While scientists, clinicians, and regulators play critical roles in understanding and communicating the benefits and risks of drugs/medical treatments, only patients live with their medical conditions and make choices regarding their personal care. They provide a unique voice and unique perspective. In recent years, more and more studies are focusing on patient reported outcomes (PROs). As a result, increasing number of patients are becoming aware of the healthcare outcomes perspective. However, the exact role of the PROs in understanding the patient-centered care is a little unclear.
There come into existence the patient preference studies, which are a distinct class of methods being extensively used in medicine as they’re related to PROs, health related quality of life and the expected-utility methods used to motivate quality adjusted life years (QALYs). “Patient perspectives” refer to a type of patient input, and includes information relating to patients’ experiences with a disease or condition and its management. This may be useful for better understanding the disease or condition and its impact on patients, identifying outcomes most important to patients, and understanding benefit-risk tradeoffs for treatment. This guidance focuses on “patient preference information” as one specific type of patient perspective. Patient preference studies are far more grounded in economic theory and far more patient-centered but more importantly, they should be really flexible to capture interests of most of the outcome researchers.
Patient preference studies can be designed in several ways; they can focus either on the total value of medical interventions; they can be used to evaluate hypothetical treatments; they can address issues of patient choice, and hence can be used to understand diseases like obesity, diabetes, and coronary-artery disease where long term prognosis depends directly on patient lifestyle choice; they can evaluate patient adherence or process-related aspects of healthcare. Therefore, patient preference studies can provide an alternative method for characterizing patients’ needs and wants. However, although they complement the randomized clinical trials, patient preference studies do not replace them. This is because more patients with treatment preferences in a trial may affect the randomization process and the absence of such patients may not provide generalizable results as participants may not be representative.
Having said that, measuring patient preferences within a fully randomized design deserves further use as this conserves all the advantages of a fully randomized design with the additional benefit of allowing for the interaction between preference and outcome to be assessed. Furthermore, preference methods are flexible and adaptable to practically any health-related question and are thus suitable for quantifying the effect of treatment features on monetary valuations related to decision-making, risk-benefit tradeoffs, patient compliances, and other healthcare outcomes.
The researchers must understand the importance of patients’ preferences while decision making. It is important to acknowledge that individual patient preferences may vary and that a patient may not assign the same values to various risks and benefits as his/her healthcare professional, a family member, regulator, or another individual. Furthermore, patient preferences may vary both regarding perspective on benefits and risks, as well as in preferred modality of treatment/diagnostic procedure (e.g., often devices are one option to be considered in a treatment care path, which may include surgery or medication). Some patients may be willing to accept higher risks to potentially achieve a small benefit, whereas others may be more risk averse, requiring more benefit to be willing to accept certain risks.
It is clear that patient preference methods present an alternative method for characterizing patient needs and wants. Unlike PRO and/or HRQoL methods, the focus is on understanding the relative importance of attributes via revealed or stated preferences. Preference methods are flexible and adaptable to practically any health-related question and are thus uniquely suited to quantifying the effect of treatment features on adherence, the tradeoffs between health outcomes and other treatment features, the risk-benefit tradeoffs, and/or monetary valuations related to treatment options. Patient preference methods offer a scientifically rigorous alternative to traditional patient-centered outcomes research methods and are worth a closer look.