Published in Lab and Life Scientist June/July issue, authored by Sue MacLeman.
International Clinical Trials Day, held in May, commemorates Lind’s trials into the causes of scurvy and recognises that clinical trials are central to discovering and proving the efficacy of new treatments. But we’ve come a long way since 1747.
Today, Australia is a world-leading clinical trials destination and the sector has developed significantly in recent years, with changes in structure and growth in clinical trials activity levels. Approximately 1360 new clinical trials were commenced in Australia in 2015 and this figure has been growing at around 5% per year since 2010, outpacing that of the US, UK and even the overall global average growth rate.
The clinical trial sector is estimated to have contributed approximately $1.1 billion in 2015 to the Australian economy as direct expenditure or investment, and industry sponsors are estimated to contribute $930 million in funding for clinical trials in Australia each year. The vast majority of this is in the form of foreign investment flowing into the country from multinational medical device, biotechnology and pharmaceutical trial sponsors.
Considering this, the breadth of Australia’s clinical trials sector — level of economic activity and both the economic and health value derived from the conduct of trials — is substantial. Historic volumes suggest that Australia is still experiencing growth in the total number of trials conducted. But international competition for clinical trials is intensifying.
With other countries and populous regions like Asia striving to improve their clinical competitiveness, the question remains whether Australia can hold its dominant position. In a globally competitive marketplace, we need to not only defend our specific areas of strength, but work towards more sustainable competitive advantages. This can only be achieved through national and collaborative approaches, engaging all sector stakeholders — governments, sponsors, clinical investigators, health system managers and industry — and we’re already seeing this in action.
Trial recruitment — developing a national and global solution
Historically, Australia has faced challenges in patient recruitment and economics. Our geographically dispersed and comparatively small patient base creates difficulty in recruiting sufficient patients for trials that require large patient numbers. However, in more recent years, Australia has been increasingly competing with East Asia, Eastern Europe and South America in the conduct of trials, with those countries offering lower design complexity, lower requirement in equipment and procedures and larger participant volumes. The number of participants per trial site in Australia is generally lower than these direct competitor countries, which means the opportunity for growth in the number of clinical trials will be more challenging.
Combined with the general lack of effectiveness in referrals between sites and other healthcare entities, clinical trial costing remains complex and variable. Due to Australia’s well-structured health system, a number of diseases are routinely managed in the primary care settings (GPs) and these facilities lack recruitment data linkages to local clinical sites, which in Australia are predominately located in major hospitals. These factors impact the economics of the clinical trial site, as the set-up cost is a fixed cost that can only be spread across a limited number of patients.
These barriers call for a national planning of clinical trials programs — and there’s an opportunity for Australia to combine its reputation for patient diversity and high quality, with the development of new technologies and innovation. We’ve seen this in the success of ClinTrial Refer — a new smartphone and web-based platform that connects doctors and patients to recruiting trials across research networks. The integrated app is supporting over 19 current derivative pilot apps, links to the wide network of Australia New Zealand Clinical Trials Registry and enables doctors to be linked with patients for current trials in less than one minute. The initiative received funding from MTPConnect’s Project Fund Program.
Since the campaign was launched in 2013 in New South Wales and Canberra, ClinTrial Refer has been associated with over 60% increase in trials recruitment and has received overwhelming support from Australian pharmaceutical companies participating in the initiative that expressed the need for national approach.
Improving Australia’s attractiveness
Despite the shift to Asia and emerging markets, there are opportunities for Australia to increase the number of medicine clinical trials being undertaken locally. Australia can differentiate itself as a high-skilled, cost-effective and efficient clinical trial destination; by targeting international and local medical device, biotechnology and pharmaceutical companies that are seeking certainty around cost and time, world-class infrastructure, access to an ethnically diverse patient base and good clinicians, to ensure better data tracking and quality assurances.
Australia is globally competitive in trials in complex or rapidly changing disease areas, and performs better relative to advanced health system markets in oncology, infectious diseases, musculoskeletal, nephrology and ophthalmology trials. These trials require high-quality data and treatment environments, and high levels of key opinion leader involvement, particularly in early stages. Australia's Phase 1 specialised service providers — CMAX, Linear Clinical Research and sites such as Prince of Wales, Nucleus Network — are highly regarded in terms of their quality and speed of delivery, supported by streamlined processes and private ethics committees through Bellberry. This has attracted inbound investment and activity specifically from small and medium-sized entities in the Asia Pacific region.
A collaborative network of clinical trial units extends across Australia, and all states are courting international pharmaceutical companies, urging them to bring their early-stage trials to Australia. The Clinical Trials: Impact & Quality (CT:IQ) project is an example of this — a new national initiative that promotes a whole-sector approach to improve the quality, efficiency and impact of clinical trials in Australia. The initiative, funded by MTPConnect, brings together multiple stakeholders including Bellberry, Australian Clinical Trials Alliance (ACTA), the National Health and Medical Research Clinical Trials Centre (NHMRC CTC) and The George Institute.
Supporting future growth
It’s exciting to see the work already being undertaken by the public and private sector participants, and the calls for greater collaboration between the sectors to improve Australia’s clinical trials environment, with a view to improving health outcomes and increasing international investment in Australia.
The 2018–19 Budget has active reforms for the sector and demonstrates the Australian Government’s understanding of the value clinical trials and research, and how it supports better health outcomes for Australia. The government has delivered investment of $2.4 billion in growing Australia’s research science capability, and an additional $1.9 billion in Australia’s national research infrastructure over 12 years to ensure that we have the tools to develop and commercialise first-to-market products. As part of a National Health and Medical Industry Growth Plan, $248 million will be provided to expand the successful rare cancers, rare diseases and unmet-need clinical trials program. A ‘national clinical trials front door’ will also be established to better coordinate the Australian clinical trials sector.
A critical and welcome announcement for the sector relates to the reforms of the Research and Development Tax Incentive (RDTI) — these reforms that will encourage additional investment in R&D and ensure RDTI’s long-term sustainability. The exclusion of clinical trials from the $4 million annual cap on RDTI cash refunds illustrates the known value of clinical trials for both economic and health outcomes. The impact for R&D intensive companies is also recognised with an intensity component and increased cap to $150 million, and these measures are expected to increase R&D activity in Australia. MTPConnect is working with the Australian Government and sector on the rollout of these measures.
To date, MTPConnect has funded 34 national and industry-led, dollar-for-dollar matched projects, with over 200 consortium members — of which four projects focus on facilities to produce products at low volumes for clinical trials, four projects to assist patient recruitment for clinical trials and one project focused on upskilling graduates with trials-specific workforce skills.
By building on areas of strength and pursuing further structural improvement with a national focus, Australia could realise a considerable increase in annual expenditure by the sector. If Australia can maintain its trial growth rate, it could surpass $2 billion of annual expenditure in the next 10 years and create more than 6000 new high-skilled jobs in a sustainable sector, driving broader health and economic outcomes, and ultimately providing patients with early access to new and potentially life-saving treatments.
All this, because James Lind decided to experiment with oranges and lemons.