rare disease treatment market has been classified as drug therapy, gene therapy and cell therapy or regenerative medicine. Drug therapy treatment type segment is expected to hold prominent share in global rare disease treatment market owing to large number of approved drugs for rare indications.
On the basis of regional presence, global rare disease treatment market is segmented into five key regions viz. North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. North America will continue to dominate the global rare disease treatment market due to favorable reimbursement policies and prominent presence of key players in the region. Asia Pacific is expected to grow at highest CAGR over the forecast period partly due to entry of new players such as JCR Pharmaceuticals Co. Ltd. and increasing awareness towards rare diseases. MEA is expected to hold least share in global rare disease treatment market due to lack of coherent strategies for access to healthcare support for rare diseases.
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Some of the players operating in global rare disease treatment market Novartis AG, Shire PLC, Pfizer, Inc., Actelion Ltd., Bristol Myers Squibb, AstraZeneca PLC, Amgen Inc., Biogen Inc., Merck KGaA, Daiichi Sankyo Company, Sanofi, Eli Lilly and Company and others. Research collaborations and partnerships is emerging as a major trend among players in rare disease treatment market as development of rare disease is costly and less profitable. For instance, Protalix Biotherapeutics Inc. and Zacharon Pharmaceuticals collaborated with Pfizer Inc. for development of rare disease treatment. Mergers and acquisitions in rare disease treatment market are preferred by the leading players to gain immediate growth effect. For instance, in January 2015 Shire plc. acquired Dyax Corp. for US$ 5.9 Bn and expanded its hereditary angioedema (HAE) portfolio – a rare disease of blood. Subsequently in 2016, Shire plc. acquired Baxalta Incorporated for US$ 32 Bn and became a leader in global rare disease in treatment market.
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