What is Patient-Centered Outcomes Research?

Patient-Centered Outcomes Research (PCOR) is research that provides patients and their clinicians with evidence-based information in order to help them make more informed health care decisions and allowing them to express what is most important to them. This research is designed to answer questions about the potential benefits and harms of different care options, given a patient’s personal characteristics, conditions, and preferences.

Where We Started

In 2016, we launched a caregiver survey to identify characteristics, measure comorbidities, and prioritize caregiver concerns regarding Dravet syndrome. Over 250 caregivers responded, and we published the results in Epilepsy & Behavior. What we discovered, in addition to clear patient data trends, was that caregivers’ top concerns, aside from seizure control, were: speech/communication, sibling impacts, cognition/developmental delay/regression, and behavioral issues. Each of these concerns lends itself well to patient-centered outcomes research, so we organized a group of stakeholders including caregivers, clinicians, basic scientists, and industry representatives to determine how to design a study that would address these concerns.

Read the paper here.

Our Experience with PCOR

In 2016, DSF was awarded a Pipeline to Proposal (P2P) award from the Patient-Centered Outcomes Research Institute (PCORI) to develop comparative effectiveness research in Dravet syndrome. The purpose of the P2P Awards program is to help people form new collaborations with the goal of developing proposals for research with sound scientific rigor and robust patient engagement.

In Tier I of our P2P project, a stakeholder group (including family members, clinicians, and scientists) was established. The stakeholder group met on a monthly basis to dicuss and understand the primary concerns of the community and to encourage development of collaborative research to identify comparative effectiveness research (CER) goals for people living with Dravet syndrome.

In Tier II of our P2P project, the focus was to further refine the potential CER questions developed in Tier I down to a few strong and viable options with relevance to the Dravet syndrome community. This was accomplished through additional outreach to caregivers and community stakeholders, including focus sessions and surveys. In addition, we developed a communications plan and a stakeholder engagement plan to allow dissemination of the CER progress.

After completing Tier II of our P2P project, we applied for and were awarded a Eugene Washington PCORI Engagement Award. These awards support projects that encourage active integration of patients, caregivers, clinicians, and other healthcare stakeholders as integral members of the patient-centered outcomes research/clinical effectiveness research (PCOR/CER) enterprise. Our project was on expanding patient engagement in the Dravet syndrome community and developing a best practice paradigm

Tools developed from our PCOR work

Stakeholder Working Group

The challenges of a rare disease such as Dravet syndrome require multi-stakeholder collaboration to solve. Many rare diseases have an onset in infancy or childhood and present as a life-long condition with a variety of clinical phenotypes and multiple health issues that fall under the domain of a variety of specialists. Patients and caregivers are often the experts in their own rare disease and have unique knowledge about living with that disease in a field where clinical evidence may be sparse at best. Many patients and patient advocacy groups are keen to contribute meaningfully to research and the development of new treatment options that focus on associated health issues, preferred treatments, and health outcomes that are important to them.

By establishing and working in partnership with a multi-stakeholder group (including but not limited to patients, caregivers, healthcare providers, researchers, payers, and industry members) there is a unique opportunity to address the many challenges faced in research and healthcare decision-making. We first established our Dravet Syndrome Stakeholder Group in 2017 as part of our P2P Tier I award, with the goal of identifying and discussing the top caregiver concerns in the Dravet syndrome community. From there, the group has grown and evolved and continues to meet quarterly. It brings together our stakeholders to continue to identity patient needs, share projects, form collaborations, and visualize a better future for our patient community.

Webinars

Because there is an increasing recognition of the importance of the involvement of patients in the development of research that will impact their disease state, DSF developed these webinars, in English and in Spanish, to help educate the Dravet syndrome community on the importance of the patient voice. They can also be used as a guide for other rare disease communities.

White Papers

A white paper is an informational document meant to help readers understand an issue. It is intended to provide factual evidence to approaching or solving a problem or challenge. Throughout our PCORI projects, we produced three white pages to share our project experiences and outcomes.

Day of Dravet Summary and White Paper
Sodium Channel Coalition Lay Summary
P2P Tier 1 Summary

Best Practice Paradigm

As part of a strategic work plan that was made possible through a Patient-Centered Outcomes Research Institute (PCORI) Eugene Washington PCORI Engagement Award (7881-DSF), DSF has developed this Best Practice Paradigm for Expanding Engagement to assist other rare diseases to help them organize and prepare their patient communities for PCOR. Through our time with PCORI, we have learned several valuable lessons including the fact that an organized, easily-reached stakeholder community is vital to any project requiring patient engagement and that stakeholders need to receive information in several different formats at more than one time to understand the process.

Best Practice Paradigm for Engagement