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    PRO data

    Curbing non-adherence with PRO data

    On average, it is estimated that over 50% of patients eventually abandon their treatment. Patient-reported outcomes (PROs) could facilitate efforts to reduce these figures. Defined as any data coming directly from patients about how they feel or function in relation to a health condition or its treatment – with no clinician interpretation to remove the patient ‘voice,’ PROs could help health organisations better optimise their ability to deliver high quality care to each individual who needs it. When patients do not take their medication as intended, patients do not meet their therapeutic goals, biopharmaceutical companies cannot maximise their commercial potential, and payers are burdened with increasing healthcare costs.

    With the transformation from a provider-dominated market to one where the patient is central, there is an increasing emphasis on subjective, rather than objective, patient outcomes. Therapies need to better reflect patient needs if drug developers and healthcare providers hope to align with the reward-for-outcomes that payers insist upon and might place a premium on. PROs are proving to be a great source for insights on patient needs and thus potentially reveal effective solutions to curbing non-persistence with medication. PROs provide information on health, including symptom status, physical function, social function, and wellbeing as reported by the patient.

    PRO data

    Today, data analytics have advanced to the point that PRO data can be used to predict which patients have a relatively high risk for non-adherence. Creating data sources such as patient registries is therefore an important factor in conducting PRO studies and leveraging insights from PRO data. The Swedish rheumatology quality registry, for example, contains information on over 66,000 rheumatoid arthritis patients in Sweden. Patients report on disease-specific and other health and daily function measures, which are tracked simultaneously with on-going treatment. Results inform clinicians on how to deliver improved patient-focused care. Another example is the ImproveCareNow community in the US, which adopts an “all teach, all learn” approach to leveraging patient-sourced insights. This approach is based on collaborative communities where patients, parents, clinicians, and researchers work together to improve the health of youth. Specifically, the community utilises PROs and clinical data to reveal and share better ways to care for children with Crohn’s disease and ulcerative colitis, which has helped improve remission rates across the country.

    PROs and non-adherence assessment
    When certain non-adherence factors are unknown, this could lead to an underestimation of treatment effects. However, by asking questions that reveal a patient’s medication taking behaviour, beliefs about medicines and treatment, and barriers to use of medication, PROs can help detect and assess non-adherence. For instance, one narrative utilised PROs to evaluate health outcomes and thereby determine the costs of multiple sclerosis (MS) treatment. Consequently, PROs were used to measure patient satisfaction and identify patient preferences for care, which were found to be determinants of adherent behaviour. Understanding factors affecting patient satisfaction, treatment preference, and adherence can guide care providers in designing therapies that are tailored to the needs of patients.

    So, why are patients non-adherent or non-persistent? Some of the identified predictors for non-adherence include low satisfaction with medical information, poor perception of disease, personal beliefs about drugs, and having a poor relationship with one’s physician. Patients who perceive side-effects more negatively, report lower levels of quality of life, or who are overwhelmed by the complexity of the medication regimen and the required lifestyle modification are also likely to fall behind in their treatment.

    Consequently, PROs may not only reveal which patients are at risk during treatment, but also those who are at risk prior to starting a treatment. A study that evaluated PRO data from patients with the hepatitis C virus (HPC) suggested that unemployment, depression, being treatment-naïve, and having low social well-being were predictors of non-adherence.

    Non-adherence, especially when it is intentional, can be taken as an indication that better medicines and support access opportunities are in order; and PRO insights may be used to support better adherence. Gaining insights from patients is especially important since factors such as perception of symptoms that influence non-adherence can vary across medicine/disease types and patient demographics. Indeed, a Pan-European study on people suffering from chronic obstructive pulmonary disease (COPD) found that patients experience tremendous variability in how they perceive their symptoms. Pain is also perceived differently across cultures. Those with Mediterranean and Hispanic backgrounds are commonly expressive of pain, while those with Asian and Northern European backgrounds tend to be less expressive, which can affect the outcomes reported and the medications and treatments preferred.

    In addition, although researchers prefer to use PROs for their ease of administration, clinicians on the other hand often find PROs a hassle. With the possible physician hurdle and the intricacies across patient groups, the design, administration, analysis and reporting of PROs must carefully be considered and planned to ensure that non-adherence factors are accurately identified. Any gaps in the outcomes reported could compromise the statistical methods conducted and serve as barriers to generating valuable PRO insights. The ideal PRO meets the following criteria:

    • Specific to the concept being measured
    • Based on end-point model
    • Based on the conceptual framework
    • Contains an optimum number of items
    • Is easy to score and easy to interpret
    • Has evidence for a conceptual framework
    • Maintains the confidentiality of the patient
    • Is reproducible

    A tool for precision medicine
    Pharma companies utilise PRO insights to evaluate how their products are performing against competitor drugs and whether health targets are being met. However, at-risk patients will need a different approach and PRO insights can also be used as an ingredient for healthcare marketers or pharma companies in deciding which patients or patient types are in need of more focused support services and engagement efforts.

    Undeniably, human behaviour plays a role in non-adherence. Two patients could be faced with the same set of non-adherence risk factors, but they could still respond or behave in a different manner. Given the subjective nature of PROs, however, these tools are suited to enable companies to better understand patients as individuals. Furthermore, PRO insights may be used to inform the creation of treatment pathways that are specific to an individual’s behaviour and clinical response; however not all PRO measures have sufficient accuracy to be used at the level of the individual. Insights from patient reports can therefore be used to justify and promote the efforts of healthcare companies to create personalised, targeted services.

    Personalised treatment includes the development and matching of drugs and therapies with the genomic profile of an individual patient, which helps ensure positive treatment effects. Personalised treatment is showing much promise in certain therapeutic areas, such as oncology, but is still experiencing a slow uptake in other areas.

    Medication adherence remains the core solution to ensuring better patient outcomes. Additionally, even if patients are given personalised treatments (i.e. they are given treatments and medications that are reportedly more compatible with their genetic makeup), if they do not show up to treatment sessions or take their medication, then the promise of personalised medicines is rendered moot. As filmmaker Woody Allen once said, “Eighty percent of success is showing up.” With lack of adherence, any effort to deliver quality treatments is thwarted.

    The decision to become conscious partners in the treatment process ultimately rests with patients. However, to combat non-adherence and truly make the treatment journey a personalised one, healthcare and pharma companies will need to enlist the help of physicians, behavioural scientists, and data experts, who can aid in utilising PRO insights to design user-centric treatments that ensure compliant behaviour and improved health outcomes.

    To learn how our experts at Valid Insight can help you understand how to select and create the right PRO to support your product, contact us at

    In addition, to learn more about using patient-reported outcomes in clinical trials and studies, join our experts in our upcoming webinar:

    Patient-reported outcomes in clinical trials and studies

    Monday 10 October
    15:00 BST, 16:00 CEST, 10:00 EDT

    Register here for this free webinar:


    1. World Health Organisation. 2003. Adherence to long terms therapies: Evidence for action. [Online]. [Accessed 21 September 2016]. Available from:
    2. Cramer, J.A., Roy, A., Burrell, A., Fairchild, C., Fuldeore, M.J., Ollendorf, D.A., Wong, P.K. 2008. Medication compliance and persistence: Terminology and definitions. International Society for Pharmacoeconomics and Outcomes Research (ISPOR). 1098-3015/08/44. pp. 44–47. [Accessed 21 September 2016].
    3. 2009. Guidance for industry. Patient-reported outcomes measures: Use in medical product development to support labelling claims. [Online]. [Accessed 21 September 2016]. Available from:…/Guidances/UCM193282.pdf


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