Regenerative Medicine

Introduction Chapter 1. Regulation of RMs in the global 1.1. Regulation of RMs in each country 1.1.1. European Union (EU) 1.1.2. United States 1.1.3. Japan 1.1.4. Australia 1.1.5. Canada 1.1.6. South Korea 1.1.7. China 1.1.8. Singapore 1.1.9. India 1.1.10. New Zealand 1.2. Comparisons between countries 1.2.1. International classification and definitions of RMs 1.2.2. RMs legislation and frameworks 1.2.3. International RMs market authorization procedures 1.2.4. Regulatory assessment considerations for RMs 1.2.5. Acceptance of alternative evidence generation 1.2.6. Expedited MA assessment programs for RMs 1.2.7. Accelerated approval programs for RMs 1.2.8. Post-market requirements for RMs approved 1.2.9. RMs sponsor incentives 1.3. Challenges and recommendations 1.3.1. Discrepancies between RM regulatory requirements 1.3.2. The significance of post-market evidence generation 1.3.3. Prospects and recommendations 1.4. Conclusion Chapter 2. Health technology assessment of RMs 2.1. Country specific HTA decisions for individual products 2.1.1. Glybera® (Alipogene tiparvovec) 2.1.2. Kymriah® (Tisagenlecleucel) 2.1.3. Luxturna® (Voretigene neparvovec) 2.1.4. Imlygic® (Talimogene laherparepvec) 2.1.5. Strimvelis® 2.1.6. Yescarta® (Axicabtagene ciloleucel) 2.1.7. Zynteglo® (Betibeglogene autotemcel) 2.1.8. Libmeldy® (Atidarsagene autotemcel) 2.1.9. Zolgensma® (Onasemnogene abeparvovec) 2.1.10. Tecartus® (brexucabtagene autoleucel) 2.2. Summary of study limitation 2.2.1. Comparative study 2.2.2. Study population 2.2.3. Study endpoint 2.2.4. Statistical analysis 2.2.5. Confounding factors 2.2.6. Data transferability and generalizability 2.2.7. Indirect treatments comparison 2.3. Discussion and recommendations 2.3.1. Discrepancy in the Health Technology Assessment 2.3.2. Value Determination 2.3.3. Reimbursement and Affordability Strategies 2.3.4. Recommendations for clinical trials for RMs Chapter 3. Challenges for market access of RMs 3.1. Introduction 3.2. Challenges in the Health Technology Assessment 3.2.1. Uncertainties in the Clinical Evidence 3.3. Challenges in Economic Analysis 3.3.1. Challenges in value assessment 3.4. Challenges in Pricing, Reimbursement and Affordability 3.4.1. Controversial Surrounding the High Price of RMs 3.4.2. High Up-Front Costs Threaten Existing Payment System 3.5. Challenges in Innovative Payment Strategies 3.5.1. Outcome-based payment 3.5.2. Annuity payment/instalment payment 3.6. Discussion and Recommendations 3.6.1. Summary of the Study Findings 3.6.2. Future Recommendations 3.7. Conclusion Chapter 4. Impact of COVID-19 on the RMs industry 4.1. Introduction 4.2. Tremendous disruptions for all activities 4.2.1. Supply chain and manufacturing 4.2.2. Clinical trials 4.2.3. Delivering the therapies to patients 4.2.4. Small business 4.2.5. Regulatory activities 4.2.6. Health technology assessment 4.3. Progress made during the pandemic 4.3.1. New treatment opportunity 4.3.2. Financing raised 4.3.3. Regulation milestones 4.4. Conclusion Chapter 5. Partnerships to expediate successful market access of RMs 5.1. Introduction 5.2. Overall description for the partnership for RMs 5.3. Key players of Partnership Agreements 5.4. Discussion 5.4.1. Overall Description 5.4.2. Types of Partnership Agreements 5.4.3. Future Trends and Strategies 5.5. Case analyses: partnerships for approved RMs 5.5.1. RMs commercialized on the market 5.5.2. RMs withdrew from the market 5.5.3. Summary 5.6. Conclusion Chapter 6. Business models and commercial considerations for gene and cell therapies 6.1. Forecast of cell and gene therapy market 6.2. Increasing investment will bring fast-growing revenue 6.2.1. Cell and gene therapy market forecast for US 6.2.2. Cell and gene therapy market forecast for Europe 6.2.3. Cell and gene therapy market forecast for China 6.3. More challenges in the manufacturing and delivery 6.3.1. Challenges in manufacturing 6.3.2. Challenges in patient selection 6.3.3. Challenges in logistic and product delivery 6.3.4. Challenges in manufacturing allogeneic therapies 6.4. Why cell and gene therapies require a unique business model 6.4.1. Collaboration to navigate manufacturing challenges 6.4.2. Limited role of regional affiliates 6.4.3. Disruptions to traditional process of administration and monitoring 6.4.4. The time of approval is critical to secure market success 6.4.5. Different business strategies for cell and gene therapies 6.5. Partnerships to drive the CGT landscape progression 6.5.1. The advantage of partnership 6.5.2. Key cell and gene therapy participants 6.6. New payment models are needed for such innovative and costly therapies 6.6.1. The efficacy and safety of CGTs are not fully understood 6.6.2. Different value profiles for different patient population 6.6.3. High price of cell and gene therapies raised payment barriers 6.6.4. Innovative payment strategies to address payment challenges 6.6.5. Obstacles in implementing innovative payment strategies 6.7. Conclusions Chapter 7. Summary of recommendations and perspective for future research 7.1. Summary of key findings 7.2. Recommendations for manufacturers 7.2.1. Partnerships to streamline the market access of RMs 7.2.2. Strategies to strengthen the quality of clinical evidence 7.2.3. Active engagements with other stakeholders 7.3. Implications for future research efforts 7.3.1. From the perspective of market authorization 7.3.2. From the perspective of HTA and affordability References