W-27: The Balance between Compassion, Cost, & Commercialization in Expanded Access Programs: Managing Complexities
Clinical Project Leader
QuintilesIMS United States
Understand the complexities involved with expanded access programs and balance them to achieve the best outcomes for patients, practitioners, public and private stakeholders.
From retrospective review since 2010: Key considerations to the design and conduct of expanded access programs will be described including: how to fill an unmet need in an underserved patient population while serving all community and corporate stakeholders and creating optimal product access.
Expanded access programs (EAPs), typically driven by compassion, provide patients early access to drugs prior to market approval. Patients with life-threatening conditions, such hematological malignancies, have no treatment alternatives in some cases other than a promising investigational drug, i.e. Compassionate Use. EAPs provide practitioners an additional opportunity to gain incremental clinical experience and safety information with the investigational product in a practical setting on a wider range of patients than in a clinical trial but this comes with cost implications. There are also commercial consequences and benefits to offering an EAP which drive product access. Drug developers should consider the 3 Cs in considering and designing an EAP – Compassion, Cost, and Commercialization. In doing so, significant complexities are well-managed during planning, execution, and closing an EAP.
EAPs offer incremental and significant insights into an investigational drug’s utility and safely. EAPs should be designed in such a way to capture information that aids in this better understanding of the drug performance in a comprehensive manner. The 3 Cs should be considered and balanced in an EAP to provide the greatest benefit possible for the patient and practitioner.