An analysis of member retention patterns for adult rare disease cohorts to support evaluating multiyear payment arrangements for novel therapies.

BACKGROUND: Traditionally, treatment for chronic conditions addressed symptoms or was disease modifying and required lifelong periodic administration and recurring costs. Cell and gene therapies for rare diseases often require a short administration period relative to their expected long-term clinical benefit. Costs have historically been recognized when the service or treatment is administered, resulting in the potential for the cost associated with the possible long-term clinical benefit of cell and gene therapies being incurred during a short administration period. Innovative payment arrangements have been proposed to improve the synchronization of the payment and the emergence of the clinical benefit. Expected payments associated with a multiyear payment arrangement will depend on many factors, but key drivers of the payments include efficacy, durability of effect, mortality, and member retention. This research extends a previous study by analyzing member retention for adult patients with certain rare diseases. OBJECTIVE: To develop member retention estimates from a US commercial payer's perspective for adults diagnosed with certain rare diseases during a 10-year period. METHODS: Four population cohorts were examined: (1) self-insured - all subscribers, (2) self-insured - rare medical condition, (3) fully insured - all subscribers, and (4) fully insured - rare medical condition. Seven rare medical conditions were prospectively selected: cerebral palsy, cystic fibrosis, Gaucher disease, hemophilia, sickle cell disease, spina bifida, and thalassemia. We limited the study cohort to members who were either the subscriber or the subscriber's partner and were aged 18 years or older; dependent children were excluded from the analysis, regardless of age. The IBM MarketScan Commercial Claims and Encounters research database for the 10 years ending December 31, 2016, was used as the basis for the analysis. The analysis was completed using the lifetest procedure available in version 9.4 of the SAS Software System for Windows. The Kaplan-Meier method was used to produce retention rates. A log-rank test with chi-square statistic was used to determine statistically significant differences between pairs of curves. RESULTS: The study found that the subscriber retention for the rare medical condition cohort is significantly higher than the all-subscribers cohort by at least 12 points at each 1-year period. The finding was statistically significant (P < 0.0001) for the self-insured and fully insured cohorts. At year 5, approximately 20% more of the rare medical condition cohort was retained as compared with the all-subscribers cohort regardless of payer type. In addition, the study found that the probability of retention for adults with each rare medical condition in the rare disease cohort was also statistically significantly higher than all subscribers regardless of payer type. CONCLUSIONS: In multiyear payment arrangements, it may be important to set expectations for member retention based on studies specific to particular member cohorts. Health insurers and plan administrators may have inaccurate expectations if standard assumptions based on all member populations are used. This study found that adults diagnosed with 1 of 7 rare medical conditions are retained longer, on average, than all adult subscribers. DISCLOSURES: Milliman received funding from bluebird bio for the conduct of this study and fees from AveXis, outside the submitted work. Jackson, Runyan, and Metz are employed by Milliman. Jackson and Metz are members of the American Academy of Actuaries and meet the qualification standards for performing the analyses in this report. Kenney is an independent managed care consultant and received consulting fees from Milliman during the conduct of this study; Kenney also serves as preceptor for the Massachusetts College of Pharmacy and Health Sciences, is immediate past president of the Academy of Managed Care Pharmacy, and is a member of the Massachusetts Pharmacists Association Legislative Committee.

The Effect of a Pediatric Rare Disease on Subscriber Retention Rates for Commercial Health Insurers in the United States.

BACKGROUND: The advent of personalized medicine creates opportunities for regenerative therapies to deliver extended clinical benefit from a single administration. Policymakers and health insurers in the United States are evaluating coverage and payment arrangements for these therapies. One challenge involved in these evaluations is the perception that subscribers change insurers relatively often. However, the effect of pediatric rare diseases on retention rates for commercial health insurers has not been well studied. OBJECTIVE: To develop estimates for subscriber retention by a commercial insurer for up to 10 years. METHODS: Three population cohorts were examined: (1) all subscribers, (2) subscribers with any dependent aged 16 years or younger, and (3) subscribers with any dependent aged 16 years or younger diagnosed with a chronic rare disease that typically results in a debilitating state or high mortality, usually associated with high health care costs regardless of whether a treatment is available. The analysis was conducted for a sample of fully insured and self-insured group business within the commercial health insurance market for these study cohorts. The MarketScan Commercial Claims and Encounters research database covering the time period from January 1, 2007, through December 31, 2016, was used as the basis for the analysis. Subscribers were included in the family-based cohorts beginning with the first observed month with a dependent aged 16 years or younger and were retained in the analysis until the subscriber or insurer withdrew from the dataset (whichever came first). Subscribers were included in the family-based rare disease cohorts if their qualifying dependent was reported with at least 2 occurrences for any of the rare diseases studied. A Kaplan-Meier estimator was used to produce retention rates for all populations for up to 120 months. An adjustment for interval censoring was applied to the family-based cohorts. A log rank test with chi-square statistic was used to determine statistical significance. RESULTS: The analysis found that the subscriber retention rate within the self-insured groups was higher than within the fully insured groups (P < 0.0001). In addition, the probability of retaining subscribers with a dependent aged 16 years or younger compared with all subscribers was significantly greater (P < 0.0001). The analysis also found the probability of retaining subscribers with a qualifying dependent with a rare disease compared with subscribers with any dependent aged 16 years or younger was significantly greater (P < 0.0001). CONCLUSIONS: This study demonstrated that families with a child with a rare disease remained with their commercial health insurer longer than families who did not have a child with a rare disease. The analysis will be a useful resource when evaluating alternative payment arrangements and cost/benefit analyses of regenerative therapies that offer an extended duration of clinical benefit. DISCLOSURES: This study was sponsored by AveXis, which provided input into the study design, decided to submit the study results for publication, and performed an editorial review of the manuscript. Kuester, Jackson, and Runyan received consulting fees from AveXis during the conduct of this study. Pezalla and Nussbaum received consulting fees from Milliman during the conduct of this study. Nussbaum reports consulting fees from Sarepta Therapeutics and Ultragenyx Pharmaceutical outside of this study and serves on the Commercial Advisory Board of Gilead Sciences. A variation on this topic was presented at the Academy of Managed Care Pharmacy Nexus 2018; October 22-25, 2018; Orlando, FL.

Persistence of morbidity and cost differences between late-preterm and term infants during the first year of life.

BACKGROUND: Late-preterm infants are known to have greater morbidity and costs compared with term infants during the neonatal period, but less is known about whether these differences continue beyond this period. OBJECTIVE: The purpose of this study was to examine the most common causes and costs of rehospitalization and other health care use among late-preterm and term infants throughout the first year of life. METHODS: We conducted a retrospective cohort study of late-preterm (33-36 weeks' gestation) and term infants born in 2004 with > or =1 year of enrollment in a large national US database of commercially insured members. All of the reported health care services and costs were examined from the birth hospitalization through the first year of life. RESULTS: We evaluated 1683 late-preterm and 33 745 term infants. The average length of stay of the birth hospitalization for term infants was 2.2 days, and the average cost was $2061. Late-preterm infants had a substantially longer average stay of 8.8 days and average cost of $26 054. Total first-year costs after birth discharge were, on average, 3 times as high among late-preterm infants ($12 247) compared with term infants ($4069). Late-preterm infants were rehospitalized more often than term infants (15.2% vs 7.9%). A subset of late-preterm infants that were discharged late from their birth hospitalization had the highest rates of rehospitalization and total health care costs. Higher costs during rehospitalization of late-preterm infants, especially those with a late discharge, indicate their propensity to have more severe illness. CONCLUSIONS: Late-preterm infants have greater morbidity and total health care costs than term infants, and these differences persist throughout the first year of life. Management strategies and guidelines to reduce morbidity and costs in late-preterm infants should be investigated.

Medicare Part D formulary coverage since program inception: are beneficiaries choosing wisely?

OBJECTIVE: To evaluate how Medicare Part D formulary composition has changed since program inception, including comparison of plans eligible for full premium subsidy (ie, benchmark plans) with their counterparts. STUDY DESIGN AND METHODS: The study used publicly available data released by the Centers for Medicare & Medicaid Services to generate snapshots of formulary coverage and enrollment levels in each plan year. The analysis included all Part D plans and tracked formulary coverage of 152 of the most common brand name and generic drugs prescribed to seniors. RESULTS: Since 2006, the number of products available without restriction has increased and the number of drugs not on formulary has decreased. However, it appears that beneficiaries (subsidized beneficiaries in particular) may not be using their open-enrollment periods to reevaluate the available plan offerings. CONCLUSIONS: Beneficiaries need to reevaluate the Part D options available on an annual basis to maintain enrollment with the most appropriate plan available. Although all plans meet the proscribed formulary requirements, some plans offer richer drug coverage with more drugs available on an unrestricted basis. Benchmark plan status allows Part D plans to maintain or gain significant Medicare enrollment from year to year. Careful oversight should be provided to ensure that the level of formulary coverage offered at benchmark and other plans remains consistent.

The impact of olanzapine, risperidone, or haloperidol on the cost of schizophrenia care in a medicaid population.

OBJECTIVE: The objective of this study was to assess the impact of medication treatment on Medicaid costs for persons with schizophrenia. METHODS: Michigan Medicaid claims from January 1995 through September 1998 were analyzed for persons with schizophrenia diagnoses who initiated olanzapine (n = 458), risperidone (n = 481), or haloperidol (n = 252) treatment between January 1996 and September 1997. Total and component Medicaid payments were compared for the year after treatment initiation, with simultaneous adjustment for patient demographics, comorbid conditions, prior medication use, prior service use, and prior year costs. RESULTS: Significant baseline differences existed between the groups in prior medication and service use. Adherence to index medication varied between the groups (O = 60%; R = 54%; H = 37%; P < =.01 for each pairwise comparison). Average postperiod costs were US dollars 14512 per subject. After baseline adjustment, there were no significant differences in mean total cost. Excluding index medication costs, the olanzapine group's average cost was significantly lower than risperidone (-US dollars 1791, P =.002) and haloperidol (-US dollars 2080, P =.003), whereas the risperidone and haloperidol groups were not significantly different. The differences were driven by significantly lower cost for inpatient services for other medications among the olanzapine group. CONCLUSION: Total costs of schizophrenia care associated with olanzapine, risperidone, or haloperidol were similar, but component costs differed. Relative to risperidone or haloperidol, olanzapine may have a higher acquisition cost, but may decrease inpatient costs and be associated with more optimal medication use patterns. Use of risperidone may also increase pharmacy costs and be associated with greater persistence, relative to haloperidol.