Rare Diseases Treatment Market to Rise High at US$346.5 Bn by 2029 End, Projects Fairfield Market Research

Fairfield Consultancy Services OPC Pvt Ltd
Fairfield Consultancy Services OPC Pvt Ltd

London, Oct. 03, 2022 (GLOBE NEWSWIRE) -- Projected to exhibit robust 10.4% growth between 2022 and 2029, global rare diseases treatment market will possibly reach the revenue of around US$346.5 Bn toward the end of forecast year. The expediting pace of orphan drug development is expected to predominantly drive the growth of rare diseases treatment market in addition to an increasing number of promotional and awareness programs around rare and orphan diseases, and growing advocacy efforts. However, the report also highlights that the higher treatment costs, limited cure, lack of specialized treatment centres, poor check on drug prices, and deficient regulatory and legal marks will remain the major growth impediments facing rare diseases treatment market, besides the lack of awareness.

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Key Research Insights

  • The global valuation of rare diseases treatment market exceeded US$155 Bn in 2021

  • The US market will continue to spearhead, at an estimated CAGR of 10% through 2029

  • Oncology has been the dominant market segment, accounting for more than 48% revenue share

Insights into Segmental Insights

The report indicates that the rare oncology segment continues to lead the pack with a substantial chunk in the overall revenue generation of orphan drugs. While this category accounted for more than 48% revenue share in the year 2019, the trend will prevail throughout the period of forecast. The study attributes this to the considerably high prevalence of rare cancers that roughly make up 27% of all the cancers. Rare diseases treatment market will thus remain influenced by the early-stage investments that will most likely lead to life-changing outcomes for a large patient population.

Key Report Highlights

  • Against non-biologics, the biologics are expected to represent the dominant market category

  • The consistently soaring number of rare disease patients, and the significant rise in overall orphan designations will raise the number of drug and biologics approvals

  • Paediatric orphan drug development has been facing challenges during trials. However, regulatory initiatives, and favourable public health policies are likely to facilitate the same

Insights into Regional Analysis

The report suggests that the US will most likely remain the dominant regional market for rare diseases treatment throughout the period of forecast. Clearly attributing to the pioneering status of the US in orphan drug development, the US market will continue to reign supreme. On the other hand, developing markets across Asia Pacific are likely to develop a lucrative market for rare diseases treatment providers. India, and some of the southeast Asian countries represent a sizeably large patient population of rare disease(s). As the legislative and regulatory enactment regarding rare diseases and orphan drugs has seen considerable progress over the recent past, the region is expected to witness an influx of opportunities in the near future, especially in India, China, Japan, Taiwan, and South Korea.

Key Players in Global Rare Diseases Treatment Market 

Novartis, Bristol Myers Squibb, Johnson & Johnson, Abbvie, Roche, Vertex Pharmaceuticals, Sanofi, Takeda, Pfizer, and Astra Zeneca constitute the top 10 players steering the global rare diseases treatment market competition.

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Report Attributes


Base Year



Forecast Year

2022 – 2029

Market Size in 2021

US$155.4 Bn

Estimated Market Size in 2029

US$346.5 Bn




Key Players

Novartis, Bristol Myers Squibb, Johnson & Johnson, Abbvie, Roche, Vertex Pharmaceuticals, Sanofi, Takeda, Pfizer, and Astra Zeneca

Market Segmentation

Therapeutic Area

  • Oncology

  • Hematology

  • Neurology

  • Respiratory

  • Cardiovascular

  • Other


  • Biologics

  • Non-biologics

Patient Type

  • Adults

  • Paediatrics

  • Geriatrics

Leading Companies

  • Bristol Myers Squibb

  • Bayers AG

  • Johnson & Johnson

  • Novartis AG

  • Takeda Pharmaceutical Company Limited

  • Sanofi

  • F. Hoffmann-La Roche Ltd

  • Astra Zeneca

  • AbbVie

  • Vertex Pharmaceuticals

  • Pfizer Inc.

  • Novo Nordisk A/S

  • BioMarin Pharmaceuticals Inc.

  • Others

Report Inclusions

  • Market Estimates and Forecast

  • Market Dynamics

  • Industry Trends

  • Competition Landscape

  • Therapeutic Area-wise Analysis

  • Type-wise Analysis

  • Patient Type-wise Analysis

  • Region-wise Analysis

  • Country-wise Analysis

  • Key Trends Analysis

  • COVID-19 Impact Analysis

About Us

Fairfield Market Research is a UK-based market research provider. Fairfield offers a wide spectrum of services, ranging from customized reports to consulting solutions. With a strong European footprint, Fairfield operates globally and helps businesses navigate through business cycles, with quick responses and multi-pronged approaches. The company values an eye for insightful take on global matters, ably backed by a team of exceptionally experienced researchers. With a strong repository of syndicated market research reports that are continuously published & updated to ensure the ever-changing needs of customers are met with absolute promptness.


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