Eleanor M. Perfetto

Patient-Focused Drug Development: A New Direction for Collaboration.

Context:Patient-Focused Drug Development (PFDD) is a new initiative from the Food and Drug Administration (FDA) intended to bring patient perspectives into an earlier stage of product development. The goal is that patients will be able to provide context for benefit-risk assessments and input to review divisions, and also aid in the development of new assessment tools, study endpoints, and risk communications. This paper provides a summary on what is known to date about FDA’s PFDD initiative and describes implications for patients, researchers, payers, and the biopharmaceutical industry. It also provides a roadmap for stakeholders to consider in defining their role in and in shaping PFDD’s direction, and for expanding PFDD principles to conditions beyond the current 20 under FDA consideration. Methods:A search was conducted of the peer-reviewed and gray literature using PubMed and Google. This included laws, FDA guidance documents, the peer-reviewed literature, and FDA presentations for content relevant to the search term “patient-focused drug development.” Findings:Currently, FDA activities within PFDD are limited to gaining patient insights through 20 disease-specific meetings. However, many stakeholders see the initiative much more generally as representing a broad shift toward patient centeredness in biopharmaceutical product development. Conclusions:Depending upon the trajectory taken and whether or not all PFDD aims are eventually addressed, the initiative has the potential to change product development in fundamental ways. Further research should explore how patient input on disease manifestation and treatment options is best ascertained from patients and documented before initiating and during drug development.

What Are Outcomes

Understanding the important aspects of measuring outcomes will prepare pharmacists for changes in the marketplace and help them to assess the results of studies that will appear in subsequent issues of the Journal of the American Pharmaceutical Association and the research journals of other health services professions. In any evaluation of quality, all three quality measures (structure, process, and outcomes) must be considered. No one measure, in isolation, can describe the quality of care provided. Thus, the best patient care will come from health care practitioners who document structure, process, and outcomes and who evaluate these measures to provide appropriate care. The pharmacist cannot dispense prescriptions or provide pharmaceutical care without a pharmacy, drug inventory, or patient profiles, all of which are important structural inputs. The pharmacist takes a medication history, monitors the drug regimen, and counsels the patient on the appropriate way to use the treatment, all vital process activities. The objective of these inputs and actions is to improve the health status of the patient. Outcomes, therefore, are the intended endpoints of care, and occasional unintended effects (e.g., adverse drug reactions) as well. The future of pharmacy rests on demonstrating the positive effect of pharmaceutical care on patient outcomes. Pharmacists have always been part of the system of checks and balances in health care delivery. As the provision of health services changes, opportunities to assess the process and structure of care and to document patient outcomes will increase. Pharmacists are well positioned to intervene in patient care, but if they do not demonstrate the value of pharmacy services, they will face competition from alternative providers. Some have even suggested less expensive alternatives to having pharmacists dispense medications. Pharmacists must establish that their role goes beyond dispensing to include cognitive services such as compliance programs, screening services, glucose monitoring, and disease management programs. Pharmacists must document their role and its effects on patient outcomes.

Moxifloxacin versus levofloxacin for treatment of acute rhinosinusitis: a retrospective database analysis of treatment duration, outcomes, and charges

ABSTRACT Objective: Antibiotics are clinically indicated for acute bacterial rhinosinusitis, but they may be prescribed inappropriately. This retrospective study examined how labeled recommendations for duration of moxifloxacin and levofloxacin treatment of acute bacterial rhinosinusitis compare with real-world practice, and compared the failure and recurrence rates, and associated charges. Methods and main outcome measures: The PharMetrics Patient-Centric claims database was searched over a 3‐year period for episodes of acute rhinosinusitis treated within 5 days with moxifloxacin or levofloxacin. The duration of antibiotic treatment prescribed was compared with the labeled recommendation. Failure rates (a second antibiotic claim to treat acute rhinosinusitis within 30 days of the first claim), recurrence rates (subsequent antibiotic claims to treat any rhinosinusitis more than 30 days after the original or second [in the case of failure] claim), and treatment charges from the perspective of the payer (health insurer) were also compared using multivariate analysis. Results: The initial duration prescribed of moxifloxacin was shorter than for levofloxacin (–1.65 days, p < 0.0001), reflecting the shorter labeled recommendation (10 days versus 10–14 days). The durations of monotherapy (–2.06 days, p < 0.0001) and of all antibiotic treatment (–1.97 days, p < 0.0001) were also significantly shorter for episodes treated initially with moxifloxacin. The odds ratio for treatment failure (0.718; 95% confidence interval = 0.598–0.863; p = 0.0004) and the hazard ratio for recurrence (0.652; p = 0.0005) were both significantly lower for moxifloxacin than for levofloxacin, and resulted in lower total treatment charges (–

What Are Outcomes?: The authors define outcomes and outcomes research and discuss why pharmacists should care about them.

37.94 ± 13.65; p = 0.0055). Conclusion: The shorter treatment durations seen for moxifloxacin in this database of real-world care reflect the label-recommended duration for acute rhinosinusitis. Despite this shorter duration of therapy, moxifloxacin resulted in better outcomes than levofloxacin in terms of the risk of treatment failure and recurrence. In addition, the total charges were lower for patients treated with moxifloxacin.

Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report

Understanding the important aspects of measuring outcomes will prepare pharmacists for changes in the marketplace and help them to assess the results of studies that will appear in subsequent issues of the Journal of the American Pharmaceutical Association and the research journals of other health services professions. In any evaluation of quality, all three quality measures (structure, process, and outcomes) must be considered. No one measure, in isolation, can describe the quality of care provided. Thus, the best patient care will come from health care practitioners who document structure, process, and outcomes and who evaluate these measures to provide appropriate care. The pharmacist cannot dispense prescriptions or provide pharmaceutical care without a pharmacy, drug inventory, or patient profiles, all of which are important structural inputs. The pharmacist takes a medication history, monitors the drug regimen, and counsels the patient on the appropriate way to use the treatment, all vital process activities. The objective of these inputs and actions is to improve the health status of the patient. Outcomes, therefore, are the intended endpoints of care, and occasional unintended effects (e.g., adverse drug reactions) as well. The future of pharmacy rests on demonstrating the positive effect of pharmaceutical care on patient outcomes. Pharmacists have always been part of the system of checks and balances in health care delivery. As the provision of health services changes, opportunities to assess the process and structure of care and to document patient outcomes will increase. Pharmacists are well positioned to intervene in patient care, but if they do not demonstrate the value of pharmacy services, they will face competition from alternative providers. Some have even suggested less expensive alternatives to having pharmacists dispense medications. Pharmacists must establish that their role goes beyond dispensing to include cognitive services such as compliance programs, screening services, glucose monitoring, and disease management programs. Pharmacists must document their role and its effects on patient outcomes.

Value to Whom? The Patient Voice in the Value Discussion

BACKGROUND Rare diseases (RDs) affect a small number of people within a population. About 5000 to 8000 distinct RDs have been identified, with an estimated 6% to 8% of people worldwide suffering from an RD. Approximately 75% of RDs affect children. Frequently, these conditions are heterogeneous; many are progressive. Regulatory incentives have increased orphan drug designations and approvals. OBJECTIVE To develop emerging good practices for RD outcomes research addressing the challenges inherent in identifying, selecting, developing, adapting, and implementing patient-reported outcome (PRO) and observer-reported outcome (ObsRO) assessments for use in RD clinical trials. GOOD PRACTICES FOR OUTCOMES RESEARCH This report outlines the challenges and potential solutions in determining clinical outcomes for RD trials. It follows the US Food and Drug Administration Roadmap to Patient-Focused Outcome Measurement in Clinical Trials. The Roadmap consists of three columns: 1) Understanding the Disease or Condition, 2) Conceptualizing Treatment Benefit, and 3) Selecting/Developing the Outcome Measure. Challenges in column 1 include factors such as incomplete natural history data and heterogeneity of disease presentation and patient experience. Solutions include using several information sources, for example, clinical experts and patient advocacy groups, to construct the conditions natural history and understand treatment patterns. Challenges in column 2 include understanding and measuring treatment benefit from the patients perspective, especially given challenges in defining the context of use such as variations in age or disease severity/progression. Solutions include focusing on common symptoms across patient subgroups, identifying short-term outcomes, and using multiple types of COA instruments to measure the same constructs. Challenges in column 3 center around the small patient population and heterogeneity of the condition or study sample. Few disease-specific instruments for RDs exist. Strategies include adapting existing instruments developed for a similar condition or that contain symptoms of importance to the RD patient population, or using a generic instrument validated for the context of use. CONCLUSIONS This report provides state-of-the-art solutions to patient-reported outcome (PRO) and observer-reported outcome (ObsRO) assessments challenges in clinical trials of patients with RDs. These recommended solutions are both pragmatic and creative and posed with clear recognition of the global regulatory context used in RD clinical development programs.

Economic burden of uncomplicated urinary tract infections: direct, indirect and intangible costs.

BACKGROUND Professional societies and other organizations have recently taken a visible role trying to define treatment value via value frameworks and assessments, providing payer or provider recommendations, and potentially impacting patient access. Patient perspectives routinely differ from those of other stakeholders. Yet, it is not always apparent that patients were engaged in value framework development or assessment. OBJECTIVES To describe the development and content of the National Health Councils (NHCs) Rubric, a tool that includes criteria for evaluation of value frameworks specifically with regard to patient-centeredness and meaningful patient engagement. METHODS The NHC held a multistakeholder, invitational roundtable in Washington, DC, in 2016. Participants reviewed existing patient-engagement rubrics, discussed experiences with value frameworks, debated and thematically grouped hallmark patient-centeredness characteristics, and developed illustrative examples of the characteristics. These materials were organized into the rubric, and subsequently vetted via multistakeholder peer review. RESULTS The resulting rubric describes six domains of patient-centered value frameworks: partnership, transparency, inclusiveness, diversity, outcomes, and data sources. Each domain includes specific examples illustrating how patient engagement and patient-centeredness can be operationalized in value framework processes. CONCLUSIONS The NHC multistakeholder roundtables recommendations are captured in the NHCs Rubric to assess value framework and model patient-centeredness and patient engagement. The Rubric is a tool that will be refined over time on the basis of feedback from patient, patient group, framework developer, and other stakeholder-use experiences.

Adherence to Antipsychotic Therapy: Association With Hospitalization and Medicare Spending Among Part D Enrollees With Schizophrenia

A better understanding of how antimicrobial therapies affect the total cost of lower uncomplicated urinary tract infection, including direct (e.g., doctor visits), indirect (e.g., productivity) and intangible (e.g., pain) costs would facilitate selection of an optimal therapeutic approach. The results of this literature review indicate that the there is a considerable societal burden from uncomplicated urinary tract infection, with approximately US

Increasing uptake of comparative effectiveness and patient-centered outcomes research among stakeholders: Insights from conference discussion

1 billion in indirect and over

Economic and patient-reported outcomes of oral triptans in the treatment of migraine

600 million in direct costs in 1995. However, no single identified study incorporated all three cost components, there are gaps in the knowledge concerning the current extent of these costs, and there are no comparative assessments based on total cost. Research is needed to provide current insights on the burden of uncomplicated urinary tract infection in terms of direct, indirect and intangible costs.