July 21, 2010 by Craig Lipset
The rise of the e-patient brings great opportunities — and some unique challenges — to clinical research and the conduct of clinical trials.
Rise of the e-Patient
Much has been written elsewhere about the rise of the e-patient. The white paper “e-Patients: How They Can Help Us Heal Healthcare” defines the e-patient as “a new breed of informed health consumer, using the Internet to gather information about a medical condition of particular interest to them.” Tom Ferguson MD, lead author of the e-patient white paper, described the e-patient as “empowered, engaged, equipped and enabled.”
Some key points about the e-patient:
e-Patients Are Everywhere
According to Pew Research Center’s Internet & American Life Project:
- 61% of American adults look online for health information (the “e-patient”)
- 60% of e-patients say they or someone they know has been helped by following medical advice or health information found on the internet (just 3% say they or someone they know was harmed)
e-Patients Are Organized
Last year The Society for Participatory Medicine was formed to organize a field defined as “a movement in which networked patients shift from being mere passengers to responsible drivers of their health, and in which providers encourage and value them as full partners.”
e-Patients Are Not a Fad
Earlier this year, the National Library of Medicine hosted their 2010 conference “The ePatient: Digital and Genomic Technologies for Personalized Health Care”. Leaders from academia and government agencies gathered at the National Institutes of Health (NIH) to discuss the implications of genomic, digital and wireless technologies – increasingly patient-facing tools – in the context of comparative effectiveness, innovation and personalized medicine.
In many ways, the e-patient is the future. Their rise brings disruptive change in the delivery of healthcare, and brings similar implications for clinical trials.
e-Patients Are Organizing Around Clinical Research
Today, many patients participate in a variety of online communities. As the New York Times noted earlier this year (“When Patients Meet Online, Are There Side Effects?”), in some of these communities patients have moved beyond chatting or posting free text. Patients are increasingly sharing structured, longitudinal health information with one another. This structured information is databased and used for searching, but is also aggregated and available for research.
In these communities patients are essentially initiating their own research (prompting buzzwords such as Research 2.0 and Crowdsourced Research). They are filling out forms that look very much like the case report forms (CRFs) used in academic clinical research studies. But the patients are not waiting for academics or industry to take interest in their disease – they are self-organizing, collecting data, and analyzing the resulting database. In some cases, these communities have even published results in peer-reviewed journals.
We may be seeing the tip of the iceberg in patient communities publishing scientific findings. Earlier this month, one organization published a paper entitled “Web-Based, Participant-Driven Studies Yield Novel Genetic Associations for Common Traits” in PLoS Genetics. The study was described as a novel participant-driven research program, building a community of patients sharing both self-reported clinical data and genetic information.
Patients sharing data in a community are conducting a form of clinical research, but this should not be confused with a clinical trial. Clinical trials involve prospectively defined protocols (including eligibility criteria and other rules) and participants are often randomly assigned to one of several treatment options. Data generated by patient communities are an important step in the continuum of evidence and knowledge, but may be difficult to compare to a randomized clinical trial.
e-Patients: Opportunities (and Challenges) for Clinical Trials
While e-patients themselves are gathering in communities and may be generating their own research findings, they also bring potential opportunities and challenges for traditional clinical trials.
Patient community initiated research is different from a clinical trial (as noted above), but the roles and tools may be very similar:
- Both rely on an engaged patient as a participant
- Both rely on the participant being interested in self-tracking and reporting
- Both rely on the participant being willing to share data
#1. The engaged e-patient as a clinical trial participant
The most often-cited opportunity with e-patients in clinical trials is around engagement – reaching these patients, informing them about clinical trials, recruiting them to participate. There are many companies offering services in this space, and a seemingly endless number of conferences on the topic. As an example, Pfizer has a website to help patients match for trials, is using YouTube to share information with potential participants, and is building an online community to increase awareness and connect patients to investigators while ensuring privacy. Certainly online patient engagement is one tool to reach patients in support of trial recruitment. But this will not be a silver bullet, and should be considered one of many tools used to execute a recruitment strategy.
#2. The participant self-tracking (quantified-self)
There is an exciting trend in healthcare described as ‘quantified self’ – well described in Wired magazineand online at QuantifiedSelf.com characterized by individuals who self-track a range of information about themselves (often in the context of measuring their health and wellness). Many of the tools these self-trackers are using begin to resemble the electronic patient reported outcomes (ePRO) or electronic diaries we look to use in clinical trials, just requiring validation.
#3. Participants sharing their health data
Similar to patients sharing data in online communities, clinical trials have always been dependent upon the participant being willing to share data to improve scientific knowledge and public health, often without any direct benefit conferred back to the participant. Today’s e-patient, however, is highly engaged and informed in their health, appreciating their role as owner of their own health data.
Understand the Risks
The e-patient who advocates being an informed participant in their healthcare, may not be comfortable with many traits of a randomized and blinded clinical trial. This raises unique challenges:
- How can we keep the e-patient engaged in a clinical trial if we are unable to share traditional “participatory” information (or even their treatment assignment)?
- What happens when the e-patient is engaged with communities of patients online? Are they sharing information that may provide clues to treatment assignment? Are they sharing suspected adverse events (that may create a cascade of other participants reporting symptoms)? How does the pharmaceutical sponsor educate the study participants, or monitor or engage in this conversation?
- Is the e-patient more likely to show a response to placebo (because they are more highly engaged in their health)?
I have seen online patient communities with threads dedicated to discussing participating in specific studies. Some of these discussions have included:
- Coaching on how to adjust certain subjective tests to meet eligibility criteria
- Sharing potential symptoms that may indicate who is on active drug (vs. placebo)
The e-patients in these instances are doing what they do best – sharing information with one another. But the scientific implications for the study can include enrolling ineligible patients, unblinding, and influencing adverse event reporting rates. Disrupting the ability to answer important questions is not in the interests of the study sponsor, investigator or the participant.
New Opportunities, New Horizons
Moving forward we cannot ignore the role of the e-patient in a clinical trial.
Some questions to consider:
- How can we participate in the online discussions and communities to answer questions about studies? Can we educate participants about online conversations that may impact the scientific integrity of the study?
- Are there new opportunities for engagement with the e-patient that can help to mitigate some of the sacrifice they are making serving as a blinded participant? Specifically:
- Can we bridge the gap between the tools a self-tracking e-patient (“quantified self”) is using and the validated tools we are using in clinical trials?
- How can we best share the learnings from a clinical trial back with the participant?
There are clearly more questions than answers. But there are incremental solutions underway. One example is Pfizer’s work with the Center for Information & Study on Clinical Research Participation (CISCRP). Working together, Pfizer and CISCRP recently demonstrated a new way to share clinical research learnings and results with participants. This should be just the beginning of a new relationship between participants and clinical trials.
Gilles Frydman said:
Thanks for the very nice summary of what networked patients are doing and how some of them have been impacting the healthcare system for now more than 15 years.
The clinical trial section is of particular interest since we both know that ACOR communities were involved in discussing clinical trials of Sutent (among many other new molecules) while the drug was tested in early phase trials. You are raising valid concerns about the impact of medical online communities on the entire clinical trial enterprise. But we disagree on what needs to be done about it.
The rise in number and significance of the networked patients and their communities has not destroyed science, it has only shown the limitations of the double blind trials. The networked patients should not be expected to limit the sharing they do online. Instead science has to evolve to fit this new world. The patients role will never be again to simplify the work of scientists. The Internet will completely disrupt the work of CROs, the ways to conduct research and the practice of medicine, just as it has disrupted all the industries related to entertainment.
We all have to work together to create the optimal environment to generate high-quality, unbiased science from this new world of transparency, openness and unlimited sharing.
Thanks again for raising these very significant issues. The sooner we actively discuss them among all participants the sooner we’ll figure out new ways to maximize the scientific impact of patient involvement in research, in the age of the network society.
on July 23, 2010 at 1:34 pm
Alexandra Carmichael said:
Thanks for posting this, Craig! You raise some excellent questions.
For me, patient-run studies are to formal trials as Genome-Wide Association Studies (GWAS) are to traditional genetics research. “Hypothesis-free” has value but is complex. (see http://www.ncbi.nlm.nih.gov/pubmed/19766959)
At the moment, crowdsourced trials have great promise for:
1. idea generation – finding new treatment ideas or finding co-morbidities or finding new ways to segment populations of patients that haven’t been thought of before, rather than testing a particular hypothesis
2. studying lifestyle changes that are free or low-cost and not market-driven
3. learning on a larger scale at much lower cost than traditional trials
In future, I see crowdsourced trials approaching greater clinical significance, including randomization etc. And I see formal clinical trials opening up to involve more elements of crowdsourcing, especially as the pharmaceutical industry moves away from the blockbuster drug model towards more personalized medicine.
We will meet each other somewhere halfway.
on July 24, 2010 at 11:45 am
Craig, great post which should open up a lot of eyes to these issues. I’ve seen growing skepticism and suspicion from e-patient groups and clinical trial professionals alike.
Many scientists have rolled their eyes as I describe the patient-driven efforts on PatientsLikeMe or CureTogether. “What about selection bias, nothing is blinded, and the placebo effect will be multiplied,” they exclaim.
Similarly, I know many e-patients who react angrily to the untold clinical studies that will never be done because they don’t make commercial (ie, financial) sense, and to the length of time it now takes to recruit patients and centers, complete the trial and study and publish the results. “We don’t have the time!” Andy sadly many of them don’t.
As you acknowledge, figuring how to integrate what e-patients have to offer with the rigors of traditional trials is not yet known. I’m thankful you and Pfizer are working on the answers.
on August 4, 2010 at 7:47 pm
Craig Lipset said:
I am very grateful to the “ePatient KOLs” for sharing reaction. I wanted to share some additional information for TSN readers who are not familiar with the ePatient community.
Gilles Frydman raises some excellent points from the perspective of the ePatient (evolve the science to keep up with the epatient movement). Few know this perspective as well as Gilles, who founded of the largest online social network for cancer patients (Association of Cancer Online Resources – ACOR). Take a look at his review article in Journal of Participatory Medicine for more on his views onPatient-Driven Research.
Alexandra Carmichael does a great job highlighting where crowdsourced trials add value today — and where they may tomorrow. As co-founder of CureTogether, Alex is a pioneer in empowering patients to track their health data and contribute to “open source” research. See her blog for more perspective.
Kevin Kruse points out the gap that exists between e-patients and scientists today. I am looking forward to further exploring that topic as a speaker at Kevin’s “e-Patient Connections 2010” conference this September in Philadelphia.
Falling back to the point raised by Gilles, the Internet is clearly a disruptive force. To-date the impact of the Internet on clinical trials has been largely how we collect data (web-based electronic data capture from investigators, web-based patient-reported endpoints from participants) and how we disseminate information among study teams (use of web-based portals for real-time decision-making). The next wave of disruption is being driven by the impact of the Internet on patients – making it vital that we keep the conversation open.
on August 5, 2010 at 10:11 am
Original article at https://science.pfizer.com/content/epatients-and-clinical-trials/