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).

CTTI is pleased to announce that its Executive Committee will welcome five new distinguished thought leaders. These experts hold impressive track records in implementing change at many levels of the health care and research system, from regulatory bodies to thoe drug development pipeline.

• Steven K. Galson has spent more than 20 years in government service at regulatory agencies.
• Michael Kolodziej is an expert on payment reform, personalized medicine, and practice care delivery transformation in oncology.
• Theodore Lystig provides leadership and guidance in the use of robust statistical and research design methods.
• Rod MacKenzie focuses on global clinical development and advancement of medicines.
• Pierre Meulien works to improve and accelerate the drug development process across Europe.

“These new Executive Committee members are leaders in their fields,” said CTTI Executive Director Pamela Tenaerts. “Their guidance and passion to improve clinical trials will help ensure CTTI can achieve its goals in a rapidly evolving ecosystem.”

The five new Executive Committee members join a group of thought leaders whose knowledge and experience enables CTTI to make meaningful improvements in clinical research.

“These experts will broaden and strengthen the committee’s knowledge in a number of key areas, from clinical research design to quality assurance to drug development,” said CTTI Executive Committee Chair Mark McClellan. “We are confident that they will make a significant contribution to CTTI’s work to move the clinical trials enterprise forward.”

   Steven Galson                 Michael Kolodziej           Theodore Lystig           Rod MacKenzie           Pierre Meulien

The Reagan-Udall Foundation for the FDA presented CTTI with the Innovations in Regulatory Science Award on Tues., Dec. 4, in recognition of its achievements and continued contributions to regulatory science. CTTI was one of three recipients of the annual award.

“This great honor caps off a momentous year for CTTI,” said Pamela Tenaerts, CTTI executive director. “We celebrated our 10-year anniversary at the beginning of 2018, so this award affirms that our efforts over the last decade have led to meaningful positive change in the quality and efficiency of clinical research.”

 

The Reagan-Udall Foundation for the FDA is an independent nonprofit organization created by Congress for the purpose of advancing regulatory science that is critical to the FDA’s mission.

 

“We are pleased to give CTTI proper recognition for advancing public health by streamlining and improving clinical research,” said Garry Neil, MD, awards committee chair and chief scientific officer for Aevi Genomic Medicine. “CTTI not only identifies the most critical areas that need to be addressed in order to achieve better and more efficient clinical trials—it also provides actionable road maps to help stakeholders realize this vision.”

 

Many agencies and organizations have applied CTTI’s more than 20 existing recommendations and associated resources to make better clinical trials a reality. A few examples include:

• CTTI’s Quality by Design work has been cited in FDA and EMA guidance and incorporated into Good Clinical Practice (GCP) guidelines.
• Its Single IRB recommendations and resources have been used by organizations such as Northwell Health and the National Institute of Neurological Disorders and Stroke.
• The Duke Clinical Research Institute (DCRI) developed a new program for engaging patients in the research process based on CTTI’s Patient Groups & Clinical Trials work.

CTTI joined other awardees Richard Pazdur, MD, director of FDA’s Oncology Center of Excellence (OCE), and Robert O’Neill, PhD, former senior statistical advisor in the Office of Translational Sciences at the FDA’s Center for Drug Evaluation and Research.

The awards dinner took place at the Kaiser Permanente Center for Total Health in Washington, D.C.

CTTI is pleased to announce the addition of a leader in the National Institutes of Health’s (NIH) research initiatives to its Executive Committee, as well as the renewal of the appointments of three current members.

New EC member Jodi Black is the deputy director of the NIH Office of Extramural Research, where she oversees and supports initiative development, grants management policy and processes, and the small business and extramural technology development programs.

“CTTI’s work relies on input, participation, and collaboration across a range of public and private stakeholders in clinical research and development,” said CTTI Executive Director Pamela Tenaerts. “Jodi’s passion for challenging the status quo, as well as her experience building strategic alliances between academia, healthcare, and industry, will be invaluable to furthering CTTI’s efforts and advancing our mission to improve the efficiency and quality of clinical trials.”

CTTI is also delighted to extend the terms of current members Hans-Georg Eichler of the European Medicines Agency (until Dec. 31, 2020), Dalvir Gill of TransCelerate BioPharma Inc. (until Dec. 31, 2019), and Louis Jacques of ADVI (until Dec. 31, 2018).

“We are grateful for the continued commitment of Hans-Georg, Dalvir, and Louis, and excited for the valuable expertise Jodi brings to our committee,” said CTTI Executive Committee Chair Mark McClellan. “I’m confident that their leadership and insight will significantly contribute to the overall direction and strategy of CTTI and its impact on clinical research.”