Patient Reported Outcomes

A patient reported outcome (PRO) is a set of questions that are used to ask patients to assess their own health status. PRO is often referred to as PROMs – patient reported outcome measures. PROMs data are often used to estimate the Quality Adjusted Life Years (QALYs), which is a key element for cost-effectiveness analysis in economic evaluation studies, HTA submissions (meeting the requirements of agencies such as NICE, SMC, CDR, and PBAC), value demonstration, and publications

The use of PROMs have increased rapidly over the past decades, both in clinical trials and observational studies e.g. patient registry and population health survey. PROMs provides vital part of the evidence that is required in the process of approving new health care technologies and making pricing and reimbursement decisions. Well-established HTA bodies, such as UK’s National Institute for Health and Care Excellence (NICE) requires PROMs evidence to be submitted together with other clinical evidence. Similar HTA process are also in place in many other countries (1).  

There are two types of PROMs. The generic instrument that measures patient’s health status in a general way, which classify health status into different dimensions such as physical function, mental well-being and cognitive behavior. The mostly applied generic instruments are EQ-5D, SF-36 and HUI. The condition-specific instrument measures health status for patients suffer from a specific disease, and are more focused on a tightly defined aspect of health. There are thousands of instruments designed for specific conditions, such as EQRTC QLQ-C30 for cancer patients and Diabetes Care Profile (DCP). It is usually recommended that both a generic instrument and condition-specific instrument to be used in one study. The condition-specific instrument provides a detailed picture of a patient’s health status and is most likely to be relevant in clinical practice. While the generic instrument provides a common currency that allow comparisons across completely different patient groups and health services.

Synergus can provide you with comprehensive support from clinical development to post-marketing, complete or selective support for planning and application of PROMs including:

Measurement strategy

  • Literature and instrument reviews to select the best-fit PROMs for a specific study setting
  • To contact various PROMs bodies on behalf of the client, ensuring that the selected PROMs are the best value for money, and the terms/conditions of using PROMs are followed.
  • Provide guidance regarding how to incorporate PROMs into a specific study

Analysis strategy

  • Providing guidelines to optimize the analysis of PROMs data, as well as the value of results based on PROMs endpoints
  • Gap analysis of data supporting the use of PROMs for your intended purpose
    • Basic package
      • Descriptive statistics of PROMs at endpoints
      • Picture illustration of analysis
    • Advanced package
      • Insight of PROMs data (data quality demonstration)
      • Data imputation
      • Advanced statistical analyses to investigate deeply how outcomes change over time, e.g. multilevel model for change

Utility assessment strategy

  • Plan for the collection of utility data within a clinical trial and product R&D program
  • Identification of appropriate assessment of health state utility measures
  • Collect and analyze primary utility data during clinical trials through protocol development, statistical analysis plans, analyses, and reporting results
  • Perform stand-alone observational studies designed to derive utility data using a generic instrument e.g. EQ-5D, SF-6D and HUI
  • Perform mapping to derive health utility estimates from a condition-specific measure
  • Perform health state valuation studies using direct method e.g. time trade-off, standard gamble and rating scale

Large data-base analyses for PROMs

  • Identification of appropriate PROMs database e.g. patient registry, health survey, hospital documentation
  • Monitoring disease development, comparing treatment options/products by using PROMs database
  • Provide insights to how PROMs is linked to different HTA processes and practices for different countries and areas of technologies

1.                          Appleby J, Devlin NJ, Parkin DW. Using patient reported outcomes to improve health care. Chichester, West Sussex ; Hoboken, NJ: John Wiley & Sons Inc; 2016.