Rare Disease Treatment Market | Regional Growth Forecast-2027

Rare Disease Treatment Market | Regional Growth Forecast-2027

The global rare disease treatment market is poised to gain massive traction over the upcoming years on account of increasing cases of rare diseases across the globe. As per the National Institutes of Health, nearly 30 million residents of the U.S. suffer from one of the 7,000 known rare disorders and out of which only 5% of disorders have treatments. Moreover, as new rare genetic disorders are cropping up, research on rare disorders has significantly increased over the last few years which is helping in the development of various disease specific therapeutic drugs for rare disorders, thereby positively influencing the market growth. According to the Global Market Insights Inc. report, rare disease treatment market size is estimated to surpass USD 317 billion by 2026.

Citing an instance regarding the development of rare disease drugs, in 2019, Pfizer Inc. received FDA approval for two of its solutions namely VYNDAQEL® and VYNDAMAX™ for the treatment of a rare hereditary transthyretin-mediated amyloidosis (ATTR-CM) disease in adults. According to the company, these drugs can help in reducing cardiovascular mortality hospitalizations.

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Prominent players operating in the industry such as Bayer AG, AbbVie Inc., Alexion Pharmaceuticals, Inc., Celgene Corporation, Johnson & Johnson Services, Inc., Novartis AG, Pfizer Inc, Sanofi, etc. are undertaking strategic initiatives such as acquisitions, mergers, collaborations and partnerships in order to bolster their market position. For instance, in 2019, Pfizer Inc. announced the acquisition of Vivet Therapeutics for developing VTX-801 for the treatment of Wilson’s disease. This strategic move assisted the company in expanding its product portfolio and gain a competitive edge in the industry.


The biologics segment is projected to register a notable CAGR of over 10% through 2026. This is majorly due to the higher effectivity of biological drugs as compared to other drugs. Biological drugs are capable of providing precise and effective treatment to the patients by implementing gene therapy. Further, the cost of various biologic drugs is covered by medical insurance, thereby providing the patients with easier access to effective treatment procedures.

The musculoskeletal segment is likely to grow exponentially over the subsequent years on account of the rising need for effective therapies for musculoskeletal disorders which are the leading cause of disability around the globe. This has urged several research and development institutions and biopharmaceutical companies to collaborate for the development of new effective drugs. Besides, the increasing need for better management of adult patients with rare musculoskeletal disorders is also stimulating the development of novel therapies that will foster the segment growth further. For the records, the segment had secured a business share of USD 5.4 billion in 2019 and is estimated to grow profusely over the span of the upcoming six years.    

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The pediatric segment is anticipated to witness a substantial growth rate of 12.5% through 2026 considering the rising prevalence of rare diseases among pediatric patients. According to credible sources, the majority of the patients with rare diseases are children as 70% of these disorders show pediatric onset. Pediatric patients suffering from rare disorders are more susceptible to face late diagnosis, life-threatening challenges and fewer approved treatment procedures which provide a wider scope for the development of new treatment for rare diseases which in turn will complement the segment growth in the coming years.

The oral route of administration segment had recorded an appreciable valuation of over USD 80.2 billion in 2019 and is projected to grow over time owing to increased consumer inclination towards oral administration route for drug consumption as it is non-invasive, convenient and cost-effective in comparison with injectable administration. Moreover, it offers ease of ingestion and more patient safety and does not lead to pain due to which the adoption of this route of administration is increasing considerably among the patient population, leading to the development of new oral drugs for rare diseases.

Brazil had dominated the LATAM rare disease treatment industry in the past and is expected to observe a significant CAGR of 12.8% through 2026. This can be credited to the enhanced public healthcare system in the country. As per an article published in Orphanet Journal of Rare Diseases, at present, over 25% of Brazil’s population has health insurance coverage. This provides the patient population with easy access to available diagnostic procedures and treatment of rare diseases. As a result, major companies in the country are focusing on developing novel therapeutic drugs.

Moreover, government authorities in the country are also undertaking several initiatives focusing on rare diseases treatment. For instance, the Brazilian Health Authorities (ANVISA) has formulated a procedure for approving clinical trials pertaining to the diagnosis, treatment and prevention of rare diseases which is expected to have a positive impact on the industry growth in the years to follow.

Partial Chapter of the Table of Content

Chapter 2   Executive Summary

2.1    Rare disease treatment industry 3600 synopsis, 2015 - 2026 (USD Million)

2.1.1    Business trends

2.1.2    Drug type trends

2.1.3    Therapeutic area trends

2.1.4    Patient trends

2.1.5    Route of administration trends

2.1.6    Regional trends

Chapter 3   Rare Disease Treatment Industry Insights

3.1    Industry segmentation

3.2    Industry landscape, 2015 - 2026 (USD Million)

3.3    Industry impact factors

3.3.1    Growth drivers    Increase in number of rare disease cases across the globe    Favourable government policies    Growing number of new drug launches    Increasing R&D activities for novel therapeutics and drugs

3.3.2    Industry pitfalls & challenges    Lack of awareness regarding rare disease treatment    High cost of the drugs and therapy

3.4    Growth potential analysis

3.4.1    By drug type

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