A "Crawl, Walk, Run" Approach to Large Real World Studies
PRA Health Sciences
PRA Health Sciences

Adam Halbridge, MBA, Vice President Business Development Parallel6, spoke at the 2018 SCOPE conference in Orlando. He's added some thoughts to our earlier post on real world studies:

Historically, regulatory approval was the primary focus of drug development, and everything drove toward achieving that milestone. In today’s world, the attention has shifted well beyond product approval and launch. Payers, Regulatory Authorities, physicians, patients, caregivers and sponsors want to know how a product performs in the real world. They’re looking for a clear picture of real drug performance in a diverse population of patients. The emphasis is now on the evaluation of what actually happens regarding safety and effectiveness when patients are exposed to a newly launched product. Product success throughout the development continuum depends on the right team accessing and interpreting the right data to bridge the gap between clinical trials and patient care. Although this may sound complicated, PRA Health Sciences (PRA) guides our clients through the intricacies of real world research throughout the drug development life cycle.

At PRA, we have a two-fold approach to collection and analysis of real world data. We first look at our extensive secondary data assets to determine if we can answer some of the clinical questions from data that we already have. For additional evidence needs, we implement studies to collect specific primary data. In this case, our teams take advantage of new technology to make it easy for patients to participate in these studies. For example, we use an interactive platform on the patient’s mobile device for eConsent, ongoing engagement, wearable data collection and report outcome data on their phone, laptop or iPad. They participate in the research on their own time, in the privacy of their homes. Using innovative and efficient Bring-Your-Own-Device (BYOD) technologies to optimize patient engagement and collect real-time safety events will shift the focus of participating sites from low-value to high-importance activities (such as safety event follow-up). Implementing technologies in active surveillance also provides audit-ready and real-time documentation and traceability of activities to meet any regulatory commitments. Real world case studies and best practices will be reviewed and presented.

We know that before a drug makes it to market it is carefully studied in a randomized clinical trial. These trials are the standard for assessing a drug or medical devices’ safety and efficacy. But what happens outside a controlled clinical environment with “real world” participants? Information gained in a study doesn’t necessarily “translate” to a broader participant group.

Real world studies determine how new drugs or devices perform beyond the scope of clinical trials and take into consideration how a treatment performs with different age groups, races, and gender. Real world data can help fill the knowledge gap.

Today, there are still many stakeholders who take a traditional approach to large real world studies. For example, studies that include participants across many sites in multiple countries still largely rely on paper methods for recruitment, phone calls, and paper logs for weekly and daily end-point capture, as well as cumbersome third-party transcription for electronic data capture.

This approach is costly for sites. Some of those costs include:

  • Currency – Phone calls, transcription service fees.
  • Compliance – Possible errors related to missing events, mistyped end points, attrition, etc.
  • Participant – The need for constant site contact/interruptions which can take away from daily work and family obligations. This is particularly challenging in developing countries where many studies take place.

So how can we save costs, improve study compliance, and make studies more participant-friendly to help reduce drop-out rates? The answer is to adopt a virtual trial model starting with digital qualification and enrollment. Using the internet or a smart phone device we can ease many study barriers. Many countries accept digital enrollment while others have not yet provided guidance. If people are willing to do their banking online why can’t we replicate this model for clinical studies?

Enrolling participants digitally not only initiates the digital record for each study participant but also allows for an agile enrollment process that promotes recruitment for a much larger radius from each site. This enables a faster recruiting process and leverages omni-channel approaches to recruitment in addition to on-site recruitment. This approach significantly drops costs in the reduction of paper, shipment costs, number of overall sites, and possible phone calls needed for recruitment.

Using their own mobile device and customized trial app, study participants can receive push alerts on a daily, weekly, or configured basis to remind them to data capture events such as filling out a daily or weekly diary, or even having a video tele-conference with the site. For those studies where participants need to have blood drawn, they can quickly reference their geo-location on their mobile device and select the nearest authorized clinic. No more missed events or mistyped transcribed end-points. Best of all, this approach allows participants to live their lives with minimal disruption.

Imagine a participant at their child’s birthday party. Maybe a sweet 16 birthday or a Quinceanera. In the traditional study participant model those phone calls may not get answered or returned. But now imagine that the same participant at the same party receives a push notification and between dancing and a refill, opens the study mobile app and clicks through the eDiary in seconds, automatically logging the date, time, and other relevant end-point data directly and securely on their mobile device and then sends back this information in real-time from anywhere in the world.

Best of all, this approach allows participants to live their lives with minimal disruption.

Now imagine that same scenario securely storing the end-point data even when the participant has no signal… then transmitting the secured end-point data, automatically and immediately when signal is restored. At any time, the participant can check their trial progress and study resources while waiting for their food at a restaurant, checking their text messages or confirming a hotel reservation. Using a global enterprise platform, critical study information can be updated in multiple countries and in multiple languages through a web page, allowing study participants from around the world to see relevant information intended only for them. This consumer-friendly approach seamlessly integrates into a participant’s life.

So how do we “Crawl, Walk, Run” into the future using technology? Short of a full study redesign, there are modular, geographic, and demographic approaches:

  • Modular – Digital enrollment for a five year vaccine safety surveillance study may not make sense since enrollment timelines are usually quite efficient, but a mobile app would minimize the need for phone calls and would provide references for approved clinics for blood draws. This would help increase overall compliance.
  • Geographic – Customizing an approach that takes into consideration each study country’s regulatory requirements and unique needs, such as connectivity, average participant radius to sites, historical experience with traditional vs other approaches, etc.
  • Demographic – This approach allows participants the option of enrolling in a traditional arm or a digital arm country by country and age group by age group. This model, however, presents some challenges for participants under the age of 18.

An enterprise platform with the right design and technology elements can significantly drop study costs while allowing stakeholders the ability to “de-risk” the study in real time and across multiple countries all while managing real time measured compliance with the protocol. The advancement of connected devices allows real world studies to easily integrate into participant lives. Most developing countries have great mobile smartphone penetration and while many may not have computers their smartphones keep them connected. So, let’s put to bed those pesky notions about whether or not developing countries have access to the internet.

With a little creative confidence, we can “Crawl, Walk, Run” into the future. By embracing technology, we can significantly improve the drug development process for everyone involved—clinicians, doctors, CROs, drug sponsors, and most importantly, participants.

RWS Polaroid v2r1