Welcome back to TeloChain’s Real-World Healthcare Insights! This is the seventh in a series on recruiting for your real-world (pragmatic) clinical study. We’re homing in on a particular type of study we call the Widely-Distributed Pragmatic Study (WDPS), which bears the potential for actionable insights on how a therapy is used and how well it works over a wide variety of (clinically-appropriate) patients and healthcare settings. We’ve been discussing the data and processes involved in the critical second ‘phase’ of a study—recruiting physicians who in turn recruit and enroll ‘subjects’ (patients). (The first stage is designing the study and formalizing how the study will work in a recipe called a protocol)

To enable, track and report on recruiting-related processes requires a platform: a technology, database and user interfaces. Think of platform as the stage upon which the study’s actions can be recorded, monitored, and in some cases performed (for example, sending a lab requisition or questionnaire to a patient). The platform makes sure the work gets done at the right time, in the right sequence, by the right people.

Today, we dive into what a study platform must do to enable the various roles and tasks related to recruiting, focusing especially on enabling recruiting in the WDPS. This is an emerging study design with, potentially, great opportunity. We welcome your thoughts in the Comments section or directly, HERE.

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Cut to the punchline: A real-world pragmatic study—especially the widely-distributed kind (WDPS)—studies the use and outcomes of treatments over a wide spectrum of physicians, healthcare delivery scenarios and patients. This stands in contrast to the prospective randomized controlled trials used to demonstrate a treatment’s efficacy in a controlled setting with a limited number of experienced practice sites and physicians. The technology platform used for recruiting and qualifying patients and storing information about them in a clinical trial may not meet needs particular to the WDPS. We examine what kinds of activities a platform must enable or support, and what evolutionary steps a pragmatic study platform may need to take to make for a successful and efficient WDPS.

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Why a redesigned platform for WDPS? In prospective randomized controlled trials (PRCTs) and randomized pragmatic trials, the platform must serve “only” a relatively small well-defined number of study sites. By contrast, WDPS can span many dozens or hundreds of ‘sites’ each with one to many physicians. A WDPS platform must enable all these disparate sites to follow the same instructions and protocol, record the same data in the same structure, and track all tasks related to recruiting (and to the actual study, potentially).

Our journey today: With our recruiting map in mind, we’ll ask “What does a platform need to support?” Spoiler alert: People (and their roles in the study), Processes and workflow (sequence of processes performed by people or computers)--as specified in the study’s Protocol. Then we’ll home in on what WDPS-specific elements of recruiting must be supported, and wonder about the value of blockchain-connected platform in a WDPS.  

The recruiting map (bird’s eye view):

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Mind your three Ps for a perfect pragmatic: Good study design is implemented through a cleared shared recipe (Protocol), People and their roles; and a technology Platform. Since Platform enables the Protocol to be implemented by People and processes...let’s take a closer look at each.

Protocol: Bake me a study

I think of the protocol as the detailed ‘recipe’ for the study’s soup to nuts. The recipe should include the following ingredients and clear instructions for how, when and in what order they should be combined:

• The study’s rationale (what gaps in our knowledge about a condition or drug do we hope to fill, and how do we believe the study’s insights will benefit patients?)
• Objectives and research questions
• Definition of the clinical scenario and patient populations it will address)
• Data to be used to identify the candidate patients (with a statement about the strengths and limitations of the types of data to be used); by what inclusion and exclusion criteria the candidates will be screened using that data
• How candidates will be further screened by clinicians to determine their eligibility
• How the screened candidates will be recruited for, educated about and offered the study
• The overall design (prospective vs. retrospective; intervention and comparison groups; description of the intervention(s); timeframe over which the groups being compared will be observed; what measurements will take place; which of these measurements constitute primary and secondary outcomes
• What safety metrics and processes will be established
• What the interim and final reports will include
• If applicable, any compensation for patients, physicians, or participating healthcare delivery entities. Compensation considerations include payment for time and effort, additional support staff, time or materials related to the study, and what portion (if any) of healthplan or patient cost may be defrayed for tests, visits, procedures, drugs or medical devices.

For more on protocols, see endnotes (2) and (3).

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People and their roles in a clinical study

A useful platform must bring together the people involved in a study, enabling them to play their protocol-assigned roles in delivering, monitoring, understanding and responding to the results of the study’s many processes. The primary groups involved in conducting a clinical trial or study include sponsors, investigators and the institutional research board (IRB). Of course, regulatory agencies and patients are also involved! The table below indicates key people and their roles, but is not intended to be a complete list or authoritative definition of roles. You’ll work with your sponsoring organization and CRO for that.

Roles in a clinical trial for a new drug or device are defined in the Code of Federal Regulations (CFR) Title 21 (Food and Drugs), Part 312.3 (Investigational New Drug Application--Definitions and Interpretations). (6)

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Platform: Technology to make it work

Platform is the technology used to manage the elements of the recruiting/ enrollment and study-period phases of the study. While the underlying technology can be anything from paper to electronic with embedded artificial intelligence; we’ll assume it’s electronic so that it can operate on data with rules. There may be separate technical platforms for recruiting, enrollment and patient tracking - ultimately though they must add up to a seamless whole to prevent capture of data or delivery of care from falling ‘through the cracks.’

Platform enables:

• Data to be retrieved, displayed, entered, validated, and queried
• Workflow: Appropriate data to be sent to designated processes and people
• Assignment of tasks to the right people (patients, clinicians, labs, websites such as patient portals) at the right time in the right sequence
• Exception checking (is a task overdue? Are there missing responses in a patient questionnaire?)
• Interface (display and how data may be entered) customized to user role
• Ability of user to respond when appropriate (e.g. for a patient to enter responses to a questionnaire, or physicians to mark that a patient has enrolled)
• Secure assignment of permissions to see and enter data based on roles
• Appropriate levels of privacy, security and confidentiality

The WDPS platform must serve most of the same needs as the PRCT, and even the more-formal randomized pragmatic trials that are limited to one or a few healthcare delivery systems; in addition, it must serve needs that are unique to or different in the WDPS:

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Specifically for recruiting: The platform must track:

• Physician outreaches (if a type 2 study, must send physician the candidate patient list from initial data screen so that physician can mark patients who meet all qualifications, and who enroll)
• Pre-enrollment contacts with physicians, including around information about the study (may also deliver informational content in the form of documents or videos)
• Physician enrollment (including contracts)
• Physicians’ designation of the final candidates (platform must handle any additional data collection needed to qualify patients, such as lab tests or self-reported data)
• Documentation (and perhaps delivery) of study-related patient content that supports their decision whether to enroll in the study
• Patient informed consent (if needed)

‘Track’ or ‘Store?’ The platform need not actually store data; it may be best to store the data elsewhere while Platform enables viewing or adding to the data. For example, the physician or support staff may use a tablet to enter data, but none of this data resides on the tablet, which functions as a portal to that data.

Blockchain?

Because of its inherent tamper-proof enabling and recording of transactions, and its strengths in data security and confidentiality, is blockchain technology a natural fit for the WDPS platform? We think it’s worth exploring, and propose contracts and consent as a good place to start. We welcome your thoughts in the Comments section or you can email us directly.

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Closing remarks | Summary

• While the PRCT platform must serve “only” a relatively small well-defined number of study sites, the much broader WDPS may span dozens or hundreds of ‘sites’ and physicians (just like you expect in the real world these studies evaluate)
• A platform needs to support People (and their roles in the study), Processes, and Protocol. That’s true for any prospective study, whether it’s a PRCT or pragmatic.
• The study’s Platform must be consistent with and facilitate execution of its Protocol, which specified the research questions, the clinical scenarios (inclusion and exclusion criteria), data used to identify candidate patients, how they’ll be further qualified for the study, the materials to be used in helping qualified patients decide whether to participate, the informed consent form, the overall type of design, the outcomes and safety metrics, the statistical analysis plan, and what the reports will look like (and how the report’s metrics will be calculated)
• Platform must enable the study’s People to perform their roles
• We looked at what a study Platform must be able to do, including the requirements particular to a WDPS
• We also raised the question of whether blockchain technology might help facilitate getting data into and out of Platform…and said we’d visit that question another day

Want to know more? Connect with us directly HERE.