CAR-T Cell Therapies Data Case Studies
Despite a thorough search of scientific journals and healthcare & technology industry sources, we are unable to locate any case studies in which digital technology was used to gather real-world evidence (RWE, aka real-world data) to receive reimbursement for CAR-T cell therapies, Luxturna, or any similar therapy. While there is a widely-held belief that digital tools (wearables, etc.) to harvest and analyze RWE in a variety of applications, including insurance processing, even articles written in 2019 by institutions as prodigious as MIT refer to the use of digital-driven RWE in the future tense. Below is an outline of our findings and research strategy.
- A 2018 report by the Institute for Clinical and Economic Review and the Office of Health Economics on "the potential opportunities and important challenges and limitations that must be addressed in considering options for using RWE [real-world evidence] to inform insurer coverage decisions."
- The paper, in discussing the possibility of insurers tying reimbursement to "real-world clinical performance," describes real-time data collection as a "key opportunity for the future."
- Other references to data collection devices are likewise referred to in the future tense, e.g., "Wearables and the Apple Research Kit, for example, will enable some data collection to become routine."
- This strongly indicates that, at least as of last year, insurers are not using such data collection on any kind of scale.
- Likewise, an MIT white paper states, "It is thought that data from claims, medical records, registries, PROs, and monitors/wearables could be used to provide real-world evidence. The availability of this data will help inform decisions on appropriate payer financing tools."
- The paper also describes a "synthetic case study of a CAR-T treatment ... created and analyzed by the Oncology TAG" to test four different financial system scenarios.
- If any actual case studies were available on the use of real-world data in determining the best reimbursement scheme, it seems extremely likely that MIT would have access to it and would not have needed to create a synthetic case study.
- Another MIT white paper, written in 2019, discusses difficulties in obtaining reimbursement for CAR-T therapies but again describes the use of digital technologies to gather real-world evidence as a future possibility rather than as a present reality.
- According to an article in PYMNTS, some insurers, most notably Humana and John Hancock, are taking interest in data collection via wearables; however, the article indicates that insurers are more interested in data on their clients to help set premiums based on how healthy a lifestyle they live.
- This potentially-invasive use of wearables was also picked up by other media sources, such as Digitalist Magazine.
- Finally, a McKinsey & Company article notes, "The role of RWE in drug development is expanding, driven in part by biotechnology and pharmaceutical manufacturers’ embrace of digital solutions to realize gains in speed and efficiency from innovation." However, the article is focused on gathering data at the clinical trial stage and, in any case, discusses digital advances in the future tense.
From the outset, we had concerns about the level of specificity required for this project. An initial sweep for articles in which various insurance terms, data collection devices (e.g., wearables), and CAR-T cell therapies and/or Luxturna were found together turned up a few lists of innovations in the healthcare industry, but none in which there was a direct connection.
Likewise, a survey of Google's Scholar database did not uncover any pilot programs that fit the research criteria. While there were certainly examples of clinical trials and other studies on the effectiveness of CAR-T and Luxturna based on real-world evidence (example), none for which we could access the full study described a digital technology solution involved in collecting the data. (We allow for the possibility that a case study may be buried in one of the many papers for which we could access only the abstract.)
Therefore, we took a step back and pulled recent articles highlighting the use of data collection devices in a medical insurance context without reference to specific diseases or treatments. This yielded several sources from both healthcare and technology perspectives and many of our useful findings, including research from the Office of Health Economics and MIT. However, it did not yield any useful case studies relevant to the criteria.
Based on the aforementioned report and other sources and for reasons explained in our findings, it is our understanding that using digital technologies to gather real-world evidence for use in determining reimbursement by insurers has not taken place on any scale as yet. That being the case, case studies likely do not exist yet or, if one or more do, are considered proprietary and the results have not yet been released to the public.