Cell and Gene Therapy medicinal products need a tailored and integrated approach

Abstract

Background & Aim Advanced Therapy Medicinal Products (ATMPs), that include gene and cell therapy products, belong to a rapidly developing and state-of-the-art field of science and technology. Due to their complex nature, product development can be challenging. While they are being regulated under the same principle as for all medicinal products, that is where the similarity ends. From start to finish, these products need a personalised approach which considers 1. The scientific complexity 2. The regulatory expectations 3. The reimbursement challenges While a conventional medicinal product such as a small molecule or even a biological product could follow a stepwise approach to achieve success in development, regulatory approval and commercialisation and reimbursement, such an approach for cell and gene therapies might not be efficient and appropriate. Further, the scientific model of drug development starting from quality/ CMC through to non-clinical and clinical development to achieve licensing may be inefficient at best and misleading at worst. To ensure that the right steps are taken for development, there are a number of regulatory procedures available. One such procedure is scientific advice from regulatory agencies. It is strongnly recommended to consider this as early as possible. This can also include joint advice with both regulatory agencies and payers to ensure that both are taken into account for planning the development. Methods, Results & Conclusion Thus, the ATMP development program should aim to streamline development and avoid known pitfalls in order to produce a viable product that will fulfil its potential in the real-world setting. For this reason, an integrated approach that includes involvement of all the stakeholders from beginning to end is needed if these products with their enormous potential including curing a complex and devastating condition are to achieve their full potential to the patients with serious and unmet medical need and who are ultimately the real beneficiaries. Advanced Therapy Medicinal Products (ATMPs), that include gene and cell therapy products, belong to a rapidly developing and state-of-the-art field of science and technology. Due to their complex nature, product development can be challenging. While they are being regulated under the same principle as for all medicinal products, that is where the similarity ends. From start to finish, these products need a personalised approach which considers 1. The scientific complexity 2. The regulatory expectations 3. The reimbursement challenges While a conventional medicinal product such as a small molecule or even a biological product could follow a stepwise approach to achieve success in development, regulatory approval and commercialisation and reimbursement, such an approach for cell and gene therapies might not be efficient and appropriate. Further, the scientific model of drug development starting from quality/ CMC through to non-clinical and clinical development to achieve licensing may be inefficient at best and misleading at worst. To ensure that the right steps are taken for development, there are a number of regulatory procedures available. One such procedure is scientific advice from regulatory agencies. It is strongnly recommended to consider this as early as possible. This can also include joint advice with both regulatory agencies and payers to ensure that both are taken into account for planning the development. Thus, the ATMP development program should aim to streamline development and avoid known pitfalls in order to produce a viable product that will fulfil its potential in the real-world setting. For this reason, an integrated approach that includes involvement of all the stakeholders from beginning to end is needed if these products with their enormous potential including curing a complex and devastating condition are to achieve their full potential to the patients with serious and unmet medical need and who are ultimately the real beneficiaries.