Existing Data

Seeking Existing Patient Impact Data 

Within some disease communities or populations, previous efforts have already amassed data from which the impacts most important to patients could be gleaned. That is, information has already been collected directly from patients about what they identify as important about their disease(s) and/or treatments.  It is not information other stakeholders (e.g., clinicians, researchers) have identified as important to patients, which may exist but is not included here as the reports must come directly from patients. 

The patient-reported information may exist in a variety of formats and types such as qualitative research projects, Voice-of-the-Patient Reports, registries, social media listening, and other publications. To date, this type of information will only rarely be found in a peer-reviewed publication. This paradigm shift, to beginning with patient-generated data rather than beginning with peer-reviewed literature, may require some flexibility and creativity to locate existing sources.  

Examples of existing patient-provided impact data on an array of conditions include: 

  • U.S. Food and Drug Administration (FDA) Patient-Focused Drug Development (PFDD) website
  • Disease-specific, patient-group led Voice-of-the-Patient Reports identified on patient group websites, e.g. National Kidney Foundation, Arthritis Foundation 
  • Disease-specific registries such as RARE-X Patient-Powered registry or Duchenne Data registry 
  • Patient-Focused Medicines Development’s (PFMD) Synapse, includes information on engagement initiatives around the world 
  • The Core Outcome Measures in Effectiveness Trials (COMET) database includes initiatives that include some patient-generated data 

One potential source of existing patient impact data is a previously developed Core Outcome Set (COS).  Whether or not a COS can be readily translated into a PC-CIS is not easy to determine and depends on how patient centered the existing COS is, how comprehensive the data gathering from patients was when capturing the impacts considered, and if the prioritization process kept the patient voice intact. An existing COS that is highly patient centered may be ideal to incorporate into Patient-Centered Core Impact Sets (PC-CIS) or might simply be adopted as is. Others may require significant adaptation and others might not be useable at all.

Evaluating Existing Impact Data Reported by Patients

Existing data will need to be evaluated by patients and other members of the PC-CIS team to determine if it is patient centered enough to be considered as a sound starting point. That is, how will you evaluate whether what has been collected in the past captures what is important to the patient community from the patient community perspective?  In some cases, the description of a project or effort does not provide enough detail to evaluate the patient-centered aspects or relevance and representativeness. It may be worth having a conversation with the patients and/or project leads (which may be the same people) who were involved to better understand how data were captured. You also will need to assess whether or not the data are recent enough to still be relevant.  For example, if therapy for a specific condition has changed dramatically over the past decade, it would be important to have data that is reflective of patients’ experiences with the current suite of therapeutic options.

This tool, the Is It Good Enough? guide is a checklist for evaluating the quality and patient centeredness of existing data reported by patients.

Additional guidance for assessing patient centricity, quality, and representativeness of data includes:

  • The National Health Council Rubric to Capture the Patient Voice
  • The National Health Council Representativeness Rubric and Roadmap
  • The Core Outcome Set- STAndards for Reporting (COS-STAR) guidance to describe geographic diversity of stakeholders.
  • The Outcome Measures in Rheumatoid Arthritis Clinical Trials (OMERACT) Handbook Chapter 3, Section C

Arthritis Use Case

Arthritis Foundation, Existing Patient Impact Data