Evaluating Medical Devices in Nephrology Using Patient-Reported Outcome and Experience Measures.

Incorporating the patient's perspective into the entire product life cycle of medical device development is paramount for ensuring patient-centric evaluation. By prioritizing patient-centric evaluation, medical device developers can better address patient needs and enhance the quality and effectiveness of health care solutions. Patient-reported outcomes (PROs), patient preference information (PPI), and qualitative inquiry are methodologies to incorporate and amplify the patient's voice. In nephrology, unlike in other clinical domains, the utilization of PROs, PPI, and qualitative inquiry in medical device development has been notably sparse. Consequently, a glaring absence of patient involvement in the development of devices leaves the impact of these devices on patient well-being and functionality largely unexplored. Many forward-thinking programs as well as Food and Drug Administration guidance on the use of PROs and PPI are effectively bringing PROs into nephrology device development. Many resources exist to help researchers select high-quality PROs. There are unique considerations for using PROs and PPI to support regulatory decision-making, including fit-for-purpose, concepts of interest, context of use, and least burdensome selection. The rapid evolution of patient-centric initiatives in nephrology will serve to ensure that medical devices meet the needs of people with kidney disease and improve the quality of care.

Adaptation of the WOMAC for Use in a Patient Preference Study.

OBJECTIVES: To adapt a patient-reported outcome (PRO) measure, the Western Ontario McMaster Universities Osteoarthritis Index (WOMAC), into efficacy attributes for a discrete choice experiment (DCE) survey designed to quantify the relative importance of endpoints commonly used in knee osteoarthritis (KOA) trials. METHODS: The adaptation comprised four steps: (1) selecting domains of interest; (2) determining presentation and framing of selected attributes; (3) determining attribute levels; and (4) developing choice tasks. This process involved input from multiple stakeholders, including regulators, health preference researchers, and patients. Pretesting was conducted to evaluate if patients comprehended the adapted survey attributes and could make trade-offs among them. RESULTS: The WOMAC pain and function domains were selected for adaption to two efficacy attributes. Two versions of the discrete choice experiment (DCE) instrument were created to compare efficacy using (1) total domain scores and (2) item scores for "walking on a flat surface." Both attributes were presented as improvement from baseline scores by levels of 0%, 30%, 50%, and 100%. Twenty-six participants were interviewed in a pretest of the instrument (average age 60 years; 58% female; 62% had KOA for ≥ 5 years). The participants found both versions of attributes meaningful and relevant for treatment decision-making. They demonstrated willingness and ability to tradeoff improvements in pain and function separately, though many perceived them as inter-related. CONCLUSIONS: This study adds to the growing literature regarding adapting PRO measures for patient preference studies. Such adaptation is important for designing a preference study that can incorporate a clinical trial's outcomes with PRO endpoints.

An overview of critical decision-points in the medical product lifecycle: Where to include patient preference information in the decision-making process?

BACKGROUND: Patient preference (PP) information is not effectively integrated in decision-making throughout the medical product lifecycle (MPLC), despite having the potential to improve patients' healthcare options. A first step requires an understanding of existing processes and decision-points to know how to incorporate PP information in order to improve patient-centric decision-making. OBJECTIVES: The aims were to: 1) identify the decision-making processes and decision-points throughout the MPLC for industry, regulatory authorities, and reimbursement/HTA, and 2) determine which decision-points can potentially include PP information. METHODS: A scoping literature review was conducted using five scientific databases. Semi-structured interviews were conducted with representatives from seven European countries and the US, including industry (n = 24), regulatory authorities (n = 23), reimbursement/HTA (n = 23). Finally, validation meetings with key stakeholders (n = 11) were conducted. RESULTS: Six critical decision-points were identified for industry decision-making, three for regulatory decision-making, and six for reimbursement/HTA decision-making. Stakeholder groups agreed that PP information is not systematically integrated, either as obligatory information or pre-set criteria, but would benefit all the listed decision-points in the future. CONCLUSION: Currently, PP information is not considered as obligatory information to submit for any of the MPLC decision-points. However, PP information is considered an important component by most stakeholders to inform future decision-making across the MPLC. The integration of PP information into 15 identified decision-points needs continued discussion and collaboration between stakeholders.

Patient Preferences for the Treatment of Shoulder and Proximal Biceps Disorders Are Associated With Patient Age, Race, Sex, and Activity Level.

BACKGROUND: Patient preference information has become increasingly more important in clinical decision making. PURPOSE: To assess patient preferences when making treatment decisions in the shoulder to determine which features are more important according to patient age, race, activity level, and sex. STUDY DESIGN: Cross-sectional study; Level of evidence, 3. METHODS: Study participants aged ≥18 years were surveyed as to their preferences regarding surgical treatment for shoulder and proximal biceps abnormalities. Survey features included time to return to unrestricted activities, time to return to work, size and appearance of scars, the potential for persistent pain in the upper arm or shoulder, fatigue in the biceps with repetitive lifting, potential for muscle cramping, and deformity in the proximal biceps. Participants also completed a validated shoulder activity scale, and demographic data regarding age, race, and sex were collected. RESULTS: A total of 349 participants (166 female, 183 male) with a mean age of 45 years (range, 18-81 years) completed the survey. Overall, time to return to unrestricted activities and residual pain were considered very important to the majority of the respondents, while the size and appearance of surgical scars were of little importance. Prior shoulder pain (ρ = -0.17; P = .01) and prior shoulder surgery (ρ = -0.16; P = .03) correlated to concern about time to return to unrestricted activities. Younger age (ρ = -0.11; P = .04) and a higher level of education (ρ = 0.14; P = .03) correlated with greater concern for time to return to work. Women and African Americans were more concerned about the size and appearance of surgical scars (ρ = -0.28; P < .0001 and ρ = -0.20; P = .0002, respectively) and biceps deformity. Respondents with a higher activity level (ρ = 0.20; P = .0002) and men (ρ = 0.11; P = .04) were more concerned about fatigue. CONCLUSION: Concerns about residual pain, time to return to unrestricted activities, and time away from work are important to patients when considering the treatment for shoulder lesions. Patient preferences are associated with age, sex, race, and shoulder activity level. These features should be considered when discussing treatment options for shoulder and proximal biceps tendon disorders.