Pharmaceutical companies face a very real and immediate challenge to achieve sustainable Research and Development (R&D) models and produce affordable treatments. The rising cost of drug development, highlighted in Deloitte’s return on pharmaceutical innovation research, is a key part of this challenge which could be addressed through transforming the way clinical trials are conducted.
Clinical trials are the most expensive and time consuming part of drug development, indeed Phase III trials alone are estimated to account for over 90 per cent of total development cost. The current approach involves patients traveling considerable distances to study sites for treatment and ongoing monitoring activities.
This approach to clinical trials, established over 65 years ago to develop a drug for tuberculosis, has remained largely the same. As a result there has been limited response to recent developments in science, technology and the rising expectations of patients, payers and regulators. The aforementioned developments are either drivers of increasing cost, for example the extra effort needed to demonstrate drug value to payers on top of clinical-effectiveness, or opportunities to become more cost effective. A ‘virtual clinical trial’ methodology represents one of these opportunities.
The concept of a virtual clinical trial uses electronic health records and mobile health technology (apps, monitoring devices etc) to conduct each stage of a clinical trial, from patient identification and recruitment, through to measuring clinical endpoints and adverse reactions, from the comfort of a patient’s own home. Key benefits include:
- Enhancing enrolment – often the longest stage of a clinical trial with almost 80 per cent of trials failing to meet initial targets. The convenience of a virtual methodology alone will increase numbers of patients willing and able to enroll. Also, electronic health records can help identify increasingly targeted trial subjects and online patient support networks could be used more to raise awareness of trials and directly recruit subjects.
- Maintaining engagement – more than 30 per cent of Phase III trial subjects can become disengaged and drop out due, for example, to the inconvenience of traveling to study sites, or the complexity of the trial design and data collection. Virtual clinical trials could remove the need for frequent travel to study sites and automate data collection, increasing patient engagement and retention.
- Reducing risk – data from remote monitoring devices could be accessed by trial investigators in real time. Opening up possible efficiencies in data cleaning, which could move to an on-going process rather than cyclical. It could also facilitate an adaptive clinical trial approach, de-risking drug development by allowing improvements in trial design based on accumulating data. Decisions to terminate a drug’s development could also be made faster, improving patient safety and reducing expenditure on failed trials.
- Improving ROI – by increasing clinical trial efficiency the overall cost should decrease, patients should be able to access treatments sooner and drug companies ought to be able to lengthen their market exclusivity, thereby increasing ROI.
While there has been interest from pharmaceutical companies to adopt a 100 per cent virtual trial methodology, in reality there has been limited progress. Largely because it involves: radical changes in procedures; a reliance on unfamiliar technology; and a flexible regulatory environment. A more realistic expectation is the gradual decentralisation of trial activities in which a single study centre is responsible for oversight, receiving and storing data and distribution of drugs to patients. The centre would then collaborate with multiple trusted health services and health care practitioners local to the trial’s subjects to administer treatment and monitor progress. Telehealth could also provide a direct link to the trial subjects when necessary. Decentralisation however, has its own significant challenges:
- Collection, transfer and storage of data – will pharmaceutical companies be able to manage multiple sources of data inherent in this approach? Contract research organisations (CROs) may be better placed to handle this task.
- Trust and understanding – the need to build patient trust by making trial processes and progress transparent and for a unified, patient friendly, interface to promote compliance.
- Regulations and legal implications – concerns around patient privacy, data ownership and monitoring adverse events are already being seen in other areas of innovation and have potential to put the brakes on progress.
- Scalability – will there be a limit to the type of trial that can become decentralised? The nature of some therapy areas may require complex monitoring equipment or come with significant risks that cannot be catered for in the community or home.
A high risk, high reward approach would be to apply a decentralised methodology to the most costly development phase of clinical trials – Phase III. However, this may also be the most difficult phase to implement due to large patient populations and complex trial design. A lower risk, lower reward approach would be to apply a decentralised methodology when expanding indications for existing products, where safety profiles are well understood or in Phase II trials, which contain fewer subjects and are less complex than Phase III. This would speed up the go/ no-go decision and therefore, the critical path of the drug development process.
Whichever approach is chosen pharmaceutical companies, and third party contractors involved in running clinical trials, need to gain experience with virtual methodologies to help tackle the complexities and cost of drug development.