Osteogenesis Imperfecta (OI) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Osteogenesis Imperfecta (OI) Market Outlook and Forecast

Osteogenesis Imperfecta (OI) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Published Date : 2023-11-10

Updated On : 2024-08-22

Pages : 156

Osteogenesis Imperfecta (OI) Market Outlook

Thelansis’s “Osteogenesis Imperfecta (OI) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Osteogenesis Imperfecta treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Osteogenesis Imperfecta (OI) Overview

Osteogenesis Imperfecta (OI), or brittle bone disease, is a genetic disorder affecting connective tissues due to abnormalities in the synthesis or processing of type I collagen. This condition leads to increased susceptibility to bone fractures and reduced bone density. Other symptoms include blue sclerae, dentinogenesis imperfecta, short stature, and adult-onset deafness. Reports also indicate valvular insufficiencies and aortic root dilation. Milder symptoms include generalized laxity, easy bruising, hernias, and excessive sweating. Clinical manifestations range from nearly asymptomatic forms to severe cases (such as infants with crumpled ribs, fragile cranium, and long bone fractures incompatible with life) that increase perinatal mortality. OI is a rare genetic disease, often stemming from mutations in the COL1A1 and COL1A2 genes. The International Society of Skeletal Dysplasias classifies OI based on the mode of inheritance and genes involved:

Osteogenesis Imperfecta Type Inheritance Genes
Nondeforming OI (Type I) AD, X-linked COL1A1, COL1A2, PLS3
Perinatal (Type II) AD, AR COL1A1, COL1A2, CRTAP, LEPRE1, PPIB, BMP1
Progressively Deforming (Type III) AD, AR
COL1A1, COL1A2, CRTAP, LEPRE1, PPIB, FKBP10, SERPINH1, SERINF1, WNT1
Moderate (Type IV) AD, AR
COL1A1, COL1A2, CRTAP, FKBP10, SP7, SERPINF1, WNT1, TMEM38B
Calcification of Interosseous Membrane or Hypertrophic Callus (Type V) AD

IFITM5

Management varies according to the age, severity, and functional status of patients:

  • Mild disease: Subtle restriction, avoid contact sports, treatment for any fractures.
  • Moderate to Severe disease: Rehabilitation and orthopedic interventions, management of acute fractures and scoliosis.
  • Severe disease: Use of an intramedullary rod with osteotomy to correct severe bowing of long bones.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2023-2033

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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Osteogenesis Imperfecta (OI) Competitive Landscape

S. no Asset Company Stage
1 Setrusumab (UX143) Ultragenyx Pharmaceutical Inc Phase 3
2 Romosozumab Amgen Phase 3
3 AGA2115 Angitia Incorporated Limited Phase 1
4 SAR439459 Sanofi Phase 1

KOLs across 8 MM market from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

COUNTRY No. Of KOLs
USA 17
GERMANY 4
UK 4
SPAIN 3
FRANCE 2
ITALY 3
JAPAN 3
CHINA 4

Osteogenesis Imperfecta (OI) Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

Data Inputs with sourcing, Market Event, Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

Osteogenesis Imperfecta (OI) Market Forecast

1.  Osteogenesis Imperfecta (OI) – Key Findings Summary

         1.1.    Clinical findings
                     1.1.1. Disease overview
                     1.1.2. Therapeutic practices
                     1.1.3. Future outlook
         1.2.    Commercial findings
                     1.2.1. Osteogenesis Imperfecta (OI) market scenario 2023
                     1.2.2. Osteogenesis Imperfecta (OI) market scenario 2028
                     1.2.3. Osteogenesis Imperfecta (OI) market scenario 2033

2. Osteogenesis Imperfecta (OI) Overview

         2.1.    Disease Introduction
         2.2.    Pathophysiology
         2.3.    Signs and Symptoms
         2.4.    Risk Factors
         2.5.    Etiology
         2.6.    Classification
         2.7.    Pathogenesis
         2.8.    Diagnosis
         2.9.    Complications
         2.10. Treatment Algorithm
                     2.10.1.    Treatment in US (guidelines)
                     2.10.2.    Treatment in EU-5 (guidelines)
                     2.10.3.    Treatment in Japan (guidelines)
                     2.10.4.    Treatment in China (guidelines)
         2.11. Treatment Goals for Osteogenesis Imperfecta (OI)
         2.12. Referral Patterns
                     2.12.1.    Referral Scenario in US
                     2.12.2.    Referral Scenario in EU-5
                     2.12.3.    Referral Scenario in Japan
                     2.12.4.    Referral Scenario in China
         2.13. Osteogenesis Imperfecta (OI) Prognosis
         2.14. Healthcare burden
                     2.14.1.    Healthcare burden in US
                     2.14.2.    Healthcare burden in EU-5
                     2.14.3.    Healthcare burden in Japan
                     2.14.4.    Healthcare burden in China
         2.15.  Unmet Needs in Osteogenesis Imperfecta (OI) management
         2.16.  Market Opportunity for Osteogenesis Imperfecta (OI)
         2.17. KOL Comments on current and upcoming/expected treatment practices in Osteogenesis Imperfecta (OI)

3.       Epidemiology

         3.1.    Epidemiology Overview
         3.2.    Epidemiology by Geography
                     3.2.1. Osteogenesis Imperfecta (OI) Epidemiology in US (2023-2033)
                             3.2.1.1.              Incidence of Osteogenesis Imperfecta (OI)
                             3.2.1.2.              Diagnosed cases
                             3.2.1.3.              Treatable Patient Pool
                             3.2.1.4.              Epidemiology Trends
                     3.2.2. Osteogenesis Imperfecta (OI) Epidemiology in EU-5 (2023-2033)
                             3.2.2.1.              Incidence of Osteogenesis Imperfecta (OI)
                             3.2.2.2.              Diagnosed cases
                             3.2.2.3.              Treatable Patient Pool
                             3.2.2.4.              Epidemiology Trends
                     3.2.3. Osteogenesis Imperfecta (OI) Epidemiology in Japan (2023-2033)
                             3.2.3.1.              Incidence of Osteogenesis Imperfecta (OI)
                             3.2.3.2.              Diagnosed cases
                             3.2.3.3.              Treatable Patient Pool
                             3.2.3.4.              Epidemiology Trends
                     3.2.4. Osteogenesis Imperfecta (OI) Epidemiology in China (2023-2033)
                             3.2.4.1.              Incidence of Osteogenesis Imperfecta (OI)
                             3.2.4.2.              Diagnosed cases
                             3.2.4.3.              Treatable Patient Pool
                             3.2.4.4.              Epidemiology Trends
         3.3.    Epidemiology Trends (World-wide)

4.       Market Outlook

         4.1.    US Osteogenesis Imperfecta (OI) Market Forecast 2023-2033
                     4.1.1. Market Progression (Futuristic)
                     4.1.2. Market Trends and Expectations
                             4.1.2.1.              Worst case scenario
                             4.1.2.2.              Base Case Scenario
                             4.1.2.3.              Best Case Scenario
                     4.1.3. Drivers and Barriers
         4.2.    UK Osteogenesis Imperfecta (OI) Market Forecast 2023-2033
                     4.2.1. Market Progression (Futuristic)
                     4.2.2. Market Trends and Expectations
                             4.2.2.1.              Worst case scenario
                             4.2.2.2.              Base Case Scenario
                             4.2.2.3.              Best Case Scenario
                     4.2.3. Drivers and Barriers
         4.3.    France Osteogenesis Imperfecta (OI) Market Forecast 2023-2033
                     4.3.1. Market Progression (Futuristic)
                     4.3.2. Market Trends and Expectations
                             4.3.2.1.              Worst case scenario
                             4.3.2.2.              Base Case Scenario
                             4.3.2.3.              Best Case Scenario
                     4.3.3. Drivers and Barriers
         4.4.    Germany Osteogenesis Imperfecta (OI) Market Forecast 2023-2033
                     4.4.1. Market Progression (Futuristic)
                     4.4.2. Market Trends and Expectations
                             4.4.2.1.              Worst case scenario
                             4.4.2.2.              Base Case Scenario
                             4.4.2.3.              Best Case Scenario
                     4.4.3. Drivers and Barriers
         4.5.    Italy Osteogenesis Imperfecta (OI) Market Forecast 2023-2033
                     4.5.1. Market Progression (Futuristic)
                     4.5.2. Market Trends and Expectations
                             4.5.2.1.              Worst case scenario
                             4.5.2.2.              Base Case Scenario
                             4.5.2.3.              Best Case Scenario
                     4.5.3. Drivers and Barriers
         4.6.    Spain Osteogenesis Imperfecta (OI) Market Forecast 2023-2033
                     4.6.1. Market Progression (Futuristic)
                     4.6.2. Market Trends and Expectations
                             4.6.2.1.              Worst case scenario
                             4.6.2.2.              Base Case Scenario
                             4.6.2.3.              Best Case Scenario
                     4.6.3. Drivers and Barriers
         4.7.    Japan Osteogenesis Imperfecta (OI) Market Forecast 2023-2033
                     4.7.1. Market Progression (Futuristic)
                     4.7.2. Market Trends and Expectations
                             4.7.2.1.              Worst case scenario
                             4.7.2.2.              Base Case Scenario
                             4.7.2.3.              Best Case Scenario
                     4.7.3. Drivers and Barriers
         4.8.    China Osteogenesis Imperfecta (OI) Market Forecast 2023-2033
                     4.8.1. Market Progression (Futuristic)
                     4.8.2. Market Trends and Expectations
                             4.8.2.1.              Worst case scenario
                             4.8.2.2.              Base Case Scenario
                             4.8.2.3.              Best Case Scenario
                     4.8.3. Drivers and Barriers
         4.9.    Key Expected Milestones (world-wide) Impacting the Market

5.       Competitive Landscape

         5.1.    Pipeline Therapies Overview
                     5.1.1. Phase III Therapies
                                      5.1.1.1.                     Current Status
                                      5.1.1.2.                     Trial details, results
                                      5.1.1.3.                     Approval Timeline
                                      5.1.1.4.                     Likelihood of approval
                                      5.1.1.5.                     Expected Product Positioning
                             5.1.1.2.              All other Phase III Therapies …..
                             5.1.1.3.              Attribute Analysis of Phase III molecules
                     5.1.2.  Phase II and Phase I/II Therapies
                                      5.1.2.1.                     Current Status
                                      5.1.2.2.                     Trial details, results
                                      5.1.2.3.                     Approval Timelines
                     5.1.3. List of active Pre-clinical Therapies
                                      5.1.3.1.                     Status in Osteogenesis Imperfecta (OI)
                                      5.1.3.2.                     Company positioning
                             5.1.3.2.              All other pre-clinical therapies
                     5.1.4. List of Inactive/discontinued assets
                             5.1.4.1.              Business impact of discontinuations on current pipeline
                     5.1.5. Potential winners from Osteogenesis Imperfecta (OI) Pipeline
                             5.1.5.1.              Potential Blockbusters across the pipeline

6.       Regulatory/Approval Scenario

         6.1.    Regulatory/Approval Framework in US
                     6.1.1. Policy Framework
                     6.1.2. Payer Expectations
         6.2.    Regulatory/Approval Framework in UK
                     6.2.1. Policy Framework
                     6.2.2. Payer Expectations
         6.3.    Regulatory/Approval Framework in France
                     6.3.1. Policy Framework
                     6.3.2. Payer Expectations
         6.4.    Regulatory/Approval Framework in Germany
                     6.4.1. Policy Framework
                     6.4.2. Payer Expectations
         6.5.    Regulatory/Approval Framework in Italy
                     6.5.1. Policy Framework
                     6.5.2. Payer Expectations
         6.6.    Regulatory/Approval Framework in Spain
                     6.6.1. Policy Framework
                     6.6.2. Payer Expectations
         6.7.       Regulatory/Approval Framework in Japan
                     6.7.1. Policy Framework
                     6.7.2. Payer Expectations
         6.8.       Regulatory/Approval Framework in China
                     6.8.1. Policy Framework
                     6.8.2. Payer Expectations

7.       Clinical Trial Assessment – Current and Future Paradigm

         7.1.    Distribution of Primary Endpoints across trials
         7.2.    Distribution of Secondary Endpoints across trials
         7.3.    Evolution and acceptance of surrogate endpoints
         7.4.    Key Investigator initiated trials
         7.5.    Attrition analysis
                     7.5.1. Suspended/Discontinued Assets
                     7.5.2. Failed Trials, Reasons and Business Impact
                     7.5.3. Terminated Trials, Reasons and Business Impact
                     7.5.4. Withdrawn Trials, Reasons and Business Impact
         7.6.    Trial enrollment scenario and challenges
         7.7.    Clinical Trial Guidance (across geographies)

8.       Thelansis Commentary

         8.1.    Key Unmet needs in Osteogenesis Imperfecta (OI)
         8.2.    Possible Best-case Clinical Trial Strategies
         8.3.    Possible Best Case Targeted Product Profile (TPP)
         8.4.    Possible Best-case Market positioning strategies
         8.5.    Possible Best-case Market Access Strategies
         8.6.    Possible Best-case LCM Strategies
         8.7.    Overall View on Osteogenesis Imperfecta (OI) Market in Dollar Value

9.       Report Methodology

         9.1.    Secondary research
         9.2.    Primary research
         9.3.    Data collation
         9.4.    Insight Generation

10.   About Thelansis

         10.1.    Our Capabilities
         10.2.    Our Services
         10.3.    Our Contacts
         10.4.    Disclaimer