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Background and objectivesDurvalumab and atezolizumab are approved as first-line therapy in extensive-stage small-cell lung cancer. Although cost-effectiveness analyses compared these immunotherapy drugs with standard chemotherapy-alone regimens, no head-to-head cost-effectiveness comparisons for these treatments exist. The aim of the present analysis is to determine the cost-effectiveness of durvalumab and atezolizumab as first-line therapy for extensive-stage small-cell lung cancer from the US payers' perspective.MethodsThis study is based on two placebo-controlled, phase 3 clinical trials: CASPIAN and IMpower133. A Markov model was developed to simulate the three health states: progression-free survival, progressed disease, and death in patients with extensive-stage small-cell lung cancer. Transition probabilities were estimated from the clinical trial survival curves and extended with life-time modelling. Health utilities and direct costs of adverse event treatment were included. Main outcome was the incremental cost-effectiveness ratio (ICER) using quality-adjusted life-years saved (QALYS). Sensitivity analysis was performed to assess the impact of variables on the ICER.ResultsDurvalumab group has a cost of $187,503 with an effectiveness of 1.08 while atezolizumab has a cost of $160,219 and an effectiveness of 0.932. Durvalumab is not cost-effective compared to atezolizumab with an ICER of $165,182 QALYS, which is over the willingness-to-pay threshold of $150,000. The model was most sensitive to durvalumab cost and the cost of treating durvalumab adverse effects.ConclusionsWith the ICER of durvalumab treatment group being very close to $150,000, setting a higher willingness-to-pay threshold or decreasing the drug cost through contract pricing can increase the cost-effectiveness of durvalumab compared to atezolizumab.

Due to the complexity and fragility of biological drug products, several challenges exist in their formulation development. Excipients are added to increase product stability, maintain tonicity, and facilitate drug delivery. The potential implications of these additive substances merit clinical consideration. We assessed the safety risk of excipients on the basis of their type and variability through an assessment framework, which quantifies excipient complexity in 230 biological formulations, and identifies excipient-related adverse events through published case reports. A biologic on average contained 4.45 excipients, half of that found in oral medications. The frequency distribution was heavily skewed towards the most commonly occurring excipients: water (40.4%), sodium chloride (38.3%), polysorbate 80 (28.7%), sucrose (24.4%), and mannitol (20.9%), with 44.4% of formulations not listing the concentration of the most commonly occurring inactive ingredients. A literature search revealed only 17 case reports of excipient-related adverse events, suggesting the need for more clarity for clinicians on the safety of chemical additives. These cases included injection site reactions, anaphylaxis, hyperglycemia, and acute renal failure. With the expansion of the biopharmaceutical market, it is important to consider the safety data of biologic excipients, so that therapy can be tailored appropriately for a specific patient.

Introduction: Understanding individual patient preferences for chronic low back pain (cLBP) outcomes is essential for targeting available therapeutic options; yet tools to elicit patient outcome preferences are limited. Objective: To develop and test a choice‐based conjoint (CBC) measure, commonly used in behavioral economics research, to elicit what outcomes patients with cLBP want to achieve and avoid. Design: We developed a survey‐based CBC measure to allow patients to make risk/benefit trade‐off choices between possible treatment outcomes. After extensive literature, clinician, and patient input, our measure included seven attributes: fatigue, anxiety/depression, difficulty thinking/making decisions, pain intensity, physical abilities, change in pain, and ability to enjoy life despite pain. Random‐parameters logit models were used to estimate strength of preferences, and latent class analysis was used to identify patient characteristics associated with distinct preference. Setting: Online study using the Sawtooth web‐based platform. Participants: Two hundred eleven individuals with cLBP recruited from online advertising as well as at clinical sites across multiple academic and private institutions. Interventions: Not applicable. Results: The most valued outcome was the highest level of physical activity (β = 1.6–1.98; p <.001), followed by avoiding cognitive difficulties (β = −1.48; p <.001). Avoidance of severe pain was comparable to avoiding constant fatigue and near‐constant depression/anxiety (β = −0.99, −1.02); p <.001). There was an association between preferences and current pain/disability status; patients with higher pain had a stronger preference to avoid severe pain, whereas those with higher disability have stronger preferences for achieving physical activity. The latent class analysis identified two distinct groups: (1) more risk‐seeking and willing to accept worse outcomes (56%); and (2) more risk‐averse with a stronger preference for achieving maximum benefits (44%). Conclusions: Our study illuminated cLBP patient preferences for treatment outcomes and heterogeneity in these preferences. Patients stressed the importance of reaching high physical activity and avoiding cognitive declines, even over a desire to avoid pain. More work is needed to understand patient preferences to aid informed, shared decisions. [ABSTRACT FROM AUTHOR]

Introduction: Understanding individual patient preferences for chronic low back pain (cLBP) outcomes is essential for targeting available therapeutic options; yet tools to elicit patient outcome preferences are limited., Objective: To develop and test a choice-based conjoint (CBC) measure, commonly used in behavioral economics research, to elicit what outcomes patients with cLBP want to achieve and avoid., Design: We developed a survey-based CBC measure to allow patients to make risk/benefit trade-off choices between possible treatment outcomes. After extensive literature, clinician, and patient input, our measure included seven attributes: fatigue, anxiety/depression, difficulty thinking/making decisions, pain intensity, physical abilities, change in pain, and ability to enjoy life despite pain. Random-parameters logit models were used to estimate strength of preferences, and latent class analysis was used to identify patient characteristics associated with distinct preference., Setting: Online study using the Sawtooth web-based platform., Participants: Two hundred eleven individuals with cLBP recruited from online advertising as well as at clinical sites across multiple academic and private institutions., Interventions: Not applicable., Results: The most valued outcome was the highest level of physical activity (β = 1.6-1.98; p < .001), followed by avoiding cognitive difficulties (β = -1.48; p < .001). Avoidance of severe pain was comparable to avoiding constant fatigue and near-constant depression/anxiety (β = -0.99, -1.02); p < .001). There was an association between preferences and current pain/disability status; patients with higher pain had a stronger preference to avoid severe pain, whereas those with higher disability have stronger preferences for achieving physical activity. The latent class analysis identified two distinct groups: (1) more risk-seeking and willing to accept worse outcomes (56%); and (2) more risk-averse with a stronger preference for achieving maximum benefits (44%)., Conclusions: Our study illuminated cLBP patient preferences for treatment outcomes and heterogeneity in these preferences. Patients stressed the importance of reaching high physical activity and avoiding cognitive declines, even over a desire to avoid pain. More work is needed to understand patient preferences to aid informed, shared decisions., (© 2023 The Authors. PM&R published by Wiley Periodicals LLC on behalf of American Academy of Physical Medicine and Rehabilitation.)