Evolving oncology care management trends in the United States: A survey among health care decision makers.

BACKGROUND: There is limited knowledge of how US managed care professionals view and prioritize quality metrics/performance measures, care models, alternative payment models, and clinical pathways in oncology settings. OBJECTIVE: To characterize payor perspectives on, and the use of, oncology clinical pathways and performance measures in their reimbursement/access decision-making process. METHODS: A survey was implemented via SurveyMonkey software and distributed electronically to a national sample of the Academy of Managed Care Pharmacy (AMCP) Market Insights Panel members from July 11 through August 5, 2022. The survey was created by a steering committee based on literature reviews of the current and future oncology care landscapes. The survey consisted of 47 questions, including those to establish respondents' position, responsibilities, and demographics. The results are presented as descriptive statistics for 7 key questions that covered the perceptions and use of quality metrics/performance measures, alternative payment models, and oncology care pathways as prioritized by the steering committee. RESULTS: Among the 695 AMCP panel members who were sent the survey, 73 responded (response rate 10.5%), 54 were eligible to continue, and 31 completed the entire questionnaire; the low response rate may limit generalizability of the survey results. Specific oncology clinical and economic measures of performance were currently used (70%-88%) but generally received less endorsement for future use (39%-49%) except for chemotherapy during end of life, which was considered for future use by 80% of respondents but was only currently used by 31%. Benchmarking was the primary reason for the use of performance measures; only 27% used these to inform value-based agreements. Real-world data tracked by respondents' institutions primarily focused on managed care and pharmacy utilization (39%-85%), with patient-reported and clinical outcomes tracked by only 17%-34%. Almost one-third (31%) did not use clinical oncology pathways, and among those who did, fewer than half (48%) reported that their organization tracks whether treatment decisions agree with the oncology care pathways, and only 26% reported feedback to oncology providers on how often their treatment decisions agree with the pathways. When considering alternative payment models, patient-related components received lower rankings in importance than clinical relevance, actionability, and costs. CONCLUSIONS: Variation among payors regarding current trends in oncology care management, including on the importance of patient-centric outcomes and the use of oncology clinical pathways, suggests the need to focus on value-based health care and greater uptake of oncology clinical pathways.

Clinical characteristics and treatment patterns of patients with NTRK fusion-positive solid tumors: A multisite cohort study at US academic cancer centers.

BACKGROUND: Neurotrophic tyrosine receptor kinase (NTRK) gene fusions are rare oncogenic drivers prevalent in 0.3% of solid tumors. They are most common in salivary gland cancer (2.6%), thyroid cancer (1.6%), and soft-tissue sarcoma (1.5%). Currently, there are 2 US Food and Drug Administration-approved targeted therapies for NTRK gene fusions: larotrectinib, approved in 2018, and entrectinib, approved in 2019. To date, the real-world uptake of tyrosine receptor kinase inhibitor (TRKi) use for NTRK-positive solid tumors in academic cancer centers remains largely unknown. OBJECTIVE: To describe the demographics, clinical and genomic characteristics, and testing and treatment patterns of patients with NTRK-positive solid tumors treated at US academic cancer centers. METHODS: This was a retrospective chart review study conducted in academic cancer centers in the United States. All patients diagnosed with an NTRK fusion-positive (NTRK1, NTRK2, NTRK3) solid tumor (any stage) and who received cancer treatment at participating sites between January 1, 2012, and July 1, 2023, were included in this study. Patient demographics, clinical characteristics, genomic characteristics, NTRK testing data, and treatment patterns were collected from electronic medical records and analyzed using descriptive statistics as appropriate. RESULTS: In total, 6 centers contributed data for 55 patients with NTRK-positive tumors. The mean age was 49.3 (SD = 20.5) years, 51% patients were female, and the majority were White (78%). The median duration of time from cancer diagnosis to NTRK testing was 85 days (IQR = 44-978). At the time of NTRK testing, 64% of patients had stage IV disease, compared with 33% at cancer diagnosis. Prevalent cancer types in the overall cohort included head and neck (15%), thyroid (15%), brain (13%), lung (13%), and colorectal (11%). NTRK1 fusions were most common (45%), followed by NTRK3 (40%) and NTRK2 (15%). Across all lines of therapy, 51% of patients (n = 28) received a TRKi. Among TRKi-treated patients, 71% had stage IV disease at TRKi initiation. The median time from positive NTRK test to initiation of TRKi was 48 days (IQR = 9-207). TRKis were commonly given as first-line (30%) or second-line (48%) therapies. Median duration of therapy was 610 (IQR = 182-764) days for TRKi use and 207.5 (IQR = 42-539) days for all other first-line therapies. CONCLUSIONS: This study reports on contemporary real-world NTRK testing patterns and use of TRKis in solid tumors, including time between NTRK testing and initiation of TRKi therapy and duration of TRKi therapy.

Modeling the population health impact of incorporating a multi-cancer early detection (MCED) test to existing cancer screening among immunocompromised individuals.

OBJECTIVE: To assess the screening efficiency of an multi-cancer early detection (MCED) test added to standard of care (SoC) screening, compared to SoC screening alone, among immunocompromised individuals, and to estimate the diagnostic workup costs associated with positive screening results. METHODS: We estimated the potential impact of cancer screening among immunocompromised individuals aged 50-79 years within the University of Utah Health system who underwent a stem cell/solid organ transplant or were diagnosed with a primary or secondary immunodeficiency disorder between January 2000 and February 2018. We derived cancer incidence rates from the Huntsman Cancer Institute Tumor Registry, and screening performance of SoC screening and an MCED test from published literature. Outcomes of screening efficiency included the true-positive to false-positive (TP:FP) ratio, diagnostic yield (DY), and cancer detection rate (CDR) for SoC screening alone and an incremental MCED test. Scenario and probabilistic sensitivity analyses were conducted. RESULTS: Among 4932 immunocompromised individuals aged 50-79 years, we estimated that 2595 tests would be done under SoC screening and assumed that all individuals received an additional MCED test. Adding an MCED test to SoC screening substantially improved screening efficiency (TP:FP = 1:1, DY = 5.15/1000 tests, CDR = 42.0%), compared to SoC screening alone (TP:FP = 1:99, DY = 1.23/1000 tests, CDR = 5.3%), assuming an MCED test with 100% uptake. Our findings were also robust to parameter uncertainty. CONCLUSION: Adding an MCED test to complement existing screening may be a highly efficient strategy to increase the detection of cancers among immunocompromised individuals. These results could help to improve cancer prevention and detection efforts among individuals with multiple cancer risk factors.

Potential of pharmacogenetics in minimizing drug therapy problems in cystic fibrosis.

BACKGROUND: With advancements in CF drug development, people with cystic fibrosis (PwCF) now take a median of seven medications daily, increasing treatment complexity, risk of drug therapy problems (DTPs), and interference with treatment goals. Given that some of these DTPs can be prevented with preemptive pharmacogenetic testing, the overall goal of this study was to test the clinical utility of a multi-gene pharmacogenetics (PGx) panel in potentially reducing DTPs in PwCF. METHODS: A population based retrospective study of patients with CF was conducted at the University of Utah Health Care System. The patients were genotyped for CYP450 enzymes using a pharmacogenomic assay, and their drug utilization information was obtained retrospectively. This pharmacogenomic information was combined with clinical guidelines to predict the number of actionable PGx interventions in this patient cohort. RESULTS: A total of 52 patients were included in this study. In the patient sample, a minimum of one order of actionable PGx medication was observed in 75 % of the cases. Results revealed that 4.2 treatment modifications per 10 patients can be enabled with the help of a PGx intervention in this patient population. Additionally, our findings suggest that polymorphisms in CYP2D6 and CYP2C19 are most likely to be the primary contributors to DTP's within PwCF. CONCLUSION: This study provides evidence that the PGx panel has the potential to help alleviate the clinical burden of DTPs in PwCF and can assist in informing pharmacotherapy recommendations. Future research should validate these findings and evaluate which subgroups of PwCF would most benefit from pharmacogenetic testing.

Gastrointestinal adverse effects associated with the use of intravenous oliceridine compared with intravenous hydromorphone or fentanyl in acute pain management utilizing adjusted indirect treatment comparison methods.

Background: In the absence of head-to-head comparative data from randomized controlled trials, indirect treatment comparisons (ITCs) may be used to compare the relative effects of treatments versus a common comparator (either placebo or active treatment). For acute pain management, the effects of oliceridine have been compared in clinical trials to morphine but not to fentanyl or hydromorphone. Aim: To assess the comparative safety (specifically differences in the incidence of nausea, vomiting and opioid-induced respiratory depression [OIRD]) between oliceridine and relevant comparators (fentanyl and hydromorphone) through ITC analysis. Methods: A systematic literature review identified randomized clinical trials with oliceridine versus morphine and morphine versus fentanyl or hydromorphone. The ITC utilized the common active comparator, morphine, for the analysis. Results: A total of six randomized controlled trials (oliceridine - 2; hydromorphone - 3; fentanyl - 1) were identified for data to be used in the ITC analyses. The oliceridine data were reported in two studies (plastic surgery and orthopedic surgery) and were also reported in a pooled analysis. The ITC focused on nausea and vomiting due to limited data for OIRD. When oliceridine was compared with hydromorphone in the ITC analysis, oliceridine significantly reduced the incidence of nausea and/or vomiting requiring antiemetics compared with hydromorphone (both orthopedic surgery and pooled data), while results in plastic surgery were not statistically significant. When oliceridine was compared with hydromorphone utilizing data from Hong, the ITC only showed a trend toward reduced risk of nausea and vomiting with oliceridine that was not statistically significant across all three comparisons (orthopedic surgery, plastic surgery and combined). An ITC comparing oliceridine with a study of fentanyl utilizing the oliceridine orthopedic surgery data and combined orthopedic and plastic surgery data showed a trend toward reduced risk that was not statistically significant. Conclusion: In ITC analyses, oliceridine significantly reduced the incidence of nausea and/or vomiting or the need for antiemetics in orthopedic surgery compared with hydromorphone and a non-significant trend toward reduced risk versus fentanyl.

Real-world evidence in the use of Bevacizumab in age-related macular degeneration (ArMD): a scoping review.

PURPOSE: Pharmacological treatments for age-related macular degeneration (ArMD) include anti-vascular endothelial growth factor therapies. Bevacizumab is used off-label, as it has no indication for ArMD. This study aims to identify and describe literature on real-world evidence of bevacizumab (originator or biosimilars) use in ArMD. METHODS: A scoping review was conducted in Medline, CINAHL and Embase databases. Studies published in English after September 2017, conducted in USA, including adults (≥ 18 years old) with ArMD who received treatment with bevacizumab for ArMD were included. The review was further limited to peer-reviewed observational studies that quantitatively analyze either clinical or patient-reported outcomes among patients treated with bevacizumab for ArMD. RESULTS: The search strategy retrieved 543 studies. After title and abstract screening, a total of 142 studies were selected for full-text review leading to a total of 12 studies qualifying for data charting. All were retrospective studies. Five (41.6%) of the studies had less than 500 eyes included in the analysis, and the rest had over a thousand eyes. All except one study reported clinical outcomes (visual acuity was the main outcome in 8 (66.6%) studies). There were 3 (25%) studies reporting adverse events of bevacizumab intravitreal injections. None of the studies specified using biosimilars for bevacizumab and none mentioned patient-reported outcomes. CONCLUSION: The lack of studies aiming to study the patient-reported outcomes as well as the use of biosimilars of bevacizumab in ArMD makes this field a potential for future research. The different exposures and times to follow-up make it difficult to compare results among the selected studies.

The implementation of value-based frameworks, clinical care pathways, and alternative payment models for cancer care in the United States.

BACKGROUND: Cancer treatment is a significant driver of rising health care costs in the United States, where the annual cost of cancer care is estimated to reach $246 billion in 2030. As a result, cancer centers are considering moving away from fee-for-service models and transitioning to value-based care models, including value-based frameworks (VBFs), clinical care pathways (CCPs), and alternative payment models (APMs). OBJECTIVE: To assess the barriers and motivations for using value-based care models from the perspectives of physicians and quality officers (QOs) at US cancer centers. METHODS: Sites were recruited from cancer centers in the Midwest, Northeast, South, and West regions in a 15/15/20/10 relative distribution. Cancer centers were identified based on prior research relationships and known participation in the Oncology Care Model or other APMs. Based on a literature search, multiple choice and open-ended questions were developed for the survey. A link to the survey was emailed to hematologists/oncologists and QOs at academic and community cancer centers from August to November 2020. Results were summarized using descriptive statistics. RESULTS: A total of 136 sites were contacted; 28 (21%) centers returned completed surveys, which were included in the final analysis. 45 surveys (23 from community centers, 22 from academic centers) were completed: 59% (26/44), 76% (34/45), and 67% (30/45) of physicians/QOs respondents had used or implemented a VBF, CCP, and APM, respectively. The top motivator for VBF use was "producing real-world data for providers, payers, and patients" (50% [13/26]). Among those not using CCPs, the most common barrier was a "lack of consensus on pathway choices" (64% [7/11]). For APMs, the most common difficulty was that "innovations in health care services and therapies must be adopted at the site's own financial risk" (27% [8/30]). CONCLUSIONS: The ability to measure improvements in cancer health outcomes was a large motivator for implementing value-based models. However, heterogeneity in practice size, limited resources, and potential increase in costs were possible barriers to implementation. Payers need to be willing to negotiate with cancer centers and providers to implement the payment model that will most benefit patients. The future integration of VBFs, CCPs, and APMs will depend on reducing the complexity and burden of implementation. DISCLOSURES :Dr Panchal was affiliated with the University of Utah at the time this study was conducted and discloses current employment with ZS. Dr McBride discloses employment with Bristol Myers Squibb. Dr Huggar and Dr Copher report employment, stock, and other ownership interests in Bristol Myers Squibb. The other authors have no competing interests to disclose. This study was funded by an unrestricted research grant from Bristol Myers Squibb to the University of Utah.

Caregiver survey in glioblastoma focused on cognitive dysfunction: development and results from a multicenter study.

Aim: To develop a cognitive dysfunction (CD) focused questionnaire to evaluate caregiver burden in glioblastoma. Materials & methods: The survey was developed from stakeholder consultations and a pilot study, and disseminated at eight US academic cancer centers. Caregivers self-reported caring for an adult with glioblastoma and CD. Results: The 89-item survey covered demographics, CD symptoms and caregiver burden domains. Among 185 caregivers, most were white, educated females and reported memory problems as the most common CD symptom. An exposure-effect was observed, with increase in number of CD symptoms significantly associated with greater caregiver burden. Conclusion: This questionnaire could guide caregiver interventions and be adapted for use longitudinally, in community cancer settings, and in patients with brain metastases.

Glioblastoma (GBM) is a very aggressive brain cancer. People who have GBM have trouble remembering things and are unable to do things they used to do. These changes can be very hard. Researchers are trying to better understand what it is like for people who take care of people with GBM (or caregivers). In this study, researchers created a new survey for caregivers. The survey included questions about what caregivers see happening in their loved one with GBM. Caregivers said that memory problems were common. Also, when the patient had more problems the caregiver had a harder time, too. Researchers hope to improve the survey and use it in the future for more studies.

Patient Perspectives on the Use of Digital Technology to Help Manage Cystic Fibrosis.

Background: Digital health technologies (DHTs) have shown potential to improve health outcomes through improved medication adherence in different disease states. Cystic fibrosis (CF) requires care coordination across pharmacies, patients, and providers. DHTs can potentially support patients, providers, and pharmacists in diseases like CF, where high medication burden can negatively impact patient quality of life and outcomes. Methods: In this prospective cohort study, a CF-specific mobile application (Phlo) was distributed to adults with CF who received care at the University of Utah Cystic Fibrosis Center, used an iPhone, and filled prescriptions through the University of Utah Specialty Pharmacy services. Participants were asked to use Phlo for 90 days with an optional 90-day extension period. Participants completed four surveys at baseline and after 90 days. Changes in patient-reported outcomes, adherence, clinical outcomes, and healthcare resource utilization from baseline to 90 days were tracked. Results: Phlo allowed users to track daily regimen activities, contact their care team, receive medication delivery reminders, and share progress with their healthcare team. A web-based dashboard allowed the care team to review reported performance scores from the app. Most patients (67%) said the app improved confidence in and motivation for continuing their regimen. The most important reported benefit of Phlo was having a single location to manage their whole routine. Conclusions: Phlo is a mobile health technology designed to help patients with CF manage their treatment regimen and improve patient-provider communication.

Cancer incidence in immunocompromised patients: a single-center cohort study.

BACKGROUND: Diminished immune defense plays an important role in cancer development. Cancer risk in immunocompromised patients may differ. Identifying individuals with elevated cancer risk can inform strategies for routine cancer screening. This study aimed to understand and compare cancer incidence and risk in three patient groups: recipients of solid organ transplant (SOT) or hematopoietic stem cell transplant (HSCT); diagnosis of primary or secondary immunodeficiency disorder (PID/SID); and recipients of tumor necrosis factor inhibitor (TNF-i) therapy. METHODS: This retrospective cohort study used the University of Utah Health System database and Huntsman Cancer Institute tumor registry. Patients aged ≥18 years with SOT/HSCT, PID/SID or ≥ 3 months of TNF-i therapy were included. The date of transplant, diagnosis of PID/SID, or 1st TNF-i medication order date was defined as the index date. We calculated cumulative cancer incidence by Kaplan-Meier method. A Cox-proportional hazard regression model with a stepwise variable selection process was used to identify independent risk factors associated with the time to onset of a new primary cancer. RESULTS: In total, 13,887 patients were included which comprised of 2982 (21%) SOT/HSCT, 7542 (54%) PID/SID and 3363 (24%) patients receiving TNF-i. The mean (SD) age ranged from 46.8 (15) years - 50.4 (18.2) years. The proportion of white patients ranged from 72.3-84.8%. The estimated cumulative cancer incidence was 11.5% in the SOT/HSCT cohort, 14.3% in the PID/SID cohort, and 8.8% in the TNF-i cohort. The multivariable model adjusted for age, benign in-situ disease, Charlson Comorbidity Index, hypertension/cardiovascular disease/end stage renal disease, gender, race/ethnicity, and renal cyst as significant risk factors. The adjusted hazard ratios for cancer development in SOT/HSCT and PID/SID cohorts compared to the TNF-i cohort over the full follow-up period were 1.57 (95% CI: 1.16-2.13) and 2.14 (95% CI: 1.65-2.77), respectively. CONCLUSION: A significantly increased risk of cancer was observed in PID/SID patients and SOT/HSCT patients compared to TNF-i patients. Age ≥ 50 years, male gender, and clinical comorbidities were additional factors impacting cancer risk. PID/SID and SOT/HSCT patients may benefit from more intensive cancer screening.

Real-world survival analysis by tumor mutational burden in non-small cell lung cancer: a multisite U.S. study.

BACKGROUND: Tumor mutational burden (TMB) is a potential biomarker to predict tumor response to immuno-oncology agents in patients with metastatic non-small cell lung cancer (NSCLC). MATERIALS AND METHODS: A multi-site cohort study evaluated patients diagnosed with stage IV NSCLC between 2012 and 2019 who had received comprehensive genomic profiling (CGP) and any NSCLC-related treatment at 9 U.S. cancer centers. Baseline characteristics and clinical outcomes were compared between patients with TMB <10 and TMB ≥10. RESULTS: Among the 667 patients with CGP results, most patients received CGP from Foundation Medicine (64%) or Caris (20%). Patients with TMB ≥10 (vs. TMB <10) were associated with a positive smoking history. TMB was associated with ALK (p = 0.01), EGFR (p < 0.01), and TP53 (p < 0.05) alterations. TMB >10 showed a significant association towards longer overall survival (OS) (HR: 0.43, 95% CI: 0.21-0.88, p = 0.02) and progression-free survival (PFS) (HR: 0.43, 95% CI: 0.21-0.85, p = 0.02) in patients treated with first-line immunotherapy and tested by Foundation Medicine or Caris at treatment initiation. CONCLUSIONS: TMB levels greater than or equal to 10 mut/Mb, when tested by Foundation Medicine or Caris at treatment initiation, were significantly associated with improved OS and PFS among patients treated with first-line immunotherapy-containing regimens. Additional prospective research is warranted to validate this biomarker along with PD-L1 expression.

Payer perceptions on the use of patient-reported outcomes in oncology decision making.

BACKGROUND: In oncology, especially with accelerated regulatory approvals and niche populations, US payers appreciate all evidence that can help support formulary decision making, including evidence beyond traditional safety and efficacy data from clinical trials. Research suggests payers incorporate patient-reported outcome (PRO) evidence in their decision making and expect the importance of PRO evidence to grow. Greater understanding on payers' use of PRO information in oncology is needed. OBJECTIVE: To assess US payer perceptions regarding the use of PRO evidence in informing oncology formulary decision making. METHODS: A multidisciplinary steering committee involving a measurement specialist, health economics and outcomes research experts, and payers developed a survey containing single-answer, multiple-answer, and free-response questions. The pilot survey was tested at a mini-advisory board with 5 US payers and revised based on feedback. In February 2020, the survey was distributed to 221 US payers through the AMCP Market Insights program and 10 additional payer panelists who were invited to discuss and contextualize the survey results. Results were presented primarily as frequencies of responses and evaluated by plan size, type of health plan, and geography (regional vs national). Differences in categorical data responses were compared using Pearson chi-square or Fisher exact tests. Two-tailed values are reported and a P value less than or equal to 0.05 was used to indicate statistical significance. RESULTS: Overall, 106 of 231 payers (45.9%) completed the survey; 45.5% represented small plans (< 1 million lives), and 54.5% represented large plans (≥ 1 million lives). Respondents were largely pharmacists (89.9%), with 55.6% of all respondents indicating their job was pharmacy administrator. The majority of payers (60.0% of small health plans and 57.8% of large plans) felt PRO evidence from clinical trials is useful. Similarly, the majority of payers (57.8% of small plans and 51.9% of large plans) felt PRO evidence from real-world studies is useful. Almost half (47.1%) suggested formulary review would be influenced by a lack of PRO evidence from oncology clinical trials either somewhat, much, or a great deal. Most payers (78.2%) thought PRO evidence is useful for providing additional context for safety of oncology therapies. More than one-third of payers (34.3%) valued PRO evidence when comparing 2 similar therapies, and 51.5% felt PRO evidence may help in measuring value for value-based agreements. Panelists indicated PRO evidence can be useful for developing treatment pathways for addressing health-related quality of life, informing provider-patient dialogues, and defining progression-free survival length and quality. CONCLUSIONS: US payers view PRO evidence from both clinical trials and real-world studies as useful for supplementing traditional clinical trial data when making oncology formulary decisions and for refining treatment pathways and care delivery models. Manufacturers of oncology therapies should collect and consider leveraging PRO evidence from both settings when engaging with US payers. DISCLOSURES: Pfizer provided funding for this research, and employees of Pfizer contributed to the development of the survey instrument, were involved in the interpretation of the data, and contributed to the discussion and output as authors. Biskupiak, Oderda, and Brixner are managers of Millcreek Outcomes Group and were paid as consultants on this project. Burgoyne was a consultant for Pfizer on this project. Arondekar, Deal, and Niyazov are employees of Pfizer and own Pfizer stock. Qwek was an employee of Pfizer at the time of this project and owns Pfizer stock.

Caregiver burden by treatment and clinical characteristics of patients with glioblastoma.

BACKGROUND: Glioblastoma is an incurable disease with a poor prognosis. For caregivers of people with glioblastoma, the burden of care can be high. Patients often present with different clinical characteristics, which may impact caregiver burden in different ways. This study aimed to evaluate associations between patient clinical characteristics and caregiver burden/quality of life (QoL). METHODS: Caregiver-patient dyads were enrolled at 7 academic cancer centers in the United States. Eligible caregiver participants were self-reported as the primary caregiver of an adult living with glioblastoma and completed a caregiver burden survey. Eligible patients were age ≥ 18 years at glioblastoma diagnosis and alive when their respective caregiver entered the study, with the presence of cognitive dysfunction confirmed by the caregiver. Data were analyzed with descriptive statistics and multivariable analyses. RESULTS: The final cohort included 167 dyads. Poor patient performance status resulted in patient difficulty with mental tasks, more caregiving tasks, and increased caregiving time. Language problems were reported in patients with left-sided lesions. Patient confusion was negatively associated with all caregiver domains: emotional health, social health, general health, ability to work, confidence in finances, and overall QoL. Better caregiver QoL was observed in patients with frontal lobe lesions versus non-frontal lobe lesions. CONCLUSION: This study reinforced that patient performance status is a critical clinical factor that significantly affects caregiver burden, caregiving tasks, and caregiver time. Additionally, patient confusion affects multiple facets of caregiver burden/QoL. These results could be used to support guided intervention for caregiver support, customized to the patient experience.

Payer perceptions on the use of economic models in oncology decision making.

BACKGROUND: To support oncology formulary decisions, especially with accelerated regulatory approvals and niche populations, payers desire data beyond what regulators review. Economic models showing financial impact of treatments may help, but data on payers' use of economic models in oncology are limited. OBJECTIVE: To assess payer perceptions regarding use of economic models in informing oncology formulary decisions. METHODS: A multidisciplinary steering committee involving health economists and payers developed a survey containing singleanswer, multiple-answer, and free-response questions. The pilot survey was tested at a mini-advisory board with 5 US payers and revised based on feedback. In February 2020, the survey was distributed to 221 US payers through the AMCP Market Insights program and 10 additional payer panelists, who were invited to discuss survey results. Results were presented primarily as frequencies of responses and evaluated by plan size, type of health plan, and geography (regional vs national). Differences in categorical data responses were compared using Pearson chi-square or Fisher's exact tests. Two-tailed values were reported and an alpha level of 0.05 or less was used to indicate statistical significance. RESULTS: Overall, 106 of 231 payers completed the survey (45.9%); 45.5% represented small plans (< 1 million lives), and 54.5% represented large plans (≥ 1 million lives). Respondents were largely pharmacists (89.9%), and 55.6% indicated that their job was pharmacy administrator. Payers indicated moderate/most interest in cost-effectiveness models (CEMs; 85.3%) and budget impact models (BIMs; 80.4%). Overall, 51.6% of respondents claimed oncology expertise on their pharmacy and therapeutics committees. Large plans were more likely to have expertise in reviewing oncology economic models than small plans (55.6% vs 31.1%, P = 0.015). The most common reasons for not reviewing economic models included "not available at time of review" (44.1%) and "potential bias" (38.2%). Overall, 43.1% of payers conduct analyses using their own data after reviewing a manufacturer-sponsored economic model. To inform formulary decisions, 62.7% of payers use BIMs and 66.7% use CEMs sometimes, often, or always. When comparing therapies with similar safety/efficacy profiles, 68.6% of payers reported economic models as helpful a moderate amount, a lot, or a great deal. Over one-third of payers (37.3%) were willing to partner with manufacturers on economic models using their plans' data. Payers valued preapproval information, data on total cost of care, and early access to models. Concerns remained regarding model transparency and assumptions. CONCLUSIONS: Most US payers reported interest in using economic models to inform oncology formulary decision making. Opportunities exist to educate payers in assessing economic models, especially among small health plans. Ensuring model availability at launch, transparency in model assumptions, and payer-manufacturer partnership in model development may increase the utility of oncology economic models among US payers. DISCLOSURES: Pfizer provided funding for this research, and Pfizer employees led the development of the survey instrument, were involved in the analysis and interpretation of the data, and contributed to the manuscript as authors. Arondekar and Niyazov are employed by Pfizer. Biskupiak, Oderda, and Brixner are managers of Millcreek Outcomes Group and were paid as consultants on this project. Burgoyne was a consultant for Pfizer on this project.

Payer perceptions of the use of real-world evidence in oncology-based decision making.

BACKGROUND: Randomized controlled trials (RCTs), the gold standard of safety and efficacy evidence, are conducted in select patients that may not mirror real-world populations. As a result, healthcare decision makers may have limited information when making formulary decisions, especially in oncology, given accelerated regulatory approvals and niche patient populations. Real-world evidence (RWE) studies may help address these knowledge gaps and help inform oncology formulary decision making. OBJECTIVE: To assess US payer perceptions regarding the use and relevance of RWE in informing oncology formulary decisionmaking. METHODS: A national survey containing single-answer, multiple-answer, and free-response questions evaluated 4 key areas: (1) the value of RWE, (2) barriers to RWE, (3) sources of RWE, and (4) use of RWE in outcomes-based contracting. The survey was distributed to 221 US payers through the Academy of Managed Care Pharmacy (AMCP) Market Insights program in February 2020. Ten additional respondents were invited to discuss the survey results. The survey results were presented primarily as frequencies of responses and were evaluated by the respondent's plan size, type, and geography (regional vs national). Differences in responses for categorical data were compared using a Pearson Chi-Square or a Fisher's Exact test. Two-tailed values are reported and a level of ≤ 0.05 was used to indicate statistical significance. RESULTS: The national survey had a 45.9% response rate, with 106 payers responding. Most were from managed care organizations (MCOs; 47.5%) and pharmacy benefit managers (PBMs; 37.4%), with 54.5% from large plans (≥ 1 million lives) and 45.5% from small plans (< 1 million lives). Respondents were largely pharmacists (89.9%), with 55.6% overall indicating their job was a pharmacy administrator. Most (84.9%) used RWE to inform formulary decisions in oncology to support comparative effectiveness in the absence of head-to-head clinical trials (4.1 on a scale of 1 = Not At All Useful to 5 = Extremely Useful) and validation of National Comprehensive Cancer Network (NCCN) recommendations (4.0). Almost half (41.5%) used RWE results to inform off-label usage decisions. Payers valued RWE pre-launch to inform formulary and contracting decisions and desired real-world comparative effectiveness data post-launch to validate coverage decisions. However, the majority of payers (54.7%) did not conduct their own real-world studies. Commonly considered RWE sources included claims data (79.2%), medical records (68.9%), prospective cohort studies (60.4%), patient registries (36.8%), and patient outcome surveys (33.0%). Barriers to conducting internal RWE studies included the lack of resources and personnel, analytic capabilities, appropriate in-house data, and perceived value in conducting analyses. Payers expressed interest in using outcomes-based contracting in oncology; few have direct experience, and operationalizing through value measurement is challenging. CONCLUSIONS: RWE providing comparative treatment data, validation of NCCN treatment recommendations, and information on off-label usage are appreciated pre launch with post launch validation. DISCLOSURES: Pfizer provided funding for this research, and employees of Pfizer led the development of the survey and contributed to the manuscript as authors. Arondekar and Niyazov are employees of Pfizer; Oderda, Biskupiak, and Brixner are managers of Millcreek Outcomes Group and were paid as consultants on this project. Burgoyne was a consultant for Pfizer on this project. Malone was paid by Millcreek Outcomes as a consultant on this project.

Can Cost-effectiveness Analysis Inform Genotype-Guided Aspirin Use for Primary Colorectal Cancer Prevention?

BACKGROUND: Inherited genetic variants can modify the cancer-chemopreventive effect of aspirin. We evaluated the clinical and economic value of genotype-guided aspirin use for colorectal cancer chemoprevention in average-risk individuals. METHODS: A decision analytical model compared genotype-guided aspirin use versus no genetic testing, no aspirin. The model simulated 100,000 adults ≥50 years of age with average colorectal cancer and cardiovascular disease risk. Low-dose aspirin daily starting at age 50 years was recommended only for those with a genetic test result indicating a greater reduction in colorectal cancer risk with aspirin use. The primary outcomes were quality-adjusted life-years (QALY), costs, and incremental cost-effectiveness ratio (ICER). RESULTS: The mean cost of using genotype-guided aspirin was $187,109 with 19.922 mean QALYs compared with $186,464 with 19.912 QALYs for no genetic testing, no aspirin. Genotype-guided aspirin yielded an ICER of $66,243 per QALY gained, and was cost-effective in 58% of simulations at the $100,000 willingness-to-pay threshold. Genotype-guided aspirin was associated with 1,461 fewer polyps developed, 510 fewer colorectal cancer cases, and 181 fewer colorectal cancer-related deaths. This strategy prevented 1,078 myocardial infarctions with 1,430 gastrointestinal bleeding events, and 323 intracranial hemorrhage cases compared with no genetic testing, no aspirin. CONCLUSIONS: Genotype-guided aspirin use for colorectal cancer chemoprevention may offer a cost-effective approach for the future management of average-risk individuals. IMPACT: A genotype-guided aspirin strategy may prevent colorectal cancer, colorectal cancer-related deaths, and myocardial infarctions, while minimizing bleeding adverse events. This model establishes a framework for genetically-guided aspirin use for targeted chemoprevention of colorectal cancer with application toward commercial testing in this population.

Higher Medication Adherence and Lower Opioid Use Among Individuals with Autoimmune Disease Enrolled in an Adalimumab Patient Support Program in the United States.

INTRODUCTION: Opioid use is prevalent among patients with autoimmune conditions, despite not being a recommended treatment. Tumor necrosis factor inhibitor (anti-TNF) therapy is an effective treatment for these autoimmune conditions, and patient support programs (PSPs) have been developed to help patients manage their prescribed treatments. This study was conducted to evaluate the impact of PSPs on anti-TNF adherence and opioid use using data on adalimumab (ADA), an anti-TNF. METHODS: The study used insurance claims data linked to ADA PSP data on patients who initiated ADA after 01/2015, were commercially insured, and had data coverage for 1 year before and after (i.e., during the follow-up period) ADA initiation. Patients with opioid use in the 3 months before ADA initiation were excluded. PSP patients enrolled in the PSP within 30 days of ADA initiation and had 2+ PSP nurse ambassador interactions; non-PSP patients had no PSP engagement. ADA adherence [proportion of days covered (PDC), persistence], opioid initiation, 2+ opioid fills, and opioid supply during follow-up were compared between cohorts using regression models that controlled for patient characteristics. RESULTS: Results were obtained for 1952 PSP and 728 non-PSP patients. PSP patients demonstrated better adherence to ADA than non-PSP patients, including higher PDC and persistence (all p < 0.001). PSP patients were 13% less likely to initiate opioids and 26% less likely to have at least 2 fills than non-PSP patients, and they had fewer days of opioid supply (all p < 0.01). CONCLUSIONS: This study supports the benefit of PSPs and suggests that the ADA PSP is associated with improved adherence and potentially lower opioid use.

Budget impact of capmatinib for adults with metastatic non-small cell lung cancer harboring a MET exon 14 skipping mutation in the United States.

AIMS: To estimate the budget impact of adding capmatinib, the first FDA approved MET inhibitor, to a US commercial or Medicare health plan for patients with metastatic non-small cell lung cancer (mNSCLC) whose tumors have a mutation that leads to MET exon 14 (METex14) skipping. METHODS: Target population size was estimated using published epidemiology data. Clinical data were obtained from the GEOMETRY mono-1 capmatinib trial and published trials. Treatments in the market mix included crizotinib, pembrolizumab, ramucirumab, and chemotherapy. Uptake of capmatinib and testing rates were based on market research. All costs (drug acquisition and administration, pre-progression, progression, terminal care, adverse event, and testing) were estimated based on public sources (2020 USD). RESULTS: The number of patients eligible for capmatinib in the first three years was estimated to be 2-3 in a hypothetical 1 million member commercial plan and 34-44 in a hypothetical 1 million member Medicare plan each year. The estimated total budget impact ranged from $9,695 to $67,725 for a commercial plan and $141,350 to $985,695 for Medicare. With capmatinib included, a marginal per member per month budget impact was estimated (commercial: $0.0008 to $0.0056; Medicare: $0.0118 to $0.0821). Capmatinib inclusion resulted in lower medical costs (commercial: -$0.0003 to -$0.0007; Medicare: -$0.0037 to -$0.0106), partially offsetting increased drug costs ($0.0011 to $0.0064; $0.0154 to $0.0928, respectively), and were primarily driven by reductions in progression and terminal care costs (-$0.0003 to -$0.0009; -$0.0037 to -$0.0125, respectively). The results were most sensitive to capmatinib market share, capmatinib price, and treatment duration. LIMITATIONS: Certain assumptions were applied to the model to account for inputs with limited evidence. CONCLUSIONS: The estimated budget impact of including capmatinib for mNSCLC with a METex14 skipping mutation is minimal, and the increased drug costs were partially offset by savings in AEs, and progression-related and terminal care costs.