The Importance of Patient Reported Outcomes in Compliance

Rob Arbuckle, associate director at Mapi Values, explains how patient reported outcomes impact adherence.



Rob Arbuckle, associate director at Mapi Values, explains how patient reported outcomes impact adherence.



The FDA has defined patient reported outcomes (PROs) as the measurement of any aspect of patient health that comes directly from the patient. Rob Arbuckle, associate director at Mapi Values, explains that this is usually done using a questionnaire. Example measures are pain and quality of life.


Pain will typically be a primary efficacy variable, whereas quality of life (QoL) will usually be secondary. Other outcomes include patient satisfaction and treatment adherence.


Quality adjusted life years (QALYs) are not familiar to US audiences, but are widely used in Europe for reimbursement decisions. Such measures stand alongside established ones, such as cardiovascular and respiratory parameters.


Why use PROs?


The primary reason, says Arbuckle, is to assess treatment effects that are known only to the patient. Pain is the classic example. PROs can also support established measures, by assessing the impact of an intervention on daily life.


Indeed, sometimes PROs show that the supposedly objective measure is not reflecting the impact correctly. Arbuckle also makes the important point that, whereas the objective measure may well be aimed at what you put on your label, the PRO will put that into the context of real life, and be highly relevant to marketing.


PROs and adherence


Because patient experience is the key to adherence, PROs are a way to measure that experience. The development of a PRO instrument is a very systematic and rigorous process, according to Arbuckle, resulting in a validated questionnaire.


He illustrates this process with a questionnaire called Self-Assessment and Goal Achievement (SAGA) in the field of urinary symptoms. It is patient-completed and physician-reviewed.


Most questionnaires include a standard list of questions for all patients, but SAGA selects questions that mean the most to each particular patient. SAGA is related to QoL but does not replace it. Arbuckle explains that the patient completes the questionnaire before seeing the doctor, who then uses it to structure the consultation. It helps the doctor manage the patient's expectations, which in turn strongly impacts adherence.


PROs and reimbursement


Arbuckle describes a patient study that demonstrates this link. Some patients who have frequent blood transfusions accumulate iron, and this has to be removed using chelation therapy. This usually has to be done several times a week, using subcutaneous injections, so has a substantial impact on the patient.


The availability of an oral treatment clearly has advantages, and this was the model for the study.  The first step in the project was to collect data on patient impacts, including QoL, and to publish this widely to encourage criticism and consultation. Analysis showed that these impacts did hinder treatment compliance, and ultimately had an effect on survival.


Although the data didn't make it into the regulatory submission, says Arbuckle, because we published it, the reimbursement authorities did seem to take it into account.


This was very clear in France, where a price premium was achieved, and strongly indicative in the UK, he emphasizes: The message is, if you have the data and you publish it, even if it's not going to be part of your submission, it can still impact on these decisions.


Raising awareness


Arbuckle gives the example of a pediatric drug, for which slow uptake seemed to be related to adherence. In this case, a Web-based symptom measure for asthma was used. Again, symptom scores were collected before the consultation, enabling the doctor to assess more effectively the level of symptom control.


But Arbuckle clarifies that such tools are not always disease-specific, highlighting a generic questionnaire that can be used to identify actionable items to improve adherence.


PROs can also be used to raise disease awareness, with restless legs syndrome (RLS), for example. Contrary to lay media claims, PROs have exposed real impacts on patients from RLS, and it clearly is not an invented disease.


In particular, Arbuckle reports that having qualitative data on the website helps patients describe the sensation that they have in their legs. Public sector bodies endorse such tools.


PROs have various audiences, and Arbuckle emphasizes that one size does not fit all. Ultimately, it is all about talking to patients.