We started our conversations on the digital opportunity for clinical trials at the HealthXL Global Gathering in September 2019. And after many months of exchanges, debates and conversation, our long awaited report on Clinical Trial Optimization is finally here!
Why is this important? Some stats and facts.
- 30% drop-out rates in clinical trials and delays due to recruitment. 11% of sites fail to enroll a single patient and 37% under enroll.
- The rise of niche drugs has led to more pressure to identify from smaller (albeit more strictly defined) pools of patients with rare conditions. Luckily, however, patients with rare diseases tend to be part of strong communities dedicated to advancing scientific research.
- Going ‘paperless’ in trials is not opportunistic enough (e.g., electronic case report forms have 100% adoption across trials today, whereas smartphones only have 45%, wearables 29% and social media a mere 6%).
- Mortality, clinical symptomatology, hospitalization, and laboratory biomarkers have traditionally been the types of end-points being measured in patients during clinical trials. Some of these outcomes could take months or years to collect. New guidelines now recognize the importance of patient-centric endpoints like quality of life and function. New digital biomarkers are needed to quantify these.
- Perception of acute vs chronic disease: For chronic conditions that patients live with long-term, digital tools may have a higher value due to the perceived ‘lowstakes’ of their disease. In comparison, oncology patients may feel that digital tools are too light touch for the severity and acuity of their condition.
Our Q1 report is completely expert led, oozing with opinions from the catalysts of the industry including:
What is the real future for digital in clinical trials? Has digital made clinical trials cheaper and faster? Has regulation changed to support these ‘new age’ trials? Do we understand what digital signals correspond to clinical end points? These questions have inspired this HealthXL report on Clinical Trial Optimization. We explore the hype vs reality of this opportunity under 5 key themes that were handpicked by HealthXL members. Below we summarize our key takeaways:
Can Digital Strategies improve Clinical Trial Recruitment and Retention?
Digital is helping pharma reach new cohorts of patients where they share their stories: online. It is also enabling pharma to co-create new strategies with healthcare providers that allow patients to partake in trials from their homes. However, it is too early to prove that a full online-only engagement modality will dramatically increase patient enrollment into clinical trials or retain patients in clinical trials. We also want to steer away from the use of the word ‘siteless’ trial and promote the use of ‘decentralized’ or ‘virtual’ trial.
Can Digital Health Lower Costs of Clinical Trials?
We believe technology will enable ‘cost shifting’ rather than gross cost reduction, by helping to better manage the cost of routine tasks and logistics. This may lead to new business models or additional profits based on expanded data collection. Short term cost escalation will yield longer term benefits.
Will Digital Health Expand Clinical Endpoints for Clinical Trials?
Even if we lack best practices at this moment in time, digital biomarkers allow for continuous collection of real-world patient data in clinical trials. We are attempting to understand what digital biomarkers are, and which ones can be captured for various indications, and through what devices.
Who is Driving the Changes to Regulation Necessary to Adopt Digital Tools in Clinical Trials?
We believe that pharma, consortia of pharma (e.g. Neuroscience Consortium) and digital health innovators will lead the way through R&D and piloting.
What Areas are Ripe for Optimization with Digital Health?
Much hope has been placed on the utilization of digital health technologies to manage and treat chronic conditions. Cardiometabolic conditions are regarded as one area ripe for disruption due to the high burden as well as large populations impacted along with many devices aimed at that sector currently. Pharma is increasingly interested in how similar offerings could be expanded towards more high risk and rare conditions in neurology and oncology.
Therapy areas: Cardiology, Oncology, and Neurology
Clinical Trial Pathway: Recruitment, Retention, Logistics
Grounded in Data and Evidence
We’ve identified 119 (and counting) start-ups working to optimize clinical trials along the pathway (protocol design and review, patient recruitment, site selection, patient engagement, data collection, as well as operational management and risk monitoring) to help reach the promise of faster, cheaper, better. We have also picked out ongoing studies, pilots and initiatives already in play to make tech enabled trial optimization a reality.
Next Steps: Our Future Expectations
- Patient Identification, Recruitment, Site-matching and Retention: It is likely that in the near future we will see studies and market data that demonstrate which digital strategies are successful in increasing patient recruitment and retention in clinical studies. We will see the decentralization and reduced dependency on physical sites, but we will never do away with sites, or with investigators.
- Costs: Anything innovative or new, especially in research and industry, will not lead to immediate cost savings but may in fact increase the cost. Digital health will probably add more costs and work for clinical researchers. However, collecting more data will ultimately add greater value to the company as a whole. Short term, pharma should focus on cultural change to set the scene for these longer term benefits.
- Digital Biomarkers and Novel Endpoints: In time we will understand the viability of wearables and sensors in collecting meaningful clinical endpoints and whether this is more cost effective than standard practice. FDA regulation on how to collect and use digital endpoints in clinical trials can be expected in the coming years.
- Regulation: Pharma and digital health innovators will lead the way. With a number of pharma organizations and digital health companies already setting a precedent by working with regulators (e.g. Biofourmis - Novartis - FDA) we may expect future collaborations of this kind to be somewhat fast-tracked. Expect Germany and China to follow on the heels of the US is achieving regulatory milestones.
- Areas ripe for action: Pharma partnerships with start-ups and non-traditional healthcare organizations to bring technology they lack onto their workbench. An opportunity to reframe the place of digital in oncology trials and treatment to make greater impact and deliver better patient outcomes based on learnings from cardiometabolic interventions.
Do you have experience leveraging digital solutions to improve clinical trials? Are you a PI looking to identify an appropriate digital solution to help you overcome an investigation challenge? Do you agree/disagree with our findings?We’d love to hear from you, and of your initiatives in translating hype to pragmatic reality.