North America Rare Disease Therapeutics Market Set for Significant Growth, with Projected 10.88% CAGR Through 2028

Dublin, March 20, 2024 (GLOBE NEWSWIRE) -- The "North America Rare Disease Therapeutics Market, Competition, Forecast & Opportunities, 2018-2028" report has been added to's offering.

The North America Rare Disease Therapeutics Market is experiencing a period of significant growth, with an expected Compound Annual Growth Rate (CAGR) of 10.88% through 2028. This market, which includes a dynamic ensemble of treatments tailored for rare and orphan diseases, has reached a value of USD 40.38 billion as of 2022.

The market is benefitting from a combination of factors, including an increase in the prevalence of rare diseases, a supportive regulatory environment, advancements in biotechnology and pharmacology, and a burgeoning patient advocacy movement.

Driving Factors Behind Market Growth

The market is fueled by several key drivers, chief among them being the increasing prevalence and awareness of rare diseases. The advancements in genetic testing and precision medicine contribute substantially to the diagnosis and treatment of these rare conditions. The supportive regulatory environment in North America, with incentives for drug development under the U.S. Orphan Drug Act, such as market exclusivity, tax credits, and prioritized review, further stimulates the growth of this specialized therapeutics sector.

Investments in biotech innovations have also catalyzed the development of gene and cell therapies, which hold promise for addressing the unmet medical needs of rare disease patients. Additionally, the concerted efforts of patient advocacy groups have played a crucial role in promoting research and ensuring access to necessary treatments.

Challenges to Tackle

Despite significant growth drivers, the Rare Disease Therapeutics Market faces challenges, including a limited patient pool for clinical trials, the high cost of drug development, and challenge regarding patient accessibility and affordability of treatments. Companies operating within this sector continue to work on overcoming these barriers to translate scientific advances into therapies that reach patients in need.

Emerging Market Trends

Trends shaping the future of the Rare Disease Therapeutics Market include the rise of precision medicine, with increasingly personalized treatment strategies. The development of gene and cell therapies also exemplifies the innovative approaches making inroads in the treatment of rare diseases. Patient-centric care has become the focus, with a significant rise in advocacy and community engagement driving market developments. The integration of advanced genomics into diagnostics and treatments further exemplifies the shift towards more individualized therapies. The use of biomarkers is also increasing, enabling more accurate diagnosis and monitoring of rare diseases.

Leadership Role of the United States

Within North America, the United States holds the lion's share of the Rare Disease Therapeutics Market. With its robust healthcare infrastructure, extensive biopharmaceutical R&D activities, and favorable regulatory environment, the U.S. is at the forefront of this market's expansion.

Rapid Expansion in Specific Market Segments

  • The Hematologic Diseases segment dominates the market, propelled by the prevalence and intensity of research into these conditions.
  • Injectable Therapies maintain their lead due to their suitability for the delivery of targeted biologics and gene therapies.
  • The Biologics segment leads drug type categories with its precision-targeted treatment capabilities.
  • Specialty Pharmacies are gaining ground due to their expertise in rare disease medications and support services.

Canada: Fastest-Growing Market with R&D Incentives

Canada shows potential for rapid growth in the Rare Disease Therapeutics Market. Contributing factors include increased R&D activity, heightened rare disease awareness, and concerted efforts in healthcare investment, all of which provide fertile ground for market expansion.

Specialty Pharmacies to Spearhead Distribution

Forecasted to grow significantly, specialty pharmacies are set to play a pivotal role in the distribution of rare disease therapeutics, underpinned by their specialized services and patient-centric model.

In summary, the North America Rare Disease Therapeutics Market is set to witness considerable growth, propelled by advancements in precision medicine, strong regulatory support, and the enduring commitment of the rare disease community. This growth trajectory highlights the market's critical role in addressing the complex healthcare needs of rare disease patients across North America.

Key Attributes

Report AttributeDetails
No. of Pages120
Forecast Period2022 - 2028
Estimated Market Value (USD) in 2022$40.38 Billion
Forecasted Market Value (USD) by 2028$74.27 Billion
Compound Annual Growth Rate10.8%
Regions CoveredNorth America

A selection of companies mentioned in this report includes

  • Amgen Inc
  • BioMarin Pharmaceutical Inc
  • Alexion Pharmaceuticals Inc
  • Spark Therapeutics Inc
  • Ultragenyx Pharmaceutical Inc
  • Vertex Pharmaceuticals Inc
  • Novartis AG
  • Sanofi SA
  • Bristol-Myers Squibb Co
  • Bayer AG

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