Patient engagement and patient-centricity are hot topics in clinical research and study teams are trying creative approaches to involve patients in the research process. These teams can look to the ADAPTABLE (Aspirin Dosing: A Patient-centric Trial Assessing Benefits and Long-Term Effectiveness) trial as an emerging model for patient engagement in randomized trials. Patient leaders, study investigators, and operations and research staff gathered together on October 28, 2016 in Washington D.C. to discuss ideas and strategies for making this novel, pragmatic study a success.
The meeting kicked off with welcome remarks from the Co-Principal Investigators, Matthew Roe, MD, MHS and Adrian Hernandez, MD, MHS from the Duke Clinical Research Institute Coordinating Center. In opening comments, Joe Selby, MD, MPH, Executive Director of the Patient-Centered Outcomes Research Institute (PCORI) and Russell Rothman, MD, MPP, ADAPTABLE Study Co-Chair, provided the framework for how the ADAPTABLE study is breaking ground in the conduct of pragmatic trials. Selby discussed how studies like ADAPTABLE are demonstrating the power of PCORnet, the National Patient-Centered Clinical Research Network, whose mission is to “ enable people to make informed healthcare decisions by efficiently conducting clinical research relevant to their needs.” “You are laying down the tracks to pragmatic research while driving ADAPTABLE forward as a model of faster, less expensive, and people-centered approach to clinical research,” said Selby. Rothman added, “The ADAPTABLE study represents an innovative opportunity to revolutionize clinical research by leveraging health information technology that can lead to rapid participant identification, contact, consent, and data collection. In addition, the engagement of patients, clinicians and other key stakeholders in all aspects of ADAPTABLE from design through dissemination will allow us to do research that is improving healthcare and is of greatest value to the community.”
Rachael Fleurence, PhD, PCORI, Program Director and Chair of the PCORnet Executive Committee congratulated the Clinical Data Research Networks (CDRNs) who are currently enrolling participants and acknowledged that the remaining networks are on the cusp of enrolling. “When you are breaking new ground, it can take time to overcome challenges and see progress, but I can tell ADAPTABLE is picking up steam,” said Fleurence.
Moving the Needle from Traditional to Pragmatic Research
Robert Harrington, MD, ADAPTABLE Study Co-Chair described how pragmatic trials are evolving from the model of traditional trials. Traditional trials are expensive, long, and often don’t provide critical evidence. With costs typically at least hundreds of millions of dollars and taking more than five years to obtain results, the traditional model of research is not sustainable. In addition, these trials often do not answer questions people have about their health care. Harrington and meeting attendees agreed that while results from traditional trials are published in scientific journals and presented at professional meetings, the participants ─ who agreed to be in the study, underwent the protocol procedures, and provided their data ─ often never learn about study results.
Enter ADAPTABLE, a new model of pragmatic research with a focus on transparency and engagement. ADAPTABLE will compare the effectiveness of two different daily doses (81 mg and 325 mg) of aspirin widely used to prevent heart attacks and strokes in individuals living with heart disease. Using electronic health records (EHRs) to screen and identify potential participants against key indicators, efficiencies gained in ADAPTABLE include: 1) Elimination of data-entry redundancies by obtaining information directly from EHRs and data in the PCORnet Common Data Model; 2) Reporting outcomes data by participants directly via the ADAPTABLE web portal; 3) Elimination of monitoring costs.
How do we answer the age-old aspirin question?
Adrian Hernandez, MD, MHS, ADAPTABLE Study Co-PI and PCORnet Co-PI started the operations update presentation with a question he hears from his mother who has cardiovascular disease, “How do you not know the right dose of aspirin?” It was no surprise to the meeting attendees that although doctors prescribe either low-dose aspirin (81 mg) or regular-strength aspirin (325mg daily) to help prevent a future heart attack, stroke, or death, there is no clear, long-term evidence to delineate the best dose of aspirin to prescribe for patients with cardiovascular disease.
“It would be too expensive and take too long for a company from the pharmaceutical industry to answer this aspirin dose question. However, it is a perfect question to show the power of PCORnet to harness massive amounts of health data to answer questions such as this one and other that matter to people,” said Hernandez. Read more about PCORnet here.
Success in the Making
ADAPTABLE Study Co-PI, Matthew Roe, MD, MHS reviewed metrics highlighting early successes and challenges. While ADAPTABLE start up timelines are slightly longer for Institutional Review Board (IRB) approval and site activation compared with traditional cardiovascular outcomes trials, the average monthly enrollment rate per site is significantly higher than the average monthly enrollment rate for a site in a traditional trial. IRBs often have little experience with pragmatic trials so longer trial reviews are not unusual.
Each CDRN presented their site-level metrics, recruitment approaches, and enrollment plans. These presentations were followed by an engaging group discussion of lessons learned. The attendees discussed topics such as: How pragmatic do we need to be when facing an enrollment goal of 20,000? The group agreed that pragmatism is a spectrum and that each CDRN will need to evaluate recruitment approaches and find what fits best at their site.
The group overwhelmingly agreed that a broad, multi-touch approach is needed for the outreach to potential participants to achieve the trial enrollment goals. This type of approach includes contacting eligible patients in a variety of ways, including by email, phone, direct mail, and facilitated discussions during clinic encounters with tablets and other mechanisms. Participants also stressed the importance of clinician engagement. “Primary care providers and cardiologists can support their patients and ADAPTABLE by having discussions with their colleagues about ADAPTABLE and why it is important to have broad support,” said Roe. Just as in traditional trials, patients like to hear about studies from their physician and know that their physician is in support of their participation. The challenge will be how to optimize that message with the electronic recruitment approaches.
Real People Shaping Research
Without question having the Adaptors ─ the ADAPTABLE patient leaders and partners ─ engaged in the meeting discussions and during panel discussions was the highlight of the meeting for all attendees. The Adaptors are patient investigators from each of the eight participating CDRNs who have already provided protocol feedback, review of the consent form, and input on the study portal and materials. The Health eHeart Alliance PPRN supports and facilitates the Adaptors Team via bi-weekly check-in calls.
This meeting provided a face-to-face opportunity for researchers and Adaptors to interact and all agreed that it was an amazing experience. Seven of the eight ADAPTORS were able to attend the kick-off meeting and participated in a facilitated panel discussion. “Prior to my cardiac event, I had lost three close friends to heart disease,” shared Ken Gregoire from the REACHnet CDRN. “When I was introduced to ADAPTABLE by Oschner, I thought the results from this study might benefit someone I know or even someone I don’t know. I wanted to be involved to get the word out about ADAPTABLE.”
By being present at the meeting, the Adaptors heard of the recruitment challenges and learned of opportunities where they could be involved as study ambassadors by making other patients aware of ADAPTABLE. Linda Brown from the Mid-South CDRN, whose father and brothers all died from heart disease before they were 65 years old, is grateful to be involved to help support her husband and other patients with heart disease. “I think the Adaptors can play a pivotal role in publically sharing study updates and results in lay terms so that other patients will have a better understanding of what the results mean.” Brown said. “The act of sharing study progress may encourage more people to enroll in studies knowing that they will be informed of the results and how their participation contributed.”
Adaptors Team Co-Chairs, Jacqueline Alikhaani from pSCANNER CDRN and Henry Cruz from NYC-CDRN echoed the importance of including the patient voice in clinical research. “We cannot improve patient care without involving patients in the process,” said Alikhaaini. “In ADAPTABLE, we have the opportunity to develop innovative ways to engage the patient community.” Cruz, who is an advocate for diabetes education after his mother and nine siblings died from complications of diabetes said, “Providing the patient perspective to health research is my passion. Telling my personal story makes me vulnerable but I know in my heart that clinical research has the potential to save lives.”
The Path Forward
As with all clinical trials, traditional or pragmatic, opportunities to establish and deepen personal relationships can play a significant role in the success of a study. The probing questions, thought-provoking presentations and high-level dialogue at the kick-off meeting help to further develop the personal relationships that are critical for the success of clinical trials, especially a novel, pragmatic trial like ADAPTABLE. Continue to follow the success of ADAPTABLE by visiting the website and following on Twitter @ADAPTABLEstudy.
The ADAPTABLE study is funded through a Patient-Centered Outcomes Research Institute (PCORI) Award (Contract Number: ASP-1502-27079).ADAPTABLE is the first demonstration project to be conducted through PCORnet, the National Patient-Centered Clinical Research Network. PCORnet is a PCORI-funded initiative designed to improve the nation’s capacity to conduct comparative clinical effectiveness research. More information about PCORnet can be found here.