The changing landscape of expanded access to investigational drugs for patients with unmet medical needs: ethical implications
Abstract
When patients are told that standard medical treatment options have been exhausted, their treating physicians
may start looking for promising new drugs that are not yet approved, and still under investigation. Some patients
can be included in clinical trials, but others cannot. It is not widely known that these patients might still be eligible
for trying investigational drugs, in a therapeutic context. Worldwide, public and private parties are seeking to
change this by informing patients and physicians about opportunities for expanded access and/or by facilitating its
processes. When expanded access becomes available to larger groups of patients, ethical issues gain prominence,
including informed consent, funding issues, disparities in access, and potential adverse effects on clinical drug
development. Physicians, patients and policy-makers should not shift the responsibility to address these issues to
pharmaceutical companies, but work together to resolve them