Forum on Regenerative Medicine – författare

Regenerative medicine products, which are intended to repair or replace damaged cells or tissues in the body, include a range of therapeutic approaches such as cell- and gene-based therapies, engineered tissues, and non-biologic constructs. The current approach to characterizing the quality of a regenerative medicine product and the manufacturing process often involves measuring as many endpoints as possible, but this approach has proved to be inadequate and unsustainable.

The Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine convened experts across disciplines for a 2-day virtual public workshop to explore systems thinking approaches and how they may be applied to support the identification of relevant quality attributes that can help in the optimization of manufacturing and streamline regulatory processes for regenerative medicine. A broad array of stakeholders, including data scientists, physical scientists, industry researchers, regulatory officials, clinicians, and patient representatives, discussed new advances in data acquisition, data analysis and theoretical frameworks, and how systems approaches can be applied to the development of regenerative medicine products that can address the unmet needs of patients. This publication summarizes the presentation and discussion of the workshop.

Regenerative medicine holds the potential to create living, functional cells and tissues that can be used to repair or replace those that have suffered potentially irreparable damage due to disease, age, traumatic injury, or genetic and congenital defects. The field of regenerative medicine is broad and includes research and development components of gene and cell therapies, tissue engineering, and non-biologic constructs. Although regenerative medicine has the potential to improve health and deliver economic benefits, this relatively new field faces challenges to developing policies and procedures to support the development of novel therapies are both safe and effective.

In October 2016, the National Academies of Sciences, Engineering, and Medicine hosted a public workshop with the goal of developing a broad understanding of the opportunities and challenges associated with regenerative medicine cellular therapies and related technologies. Participants explored the state of the science of cell-based regenerative therapies within the larger context of patient care and policy. This publication summarizes the presentations and discussions from the workshop.

On June 26, 2017, the Forum on Regenerative Medicine hosted a public workshop in Washington, DC, titled Navigating the Manufacturing Process and Ensuring the Quality of Regenerative Medicine Therapies in order to examine and discuss the challenges, opportunities, and best practices associated with defining and measuring the quality of cell and tissue products and raw materials in the research and manufacturing of regenerative medicine therapies. The goal of the workshop was to learn from existing examples of the manufacturing of early-generation regenerative medicine products and to address how progress could be made in identifying and measuring critical quality attributes. The workshop also addressed the challenges of designing and adhering to standards as a way of helping those who are working to scale up processes and techniques from a research laboratory to the manufacturing environment. This publication summarizes the presentations and discussions from the workshop.

Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.