P60 Developing a decision-making framework for expanded access to gene therapy in rare neuromuscular diseases

Expanded access (EA) to investigational medicines offers a potential lifeline for patients suffering from a serious or life-threatening disease, who have exhausted other comparable or satisfactory alternative therapeutic options and are not eligible to enter a clinical trial. The 21st Century Cures Act requires that a company developing investigational medicines should make a policy regarding EA public and readily available. Navigating through EA can be challenging due to a number of considerations, such as the stage of clinical development, the safety profile of the investigational medicine, commercialization plans and the needs of different stakeholders.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research