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Rare Disease Drugs Market 2020 Global Trends, by Test Type, Disease, Method, Key Players, Shares and industry Demand

Global Rare Disease Drugs Market – Insights

Rare disease, also known as orphan disease, is condition that is impacts a low number of people at any given time. The U.S. Food & Drug Administration (FDA) defines a rare disease as condition affecting less than 200,000 people in a given country. In the U.S., the Orphan Drugs Act allows manufacturer to request the U.S. FDA to grant an orphan drug status to drugs intended for the treatment of a rare disease. According to the U.S. FDA, 2017, number of orphan drug designation requests have steadily increased in the recent past.

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Various uncertainties associated with rare diseases have failed to offer lucrative growth opportunities for market players. Moreover, low prevalence of these diseases and huge differentiation and heterogeneity among the group of diseases also limits R&D, which further leads to poor understanding of these diseases. Such scenario limits product development. However, introduction of some expensive orphan drugs e.g. spinal muscular atrophy drug Spinraza priced at US$ 125,000 per vial and high unmet needs have led market players to enhance their market share in the global rare disease drugs market.

Competitive Analysis of Top 10 Market Players in the Global Rare Disease Drugs Market

The total revenue of top 10 market players for 2016 accounted for US$ 62.5 billion. The top 10 market players in 2016, based on the 2016 revenue include:

  • Novartis AG
  • Hoffmann-La Roche Ltd.
  • Celgene Corporation
  • Bristol-Myers Squibb Company
  • Shire plc.
  • Pfizer, Inc.
  • Sanofi S.A.
  • Bayer
  • Alexion Pharmaceuticals, Inc.
  • Biogen, Inc.

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  1. Novartis AG — It is a pharmaceutical company focused on development and commercialization of cancer therapies and ophthalmic products. The company operates its business through Innovative Medicines Division, which consists of oncology business unit and Novartis Pharmaceutical Business Units. Innovative Medicines Oncology division offers products for cancer and rare diseases and the pharmaceutical business consists of ophthalmology, neuroscience, immunology and dermatology, respiratory, cardio-metabolic, and established medicines. In 2015, Novartis lost patent protection for its orphan drug Gleevec for the treatment of chronic myeloid leukemia (CML). In 2016, Sun Pharma launched generic Gleevec, hindering sales of Gleevec.
  2. Celgene Corporation — The company develops and commercializes therapies for cancer and inflammatory diseases such as Revlimid, Pomalyst, and Otezla. The company is focused on expanding its product portfolio using its robust pipeline of cancer therapies. For instance, Revlimid (lenalidomide) is in phase III clinical trials for diffuse large B-cell lymphoma, indolent lymphoma, and follicular lymphoma. The company developed its own target-identification and drug-discovery technology platforms.
  3. Shire Plc. – The company grossed US$ 11 billion in orphan drug products category (excluding oncology products) in 2016. In the same year, it acquired Dyax and Baxalta and its three therapeutic segments: hematology, immunology, and oncology. The company invested US$ 1.4 billion in R&D of therapies for rare diseases. The company has 37 therapies in the pipeline.

Top 10 Orphan Drugs U.S. Sales (US$ Mn) in Rare Disease Drugs Market (2016):

Orphan Drug Product Company U.S. Sales,(US$ million) 2016
Revlimid Celgene 4,417
Rituxan Roche 3,970
Copaxone Teva Pharm 3,257
Opdivo Bristol-Myers Squibb 2,664
Avonex Biogen 1,675
Imbruvica AbbVie 1,580
Sensipar Amgen 1,240
Gleevec Novartis 1,214
Velcade Takeda 1,133
Xyrem Jazz Pharmaceuticals 1,114

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Global Rare Disease Drugs Market – Trends  

Wide gap between market needs and potential market offerings has primarily led manufacturers to focus on R&D of therapies for the treatment of rare diseases. For instance, according to the U.S. Food & Drug Administration (FDA), in 2016, 582 requests for orphan drug designation were filed by the biopharma companies. Moreover, availability of highly effective drug therapies has led to increase in sales of orphan drugs. There are several potential drug therapies in the pipeline for various cancers and rare diseases. For instance, Niraparib by Tesaro, Inc. is in phase I clinical trial for the treatment of ovarian cancer, Emicizumab by Roche is in phase III clinical trial for the treatment of hemophilia, and LentiGlobin by Bluebird Bio in different phases for the treatment of sickle cell anemia and beta-thalassemia major. Positive outcome from these clinical trials is expected to boost growth of the market over the forecast period.

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Last Updated: 16-Mar-2021