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The right patient reported outcome

Case study: Data derived from patient reported outcome measures can provide added value in supporting key biomedical endpoints.

Right patient reported outcomes

Case study: Data derived from patient reported outcome measures can provide added value in supporting key biomedical endpoints.

Patient non-adherence is only likely to be surmounted if patients believe that taking medication will give immediate benefits through reduction of symptoms, improvement in physiological functioning and quality of life.

The diabetes mellitus (DM) marketplace is becoming saturated with multiple medications in both the insulin and pre-insulin space, particularly as analogues start to lose their patents. Differentiation in clinical outcomes within classes is often unclear or minimal. So, differentiation of therapeutic options is likely to focus more on frequency, mode and method of administration, as opposed to statistically significant differences in glucose control, which are clinically relevant.

There is no consensus in the industry on the most appropriate population-based balance between diabetes-related physiological outcomes such as blood glucose levels (HbA1c) and measurement of quality of life (QoL).

Therefore a workshop was developed for a pharma company client to demonstrate that data derived from patient reported outcome (PROs) measures could provide added value in supporting key biomedical endpoints.

The challenge was to present the case for investment in the selection and implementation of PROs for clinical trials through the development of an effective PRO measurement strategy which was mindful of current regulatory concerns.

Pharma companies are faced with a plethora of PROs, each purporting to measure a range of health outcomes, often without a sound theoretical or measurement model.

As a consequence, outcomes teams often make their choice of a PRO according to whether it has been used in previous studies or its name appears to be appropriate for the intended use. There is also the tendency in the industry to treat the more commonly measured health outcomes such as QoL and health status as interchangeable in the selection process; they are not.

There is no universally accepted definition of QoL, for example, but there is general consensus that it is based on the individual's own subjective evaluation of the psychological, physical and social aspects of his or her life.

Measurement of QoL contrasts with the commonly used measures of health status, such as the SF-36 and EQ-5D, which are frequently referred to as indicators of QoL, which they are not.

The client was aware of these potential pitfalls in the selection of PROs, in phase III trials. Prior to considering expenditure on a programme of PRO implementation, the main requirement was for the outcomes team to have a much clearer insight into best practice. This would enable it to provide evidence of treatment effectiveness and benefit from the patients' perspective across the DM product's life cycle in the context of new regulatory requirements.

A one-day workshop was convened for the members of the Outcomes Team, the primary aim of which was to give them a practical measurement strategy, which would assist in the selection of appropriate PROs for their phase III and late phase DM studies. The workshop addressed three specific questions posed by the company:

What is achievable using a PRO? 

How can we distinguish between the different measured endpoints? 

How can we understand a PRO score in relation to clinical endpoints? 

The workshop was initiated with a comprehensive and interactive overview of the benefits to the company through the patient's perspective using PROs. This was followed by an in-depth exploration of key stages of the strategy including:

Making explicit the expected treatment effects eg. primary biomedical endpoint(s)

Linking these, through the articulation of an endpoint model, to outcome domains relevant to the patient and disease from which the most appropriate PRO could be selected.

Differentiation between the various secondary endpoints was also explored, such as whether the EQ-5D and SF-36 measured QoL or health status. 

The PRO's conceptual framework, the linkage between the PRO's item content and its specific measurement domains, was emphasised to the team, as was its relationship with the expected primary treatment outcome(s) and study objectives.

Generic and disease-specific PROs, together with their strengths and weaknesses, were also explored. Ways to increase and aid interpretation of the relationships between clinical endpoints and PRO scores were also taken into consideration. 

Selecting the most appropriate PRO to provide evidence of treatment effectiveness, based on the patient's perspective, is a complex process. PROs are frequently and inappropriately selected on the basis of what they appear to be measuring.

The aim of the one-day workshop was to provide the team with a deeper insight into the concepts and tools needed to develop an explicit and successful measurement strategy, to enable the selection of an appropriate PRO to meet the requirements of a specific clinical trial.

Following the workshop, the participants were able to differentiate more clearly between the endpoints purported to be measured by PROs, such as QoL, health status and HRQoL, and select the most appropriate one for a given purpose. 

The team also gained a clearer understanding of the importance of developing an explicit endpoint model to ensure a strong link between the PRO's item content, what it should measure and the objectives of the study for identifying potential treatment benefits. 

The Author
Dr Keith Meadows
is director, DHP Research & Consultancy

28th June 2011


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