RTI Health Solutions Research Articles

1386. Current Estimates of the Impact of Routine Childhood Immunizations in Reducing Vaccine-Preventable Diseases in the United States

BackgroundRoutine immunizations for children aged 10 years and younger in the United States (US) currently cover 14 diseases. Updated estimates of public health impact are needed, given changes in disease epidemiology, evolving recommendations, and the dynamic nature of compliance with the immunization schedule.MethodsPre-vaccine disease incidence was estimated before each routine vaccine was recommended, with average values across multiple years obtained directly from published literature or calculated based on disease surveillance data or annual case estimates from the published literature. Pre-vaccine incidence then was compared to current, post-vaccine incidence, which was generally calculated as average values over the most recent 5 years of available incidence data. Overall incidence estimates and estimates by age group were calculated. Differences in pre- and post-vaccine disease incidence rates were used to calculate the annual number of cases averted, based on 2019 US population estimates. This analysis did not separately estimate the proportion of disease incidence reduction that may be attributed to adult vaccines or booster doses.ResultsPost-vaccine disease incidence decreased overall and for all age groups across all diseases evaluated (Table 1). Decreases ranged from 17.4% for influenza to 100.0% for polio (Figure 1). Over 90% reduction in incidence was achieved for 10 of the 14 diseases evaluated (including reduction in incidence of rotavirus hospitalizations). Overall post-vaccine disease incidence estimates were highest for influenza, rotavirus, and varicella. Estimated annual cases averted by vaccination in 2019 ranged from 1,269 for tetanus to more than 4.2 million for varicella.Table 1. Pre- and Post-Vaccine Disease Incidence Estimates, Annual Cases, and 2019 Cases Averted, by Disease Figure 1. Percentage Reduction in Disease Incidence Post-Vaccine, by Disease ConclusionRoutine childhood immunization in the US continues to result in high, sustained reduction in disease across all vaccines and for all age groups evaluated.DisclosuresElizabeth M. La, PhD, RTI Health Solutions (Employee) Justin Carrico, BS, GlaxoSmithKline (Consultant) Sandra E. Talbird, MSPH, RTI Health Solutions (Employee) Ya-Ting Chen, PhD, Merck & Co., Inc. (Employee, Shareholder) Mawuli K. Nyaku, DrPh, Merck & Co. Inc. (Employee, Shareholder) Cristina Carias, PhD, Merck (Employee, Shareholder) Gary S. Marshall, MD, GlaxoSmithKline (Consultant, Scientific Research Study Investigator)Merck (Consultant, Scientific Research Study Investigator)Pfizer (Consultant, Scientific Research Study Investigator)Sanofi Pasteur (Consultant, Grant/Research Support, Scientific Research Study Investigator, Honorarium for conference lecture)Seqirus (Consultant, Scientific Research Study Investigator) Craig S. Roberts, PharmD, MPA, MBA, Merck & Co., Inc (Employee, Shareholder)

996. The Potential for Reducing Opioid and Analgesic Prescriptions Via Herpes Zoster Vaccination

BackgroundHerpes zoster (HZ), or shingles, is a common neurocutaneous disease caused by the reactivation of latent varicella zoster virus that often includes rash and neuropathic pain that may last for months. Opioids and other analgesics may be prescribed. Recombinant zoster vaccine (RZV) is preferentially recommended for the prevention of HZ in adults aged 50 years and older. This study aimed to assess the impact of RZV vaccination on opioid and other analgesic prescription-related outcomes.MethodsEstimates of analgesic prescription rates (opioids, benzodiazepines, and other analgesics) among HZ cases were established using Truven claims data from 2012-2018 for adults aged 50 years and older. HZ case avoidance with RZV vaccination was calculated using a previously published cost-effectiveness model. This data was included in a calculator assessing the impact of RZV vaccination on analgesic prescription-related outcomes (compared to no vaccination).ResultsBetween 24.4% and 28.0% of HZ cases in the observed claims had at least one opioid prescription, dependent on age group (4.5%-6.5% and 8.6%-19.6% for benzodiazepines and other analgesics, respectively). The mean number of opioid prescriptions per person in each age group with at least one opioid prescription was between 1.7 and 1.9 (1.7-2.3 and 1.7-2.0 prescriptions for benzodiazepines and other analgesics, respectively). Assuming a 1-million-person population and 65% RZV coverage, the calculator predicts RZV vaccination will prevent 75,002 cases of HZ and will prevent 19,311 people from being prescribed at least 1 HZ-related opioid, 4,502 people from being prescribed benzodiazepines, and 12,201 people from being prescribed other analgesics. Additionally, 34,520 HZ-related opioid prescriptions will be avoided (9,413 benzodiazepine prescriptions; 22,406 other analgesic prescriptions).ConclusionHZ is associated with high levels of opioid, benzodiazepine, and other analgesic use. Primary prevention of HZ by vaccination could potentially reduce opioid and other medication exposure.DisclosuresJean-Etienne Poirrier, PhD, MBA, The GSK group of companies (Employee, Shareholder) Justin Carrico, BS, GlaxoSmithKline (Consultant) Jessica K. DeMartino, PhD, The GlaxoSmithKline group of companies (Employee, Shareholder) Katherine A. Hicks, MS, BSPH, GlaxoSmithKline (Scientific Research Study Investigator, GSK pays my company for my contractual services.) Saurabh P. Nagar, MS, RTI Health Solutions (Employee) Juliana Meyers, MA, GlaxoSmithKline (Other Financial or Material Support, This study was funded by GlaxoSmithKline.)

Paroxysmal nocturnal hemoglobinuria: role of the complement system, pathogenesis, and pathophysiology.

The complement system is part of the innate immune response system, which comprises more than 50 distinct plasma and serum proteins that interact to opsonize pathogens (i.e., mark pathogens for destruction) and induce inflammatory responses to fight infection. The role of the complement system is 2-fold: immune surveillance and host defense. Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, acquired, hematologic disease caused by somatic mutations in the gene PIGA in the hematopoietic stem cells. These stem cells produce abnormal clone blood cells that lack the complement regulatory proteins CD55 and CD59, causing the body to recognize these otherwise healthy red blood cells as damaged. The complement system destroys cells without these protective proteins, resulting in general hemolysis. PNH is characterized by fatigue; hemolytic anemia that can be severe and debilitating; increased lactic dehydrogenase level, reticulocyte count, and bilirubin level; propensity for thrombotic events; and renal dysfunction. Epidemiologic data, while sparse, suggest that an estimated 5,000-6,000 individuals in the United States are affected by PNH. If left untreated, PNH has a 10-year mortality rate of 29%, although the natural history of this disease has been recently altered by the introduction of complement inhibitors for the treatment of PNH. DISCLOSURES: This research was developed under a research contract between RTI Health Solutions and Apellis Pharmaceuticals and was funded by Apellis Pharmaceuticals. Bektas, Copley-Merriman, and Khan are employees of RTI Health Solutions. Sarda is an employee of Apellis Pharmaceuticals. Shammo consults for Apellis Pharmaceuticals.

Paroxysmal nocturnal hemoglobinuria: current treatments and unmet needs.

The current standard of care for paroxysmal nocturnal hemoglobinuria (PNH) are the C5 inhibitors eculizumab and ravulizumab, both monoclonal antibodies designed to target the complement protein C5, thereby preventing its cleavage and the formation of the terminal attack complex. C5 inhibitors have yielded substantial improvements in the treatment of PNH and changed the mortality and morbidity, as well as health-related quality of life of patients with the disease. These treatments target underlying intravascular hemolysis; however, they do not address extravascular hemolysis, resulting in incomplete response and remaining symptoms in some patients. Therefore, despite treatment with a C5 inhibitor, some patients still experience anemia with associated fatigue, transfusion needs, and impaired health-related quality of life. DISCLOSURES: This research was developed under a research contract between RTI Health Solutions and Apellis Pharmaceuticals and was funded by Apellis Pharmaceuticals. Bektas, Copley-Merriman, and Khan are employees of RTI Health Solutions. Sarda is an employee of Apellis Pharmaceuticals. Shammo consults for Apellis Pharmaceuticals.

Paroxysmal nocturnal hemoglobinuria: patient journey and burden of disease.

Patients with paroxysmal nocturnal hemoglobinuria (PNH) often experience a lengthy path to diagnosis. Fewer than 40% of patients with PNH receive a diagnosis within 12 months of symptom onset, and 24% of all PNH diagnoses can take 5 years or longer. Diagnostic delay is a source of distress and can affect emotional well-being for patients with PNH. In PNH disease management, patients and care providers focus on risk of organ failure and mortality related to disease progression; nonetheless, patients' health-related quality of life (HRQOL) is largely affected by extensive treatment requirements and nonfatal complications of disease, such as fatigue. In particular, thrombosis is associated with significant impairments in physical and social functioning and global health status and significant fatigue. Among patients with anemia who are transfusion dependent, the burden of transfusion is considerable. Transfusion dependence has a negative effect on HRQOL; is associated with risks and complications, including iron overload; and results in lost productivity due to travel times to and time spent at infusion centers. DISCLOSURES: This research was developed under a research contract between RTI Health Solutions and Apellis Pharmaceuticals and was funded by Apellis Pharmaceuticals. Bektas, Copley-Merriman, and Khan are employees of RTI Health Solutions. Sarda is an employee of Apellis Pharmaceuticals. Shammo consults for Apellis Pharmaceuticals.

Real-World Clinical Outcomes of Patients with Myelofibrosis Treated with Ruxolitinib: Evidence from a Multinational Medical Record Review

Real-World Clinical Outcomes of Patients with Myelofibrosis Treated with Ruxolitinib: Evidence from a Multinational Medical Record Review

Patient-Reported Disease Burden: In-Depth Interviews of Patients with CAD

Introduction Cold agglutinin disease (CAD) is a rare disorder (a prevalence of 16 cases per 1 million people), accounting for approximately 15%-25% of all autoimmune hemolytic anemias (Berentsen. Semin Hematol. 2018). Currently, there is no approved drug for the treatment of CAD. Debilitating fatigue that greatly affects quality of life is common among patients with CAD (Roth et al. Blood. 2019). The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Scale has recently been used to quantify fatigue in patients with CAD (Roth et al. Blood. 2019). However, the appropriateness of FACIT-F in these patients has not been assessed. In-depth individual interviews of adults with CAD were conducted primarily to identify the symptoms and effects of CAD reported as important by patients and to better understand their experiences with CAD. The secondary objective was to obtain patient feedback regarding the relevance and importance of the items on the FACIT-F. Methods Participants were identified and recruited from a proprietary panel of patients with CAD. Of 1,250 panel members, approximately 600 members from the United States (US) were invited to participate in an in-depth interview. Systematic random sampling was used to select every nth panel member from each of 4 US regions to receive an email invitation. A semi-structured interview guide was used to ensure consistent systematic collection of data pertaining to study objectives, while maintaining a conversational tone and encouraging spontaneity of answers. Results Twenty-two members expressed interest in participating; of these, 18 could be reached for screening and 16 were eligible for interview. The sample was 100% White and non-Hispanic, 62.5% female, and had an average age of 67.8 years. Participants resided in the West (37.5%), Midwest and South (both 25%), and Northeast (12.5%). The date of CAD diagnosis averaged 6 years before the interview, and 68.8% of patients had received their diagnosis within the last 1.5 years. Seven (43.8%) patients reported that they were not taking any prescription or over-the-counter (OTC) medication for CAD, whereas 9 (56.3%) were taking prescribed or OTC folic acid for CAD (of these, 4 also took prescription strength vitamin B12). In this sample, no patient reported current use of other prescription medications for CAD. A few patients spontaneously reported previous treatments for CAD (eg, transfusions and rituximab or other injections). However, previous CAD treatment was not systematically collected for all subjects and is a limitation of the study. Concept-elicitation of symptoms The most frequently reported symptoms were fatigue/tiredness/lack of energy (93.8%), reaction to cold environments (93.8%), breathlessness (68.8%), and trouble thinking/concentrating (68.8%). When probed, 43.8% of patients reported sleep disturbances (Figure 1A). Concept-elicitation of effects Most participants (68.8%) reported lifestyle or behavioral changes to help treat CAD symptoms (eg, taking gloves/extra clothing to places that may be cold). Others reported giving up/limiting enjoyable or outdoor activities and making major changes (eg, discontinuing outdoor activities, restricting travel). Other CAD-related effects included impairment in daily activities, overall health/physical activities, and social/leisure activities/relationships (Figure 1B). For the evaluation of the FACIT-F in this CAD population, participants reported that FACIT-F instructions and items were clear and easy to understand, response options were distinct from each other, and they could recall their fatigue over the previous 7 days. They reported that FACIT-F measured one or more concepts personally important to them and important concepts were not missing. This provides support for the content validity of the FACIT-F in patients with CAD. Conclusions Patients with CAD experienced negative effects on daily activities, physical health/activities, social/ leisure activities/relationships, the ability to concentrate/recall, and mood/emotions mostly owing to fatigue/lack of energy and reactions to cold environments. Most patients required modifying their lifestyle/behavior to manage CAD symptoms. FACIT-F measured concepts important to patients with CAD, which supports its content validity and use in this patient population. Disclosures Su: Sanofi: Current Employment. Kosa:Sanofi: Other: RTI Health Solutions was contracted by Sanofi to conduct this work; RTI Health Solutions: Current Employment. DiBenedetti:Sanofi: Other: RTI Health Solutions was contracted by Sanofi to conduct this work; RTI Health Solutions: Current Employment, Other.

The Patient Experience of Relapsed Refractory Multiple Myeloma and Its Treatment: A Social Media Review

Background: Patients with relapsed refractory MM (RRMM) report a decline in HRQOL with disease progression and after each relapse, may experience cumulative toxicities from exposure to several lines of therapy. Social media (SM) platforms are rich databases of unfiltered, real-world evidence, where patients share their experiences about diseases and treatments. These platforms provide researchers with a novel approach to understanding the patient perspective. This study aimed to review unsolicited, publicly available patient-reported information (PRI) shared on SM platforms to gain insights into the patient experience of RRMM and its treatment, specifically CAR-T therapy. Methods: Purposeful sampling of PRI were collected from two SM sources (YouTube [video data] and a patient advocacy website Patient Power [patient blogs; https://patientpower.info]) to identify stories of patients living with RRMM. SM posts were included for review if individuals with a self-reported MM diagnosis described their experience with RRMM and provided commentary on CAR-T therapy. Video/blog data were manually reviewed and content analyzed thematically to address experiences of living with MM, and perceptions and experiences of treatment including CAR-T. Demographic information (e.g., sex and age) were extracted from SM posts along with any accompanying disease information, where data were available. Results: Of 43 posts initially identified from the SM sources, 14 were considered relevant to the study objective: 10 videos / 4 blogs connected to 19 unique patients with RRMM (9 male/10 female, patient age range, 51-72 years). One patient reported receiving CAR-T therapy (female, aged 51 years) and was the subject of 5 videos and all 4 blogs. RRMM Impacts: Key symptoms reported included: pain, fatigue, bone fractures, infections, and fever. Patient-reported HRQOL impacts included reduced mobility, impeded ability to work, negative impact on family activities, low mood, anxiety, and a loss of independence. Patients used different strategies (e.g., slower pace of life, planning, and self-advocacy) to adapt to their progressing disease. Treatment Experience: Patients reported switching from one continuous therapy to another in order to achieve remission with varying degrees of success. Positive treatment outcomes were unpredictable; patients who achieved remission were uncertain how durable the remission period would be and expected to relapse. Treatment Decision Drivers: treatment toxicity, impact on daily life, time required for in-hospital treatment, and desire to attain life milestones. Treatment Outcomes: Patients prioritized HRQOL impacts over effectiveness; benefits of treatment-free periods included ability to travel or attending key life events (e.g., family weddings). CAR-T Patient Experience: Patient reported 16 lines of therapy and two stem-cell transplants but never achieved complete remission from these prior treatments. The CAR-T procedure (received in 2018) was described as quick / preferable to previous treatments (e.g., stem cell transplants / continuous chemotherapy) despite a serious cytokine release storm. The patient experienced a rapid improvement, within 1 month of receiving CAR-T, and achieved complete remission which she described as life changing/lifesaving: remission allowed her to return to living a 'normal' life (i.e., working and exercise) and reportedly reduced the burden on family. Conclusions: This study provides valuable insights on the patient perceptions and experience of MM and its treatments using an innovative method for collecting PRI. Although remission was a key treatment outcome for patients, HRQOL and the ability to live their lives on their own terms were often prioritized over treatment effectiveness. The patient who underwent CAR-T therapy experienced a return to a sense of normality as a result of her CAR-T therapy, if for a brief period, which was life changing for both patient and family. Insights generated from this SM review can be used to inform patient-reported outcome measurement strategies for novel treatment clinical development programs. Purposeful sampling of PRI on 2 SM sources limits external validity of insights gathered. The CAR-T experience was a singular experience of one patient; additional insights are to be learned as more patients undergo CAR-T for their RRMM. Disclosures Crawford: RTI Health Solutions: Current Employment. Gries:Janssen: Current Employment, Current equity holder in publicly-traded company. Morrison:RTI Health Solutions: Current Employment. Valluri:Janssen: Current Employment. Doward:RTI Health Solutions: Current Employment. Fastenau:Janssen: Current Employment, Current equity holder in publicly-traded company.

Patient Expectations and Perceptions of Treatment in CARTITUDE-1: Phase 1b/2 Study of Ciltacabtagene Autoleucel in Relapsed/Refractory Multiple Myeloma

Patient Expectations and Perceptions of Treatment in CARTITUDE-1: Phase 1b/2 Study of Ciltacabtagene Autoleucel in Relapsed/Refractory Multiple Myeloma

Assessing Patient Experiences with Prophylactic Treatments for Hemophilia a: Concept Elicitation for Gene Therapy

Background: Gene therapy for hemophilia A is designed to be a one-time infusion to deliver functional copies of the defective factor VIII (FVIII) gene, to facilitate the endogenous production of therapeutic FVIII levels. The aim is to achieve long-term protection from bleeds without the burden of regular infusions. Aims: To better understand patients' experiences of living with hemophilia A, the impact of traditional hemophilia A treatments, and patients' perceptions of the potential value of gene therapy versus traditional prophylactic treatments. Methods: Patients were identified from the database of a US rare diseases patient organization, who also recruited and screened patients for the study using materials developed by the outcomes research organization (RTI-HS) and Takeda. Adult males aged ≥18 years with a self-reported diagnosis of moderate or severe hemophilia A, who reported using factor or nonfactor prophylactic treatment and were not currently receiving treatment for inhibitors, were eligible for the study. Eligible patients provided verbal informed consent to participate in a semi-structured, 60-minute telephone interview conducted in English by 2 members of RTI-HS who did not have access to any patient-identifying information at any time during the study. Targeted questions probed perceptions of treatment burden, impact of hemophilia A on daily life, and time spent on treatment. Additionally, questions were posed to assess patients' perceptions of the impact of traditional treatments and the potential benefits they anticipate from gene therapy. Results: Nineteen patients aged 19-55 years with moderate (n = 1) or severe (n = 18) hemophilia A were interviewed. Most (16/19, 89.5%) received prophylactic FVIII therapy, (3/19, 15.8%) were receiving nonfactor prophylactic treatment, of which 1 patient also used FVIII treatment. The aspects of current or past treatments most frequently disliked by patients were lack of efficacy, frequency of infusions, intravenous administration, vein health/scar tissue, and dosing volume. Most patients expressed satisfaction with their current treatment (18/19; 94.7%), though all listed ≥1 negative treatment impact, most frequently related to difficulties with travel (13/19; 68.4%), mood/emotions (12/19; 63.2%), day-to-day activities (10/19; 52.6%), and physical health/activities (7/19; 36.8%), including having to give up or reduce particular activities because of their treatment and needing to be more cautious, especially on nontreatment days. When presented with a hypothetical scenario for gene therapy - a one-time long-acting intravenous infusion to provide a constant level of FVIII that could reduce future bleeds - all patients stated they would choose gene therapy over their current therapy, although several said they would have initial questions regarding safety, efficacy, and duration of protection. Commonly expressed reasons for preferring gene therapy (Figure 1) included fewer infusions and less worry about the need to infuse. All 19 patients said they expected to be highly satisfied with this treatment, largely because of the long-term protection from bleeds, fewer infusions, and less concern about inhibitors. The most commonly anticipated improvements were in mood/emotions (15/19; 78.9%), specifically related to reduced concern about infusions and bleed protection. Other commonly anticipated improvements included gain in time usually spent infusing (13/19; 68.4%), easier travel (12/19; 63.2%), and improved physical health and ability to perform activities (10/19; 52.6%). Conclusions: This study identified specific patient priorities, including treatment convenience, long-lasting bleed protection, frequency of intravenous infusions, and infusion volumes. The results suggest that gene therapy clinical trials should consider evaluating patient concerns in relation to the level of patient confidence in bleed protection. A study limitation is that, at the time of the survey, data on the efficacy and safety of gene therapy were limited. In the future, the study will be expanded to include a larger population of patients with hemophilia. Disclosures Sun: Takeda Pharmaceutical Company Ltd.: Current Employment. Olayinka-Amao:Takeda Pharmaceutical Company Ltd: Other: RTI-HS was contracted by Takeda Pharmaceutical Company Ltd to conduct this work; RTI Health Solutions: Current Employment. DiBenedetti:RTI Health Solutions: Current Employment; Takeda Pharmaceutical Company Ltd: Other: RTI-HS was contracted by Takeda Pharmaceutical Company Ltd to conduct this work.

Incremental clinical and economic impact of recombinant zoster vaccination: real-world data in a budget impact model.

In 2017, the FDA approved the adjuvanted recombinant zoster vaccine (RZV) for the prevention of herpes zoster (HZ) in immunocompetent adults aged 50 years and older. RZV joined zoster vaccine live (ZVL) as U.S.-marketed vaccines against HZ. The Advisory Committee on Immunization Practices preferentially recommended use of RZV over ZVL. In order to inform population-based decision makers (PBDMs) about the incremental clinical and economic impact of RZV adoption, budget impact (BI) models may be used. Populating such models with national data can inform PBDMs about the incremental value of RZV adoption nationally; however, heterogeneity across health plans requires the inclusion of plan-specific data to ensure the relevance of modeling outcomes for plan-specific decision makers. To investigate the clinical and economic outcomes associated with the adoption of RZV in nationally representative populations with commercial and Medicare coverage and to demonstrate the effect of the heterogeneity of health plans using real-world data from a large, integrated delivery network (IDN). We used a publicly available BI model. The model accounts for national and IDN-collected population characteristics (size, age distribution) and epidemiological data (incidence of HZ and complications, HZ recurrence rate), vaccine characteristics from randomized controlled trials and observational studies (efficacy, waning, second dose compliance for RZV, adverse event rate), national costs (vaccine, direct medical for HZ, complications, and vaccine adverse events), and current and anticipated vaccine coverage. We assessed incremental clinical (HZ cases and complications) and economic (per-member-per-month [PMPM] costs) impact at 5-year to 15-year time horizons, comparing scenarios where RZV is solely implemented with one where only ZVL is utilized. Following the adoption of RZV, the incremental HZ cases avoided over 5 and 15 years were estimated to be 1,800 and 15,000 for a commercial plan, 3,800 and 21,000 for a Medicare plan, and 8,600 and 71,000 for a specific IDN. The incremental PMPM budget impact over the same time horizons was estimated to be $0.42 and $0.31, respectively, for a commercial plan, $0.35 and $0.10 for a Medicare plan, and $0.39 and $0.25 for a specific IDN. The differences in results across plans resulted from the population age distribution, the vaccine copay (applied in the Medicare scenario only), the vaccine coverage in the plan, and other plan-specific factors affecting disease epidemiology and costs per case of HZ. Model projections indicated that RZV adoption avoided HZ cases and related complications, with the PMPM budget impact dependent on plan-specific factors. As health gains increased over time, the incremental costs incurred were found to decrease as the shorter-term costs of adopting the new vaccine were increasingly offset by the longer-term benefits of vaccination. GlaxoSmithKline Biologicals SA funded this study (GSK study identifier: HO-17-18378) and was involved in all stages of study conduct, including analysis of the data. GlaxoSmithKline Biologicals SA also paid all costs associated with the development and publication of this manuscript. Patterson, Van Oorschot, and Curran are employees of the GSK group of companies and hold shares in the GSK group of companies. Herring, Carrico, and Zhang are employees of RTI Health Solutions, which received funding via a contractual agreement with the GSK group of companies to perform the work contributing to this research. Ackerson, Bruxvoort, Sy, and Tseng are employees of Kaiser Permanente Southern California, which was contracted by the GSK group of companies for the conduct of this study and were members of the KPSC study team. Ackerson, Bruxvoort, Sy, and Tseng report research contracts with the following pharmaceutical companies unrelated to this study: Dynavax (Ackerson, Bruxvoort, and Sy); the GSK group of companies (Ackerson, Bruxvoort, Sy, and Tseng); Novavax (Ackerson, Sy, and Tseng); and Seqirus (Ackerson, Bruxvoort, Sy, and Tseng). Tseng reports having served as a paid consultant for the GSK group of companies. The authors declare no other financial and nonfinancial relationships and activities. Findings from this study were presented at AMCP Nexus 2019; October 29-November 1, 2019; National Harbor, MD.

Understanding Payer Perspectives on Value in the Use of Pharmaceuticals in the United States.

In recent years, value assessment frameworks have been introduced to inform discussions about how to define and assess value in the U.S. health care system. However, there is uncertainty as to how value assessment frameworks and other approaches to achieve value such as outcomes-based contracting are perceived and used in coverage decisions. To understand how U.S. payers determine value in the use of pharmaceuticals and how it differs from payers outside the United States. Qualitative in-depth phone interviews with 13 executive-level public and private U.S. managed care representatives and 6 health technology assessment advisors outside the United States were conducted from September to November 2017. Despite various mechanisms used by U.S. payers to assess value, no consistent definitions of value were provided, and U.S. payers felt limited in what they can do to achieve value in pharmaceutical decision making. Value assessment frameworks are not formally considered in formulary and reimbursement decisions but are used as a reference as they become available by most or all U.S. health plans. U.S. payers expressed concerns, including limited control over pharmaceutical pricing and budget caps, and limited ability to use incremental cost per quality-adjusted life-year thresholds. Outcomes-based contracting could have some utility in specific cases where the treatment has a particularly high cost and a clear outcomes measure, but payers indicated that outcomes-based contracts can be difficult to operationalize, and determination of savings was uncertain. Payers outside the United States-who are enabled by government health care bodies, policy tools, and analytical frameworks that have no counterpart in the United States-have a wider array of instruments at their disposal. U.S. payers were largely open to learning from other health care systems outside the United States, particularly the German health care system, where patient-relevant benefit compared with a predetermined treatment comparator is the primary determinant for price negotiations. Although there is interest in including value assessment frameworks during the decision-making process in the United States, there are significant challenges to operationalizing them. The current environment in the United States restricts payers' ability to make favorable contracts with manufacturers, and changes to the U.S. health system design are needed to facilitate this effort. Adoption of a value assessment framework in Medicare or Medicaid would accelerate adoption of these tools by private payers in the United States. This study was conducted by RTI Health Solutions under the direction of The Pew Charitable Trusts and was funded by The Pew Charitable Trusts. Vekaria is employed by RTI Health Solutions. Reynolds and Coukell are employed by The Pew Charitable Trusts. Brogan and Hogue have nothing to disclose.

Overall Survival, Adverse Events, and Economic Burden in Medicare Patients with Chronic Lymphocytic Leukemia Receiving Cancer-Directed Therapy

Background: Data on overall survival (OS) and adverse events (AEs) in patients with chronic lymphocytic leukemia (CLL) are mostly available from controlled trials, with limited data from routine clinical practice. We therefore conducted a population-based retrospective cohort study to assess OS, incidence of AEs, and economic burden in patients treated for CLL. Methods: Patients with CLL receiving ≥ 1 systemic therapy from 2013-2015 were selected from the Medicare claims database and followed through 2016. The date of the start of first observed therapy served as the index date. Patients were required to have at least 12 months of continuous Medicare enrollment with no evidence of systemic therapy for CLL and/or SCT before the index date. An observed therapy regimen was defined as the combination of all agents received within 35 days after (and including) the first claim for a systemic therapy drug. Therapy was considered ended upon switch to a different regimen or a gap ≥ 90 days after the last treatment. OS was assessed from the index date until the last follow-up or death using the Kaplan-Meier method. Incidence of hematologic and nonhematologic AEs during treatments were assessed for the most commonly observed regimens. The incidence of AEs was based on the presence of at least one claim containing an AE-specific diagnosis code during the treatment, regardless of any history of the AE before treatment initiation. All-cause direct medical costs were assessed from the Medicare's perspective. Results: We analyzed 7965 patients (median age=76 years) who met the inclusion criteria. In the overall study follow-up (across all observed therapy lines), ibrutinib monotherapy (Ibr; n=2708) was the most frequent regimen, followed by chlorambucil monotherapy (Clb; n=1620) and bendamustine/rituximab (BR; n=1485). Median length of follow-up from the index date was 19 months for Ibr, 21 months for Clb, and 24 months for BR. Median OS was reached only for Clb (40.8 months [95% CI = 38.6-not reached]). 24-month OS rates for Ibr, Clb, and BR recipients were 69% (95% CI = 68%-71%), 68% (95% CI = 65%-71%), and 79% (95% CI = 77%-81%), respectively. The incidence of the most frequent AEs (occurrence in >10% of patients) are presented in Table1 1; estimates in bold indicate that the incidence of the AE was higher by ≥ 5 percentage points than in the noted trials (Woyach, 2018, N Engl J Med; Burger, 2015, N Engl J Med). The mean per patient per month costs, among all patients, were $1,915 (SD = $2,453) during the baseline period and $8,974 (SD = $11,562) during the period after initiation of the first observed CLL-directed systemic therapy. Mean monthly all-cause costs increased by the number of AEs (from $5,144 [SD = $5,409] among those with 1-2 AEs to $10,077 [SD = $12,542] among those with ≥6 AEs). Conclusion: To our knowledge, this is the largest contemporary observational study reporting outcomes among CLL patients initiating treatments in a real-world setting. Over two-thirds of patients survived ≥2 years after start of the first observed therapy during the study period. Incidence for several hematologic and nonhematologic AEs during the common CLL therapies observed in this study appear to be higher than those reported in the noted clinical trials, highlighting potentially greater susceptibility to these AEs and an unmet medical need in Medicare patients with CLL treated in routine practice. This study also highlights a substantial economic burden that exists in the period after initiation of treatment for CLL. Disclosures Goyal: RTI Health Solutions: Employment. Nagar:RTI Health Solutions: Employment. Kabadi:AstraZeneca: Employment, Equity Ownership. Davis:RTI Health Solutions: Employment. Le:AstraZeneca: Employment, Other: Stocks. Kaye:RTI Health Solutions: Employment.

Overall Survival, Adverse Events, and Economic Burden in Medicare-Insured Patients with Mantle Cell Lymphoma Receiving Cancer-Directed Therapy

Background: Most data on overall survival (OS) and adverse events (AEs) in patients with mantle cell lymphoma (MCL) are from controlled trials in academic centers; data from real world management and outcomes in patients with MCL are sparse. We therefore conducted a population-based retrospective cohort study of patients with MCL in the Medicare database to assess treatment patterns, OS, AEs, and economic burden. Methods: Patients with MCL who received any systemic cancer-directed treatment from 2013 to 2015 were selected from the nationwide Medicare claims database and followed through 2016. The date of the first observed systemic therapy defined each patient's index date. Patients were included if they (a) were ≥ 18 years of age at the index date; (b) had ≥ 12 months of continuous Medicare enrollment before the index date (baseline period); and (c) had no evidence of prior MCL-directed treatment (systemic therapy and/or SCT) at any time before the index date (i.e., during at least the previous 12 months). An observed line of therapy was defined as all agents received on or within 35 days after the first claim for a systemic therapy drug; the observed therapy line was considered ended upon switch to another regimen or a gap ≥ 90 days after the last treatment. OS was estimated by the Kaplan-Meier method from the index date (start of first observed line of therapy) until the last follow-up or death. We also calculated rates of occurrence for hematologic and nonhematologic AEs often associated with the most commonly observed regimens (irrespective of observed line of therapy). The occurrence of AEs was defined based on the presence of at least one claim containing an AE-specific diagnosis code during the treatment, regardless of any history of the AE before treatment initiation. All-cause health care costs were assessed from Medicare's perspective. Multivariable models were fitted to assess the association between number of AEs and average costs during the first observed therapy. Results: We analyzed 1,465 patients who met the inclusion criteria (median age=74 years; 68% male; 93% white). Across all observed lines of therapy, ibrutinib monotherapy (Ibr) (n=588 [40%]) was the most frequently used regimen, followed by bendamustine/rituximab (BR) (n=527 [36%]). Ibr recipients had a median age of 75 years, median Charlson Comorbidity Index (CCI) score of 4.0, and were followed for a median duration of 15 months; 52% died during the study period. BR recipients had a median age of 75 years, median CCI score of 3.0, and were followed for a median duration of 21 months; 28% died during the study period. In Ibr recipients, median OS was 22 months (95% CI = 16.9-28.6) and 24-month OS was 47% (95% CI = 42.9%-50.5%). In BR recipients, median OS was not reached while OS at 24 months was 73% (95% CI = 69.4%-76.0%). The occurrence of common AEs during Ibr and BR therapies are presented in Tables 1 and 2. The average per patient per month costs, among all patients, were $2,501 (SD = $2,818) during the baseline period and $12,604 (SD = $14,437) during the period after initiation of the first observed MCL-directed systemic therapy. Multivariable analysis showed that the patients with 3 or more AEs had nearly 4 times higher monthly per patient costs (cost ratio = 4.12, 95% CI = 3.53-4.82) compared with those with 0-2 AEs. Conclusions: Two-year survival rates observed in this study are comparable to those reported in clinical trials (47% for Ibr in the relapsed disease setting [Wang, 2015, Blood]) and nearly 75% for BR in patients with relapsed indolent disease and MCL [Rummel, 2016, Lancet]). Rates of AE occurrence in Ibr- and BR-treated patients in this study highlight the substantial burden and susceptibility to AEs among Medicare patients in the real-world setting. These findings also demonstrate a substantial increase in the economic burden from the baseline period to the period after MCL treatment initiation and as the number of AEs increased. Disclosures Kabadi: AstraZeneca: Employment, Equity Ownership. Goyal:RTI Health Solutions: Employment. Nagar:RTI Health Solutions: Employment. Davis:RTI Health Solutions: Employment. Le:AstraZeneca: Employment, Other: Stocks. Wang:Dava Oncology: Honoraria; Guidepoint Global: Consultancy; BioInvent: Consultancy, Research Funding; VelosBio: Research Funding; Loxo Oncology: Research Funding; Celgene: Honoraria, Research Funding; Juno Therapeutics: Research Funding; Aviara: Research Funding; Kite Pharma: Consultancy, Research Funding; Janssen: Consultancy, Honoraria, Research Funding, Speakers Bureau; Pharmacyclics: Honoraria, Research Funding; AstraZeneca: Consultancy, Honoraria, Research Funding, Speakers Bureau; MoreHealth: Consultancy, Equity Ownership; Acerta Pharma: Consultancy, Research Funding. Kaye:RTI Health Solutions: Employment.

Patient-Reported Disease Burden and Age: Results from a National Patient Advocacy Survey of Patients with Chronic Lymphocytic Leukemia, Diffuse Large B-Cell Lymphoma and Follicular Lymphoma

Introduction: Evaluation of patients' psychosocial burden related to their cancer and its treatment is important for shared decision making by patients and their healthcare team. We aimed to better understand the impact of age on this burden as reported by patients with chronic lymphocytic leukemia (CLL), diffuse large B-cell lymphoma (DLBCL), and follicular lymphoma (FL) using data from a large US patient advocacy survey. Methods: We developed a survey to understand patients' perceptions of disease burden and impact on physical and emotional health. The survey was designed through consultation with medical experts, patient advocacy organizations and research team members. Concept elicitation and cognitive pretesting were conducted with patients to inform the survey content. The survey was administered electronically to patients with CLL, DLBCL, or FL who had received either initial or subsequent treatment within the past year. The survey consisted of categorical Likert options that quantified the impact of disease on physical function, sleep, cognition, work, emotional health and quality of life (QoL). The survey data were analyzed descriptively by patients' reported age: 60 years or older versus less than 60 years. Results: The survey was completed by 424 patients who were associated with The Leukemia & Lymphoma Society and/or the Lymphoma Research Foundation (309 patients with CLL, 59 patients with DLBCL and 69 patients with FL). Respondents had a mean age of 66 years (range: 22-95 with five patients electing not to report their age), 79% were 60 years or older, and 51% were female. A greater proportion of younger patients (<60 years) reported negative impact of the disease on various aspects of their lives as compared with older patients. Negative impact on emotional health, on personal relationships and on overall health-related QoL, as well as worry about disease returning or getting worse are highlighted in the following Table. Physical impacts of diseases were also reported in a greater proportion of younger patients versus older patients, specifically in reports of nausea, pain, appetite, sleep patterns, concentration and multi-tasking. Additionally, 95% of patients overall, regardless of age, agreed that delaying disease progression was important to them. Conclusions: Evaluation of disease burden is important as patients and healthcare providers are empowered to take more active roles in shared decision making for treatments that are aligned with patients' concerns and priorities. While patients with CLL, DLBCL, and FL report substantial disease burden, the negative impact of disease was reported in a greater proportion of younger patients (<60 years) than in older patients (≥60 years). Differences in patient-reported disease burden based on age and other patient circumstances should be explored as potential key concerns of patients for education of the clinical community. Disclosures Dawson: Roche/Genentech: Equity Ownership; Genentech: Employment. Price:RTI Health Solutions (RTI-HS): Employment. Sae-Hau:Genentech: Other: The Leukemia & Lymphoma Society received funding from Genentech to participate in this research and also receives programmatic funding from Genentech.. Weiss:Genentech: Other: The Leukemia & Lymphoma Society received funding from Genentech to participate in this research and also receives programmatic funding from Genentech.. Mange:Research Triangle Institute d/b/a RTI Health Solutions: Employment. Mansfield:RTI Health Solutions (RTI-HS): Employment. Comenencia-Ortiz:Genentech, Inc.: Employment. Masaquel:Roche: Equity Ownership; Genentech: Employment. Ravelo:Genentech: Employment, Equity Ownership.

Real-World Treatment Patterns and Adverse Events in Patients with Chronic Lymphocytic Leukemia Treated with Ibrutinib in the UK: A Preliminary Analysis

Introduction: Bruton tyrosine kinase (BTK) is a critical component of the B-cell receptor pathway, and is a validated target for the treatment of chronic lymphocytic leukemia (CLL). Ibrutinib is a first-generation, covalent, small molecule BTK inhibitor approved for the treatment of CLL. We present a preliminary analysis of treatment patterns and adverse events (AEs) in patients with CLL treated with ibrutinib in a real-world setting. Methods: A retrospective chart review is being conducted among patients diagnosed with CLL and treated with ibrutinib in oncology centers throughout the UK; the target sample size for the study is 250 patients. Patients are eligible if they initiated ibrutinib after diagnosis of CLL, between January 2017 and June 2018, with at least 12 months of follow-up data available, with the exception that patients who died less than 12 months after ibrutinib initiation remained eligible. Hematology/oncology physicians reviewed medical records and completed web-based data collection forms. Baseline medical history information and data on treatment characteristics and AEs were collected. By June 2019, a total of 151 medical records (60% of the target sample size) had been abstracted. All analyses were descriptive in nature and were performed in SAS v9.4 or later (Cary, NC, USA). Results: Twenty-two physicians from specialist cancer centers or tertiary referral treatment centers (45.5%), teaching hospitals (31.8%) and non-teaching hospitals (22.7%) submitted data on ibrutinib-treated patients. The median follow-up for this interim sample of 151 patients was 16.1 months (range: 2.8-27.5 months) from ibrutinib initiation (index date) and 61.7 months (range: 11.6-264.1 months) from initial CLL diagnosis (Table 1). Median age was 71 years, 56% were male, 22.5% of patients had del(17p) mutation and 24.5% had TP53 mutations/aberrations. Of the 151 patients, 24.5% (n=37) initiated ibrutinib as first-line therapy while 75.5% (n=114) initiated ibrutinib as second- or later-line treatment. Median time to initiation of ibrutinib was 3.8 months (range: 0.3-123.7 months) for first-line therapy after initial CLL diagnosis and 22.3 months (range: 0.2-242.2 months) for second-line therapy after end of first-line therapy. Other therapies that patients received besides ibrutinib included the combination of fludarabine, cyclophosphamide, and rituximab (first-line, 22.5%; second-line, 1.7%), bendamustine plus rituximab (first-line, 19.9%; second-line, 15.5%), and chlorambucil plus rituximab (first-line, 10.6%; second-line, 1.7%). The most common AEs observed during ibrutinib therapy were bruising (19.9%), cytopenias (18.5%), diarrhea (15.2%), and arthralgia (11.3%) (Table 2). Conclusion: This preliminary analysis describes patient characteristics and treatment patterns in ibrutinib-treated patients in the UK. We found that the majority of ibrutinib use was in the second-line or later, reflecting the current UK public reimbursement situation. AEs such as bruising and cytopenias were commonly reported in patients treated with ibrutinib, and future analyses from this study will determine how these AEs and others affect dosing, treatment discontinuation and healthcare resource utilization. Disclosures Xie: AstraZeneca: Employment. Yong:AstraZeneca: Employment, Equity Ownership. Waweru:AstraZeneca: Employment, Equity Ownership. Sorof:Acerta Pharma: Employment. Goyal:RTI Health Solutions: Employment. Davis:RTI Health Solutions: Employment. Follows:Roche: Consultancy, Honoraria, Speakers Bureau; Janssen: Consultancy, Honoraria, Speakers Bureau; Abbvie: Consultancy, Honoraria, Speakers Bureau; AstraZeneca: Consultancy, Honoraria, Speakers Bureau. Hillmen:Apellis: Research Funding; Gilead: Research Funding; Roche: Research Funding; AbbVie: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Travel Expenses, Research Funding; Janssen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Travel Expenses, Research Funding; Pharmacyclics LLC, an AbbVie Company: Consultancy, Honoraria, Research Funding; Acerta: Membership on an entity's Board of Directors or advisory committees.

Treatment Patterns, Adverse Events, and Economic Burden in a Privately Insured Population of Newly Diagnosed Patients with Chronic Lymphocytic Leukemia in the United States

Introduction: Contemporary data describing treatment patterns, adverse events (AEs) and outcomes in CLL patients in clinical practice is lacking. We conducted a retrospective cohort study and assessed treatment patterns, AEs, health care resource use (HCRU), and costs in patients with newly diagnosed CLL.Methods: Using a nationally representative population of privately insured patients in the US, adult patients with CLL were selected if they had continuous health plan enrollment for ≥12 months before the first CLL diagnosis without any evidence of any CLL-directed treatment. Treatment patterns up to 4 lines of therapy (LOT) and occurrence of AEs during CLL therapies were assessed. Mean per-patient monthly HCRU and costs were assessed overall and by number of unique AEs.Results: Of all patients meeting the selection criteria (n=7,639; median age, 66 years), 18% (n=1,379) received a systemic therapy during study follow-up. The most common systemic therapy regimens, regardless of therapy line, were bendamustine/rituximab (BR) (32%), rituximab monotherapy (24% [including maintenance]), ibrutinib monotherapy (15%), and fludarabine/cyclophosphamide/ rituximab (FCR) (14%). Of these, BR was the most common LOT-1 regimen (28.1%), while ibrutinib was the most common regimen in LOT-2 (20.8%) and in LOT-3 (25.5%). Use of idelalisib was limited to 1.6% of all patients receiving systemic therapy; however, an increasing trend was observed as patients moved from first to fourth LOT (<1% in LOT-1, 3.1% in LOT-2, 4.7% in LOT-3, and 8.6% in LOT-4). AEs identified during the most common treatments for CLL are presented by treatment regimen in Table 1. The mean monthly all-cause and CLL-related costs, among patients treated with a systemic therapy, were $7,943 (SD=$15,757) and $5,185 (SD=$9,935), respectively. Figure 1 depicts mean monthly costs by care setting and number of AEs, among all patients. Mean (SD) monthly all-cause costs during the post-index date follow-up were $905 ($1,865) among those with no AEs, $1,655 ($5,364) among those with 1-2 AEs, $2,883 ($8,483) among those with 3-5 AEs, and $6,032 ($13,290) among those with ≥6 AEs.Conclusions: This population-based study yielded recent real-world evidence on treatment patterns, AEs, HCRU, and costs in patients enrolled in health plans in the US. Immunochemotherapy, particularly BR, remained the preferred frontline therapy for CLL, whereas ibrutinib was the preferred therapy in LOT-2 and LOT-3, during the study period. This study demonstrates that the AE burden associated with current treatment alternatives for CLL is substantial, and the management of AEs occurring during treatments may have a significant impact on the overall health care costs. [Display omitted] DisclosuresKabadi:AstraZeneca: Employment. Goyal:RTI Health Solutions: Employment, Research Funding. Nagar:RTI Health Solutions: Employment, Research Funding. Kaye:RTI Health Solutions: Employment, Research Funding. Davis:RTI Health Solutions: Employment, Research Funding. Mato:Celgene: Consultancy; AstraZeneca: Consultancy; Pharmacyclics: Consultancy, Honoraria, Research Funding; Abbvie: Consultancy; Sunesis: Honoraria, Research Funding; TG Therapeutics: Research Funding; Janssen: Consultancy, Honoraria; Acerta: Research Funding; Prime Oncology: Speakers Bureau; Regeneron: Research Funding.

683. Cost Calculator for Mass Vaccination Response to a US College Campus Outbreak of Serogroup B Meningococcal Disease

BackgroundUS college students are at increased risk for serogroup B meningococcal disease (MenB). MenB caused ~57% of meningococcal disease cases among 16- to 23-year-olds in 2016, and was responsible for 10 US college outbreaks from 2011−2017 involving 41 cases and an at-risk population of ~182,000 enrolled undergraduates. Outbreaks cause disruptive anxiety among university communities and implementing a mass vaccination response imposes an often unforeseen financial burden. This study aimed to enumerate costs incurred during a points-of-dispensing, mass vaccination response to a US campus MenB outbreak.MethodsThe 2015 MenB outbreak at Providence College was used as a case study to develop an Excel-based (Microsoft, Redmond, WA) cost calculator to capture costs and resources associated with a MenB outbreak response. The calculator has user-modifiable inputs related to the vaccine-eligible population, accounts for each vaccination event and vaccine dose (Figure 1), and estimates direct costs (2016 USD) during 18 months post-outbreak. Potential/expected costs computed (assuming 100% vaccine coverage) were compared with estimated actual costs incurred during the outbreak, using a micro-costing approach.ResultsThe estimated total cost for full vaccination of 4,795 eligible individuals was $1,798,399 ($375.06/person); based on actual vaccinations received, the cost calculator computed $1,350,963 in aggregate direct costs ($636.05/person fully vaccinated) (Table 1). In both analyses, medical supplies were the majority of costs (88–89%), followed by labor resources (7–9%).ConclusionThis cost calculator quantifies the direct cost of a mass vaccination response to one campus MenB outbreak. Although the cost estimates herein are higher than previously reported, the calculator does not account for follow-up costs or productivity losses and therefore underestimates the true economic burden of a campus MenB outbreak. This outbreak response cost calculator can be used to aid in response planning and highlights the need to shift the public health response from outbreak control to prevention by proactive, pre-emptive vaccination using available licensed meningococcal vaccines.Figure 1.Timeline of vaccination clinicsDisclosures E. M. La, RTI Health Solutions (RTI-HS): Employee and RTI-HS is an independent scientific research organization which was retained pursuant to a contract with Pfizer to conduct the research services which are the subject of this presentation/abstract., Salary and The RTI-HS employees who worked on this project did not receive compensation from Pfizer or any other organization, other than RTI-HS salaries.. S. E. Talbird, RTI Health Solutions (RTI-HS): Employee and RTI-HS is an independent scientific research organization which was retained pursuant to a contract with Pfizer to conduct the research services which are the subject of this presentation/abstract., Salary and The RTI-HS employees who worked on this project did not receive compensation from Pfizer or any other organization, other than RTI-HS salaries.. J. Fain, Pfizer Inc.: Employee at time of Study and Employee, Salary. L. Huang, Pfizer: Employee and Shareholder, Salary and Stocks. A. Srivastava, Pfizer: Employee and Shareholder, Salary and Stocks.

Higher Psoriasis Skin Clearance Is Associated with Lower Annual Indirect Costs in the United States: A Post Hoc Analysis from the CLEAR Study.

Psoriasis is associated with a high economic burden to society. New psoriasis systemic treatments offer the potential for improved skin clearance. Whether a higher degree of clearing translates into economic benefit through decreased work impairment has not been fully determined. To assess whether more complete clearing of psoriasis is associated with a reduction in disease-related indirect costs. Pooled data from employed patients included in the CLEAR study, a phase 3b study comparing the efficacy and safety of secukinumab (337 subjects) versus ustekinumab (339 subjects), were classified into 4 levels of skin clearance improvement at weeks 16 and 52: Psoriasis Area and Severity Index (PASI) improvement from baseline of < 50% (PASI < 50), 50%-74% (PASI 50-74), 75%-89% (PASI 75-89), and ≥ 90% (PASI ≥ 90). Patients completed the Work Productivity and Activity Impairment questionnaire for psoriasis (WPAI-PSO), which assessed absenteeism, presenteeism, and a composite overall work impairment over the previous 7 days at weeks 16 and 52. U.S. Department of Labor data were used to calculate annual indirect costs due to work productivity loss. In the CLEAR study, 452 (67%) were employed at baseline and included in this analysis. At week 16, mean overall work impairment significantly decreased with higher PASI improvements: 22.8% for PASI < 50, compared with 13.3% for PASI 50-74 (P = 0.001); 6.4% for PASI 75-89 (P < 0.001); and 4.9% for PASI ≥ 90 (P < 0.001), with the majority of work impairment related to presenteeism. Calculated mean work hours lost by overall work impairment decreased with higher PASI improvements: 8.2 hours lost/week (429 hours/year) for patients with PASI 50; 4.6 hours lost/week (251 hours/year) for PASI 50-74; 2.3 hours lost/week (121 hours/year) for PASI 75-89; and 1.8 hours lost/week (93 hours/year) for PASI ≥ 90. Associated mean annual indirect costs due to work productivity loss per worker decreased with higher PASI improvements: $10,318 for PASI < 50, $6,042 for PASI 50-74, $2,901 for PASI 75-89, and $2,233 for PASI ≥ 90. Similar results were observed at week 52. Mean overall work impairment decreased with higher PASI improvements, ranging from 26.3% for PASI < 50 to 6.9% for PASI ≥ 90. A decrease in overall work hours lost (ranging from 9.5 hours lost/week [495 hours/year] for PASI < 50 to 2.5 hours/week [130 hours/year] for PASI ≥ 90), as well as associated annual indirect costs due to work productivity loss (ranging from $11,906 for PASI < 50 to $3,125 for PASI ≥ 90), were also shown with higher PASI improvements at week 52. Among working patients with moderate to severe psoriasis, higher PASI improvements were associated with lower work productivity loss and reduced annual indirect costs. By improving and sustaining skin clearance, psoriasis treatments may contribute to increased work productivity and decreased societal economic burden. Funding for this study was provided by Novartis Pharmaceuticals. Zhao and Herrera are employed by Novartis. Gilloteau is employed by Novartis Pharma AG. McBride, Graham, and Miles are employed by RTI Health Solutions, which provides consulting and other research services to pharmaceutical, device, governmental, and nongovernmental organizations and received funding from Novartis for manuscript development, analysis development, and general consultation. Feldman reports grants and personal fees from Novartis, Abbvie, Janssen, Lilly, and Celgene, along with personal fees from Amgen and Valeant.

The Effect of Medical Technology Innovations on Patient Outcomes, 1990-2015: Results of a Physician Survey.

Developments in diagnostics, medical devices, procedures, and prescription drugs have increased life expectancy and quality of life after diagnosis for many diseases. Previous research has shown that, overall, increased investment in medical technology has led to increased health outcomes. In addition, the value of investment in specific innovations, particularly in new pharmaceuticals or biopharmaceuticals, has frequently been shown through an evaluation of the associated health outcomes and costs. Value assessments for all medical technologies and interventions are an important consideration in current debates on access and affordability of health care in the United States. To identify practicing physician impressions of the historical effect of postdiagnosis innovations in medical technology on patient outcomes within the 8 health conditions that have the largest effect on health in the United States. National statistics were used to identify the 8 conditions responsible for the most mortality and morbidity within the United States between 1990 and 2014. A physician survey was developed for each major condition to obtain physician opinion on the extent to which pharmaceuticals and biopharmaceuticals, medical devices, diagnostics, and surgical procedures contributed to improvements in postdiagnosis mortality and morbidity outcomes over the evaluated period. Respondents were provided with a fifth category, "cannot allocate," to account for postdiagnosis outcome gains resulting from other factors such as public health interventions. The conditions identified as having the greatest effect on morbidity and mortality since 1990 were breast cancer, ischemic heart disease, human immunodeficiency virus infection, diabetes, unipolar depression, chronic obstructive pulmonary disease, cerebrovascular disease, and lung cancer. After excluding other factors, physicians specializing in these conditions, with a mean of 21.4 years in practice, considered pharmaceuticals and biopharmaceuticals as having the greatest postdiagnosis effect across all 8 conditions, with 56% of outcome gains attributed to this innovation category. Diagnostics was the second biggest contributor at 20%. Physician perceptions indicated that attention should be paid to value assessments of innovative diagnostics, devices, and surgical procedures, as well as to pharmaceuticals and biopharmaceuticals, before goals for allocating health care expenditures among the different innovations are determined. Funding for this study was provided by the National Pharmaceutical Council, a health policy research group that receives its funding from biopharmaceutical manufacturers. Wamble is employed by RTI Health Solutions, which received funding from the National Pharmaceutical Council to conduct this research. Ciarametaro and Dubois are employed by the National Pharmaceutical Council.