A Global Perspective on Compassionate Use and Expanded Access.

Whilst the complex ethical and benefit/risk questions that need to be considered when determining whether an investigational treatment should be given to a patient remain constant throughout the world, the practical and logistical realities of running an Early Access Program (EAP) differ widely globally presenting a whole range of challenges if the objective is to find solutions that are in the patients best interest and are as fair and equitable as possible. Some of the complexities can create extreme challenges for Pharma Companies looking to set up global programs, but with the right planning and strategy it is possible to overcome hurdles allowing patients to access critical treatments they desperately need. The exact design and scope of any global EAP will depend on country scope, expected demand, regulatory feasibility, the license status of the product, necessary drug pricing structure, as well as company strategy, costs, and product supply. Having worked in this space for many years, I am constantly reminded of the dramatic positive impact early access to critical treatments can have on the lives of patients and their families. I am also well aware of the potential risks that need to be well thought through and managed in order to provide access in a timely and compliant manner to the right patients and to compliment and support, rather than disrupt, traditional development pathways.