Co-creation of the Global Patient Experience Data Navigator: a multi-stakeholder initiative to ensure the patient voice is represented in health decision-making.

BACKGROUND: Putting patients' needs and priorities at the forefront of healthcare initiatives and medical product development is critical to achieve outcomes that matter most to patients. This relies on the integration of early, meaningful patient engagement (PE) to learn what is important to patients, and collection of representative patient experience data (PXD). The increased number of PE/PXD efforts across global regulatory, health technology assessment, and healthcare systems is an important step forward to deliver improved health outcomes for patients. However, these initiatives are fragmented and lack integration, which is necessary to maximize efforts and reduce burden on patients. To overcome these challenges, the Global Patient Experience Data Navigator has been co-created by Patient Focused Medicines Development to provide practical resources that can facilitate and optimize PXD generation, collection, analysis, and dissemination for patient benefit and aims to be applicable across all therapeutic areas for all stakeholders. METHODS: Co-creation of the Navigator took place through an iterative process of validation and formalization driven by a diverse, multi-stakeholder working group with individuals who have varying knowledge/experience in PE/PXD. RESULTS: A series of workshops took place to conduct a gap analysis, develop a taxonomy model, and integrate existing frameworks. The collective insights led to the development of the Navigator consisting of four specific tools in the form of downloadable templates, which can be used to: (1) prioritize outcomes that matter most to patients and their caregivers; (2) select appropriate measurement methods for these outcomes; (3) identify when and why PXD is used throughout the product development cycle for each stakeholder; (4) identify when and why PXD is used throughout the healthcare process for each stakeholder. A public consultation was carried out to collect user feedback before the Navigator was made publicly available in December 2022. CONCLUSION: To our knowledge, the Global Patient Experience Data Navigator is the only publicly available toolkit developed with a multi-stakeholder and disease-agnostic approach providing taxonomically grouped resources to optimize the collection and collation of PXD for patient benefit. Future work will aim to further engage patients by adding a PE dimension to the Navigator.

Engaging patients at the start of healthcare and medical product development projects can help better understand their experiences and what is most important to them. Ultimately this will achieve the best outcomes. However, if not carefully planned, projects that engage patients can lead to inefficiencies, such as patients being asked for the same information repeatedly. The collection of patient experience data­information related to patients' experiences, needs, and priorities­also needs to be carefully managed. To help solve this problem, Patient Focused Medicines Development developed a publicly available "toolkit" called the Global Patient Experience Data Navigator. The Navigator has downloadable templates that can be filled in and used for projects in any disease area and by anyone collecting patient experience data. To represent different perspectives, individuals with a range of experiences and understanding of patient engagement projects worked together to co-create the toolkit. Several meetings took place to understand what the toolkit needed to do and to help provide a structure for the templates. There are four templates in the toolkit. These can be used to: (1) prioritize outcomes that matter most to patients and their caregivers; (2) select the best ways of measuring these outcomes; (3) identify when, why, and by whom patient experience data is used throughout the product development cycle; and (4) identify when, why and by whom patient experience data is used throughout the healthcare process. Future work will utilize public feedback to make the toolkit more user-friendly and provide education on how the Navigator can be used.

Measuring intraoral pressure: adaptation of a dental appliance allows measurement during function.

This article introduces a new way of recording intraoral pressures from a range of locations within the oral cavity. To measure pressure flow dynamics during swallowing, we fitted eight miniature pressure transducers capable of measuring absolute pressures to a chrome-cobalt palatal appliance with a labial bow. Unlike previous devices, our design provides a rigid, custom-fitted platform for the simultaneous recording of pressures at eight locations within the oral cavity during function. We placed an anterior pair of gauges to measure lingual and labial contact against the left central incisor tooth, and two pairs of gauges to measure pressure contributions of the lateral tongue margin and cheeks on the canine and first molar teeth. Finally, lingual pressure on the midline of the palate was measured by two gauges, one at the position of the premolars and one on the posterior boundary of the hard palate. We then recorded intraoral pressures in five adult volunteers seated in an upright position and asked to swallow 10 ml of water. Labial pressures on the canine rose rapidly from a resting level of 10 kPa to 33 kPa, while pressure profiles from the labial aspects of the incisor and first molar teeth followed a negative pattern, peaking at -12 kPa for the incisor and -15 kPa for the molar sensor. Pressure profiles recorded from the palatal aspects of the first molar and the canine appeared to be similar, but the former fell to -13 kPa before rising to 9 kPa, and the canine pressure rapidly increased to 22 kPa before returning to its resting level of 4 kPa. The pressure profile of the palatal aspect of the central incisor was strikingly different; at the start of the swallow, pressure dropped precipitously to -20 kPa, before slowly rising to 10 kPa. It then followed the general pattern of the other two sensors, before peaking again at 10 kPa and then returning to a resting level of 4 kPa. We also showed that there were significant negative pressures in the mouth during function, and that pressure profiles varied markedly between individuals.

The investigation and correction of recall bias for an ordinal response in a case-control study.

Consider a case-control study designed to investigate the possible association between development of a particular disease and the value of a putative risk factor measured on an ordinal scale. Let E denote a subject's true risk factor value and let E* denote a subject's recorded risk factor value. Misclassification bias occurs if conclusions reached regarding the relationship between disease status and E* do not also apply to the relationship between disease status and E. We propose a model for the conditional probability distribution of E* given E. We show how the model may be used to investigate misclassification bias in a validation study where measurements of E* and E are available for both cases and controls and apply the methods developed to data from a test-retest study of recall bias in the context of screening for hypertension. We also consider a situation where the validation study is carried out on a subset of the subjects within a larger case-control study. In that case, values for E* are available for all subjects but values for E are available only for those subjects included in the validation study. We show how correct likelihood-based inference concerning association between disease status and risk factor value may be carried out using all of the available data. A Monte Carlo study shows how the inclusion of a validation study leads to a correction of recall bias problems at the cost of an increased standard error for the estimated association parameter.