Fact.MR’s latest study reveals that the global rare disease treatment market size is forecasted to increase from a value of US$ 182.28 billion in 2024 to US$ 412.59 billion by the end of 2034. Market expansion has been projected at a CAGR of 8.5% from 2024 to 2034. Increasingly efficient diagnostic procedures, advancements in genetic and molecular research, and the development of novel therapeutic methods are among the key factors pushing the rare disease treatment market forward.

Personalized medicine, genomics, and biotechnology advancements are creating new avenues for the treatment of rare diseases. Favorable policies that encourage pharmaceutical companies to develop treatments for rare diseases include fast-track approvals and orphan drug designations. The use of multiple distribution channels, such as online pharmacies, specialty pharmacies, and hospital pharmacies, is driving up demand for rare disease treatment. This streamlines the development and commercialization processes.

Introduction of novel drugs and cutting-edge therapies, as well as the increase in rare disease cases and immunization campaigns, are all contributing factors to market expansion. Demand for rare disease treatments is not limited to developed countries but also extends to developing countries. The increasing involvement of businesses in the industry is one factor contributing to the rise in demand for treatments for rare diseases.

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Key Takeaways from the Market Study:

• The global market for rare disease treatment is projected to reach US$ 182.28 billion in 2024.
• By the end of 2034, the market is forecasted to climb to US$ 412.59 billion, expanding at a CAGR of 8.5% through 2024.
• The market in the United States is forecasted to reach a value of US$ 63.37 billion in 2024.
• Brazil occupies a 38% share of the Latin American market in 2024.
• Revenue from rare disease treatment in Canada is estimated at US$ 8.9 billion in 2024.
• The Latin American market is forecasted to expand at a CAGR of 8.6% through 2034.

“Increased research funding, advancements in personalized medicine, and supportive regulatory policies are fueling the growth of the rare disease treatment market. This is driving innovation and enhancing treatment accessibility,” says a Fact.MR analyst.

Originator Drugs Facilitating Targeted Treatment for Specific Inherited or Uncommon Disorders

Originator drugs are exclusive to the pharmaceutical company that discovered them and are covered by a patent. These drugs are used to treat uncommon illnesses for which there were no known effective treatments in the past. Rare diseases often have no effective treatment because they are not commonly observed. Originator medications bridge this gap by providing targeted treatment for specific inherited or uncommon disorders.

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More Valuable Insights on Offer

Fact.MR, in its new offering, presents an unbiased analysis of the rare disease treatment market for 2018 to 2023 and forecast statistics for 2024 to 2034.

The study divulges the rare disease treatment market based on therapeutic area (hematology, genetic diseases, neurological diseases, oncology, infectious diseases, inflammatory diseases, metabolic diseases, pulmonary diseases, kidney diseases, musculoskeletal diseases, endocrine diseases, topical diseases), drug type (originator drugs, generic drugs), drug class (biologics, organic compounds), mode of administration (injectables, oral), and distribution channel (hospital pharmacies, specialty treatment pharmacies, retail pharmacies), across six major regions of the world (North America, Latin America, Europe, East Asia, South Asia & Oceania, and MEA).

Key Companies Profiled:

Novartis AG; AbbVie; Bristol Myers Squibb; BioMarin; Amgen Inc.; PTC Therapeutics; AstraZeneca; GlaxoSmithKline Plc; Amgen Inc.; Pfizer Inc.; Johnson & Johnson

Industry News:

As per the Canadian Organization for Rare Disorders (CORD) Report 2021, approximately 1 in 12 individuals in Canada is impacted by rare diseases annually, with approximately 80% attributed to genetic mutations. Due to the substantial prevalence of rare diseases in the country, demand for effective treatment solutions is projected to rise over the forecast period.

• A study reported that as of May 2022, 30 million Americans, or 1 in 10 of the population, suffered from a rare disease, and there are about 7,000 known rare diseases, according to the Genetic and Rare Diseases (GARD) Information Center.
• In November 2023, the Union Health Ministry of India introduced generic medications to support the care and treatment of four conditions: Wilson's disease, Gaucher's disease, Tyrosinemia-Type 1, and Dravet- Lennox-Gastaut syndrome. This initiative aims to alleviate the burden on patients with rare diseases across India, offering these medications at a fraction of their current market prices, ranging from 60 to 100 times less expensive.

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