Orphan Drugs Market Size, Share & Trends Analysis Report By Drug Type (Biological, Non-biological), By Disease Type (Oncology, Hematology, Neurology, Cardiovascular, Others), By Distribution Channel (Hospital, Pharmacy, Online Sales, Others), By Region, And Segment Forecasts, 2023 To 2030

Rise in rare diseases, beneficial government policies, and exclusive market access for orphan drug manufacturers fuel the expansion of the orphan drugs market. Nevertheless, the limited availability of patients for clinical trials and product promotion and the high expenses associated with patient treatment may impede market growth during the forecast period. Despite this, prospects for profitable expansion in emerging economies still need to be fully tapped, as well as explore new indications for established orphan drugs.

A key driving force in the global market is the escalated investment in research and development by prominent industry players toward developing innovative orphan drugs. As knowledge and awareness regarding rare diseases continue to grow, major clinical-stage biopharmaceutical organizations and established market players have amassed an extensive pipeline of potential orphan drug candidates undergoing clinical trials at various stages. It is because major pharmaceutical breakthroughs are more likely to result in blockbuster drug developments in rare disorders than traditional pharmaceutical portfolios. Additionally, pharmaceutical companies must conduct more extensive outcome studies for traditional therapeutics for diseases such as diabetes and coronary artery disease to gain regulatory approvals compared to rare diseases. Such factors are anticipated to drive market growth during the forecast period.

The global prevalence of rare diseases is on the rise. Both developed, and developing countries have implemented regulations to promote the development of drugs for these diseases and ensure their accessibility to patients. According to the Genetic and Rare Diseases (GARD) Information Center and GlobalGenes, rare diseases affect a significant portion of the global population, with approximately 7,000 known rare diseases and 80% having genetic origins. As per a study titled "Rare diseases: maintaining momentum," drug companies have spent USD 22.9 billion on rare disorder research in 2021, indicating a 28% increase from the previous year. This growing prevalence of rare diseases and increased research and development spending are expected to boost the market for innovative orphan drugs in the upcoming years.

Furthermore, favorable government policies for approving and producing orphan drugs also contribute to market growth. Various countries, including the U.S., Japan, Australia, and several other European countries, have established well-recognized orphan drug acts (ODA) to support the development of drugs for rare diseases. The European Medicines Agency (EMA) facilitates developing and authorizing medicines for rare diseases in Europe. Approvals across the European Union have been consistently high over the past five years.

Orphan Drugs Market Segmentation

• By Drug Type

  • Biological

  • Non-biological

• By Disease Type

  • Oncology

  • Hematology

  • Neurology

  • Cardiovascular

  • Others

• By Distribution Channel

  • Hospital

  • Pharmacy

  • Online sales

  • Others

Key players

• Novartis AG

• Pfizer Inc.

• Sanofi

• Bristol-Myers Squibb Company

• GSK plc

• Alexion Pharmaceuticals, Inc.

• AbbVie Inc.

• Merck & Co., Inc.

• Johnson & Johnson Private Limited

• F. Hoffmann-La Roche Ltd