Using patient-reported outcome assessments in clinical practice and their importance in risk management

Abstract

Patient-reported outcome (PRO) instruments are related to risk management programmes in that they are tools to measure the benefits and risks of exposure to pharmaceutical products from the patient's perspective. Clinical measures of improvement of certain conditions may not necessarily correlate with improvements in a patient's ability to perform daily activities. PRO data, when properly administered, collected, analysed and returned to physicians are a very useful source of information. This will ultimately address safety concerns, facilitate the physician–patient relationship and improve patients' compliance to treatment in routine patient care. In this article we stress the importance of PRO in risk management.

Key words::

• patient-reported outcome assessment
• risk management

Dear Sir,

The need for balance between the risks to a study subject and the potential benefits to that subject or to society is an important principle in clinical research¹. The European Medicines Agency (EMEA), the Food and Drug Administration (FDA) and regulatory agencies across the globe are placing more emphasis on post-approval safety efforts. Unsolicited (i.e., spontaneous) reporting has been a traditional method of assessing the safety of medical products in the period after regulatory approval. Currently, risk management activities include, in addition to spontaneous reporting, risk minimisation action plans (risk-MAPs) and recently-introduced risk evaluation and mitigation strategies (REMS). In general, risk-MAPS involve patient education and outreach programmes, reminder systems, and performance-linked access systems, while REMS are risk-management strategies that specifically address management of known or potential serious risk associated with a medical product, describe precisely how it will be prescribed to help assure safe use, and outline the mechanisms by which physicians and patients will be warned of the major risks associated with the use of a medical product. Instruments measuring patient-reported outcomes (PRO) are related to risk management programmes in that they measure the benefits and risks of exposure to pharmaceutical products from the patient's perspective. Over the last two decades, PRO assessments have gained high value in clinical research². However, the use of PRO instruments, especially those designed to detect negative treatment-related symptoms has not gained widespread acceptance in routine patient care. It has been reported that an alarmingly high percentage of patients (over 30%) do not spontaneously report to their clinician any side-effects they experience during their treatment³. This may lead to physicians underestimating the frequency and impact of side-effects experienced by patients. Furthermore, patients who perceive that the side-effects they experience are treatment-related may become non-adherent to the treatment, leading to economic consequences due to increased hospitalisation and productivity losses⁴. Good communication between healthcare professionals and patients is recognised to be central to disease management. Previous research suggests that retrieval of PRO data from individual patients facilitates physician–patient communication, helps to monitor treatment of disease over time, and therefore improves the delivery of medical care^5–7. Real-time PRO measurements are feasible even in busy clinical practices⁸. Technological advances such as the use of PRO assessment via telephone interactive systems and the internet to monitor treatment and guide treatment recommendations are an effective way of enhancing doctor–patient communications⁹, especially in health systems with reduced contact time with physicians because of time constraints. For use of PRO measures in routine clinical practice it is necessary to have publicly available, clear and easy-to-use PRO instruments with several alternative modes of administration for the patients. PRO administration to patients, electronic flow of responses from patients to physicians and automated analysis of PRO data having a one-page summary of the patient-reported data for the physician to review in an easily understandable format, which flags any significant symptom changes, would result in a better and early quantification of clinically important symptoms and outcomes that are currently difficult to capture in a routine clinical practice. Such PRO administration and data management should be cheap enough not to drive up health insurance costs further. PRO data, when properly administered, collected, analysed and returned to physicians are a very useful source of information. This will ultimately address safety concerns, facilitate the physician–patient relationship and improve patients' compliance with treatment in routine patient care. Pilot projects are needed to investigate ways in which physicians can effectively use PRO measures in routine clinical practice and how use of PRO can improve risk management in clinical practice cost-effectively.

Funding: none.

Declaration of interest: none declared.