Global Skeletal Dysplasia Market: Overview
Skeletal dysplasia is a rare disease with more than 450 types. It is also widely known as osteochondrodysplasias. It affects the growth and development of the bone and cartilage. The level of severity of this disorder varies from neonatal lethality to growth differences experienced in adulthood. Skeletal dysplasia is difficult to diagnosis, as it is a rare disorder. However, certain symptoms include slow growth, deformities in large head, short upper arms or thighs, curved bones, arthritis or joint pain, and others. The condition is highly prevalent in children and early diagnosis helps creating awareness. This condition is a result of defective genes, which are either inherited from parents or mutated during the development of fetus. The defective gene is extremely difficult to identify. Hence, companies are making significant investments in research & development of treatment for this rare disease.
Global Skeletal Dysplasia Market: Key Trends
Rise in awareness and early diagnosis of skeletal dysplasia drive the global skeletal dysplasia market. Technological advancements is another factor anticipated to propel the global market in the next few years. Key players are investing in the development of the treatment for this condition. Moreover, prevalence of skeletal dysplasia in children is increasing. These factors are expected to drive the global market. However, less awareness about skeletal dysplasia and poor diagnosis hamper the growth of the global market, especially in developing regions.
According to a journal published on UpToDate, prevalence of skeletal dysplasia is estimated to be 2.4 per 100,000 births globally every day. The prevalence of skeletal dysplasia estimated during pregnancy is 7.5 per 100,000 women screened through ultrasound. It is a rare disease; however, there are 450 different forms of skeletal dysplasia. Patients with this condition undergo extensive medical and surgical treatments throughout childhood and adulthood.
Global Skeletal Dysplasia Market: Segmentation
The global skeletal dysplasia market can be segmented based on type, treatment, end-user, and region. In terms of type, the market can be classified into achondroplasia, hypochondroplasia, thanatophoric dysplasia, osteogenesis imperfect, and others. Achondroplasia is the most common type of skeletal dysplasia. According to research, prevalence rate of achondroplasia is 5 in 100,000 and incidence is 1 in 20,000 live births. Based on treatment, the global skeletal dysplasia market can be categorized into medication, surgery, and others. There is no cure for the disorder; however, treatments are recommended for orthopedic and medical conditions caused by skeletal dysplasia. Medications include growth hormones, muscle relaxants, parathyroid hormones, and others. Surgical options include inserting drainage tubes to avoid chronic ear infections, tracheotomy, widening spinal canal process, and correcting physical deformities. In terms of end-user, the market can be divided into hospitals, ambulatory surgical centers, and others.
Global Skeletal Dysplasia Market: Regional Analysis
In terms of region, the global skeletal dysplasia market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America dominated the global market in 2018, and the trend is projected to continue during the forecast period. This is due to rise in awareness about the treatment of orthopedic and medical problems, increase in health care expenditure, and presence of key players involved in the clinical trials for development of therapies to cure skeletal dysplasia. Moreover, advanced health care facilities, awareness about early diagnosis, and recognition of orphan drugs by governments boost the growth of the skeletal dysplasia market in the U.S. According to research, the estimated prevalence of this rare bone disease is 1 in 1,600. In Europe, the rare disease affects a small population. It affects 30 million people, of which 6,800 have been identified. The skeletal dysplasia market in Asia Pacific is anticipated to grow at a rapid pace during the forecast period. Prevalence of rare disorders including skeletal dysplasia, early diagnosis, and prevalence in children are the factors fueling the growth of the market in the region. Expansion of national and international collaborations and technological advancements are expected to augment the skeletal dysplasia market in Asia Pacific.
Currently, there are no therapies for skeletal dysplasia. Major companies involved in the development of therapies in clinical trials include BioMarin Pharmaceuticals, Clementia Pharmaceuticals, Regeneron Pharmaceuticals, Ultragenyx Pharmaceutical, and Alexion Pharmaceuticals.
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.
2. Insights and Tools
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.
3. Actionable Results
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.