The growing demand for rare genetic disorders treatments can be attributed due to the surge in the prevalence of rare cancers and other diseases. According to an article shared by the American Cancer Society, in the US in January 2022, there are approximately 54,000 new cases of oropharyngeal or oral cancer each year. Oropharyngeal or oral cancer claims the lives of 11,230 people. Moreover, rising healthcare expenditure is also acting as major driver for the growth of the market in region.
Further,the North AmericaRare Genetic Disorders Treatment Marketis expected to grow at a strong CAGR of 11.5% during the forecast period (2022-20230).North America held a dominant share of the market in 2021 as the regional market that is focused on providing innovative therapies and treatments for individuals with rare genetic disorders. Rare genetic disorders are conditions that affect a small number of individuals, typically less than 200,000 people in the US. The market for rare genetic disorders treatment in the US is primarily driven by the increasing prevalence of rare genetic disorders, advancements in technology and research, and the growing demand for personalized medicine. According to the National Institutes of Health, there are over 7,000 known rare genetic disorders affecting an estimated 25 to 30 million people in the US. Moreover, presence of key players and rising investment is also driving the regional growth. For instance, in January 2021, Roche announced a collaboration with Sarepta Therapeutics to develop and commercialize therapies for Duchenne muscular dystrophy (DMD) and other rare neuromuscular diseases.
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FIG. 1 North America Rare Genetic Disorders Treatment Market Revenue (2020-2030)- USD Mn
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Based on that is focused on providing innovative therapies and treatments for individuals with rare genetic disorders. Rare genetic disorders are conditions that affect a small number of individuals, typically less than 200,000 people in the US. The market for rare genetic disorders treatment in the US is primarily driven by the increasing prevalence of rare genetic disorders, advancements in technology and research, and the growing demand for personalized medicine. According to the National Institutes of Health, there are over 7,000 known rare genetic disorders affecting an estimated 25 to 30 million people in the US. Moreover, presence of key players and rising investment is also driving the regional growth. For instance, in January 2021, Roche announced a collaboration with Sarepta Therapeutics to develop and commercialize therapies for Duchenne muscular dystrophy (DMD) and other rare neuromuscular diseases.
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By drug type, the market is bifurcated into biologics biosimilars, and non-biologics. The biologics biosimilar segment held a dominant share of the market in 2021 owing to their high specificity towards the disease. Biologics biosimilars drugs are increasingly being used for the treatment of rare genetic disorders, including hematology diseases. Biologics are drugs that are derived from living cells, and include proteins, antibodies, and other complex molecules. Biosimilars are similar to biologics, but are developed after the patent for the original biologic drug has expired. Several biologics have beend approved for the management of rare genetic disorders including Rituximab, Adalimumab, zynteglo, and others. Apart from this, rising product pipelines and their encouraging response in clinical trials is also suggesting positive future growth of this segment. For instance, in 2020, Regenreon Pharmaceuticals announced encouraging results from LUMINA-1, a 44-patient, Phase 2, double-blind placebo-controlled trial evaluating garetosmab (REGN2477) in patients with fibrodysplasia ossificans progressiva (FOP).
On the basis of distribution channel, the market is segmented into hospital pharmacy, online pharmacy, and retail pharmacy. The online pharmacy is expected to witness high CAGR during the forecast period. Online pharmacies are also gaining considerable momentum in the market owing to their convenience. Consumers can order medications from the comfort of their own homes and have them delivered directly to their doorsteps. This is especially beneficial for those who live in rural areas or have mobility issues, as it eliminates the need for them to travel to a physical pharmacy. Moreover, online pharmacies often offer lower prices than traditional brick-and-mortar pharmacies due to lower overhead costs. In addition, online pharmacies may offer discounts, promotions, and loyalty programs to attract and retain customers. According to a survey conducted by the National Community Pharmacists Association, 61% of respondents said that cost was the primary reason they chose to use an online pharmacy.
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Global Rare Genetic Disorders Treatment Market Segmentation
Market Insight, by Application
Cancer
Neurological Disorders
Cardiovascular Diseases
Metabolic Disorders
Hematology Diseases
Others
Market Insights, by Drug Type
Biologics Biosimilars
Non-Biologics
Market Insights, by Distribution Channel
Hospitals Pharmacy
Online Pharmacy
Retail Pharmacy
Market Insight, by Region
North America
o U.S.
o Canada
o Rest of North America
Europe
o Germany
o U.K.
o France
o Spain
o Italy
o Rest of Europe
APAC
o China
o Japan
o India
o Rest of APAC
Rest of the World
Top Company Profiles
F. Hoffmann-La Roche Ltd.
Pfizer, Inc.
AstraZeneca
Bristol Myers Squibb
Takeda Pharmaceutical Company Limited
Novartis AG
AbbVie Inc.
GlaxoSmithKline Plc
Amgen Inc.
Johnson Johnson Inc.
