North America Rare Disease Therapeutics Market to Grow with a CAGR of 10.88% through 2028
Increase in the
incidence of Rare Disease and increase in the demand of biologics drugs is
expected to drive the North America Rare Disease Therapeutics Market growth in
the forecast period, 2024-2028.
According to
TechSci Research report, “North America Rare Disease Therapeutics Market – Industry
Size, Share, Trends, Competition Forecast & Opportunities, 2028”,
the North America Rare Disease Therapeutics Market stood at USD 40.38 billion
in 2022 and is anticipated to grow with a CAGR of 10.88% in the forecast
period, 2024-2028. The awareness among individuals regarding Rare Disease
Therapeutics has led to favorable market conditions for the North America Rare
Disease Therapeutics market. Several factors contribute to the growth of
various Rare Disease Therapeutics products.
The shift
towards precision medicine is increasingly emphasizing personalized treatments
that cater to the unique genetic profiles of individual patients. This trend is
rooted in the acknowledgment that rare diseases can exhibit varying
manifestations among patients, even when stemming from the same genetic
mutation. Personalized therapies are designed to optimize effectiveness while
minimizing potential side effects. Additionally, there is a growing prevalence
of biomarkers, such as genetic indicators or specific protein levels, in the
realm of rare disease diagnosis. These biomarkers play an expanding role in
early disease detection, patient classification, and the monitoring of
treatment responses, thereby streamlining more efficient and timely
interventions.
Rare hematologic
diseases encompass a diverse spectrum of conditions, including different forms
of anemia, hemophilia, as well as specific uncommon blood cancers like multiple
myeloma and certain types of leukemia. The prevalence of these rare hematologic
disorders is a key factor in elevating the importance of this segment. Given
the intricacy of hematologic diseases and the difficulties in their diagnosis
and treatment, there is a significant focus on intensive research and the development
of novel therapies within this field, which will further fuel market growth.
In May 2023,
Amgen announced that it had submitted a new drug application (NDA) to the US
Food and Drug Administration (FDA) for its investigational gene therapy
asfotase alfa for the treatment of hypophosphatasia (HPP). HPP is a rare
genetic disorder that affects the body's ability to use and store phosphorus.
Asfotase alfa is designed to replace the missing enzyme that causes HPP.
Gene therapy has
emerged as a groundbreaking approach in the treatment of certain rare diseases
caused by single-gene mutations. These therapies involve the delivery of
functional copies of the defective gene or the correction of the mutated gene
itself, and growing awareness of the health benefits of gene therapy are
expected to positively impact the North America Rare Disease Therapeutics
market.
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In today's
fast-paced world, Rare disease therapeutics are often precision medicines,
tailored to the specific genetic or molecular abnormalities underlying these
conditions. This approach allows for highly targeted and efficient treatments,
minimizing trial and error and optimizing patient outcomes. Advanced
diagnostics and genetic testing are increasingly used in rare disease diagnosis.
This speeds up the identification of rare diseases, allowing for earlier
interventions and reducing the time patients spend searching for answers. The
development of rare disease therapies is increasingly patient-centric. Patients
and their advocates are actively involved in research, clinical trials, and
treatment decisions. This collaborative approach ensures that therapies meet
the unique needs and preferences of individuals. Rare disease research and
therapeutics benefit from international collaboration. Researchers, healthcare
professionals, and pharmaceutical companies work across borders to advance
knowledge and share best practices, accelerating progress in the field. Rare
disease therapeutics often leverage cutting-edge technologies, such as gene
editing and cell therapies. These innovations are not only transforming rare
disease treatment but also contributing to broader advancements in medical
science.
The growing
awareness among consumers regarding health and wellness has shifted the focus
towards the ideology of "prevention is better than cure." As a
result, more individuals are relying on nutraceuticals, functional foods, and
dietary and Rare Disease Therapeutics to lead a healthy and disease-free life.
In recent times,
advancements in medical science, particularly in genomics and molecular
biology, have enabled the development of precision medicines tailored to the
specific genetic or molecular abnormalities underlying rare diseases. This
targeted approach enhances treatment effectiveness while minimizing side
effects. In recent years, there has been a growing emphasis on early diagnosis
of rare diseases through improved diagnostics and genetic testing. This early
detection allows for timelier interventions and reduces the diagnostic odyssey
that patients often endure. The era of personalized medicine has dawned, with
rare disease therapeutics leading the way. These treatments are customized to
individual patients based on their unique genetic profiles, optimizing outcomes
and minimizing adverse effects. However, the Small Target Populations may
hinder market growth. Moreover, challenges related to Research and Development
Challenges and Disease Heterogeneity may pose obstacles to the Rare Disease
Therapeutics market in the near future.
The North
America Rare Disease Therapeutics Market is segmented into therapeutics area, route
of administration, drug type, distribution channel, country distribution, and
company.
Based on its therapeutics
area, the Hematologic rare diseases encompass a wide range of disorders,
including various types of anemia, hemophilia, and certain rare blood cancers
like multiple myeloma and certain types of leukemia. The prevalence of these
rare hematologic disorders contributes significantly to the segment's
prominence. Hematologic diseases can be complex and challenging to diagnose and
treat, making them an area of intensive research and development for innovative
therapies. Many rare hematologic diseases have limited treatment options
available. Patients with these conditions often have unmet medical needs,
creating a strong demand for novel and effective therapeutics. The scarcity of
treatments for specific hematologic rare diseases can lead to market
exclusivity for companies that develop effective therapies, providing a competitive
advantage. Hematologic rare diseases often qualify for orphan drug designation,
which comes with incentives such as extended market exclusivity, tax credits,
and research grants. These incentives encourage pharmaceutical companies to
invest in research and development for rare hematologic disease therapies.
Advances in biotechnology, including gene therapies and precision medicine
approaches, have revolutionized the treatment landscape for rare hematologic
diseases.
Based on region,
United States segment is expected to grow during the forecast
period. The United States boasts a highly developed healthcare
infrastructure, including renowned medical institutions, specialized treatment
centers, and a well-established pharmaceutical industry. This infrastructure is
well-suited for the diagnosis, treatment, and management of rare diseases. The
U.S. is a North America hub for biopharmaceutical research and development,
with numerous pharmaceutical and biotech companies dedicated to rare disease
therapies. This has resulted in a robust pipeline of innovative treatments. The
U.S. has a favorable regulatory environment that incentivizes the development
of orphan drugs for rare diseases. These incentives, such as extended market
exclusivity and research grants, encourage investment in rare disease
therapeutics. The U.S. has a sizable population, including patients with rare
diseases. This large patient pool creates a significant market for rare disease
therapeutics. The U.S. has a strong network of patient advocacy groups and rare
disease organizations that actively support research, raise awareness, and
advocate for improved access to treatments. Health insurance coverage in the
United States often includes rare disease therapies, making these treatments more
accessible to patients.
The Canada
market is poised to be the fastest-growing market, offering lucrative growth
opportunities for Rare Disease Therapeutics players during the forecast period.
Factors such as Canada is increasingly becoming a destination for
biopharmaceutical research and development. The country offers incentives for
clinical trials and innovative drug development, making it an attractive
location for rare disease research. Awareness of rare diseases and their
treatment options is on the rise in Canada. This awareness is driving demand
for rare disease therapies. The Canadian government has been investing in
healthcare infrastructure and research, including rare disease research. This
investment is expected to boost the availability of rare disease treatments.
Canada's proximity to the United States allows for collaboration and access to
the extensive U.S. rare disease therapeutics market, contributing to growth
potential. Canada has an active rare disease patient community, advocating for improved
access to treatments, which can further drive market growth.
Major
companies operating in North America Rare Disease Therapeutics Market
are:
- 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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“The scientific
advancement and increasing awareness and diagnosis are key drivers of the Rare
Disease Therapeutics market. In recent years, Active involvement of patient
advocacy groups in research, awareness campaigns, and policy advocacy drives
progress in rare disease therapeutics. Moreover, the growing consumer emphasis
on preventative healthcare products has enhanced production and marketing
efforts. To meet the demands of the market, manufacturers are increasingly
incorporating cutting-edge technologies with high production efficiency. Rising
consumer acceptance and continuous product innovation will further ensure the
growth of the Rare Disease Therapeutics and supplements market in the coming
years.,” said Mr. Karan Chechi, Research Director with TechSci Research, a
research-based management consulting firm.
“North America
Rare Disease Therapeutics Market Segmented By Therapeutics Area (Hematologic
Diseases, Cancer, Infectious Diseases, Cardiovascular Diseases, Metabolic
Diseases, Endocrine Diseases, Musculoskeletal Diseases, Others), By Route of
Administration (Injectable, Oral, Others), By Drug Type (Biologics, Biosimilar,
Small Molecules), By Distribution Channel (Specialty Pharmacies, Hospital
Pharmacies, Online Pharmacies), By Country, Competition, Forecast &
Opportunities 2018-2028”, has evaluated the future growth potential of North
America Rare Disease Therapeutics Market and provides statistics &
information on market size, structure and future market growth. The report
intends to provide cutting-edge market intelligence and help decision makers
take sound investment decisions. Besides, the report also identifies and
analyzes the emerging trends along with essential drivers, challenges, and
opportunities in North America Rare Disease Therapeutics Market.
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