Lloyd Price
- Feb 29

Platform Trials emerging as new study types for evaluating Digital Health solutions

Exec Summary:

A platform trial is a specific type of clinical trial designed for efficiency and adaptability, making it well-suited for evaluating rapidly evolving interventions like digital health solutions.

Platform trials are not a complete replacement for clinical trials, they offer a valuable addition to the clinical research toolbox, particularly in the fast-paced and data-rich landscape of digital health.

They provide a way to accelerate the development and optimisation of effective digital health solutions while ensuring their real-world effectiveness and broader applicability.

What are Platform Trials?

Traditional Clinical Trials: Focus on comparing one treatment to a placebo or standard care in a strictly defined timeframe.
Platform Trials: Test multiple treatments against a shared control group. They have an adaptive design, meaning they can add or remove treatments, or modify the trial as it progresses, based on results.

Why Platform Trials are Ideal for Digital Health

Rapid Evolution: Digital health solutions change rapidly. Traditional trials can be outdated by the time they are completed. Platform trials, with their adaptive nature, can keep pace with updates and new versions of digital tools.
Multiple Interventions: In digital health, various interventions might be worth exploring (different app features, behavioural nudges, etc.). Platform trials can simultaneously test these, increasing efficiency and speed.
Real-World Data: Digital health solutions generate data in real-time. Platform trials can integrate this data for ongoing analysis, allowing quicker identification of what's working and what's not.
Patient-Centricity: Platform trials can be designed to enroll a broader, more representative population, mirroring the real-world users of digital health apps. This can enhance the generalizability of results.

Examples:

COVID-19: Platform trials were instrumental in rapidly evaluating treatments for COVID-19, such as the RECOVERY trial in the UK.
Mental Health: Platform trials are being explored to test various digital mental health interventions to find the optimal approaches for different individuals.
Chronic Diseases: Platform trials can evaluate digital health tools for managing chronic conditions like diabetes or heart disease.

Challenges and Considerations

Complexity: Platform trials are more complex to design and execute than traditional trials.
Statistical Considerations: Analyzing data from multiple interventions and adapting the trial requires sophisticated statistical methods.
Regulatory: Regulatory pathways for platform trials, especially those involving digital health might need adapting for these novel designs.

Platform trials offer a flexible and efficient approach well-suited to the rapidly evolving nature of digital health solutions. As platform trials become more common, we can anticipate rapid acceleration in the development and optimisation of effective digital health interventions.

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Platform Trials v Clinical Trials

'Platform trials are different in that they are open ended, meaning new interventions can be added, assessed, and removed as time goes on, without having to specify what they might be at the start.

Compared to a more traditional intervention focussed trial design (is this drug better than usual care / placebo?), a platform trial can be better thought of as disease focussed (what is the best drug for this disease?).

As well as being able to add new interventions as time goes on, platform trials also have the flexibility to update the control, or ‘usual care’ group as the study progresses. This is useful for when, for example, the platform trial shows a drug it’s testing to be much more effective that ‘usual care’, then that drug can be rolled out to benefit patients right away and become the new ‘usual care’ against which all new drugs are tested in the trial, creating a potential cycle of improvement.'

Source: https://www.phctrials.ox.ac.uk/platform-trials-an-explainer

Challenges in generating evidence in digital health

'The traditional methods of evidence generation, such as randomized controlled trials (RCTs), are not the best fit for digital health solutions. Digital health solutions evolve quickly, and drug trials demand long-term stability and conformity. Moreover, these trials often have strict criteria for who can participate, which might not reflect the diversity of real-world populations that will actually use the technology in everyday life. Additionally, these trials can be very costly and time-consuming.

Solutions and novel approaches to generate evidence in digital health

Simulation studies evaluate new digital health solutions safely, efficiently, and cost-effectively before introducing them to the real world.
Real-world evidence (RWE) generated by digital health solutions adds value in the early stages of evaluation and during monitoring after introduction to the market. For example, Germany's Fast Track process for digital health solutions highlights the growing importance of RWE in supporting the diagnosis and treatment of diseases as well as patient-initiated lifestyle changes.
Platform trials (PTs) are a novel study type that may be useful for evaluating quickly evolving digital health solutions as these trials are designed to be adaptive, allowing for interventions to be modified or changed completely over time'

Source: https://www.roche.com/stories/evidence-in-digital-health

Future of Platform Trials for Digital Health

The future of platform trials for digital health is promising and holds the potential to revolutionize how we evaluate and develop these rapidly evolving solutions. Here are some key trends and predictions:

Increased Adoption:

As the benefits and efficiencies of platform trials become more widely recognized, we can expect increased adoption across the digital health landscape.
This will likely be driven by regulatory bodies adapting their frameworks to accommodate platform trials and by researchers and developers embracing their potential for faster and more comprehensive evaluations.

Focus on Specific Areas:

Platform trials are likely to be particularly effective in specific areas of digital health, such as:
Mental health: Testing various digital interventions and tailoring them to individual needs.
Chronic disease management: Evaluating the effectiveness of digital tools in managing conditions like diabetes or heart disease.
Preventative healthcare: Studying the impact of digital tools on promoting healthy behaviors and preventing disease.

Integration with AI and Machine Learning:

Artificial intelligence (AI) and machine learning (ML) can play a crucial role in platform trials by:
Analysing massive datasets generated in real-time to identify trends and optimize interventions.
Predicting patient outcomes and tailoring interventions accordingly.
Identifying new potential interventions based on data analysis.

Enhanced Patient Engagement:

Platform trials can be designed to improve patient engagement through:
Remote monitoring and data collection using digital tools, reducing the burden of in-person visits.
Gamification elements to increase participation and adherence to the study protocol.
Real-time feedback and insights for participants, allowing them to actively participate in their health journey.

Challenges and Considerations:

While the future looks bright, some challenges need to be addressed:

Complexity: Ensuring rigorous design and execution of platform trials requires specialized expertise.
Data Sharing and Privacy: Addressing data security and privacy concerns will be crucial, especially with the integration of real-world data.
Ethical Considerations: Ensuring ethical conduct and informed consent in the context of adaptive designs and real-time adjustments.

Overall, platform trials are poised to play a transformative role in the future of digital health. Their ability to accelerate innovation, improve efficiency, and enhance patient engagement will be crucial in developing effective and accessible digital health solutions for a healthier tomorrow.