Patient participation in regulatory decisions regarding regenerative medicine

      Regulatory agencies responsible for ensuring the safety, efficacy, and quality of medical products for human use are faced with many challenges in appropriately regulating novel medical technologies, including emerging regenerative medicine treatments such as stem cell-based therapies. These agencies have the difficult task of assessing whether the balance of risks and benefits associated with a new treatment justifies approval, in the context of substantial uncertainty. In making these decisions, they are increasingly asked to take into account the perspectives of patients and patient advocacy organizations. These groups may have important information about the risks and benefits of a new treatment and perspectives on how they should be balanced, but their participation in regulatory decision making raises many questions. What type of input should be sought or received from patients and at what stages in the regulatory process? Who should speak for patients and how should the diversity of interests and perspectives among patients be addressed in processes for consultation or input? In what ways should information and views from patients be used in regulatory decisions and how much weight should they be given? Are there special considerations regarding patient participation in the context of novel forms of treatment, such as stem cell-based therapies, or particular types of conditions, such as rare diseases? This paper examines existing and proposed models for patient input into regulatory decision making, seeking to determine how different systems have answered the questions above. It will identify the various approaches used and their advantages and disadvantages, with a view to formulating a set of recommendations or best practices for regulatory agencies and patient organizations to consider.
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