What I wish I knew before starting a clinical trial

You’ve piloted your new idea to detect, prevent or treat disease and the results look promising. Congratulations! The next step may be to conduct a clinical trial, where the safety and efficacy of your health intervention is tested in humans.

To help you progress your idea, public health scientists and medical researchers from the University of Sydney have come together to share their top tips on starting and managing a clinical trial.

Plan, plan plan

Before starting your trial, build a project plan or study manual that outlines all the study processes and logistics for how the study will be completed – from identification of participants, recruitment, data collection and management (including what variables are being collected and how they will be recorded) to a detailed timeline. Name members of the team responsible for each task. Make sure you allow plenty of time to secure ethics approval. And make sure you track your budget from the get-go – many studies fail to meet recruitment targets simply because they run out of money.

- Dr Joanne Shaw, psycho-oncology and health communication researcher, Faculty of Science.

Know what question you are asking

Make sure you define your hypothesis clearly and collect only what you need. Work with a statistician who is experienced in the conduct, implementation and reporting of clinical trials. The quality of the data you generate at the end of the trial and the resulting impact the data have will reflect the thought and effort you put in at the beginning.

- Dr Rebekah Puls, HIV/AIDS researcher and Clinical Research Manager, Brain and Mind Centre.

Come up with creative ways to keep participants engaged

Adherence to the allocated intervention during a clinical trial is critically important. So try to think of imaginative and creative ways to maximise participant engagement and adherence. For example, you could enhance the intervention experience through health gaming, such as a video-game biofeedback for exercise, and encourage participants to discuss their progress through an eCommunity, such as Facebook.

- Professor Joshua Burns, neurological and musculoskeletal researcher, Faculty of Health Sciences.

Document decisions

For decisions that are likely to impact on any aspect of data analysis or study reporting, document the rationale for the decision at the time of decision-making. Ask yourself: ‘Why was an amendment made?’, ‘Why was a particular participant eligible/ineligible if not per protocol?’.  This will save a lot of angst when it comes to analysis as you will not remember why some decisions were made.

- Dr Joanne Shaw, psycho-oncology and health communication researcher, Faculty of Science.

Be enthusiastic and consultative

To help build engagement with the study, be enthusiastic and consultative with collaborating researchers and key stakeholders, including consumer representatives. Take on board their comments and address any questions in a timely manner.

- Associate Professor Anne Cust, cancer prevention researcher and SOAR Fellow, Sydney Medical School.

Establish relationships with key support staff

Establishing positive and productive working relationships is particularly important for multi-site research. Get to know key staff who will be supporting your research, such as pharmacists (if dispensing investigational products), psychologists, laboratory services staff (for bloods or samples taken during the clinical trial), local GPs or medical practitioners who may have patients participating in the clinical trial.

- Dr Kirsten Morley, mental health and substance use researcher, Sydney Medical School.

Clean data regularly

Conduct data cleaning on an ongoing basis – don’t leave it until the end of the study, as systematic errors that can compromise the study data integrity can develop over the course of the study if not addressed on an ongoing basis.

- Dr Joanne Shaw, psycho-oncology and health communication researcher, Faculty of Science.

Be prepared for participants to drop out

Some degree of participant drop out is difficult to avoid, particularly in the substance use and mental health field. Build this into your planning and put in extra efforts to obtain follow-up data from all participants as you go.

- Dr Kirsten Morley, mental health and substance use researcher, Sydney Medical School.

Build extra time into your plan

Don’t expect the process to run smoothly. Things can and do go wrong, but if you have spent time considering the potential risks, building extra time into your planning, approval and start-up phase, to allow for ethics, contractual and regulatory approvals, you will save yourself stress later on. 

- Dr Rebekah Puls, HIV/AIDS researcher and Clinical Research Manager, Brain and Mind Centre.

Know the standards

Remember, it’s the principal investigator’s responsibility make sure the trial adheres to all legislation from the Therapeutic Goods Act, state and federal privacy acts and the NSW Health Directives to Good Clinical Practice. Read, review, review again and ask for advice. Following these standards will ensure the safety and wellbeing of your participants.

- Jacqueline Everett, Clinical Trials Risk and Governance Manager, University of Sydney.

More information

In Australia, clinical trials are governed by national ethics guidelines and codes of conduct, such as the National Statement on Ethical Conduct in Human Research and the Australian Code for Responsible Conduct of Research. For more information on legal requirements as well as more tips, download the Australian Government’s Clinical Trials Handbook or visit australianclinicaltrials.gov.au. The NHMRC also offers free online training modules to ensure quality research outcomes and participant safety.