Case Studies

Developing Gold-Standard Instruments to Measure Patient Health and Quality of Life

Challenge

Healthcare professionals and researchers require validated, reliable instruments to accurately measure how medical conditions affect patients' health-related quality of life (HRQoL). Without condition-specific questionnaires that meet rigorous psychometric standards, it becomes difficult to quantify treatment effectiveness or understand what matters most to patients.

Approach

We led the full lifecycle development of multiple condition-specific questionnaires designed to capture the patient experience with precision. Our methodology ensures each instrument demonstrates robust validity, reliability, and responsiveness to clinical change.

Outcome

Prof Jones developed the Endometriosis Health Profile (EHP) during her doctoral research at the University of Oxford. The EHP has become the pre-eminent instrument for measuring the impact of endometriosis on HRQoL. Since its development, the instrument has been:

The EHP provides healthcare professionals and researchers with a validated tool that makes visible what matters most to patients and establishes a consistent yardstick for measuring treatment outcomes.

Validating Effectiveness for FDA Approval of a New Endometriosis Treatment

Challenge

A pharmaceutical company developing Myfembree, a novel treatment for endometriosis pain, required a patient-reported outcome measure that met the US Food and Drug Administration's stringent quality standards for demonstrating improvements in HRQoL.

Context

Endometriosis affects approximately 10% of women of reproductive age globally—an estimated 190 million women worldwide. Securing regulatory approval requires robust evidence of treatment effectiveness from the patient perspective.

Approach

The Endometriosis Health Profile (EHP) was selected as the primary outcome measure for clinical trials due to its proven psychometric properties and condition-specific focus.

Outcome

The FDA accepted the EHP as meeting its exacting quality standards for demonstrating HRQoL in women with endometriosis. Clinical trial data collected using the EHP provided the evidence necessary for Myfembree to gain FDA licensing as a treatment for endometriosis pain.

This case demonstrates the critical role that rigorously validated, condition-specific patient outcome instruments play in enabling market access for new treatments and therapies.

Measuring Patient Benefit for New Therapies in Post-Surgical Care

Challenge

Our client is developing an innovative product in a £10 billion annual global market. While research literature clearly documents shortcomings with established post-surgery therapies, existing patient questionnaires lack the sensitivity to detect these issues. This measurement gap prevents the client from demonstrating their product's superior patient benefit.

Approach

We are developing a new condition-specific questionnaire tailored to this commonplace medical treatment. Our work focuses on:

Expected Outcome

The new questionnaire will demonstrate superior psychometric properties compared to existing alternatives, providing the measurement acuity necessary to reveal meaningful differences in post-surgery outcomes. This will enable our client to quantify and communicate the genuine patient benefit their therapy delivers—evidence that is currently invisible with available instruments.

By developing an instrument capable of measuring what truly matters to patients, we are enabling better therapies to be appropriately recognized and adopted.