Volume 22, Number 4 - December 2019

Clinical trials: what they are, why they’re important, and where to find them

By
JDRF

JDRF is the leading global organisation funding type 1 diabetes (T1D) research. JDRF Australia is built on a grassroots model of people connecting in their local communities, collaborating regionally for efficiency and broader fundraising impact, and uniting on an international stage to pool resources, passion and energy.

Our mission is to accelerate life-changing breakthroughs to cure, prevent and treat T1D and its complications.

To accomplish this, JDRF has invested nearly $2 billion since our inception. We collaborate with academic institutions, policymakers, and corporate and industry partners to develop and deliver a pipeline of innovative therapies to people living with T1D.

Our staff and volunteers in seven countries are dedicated to advocacy, community engagement and our vision of a world without T1D.

Introduction

Clinical trials are an essential part of research – they are the gold standard for evidence-based evaluation of a new intervention, whether it’s a drug, therapy, device, test or methodology.  
Around 1,000 new clinical trials are initiated in Australia each year.​1​ Despite a world-class medical research and healthcare infrastructure, these trials still struggle to recruit enough participants. A 2014 survey found that only 20% of clinical trials in Australia meet their recruitment targets before the proposed deadline.​2   
One of the key reasons for low recruitment rates is a lack of awareness.​2-5 Both healthcare providers and the public are often unaware which clinical trials are available, what’s involved in them, or how to participate.2   
As trusted advisers who are central to peoples’ decision making, healthcare providers play a crucial role in raising this awareness among the public. By familiarising yourself with what clinical trials are, why they’re important, and where to find them, you can help those you work with to better understand this vital aspect of research – and what it means for them.

What is a clinical trial?  

A clinical trial is any research study in people that aims to evaluate a health-related intervention. Clinical trials are a critical part of the research pipeline – a new treatment, device or therapy cannot be made available to the public until it is shown to be safe and effective, first in cell or animal-based studies (discovery research) and then in human trials. 
There are four main phases of clinical trial, and enough evidence must be gathered in each phase before progressing to the next: 
Phase I ​trials are usually the first time an intervention has been tested in humans. They assess safety and tolerance in small groups of healthy volunteers.  
Phase II ​trials involve larger groups of people from the target population – the people who will be using the intervention once it is approved. A phase II trial usually assesses whether an intervention works as intended, as well as gathering further evidence of safety. 
In ​Phase III, ​the efficacy of the intervention is further evaluated in larger groups of people – anywhere from a few hundred to several thousand. A phase III trial usually compares the new intervention to another treatment or a placebo, to determine if it is equivalent or superior. It’s also an opportunity to monitor less common side effects that may not have been picked up in smaller groups.  
Phase IV ​trials often involve monitoring the long-term effects of an intervention in a real-world setting, and take place after the intervention has been approved 
 
After phase III, the evidence that has been gathered is submitted to the relevant regulatory body – for example, the Therapeutic Goods Association (TGA) for medications. If the regulators determine that the benefits of the intervention outweigh the risks, they will approve it for use in the target population. If not, they may request that further trials are carried out.  
Take the example of researchers who have discovered a new antibody that delays the onset of type 1 diabetes (T1D) in mice, by preserving beta cell function. Once enough evidence of the antibody’s safety and effectiveness has been gathered in the discovery phase, researchers will begin human studies with a trial in healthy volunteers (Phase I). Then, the antibody might be evaluated in family members of people with T1D who are showing early signs of beta cell loss (Phase II). Finally, researchers might compare the antibody with a similar preventative treatment that’s already on the market (Phase III).  
This entire process of making a new intervention available to the public, from discovery through to approval, takes around 10-15 years.​6    

Why clinical trials are so important  

In the mid-1990s, two landmark clinical trials – the Diabetes Control and Complications Trial and the Stockholm Intervention Study – found that maintaining blood glucose levels within an optimal range can delay or prevent the complications associated with type 1 diabetes.7, 8 These studies transformed the understanding of T1D worldwide, and saw a shift from the conventional treatment strategy of the time (one or two injections of insulin per day with daily self-monitoring) to the intensive blood glucose management that has now become standard practice. Major shifts in medical knowledge like this wouldn’t be possible without large, evidence-based studies like clinical trials. 
Today, there are many more innovative treatments, devices and preventative strategies in development, poised to make a big impact on the daily lives of people with T1D. But before these ideas can become reality for the T1D community, researchers rely on volunteers to participate in clinical trials.  

Motivators and barriers to participation 

There are many reasons people participate in clinical trials – they may be motivated by altruism and a desire to help others and move science forward, or they may take part to access new treatment or management options.​Those who do participate often find empowerment in the opportunity to take an active role in their own healthcare, and learn more about their condition.9 Clinical trials also give people access to an increased level of care from health professionals who 
are experts in their field, and there is evidence that individuals who take part have better outcomes than those with the same condition who don’t participate.​10, 11   
But despite the benefits of participating, recruitment rates for clinical trials in Australia are generally low. The reasons for this are wide-ranging – some of the problems are systemic, like a lack of coordination between clinical research centres at national and state levels.​ 2 Networks like the ​Type 1 Diabetes Clinical Research Network​ play an important role in bringing together researchers, clinicians and participants to increase the awareness, coordination and impact of clinical 
research.  
Additionally, people often have personal barriers that may prevent them from enrolling in a trial. They may be influenced by how uncomfortable or risky an intervention is perceived to be, how accessible the trial site is, or how much of a time commitment is involved.​2  
But often, people simply don’t even know that a trial exists, what’s involved, or how to enrol. This lack of awareness – among both healthcare professionals and the public – is consistently highlighted as one of the greatest barriers to clinical trial recruitment, and can only be overcome by increasing the volume and impact of communication about clinical research.​2, 3

Talking about clinical trials  

Healthcare providers play a key role in raising awareness of clinical trials among the people they work with. Providing information to help them better understand clinical trials – including what they are, what’s involved, and the benefits and risks of participating – can help them to make informed decisions about getting involved in research.  
Here are some key points to cover when talking about clinical trials:​2   
Participating in a clinical trial is entirely voluntary, and you can withdraw from a trial whenever you choose. 
There are many kinds of clinical trial – they don’t all involve testing a new drug. You might be asked to take a dietary supplement, try a different exercise routine, or even just answer some questions.  
You don’t have to pay to take part in a clinical trial, and many larger trials will reimburse expenses like travel and parking. 
Clinical trials involve medical teams that offer support and monitoring throughout the trial. You will always have access to a member of the trial team to ask questions, or let them know about any problems. 
Clinical trial teams take every precaution to minimise the risk of harm, but no clinical trial is risk-free. It’s important to talk to your healthcare team about any trials you are considering taking part in.

How to find clinical trials 

Trial Finder — Type 1 Diabetes Clinical Research Network  
Trial Finder is the best place to find clinical trials in Australia that are specific to T1D. It lists trials that are currently 
recruiting in each state, with links to more information for those interested in taking part.  
JDRF Game Changers 
JDRF’s Game Changers is a program for people who are interested in finding out about upcoming T1D trials in Australia, 
and hearing regular updates of exciting developments in T1D research. 
Australian New Zealand Clinical Trials Registry 
A registry of clinical trials across all areas of medicine, primarily those taking place in Australia and New Zealand.  
Australian Clinical Trials 
Information and resources for health professionals and the public, as well as a search engine to find clinical trials in 
Australia.  
ClinTrial Refer 
A clinical trial database with a website and app to search for open trials in Australia.  
Clinicaltrials.gov 
A database of clinical trials from around the world, created and maintained by the U.S. National Library of Medicine. 
Australian Centre for Behavioural Research in Diabetes 
The ACBRD carries out research that investigates the behavioural, psychological and social aspects of diabetes, and has a 
regularly updated page listing their current clinical trial opportunities.  
NHMRC Clinical Trials Centre 
The NHMRC Clinical Trials Centre aims to improve practices, policy and outcomes around clinical trials, and has links to 
currently available research opportunities.

References

1.

Clinical Trials Capability Report. The Australian Trade and Investment Commission (Austrade); 2018. Available from: https://www.austrade.gov.au/ArticleDocuments/2814/Clincal-Trials-Capability-Report.pdf.aspx

2.

Scoping and Analysis of Recruitment and Retention in Australian Clinical Trials. Department of Health; 2016. Available from: https://www1.health.gov.au/internet/main/publishing.nsf/Content/EE207D978A44E4B8CA257FA90081B212/$File/EY%20Final%20Report%20-%20Recruitment%20and%20retention%20in%20Australian%20clinical%20trials%2030%20June%202016.pdf

3.

Leiter A, Diefenbach M, Doucette J, Oh W, Galsky M. Clinical trial awareness: Changes over time and sociodemographic disparities. Clin Trials. 2015 Jun;12(3): 215-223.

4.

Building Australia’s Clinical Trial Expertise. Medicines Australia; 2016. Available from: https://medicinesaustralia.com.au/wp-content/uploads/sites/52/2017/02/MA-Issues-Brief_6_ClinicalTrials.pdf

5.

Analysis of Recently Conducted Clinical Trials. Department of Health; 2015. Available from: https://www1.health.gov.au/internet/main/publishing.nsf/Content/EE207D978A44E4B8CA257FA90081B212/$File/Analysis%20of%20Recently%20Conducted%20Clinical%20Trials%20Report.pdf

6.

Corr P, Williams D. The Pathway from Idea to Regulatory Approval: Examples for Drug Development. In: Lo B, Field M, editors. Conflict of Interest in Medical Research, Education and Practice. Institute of Medicine (US); 2009; Available from: https://www.ncbi.nlm.nih.gov/books/NBK22930/

7.

Nathan D. The Diabetes Control and Complications Trial/Epidemiology of Diabetes Interventions and Complications Study at 30 Years: Overview. Diabetes Care. 2014 Jan; 37(1):9-16.

8.

Reichard P, Nilsson B, Rosenqvist V. The effect of long-term intensified insulin treatment on the development of microvascular complications of diabetes mellitus. N Engl J Med. 1993; 329:304-309.

9.

Estcourt S, Epton J, Epton T, Vaidya B, Daly M. Exploring the Motivations of Patients with Type 2 Diabetes to Participate in Clinical Trials: A Qualitative Analysis. Res Involv Engagem. 2016 Dec; 2:34.

10.

Majumdar S, Roe M, Peterson E, Chen A, Gibler W, Armstrong P. Better outcomes for patients treated at hospitals that participate in clinical trials. Arch Intern Med. 2008; 168(6):657-662.

11.

Hallstrom A, Friedman L, Denes P, Rizo-Patron C, Morris M. Do arrhythmia patients improve survival by participating in randomized clinical trials? Control Clin Trials 2003; 24(3):341-52.

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