The patient’s perspective is becoming increasingly important in healthcare decision making. Patients as “end users” should be involved in the health technology assessment (HTA) process to broaden the perspectives of assessments and advice provided to decision makers . Patients can provide information on their daily experiences with diseases and treatments, their needs, their views on aspects not measured in studies, or they can state their preferences for various therapy aspects and treatment endpoints.
Inclusion of patient perspectives in health technology assessments (HTA)
HTA agencies include patient views in their appraisals in different ways and to various extents.
Patient views and preferences can be captured by engaging either patient organizations, patients themselves, or informal caregivers during the HTA process (as is done, for example, in the UK and Canada). Such patient aspects can also be explored in qualitative and quantitative studies, which are considered as part of evidentiary documentation by the assessment committees.
Patient preferences differ from patient-reported outcomes
Clinical studies often include patient-reported outcomes, such as quality of life, that report the patient’s status in several dimensions (e.g., physical, mental, functional, social and emotional). These outcomes are focused on the health state the patient is in, rather than trying to identify or evaluate how patients feel about being in that state of health. On the other hand, patient preferences are concerned with measuring how the patient values a specific component or attribute. By exploring how patients trade between various components, the relative value of each component can be determined .
About a quarter of early benefit dossiers referred to data on patient preference
GfK’s market access team explored patient preference data, how manufacturers included the data in early benefit dossiers and how the Institute for Quality and Efficiency in Healthcare (IQWiG) or the Federal Joint Committee (G-BA) subsequently evaluated the data. Data from January 1, 2011, to March 31, 2014, was reviewed, and the type of patient preference data incorporated in the early benefit dossiers along with their associated assessments was collated and summarized.
A total of 68 dossiers were analyzed: 18 dossiers (26%) included data on patient preferences. As shown in Table 1, patient preference data related to:
a) relevance of different treatment end points from the patient’s perspective in oncology, hepatitis C, diabetes, or HIV infection
b) patient preference for a specific drug administration route (e.g., oral vs. injection), administration system (e.g., different inhalation systems), or administration frequency
c) patient preference for therapy duration or type of therapy
In none of the assessment reports did the evaluating committee specifically address the evidence presented on patient preference. A comment on patient preference data was given in one assessment report only (for aclidinium bromide). The manufacturer stated that patient preference for their drug delivery system positively impacts patients’ quality of life. The assessors, however, noted that patient preference for the administration system is not a measure of quality of life, which itself relates to physical, mental, and social well-being of a person.
Data on patient preference appears not to play an important role in early benefit assessments
Surprisingly, it appears that evidence on patient preference has not been considered in the Arzneimittelmarkt-Neuordnungsgesetz (AMNOG, English translation: “Pharmaceuticals Market Reorganisation Act)early benefit assessments despite the fact that benefit to the patient is the central criterion of the AMNOG early benefit assessment.
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