Applied Clinical Trials
A countdown of the top 10 developments that have most influenced the ePRO industry over the past decade.
As the CEO of PHT Corporation, a leading provider of electronic clinical outcome assessment (eCOA) and electronic patient reported outcome (ePRO) systems used in clinical research, I'm often asked what I believe have been the most important engines driving ePRO industry growth over the last decade or so. I've identified 10 key drivers, described here in reverse order of importance.
10: EDC adoption
The growth of electronic data collection (EDC) began about a dozen years ago. Companies using EDC studies for all data other than diaries, take three to nine months to lock the database. That's unacceptable—and why major pharmas always consider ePRO if they are using EDC and endpoints in patient or clinician outcomes.
9: Critical Path Institute (C-Path)
Two influential groups within the C-Path Institute have been major influencers: the Patient-Reported Outcome (PRO) Consortium, http://c-path.org/PRO.cfm, and the Electronic Patient-Reported Outcome (ePRO) Consortium, http://c-path.org/ePRO.cfm. The PRO Consortium is significant because it is a collaboration with FDA, pharmaceutical companies, and ePRO providers.
The ePRO Consortium is a group of ePRO providers that includes Almac, Bracket, CRF Health, ERT, ICON, and PHT. The consortium provides electronic technologies for trials capturing clinical outcome assessments as endpoints; and ensure that new instruments from the PRO Consortium are validated in various electronic formats.
These two consortiums are working together to lower both the cost and the risk of using ePRO for all clinical research stakeholders.
8: ePRO integration with objective measurement devices
The advent of objective medical devices such as the PEF meter for asthmatics and the glucometer for diabetics have been landmarks. Combining objective information from these devices with the subjective information gathered by electronic diaries significantly enriches what we call "the true voice of the patient."
7: Non-compliance with paper diaries
Patient non-compliance when using paper diaries has been outlined in a number of articles. One of these articles, published in the British Medical Journal, http://www.bmj.com/content/324/7347/1193, notes that when doctors ask patients to recall recent health experiences such as pain and fatigue, "[patients'] recall is unreliable and rife with inaccuracies and biases." The problem is even more severe when patients use paper diaries.
Concerns about the use of unsupervised paper diaries are justified. The FDA has taken note and expressed its concern in some of its guidances.
6: Increase in the number of trials collecting PROs
The fact that the FDA, European Medicines Agency (EMA), and the Pharmaceuticals and Medical Devices Industry, (Japan) are all interested not just in what physicians say but in how patients feel is driving more and more PRO-based labeling claims. Given today's regulatory and economic environment, sponsors are also focused on finding meaningful ways to differentiate their drugs. Having more information from patients helps.
5: ISPOR Task Force Report
This report, written several years ago, notes that when sponsors migrate from paper to electronic data capture, the change can be categorized as minor, moderate, or substantial. The degree of change requires different types of validation. Because the report provides pragmatic guidance for PRO scientists to follow when migrating from paper instruments to electronic ones, it has speeded the adoption of ePRO.
4: Improved site and sponsor satisfaction
Many of us remember the days when ePRO was new and didn't work as well as it does today. As sponsors gained experience—particularly large pharmas looking for competitive advantage—ePRO site and sponsor satisfaction improved and adoption increased.
3: Merck case study—ROI justification
The Merck insomnia study showed the significant return on investment ePRO delivers.
The trial was executed to help Merck better understand the scientific and business benefits of ePRO versus paper for trials with patient-driven endpoints. The trial was designed so that an already-approved drug was used and patients were randomized; using both paper and electronic arms. Merck deduced that they could have 56% fewer patients using electronic data gathering and achieve exactly the same study power.
Similar reduction in data variance in trials using electronic diaries versus paper diaries has been published in other indications, including urinary incontinence, asthma, and chronic constipation, and concretely demonstrates the significant ROI ePRO delivers.
2: PRO guidance and other regulatory documents
The FDA PRO guidance covers the importance of PROs and clinical outcome assessments in research as well as the importance and benefits of implementing measures electronically. The same material is covered in the EMA reflection paper on health-related quality of life about collecting data electronically. These documents, as well as the eSource draft guidance documents, http://www.fda.gov/downloads/Drugs/.../Guidances/UCM239052.pdf, that came up earlier this year, all strongly support electronic data capture.
All these guidelines demonstrate that regulators now expect to see the use of electronic systems rather than paper methods to collect clinical outcomes.
1: Leveraging smartphones
The number one driver of ePRO: smartphones and the proliferation of consumer technologies. The PHT ePRO/eCOA system collects data on smartphones, with great success.
Author's Note: The full article is available at http://goo.gl/KH3To.
Phil Lee is the President and Chief Executive Officer of PHT Corporation, 500 Rutherford Avenue, Boston, MA, e-mail: firstname.lastname@example.org.