EU early access - regulatory framework & practical considerations

European Regulation 726/2004/EC (Article 83) and Directive 2001/83 (Article 5), provide a regulatory framework for access to investigational medicines outside the context of the clinical trial, allowing physicians to access potentially life-saving medicines that would otherwise be unavailable for their patients. Treatment with an investigational product represents an important option for patients suffering from serious or life threatening conditions where licensed alternatives are either unavailable or unsuitable for the patient. They can often be the only treatment option for disease areas of high unmet need such as rare diseases and orphan indications. Whilst the pharmaceutical industry has continued to focus on accelerating access to innovative new treatments by shortening the development timelines; increased regulatory challenges and delays due to pricing & reimbursement negotiations can result in delays of many years between positive phase III trials and commercial availability. Access Programs hence provide an important mechanism to bridge the time between clinical development, marketing authorisation and product launch. Although this EU framework exists, each member state has decided independently how and when to allow such access, and developed national rules and legislation to reflect this. As a result, there is no single, centralized European procedure for either single patient or cohort approaches; indeed there are often more differences between the member states than similarities. Generally, access is initiated by the physician, is limited to investigational products for the treatment of a serious or rare disease and where there is an absence of alternative approved treatments. The objective of this article is to provide an overview of the regulatory frameworks available in the member states, as well as practical considerations for implementation of an access program.