Patient experience and quality of life during neoadjuvant therapy for pancreatic cancer: a systematic review and study protocol.

PURPOSE: Neoadjuvant therapy (NT) is increasingly being offered to patients with pancreatic ductal adenocarcinoma (PDAC) prior to surgical resection. However, the experience and quality of life (QOL) of patients undergoing NT are poorly understood. METHODS: A systematic review of the Cinahl, Embase, Medline, Pubmed, Scopus, and Web of Science databases was conducted to evaluate the available literature pertaining to the experience and QOL of patient's undergoing NT for PDAC. RESULTS: Among 6041 articles screened, only six met criteria for full-text review including three prospective clinical trials of NT with QOL secondary endpoints. Overall, global QOL during or following NT did not significantly change from baseline. Pain scores seemed to improve during NT while the impact of NT on physical functioning varied across studies. No studies were identified evaluating other aspects of the patient experience. CONCLUSION: Although NT appears to have a minor impact on the QOL of patients with PDAC, this systematic review identified significant evidence gaps in the literature. A protocol of a prospective observational cohort study utilizing a digital smartphone app that aims to evaluate the patient experience and longitudinal QOL of patients with PDAC undergoing NT is presented.

Patient and caregiver preferences for the potential benefits and risks of a seizure forecasting device: A best-worst scaling.

BACKGROUND: Epilepsy is the 4th most common neurological disorder and is characterized by recurrent, unpredictable seizures. The ability to forecast seizures is a significant unmet need and would have a transformative effect on the lives of people living with epilepsy. In an effort to address this need, the Epilepsy Foundation has committed effort and resources to promote the development of seizure forecasting devices (SFD). OBJECTIVE: To promote user-centered design of future SFD, we sought to quantify patient and caregiver preferences for the potential benefits and risks of SFD. METHODS: A community-centered approach was used to develop a survey incorporating a novel best-worst scaling (BWS) to assess preferences for SFD. A main-effect orthogonal array was used to design and generate 18 "prototypes" that systematically varied across six attributes: seizure forecasting probability, seizure forecasting range, inaccuracy of forecasting, amount of time required to use the device, how the device is worn, and cost. The dependent variable was the attributes that respondents selected the best and worst in each profile, and a choice model was estimated using conditional logistic regression, which was also stratified and compared across patients and caregivers. Respondents also indicated that they would accept each of the prototype SFDs if it were real. These acceptance data and net monetary benefits (relative to the least preferred SFD) were explored. RESULTS: There were 633 eligible respondents; 493 (78%) completed at least one task. Responses indicated that 346 (68%) had epilepsy, and 147 (29%) were primary caregivers or family members of someone with epilepsy. The data show that short forecasting range is the most favored among experimental attributes, followed by mid forecasting range and notification of high chance of seizure. Having the device implanted is the least favorable attribute. Stated preferences differed between patients and caregivers (p < 0.001) for range of forecasting and inaccuracy of device. Caregivers preferred any range of forecasting, regardless of length, more than patients. Patients cared less about inaccuracy of the device compared to caregivers. The groups also differ in impact of fear of having seizures (versus actually having seizures) (p = 0.034) and on device acceptance. The acceptance of devices ranged from 42.3% to 95%, with caregivers being more likely to use a device (p < 0.05) for the majority of device profiles. Acceptance of devices varied with net monetary benefit of the best device being $717.44 more per month relative to the least preferred device. CONCLUSION: Our finding extends previous calls for seizure forecasting devices by demonstrating the value that they might provide to patients and caregivers affected by epilepsy and the feature that might be most and least desirable. In addition to guiding device development, the data can help inform regulatory decisions makers.

Line of therapy and patient preferences regarding lung cancer treatment: a discrete-choice experiment.

OBJECTIVE: A growing literature on patient preferences informs decisions in research, regulatory science, and value assessment, but few studies have explored how preferences vary across patients with differing treatment experience. We sought to quantify patient preferences for the benefits and risks of lung cancer treatment and test how preferences differed by line of therapy (LOT). METHODS: Preferences were elicited using a discrete choice experiment (DCE) following rigorous patient and stakeholder engagement. The DCE spanned five attributes (each with three levels): progression-free survival (PFS), short-term side effects, long-term side effects, risk of developing late-onset side effects, and mode of administration (MOA) - each defined across 3 relevant levels. A D-efficient design was used to generate 3 survey blocks of 9 paired-profile choice tasks each and respondents were asked which profile they preferred and then if they preferred to have no treatment (opt-out). A mixed logit model, controlling for opt-out, was used to estimate preferences. Preferences and trade-offs between PFS and other attributes were compared across two groups: those receiving ≤1 LOT and those receiving ≥2 LOT. RESULTS: Of the 466 participants, 42% received ≤1 LOT and 58% received ≥2 LOT. Stated preferences differed between the groups overall (p<.001) and specifically for 18 months of PFS (p<.001), moderate short-term side effects (p<.001), no long-term side effects (p=.03), and 30% chance of late-onset side effects (p=.02). Those receiving differing amounts of LOT were willing to trade different amounts of PFS to change from moderate to mild short-term side effects (p<.001), moderate to no (p<.001) and mild to no (p<.001) long-term side effects. There were also differing amounts of tradeoff acceptable between the groups for a 10% decrease in risk of late-onset side effects (p=.016), a decrease in MOA from infusion every 3 weeks to pills taken daily at any time (p=.005) and from pills taken daily without food to pills taken daily at any time (p<.001). CONCLUSION: We demonstrate differences in preferences based on experience with LOT, suggesting that patient treatment experience may have an impact on their preferences. As patient preference data become an important component of treatment decision making, preference differences should be considered when recommending therapies at different stages in the treatment journey. Understanding patient preferences regarding treatment decisions is essential to informing shared decision-making and ensuring treatment plans are consistent with patients' goals.