A three-pronged approach was followed for deducing the hemoglobinopathies market estimates and forecasts. The process has three steps: information procurement, analysis, and validation. The whole process is cyclical, and steps repeat until the estimates are validated. The three steps are explained in detail below:
Information procurement: Information procurement is one of the most extensive and important stages in our research process, and quality data is critical for accurate analysis. We followed a multi-channel data collection process for hemoglobinopathies market to gather the most reliable and current information possible.
Analysis: We mine the data collected to establish baselines for forecasting, identify trends and opportunities, gain insight into consumer demographics and drivers, and so much more. We utilized different methods of hemoglobinopathies market data depending on the type of information we’re trying to uncover in our research.
Market Research Efforts: Bottom-up Approach for estimating and forecasting demand size and opportunity, top-down Approach for new product forecasting and penetration, and combined approach of both Bottom-up and Top-down for full coverage analysis.
Value-Chain-Based Sizing & Forecasting: Supply-side estimates for understanding potential revenue through competitive benchmarking, forecasting, and penetration modeling.
Demand-side estimates for identifying parent and ancillary markets, segment modeling, and heuristic forecasting.
Qualitative Functional Deployment (QFD) Modelling for market share assessment.
Market formulation and validation: We mine the data collected to establish baselines for forecasting, identify trends and opportunities, gain insight into consumer demographics and drivers, and so much more. We utilize different methods of data analysis depending on the type of information we’re trying to uncover in our research.
Market Formulation: This step involves the finalization of market numbers. This step on an internal level is designed to manage outputs from the Data Analysis step.
Data Normalization: The final market estimates and forecasts are then aligned and sent to industry experts, in-panel quality control managers for validation.
This step also entails the finalization of the report scope and data representation pattern.
Validation: The process entails multiple levels of validation. All these steps run in parallel, and the study is forwarded for publishing only if all three levels render validated results.
The hemoglobinopathies market was categorized into four segments, namely type (Thalassemia, Sickle Cell Disease), diagnosis (Thalassemia, Sickle Cell Disease), therapy (Thalassemia, Sickle Cell Disease), and regions (North America, Europe, Asia Pacific, Latin America, Middle East & Africa).
The hemoglobinopathies market was segmented into type, diagnosis, therapy, and regions. The demand at a segment level was deduced using a funnel method. Concepts like the TAM, SAM, SOM, etc., were put into practice to understand the demand. We at GVR deploy three methods to deduce market estimates and determine forecasts. These methods are explained below:
Demand estimation of each product across countries/regions summed up to from the total market.
Variable analysis for demand forecast.
Demand estimation via analyzing paid database, and company financials either via annual reports or paid database.
Primary interviews for data revalidation and insight collection.
Used extensively for new product forecasting or analyzing penetration levels.
Tool used invoice product flow and penetration models Use of regression multi-variant analysis for forecasting Involves extensive use of paid and public databases.
Primary interviews and vendor-based primary research for variable impact analysis.
The hemoglobinopathies market was analyzed at a regional level. The globe was divided into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa, keeping in focus variables like consumption patterns, export-import regulations, consumer expectations, etc. These regions were further divided into twenty-five countries, namely, the U.S.; Canada; the UK.; Germany; France; Italy; Spain; Russia; Denmark; Sweden; Norway; India; China; Japan; Singapore; South Korea; Australia; Thailand; Brazil; Mexico; Argentina; Saudi Arabia; UAE; South Africa; and Kuwait.
All three above-mentioned market research methodologies were applied to arrive at regional-level conclusions. The regions were then summed up to form the global market.
The hemoglobinopathies market was analyzed via companies operating in the sector. Analyzing these companies and cross-referencing them to the demand equation helped us validate our assumptions and conclusions. Key market players analyzed include:
SANGAMO THERAPEUTICS: Sangamo Therapeutics is involved in developing proprietary platform technologies in gene therapy, genome editing, cell therapy, and gene regulation.
BLUEBIRD BIO, INC.: Bluebird bio is a clinical-stage biopharmaceutical company involved in developing transformative gene therapies aimed at treating cancer and severe genetic diseases. The company has developed significant capabilities in gene editing and lentiviral-based gene therapies for treating conditions such as cerebral adrenoleukodystrophy, SCD, beta-thalassemia, Hurler syndrome, and multiple myeloma.
GLOBAL BLOOD THERAPEUTICS, INC.: Global Blood Therapeutics, Inc. (GBT) is involved in research, development, and manufacture of therapies for blood-related disorders. Its product pipeline includes drug candidates for SCD therapy in pediatric, adolescent, and adult patients.
PFIZER, INC.: Pfizer manufactures and offers products for treatment of a wide range of diseases, including diabetes, immunologic conditions, cardiac disorders, cancer, and dermatological diseases. It operates through two segments: Innovative Health and Essential Health.
EMMAUS LIFE SCIENCES, INC.: Emmaus Life Sciences is involved in the processes of discovery, development, and manufacture of therapeutics, predominantly for rare & orphan disorders. The company is focused on product development for SCD and other blood disorders.
PROLONG PHARMACEUTICALS, LLC.: Prolong Pharmaceuticals is currently involved in development and manufacture of products for treatment of diseases in the areas of oncology and hematology. The company’s product pipeline in clinical development includes SANGUINATE-used in treating the comorbidities of SCD along with disorders caused by ischemia, hypoxia, and hemolysis.
CELGENE CORPORATION (BMS): Celgene Corporation is a biotechnology company involved in research & development of novel therapies for treatment of cancer & inflammatory diseases. The company is focused on development of new and more efficient drugs for patients suffering from multiple myeloma, chronic lymphocytic leukemia, myelodysplastic syndromes, beta-thalassemia, non-small cell lung cancer, and pancreatic cancer.
BIOVERATIV INC. (SANOFI): Bioverativ is involved in discovery, development, and manufacturing of therapies used in the management of hemophilia, cold agglutinin disease, and other blood disorders.
GAMIDA CELL: Gamida Cell is engaged in development of advanced cell therapies for use in the treatment of cancer and other hematologic diseases. The company uses its Nicotinamide-based (NAM) cell expansion technology to develop pipeline of products in order to address the limitations of cell therapies.
NOVARTIS AG: Novartis AG is involved in R&D, manufacturing, and marketing of healthcare products. The company operates through three divisions: Innovative Medicines, Sandoz, and Alcon.
Supply Side Estimates
Company revenue estimation via referring to annual reports, investor presentations, and Hoover’s.
Segment revenue determination via variable analysis and penetration modeling.
Competitive benchmarking to identify market leaders and their collective revenue shares.
Forecasting via analyzing commercialization rates, pipelines, market initiatives, distribution networks, etc.
Demand side estimates
Identifying parent markets and ancillary markets
Segment penetration analysis to obtain pertinent
Heuristic forecasting with the help of subject matter experts
Forecasting via variable analysis
Understanding market dynamics (in terms of drivers, restraints, & opportunities) in the countries.
Understanding trends & variables in the individual countries & their impact on growth and using analytical tools to provide high-level insights into the market dynamics and the associated growth pattern.
Understanding market estimates and forecasts (with the base year as 2022, historic information from 2018 to 2021, and forecast from 2023 to 2030). Regional estimates & forecasts for each category are available and are summed up to form the global market estimates.
The report provides market value for the base year 2022 and a yearly forecast till 2030 in terms of revenue/volume or both. The market for each of the segment outlooks has been provided on region & country basis for the above-mentioned forecast period.
The key industry dynamics, major technological trends, and application markets are evaluated to understand their impact on the demand for the forecast period. The growth rates were estimated using correlation, regression, and time-series analysis.
We have used the bottom-up approach for market sizing, analyzing key regional markets, dynamics, & trends for various products and end-users. The total market has been estimated by integrating the country markets.
All market estimates and forecasts have been validated through primary interviews with the key industry participants.
Inflation has not been accounted for to estimate and forecast the market.
Numbers may not add up due to rounding off.
Europe consists of EU-8, Central & Eastern Europe, along with the Commonwealth of Independent States (CIS).
Asia Pacific includes South Asia, East Asia, Southeast Asia, and Oceania (Australia & New Zealand).
Latin America includes Central American countries and the South American continent
Middle East includes Western Asia (as assigned by the UN Statistics Division) and the African continent.
GVR strives to procure the latest and unique information for reports directly from industry experts, which gives it a competitive edge. Quality is of utmost importance to us, therefore every year we focus on increasing our experts’ panel. Primary interviews are one of the critical steps in identifying recent market trends and scenarios. This process enables us to justify and validate our market estimates and forecasts to our clients. With more than 8,000 reports in our database, we have connected with some key opinion leaders across various domains, including healthcare, technology, consumer goods, and the chemical sector. Our process starts with identifying the right platform for a particular type of report, i.e., emails, LinkedIn, seminars, or telephonic conversation, as every report is unique and requires a differentiated approach.
We send out questionnaires to different experts from various regions/ countries, which is dependent on the following factors:
Report/Market scope: If the market study is global, we send questionnaires to industry experts across various regions, including North America, Europe, Asia Pacific, Latin America, and MEA.
Market Penetration: If the market is driven by technological advancements, population density, disease prevalence, or other factors, we identify experts and send out questionnaires based on region or country dominance.
The time to start receiving responses from industry experts varies based on how niche or well-penetrated the market is. Our reports include a detailed chapter on the KoL opinion section, which helps our clients understand the perspective of experts already in the market space.
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