AI Summary & Key Takeaways
AI InsightThe inclusion of gene and cell therapies in guidelines and legislation may improve the market access of these therapies
📌 3 Key Takeaways
Abstract
Objective: Genetic diseases constitute the majority of rare diseases. The Orphanet portal for rare diseases lists more than 5800 diseases in its database. Rare diseases of genetic origin are less common than other diseases. The current treatment costs of these diseases cause a high budget impact on health systems. Gene and cell therapies are targeting the innovating treatment methods to cure the underlying diseases rather than the symptoms. Researchers, health care industries, small and medium-sized enterprises companies, as well as major pharmaceutical companies, are paying more attention to gene and cell therapies. European countries are highly invested in gene and cell therapy research. This study aims to provide information on trends and health policies in Europe.Methods: The study followed an inductive research approach with secondary data search which was collected from different online sources to perform areviow on the regulatory process, reimbursement, pricing decisions, and regenerative therapy market.Results: The United Kingdom, Germany, Italy, Portugal, Spain, and France follow the European Medicines Agency regulatory process for market approval. Turkey does not follow the European Medicines Agency and does not have any defined regulation authority for gene and cell therapy. France, Germany, and the United Kingdom have recently published updated health technology assessment reports. Portugal and Spain do not have nationally approved gene and cell therapy practices, however, have initiated research resource on cellular and gene therapies. The United Kingdom and Germany are the most advanced in the commercialization and legalization of gene and cellular therapies in selected countries.Conclusion: European countries have variable pricing, reimbursement, and market access regulations apart from the common European Medicines Agency regulations. Improved gene and cell therapy regulations have proven the clinical effectiveness of new treatments. Academic research centers, small and medium businesses are the main components of gene and cell therapy research. The inclusion of gene and cell therapies in guidelines and legislation may improve the market access of these therapies
Özet
Objective: Genetic diseases constitute the majority of rare diseases. The Orphanet portal for rare diseases lists more than 5800 diseases in its database. Rare diseases of genetic origin are less common than other diseases. The current treatment costs of these diseases cause a high budget impact on health systems. Gene and cell therapies are targeting the innovating treatment methods to cure the underlying diseases rather than the symptoms. Researchers, health care industries, small and medium-sized enterprises companies, as well as major pharmaceutical companies, are paying more attention to gene and cell therapies. European countries are highly invested in gene and cell therapy research. This study aims to provide information on trends and health policies in Europe.Methods: The study followed an inductive research approach with secondary data search which was collected from different online sources to perform areviow on the regulatory process, reimbursement, pricing decisions, and regenerative therapy market.Results: The United Kingdom, Germany, Italy, Portugal, Spain, and France follow the European Medicines Agency regulatory process for market approval. Turkey does not follow the European Medicines Agency and does not have any defined regulation authority for gene and cell therapy. France, Germany, and the United Kingdom have recently published updated health technology assessment reports. Portugal and Spain do not have nationally approved gene and cell therapy practices, however, have initiated research resource on cellular and gene therapies. The United Kingdom and Germany are the most advanced in the commercialization and legalization of gene and cellular therapies in selected countries.Conclusion: European countries have variable pricing, reimbursement, and market access regulations apart from the common European Medicines Agency regulations. Improved gene and cell therapy regulations have proven the clinical effectiveness of new treatments. Academic research centers, small and medium businesses are the main components of gene and cell therapy research. The inclusion of gene and cell therapies in guidelines and legislation may improve the market access of these therapies