Estimating the impact of biosimilar entry on prices and expenditures in rheumatoid arthritis: a case study of targeted immune modulators.

OBJECTIVE: To model changes in prices, utilization, and expenditures of targeted immune modulators (TIMs) for rheumatoid arthritis, accounting for biosimilar entry. METHODS: Using IQVIA National Sales Perspective data between 2013 and 2019, we examined sales and expenditures of biologics and non-biological complex molecules, 20 quarters before and after patent exclusivity milestones. We estimated the impact of a molecule's exclusivity milestones and biosimilar entry on prices, using a regression discontinuity design (RDD). We then combined the RDD estimate with historical trends to assess the impact of adalimumab's exclusivity milestones on future TIM expenditures. RESULTS: Changes in average molecule prices were associated largely with biosimilar uptake. For molecules with relatively high biosimilar uptake (>60%), prices fell considerably (-21.2% to -59.3%) one year after exclusivity milestones, whereas molecules with lower biosimilar uptake (<10%) experienced smaller price decreases (-2.4% to -8.4%). Average price reduction at the molecule level after biosimilar entry was not significant (-18.6%; p = .657). When applying the RDD results after adalimumab's exclusivity milestones, its projected share of total TIM market expenditures decreased from 48.0% in 2019 to 26.0% in 2025, whereas expenditures on Janus kinase inhibitors increased from 4.0% to 34.0%. CONCLUSIONS: Biologics facing biosimilar competition may experience price decreases, potentially offering substantial savings to payers, patients, and society, although the magnitude of these estimates depends on biosimilar uptake. Formulary placement, along with manufacturer-payer dynamics, may also play a role in determining the impact on price and market uptake of biosimilars.

Health care utilization and costs associated with functional status in patients with psoriatic arthritis.

BACKGROUND: The Health Assessment Questionnaire Disability Index (HAQ-DI) has been validated and widely used in psoriatic arthritis (PsA) clinical trials for the assessment of patient functional status. Significant improvements in the HAQ-DI have been reported in response to therapeutic interventions; however, few US studies have evaluated the economic impact of functional disability in patients with PsA. OBJECTIVE: To evaluate the association of functional status with health care resource utilization (HCRU) and total health care costs in US patients diagnosed with PsA. METHODS: This retrospective study included adult patients with PsA enrolled in FORWARD between July 2009 and June 2019 who completed 1 or more HAQ-DI questionnaires between January 2010 and December 2019. Patient demographics, clinical characteristics, and patient-reported outcomes were collected from the most recent questionnaire. HCRU and total health care costs (2019 US dollars) for all hospitalizations, emergency department (ED) visits, outpatient visits, diagnostic tests, and procedures were assessed for the 6 months prior to survey completion. Negative binomial regression models (HCRU outcomes) and generalized linear models with γ distribution and log-link function (cost outcomes) were used to assess the relationship between HAQ-DI and HCRU and cost outcomes, respectively. RESULTS: A total of 828 patients with PsA who completed HAQ-DI questionnaires were included. The mean (SD) age was 58.5 (13.5) years, 72.3% were female, and 92.3% were White. The mean (SD) disease duration was 17.5 (12.4) years, and the mean (SD) HAQ-DI score at the time of the patients' most recent questionnaire was 0.9 (0.7). More severe functional disability, measured by higher HAQ-DI score, was significantly associated with increased risk (incident rate ratio [95% CI]) of hospitalizations (1.68 [1.11-2.55]), ED visits (2.09 [1.47-2.96]), outpatient visits (1.14 [1.05-1.24]), and diagnostic tests (1.42 [1.16-1.74]). There was also a significant positive association between greater HAQ-DI score and increased total annualized health care costs (incremental amount [95% CI], 1.13 [1.03-1.23]) and medical costs (1.38 [1.13-1.69]), but there was no significant association found with pharmacy costs. Total adjusted average patient medical costs increased with increasing HAQ-DI score. CONCLUSIONS: Among patients with PsA enrolled in FORWARD, more functional disability-as measured by higher HAQ-DI scores-was associated with greater HCRU and increased total health care costs. These results suggest that improving functional status in patients with PsA may reduce economic burden for health care payers and systems. DISCLOSURES: Dr Ogdie has received consulting fees from Amgen, AbbVie, Bristol Myers Squibb, Celgene, CorEvitas (formerly Corrona), Gilead, Janssen, Lilly, Novartis, Pfizer, and UCB and has received grant support from the National Institutes of Health/National Institute of Arthritis and Musculoskeletal and Skin Diseases, Rheumatology Research Foundation, National Psoriasis Foundation, Pfizer (University of Pennsylvania), Amgen (FORWARD), and Novartis (FORWARD). Dr Hwang has received consulting fees from Novartis and UCB and has received grant support (5KL2TR003168-03) from the University of Texas Health Science Center at Houston Center of Clinical and Translational Sciences KL2 program. Drs Veeranki and Shafrin were employees of PRECISIONheor at the time of this analysis. Ms Portelli and Mr Sison are employees of PRECISIONheor. Ms Pedro has nothing to disclose. Dr Hass is an employee of H. E. Outcomes, providing consulting services to Novartis. Dr Hur was an employee of Novartis at the time of this analysis. Dr Kim was a postdoctoral fellow at the University of Texas at Austin and Baylor Scott and White Health, providing services to Novartis at the time of this analysis. Dr Yi is an employee of Novartis. Dr Michaud received grant funding from the Rheumatology Research Foundation at the time of this analysis. This study was funded by Novartis Pharmaceuticals Corporation, East Hanover, NJ.

Association of health care utilization and costs with patient-reported outcomes in patients with ankylosing spondylitis.

BACKGROUND: Interventions for ankylosing spondylitis (AS) have improved patient-reported outcomes (PROs) in clinical studies. However, limited data exist associating these improvements with health care resource utilization (HCRU) or cost savings. Few studies have evaluated the economic impact of patient-reported physical status and related disease burden in patients with AS in the United States. OBJECTIVE: To assess the association of PRO measures with HCRU and health care costs in patients with AS from a national US registry. METHODS: This cohort study included adults with a diagnosis of AS enrolled in the FORWARD registry from July 2009 to June 2019 who completed at least 1 questionnaire from January 2010 to December 2019 and completed the Health Assessment Questionnaire Disability Index (HAQ-DI) (0-3) and/or Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) (0-10). Patient-reported data for demographics, clinical characteristics, and PROs were collected through questionnaires administered biannually and reported from the most recent questionnaire. Patient-reported HCRU and total health care costs (2019 US dollars) for hospitalizations, emergency department (ED) visits, outpatient visits, diagnostic tests, and procedures were captured during the 6 months prior to the most recent survey completion. The relationship between HAQ-DI or BASDAI and HCRU outcomes was assessed using negative binomial regression models, and the relationship between HAQ-DI or BASDAI and the cost outcomes was evaluated using generalized linear models with γ distribution and log-link function. RESULTS: Overall, 334 patients with AS who completed the HAQ-DI (n = 253) or BASDAI (n = 81) were included. The mean (SD) HAQ-DI and BASDAI scores at the time of patients' most recent surveys were 0.9 (0.7) and 3.7 (2.3), respectively. HAQ-DI score was positively associated with number of hospitalizations, ED visits, outpatient visits, and diagnostic tests, whereas BASDAI was not associated with HCRU outcomes. Overall annualized mean (SD) total health care, medical, and pharmacy costs for patients with AS were $44,783 ($40,595); $6,521 ($12,733); and $38,263 ($40,595), respectively. Annualized total health care, medical, and pharmacy costs adjusted for confounders increased by 35%, 76%, and 26%, respectively, for each 1.0-unit increase in HAQ-DI score (coefficient [95% CI]: 1.35 [1.15-1.58], 1.76 [1.22-2.55]; both P < 0.01 and 1.26 [1.04-1.52]; P < 0.05, respectively); BASDAI score was not significantly associated with cost outcomes. CONCLUSIONS: Higher HAQ-DI scores were associated with higher HCRU and total health care costs among patients with AS in FORWARD, but BASDAI scores were not. These findings indicate that greater functional impairment may impose an increased economic burden compared with other patient-reported measures of AS. DISCLOSURES: A. Ogdie has received consulting fees from Amgen, AbbVie, Bristol Myers Squibb, Celgene, CorEvitas (formerly Corrona), Gilead, Janssen, Lilly, Novartis, Pfizer, and UCB and has received grant support from the National Institutes of Health/National Institute of Arthritis and Musculoskeletal and Skin Diseases, Rheumatology Research Foundation, National Psoriasis Foundation, Pfizer (University of Pennsylvania), Amgen (FORWARD), and Novartis (FORWARD). M. Hwang has received consulting fees from Novartis and UCB and has received grant support (5KL2TR003168-03) from the University of Texas Health Science Center at Houston Center of Clinical and Translational Sciences KL2 program. P. Veeranki and J. Shafrin were employees of PRECISION-heor at the time of this analysis. A. Portelli and S. Sison are employees of PRECISION-heor. S. Pedro does not have anything to disclose. N. Kim was a postdoctoral fellow at the University of Texas at Austin and Baylor Scott and White Health, providing services to Novartis at the time of this study. E. Yi is an employee of Novartis. K. Michaud received grant funding from the Rheumatology Research Foundation at the time of this analysis. This study was funded by Novartis Pharmaceuticals Corporation, East Hanover, NJ.

Patient and physician perspectives guiding intra-articular treatment choice in knee osteoarthritis: stakeholders are aligned on treatment priorities but have different assessments of treatment effect.

OBJECTIVES: Knee osteoarthritis (OA) is a leading cause of health-related disability. In the absence of curative non-operative therapies, treatment goals are limited to symptom relief. Data are limited on how patients and physicians prioritise available treatment options. We assessed patients' preferences for and physicians' attitudes towards intra-articular treatments including corticosteroids (IACS), an extended-release corticosteroid (TA-ER) and hyaluronic acids (IAHA). METHODS: We conducted a prospective, IRB-exempt, double-blind survey of patients with and providers who treat knee OA. Respondents were required to have received or prescribed TA-ER in a non-trial setting. We evaluated patients' OA history, impact of knee OA and treatment preferences, and physicians' decision-making and prescribing experiences. RESULTS: Of the 97 patient participants, mean age was 56 years, 70.0% were women, 75.0% had bilateral knee OA and 46.4% were diagnosed over 5 years ago. Of the 50 physician participants, 42.0% were orthopaedic surgeons, 34.0% were rheumatologists and 60.0%, on average, treat 50+ patients with knee OA per month. Treatment selection factors considered 'very important' to patients and physicians included disease severity (88.7%, 82.0%), impact on quality of life (88.7%, 72.0%), disease extent (84.5%, 54.0%) and activity level (80.4%, 64.0%). A majority (93.8%) of patients indicated moderate to severe difficulty with their knees. Fewer patients (76.3%) reported shared decision making compared with physicians (92.0%). Half (50.5%) of the patients reported that they experienced months of pain relief with TA-ER, 27.7% with IACS and 18.8% with IAHA. Physician assessments were consistent but estimated a greater duration of treatment effects than that reported by patients across all therapies. CONCLUSION: While knee OA has a tremendous impact on patients, there are significant unmet treatment needs. The increasing use of patient-reported outcomes will allow patients and physicians to track pain and functional status over time and across therapies, improving shared decision-making.