November 2012 issue of Life Science Leader
Like it or not, the wheels of the Patient Protection Affordable Care Act 2010 (commonly referred to as “ObamaCare”) are well in motion. One of the key requirements is that by 2014, there will be nationwide use of electronic health records (EHRs) — a systematic collection and digital record of electronic health information about individual patients or populations. EHRs are a critical component of the “digital healthcare ecosystem” that captures patient data, shares that data with healthcare providers, and improves access to that data. Other emerging technologies that create opportunity for engagement around health and wellness include mobile health (mHealth) and social media exchanges.
“It’s important to understand that emerging technologies such as mobile health, remote monitoring, data analytics, and others are enabling healthcare reform this time around,” says Paulo Machado, founder and CEO of Health Innovation Partners LLC, a strategic consultancy that works with a range of healthcare stakeholders to use technology in developing next-generation business models and solutions. “The digitization of healthcare is fueling a Healthcare Renaissance that will lead us to the triple aim of better care, better health, and lower cost.”
OPPORTUNITIES IN CLINICAL TRIALS
One healthcare area that is being driven forward by technology is clinical trials. While clinical trials determine whether new drugs are effective on different groups of people, it can be complex to look at a variety of subsets of people. The ubiquity of smartphones — and the incredible amounts of data they collect — might change all that.
“There are incremental and radical opportunities that present themselves with digital technology,” says Craig Lipset, head of clinical innovation at Pfizer. “It can change how we perform studies, engage patients around recruitment, capture data, and monitor data. Each of these steps can be reinvented with digital platforms and make clinical research a more transparent and integrated part of healthcare.”
Once the healthcare information is seamlessly integrated across healthcare stakeholders, patients can be recruited faster and more efficiently. Researchers would be able to increase the likelihood of finding “better” candidates for their trials. “Theoretically, one could enter search criteria that would find how many patients exist that meet the criteria. The patients could be screened digitally to determine who is eligible for the trial-based specific criteria, then the investigators/physicians could be brought into the process,” says Machado.
Since patients are already familiar with how their smartphones operate, once they are recruited into a clinical trial, real-time data capture becomes possible. This is a paradigm shift from conventional electronic diaries that often require providing patients with an unfamiliar handheld device exclusively for use in the study and which often provide one-way data input. Now, rather than visiting a clinical trial site, patients input the data to their mobile phones. This enables trials to occur wherever the patient is located. “Using mobile platforms to support capture of electronic diary data (e.g. ePRO [electronic patient reported outcomes]) in clinical studies is a real opportunity to leverage a technology that people are already familiar with — their smartphones,” says Machado. Both ePRO and mHealth applications capture data, but most mHealth apps also confer some benefit and value to the patient, which in turn improves compliance in using the app to provide the data.
Sharing the collected data among all the trial stakeholders is an opportunity as well. “Typically, data capture and analysis of clinical trial data has been performed in buckets or silos, but emerging data management and analytics approaches would allow all interested parties access to the data for improved collaboration and faster turnaround times,” says Machado. And, once a trial closes, analyzing the data becomes a more efficient process because the data has been captured and reviewed throughout the study, he adds.
COLLABORATING IN THE DIGITAL ECOSYSTEM
From the patient to the provider to the recruiter, digitizing healthcare will recast the roles within a clinical trial and realign business models as the stakeholders will share data and collaborate in new ways. For example, rather than just collecting patient data and walking away armed with the information, researchers will engage with patients at the commencement of the study to understand their needs and share information at the end of the study that could address those needs. Or, pharmaceutical companies can share the information with insurance companies to help the payers understand what medications work best for certain patients so that proper reimbursement occurs.
mHealth also will facilitate collaboration between atypical partners. “The landscape of clinical trials will be very different in the next decade in terms of how they are done and who the players are,” says Lipset. “For a pharmaceutical company, this means being willing to collaborate with organizations that typically would be competitors, like other pharma companies. Pharma can come together to make the process more efficient because it ultimately benefits everyone.”
As proof that this movement is afoot, 10 of the biggest names in drug development are joining forces to speed up new drug creation, and the resulting nonprofit aims to work with CROs to tackle some of the most vexing troubles in pharmaceutical R&D. TransCelerate BioPharma is the moniker for the collaboration, which joins Abbott Laboratories, AstraZeneca, Boehringer Ingelheim, Bristol-Myers Squibb, Eli Lilly, GlaxoSmithKline, Johnson & Johnson, Pfizer, Roche’s Genentech unit, and Sanofi.
In a release, the conglomerate established five targets for action: developing a shared user interface for investigator site portals, mutual recognition of study site qualification and training, developing risk-based site-monitoring approaches and standards, developing clinical data standards, and establishing a comparator drug supply model. “Most of these targets are what I would consider ‘technology-enabled,’” says Lipset. “Those targets with the strongest technology alignment are around clinical data standards as well as a shared investigator site portal.
“TransCelerate BioPharma marks an important demonstration of collaboration to drive innovation,” Lipset continues. “There is an appreciation that there are problems we simply can’t address alone anymore, and there is no reason to do so because they are shared challenges. We are competitors in the eyes of Wall Street, but we can work together to address challenging areas.”
TransCelerate is not centered on mHealth or digital, but instead on collaboration across industry R&D organizations to improve the drug development process. Of the five “workstreams” live at launch, several are strongly technology-enabled, such as the investigator portal. The work around improved data standards is very consistent with other data efforts in healthcare — standards for data help make data “liquid” (i.e. easily shared and transferred across systems) between research and healthcare and ensures that users can readily share and exchange data. “When data and health information is liquid, we can enable a future state of enhancing trial recruitment and study efficiency. Because then we can readily enable identification of very specific patients for individual studies (i.e. based on detailed health information, genetic information, etc.) and enable patients to opt-in to share their data for research,” says Lipset.
TIME, MONEY, AND THE FDA
And while Machado wouldn’t put a number to the time and cost savings possibly achievable using emerging digital technologies, he is optimistic that both could be cut in half — at least. “Maybe more, depending how aggressively the technology is adopted by life sciences companies and the regulatory bodies,” he says. “We’re talking about optimizing the clinical trial process, not changing trial durations. Recruitment and analytics — the flexible components — will change, but the amount of time the patient needs to take the medicine won’t change.”
Change could, however, be coming from the FDA with regard to mHealth. At the end of 2010, the FDA issued a draft guidance on electronic source, or e-source, in clinical trials. This means that the original record of a data element is captured electronically and must be followed with a detailed audit trail. The FDA promotes eSource in clinical studies because it will help to eliminate unnecessary duplication of data, reduce the opportunity for transcription errors, promote the real-time entry of electronic source data during subject visits, and ensure the accuracy and completeness of data (e.g., through the use of electronic prompts for missing or inconsistent data).
The industry is awaiting a final draft of guidelines for mobile medical apps, but it appears that the FDA is focused on mobile medical apps that assist in the development of clinical decisions for health issues. “The FDA is being forward-thinking as it puts out guidance about using electronic tools as source documents in clinical trials,” says Lipset. “The industry needs an FDA that evolves and is creative, and so far, the agency has shown progress toward meeting that goal.”
90% OF CLINICAL TRIALS TO BE DIGITIZED
Just as the FDA evolves in working with mHealth, so too must life sciences companies. Both Lipset and Machado agree there is a learning curve but that use of digital technology in healthcare is only at the starting line. “Patients are used to using devices such as smartphones, which are the types of tools that bring value to the clinical trial process,” says Lipset. “We can collect data at unprecedented levels, develop better medicines, and better target medicines to particular patient groups.”
The U.S. will not have a completely digital ecosystem this decade, points out Machado. He does believe that 80% to 90% of what needs to be digitized will be completed by 2030. “Until then, we will continue to see clinical trials that are part digital and part manual,” he says. “We are not making the move to digital just for clinical trials. We are doing this to improve the quality of life. Better care, better quality, better value. Those three things will have a profound positive effect on clinical trials.”
Pfizer Forges On With mHealth
Last June, Pfizer set out to incorporate mobile technology in a virtual clinical study of its overactive bladder drug Detrol LA (tolterodine tartrate), with the aim of attracting 600 patients from 10 states across the U.S. The remote trial of Pfizer’s was the first randomized clinical trial that patients could participate in entirely from home by using mobile phones and Web-based technology. The trial screened patients online, provided multimedia content regarding informed consent online, and enabled extensive data capture from patients online (including efficacy and safety).
The hope was this approach would save time, produce better data, and increase patient compliance, but the trial was halted after Pfizer failed to persuade sufficient numbers of patients to take part. The project does not represent a failure for, or withdrawal from, the use of the Internet or social media for patient recruitment, says Lipset. “We routinely use the Internet as a channel for recruitment in our studies and will continue to do so wherever it is appropriate,” he wrote on the company’s blog.
One reason the trial wasn’t a complete success was because Pfizer was trying to do too much too fast. “We were testing a number of digital tools at once: online recruitment, screening, and consent; at-home study drug delivery; mobile- and Web-based tools for all data reporting; online identity verification; and a centralized investigator site,” Lipset says.
But, all hope is not lost. Pfizer plans to relaunch the trial in 2013.