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CTTI and FDA Workshop Explores Patient Partnerships at Every Step of Research Process

While many share the vision that patient engagement should be embedded throughout the research enterprise, Donna Cryer, president and CEO of the Global Liver Institute, and CTTI Executive Committee member, goes one step further: “I hope one day it’s simply called ‘research’.”


Cryer keynoted a recent public workshop, “Enhancing the Incorporation of Patient Perspectives in Clinical Trials,” convened by CTTI in collaboration with the FDA. The workshop provided an opportunity for patients, investigators, and other groups to have their voices heard and, ultimately, help shape better clinical trials. A full report on proceedings from the workshop and a summary of public comments are now available.


The workshop was divided into three main sessions:


  • Enhancing awareness and access to clinical trials
  • Designing and conducting patient-centered trials
  • Post-trial communication and engagement


Wrapping up the day’s event, a panel of multi-stakeholder thought leaders summarized their key takeaways and perspectives on the future of patient engagement:


Cryer said she was inspired by the idea that researchers should let patients push them to a place where they are uncomfortable, letting the patients chart the course. “It’s different from how medicine has been taught. It’s different from how research has been constructed,” she said. “But it’s an endeavor worth taking on.”


Michael Kurilla, clinical innovation director at the National Center for Advancing Translational Sciences, noted that the increased trend toward the use of wearables and digital media holds the potential to lessen the burden of clinical trial participation. “Use of wearables will stimulate new ideas and directions in terms of clinical trial research that wouldn’t have previously been considered,” he said.


Craig Lipset, former head of clinical innovation at Pfizer, said that patient engagement is still anecdotal and not consistent across the drug development industry. A key component that’s needed to make patient engagement consistent is empathy. “Everyone thinks they are empathetic, but that’s different from incorporating empathy into how you are designing and engaging,” he said. In addition to a call for greater consistency, Lipset also listed 5 other themes of critical importance: understanding and meeting the needs of participations before, during and after participation; strategies that can better bridge divide between research and health care; concierge-level support for participants that anticipates all the steps required to get to a study visit; remote and decentralized trials; and making return of results to participants a standard deliverable from sponsors.


Theresa Mullin, associate director for strategic initiatives at the FDA’s Center for Drug Evaluation and Research (CDER), discussed the need for companies to develop long-term relationships with patients. When companies develop partnerships, and communicate with patients transparently, patients will be generous with their time and insights.                


Peter Saltonstall, president and CEO of the National Organization for Rare Disorders, said that it is important to “keep the patient at the absolute center of the conversation and build every single thing around the patient.” He cited the need for a new culture―from enrolling patients to returning results. “That culture isn’t fully developed yet and needs to be encouraged.”


Pamela Tenaerts, executive director of CTTI, said she is hopeful about the future of patient engagement. Key to the solutions discussed throughout the workshop is the idea of people working together. Organizations in the research ecosystem need to continue to facilitate communication between sponsor companies and patients.


John Wilbanks, chief commons officer for Sage Bionetworks, outlined two potential futures for patient engagement. In one future, patient engagement could be thought of solely in terms of recruitment and participation in traditional trials that are designed “for” and not “with” patients. In another future, patients would unite to demand more from investors in clinical research. The changes needed for true engagement require deep discomfort. “It’s really hard to add a little bit of change to a totally broken system. You have to actually change the system,” he said. “It will be slower and more expensive to center patients in this system that was built to center researchers and investors.”


Panelists concluded the workshop by expressing their deep gratitude to the patient representatives who attended the meeting and encouraging all attendees to continue moving toward true patient partnerships in clinical research.


This workshop met an FDA commitment that is part of the sixth authorization of the Prescription Drug User Fee Act (PDUFA VI).