Severe Combined Immunodeficiency Treatment (SCID) Market: Introduction
- Severe combined immunodeficiency (SCID) is a group of rare disorders caused by mutation in different genes. These genes are responsible for the development and functioning of the immune system. These disorders are life-threatening or fatal usually in the first two years of a child’s life. These can be treated through immune restoring treatments, which include gene therapy, hematopoietic stem cell therapy (HSCT or bone marrow transplant), and enzyme therapy. Among these, HSCT has proven to be the only treatment that completely cures SCID in infants; however, other therapies are introduced regularly for the treatment of this disorder. Gene therapy is still under experimentation. Nevertheless, it is considered where bone marrow transplant is not advised for a patient. Around 80% of infants affected by SCID do not have a background or family history of the disorder. Early diagnosis or screening of the disorder can ensure that infants receive lifesaving SCID treatment. According to the Immune Deficiency Foundation, 13 different genetic defects can cause severe combined immunodeficiency in infants, leading to serious infections. According to scientists, gene therapy offers promising treatment for this disorder. ADA-SCID is a rare inherited disorder, also known as bubble syndrome, caused by mutation in the adenosine deaminase (ADA) gene. This type of SCID is the second most prevalent form of the disorder.
- Screening or early diagnosis plays an important role in saving the lives of infants. Addition of the IDF SCID Newborn Screening Campaign in the newborn screening panel of the U.S. resulted in higher awareness about the diagnosis of SICD. The Immune Deficiency Foundation has commended the Secretary of Health and Human Services for the inclusion of SCID new born screening obligatory in every state of the U.S. effective from December 2018.
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Key Drivers and Restraints of Global Severe Combined Immunodeficiency Treatment Market
- Severe combined immunodeficiency are rare forms of genetic disorders that can be fatal for an infant if not treated promptly. Rise in awareness about screening and diagnosis of these disorders is projected to augment the global severe combined immunodeficiency treatment market. Availability of drugs and immune-related treatments are anticipated to drive the global market. Technological advancements in the screening technique is expected to boost the growth of the global severe combined immunodeficiency treatment market. Adoption of stem cell therapy, gene therapy, and other therapies is also likely to propel the global market. Moreover, antibody infusion and bone marrow transplant are commonly recommended for the treatment of children with severe combined immunodeficiency.
- Continuous investment in research & development on the diagnosis and treatment of these disorders, including gene therapy, is projected to fuel the growth of the global severe combined immunodeficiency treatment market. High investments by key players and initiatives by governments for research & development on the treatment of severe combined immunodeficiency are anticipated to propel the global severe combined immunodeficiency treatment market. According to the National Organization for Rare Disorders, Inc. (NORD), severe combined immunodeficiency is quite rare and occurs in approximately 1 or fewer newborns in 100,000 in the U.S. Leadiant Biosciences’ innovative enzyme replacement therapy, Revcovi, was approved by the FDA for the treatment of severe combined immunodeficiency in October 2018. However, low awareness, low diagnosis or screening rate, and higher treatment cost for this rare disorder act as major restraints of the global severe combined immunodeficiency treatment market.
North America to Lead Global Severe Combined Immunodeficiency Treatment Market
- In terms of region, the global severe combined immunodeficiency treatment market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa
- North America dominated the global severe combined immunodeficiency treatment market owing to faster adoption of stem cell therapy and increase in approval of treatment for these disorders. Advanced health care system and rise in awareness about improved screening methods in the U.S. are expected to augment the market in the region. Mandatory screening of newborns for severe combined immunodeficiency in the U.S. is spreading awareness about the disease. For instance, as of December 2018, all 50 states in the U.S. now screen infants for SCID. Incidence of ADA-SCID is estimated to be 1 in 100,000 to 200,000 infants in the country.
- Europe was the second largest market for severe combined immunodeficiency treatment in 2018, followed by Asia Pacific. Clinical trials by key players and availability of advanced treatment for SCID are likely to augment the market in Europe. In 2017, the European Commission approved GSK’s ex vivo hematopoietic stem cell (HSC) gene therapy and Strimvelis products for the treatment of SCID. In 2015, around 2,910 prevalent cases ADA-SCID were identified in seven markets (the U.S., France, Germany, Italy, Spain, the U.K. and Japan). Moreover, the incidence of ADA-SCID in the U.K. is approximately 1 in 375,000 to 660,000 births. Asia Pacific is projected to be the fastest growing market for severe combined immunodeficiency treatment, followed by Latin America and Middle East & Africa. Developing health care facilities, rise in awareness about the screening of this rare disease, and investments in clinical trials are anticipated to augment the severe combined immunodeficiency treatment market in Asia Pacific.
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Key Manufacturers Operating in Global Severe Combined Immunodeficiency Treatment Market
The global severe combined immunodeficiency treatment market is consolidated, with only a small number of players offering treatments for severe combined immunodeficiency. Key players offering severe combined immunodeficiency treatments are:
- Leadiant Biosciences
- GlaxoSmithKline plc
Global Severe Combined Immunodeficiency Treatment Market: Research Scope
Global Severe Combined Immunodeficiency Treatment Market, by Disease Type
- X-linked SCID
Global Severe Combined Immunodeficiency Treatment Market, by Treatment
- Stem Cell Therapy
- Gene Therapy
- Enzyme Therapy
Global Severe Combined Immunodeficiency Treatment Market, by End-user
- Ambulatory Surgical Centers
Global Severe Combined Immunodeficiency Treatment Market, by Region
- North America
- Rest of Europe
- Asia Pacific
- Australia & New Zealand
- Rest of Asia Pacific
- Latin America
- Rest of Latin America
- Middle East & Africa
- GCC Countries
- South Africa
- Rest of Middle East & Africa
This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.
Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:
- Customer Experience Maps
- Insights and Tools based on data-driven research
- Actionable Results to meet all the business priorities
- Strategic Frameworks to boost the growth journey
The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.
The following regional segments are covered comprehensively:
- North America
- Asia Pacific
- Latin America
- The Middle East and Africa
The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.
Below is a snapshot of these quadrants.
1. Customer Experience Map
The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.
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The various insights in the study are based on elaborate cycles of primary and secondary research the analysts engage with during the course of research. The analysts and expert advisors at TMR adopt industry-wide, quantitative customer insights tools and market projection methodologies to arrive at results, which makes them reliable. The study not just offers estimations and projections, but also an uncluttered evaluation of these figures on the market dynamics. These insights merge data-driven research framework with qualitative consultations for business owners, CXOs, policy makers, and investors. The insights will also help their customers overcome their fears.
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The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.
4. Strategic Frameworks
The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.
The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:
1. What can be the best investment choices for venturing into new product and service lines?
2. What value propositions should businesses aim at while making new research and development funding?
3. Which regulations will be most helpful for stakeholders to boost their supply chain network?
4. Which regions might see the demand maturing in certain segments in near future?
5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?
6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?
7. Which government regulations might challenge the status of key regional markets?
8. How will the emerging political and economic scenario affect opportunities in key growth areas?
9. What are some of the value-grab opportunities in various segments?
10. What will be the barrier to entry for new players in the market?
Note: Although care has been taken to maintain the highest levels of accuracy in TMR’s reports, recent market/vendor-specific changes may take time to reflect in the analysis.