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Patient advocates need to process vast amounts of information to accurately and effectively represent heterogeneous patient groups and make meaningful contributions to HTA decisions. Although a wealth of data is available from a variety of sources, it is not often curated in user-friendly ways. Patient representatives have frequently requested tailored resources that allow them to mine the existing literature in preparation for their engagements. Developing such resources constitutes a complex challenge that requires contributions and scrutiny from multiple stakeholders.
We previously developed the Continuous Innovation Indicators™ (CII), an evidence-based tool to assess treatments for twelve solid tumors (freely available at www.scoringprogress.com). The foundation of the CII is a rigorous assessment of published evidence for increased overall survival. Based on feedback from patient advocates, we are expanding the framework to include information on adverse events and other patient-centered outcomes for selected prototype indications.
We present a novel, flexible framework that combines evidence of efficacy with published results on other outcomes that matter to patients. Menus and outputs are designed to facilitate dialogue between advocates, clinicians, and HTA professionals. By allowing the user to adjust settings based on known heterogeneity among subpopulations, the tool's output can be used to inform discussions about the value of new interventions for defined patient segments.
Patient representatives must frequently identify knowledge gaps in the literature before their HTA engagements and leverage this information to conduct surveys among their constituents. Our new patient advocate decision aid can support this process and facilitate a better understanding of the value of new innovations for diverse subgroups. A better definition of target populations will help to achieve balance between patient access and budget impact of new treatments. We seek feedback on our prototype from all stakeholders to further improve and maximize utility of this tool.
Aripiprazole lauroxil (AL) is a long-acting injectable atypical antipsychotic that was evaluated for the treatment of schizophrenia in a randomized, placebo-controlled, Phase 3 study. Here, we present exploratory analyses of supportive efficacy endpoints.
Patients experiencing an acute exacerbation of schizophrenia received AL 441 mg intramuscularly (IM), AL 882 mg IM, or matching placebo IM monthly. Supportive endpoints included changes from baseline at subsequent time points in Clinical Global Impression-Severity (CGI-S) scale score; Positive and Negative Syndrome Scale (PANSS) Total score; PANSS Positive, Negative, and General Psychopathology subscale scores; PANSS Marder factors (post hoc); and PANSS responder rate. Overall response rate, based on PANSS Total score and Clinical Global Impression–Improvement (CGI-I) scale score, was also analyzed.
Of 622 patients who were randomized, 596 had ≥1 post-baseline PANSS score. Patients were markedly ill at baseline (mean PANSS Total scores 92–94). Compared with placebo, CGI-S scores; PANSS Positive, Negative, and General Psychopathology subscale scores; and PANSS Marder factors were all significantly (p<0.001) improved by Day 85 with both AL doses, with significantly lower scores starting from Day 8 in most instances. Treatment response rates were significantly (p<0.001) greater with both doses of AL vs placebo.
AL demonstrated robust efficacy on CGI-S score, PANSS subscale scores, PANSS Marder factors, and response rates. Study limitations included use of a fixed dose for initial oral aripiprazole and fixed monthly AL doses without the option to individualize the oral initiation dosing or injection frequency for efficacy, tolerability, or safety.
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