December 22, 2022
The life sciences industry is flexing towards innovation in new areas, faster than ever before, and increasing patient care in astonishing ways. Decentralised oncology trials, for example, have shown actual predictive outcome value. We can now measure patient activity, steps, and movement continuously and in real time, which serves as a new potential indicator of treatment effectiveness.1 And, digital therapeutics have emerged as an effective treatment for chronic diseases, including mental illness. The rapid innovation is both exciting and disruptive, so it’s important to strategise accurately for what we can expect in the year ahead.
Prediction 1: the FDA will become more directive and collaborative on guidelines for DCTs.
Traditional protocol designs are built upon years of empirical evidence and consensus among highly rated peer reviewed journals. For new decentralised approaches, this is practically non-existent. This will lead to more pre-protocol design discussions between sponsors and CROs and regulatory bodies seeking input on new and potentially acceptable approaches to, for example, measure digital endpoints. The risk of not having these discussions is to conduct expensive trials only to discover too late that they were measuring the wrong endpoint, in the wrong way, or with a tool or technology the FDA does not accept.
Curavit is currently conducting a decentralised trial for a digital therapeutic that began with discussions with the FDA. In this case, the indication was well-known, treated for decades by traditional medical interventions, and copious peer reviewed data to call upon. However, since the route of administration itself was new – i.e., digital rather than in pill form – all stakeholders recognised how crucial it would be to involve the FDA early and often. Specifically, they discussed which patient survey format would be best, which scores to include or exclude, and acceptable means of data capture.
As this collective body of experience continues to proliferate, 2023 will see additional FDA guidance. It is likely that the FDA will address various elements associated with DCTs more granularly than ever. These may include best practices for measuring digital endpoints, biomarkers, and telemedicine and ePRO (Patient Reported Outcomes), as well as risk factors most concerning to the FDA. For instance, at a recent presentation to the Society of Quality Assurance 2022 Annual Meeting, the FDA Office of Regulatory Affairs Bioresearch Monitoring Division Director Anne Johnson emphasised that DCTs are largely reliant on electronic data integrity and therefore subject to firmly established 21 CFR Part 11 compliance.2
Prediction 2: More clinical trial services will become remote, beyond just patient monitoring.
We will see continued increases in remote clinical trial activities and services in 2023, such as remote monitoring, remote site staff training, remote site activation visits, remote quality management meetings, remote safety management meeting,s and more.
These unprecedented clinical trial activities represent a fundamental shift that is still unfolding. There was a time when Source Data Verification (SDV) by any other means than frequent and extremely costly on-site CRA visits was unthinkable. We now see continuing proof points that conducting SDV remotely can not only save time and travel costs, but can also increase quality.
Curavit recently worked on a 28-site, US-based trial where more than half of the source data verification was conducted remotely with secure, remote access to the eTMF and EDC systems. Instead of using generalised, on-site CRAs, Curavit’s therapeutic area specialists handled this significant task from afar, prioritising specific expertise over physical proximity. This had a significant impact on data quality and speed, as well as reductions in travel and costs. Curavit was also able to conduct additional Q/C measures simultaneously, which otherwise would have been a separate process.
Prediction 3: AI innovation will expand its application to enhance patient experience.
Although “Enquire Here” buttons are common for large patient enrolment providers, recent advances in Natural Language Processing (NLP) – a form of artificial intelligence or AI – allow for far greater, more meaningful, and effective patient engagement. Technologies such as BERT3 and GPT-3 catapult NLP capabilities beyond basic processing, into the realm of language intelligence. Potential applications will surpass current-day screening chat bots, which automate FAQ workflow and are only capable of answering the most basic patient questions.
In 2023, expect to see AI innovations improve enrolment agility and, most importantly, the patient experience in a clinical trial. For instance, NLP will respond to patient concerns or enquiries with more humanised responses and alert clinicians faster and automatically. This will, in turn, improve patient interest, trust, engagement, enrolment, and clinical trial retention. In addition to patient benefits, AI will enable actionable real-time metrics and analysis and a near-immediate feedback loop on the investigational drug’s impact on patients’ daily lives.
Prediction 4: digital therapeutics companies will focus on the difficult last mile to commercialisation.
Historically, digital therapeutics (DTx) companies have directed about 95% of their effort into gaining FDA approval and 5% into market access strategy. In 2023, this will shift to a 60%/40% split, with DTx companies having an earlier focus on the commercialisation process, while simultaneously working toward earning FDA approval.
This ’last mile’ is completely unchartered territory for DTx companies. They face new obstacles bringing their therapeutics to market – for example, physicians’ lack of awareness of the DTx and their inability to easily prescribe them. There are still many unknowns around getting DTx products on the formulary and how to prescribe them to patients – doctors can’t just write a prescription and send patients off to the local drugstore when prescribing the use of an app.
And, whereas traditional drug companies may spend up to $300 million to launch a product, most DTx startups don’t have the budget to spend on commercialisation strategies such as building a field team, equipping marketing teams with supporting analytics technology, and initiating patient support programs to help patients learn to use these software-based therapeutics.
Beyond 2023 – the Sovereign Patient ID
The concept of patients owning their own data has been around since the advent of electronic health records. However, 2023 will see new technologies such as blockchain and smart contracts bring the Sovereign Patient ID concept closer to reality. This will give the patient the ability to own their own identity and associated data on their own devices, as opposed to their identity being created and stored separately by each service provider: a state of error prone, redundant inefficiency. The life sciences industry will collaborate and share lessons learned with other verticals that are currently exploring the same concept, such as “Self-Sovereign Identity” (SSI).4
This problem will not be solved in 2023, but progress will be made.
References
1. Gresham G, Hendifar AE, Spiegel B, Neeman E, Tuli R, Rimel BJ, Figlin RA, Meinert CL, Piantadosi S, Shinde AM. Wearable activity monitors to assess performance status and predict clinical outcomes in advanced cancer patients. NPJ Digit Med. 2018 Jul 5;1:27. doi: 10.1038/s41746-018-0032-6. PMID: 31304309; PMCID: PMC6550281.
2. Chapman, J. An FDA Perspective on Decentralized Clinical Trials: Part 1. July 28, 2022.
3. Devlin, J, Chang, M.W. Open Sourcing BERT: State of the Art Pre-Training for Natural Language Processing. November 2, 2018.
4. Preukschat, A., Reed, D. Self-Sovereign Identity- Decentralized Digital Identity and Verifiable Credentials. Manning. 2021.
About the Author
Dr. Pam Diamond is co-founder and chief medical officer of Curavit, a virtual contract research organisation (VCRO) that designs and executes decentralised clinical trials. She is a board-certified, Boston-based otolaryngologist/head and neck surgeon who was affiliated with Harvard Medical School (HMS) and Massachusetts Eye and Ear Infirmary (MEEI) for over 30 years. At Curavit, Dr. Diamond provides medical and scientific expertise and oversight to the design and execution of Curavit’s decentralised clinical trials. She received her medical degree from the University of Rochester School of Medicine and completed her otolaryngology/head and neck surgery residency at Harvard Medical School/MEEI.
