You’re engaging patients – but how do you know if you’re being representative?

Authors: Eleanor Perfetto (Senior Vice President of Strategic Initiatives) and Jason Harris (Manager of Public Policy) at the National Health Council (NHC).

Just over 14 months ago, in March 2016, the National Health Council (NHC) released the NHC Patient-Centered Value Model Rubric, which aims to help the field better incorporate patients’ perspectives in assessments of the value of health care interventions and treatments. At a webinar to discuss and explore key issues and themes in the rubric, we asked attendees what NHC can do to help value framework developers be more patient centric. A resonating response from a value framework developer was the need to articulate and define what ‘representativeness’ is and to develop a scale or metric that they could use to gauge representativeness.
We realized that such a definition and metric wasn’t relevant solely to value assessment but to all fora where patient engagement is part of the process. This widespread need was the starting point of NHC efforts to provide direction on determining what representativeness means in patient engagement, which we kick-started by hosting a ‘Roundtable on Defining Patient Representativeness’ this month (May 8, 2017).
The multi-stakeholder roundtable meeting included participants from patient and caregiver groups, value framework development organizations, pharma, clinical professional associations, not-for-profit (non-patient membership) organizations, payer organizations, and the FDA. Five working groups met for two sessions each to brainstorm, discuss, and respond to a set of “starter” questions. We are still busy assimilating the findings and discussions but we already see key themes emerging.
Theme 1: Assessing representativeness can’t be formulaic. It can’t be a pass or fail measurement, but needs to be flexible because context matters. For example, any assessment or metric needs to take into account the goal and objectives of the activity.
Theme 2: The need for representativeness can change over time as the project evolves. If we take a pharma example, patient engagement and representativeness needs during the preclinical phase of drug development may be very different from the needs at the post-marketing phase so a ‘one-size-fits-all’ approach is not appropriate.
Theme 3: Representativeness is ­one of the desired qualities of patient engagement – other parameters also need to be optimized. Just getting representativeness ‘right’ in isolation does not guarantee meaningful and effective patient engagement.
Theme 4: Definition of representativeness needs to be transparent within the specific context. For a specific project or activity, there should be clear rationale and reasons to explain how ‘representativeness’ was decided or calculated. For example, if we take a rare-disease area, there may be a limited number of patients with that condition, which impacts representativeness. Or, in a disease area where fatigue is a common occurrence, this may be a practical barrier to the extent of patient participation possible. We need to be able to explain and be transparent about issues like these that can impact representativeness.
Theme 5: Learn from other experience rather than through trial and error. This was a resounding theme that highlighted the need for a repository of practice – good and suboptimal – so we don’t reinvent the wheel, but instead build on experience and knowledge; not just in representativeness, but in patient engagement generally. This need for a repository or hub for sharing experience and knowledge across the patient engagement community resonates with one of the core outputs of PFMD. SYNaPsE – the PFMD on-line global mapping and networking tool – aims to SYNergise Patient Engagement practices by providing a searchable resource that connects the patient-engagement landscape and allows users to find and connect with organizations and individuals active in patient engagement.
The Roundtable on Defining Patient Representativeness has provided rich and valuable insights. We are currently doing a deep-dive of the meeting outputs so that we can share these through a White Paper to help all stakeholders reach and gauge representativeness as a core quality of meaningful patient engagement.