Patient Engagement in Research

Patients are the ones who the therapy is made for, so it’s important to get their input about what therapeutic benefits actually matter to them. Patients can help researchers better understand the challenges of these therapies and clinical care for their condition. Patients can also help improve study design and educational materials to make clinical trials more effective and help other patients with the condition better understand the risks and benefits of gene therapy.

Learn more about patient engagement efforts at the Foundation for the National Institutes of Health (FNIH) here: Patient Voices | FNIH

How to Involve Patients and Caregivers in Research

The Patient-Centered Outcomes Research Institute (PCORI) required investigators to include patients (as well as other stakeholders) in the research process. Almost 2/3 of these projects used multiple methods to engage patients.

In these projects, patients helped determine the study design, selected study outcomes, improved educational materials and informed consent forms, tailored interventions for specific populations or settings, assisted in enrolling participants, and more.

Many studies reported that patient engagement made positive contributions to research feasibility, acceptability, rigor, and relevance. Most of these contributions were based on personal reflections from study authors, but some projects systematically measured impact with user feedback and statistics that measured enrollment or retention in studies.

Patient Engagement In Research: Early Findings From The Patient-Centered Outcomes Research Institute | Health Affairs

Patient-Researcher Forum (PRF) is a series that promoted “a community-engaged research approach through communication, compassion, and bi-directional research insight for both patients/caregivers and researchers”.

Each month a patient and caregiver were invited to meet with researchers and start off with a tour of the lab. Clinicians who treat the patients’ condition were also invited to explain current treatment options and unmet needs to researchers. Then there was a meeting where researchers shared about their experience and their research that is relevant to the patient. The patient shared their story and experience with the condition, and then the group had a moderated roundtable discussion to exchange ideas and perspectives.

This helped patients and caregivers learn about the research process and gave them a more positive outlook about research. Researchers found that the forums reinforced their decision to work in research, helped them see the patient perspective, and helped them practice communicating research with a non-scientific audience.

Partnering patients, caregivers, and basic scientists: An engagement model that fosters patient- and family-centered research culture – PMC (nih.gov)

Participants: How researchers select participants varies by the research area. For some researchers, it may be beneficial to work with individual patients with the condition, caregivers, clinicians who treat patients with the condition, or patient advocacy organizations.

Plan: Create a formal plan that includes time commitments, what patients should expect from the research team, the scope of engagement for both patients and researchers, and a budget to pay patients or patient costs.

Training: Plan to educate patients with basic background on health research methods to give them good starting knowledge. Train researchers who will work with patients on the value of patient engagement, communication skills, and strategies to co-build ideas with patients and other researchers.

Respect: Set a requirement for mutual respect between researchers and patients, and value patient’s experiential knowledge as expertise.

Early: Start patient engagement early within the research process, and continue it (with either the same or different patients) as your research continues.

Patient engagement: What partnering with patient in research is all about – ScienceDirect

Considerations With Patients in Research

How Patients Contribute to Research

Patients can contribute to many aspects of research, including (1-3):

  • Helping researchers gain a new patient-centered perspective
  • Improving the relevance of research to patient priorities
  • Defining research questions or aims, selecting comparators, setting inclusion or exclusion criteria, and establishing study outcomes
  • Tailoring interventions for specific populations or settings
  • Improving patient educational materials and informed consent documents
  • Anticipating barriers to recruitment, enrollment, and retention and helping provide solutions
  • Providing input into when and how data is collected from study participants
  • Improving dissemination and/or implementation of research findings

Challenges with Patient Engagement

There are some challenges to consider with patient engagement, including (4):

  • Additional costs to pay the patients or caregivers participating
  • Time issues for both patients and researchers
  • Patients feeling like tokens rather than valued team members or participants
  • Patient values leading to divergence from the research agenda
  • Concerns that patient contribution creates unfeasible study designs.
  • Concerns about true representation as patients who engage with research teams may not be representative of the patient population (in terms of education level, SES, etc.)
  • Lack of clarity for resolving disputes between patients and researchers

Calculating Patient Engagement Costs

It is important to plan for patient engagement costs. Whether that is setting aside budget or calculating the financial value to show the funding party, here are a few things to consider:

  • Calculating ENPV
    • ENPV (expected net present value) can be used to predict how patient engagement can financially impact the project. Information about calculating ENPV can be found here.
  • Ways Patients Can Financially Impact Projects
    • Patients can offer insights and suggestions to avoid protocol amendments, improve enrollment, increase adherence to protocol, and improve retention rates, which can decrease costs and help streamline projects to next trial phases.
  • Costs Associated with Patient Engagement
    • Costs can differ depending on the project, the way researchers engage with patients, the stage of research, and the frequency in which they work with patients. Costs can include preliminary research, time spent educating or creating educational materials for patients, facilitator or facility fees, travel and lodging, compensatory fees for time, and time cost for researchers to work with patients instead of working on research.

References

  1. Partnering patients, caregivers, and basic scientists: An engagement model that fosters patient- and family-centered research culture
  2. Patient engagement: What partnering with patient in research is all about
  3. Patient Engagement In Research: Early Findings From The Patient-Centered Outcomes Research Institute
  4. Patient engagement: What partnering with patient in research is all about – ScienceDirect
  5. Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI’s Patient Groups and Clinical Trials Project