Report from the Field: Patient Involvement at the US Food and Drug Administration

In this new feature, we aim to highlight the innovative and patient-centered work of other organizations at the local, regional, and national level. This is part of an ongoing effort to develop a network of Patient Advisors who are informed and educated on research and healthcare issues. For this quarter, we wanted to highlight a national effort to ensure the patient voice is represented in regulatory decisions. Do you know of an organization or initiative we should highlight? Let us know!

The US Food and Drug Administration (FDA) involves patients and patient representatives to inform efforts to ensure human drug and biologic projects are safe and effective. There are two main efforts underway at the FDA where patients are actively involved, the FDA Patient Representative program and through a new initiative for Patient-Focused Drug Development.

Patient Representatives provide the FDA with the unique perspective of both patients and family members affected by a serious or life-threatening disease. FDA Patient Representatives participate on Advisory Committees and offer the patient perspective, providing comments to assist the committee in making recommendations. Patient Representatives may also consult for review divisions when a medical product or therapy is under review or present at FDA meetings and workshops on disease-specific or regulatory and health policy issues. To learn more about the FDA Patient Representative Program check out the FDA website.

ecognizing the importance of the patient voice and the opportunity to more effectively gather patient perspectives on their disease and available treatment options, the FDA launched the Patient-Focused Drug Development Program in 2013. This initiative comes through the fifth reauthorization of the Prescription Drug User Fee Act (PDUFA V), which committed the FDA to further develop the approach for assessing the benefits and risks associated with regulatory decision making. Over a five year period, the FDA will hold a public meeting on 20 different disease areas to collect patient perspectives. Meetings planned for 2014-2015 are listed in the table below. Patients are invited to attend public meetings either in person or through web conferencing. In addition to providing input at meetings, patients can provide input through public dockets. Interested in learning more or getting involved? Learn more about meeting updates and opportunities for participation at the FDA website.

New Project in Breast Cancer Seeks Focus Group Participants

Discussions between CERTAIN Patient Advisor Vicki Diguilio and University of Washington surgeon Sara Javid resulted in the development and subsequent funding of a research project focused on improving information collected from women undergoing surgical procedures for breast cancer. In 2012, CERTAIN began collecting information about quality of life, well-being, and physical perception among women following surgery for breast cancer. The information is important to understanding how women are doing following surgery but limited in its ability to inform breast cancer quality improvement activities in an actionable way that will improve care delivery. To develop and inform a new approach, we are conducting surveys of surgeons and members of the breast cancer surgical care team to hear from them what they think are the most important aspects of quality of life (e.g., body image, physical function, emotional well-being) and treatment decision-making for them to address when treating their patients. We will also be conducting a series of focus groups with women who are up to 2 years out from having a mastectomy to treat their breast cancer, to find out what they feel is most important for surgeons to know about their patients given their experiences. If you are interested in learning more about the study, please contact us.

Back Pain Patient Advisory Group Convenes

On January 27, 2014, the CERTAIN Patient Advisory Network convened an advisory group for back pain, as part of a project funded by the Patient Centered Outcomes Research Institute (PCORI), led by Dr. Janna Friedly. The goal for the meeting was 3-fold 1) to provide an overview of current research activities underway, 2) to present preliminary findings from recent focus groups informing work in spinal stenosis, and 3) to generate discussion about collaboration with patients moving forward. The meeting provided an opportunity for patients and researchers to come together in discussion around the important issues for those who experience back pain, in a continuing effort to ensure the research we do meets the needs of patients and their clinicians and helps to inform real-world decisions. We heard important insights about how patients experience pain versus how researchers measure pain and other topics of interest, such as exercise and nutrition aspects not often covered in research. The Back Pain Patient Advisory Group currently plans to meet twice a year, with the next meeting scheduled for Fall 2014. Members of this group will learn more about current research initiatives, provide input on ongoing studies, and, if interested, collaborate on future studies. Are you someone who experiences back pain who would like to join this group? Contact us for more details.

Research 101: Patient Centered Outcomes Research Institute (PCORI)

We understand that in partnering with CERTAIN, a lot of new terms and acronyms are thrown at you! This section of the newsletter is meant to highlight a different research-related term in each edition, to help you better understand the landscape in which CERTAIN, and you as Patient Advisor partners, are operating.

As a Patient Advisor collaborating with CERTAIN, you will likely hear a lot about the Patient-Centered Outcomes Research Institute (PCORI). Indeed many of you played an instrumental role in the recent submission of funding applications! In 2010, Congress passed legislation authorizing the establishment of the PCORI as a not-for-profit, non-government entity. The mission of PCORI is to help people make informed health care decisions and achieve better health outcomes. Central to this mission is funding and promoting research guided by patients, caregivers and the healthcare community. The establishment of PCORI provided a catalyst for change in how research questions are identified, how research studies are developed and how results are reported to inform decision-making. Specifically, PCORI promotes the involvement of patients, caregivers and patient representatives as collaborators on research teams; as reviewers for PCORI’s formal review process of research funding applications; and through submission of specific questions PCORI should consider for future funding.  You can learn more about PCORI, including opportunities to provide input at a national level at

What is Patient-Centered Research? How do Patient Advisor Activities Fit Into CERTAIN?

The United States spends more money on healthcare than any other industrialized nation. And yet, in list after list, American healthcare is ranked at the bottom in measures of delivering safe, effective, and affordable care. Additionally, despite the huge amounts of money spent each year, most healthcare research is not effective in helping patients get better healthcare, because the system does not have a way to hear from patients and their doctors what information is important to have when making decisions about healthcare and treatment. CERTAIN is a program that helps doctors provide the care that patients need and helps patients be empowered and educated when making decisions about healthcare. CERTAIN talks to patients about the care they have received and gives that information back to patients and their doctors to inform discussions about healthcare decisions to improve the care received and outcomes experienced. CERTAIN actively works to include a wide range of voices and perspectives in all aspects of the research process – from development, to implementation, to dissemination and translation of study results.

The CERTAIN Patient Advisory Network is critical to the CERTAIN mission of ensuring that research aligns with issues important to patients and their caregivers. In particular, Patient Advisors help guide research teams to focus on the questions important to patients about their health, the important outcomes considered when making decisions, and how health and medical information is accessed and used. By sharing personal experiences with a specific health condition or healthcare delivery, Patient Advisors help research teams refine and improve research questions and study designs, including how study results are shared with the patient community and care providers.

CERTAIN Patient Advisors can get involved in a number of ways. Our Patient Advisory Board meets quarterly to provide input on our research programs and our approaches for supporting and involving patients as collaborators in our work. Patient research partners serve as members or consultants on specific research projects working directly with the research team throughout the life of the project – from selecting the research question to communicating the results. In addition to these activities, CERTAIN Patient Advisory Network members review research materials including patient surveys and research summaries for content and clarity as well as serve as speakers at CERTAIN events. The insight and passion Patient Advisors contribute to our work is greatly valued and critical to our continued commitment to improve healthcare in Washington State!

Patient Voices First Annual Research Symposium

2013: A Building Year
We spent 2013 building up our network of Patient Advisors and getting them connected to the work of CERTAIN. We conducted outreach to many community organizations that serve patients in order to identify ways to collaborate. In addition, our Patient Advisory Network Steering Committee composed of 5 Patient Advisors continued to provide input on CERTAIN initiatives, identify areas where CERTAIN can better support the patient involvement in research, and provided support in new research development. The Patient Advisory Network Steering Committee met quarterly in 2013, and on October 31, hosted Ms. Deborah Collyar as a keynote speaker at the first annual Patient Advisory Network Research Symposium, to kick off discussions and planning for how the Patient Advisory Network can best support patient partnership in research in 2014 and beyond.

Patient Advisory Network Director Danielle Lavallee, Research Symposium keynote speaker Deb Collyar, and meeting participantsThe October 31 Patient Advisory Network Research Symposium brought together 30 patients, researchers, and clinicians interested in working together to support patient involvement in research. Keynote speaker Deborah Collyar has been a leader in cancer patient advocacy since 1991, utilizing successful business, leadership and IT skills to bridge research gaps between science, medical providers and patients. She is the founder of Patient Advocates In Research (PAIR), an international communication network advocating the patient perspective in research. At the meeting, Ms. Collyar presented her perspective on the evolution of patient involvement in research drawing from her experience. The meeting agenda and Ms. Collyar’s slides are available on the CERTAIN website.

Moving Forward in 2014
The Patient Advisory Network Research Symposium kicked off a conversation that we will be continuing throughout 2014, namely – how can patient partnership in research be best supported, and what tools or resources are needed to support both patients and researchers in incorporating the patient perspective into research? With these questions in mind, we are launching a discussion board that we invite all Patient Advisory Network members, partners, and collaborators to provide input on. The discussion board has been seeded with two preliminary questions on which we would like your feedback, and we will be updating with additional questions for discussion over the next few months as conversation develops. Join the discussion today! (Click herefor guidance on registering for an account, or contact Sarah Lawrence with questions.)

The Patient Advisory Network Research Symposium kicked off a conversation that we will continue in 2014, namely how can we best support patient and researcher partnerships, and what strategies we can develop to break down barriers in communication and collaboration between patients and researchers. With these questions in mind, we are launching a discussion board that we invite all who are interested to provide input on. The discussion board has been seeded with two preliminary questions on which we would like your feedback. We will update with new questions regularly and share results and plans coming from the input received. Join the discussion today, and feel free to share with friends and colleagues who might also be interested.