Safety and Oversight of the Individually Tailored Treatment Approach: A Novel Pilot Study

Summary

Participation Requirements

Description

Some advanced cancers have numerous standard treatment options that have proven efficacy in clinical trials. However, in other cancers, there may be few or no standard treatment options with proven efficacy as determined in a large clinical trial. This may be particularly the case for rare cancers i...

Some advanced cancers have numerous standard treatment options that have proven efficacy in clinical trials. However, in other cancers, there may be few or no standard treatment options with proven efficacy as determined in a large clinical trial. This may be particularly the case for rare cancers in which there is a lack of clinical research. When seriously ill patients run out of standard treatment options, they will often consider non-standard treatment options (such as treatments that are currently unapproved by the regulatory agency for the given indication). The majority of clinicians and researchers agree that this is best received in a clinical trial setting as this provides ethical and clinical oversight, as well as addresses prospectively defined research questions which can be publicly reported. This allows the conclusions of the research to be available to the entire clinical and research community. In general, an access program enables patient access to a non-reimbursed therapeutic agent, outside of a clinical trial setting. Compassionate access is typically for therapeutics that are not yet approved or TGA registered, and are still considered investigational. In general, there is a negotiation between the pharmaceutical company and the clinician and patient regarding access to the therapeutic agent, as well as whether the medicine will be provided free of charge, or on some form of cost-sharing arrangement. In Australia, access to TGA non-registered medicines also requires an application via the "Special Access Scheme". For most cancer patients, the use falls under category A, for a patient defined as seriously ill. This sub-study generally pertains to compassionate access to therapeutic agents. Given the ad hoc nature of compassionate access for patients, there is relatively little reported data on clinical outcomes. Compassionate access is an established process with increasing demands. This study is designed to provide a framework for which patients treated with compassionate access therapeutics can register, so that some of the limitations of ad hoc compassionate access programs can be overcome. A study committee will prospectively assess each individual patient's detailed treatment approach in an objective and time-efficient manner. If approved, the patient may be eligible to register into the treatment phase of the study. The study committee is essential to provide a balanced approach to understanding the rationale for the study treatment, as well as potential safety issues that may arise. As previously reported, this is an essential component to improving patient oversight as well as equity

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