Tim Davis

The role of Real-World Evidence (RWE) in assessing the value of new drugs is increasingly important. RWE typically consists of many types of Real-World Data (RWD) captured by patients directly reporting on their own health outcomes as they take a new drug in their everyday lives.

These patient reported outcomes (PROs) are captured in observational studies, pragmatic trials, and commercial programs. Each of these types of RWD program involves different methods and RWD capture challenges, which are being overcome by novel technological solutions.

In previous blogs we explored this in relation to 

. In the last of this three-part series, we look at how the healthcare industry is starting to harness patient generated RWD as a valuable by-product of commercial programs.

		Pharmaceutical companies are increasingly implementing commercial programs that engage with patients to offer support as they take new treatments in the real world. These patient support programs usually occur after the new drug has been licensed and is available on the market. Commercial programs range from simple apps to more advanced programs that help patients manage their health and maintain their daily routine while taking the treatment.

		By setting up these programs, pharmaceutical companies are aiming to improve patient support and protect the integrity of their brand. An important by-product is the rich source of RWD that they generate.

		How can technology support RWD capture in commercial programs?

		Capturing data in this manner enables companies to engage with patients and support them on a personal level while protecting their brand. One of the by-products of a commercial program that employs electronic data capture is humanistic RWD. This can be used to enhance health outcomes, educate patients, and facilitate discussions between patients and care providers.

		An example of an electronic data capture platform effectively supporting commercial programs and RWD capture is TARGET My Hives, a digital health network set up to support everyone affected by chronic urticaria (hives). 

		The community that uses this digital health network can interact with others suffering from chronic urticaria to reduce feelings of isolation. The network enables patients to set goals for themselves, share their results with each other, and connects them with physicians to help manage their condition. Discussions on digital networks like these can generate valuable information about patients’ specific conditions and treatment journeys that hold the potential to support RWD programs.

		This blog series has outlined how digital platforms are enhancing the efficiency, cost-effectiveness, and reliability of RWD capture in observational studies, pragmatic trials, and commercial programs.

		Such RWD programs improve the quality of RWE, allowing healthcare stakeholders to make more effective and informed decisions that will benefit patients’ lives.

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In this three-part blog series, we look at how electronic platforms can support and enhance data capture in several types of RWD programs: observational studies, pragmatic trials, and commercial programs.

Utilizing Real-World Data Captured During Commercial Programs

A primary focus of the healthcare industry is to generate Real-World Evidence (RWE) to better inform decision making. A key source of RWE comes from patients reporting on their own health outcomes as they participate in Real-World Data (RWD) programs, including observational studies, pragmatic trials, and commercial programs. 

In the first of this three-part blog series, we explored how RWD generated by observational studies can be harnessed effectively. Here we look at pragmatic trials-which have recently re-emerged to generate valuable RWD-and assess how technological solutions are ideally placed to support RWD capture in this setting. 

	Pragmatic trials are gaining momentum in post-marketing research as they are still interventionalâyet carried out in real-world settings. Designed to evaluate the effectiveness of treatment interventions in routine general practice, pragmatic trials provide data that can be generalized and applied in real-world settings. As such, they are an increasingly valuable source of RWD. 

	What role does technology play in pragmatic trials? 

	In this digital age, patients have expectations for digital tools to be intuitive and user friendly, like those they can download and use in their everyday lives. Electronic data capture methods can support pragmatic trials through enabling patients to use their own devices, known as a ‘Bring Your Own Device’ (BYOD) approach. Digital platform solutions allow sponsors to easily collect patient reported outcomes and health-related quality of life assessments in real-time, in a regulatory compliant manner. 

	Patients benefit from the ease-of-use of a BYOD approach in pragmatic trials. They can also access a broad range of engaging and motivational digital content to further improve their experiences. For example, they can view videos about their condition, ask questions through scripted chat-bots, and even integrate other personal devices, such as activity monitors, into the data capture platform if appropriate. 

	Depending on the requirements of the sponsor, some or all of these capabilities can be implemented using the digital platform. The flexibility of a digital platform means they can be adapted to meet the specific needs of each trial, allowing study teams to balance internal validity versus real-world effectiveness. Electronic capture solutions help to ensure the collection of high quality RWD supported by a full audit trail to aid future regulatory approval processes. 

	BYOD in pragmatic trials is highlighting the importance of digital capture technology in post-approval research. Sponsors can now assess the effectiveness of a treatment in an environment that can be generalized and applied to real-world settings, while operating in a regulatory compliant manner that does not increase burden on patients or compromise the quality of the data. 

	Look out for the third blog in this series ‘Utilizing Real-World Data Captured during Commercial Programs’ next week!

Generating a Rich Source of Real-World Data Through Pragmatic Trials

The capture of patient generated Real-World Data (RWD) is increasing the influence and value of the ‘patient voice’ in RWE and healthcare decision making. The electronic capture of RWD is facilitating this by overcoming the challenges presented by paper-based capture. 

In this three-part blog series, we look at how electronic platforms can support and enhance data capture in several types of RWD programs: observational studies, pragmatic trials and commercial programs. We reveal the challenges of RWD capture in each setting and how technological solutions can overcome them. 

Here, we focus specifically on observational studies, including why they are an important source of RWD, and why electronic data capture platforms are essential to ensuring effective RWD capture in this setting.

	In post-marketing research, observational studies are used to assess health outcomes in a group of patients as they take a new treatment. They are non-interventional by design, recording what happens to patients via patient reported outcomes (PROs). These studies often involve capturing patient data from geographically diverse populations over long periods of time.

	What are the primary challenges of observational studies?

	A substantial burden can be placed on patients to complete regular and timely data entries using paper forms. This burden must be carefully balanced; too little and the integrity of the data will be compromised, while too much will tip the scales and may impact patient outcomes.

	Electronic data capture platforms used on patients’ own devices (smartphone, tablet, etc.) can help to reduce the burden placed on participants in observational studies. Since they are already familiar with their own devices, patients find it easier and quicker to provide accurate data entries. This approach enables studies to offer a light level of support, limiting intervention and the introduction of bias, while being widely accepted by ethics committees. Digital platforms also enable the use of data protection and privacy tools to ensure security and adherence to clinical rigor.

	Electronic data capture platforms overcome the limitations of paper-based data entries. For example, they allow reminders to be sent to participants to complete data entries on time, are globally scalable, and are adaptable to different healthcare systems and languages to ease RWD capture wherever patients are in the world.

	Digital platforms ensure the efficient capture of high quality RWD in observational studies by reducing the burden placed on patients and overcoming the limitations of paper-based data collection, generating a rich source of RWE that represents the voice of the patient and informs meaningful healthcare decisions.

	Check back next week for Part 2 of the series, ‘Generating a Rich Source of Data through Pragmatic Clinical Trials.’ 

In this three-part blog series, we look at how electronic platforms can support and enhance data capture in several types of RWD programs: observational studies, pragmatic trials, and commercial programs. 

Overcoming the Challenges of Real-World Data Capture in Observational Studies

	Electronic data collection has grown rapidly over the course of the last ten years and has already emerged as a common asset in clinical research for the completion of Clinical Outcome Assessments (COA), including the outcomes captured directly from patients, known as patient reported outcomes (PRO). The global growth of mobile technology over recent years has provided a new and efficient method of accessing patients, creating an ideal platform for capturing eCOA assessments.

	At its core, mobile technology is able to support the diaries, scales and questionnaires required for capture of PROs during trials, however the advantage mobile ePRO holds over paper and other types of ePRO devices lies in the provision of a simple intuitive interface for patients, that facilitates the 2-way communication so important to keeping patients engaged including educational and motivational content delivered via messaging and in-app notifications throughout their participation in a clinical trial.

	The use of mobile ePRO is now becoming commonplace, and the barriers to adoption have been broken down through the incorporation of data security measures to protect patient data, the validation of a huge range of instruments from paper entry to mobile, and the reassurance of regulatory acceptability – both through public statements such as comment last year from the FDA that the “BYOD (bring your own device) approach does not contravene 21 CFR Part 11”* and, perhaps more substantially, the use of mobile ePRO to collect primary outcomes data in numerous product approvals in Europe and the USA.

	The move away from ePRO device provisioning and towards a BYOD approach (where patients use their own mobile devices) is currently a topic of hot debate. For me, part of the cause of this debate is the mistaken belief that when introducing BYOD into a protocol, enrollment of patients with a specific type of device is the 

 option. That approach would be offering choice in the same way that Henry Ford did to the choice of color for his Model T car – you can bring any device to this study, as long as it’s an android with Ice Cream version12.2 OS – this clearly isn’t practical as it would seriously limit study eligibility criteria.

	As the only vendor with tangible experience in this field, the reality of BYOD in clinical trials is very different. We have been providing ePRO solutions for BYOD trials for many years and almost always introduce an element of provisioning in pre-approval studies. The key is to be able to identify the specifications of the patient’s device and ascertain whether it meets the requirements for the individual study, if not then a suitable device can be provisioned. I like to think of it being a flexible or ‘choice driven’ approach. The provisioning rate for such studies is decreasing year-on-year as the uptake of more advanced mobile technology increases (did you know in the US almost ¾ of adults over 65 now own a mobile phone?**) and in pre-approval studies we see rates anywhere from about 50% provisioned down to as low as 10%, depending on the study population. This provides 2 significant benefits, not only does it enable the patient to use their own device, the one they selected from all available options, but it significantly reduces the cost and logistical complexity of device provisioning for sponsors.

	I set out 10 years ago to simplify the process of collecting patient data during clinical trials through the use of mobile technology. I couldn’t have predicted back then just how far mobile technology would advance and how it would become such an integral part of daily life for people around the world. Now mobile eCOA is proven to be a highly beneficial and versatile option for data capture in clinical trials. It has been shown to significantly improve data quality of PROs and has been used – both through a provisioning and a BYOD approach – to collect primary outcomes data in Europe and US drug approvals. I believe mobile is set for further expansion and growth as the industry escalates the adoption of existing mobile ePRO as the standard approach to self-reported clinical data.

http://www.pewinternet.org/data-trend/mobile/cell-phone-and-smartphone-ownership-demographics/

Electronic data collection has grown rapidly over the course of the last ten years.

Author | Tim Davis