BACKGROUND:Vitiligo is a chronic autoimmune skin disorder characterized by loss of melanocytes, resulting in patches of skin depigmentation across the body. Vitiligo can have a substantial negative impact on patients' health-related quality of life, is associated with increased health care costs, and has no known cure. OBJECTIVE:To evaluate the prevalence and economic burden of immune-mediated and psychiatric comorbidities in patients with newly diagnosed vitiligo. METHODS:This retrospective cohort study used data from the Merative MarketScan Commercial Database. Eligible patients were aged 12 to 64 years with a new diagnosis of vitiligo (International Classification of Diseases, Tenth Revision; codes L80.x, H02.73-, or N90.89); index date recorded on a nondiagnostic medical claim between January 1, 2019, and September 30, 2024, together with 12 months of pre-index continuous medical/pharmacy enrollment. Outcomes included prevalence of immune-mediated or psychiatric comorbidities at diagnosis (evaluated using up to 3 years of pre-index data) and associated per-patient per-month (PPPM) all-cause and vitiligo-related mean health care costs and health care resource utilization, both unadjusted and adjusted for age, sex, US region, and Charlson Comorbidity Index score. Comorbidities were summarized descriptively; health care costs were compared using γ log-link models. RESULTS:Among the 14,059 patients with vitiligo, 52.8% (n = 7,430) were female and the mean (SD) age was 43 (15) years. Overall, 23.8% of patients had at least 1 immune-mediated comorbidity, most commonly atopic dermatitis (8.0%), psoriasis (5.4%), Hashimoto thyroiditis (3.8%), and alopecia areata (2.0%). At least 1 psychiatric diagnosis was present in 39.4% of patients, most commonly anxiety (20.9%), sleep disturbance (15.2%), and depression (12.7%). PPPM all-cause mean total health care costs among patients with vitiligo and at least 1 immune-mediated comorbidity were significantly higher than those among patients without an immune-mediated comorbidity ($2,112 vs $957, respectively; P < 0.001), as were costs for those with vs without at least 1 psychiatric comorbidity ($1,751 vs $896, respectively; P < 0.001). Among patients with at least 1 Janus kinase-related comorbidity, PPPM costs were $2,105, compared with $1,041 for those without such comorbidities at baseline. CONCLUSIONS:This study highlights the substantial economic burden that patients with preexisting immune-mediated and psychiatric conditions face following vitiligo diagnosis. Approximately 1 in 4 patients had a prior diagnosis of at least 1 immune-mediated condition, reflecting the immunological pathogenesis of vitiligo and that it is not a cosmetic disease. Patients with immune-mediated and psychiatric comorbidities incurred significantly higher health care costs relative to those without these comorbidities, underscoring the need for a comprehensive, multidisciplinary approach to vitiligo management.
In 2025, the Academy of Managed Care Pharmacy (AMCP) held a 2-day partnership forum on the topic of advancing precision medicine access in oncology. Precision medicine, in this context, focuses on biomarker testing required to create personalized treatment plans and procedures. The forum brought together patients; payers, including health insurers and employer groups; pharmacy benefit managers; providers; pathologists; laboratory benefit managers; and leaders from patient advocacy organizations, coalitions, and professional associations. In response to fundamental coverage challenges impacting patient access to biomarker testing, the overarching goal of the forum was to collaboratively ideate deliverables to drive a standardized approach to assessing and determining coverage. A critical deliverable from this initiative included 8 guiding principles for managed care stakeholders as it relates to the coverage of biomarker testing. This article provides detail on each guiding principle, which are considerations intended to guide managed care decision-makers on determining and monitoring coverage for biomarker-driven testing used to inform treatment decisions in oncologic indications.
Precision medicine terminology is increasingly used across clinical, laboratory, payer, regulatory, and policy settings; however, distinct terminology may be applied interchangeably or inconsistently across stakeholders, and foundational concepts may not be uniformly understood across multidisciplinary audiences with varying levels of expertise. This misalignment may create confusion for stakeholders as they attempt to navigate guideline recommendations and payer coverage policies, potentially impacting patient access to testing and treatment. In 2025, AMCP held a 2-day partnership forum on the topic of advancing precision medicine in oncology. Forum participants recommended development of a consolidated lexicon of commonly used precision medicine terms to support clearer communication and foundational understanding across stakeholders and to clarify distinctions between related concepts. Nineteen definitions were ultimately generated using a natural language processing process, an artificial intelligence approach used to extract information and derive meaning from large text samples. A comprehensive literature review was first conducted to develop an index of publicly available precision medicine terminology sources. Natural language processing-based textual analysis was then used to systematically evaluate terminology usage patterns across the index, including both consistent and inconsistent usage of terms across sources. Draft definitions generated through this process were subsequently reviewed and refined by forum participants. The resulting lexicon included relevant terms related to (1) foundational concepts in precision medicine; (2) biological foundations and molecular variation; (3) biomarker and molecular testing; (4) testing technologies, specimen collection, and regulatory classification; and (5) testing approach. The objective of this work was to organize commonly used precision medicine terms into a single reference framework, a foundational precision medicine reference for managed care stakeholders, and to clarify distinctions between related concepts where appropriate.
Establishing the clinical utility of biomarker tests is a critical component of benefit design. However, payers and laboratory benefit manager organizations may differ in how they evaluate clinical utility for a given test, including how they determine whether evaluated outcomes are clinically meaningful. These differences can create challenges for providers and manufacturers when navigating coverage policies across organizations. Based on expert insights from a partnership forum hosted by AMCP on June 24-25, 2025, in Alexandria, VA, on the topic of precision medicine in oncology, we developed a checklist of considerations to align payers and laboratory benefit managers around a shared, actionable approach to evaluating the clinical utility of biomarker tests and the types of evidence used to demonstrate it. The checklist begins with confirmation that analytical and clinical validity have been established before assessing clinical utility. Tests should then be categorized based on their intended use and whether they provide actionable information that informs clinical decision-making and improves patient health outcomes. Assessment of clinical utility includes consideration of the relevance, strength, and consistency of the supporting evidence base. After clinical utility is established, economic and operational considerations may also be evaluated. This checklist is intended to support a more transparent and structured approach to evaluating clinical utility in coverage decision-making.
BACKGROUND:The use of real-time continuous glucose monitoring (RT-CGM) is well established to support people with type 2 diabetes (T2D) in achieving adequate glycemic control. However, health care resource utilization (HCRU) studies have previously tended to focus on individuals with T2D who are receiving treatment with insulin. OBJECTIVE:To examine the impacts of RT-CGM on inpatient and emergency department (ED) HCRU in non-insulin-treated people. METHODS:This retrospective study analyzed US administrative claims data from Optum's Clinformatics Data Mart database between September 1, 2016, and December 31, 2024. Continuous glucose monitoring (CGM)-naive individuals with T2D who initiated a Dexcom G-series RT-CGM between September 1, 2017, and December 31, 2023, were included (index date: first RT-CGM claim). Change in the number of all-cause and diabetes-related encounters by service location (inpatient and ED) and the mean change in associated medical costs were assessed over a 6-month pre-index period (baseline) and in 2 consecutive 6-month segments post-index (0-6 months and 7-12 months). Stratification by baseline HCRU encounter frequency unique to service location included the following: nonutilizers, zero baseline encounters; low utilization, 1 to 2 baseline encounters; high utilization, 3 or more baseline encounters. RESULTS:The analysis included 4,463 people with T2D. Decreases were observed in the numbers of inpatient and ED encounters among those using such encounters in the baseline period. Those considered that low utilizers (1-2 baseline encounters) experienced a 66% decrease in inpatient encounters and 63% decrease in ED encounters, whereas high utilizers (≥3 encounters) experienced a 67% decrease in inpatient encounters and 68% decrease in ED encounters. These decreases coincided with significant reductions in associated costs. CONCLUSIONS:People with T2D who were not receiving insulin treatment and have had recent inpatient or ED encounters had lower observed HCRU after the initiation of RT-CGM with Dexcom G-series systems.
Barth syndrome is an ultrarare, complex, multisystem, X-linked metabolic and neuromuscular disease, which poses significant and wide-ranging burden to patients and caregivers. Pharmacologic management focuses on treatment of disease manifestations and prevention of secondary complications. Elamipretide, the first treatment indicated specifically for improving muscle strength in Barth syndrome, was approved in 2025 via accelerated approval warranting guidance for payers. To discuss managed care considerations in Barth syndrome including management of elamipretide, AMCP Market Insights virtually convened an expert panel of managed care stakeholders in March 2026. This article provides a qualitative summary of the panel discussion along with key insights and suggested payer practices meant to support informed coverage decisions and guide future work such as collaboration, research, and advocacy. Key insights highlight that there are unique challenges in generating clinical trial evidence for treatments in ultrarare conditions, which leads to difficulties determining the value of these treatments and differences in whether they are covered among payers. Additionally, there are numerous elements of care to which patients with Barth syndrome and their caregivers need equitable access, which is complicated by involving multiple specialists and fragmentation. Suggested payer practices involve education, care delivery, and coverage and benefit design.
The 2025 AMCP Foundation Symposium: Health Optimization 2.0 convened a select group of just over 70 managed care stakeholders in Dallas, Texas, on December 2-3, 2025. Via presentations and panel sessions, participants examined how precision medicine, data analytics, evolving policy, and health system design can address inequities in care delivery. Focus areas included precision medicine in oncology, vaccine hesitancy, use of data and artificial intelligence, and multiple sclerosis. Following group sessions, participants were divided into work groups and asked to highlight 1 key problem-solution pair for each of 2 assigned focus areas. This article presents a summary of the discussion for key problem-solution pairs in each of the 4 focus areas. The problem-solution pairs identified for vaccine hesitancy all related to delivering positive, community-driven messaging to rebuild eroded public trust. The other focus areas had multiple problems and solutions, which implies that driving meaningful change in these areas may be more complex.
BACKGROUND:Adalimumab is a widely used biologic for autoimmune diseases. Biosimilars to adalimumab became available in 2023 and were expected to improve affordability and access, yet real-world evidence on switching after biosimilar initiation remains limited. OBJECTIVE:To assess adoption of adalimumab biosimilars and characterize the frequency and timing of switch-backs to originator adalimumab. METHODS:This retrospective cohort study used Truveta Data, a deidentified electronic health record dataset. Adult patients were included if they had at least 1 outpatient or telehealth encounter between February 1, 2023, and August 31, 2025, and at least 2 originator adalimumab dispenses in the prior year. Switching was defined as the first biosimilar dispense following originator use, and switch-back was defined as a subsequent originator dispense after biosimilar initiation. Multivariable logistic regression identified characteristics associated with switch-back, and a Cox proportional hazards model evaluated factors associated with earlier switch-back (≤30 days). RESULTS:Among 67,594 patients treated with originator adalimumab, 17.7% (n = 11,947) switched to a biosimilar during the study period. Switching peaked in April 2024, coinciding with formulary changes. Among 9,252 biosimilar initiators, with at least 1 clinical encounter at least 90 days after a biosimilar dispense, 16.0% (n = 1,478) switched back to the originator. Older adults (≥65 years and 50-64 years) had higher odds of switching back (odds ratio [OR] = 2.05 [95% CI = 1.64-2.56] and OR = 1.23 [95% CI = 1.03-1.46], respectively), as did women (OR = 1.19; 95% CI = 1.05-1.34) and patients with ankylosing spondylitis (OR = 1.32; 95% CI = 1.10-1.58). Rural residence was associated with higher odds of early switch-back (OR = 1.80; 95% CI = 1.20-2.70). CONCLUSIONS:Biosimilar uptake increased following formulary changes, yet more than 1 in 7 biosimilar initiators returned to originator adalimumab. Variation in early switch-back and patient characteristics highlights the importance of real-world evidence to understand biosimilar adoption beyond formulary-driven transitions.
BACKGROUND:Primary adherence (PA) to a medication occurs when a patient fills a newly prescribed medication within a predefined window. Failing to fill a necessary medication can have adverse downstream clinical consequences and increased costs. The extent of PA to major depressive disorder (MDD)-related medications in the United States is not well understood. OBJECTIVE:To describe overall primary adherence rates to MDD-related medications, both the initial prescription and at each subsequent switch in the patient's treatment journey. METHODS:We used deidentified electronic health record (EHR) and claims data from the Optum Labs Data Warehouse, a national, longitudinal, real-world data asset containing data on more than 350 million patients. Within an index period (1/1/2021 to 12/31/2022), a PA event was recorded when a medication fill occurred within 30 days after the initial index prescription of a new medication class, where new classes were defined as having no prescription for that class in the preceding 180 days. A medication switch was defined as a prescription for a different medication than the immediately preceding prescribed medication. Patient characteristics and PA outcomes were summarized by (1) medication class, (2) counts of total medication switches during the index period, and (3) order of medication switches. RESULTS:Overall, PA to the index medication was 75%. PA did not vary substantively across medication classes, ranging from 74% (tricyclic class) to 77% (atypical class). Approximately 15% of the sample switched MDD-related medications during the index period, and among this sample, PA to all medications decreased as the number of switches increased, declining from 59% for 1 prescription switch to less than 37% for 4 switches. Among patients with at least 1 switch, PA declined as the number of switches increased from the first switch (70%) to the fourth switch (57%). CONCLUSIONS:PA to initial prescriptions is relatively high although adherence declined with medication switching. This pattern represents a critical clinical vulnerability, which can be addressed through approaches such as education, medication regimen management, and follow-up visits with care providers to promote PA, increase treatment efficacy, and avoid adverse long-term outcomes. Future research should identify barriers to adherence during medication optimization and develop targeted interventions to sustain treatment persistence.
BACKGROUND:Since 2021, new therapies including immune checkpoint inhibitors (ICIs) have been approved in the United States for human epidermal growth factor receptor 2 (HER2)-negative advanced gastric or gastroesophageal junction cancer (G/GEJC) in biomarker-selected populations. However, recent data on treatment patterns, health care resource utilization (HCRU), and costs remain limited. OBJECTIVE:To describe real-world patient characteristics, treatment patterns, HCRU, and costs among patients with HER2-negative advanced G/GEJC in the Medicare and MarketScan databases. METHODS:Two retrospective observational cohort studies were conducted using the 100% Medicare Fee-for-Service database and the Merative MarketScan Commercial and Medicare Databases. Patients with advanced G/GEJC initiating first-line (1L) non-HER2-targeted systemic anticancer therapy from 2021 to 2024, continuously enrolled for at least 6 months before and at least 1 month after the 1L initiation (index date), were identified. Primary outcomes including treatment patterns, HCRU, and costs were assessed from the index to death, disenrollment, or study end, whichever occurred first. Real-world overall survival (rwOS) was assessed as a secondary outcome in the Medicare database. RESULTS:We identified 2,029 and 419 patients in the Medicare and MarketScan databases, respectively. Across both databases, chemotherapy was the most common 1L regimen (approximately 50%), followed by nivolumab-containing regimens (34.7% in Medicare; 44.2% in MarketScan) and pembrolizumab-containing regimens (9.9% and 5.3%, respectively). In both databases, 38.2% of patients received second-line (2L) therapy and 11.7% received third-line (3L) therapy. Nivolumab-containing regimens were the most common 2L treatment (36.3% in Medicare; 41.3% in MarketScan), whereas ramucirumab-containing regimens were most common in 3L (36.7% and 34.0%, respectively). Outpatient visits accounted for the majority of HCRU across lines of therapy, with mean visits per patient per month (PPPM) of 3.7, 3.8, and 3.6 in Medicare and 7.7, 6.5, and 6.6 in MarketScan for 1L, 2L, and 3L, respectively. Mean total all-cause health care costs increased with line of therapy and were higher in MarketScan than Medicare: $17,191, $19,572, and $19,373 PPPM in Medicare and $47,092, $62,358, and $73,192 PPPM in MarketScan for 1L, 2L, and 3L, respectively. Outpatient costs accounted for more than half of total health care costs in both databases. Among Medicare patients, median 1L rwOS was 10.9 months. CONCLUSIONS:Analyses of recent data from the Medicare and MarketScan databases showed that most treated patients with HER2-negative advanced G/GEJC received standard chemotherapy in the 1L setting. Treatment of advanced G/GEJC remains associated with poor rwOS and high economic burden, suggesting significant remaining unmet need.
Pharmacists, as one of the most accessible health care professionals, are often cited as a solution for filling gaps in health care services due to growing shortages of primary care physicians and nurses in the United States. However, future demand forecasts for pharmacists may be low owing to systematic undercounting of pharmacists in nontraditional Managed Care, Pharmaceutical Industry, and Academia roles by the Bureau of Labor Statistics. Accurate and inclusive demand estimates are needed to best inform workforce planning for pharmacists in traditional and nontraditional roles across all industries. The urgency to have accurate demand forecasts including all types of pharmacists is difficult to overstate because PharmD program enrollment has dramatically declined to a level that challenges maintaining pharmacists in traditional community and health system roles-before the need for Managed Care, Pharmaceutical Industry, and Academia pharmacists is even considered. This viewpoint aims to consider this aspect of the demand question of "will there be enough pharmacists?" to help mitigate future pharmacist supply risk to all industries for current and yet unknown functions that will arise, regardless of pharmacist supply, for medication experts to shape the future standards of patient care.
BACKGROUND:The Washington State Prescription Drug Affordability Board (PDAB) is tasked with conducting affordability reviews to identify whether prescription drugs have excess costs, defined as either exceeding therapeutic value or being unsustainable to public and private health care systems. The PDAB also has the authority to set upper payment limits (UPLs). There has been limited reporting of the methods and decisions through which PDABs examine and choose drugs for affordability review. OBJECTIVE:To describe the processes of identifying drugs eligible for affordability review and selecting drugs for affordability review. METHODS:Branded drugs are eligible for affordability review if their wholesale acquisition cost (WAC) was at least $60,000 or their price increased above certain thresholds. Biosimilar products are eligible if their price was not at least 15% lower than the reference biologic product. Generic drugs are eligible if their WAC was at least $100 per 30-day supply and increased by at least 200% in a 12-month period. Eligible drugs were ranked according to 4 criteria with the following weights: total out-of-pocket costs (0.35), total costs (0.33), mean out-of-pocket costs among users (0.19), and total number of users (0.13). RESULTS:290 drug products were eligible in the 2023 drug review cycle. The majority (n = 166) were branded drugs, which were eligible because their WAC for a course of treatment was at least $60,000. After the weighted ranking of eligible drugs, the board chose the top 4 drugs for affordability review: Enbrel, Xtandi, Humira, and Cabometyx. CONCLUSIONS:The selected drugs have high cost burden for patients and payers in Washington. Affordability reviews of these drugs will evaluate whether they have led or will lead to excess costs, which will inform PDAB decisions regarding the possible implementation of UPLs.
BACKGROUND:The management of patients with generalized myasthenia gravis (gMG) is complex and often involves substantial health care expenses. As the treatment paradigm continues to evolve, understanding the economic burden of gMG must also reflect the availability and increasing use of newly approved and advanced therapies in a diverse patient population. OBJECTIVE:To estimate incremental health care resource utilization (HRU) and costs associated with gMG compared with non-MG controls and identify predictors of high health care costs in gMG in an insured US population cohort. METHODS:Adults with newly diagnosed gMG were identified from Komodo Research data (January 1, 2017, to September 30, 2023). Characteristics of adults without MG were weighted using entropy balancing to match the gMG cohort. The index date was the first MG diagnosis by a neurologist for the gMG cohort and a random date for the non-MG cohort. Annual per patient HRU and health care costs were compared between the weighted cohorts. Predictors of high health care costs were evaluated among the gMG cohort using a logistic regression model. RESULTS:After weighting, among 6,195 patients in the gMG cohort and 226,008 in the non-MG cohort, the mean age was 61.1 years, 49.1% were female, 53.4% were covered by commercial insurance, 41.0% were covered by Medicare Advantage, and 5.6% were covered by Medicaid. The gMG cohort (mean follow-up = 32.7 months) compared with the non-MG cohort (mean follow-up = 31.3 months) had 2.4, 1.6, and 1.9 times more inpatient days, emergency department visits, and outpatient visits annually, respectively (all P < 0.001). Mean annual all-cause total health care costs in the gMG cohort were $43,872 higher than in the non-MG cohort ($58,341 vs $14,469; P < 0.001); 59.3% of the difference was driven by pharmacy costs (mostly immunoglobulin). Prediagnosis weakness and fatigue symptoms, exacerbation or crisis, and use of immunoglobulin were important factors significantly associated with high health care costs in the gMG cohort (all P < 0.001). CONCLUSIONS:Despite advances in treatment, the economic burden of gMG remains substantial. Association of high health care costs with early symptoms and disease exacerbations underscores the need for more effective intervention strategies to improve disease control and reduce HRU and costs in patients with gMG.
BACKGROUND:The Institute for Clinical and Economic Review (ICER) aims to publish health technology assessment (HTA) reports at or near the time of drug approval by the US Food and Drug Administration (FDA) to provide a timely, independent evaluation of the benefits, risks, and economic considerations surrounding a new therapy. OBJECTIVE:To evaluate the timelines of ICER reports to inform pricing and coverage decisions, using FDA approval and list price announcement dates as a benchmark. With these data, we evaluated the availability of clinical evidence, specifically completion of pivotal trials and publication in peer-reviewed journals, at the time of ICER's review. METHODS:We included drugs that were the primary intervention of interest in ICER reviews between 2017 and 2024. We extracted information on regulatory approval and list price announcements, and publication dates of ICER reports. We also extracted dates of publication of the pivotal trials that informed each ICER assessment to evaluate the time span between trial completion and publication. RESULTS:Our analysis of 73 ICER drug assessments showed that, on average, the launch price is announced on the same day as FDA approval. The majority of the ICER draft, evidence, and final assessments were published on or before the FDA approval and price announcement date (82%, 61%, 55%, respectively). In terms of data availability, the median time from primary trial completion to peer-review publication was 15 months. There were an average of 2 pivotal trials per drug assessment. The majority (71%) of the pivotal trials informing ICER assessments were published in peer-reviewed journals before the completion of the ICER draft assessment, although these publications were, on average, published approximately 1 month prior to the completion of the assessments. CONCLUSIONS:The majority of ICER drug assessments are published before FDA approval and price announcements, making them available for early decision-making at the time of drug launch. However, data availability is still a major challenge in making HTA available immediately after approval. Prioritizing early sharing of clinical trial data would foster timely and robust HTA, ensure clinical decisions and coverage policies are well informed, and improve early patient access.
BACKGROUND:Commercial health plans may include corticosteroids in step therapy protocols for specialty drugs as part of use management. However, alignment with regulatory and clinical guidance is not well characterized. OBJECTIVE:To assess how corticosteroids are incorporated into plan-imposed step therapy protocols and evaluate alignment with US Food and Drug Administration (FDA) labeling, clinical guidelines, and pivotal trials. METHODS:We analyzed coverage policies from 18 large US commercial health plans in the Tufts Medical Center Specialty Drug Evidence and Coverage Database, reflecting policies active as of August 2024. We identified drug-indication pairs for which at least 1 plan included corticosteroids in step therapy. For each drug-indication pair, we reviewed FDA labeling to determine whether prior corticosteroid failure was consistent with FDA-recommended use. When corticosteroid use was not specified by FDA labels, we examined pivotal clinical trials and clinical guidelines to assess support for corticosteroid use in step therapy. We also examined how health plans operationalized corticosteroid use (eg, systemic vs local use, single vs multiple steps, etc). RESULTS:We identified 201 specialty drug-indication pairs and 2,767 corresponding coverage policies for which at least 1 plan included corticosteroids in step therapy. Of these drug-indication pairs, 33 (16.4%) had an FDA label indication recommending prior corticosteroids use, whereas 168 (83.6%) did not. Among 2,210 coverage policies for the 168 pairs without FDA label-indicated prior corticosteroid use, 952 (43.1%) included corticosteroids in step therapy protocols. In 21% (200/952) of these policies, corticosteroid use was a mandatory step, whereas the remaining 79% (752/952) allowed patients to meet the requirement by failing alternative agents (eg, immunomodulators). In total, 63.8% (607/952) specified the use of corticosteroids to be systemic. Few protocols defined dosage requirements (7.9%, 75/952) or treatment failure criteria (3.8%, 36/952). Among policies in which FDA labeling did not specify prior corticosteroid use, plans were significantly more likely to include corticosteroids in step therapy when corticosteroid use was an eligibility criterion in pivotal clinical trials (χ2 [1, N = 2,198] = 31.2; P < .001) or strongly recommended in clinical guidelines (χ2 [1, N = 1,434] = 128.0; P < .001). Inclusion also varied across plans, ranging from 15% to 74% of policies without FDA label-indicated corticosteroid use. CONCLUSIONS:Commercial health plans frequently include corticosteroids in step therapy, including when not specified in FDA labeling. Substantial variation in implementation highlights opportunities to improve transparency and consistency.
BACKGROUND:Understanding disease activity and therapy goals of patients with rheumatoid arthritis (RA) is important to provide patient-centered specialty pharmacy care and achieve positive therapy outcomes. The Routine Assessment of Patient Index Data 3 (RAPID3) is a validated metric to track patient-reported RA disease activity. Consistent and frequent monitoring of RAPID3 scores may provide clinically meaningful insights. OBJECTIVE:To describe the trajectory of RAPID3 scores over time and factors related to changes in RAPID3 scores among patients new to specialty medications. METHODS:Adult patients with RA who initiated specialty medications were included if they filled medications at least 3 times and had at least 2 RAPID3 scores documented in 6 months. Linear mixed effects regression models estimated trends in RAPID3 scores over time with respect to patient identifiers. Logistic regression models adjusted for baseline RAPID3 severity and estimated adjusted odds ratios (aOR) and associated 95% CIs of achieving a minimal clinically important improvement (≥3.8 points) or low severity/near remission (LS/NR) RAPID3 severity score at 6 months, by patient characteristics. RESULTS:Of 312 patients with RA, 195 (63%) had baseline high severity RAPID3 scores. Patients with baseline high severity achieved significant RAPID3 decreases over 6 months (5.9 points; P < 0.0001). Nearly half of patients (N = 147, 47%) had a clinically important improvement and 105 (34%) were LS/NR at follow-up; 67 patients (21%) met both criteria. After adjusting for baseline RAPID3 severity category, medication change (aOR, 0.39; 95% CI, 0.21-0.71) and opting out of full therapy management (aOR, 0.36; 95% CI, 0.19-0.65) were inversely associated with achieving a clinically important improvement. Being LS/NR at follow-up was also inversely associated with medication change (aOR, 0.19; 95% CI, 0.075-0.48) and opting out of full therapy management (aOR, 0.27; 95% CI, 0.12-0.63). Low medication adherence (aOR, 0.28; 95% CI, 0.11-0.72) and Medicaid insurance (aOR, 0.52; 95% CI, 0.29-0.93) were also inversely associated with being LS/NR at follow-up. CONCLUSIONS:RAPID3 is a valuable tool in the specialty pharmacy setting, helping assess patient outcomes beyond traditional metrics. Specialty pharmacies should support their patients in striving to reach clinically important improvement and/or achieving LS/NR as measures of quality pharmacy care during the crucial months after therapy initiation and throughout care. Active collaboration between patients and pharmacists is crucial in reaching positive RA disease activity outcomes. To support this, implementing programs that foster patient engagement with pharmacists may be important for optimizing care delivery.
BACKGROUND:Gaps often exist between a therapy's performance in a controlled research setting and outcomes in routine clinical practice. A prescription binocular treatment delivered via head-mounted display has demonstrated efficacy for improving visual acuity in children with amblyopia in a pivotal randomized controlled trial (RCT). A retrospective patient registry has been established to collect real-world data on the treatment. OBJECTIVE:To evaluate the real-world effectiveness of this treatment by benchmarking outcomes from a patient registry against those from the RCT. METHODS:A retrospective analysis of a multicenter US patient registry was completed. The Registry was queried for patients who matched key eligibility criteria of the RCT with respect to age, amblyopia type and severity, and prior treatment exposure. The primary outcome was the change in amblyopic eye best-corrected visual acuity from baseline to the 12-week follow-up visit. These outcomes were compared with the historical intent-to-treat cohort (n = 45) from the RCT using a two one-sided tests procedure for equivalence with an equivalence margin of ±0.75 lines. RESULTS:Among the 40 registry patients meeting query criteria, mean age was 5.7 ± 1.0 years, and 21/40 (53%) had a history of prior treatment. Amblyopic eye best-corrected visual acuity improved a mean 1.7 lines (95% CI = 1.2-2.2), consistent with statistical equivalence within the prespecified ±0.75-line margin compared with RCT participants (1.8 lines; 95% CI = 1.4-2.3; two one-sided tests P = 0.038). No adverse safety events were reported. CONCLUSIONS:Patients prescribed this treatment for amblyopia in clinical practice achieved visual acuity improvements consistent with those observed in the RCT, indicating that effectiveness generalizes to real-world settings.
BACKGROUND:As defined by the 21st Century Cures Act, patient experience data (PED) are data collected by any person with the purpose of providing information about patients' experiences with a disease or condition. To date, limited information is available on payer perspectives and the use of PED in formulary decision-making beyond patient-reported outcomes. OBJECTIVE:To assess payer decision-maker familiarity with and perspectives on PED and their use in formulary development and benefit design. METHODS:Informal interviews were conducted with payer decision-makers to guide the development of an online survey instrument. The survey was used to collect input from a broader population of payer decision-makers to understand their perspectives on and familiarity with PED, current uses of PED in their formulary decision-making processes, and barriers to incorporating PED. RESULTS:Interviews: Six individuals participated in the interviews, representing health plans (n = 4) and pharmacy benefit managers (n = 2). Participants reported varying levels of understanding of PED and its use in practice and shared mixed views regarding organizational openness to using PED and barriers to uptake. Survey: The survey was sent to 6,960 individuals, of whom 312 attempted the screener survey (4.5%). Of these, 60 respondents progressed to and completed the full survey. Respondents reported that the definition of PED from the 21st Century Cures Act "mostly" (n = 24, 40.0%) or "completely" (n = 28, 46.7%) aligned with their understanding. In an open-ended follow-up question, respondents frequently cited non-PED sources as PED, including health care provider feedback, and claims and utilization management data. Most organizations consider PED of low (n = 32; 53.3%) or medium (n = 22; 36.7%) importance. Respondents indicate they do not commonly incorporate PED into the formulary decision-making process today, with 51.7% (n = 31) and 36.7% (n = 22) of respondents reporting that they "sometimes" or "rarely" incorporate PED into decision-making processes. Perceived barriers to using PED in formulary decisions were noted to be "subjectivity of results" (n = 44; 73.3%), a "lack of scientific validity" (n = 38; 63.3%); and a "lack of standardized measures" (n = 35; 58.3%). CONCLUSIONS:Survey responses suggest understanding of PED varies and that payers place low value on PED relative to other information sources, collectively limiting the meaningful use of PED in decision-making. Results also show uptake is impacted by various perceived barriers. Additional training is required on the scientific merit of PED and the rigorous qualitative and quantitative methods used to engage the patient community to collect and leverage PED.
