Clinical trials are research studies to test new treatments, diagnostic tests, procedures, devices, health service models and other approaches that may improve the health and wellbeing of people living with medical conditions.^1,2

There are different types and phases of clinical trials. Not all trials give people access to a possible treatment and not everyone is eligible to take part. Early-phase clinical trials mainly look at if a new treatment option is safe and how it should be used. Evidence that a treatment works usually comes from later-phase clinical trials, which look at how well a treatment option helps diagnose, treat or manage a health condition.  

Not all clinical trials directly benefit the people taking part. Sometimes new knowledge from clinical trials benefits people at a later stage. 

To learn more about the different types and phases of clinical trials, speak with your health professional or visit the Australian Clinical Trials website. This Australian Clinical Trials Alliance video is also a helpful resource for understanding how clinical trials work. 

Fewer than 5% of rare diseases have an approved treatment.³ Therefore, for many people living with a rare disease, participation in a clinical trial may be the only way to access a possible treatment.^4,5 Rare diseases are often serious and progressive, so taking timely action is critical. Knowing about clinical trials early gives people time to speak with their health professional, find out if they are eligible and decide if they want to take part.

To learn more and stay up to date about relevant rare disease clinical trials, please speak with your health professional. Your health professional can also support you to understand the eligibility criteria for a particular clinical trial.

For more information about things to consider before participating in a clinical trial or other types of health and medical research, please visit the Considerations for Participating in Health and Medical Research page on the RARE Portal. Specific considerations, including a list of useful questions to ask your health professional, can be found in the Consumer Guide for Clinical Trials developed by the Australian Clinical Trials and Consumers Health Forum.

You can search for rare disease clinical trials using the following clinical trial databases:

• Australian Clinical Trials – searchable database of the Australian New Zealand Clinical Trials Registry.
• NIH ClinicalTrials.gov – database of privately and publicly funded clinical studies conducted around the world.

The Sydney Children’s Hospitals Network has produced a video demonstrating how to search for clinical trials using the Australian Clinical Trials database.

For people living with a rare disease, clinical trials can offer hope. At the same time, clinical trials involve commitments and unknowns that are important to understand before deciding if participation is right for you. Here are some key things to consider before taking part:

• Limited places
  Clinical trials only have a small number of places for participants. Interest is often high, so not everyone who wants to take part will be able to participate.
• Strict eligibility rules
  The rules for who can join a clinical trial are often very specific. This means many people who want to take part may not qualify. It helps to fully understand the eligibility criteria early, so you know what to expect.
• Placebo groups (no active treatment)
  Some clinical trials use a placebo (a substance with no active medicine) to compare outcomes between groups. This is called a randomised control trial. Participants are randomly assigned to receive either the treatment or a placebo. You can’t choose which group you are assigned to, so it is important to understand how the clinical trial is designed before joining.
• Clinical trials can change or stop
  Only a small number of clinical trials lead to approved treatments. Clinical trials can be changed or stopped as new information becomes available. This is a normal part of the research process to make sure treatments are safe and effective. Understanding this uncertainty is important when deciding whether to participate.
• Travelling for clinical trials
  Some clinical trials may only be available in another state or country. This can mean being away from home for long periods, managing costs, and dealing with unfamiliar healthcare systems without your usual support network. It is important to consider if these requirements are manageable for you and your family.
• Impact on future clinical trials
  Taking part in one clinical trial may stop you from joining others in the future. It is important to discuss both current and future options with your healthcare team so you can make an informed decision.

Taking part in a clinical trial is a personal choice and will not affect the care and support you receive from your healthcare team. It is important to think about the possible benefits and risks of a clinical trial, as well as how it may affect you and your family emotionally, socially, physically and financially. Asking questions to understand the clinical trial design and discussing options with your healthcare team can help you decide whether participation is right for you.

Taking part in a clinical trial can affect different parts of a person’s life. Having the appropriate support can help. Below is a list of support resources that may be useful:

• Australian Clinical Trials: Concerns about a clinical trial – provides information about steps to take if there are any concerns, complaints or if participants wish to leave a clinical trial.
• RARE Portal: Mental Health and Wellbeing Support for Australians Living with a Rare Disease

Your healthcare team is an important source of support and information if you are considering clinical trials.

You can consider enrolling in a registry.

A registry is a secure database of health information. Registries can help researchers understand where more research is needed and find people who may want to participate in research, including clinical trials.

The Genomics of Rare Diseases Registry at the Garvan Institute for Medical Research is a national registry aiming to better understand rare inherited diseases and connect families with new research opportunities.

There may also be registries specific to your rare disease, either in Australia or internationally. Some registries actively link participants to relevant research studies as they become available. Before enrolling in any rare disease registry, it is important to check that it is linked to a trusted health service, university, government agency or other accredited (officially approved) research institution. It’s also important to make sure you understand how your information will be used. You may also wish to review the Considerations for Participating in Health and Medical Research page on the RARE Portal to help you make an informed decision.