Clinical trials evaluate investigational agents and/or new regimens in a tightly controlled research setting. Trials provide access to new agents with the potential for clinical benefit or new methods of administration/scheduling. In the case of patients with advanced malignancy, clinical trials often provide treatment options where few or none exist. Treatment given as part of a clinical trial may utilise medications, doses and scheduling outside those familiar to staff.
Once a sufficient level of evidence of benefit and safety of a new agent has been obtained from clinical trials, access to new agents is being increasingly provided by industry via special or compassionate access schemes prior to PBS listing. Access schemes may involve part payment for the agent by the patient. Access is generally conditional on prescribing the agent in accordance with the protocol used in the clinical trial.
Whilst guidance for clinical trials is beyond the scope of this document, the same principles and safety precautions described in these guidelines also apply to the prescribing, supply and administration of investigational agents and the conduct of clinical trials.
All clinical trial protocols will require local Human Ethics and Research Committee approval before a study is opened and offered to patients.
All staff involved in the prescribing, dispensing and administration of treatment as part of the trial must be appropriately trained and educated according to the trial requirements. An up-to-date copy of the study protocol should be available and accessible to all staff involved in the trial. Staff should also have access to clinical trials staff involved in the management of cancer clinical trials open at their facility.
Fatal errors have occurred where trial protocols have been misinterpreted by staff  and doses of chemotherapy have been compromised where trial protocols have been used that are not familiar to staff.
Medicine access programs
Guiding principles are available to support the supply of medicines under Medicine Access Programs (MAP) which include responsibilities of facilities when providing access to these medicines. These guidelines define principles to ensure that patients and hospitals are not unduly exposed to clinical risk when utilising MAP.
The management of the treatment within a clinical trial should be limited to those familiar with the trial protocol.
Clinical trial protocols should clearly outline the treatment protocol; however the format may differ according to the local ethics/governance requirements. Steps should be taken to ensure that information is presented in a clear manner in the clinical trial protocol through a multidisciplinary review process.
Medicine access programs
The recommendations of the Council of Australian Therapeutic Advisory Groups should be applied to the conduct of any MAP use in the context of cancer therapy (Council of Australian Therapeutic Advisory Groups, 2015). Responsibilities of all parties involved in the provision of the MAP should be documented.
Approval should be obtained from the local Drug and Therapeutics committee (or similar) to conduct the program and there must be a process of informed patient consent. Continued access to such medications may be limited by the agreement between the Pharmaceutical company and the treating team.
Detailed information on the use of the agent and its associated toxicities should be obtained from the pharmaceutical company that sponsors the scheme or an appropriate source (e.g. the principal investigator at a centre that participated in the trial).
(Council of Australian Therapeutic Advisory Groups, 2015)
- Australian Government and Cancer Australia. Australian Cancer Trials. [homepage on the internet]; [cited 2016 Sep]. Available from: http://www.australiancancertrials.gov.au/.
- Roush W. Dana-Farber death sends a warning to research hospitals. Science 1995 Jul 21;269(5222):295-6 Abstract available at http://www.ncbi.nlm.nih.gov/pubmed/7618095.
- Independent Review Panel for SA Health. Independent review into the incorrect dosing of cytarabine to ten patients with acute myeloid leukaemia at Royal Adelaide Hospital and Flinders Medical Centre.; 2015 [cited 2016 Sep] Available from: http://www.hcasa.asn.au/documents/211-full-report-independent-review-into-the-incorrect-dosing-of-cytarabine/file.
- Council of Australian Therapeutic Advisory Groups (CATAG). Managing Medicines Access Programs: Guiding principles for the governance of Medicines Access Programs in Australian hospitals.; 2015 [cited 2016 Sep] Available from: http://www.catag.org.au/wp-content/uploads/2012/08/OKA10428-CATAG-Guiding-Principles-for-Australian-Hospitals-FINAL-pdf.pdf.