Amyloid is an abnormal protein produced in the bone marrow. Amyloidosis is a rare disease caused by extracellular and intracellular deposition of amyloid which alters the normal activities of the tissue. Commonly affected organs by amyloidosis encompass kidney, heart, nervous system, digestive tract, liver and spleen. Severe form of amyloidosis in later stages can lead to permanent organ failure and in extreme cases can be life-threatening also. There are over 30 different types of amyloid proteins discovered so far and treatment can only help in managing the symptoms and regulating the production of amyloid protein and not the permanent cure. About 90% of the amyloid formed due to the aggregation of misfolded proteins whereas, 10% of amyloidosis is due to presence of components such as apolipoprotein-E (apoE), glycosaminoglycans (GAGs) and serum amyloid P-component (SAP). According to American Society of Hematology the prevalence rate of amyloidosis in the United States is approximately 1 in 90,666 of which 60 – 65 % of the patient are men. Prominent symptoms of the disease include fatigue, weight loss, breathlessness, peripheral oedema, carpal tunnel syndrome, postural hypotension and sensory change. In the United Kingdom systemic amyloidosis is responsible for approximately one in 1,200 deaths each year.
Amyloidosis can be broadly classified into 3 major categories namely AA amyloidosis, AL amyloidosis and Transthyretin amyloidosis. In general amyloidoses are generally denoted by a capital “A” signifying the amyloid present followed by an abbreviation for the fibril protein. AA amyloidosis is a normal sequence serum amyloid - A protein produced mainly in the liver in response to multiple cytokines. AA mostly occur in various chronic inflammatory disorders and is one of the most common systemic amyloidosis across the globe. Some of the conditions associated with AA include: rheumatoid arthritis, alzheimer disease, psoriasis, tuberculosis, renal cell carcinoma, hodgkin disease and non-hodgkin lymphoma.
AL amyloidosis or light chain amyloidosis monoclonal plasma cell disorder closely related to multiple myeloma. Organs involved in the AL amyloidosis include heart, kidney, peripheral nerve, gastrointestinal tract, respiratory tract and other organs. Treatment for AL amyloidosis in most instances is similar to multiple myeloma i.e. chemotherapy. Selected patient are treated with high-dose melphalan and autologous stem-cell transplantation. Alternative therapy includes rituximab, etanercept, thalidomide, iododoxorubicin and lenalidomide. In about about 80% the cases AA amyloidosis is fetal with median survival rate of 1-2 years only. Transthyretin amyloidosis (ATTR) is one of the most frequent inherited systemic forms of amyloidosis. ATTR is an autosomal-dominant disease but is associated with at least 100 different transthyretin mutations. Other forms of amyloidosis include heavy-chain amyloidosis, beta2 -microglobulin amyloidosis, hereditary renal amyloidosis and Central nervous system amyloidosis.
Major drivers for the growth of the global amyloidosis market include favourable investment and reimbursement facilities in developed economic countries. On the other hand high cost involved in treatment and limited awareness may hinder the growth of the overall market. Biopsy is the widely accepted diagnostic tool for amyloidosis and in about two-third of the patient alkylator-based chemotherapy is effective in managing amyloidosis. Noninvasive tests such as echocardiography of the heart and X-ray of bone abnormalities may provide supportive information but are not sufficient for the diagnosis of amyloidosis. Some of the prominent players operating in global amyloidosis market include Pfizer Inc., GlaxoSmithKline plc, Celgene Corporation, Alnylam Pharmaceuticals, Inc., Takeda Pharmaceutical Company Limited, Prothena Corporation plc. Premier institutes and universities promoting the development of amyloidosis clinical trial studies include Stanford University, Boston University, IRCCS Policlinico San Matte and Umea University.
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.