Biosimilar underutilization alone does not foretell a broken biologics market.

Biosimilars offer the potential for cost savings and expanded access to biologic products; however, there are concerns regarding the rate of biosimilar uptake. We assessed the relationship between biosimilar and originator pricing, coverage, and market share by describing four case studies that fall into two categories: (1) sole preferred coverage strategy (ie, aim is to have originator product preferred; biosimilar(s) non-preferred), defined as steep average sales price (ASP) reductions for originator products (decline in net prices by at least 50% following the introduction of biosimilar competition by 2022) and (2) non-sole preferred coverage strategy (ie, aim is to have originator product preferred alongside biosimilar products), defined as moderate ASP reductions for originator products with (net prices did not decline by at least 50% of its pre-biosimilar competition value). We found that originators with sole preferred coverage strategies maintained formulary preference and market share relative to originators with non-sole preferred coverage strategies. Regardless of strategy, the market-weighted ASP for all four product families (originator and biosimilars) declined significantly in the years following the introduction of biosimilars, suggesting that biosimilar uptake alone may not be a complete measure of whether the biosimilar market is facilitating competition and lowering prices.

Balancing Evidence and Need: Variation in US Commercial Payer Coverage of Esketamine.

PURPOSE: Variations in US commercial health plan coverage policies affect how patients access medications. Plans may vary in treatment access criteria, line of therapy, and prescriber requirements. In this study, we examined coverage of esketamine hydrochloride (Spravato) for major depressive disorder (MDD) and treatment-resistant depression (TRD) to answer the following question: how do US commercial health plans cover esketamine, and how do they guide prompt patient access to the drug? METHODS: We used information from the Tufts Medical Center Specialty Drug Evidence and Coverage database, which includes coverage policies issued by 18 large commercial health plans in the United States. Esketamine coverage policies for MDD and TRD active in December 2022 were collated and analyzed. We compared coverage policies according to step therapy protocols, patient subgroup restrictions, and prescriber requirement criteria, evaluating patient access using the number of restrictions and proportion of plans including each criterion. FINDINGS: Plans more often imposed step therapy requirements for access to esketamine for TRD than for MDD, with line of treatment of ≤9 steps for MDD compared with 1 to 5 steps for TRD. Plans also varied with respect to the therapies they required patients to first try and experience treatment failure before granting access to esketamine for both indications. Clinical coverage requirements varied in thresholds and rating scales used to assess severity of depressive symptoms. IMPLICATIONS: Plans vary in terms of line of therapy and clinical coverage requirements for access to esketamine. Variation in health plan coverage policies may result in inequitable access and added complexity for patients and clinicians navigating care, which may delay access to urgent treatment. GOV IDENTIFIERS: Not applicable.

US commercial health plan coverage dynamics in pulmonary arterial hypertension therapies.

BACKGROUND: Health plan coverage is central to patient access to care, especially for rare, chronic diseases. For specialty drugs, coverage varies, resulting in barriers to access. Pulmonary arterial hypertension (PAH) is a rare, progressive, and fatal disease. Guidelines suggest starting or rapidly escalating to combination therapy with drugs of differing classes (phosphodiesterase 5 inhibitors [PDE5is], soluble guanylate cyclase stimulators [sGC stimulators], endothelin receptor antagonists [ERAs], and prostacyclin pathway agents [PPAs]). OBJECTIVE: To assess the variation in commercial health plan coverage for PAH treatments and how coverage has evolved. To examine the frequency of coverage updates and evidence cited in plan policies. METHODS: We used the Tufts Medical Center Specialty Drug Evidence and Coverage database, which includes publicly available specialty drug coverage policies. Overall, and at the drug and treatment class level, we identified plan-imposed coverage restrictions beyond the drug's US Food and Drug Administration label, including step therapy protocols, clinical restrictions (eg, disease severity), and prescriber specialty requirements. We analyzed variation in coverage restrictiveness and how coverage has changed over time. We determined how often plans update their policies. Finally, we categorized the cited evidence into 6 different types. RESULTS: Results reflected plan coverage policies for 13 PAH drugs active between August 2017 and August 2022 and issued by 17 large US commercial health plans, representing 70% of covered lives. Coverage restrictions varied mainly by step therapy protocols and prescriber restrictions. Seven plans had step therapy protocols for most drugs, 9 for at least one drug, and 1 had none. Ten plans required specialist (cardiologist or pulmonologist) prescribing for at least one drug, and 7 did not. Coverage restrictions increased over time: the proportion of policies with at least 1 restriction increased from 38% to 73%, and the proportion with step therapy protocols increased from 29% to 46%, with generics as the most common step. The proportion of policies with step therapy protocols increased for every therapy class with generic availability: 18% to 59% for ERAs, 33% to 77% for PDE5is, and 33% to 43% for PPAs. The proportion of policies with prescriber requirements increased from 24% to 48%. Plans updated their policies 58% of the time annually and most often cited the 2019 CHEST clinical guidelines, followed by randomized controlled trials. CONCLUSIONS: Plan use of coverage restrictions for PAH therapies increased over time and varied across both drugs and plans. Inconsistency among health plans may complicate patient access and reduce the proportion who can persist on PAH treatments.

Variation in Medicaid and commercial coverage of cell and gene therapies.

Growth in the availability of cell and gene therapies (CGTs) promises significant innovation in the treatment of serious diseases, but the high cost and one-time administration of CGTs has also raised concern about strain on health plan budgets and inequity in access. We used coverage information from the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) database for 18 large commercial health plans in the US and information from state Medicaid websites to examine variation in coverage of 11 CGTs in August 2021. We found that US commercial and Medicaid health plans imposed restrictions in 53.5 % and 68.3 % of their coverage policies for the 11 included CGTs, respectively. In addition, we identified significant variation in access to CGTs across commercial plans and across Medicaid plans. Coverage restrictions for certain CGTs were more common than others; clinical requirements were often (but not always) consistent with the inclusion criteria for the clinical trial central to the drug's approval. We conclude that there is variation in access to CGTs, creating differential patient access.

Varied use of step therapy among Medicare Advantage plans.

OBJECTIVES: First, to examine 7 large Medicare Advantage (MA) plans' use of step therapy. Second, to compare step therapy that health plans imposed in their MA and commercial (employer) drug coverage policies. STUDY DESIGN: Database analysis. METHODS: Using data from the Tufts Medical Center Specialty Drug Evidence and Coverage Database, we evaluated 7 large MA plans' use of step therapy in their Part B drug coverage policies. First, we determined the frequency with which different MA plans applied step therapy. Second, we determined the frequency with which plans imposed step therapy protocols across International Classification of Diseases, Tenth Revision, Clinical Modification categories. Third, we compared each step therapy protocol against each drug's corresponding FDA label indication. Fourth, we examined the consistency of step therapy protocols between the same insurer's MA and commercial lines of business. RESULTS: The frequency with which the included MA plans imposed step therapy ranged from 26.1% to 63.7%. Step therapy was most common for dermatology conditions (90.2%) and least common for oncology conditions (28.6%). On average, MA plans' step therapy requirements were consistent with the drug's FDA label indication 29.0% of the time. MA plans' and commercial plans' use of step therapy differed for the same drug-indication pairs 46.1% of the time. CONCLUSIONS: MA plans vary in the frequency with which they impose step therapy protocols in their Part B drug coverage policies. Moreover, insurers often impose different step therapy protocols in their MA plan and commercial plan offerings. Differences in plans' step therapy requirements may result in variability in patients' access to care within MA.

Improving Food and Drug Administration-Centers for Medicare and Medicaid Services Coordination for Drugs Granted Accelerated Approval.

Policy Points The increasing number of drugs granted accelerated approval by the Food and Drug Administration (FDA) has challenged the Medicare program, which often pays for expensive therapies despite substantial uncertainty about benefits and risks to Medicare beneficiaries. We recommend several administrative and legislative approaches for improving FDA-Centers for Medicare and Medicaid Services (CMS) coordination around accelerated-approval drugs, including promoting earlier discussions among the FDA, the CMS, and drug companies; strengthening Medicare's coverage with evidence development program; linking Medicare payment to evidence generation milestones; and ensuring that the CMS has adequate staffing and resources to evaluate new therapies. These activities can help improve the integrity; transparency; and efficiency of approval, coverage, and payment processes for drugs granted accelerated approval. CONTEXT: The Food and Drug Administration (FDA)'s accelerated-approval pathway expedites patient access to promising treatments. However, increasing use of this pathway has challenged the Medicare program, which often pays for expensive therapies despite substantial uncertainty about benefits and risks to Medicare beneficiaries. We examined approaches to improve coordination between the FDA and Centers for Medicare and Medicaid Services (CMS) for drugs granted accelerated approval. METHODS: We argue that policymakers have focused on expedited pathways at the FDA without sufficient attention to complementary policies at the CMS. Although differences between the FDA and CMS decisions are to be expected given the agencies' different missions and statutory obligations, procedural improvements can ensure that Medicare beneficiaries have timely access to novel therapies that are likely to improve health outcomes. To inform policy options and recommendations, we conducted semistructured interviews with stakeholders to capture diverse perspectives on the topic. FINDINGS: We recommend ten areas for consideration: clarifying the FDA's evidentiary standards; strengthening FDA authorities; promoting earlier discussions among the FDA, the CMS, and drug companies; improving Medicare's coverage with evidence development program; tying Medicare payment for accelerated-approval drugs to evidence generation milestones; issuing CMS guidance on real-world evidence; clarifying Medicare's "reasonable and necessary" criteria; adopting lessons from international regulatory-reimbursement harmonization efforts; ensuring that the CMS has adequate staffing and expertise; and emphasizing equity. CONCLUSIONS: Better coordination between the FDA and CMS could improve the transparency and predictability of drug approval and coverage around accelerated-approval drugs, with important implications for patient outcomes, health spending, and evidence generation processes. Improved coordination will require reforms at both the FDA and CMS, with special attention to honoring the agencies' distinct authorities. It will require administrative and legislative actions, new resources, and strong leadership at both agencies.

Follow-On Indications for Orphan Drugs Related to the Inflation Reduction Act.

This cohort study assesses rates at which orphan drugs approved by the US Food and Drug Administration were also approved for supplemental (follow-on) indications.

Specialty drug coverage varies between health plans' medical and pharmacy benefit policies.

BACKGROUND: In an effort to control drug spending, health plans are increasingly shifting specialty drugs from their medical benefit to the pharmacy benefit. One consequence of this trend is that some health plans have both a medical and a pharmacy coverage policy for the same drug. OBJECTIVE: To examine how frequently health plans issue medical and pharmacy benefit policies for the same specialty drug and to evaluate the concordance between plans' medical and pharmacy policies when plans issue both policy types. METHODS: We identified specialty drug coverage policies from the Tufts Medical Center Specialty Drug Evidence and Coverage Database, which includes policies issued by 17 of the largest US commercial health plans. Policies were current as of August 2020. We determined plans that issued both medical and pharmacy policies. Next, we identified drugs with "medical-pharmacy policy pairs," ie, drugs for which a plan issued both a medical and a pharmacy policy. For these pairs, we compared the plan's policies while accounting for the following coverage criteria: patient subgroups (patients must meet certain clinical criteria), prescriber requirements (a specialist must prescribe the drug), and step therapy protocols (patients must first fail alternative treatments). We considered medical-pharmacy policy pairs to be discordant if coverage criteria differed, eg, the medical policy included a prescriber requirement but the pharmacy policy did not. RESULTS: Eight plans issued separate medical and pharmacy benefit coverage policies for the same specialty drug and indication. Among these 8 plans, we identified 1,619 medical-pharmacy policy pairs. Eighty-six percent of pairs were concordant (1,386/1,619), and 14% were discordant (233/1,619). Discordance was most often due to differences in plans' application of step therapy protocols (184/233), followed by prescriber requirements (52/233) and patient subgroups (25/233). Forty pairs were discordant in multiple ways. Of discordant pairs, medical policies were more restrictive 41% (96/233) of the time; pharmacy policies were more restrictive 54% (125/233) of the time; 5% of the time (12/233), the medical policy was more restrictive in some ways, but the pharmacy policy was more restrictive in others. Overall, plans imposed coverage restrictions in their medical and pharmacy policies with similar frequencies. CONCLUSIONS: Commercial health plans' medical and pharmacy coverage policies for the same specialty drugs tended to be concordant, although we found coverage criteria to be discordant 14% of the time. Medical and pharmacy policies that are inconsistent in their coverage criteria and restrictions complicate, and potentially hinder, patients' access to specialty drugs. DISCLOSURES: Drs Kauf, O'Sullivan, and Strand were employees of Alkermes, Inc., when the study was conducted and may own stock in the company. Mx Levine, Mr Panzer, and Dr Chambers have no conflicts of interest to disclose. This research study was supported by Alkermes, Inc.

Factors Associated with Biosimilar Exclusions and Step Therapy Restrictions Among US Commercial Health Plans.

BACKGROUND: Biosimilars have been introduced with the goal of competing with high-priced biologic therapies, yet their adoption has been slower than expected and resulted in limited efficiency gains. We aimed to explore factors associated with biosimilar coverage relative to their reference products by commercial plans in the United States (US). METHODS AND DATA: We identified 1181 coverage decisions for 19 commercially available biosimilars, corresponding to 7 reference products and 28 indications from the Tufts Medical Center Specialty Drug Evidence and Coverage database. We also drew on the Tufts Medical Center Cost-Effectiveness Analysis Registry for cost-effectiveness evidence, and the Merative™ Micromedex® RED BOOK® for list prices. We summarized the coverage restrictiveness as a binary variable based on whether the product is covered by the health plan, and if covered, the difference of payers' line of therapy between the biosimilar and its reference product. We used a multivariate logistic regression to examine the association between coverage restrictiveness and a number of potential drivers of coverage. RESULTS: Compared with reference products, health plans imposed coverage exclusions or step therapy restrictions on biosimilars in 229 (19.4%) decisions. Plans were more likely to restrict biosimilar coverage for the pediatric population (odds ratio [OR] 11.558, 95% confidence interval [CI] 3.906-34.203), in diseases with US prevalence higher than 1,000,000 (OR 2.067, 95% CI 1.060-4.029), and if the plan did not contract with one of the three major pharmacy benefit managers (OR 1.683, 95% CI 1.129-2.507). Compared with the reference product, plans were less likely to impose restrictions on the biosimilar-indication pairs if the biosimilar was indicated for cancer treatments (OR 0.019, 95% CI 0.008-0.041), if the product was the first biosimilar (OR 0.225, 95% CI 0.118-0.429), if the biosimilar had two competitors (reference product included; OR 0.060, 95% CI 0.006-0.586), if the biosimilar could generate annual list price savings of more than $15,000 per patient (OR 0.171, 95% CI 0.057-0.514), if the biosimilar's reference product was restricted by the plan (OR 0.065, 95% CI 0.038-0.109), or if a cost-effectiveness measure was not available (OR 0.066, 95% CI 0.023-0.186). CONCLUSION: Our study offered novel insights on the factors associated with biosimilar coverage by commercial health plans in the US relative to their reference products. Cancer treatment, pediatric population, and coverage restriction of the reference products are some of the most significant factors that are associated with biosimilar coverage decisions.

Commercial health plans use of patient subgroup restrictions: An analysis of orphan and US Food and Drug Administration-expedited programs.

BACKGROUND: Health plans apply utilization management criteria to guide their enrollees' access to prescription drugs. Patient subgroup restrictions (ie, clinical prerequisites for drug coverage) are a form of utilization management that have not been thoroughly investigated. OBJECTIVE: To examine the frequency with which large US commercial health plans impose patient subgroup restrictions beyond the US Food and Drug Administration (FDA) label in their coverage policies for orphan drugs and for drugs included in 1 or more FDA-expedited programs. To determine how consistently these patient subgroup restrictions align with eligibility criteria specified in each drug's pivotal clinical trial(s). METHODS: The Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) database was used, which includes coverage policies issued by 17 large US commercial health plans. SPEC contained 3,786 orphan drug policies and 4,027 FDA-expedited drug policies (current as of December 2020). SPEC data on plans' patient subgroup restrictions were assessed for the first objective. Each patient subgroup restriction was benchmarked against the corresponding eligibility criteria for a drug's pivotal clinical trial(s) for the second objective. To do so, the "Clinical Studies" section of the drug's FDA label was reviewed or, if necessary, the published manuscript describing the drug's pivotal trial(s). Patient subgroup restrictions were categorized as follows: (1) "consistent," the restriction and trial eligibility criterion are equivalent; (2) "same measure, more stringent," the restriction and trial eligibility criteria depend on the same measure, but the plan coverage is more restrictive; (3) "same measure, less stringent," the restriction and trial eligibility criteria depend on the same measure, but the plan coverage is less restrictive; and (4) "not consistent," the restriction and trial eligibility criteria depend on different measures. RESULTS: Health plans imposed patient subgroup restrictions in 20.2% of orphan drug policies (frequency varied by health plan, 11.7%-36.6%), and in 21.8% of FDA-expedited drug policies (frequency varied by health plan, 11.1%-47.9%). Of the 936 patient subgroup restrictions in orphan drug policies, 60.3% were categorized as consistent; 7.3% as same measure, more stringent; 12.0% as same measure, less stringent; and 20.5% as not consistent. Of the 1,070 patient subgroup restrictions in FDA-expedited drug policies, 57.5% were categorized as consistent; 6.7% as same measure, more stringent; 16.0% as same measure, less stringent; and 19.8% as not consistent. CONCLUSIONS: Patient subgroup restrictions for orphan drugs and FDA-expedited programs varied substantially across health plans, potentially resulting in inconsistent access to a given therapy across the approved patient population. Patient subgroup restrictions tend to be consistent with eligibility criteria specified in pivotal clinical trials. DISCLOSURES: This study was funded by Sarepta Therapeutics, Inc. Alexa C Klimchak and Lauren E Sedita are employees of Sarepta Therapeutics, Inc., and may own stock/options in the company.

The association between US Food and Drug Administration-expedited review designations and health plan specialty drug coverage.

BACKGROUND: The US Food and Drug Administration (FDA) speeds approval of important clinical advancements through 4 expedited review programs: Priority Review, Accelerated Approval, Fast Track, and Breakthrough Therapy. Whether health plans prioritize coverage of expedited drugs relative to drugs that the FDA determined did not qualify from these programs is unclear. OBJECTIVES: To investigate how fast US commercial health plans issued coverage policies for drugs included in different numbers of FDA-expedited programs. Second, to examine the association between a drug's inclusion in an FDA-expedited program and plan coverage restrictiveness. METHODS: We used a separate dataset for each study objective. For the first objective, we created a dataset of policies issued by 17 large commercial health plans for 2018 FDA-approved drugs. Included policies were active exactly 1 year following each drug's FDA approval. We investigated the relationship between the speed of policy issuance and the number of expedited programs. We controlled for cancer and orphan indication. For the second objective, we analyzed a dataset of commercial health plan specialty drug coverage policies. We categorized drugs with respect to the number of expedited programs (0, 1, or 2+ programs). Coverage policies were categorized as whether plans imposed restrictions beyond a drug's FDA-approved labeling, for example, step therapy requirements. We used regression analysis to examine the association between FDA-expedited review and coverage restrictiveness when controlling for other relevant factors (eg, availability of alternatives). RESULTS: For our first objective, plans issued 62% (742/1,190) of policies within a year of a drug's FDA approval. In unadjusted analysis, policy issuance speed increased with each additional expedited program (hazard ratio=1.15; P<0.01). After controlling for cancer and orphan status, the number of expedited programs was not associated with faster policy issuance (hazard ratio=0.95; P=0.209). For our second objective, plans imposed coverage restrictions in 33% (672/2,027) of policies for drugs the FDA included in at least 1 FDA-expedited program vs 51% (870/1,706) of policies for drugs the FDA excluded from these programs. In multivariable regression, we did not find an association between FDA-expedited review and coverage restrictiveness after controlling for other decision-making factors (including disease prevalence, annual cost, etc). CONCLUSIONS: After controlling for other decision-making factors, we did not find that FDA-expedited approval was associated with faster coverage policy issuance, nor did we find that plans covered drugs the FDA included in expedited review programs less restrictively than drugs excluded from these programs. Our findings raise questions about why plans do not also accelerate access for these clinical advancements. DISCLOSURES: This study was funded by Janssen Scientific Affairs, LLC. Mr Ingham is an employee of Janssen Scientific Affairs, LLC, and is a stockholder of Johnson & Johnson. Dr Chambers reports grants from Janssen Scientific Affairs, LLC, during the conduct of the study.

Variation in Medicaid and commercial payer coverage of aducanumab for Alzheimer's disease.

INTRODUCTION: Ever since the United States Food and Drug Administration (FDA) approved aducanumab and Centers for Medicare & Medicaid Service (CMS) restricted coverage for the drug, a crucial question has been how other payers will behave. This study examined how Medicaid and commercial plans cover aducanumab. METHODS: We created a database of aducanumab coverage policies issued by Medicaid fee-for-service programs (50 states and DC) and 35 of the largest commercial plans (covering ∼ 84% of the commercially insured population). RESULTS: We found that only 41% of Medicaid fee-for-service plans have issued a publicly available coverage policy for aducanumab and that there is wide variation in these coverage criteria. Although the majority of included commercial plans have issued an aducanumab coverage policy, only five plans covered aducanumab for their enrollees. Available coverage polices showed little consistency in how to measure sufficient treatment response. DISCUSSION: Differences in coverage policies mean that Alzheimer's patients' access to aducanumab may vary across jurisdictions and across commercial insurers. HIGHLIGHTS: Less than half of state Medicaid fee-for-service plans issued a publicly available coverage policy for aducanumab. Available Medicaid coverage policies varied substantially in their coverage criteria. The majority of included commercial plans issued an aducanumab coverage policy; only five plans covered aducanumab. Available coverage polices showed little consistency in how to measure sufficient treatment response.

Examining US commercial health plans' use of The Institute for Clinical and Economic Review's reports in specialty drug coverage decisions.

BACKGROUND: The Institute for Clinical and Economic Review (ICER) has emerged in a visible role in US health care. However, it is unclear to what extent US commercial health plans use ICER value assessments in their specialty drug coverage decisions. OBJECTIVE: To evaluate the relationship between ICER's reported cost-effectiveness ratios (CERs) and coverage restrictiveness. Also, to examine the frequency with which plans have cited ICER in their coverage policies and to investigate how frequently health plans adjusted their drug coverage criteria in the 12 months after ICER's assessments. METHODS: We analyzed the Tufts Medical Center Specialty Drug Evidence and Coverage Database, which includes specialty drug coverage decisions issued by 17 large US commercial health plans. For ICER-assessed drugs, we recorded ICER's estimated CERs in the form of cost per quality-adjusted life-year (QALY) gained. First, we used multivariate logistic regression to examine the association between ICER's reported CERs and plan coverage restrictiveness, when controlling for other factors that were likely to affect decision-making. Next, we examined how often plans cited ICER's assessments in coverage decisions issued in years 2017-2020. Lastly, we examined whether plans added or removed coverage restrictions (eg, patient subgroup restrictions or step therapy protocols) in the 12 months following ICER's assessment. RESULTS: Plans tended to cover drugs with higher (less favorable) CERs more restrictively than drugs with CERs less than $100,000 per QALY: odds ratio (OR) = 4.48 if $100,000-$175,000 per QALY; OR = 2.00 if $175,000-$500,000 per QALY; and OR = 2.10 if $500,000 or more per QALY (all P < 0.01). Plans cited ICER in 0.8% (5/622) of coverage policies in 2017, 0.6% (5/833) in 2018, 1.7% (19/1,139) in 2019, and 2.4% (33/1,406) in 2020. For drugs with CERs less than $175,000 per QALY, plans adjusted coverage in 37% of cases: added restrictions in 20%, removed restrictions in 15%, and added one restriction but removed another in 2%. For drugs with CERs of $175,000 or more, plans changed coverage criteria in 29% of cases: added restrictions in 21%, removed restrictions in 5%, and added one restriction but removed another in 4%. CONCLUSIONS: We found that when controlling for other factors, health plans' specialty drug coverage decisions were associated with ICER's estimated CERs. Plans infrequently cited ICER value assessments. We did not observe a trend for plans more often narrowing coverage criteria for drugs with CERs $175,000 or more compared with drugs with CERs less than $175,000. DISCLOSURES: This research study was supported by a consortium of funders: Amgen, Genen-tech, Janssen Pharmaceuticals, Otsuka, and GSK.

Commercial coverage of specialty drugs, 2017-2021.

Health plans guide their enrollees' access to specialty drugs through coverage policies. We examined a set of health plan policies to determine if they have become more or less stringent over time. We did so by comparing the consistency of policies with Food and Drug Administration (FDA) label indications. We considered coverage policies for the same 187 specialty drugs issued by 17 large US commercial health plans from 2017 through 2021. Overall, the proportion of policies that were consistent with the FDA label declined from 57.1% in 2017 to 45.1% in 2021; the proportion of policies that were more restrictive than the FDA label increased from 39.5% to 51.7%. The proportion of policies excluding drug coverage remained approximately constant (3.4% in 2017; 3.2% in 2021). Trends in coverage restrictiveness varied across plans. For 13 plans, the proportion of policies with restrictions increased over time, while for 4 plans it declined.

State-Level Variation In Low-Value Care For Commercially Insured And Medicare Advantage Populations.

Low-value care is a major source of health care inefficiency in the US. Our analysis of 2009-19 administrative claims data from OptumLabs Data Warehouse found that low-value care and associated spending remain prevalent among commercially insured and Medicare Advantage enrollees. The aggregated prevalence of twenty-three low-value services was 1,920 per 100,000 eligible enrollees, which amounted to $3.7 billion in wasteful expenditures during the study period. State-level variation in spending was greater than variation in utilization, and much of the variation in spending was driven by differences in average procedure prices. If the average price for twenty-three low-value services among the top ten states in spending were set to the national average, their spending would decrease by 19.8 percent (from $735,000 to $590,000 per 100,000 eligible enrollees). State-level actions to improve the routine measurement and reporting of low-value care could identify sources of variation and help design state-specific policies that lead to better patient-centered outcomes, enhanced equity, and more efficient spending.

Characterizing health plan evidence review practices.

BACKGROUND: Health plans guide enrollees' access to specialty drugs through coverage policies. Practice guidelines recommend that the evidence supporting drug coverage policies should be comprehensive and routinely updated to reflect evidence-based medicine. OBJECTIVE: To examine the frequency with which health plans update their coverage criteria and supporting evidence and to determine the pattern with which plans cited relevant literature in their coverage policies. METHODS: Coverage policies from 17 large US commercial health plans were retrieved from the Tufts Medical Center Specialty Drug Evidence and Coverage database for August 2017 and August 2019. We identified drug-indication pairs (eg, infliximab for rheumatoid arthritis) for which plans had issued coverage policies in August 2017 and August 2019. We examined the frequency with which plans reissued these policies (ie, issued a new coverage document) between these 2 time points and the frequency with which plans altered coverage criteria or updated the cited evidence. A random sample of 20 drug-indication pairs was chosen to determine the comprehensiveness of cited evidence from the Specialty Drug Evidence and Coverage database. For each pair, a systematic literature search was conducted to identify relevant clinical and economic studies. A comparison of the systematic literature search with the evidence cited in each drug-indication pair's coverage policy was conducted to determine the comprehensive nature of each coverage policy's evidence. RESULTS: We identified 4,597 instances of plans issuing a coverage policy for the same drug-indication pair in both August 2017 and August 2019. Of those 4,597 instances, plans reissued an updated coverage document in 4,468 (97%). Fifteen percent of reissued policies revised both their coverage criteria and the evidence cited, 2% only their coverage criteria, 69% only the cited evidence, and 14% made no change. A total of 2,760 literature documents were identified relevant to at least one of the 20 drug-indication pairs, of which at least one plan cited 146 of these documents at least once (5.3%). Plans cited health technology assessments, randomized controlled trials (RCTs), systematic reviews/meta-analyses, and clinical guidelines most comprehensively. CONCLUSIONS: Health plans reissued most of their specialty drug coverage policies over a 2-year period. When plans revised their drug coverage criteria, they also tended to revise the evidence cited in their coverage polices. Of all the evidence found in our systematic review, plans more comprehensively cite health technology assessments, RCTs, and systematic reviews/meta-analyses. DISCLOSURES: This study was funded by the National Pharmaceutical Council.

Patients' access to 2018 FDA-approved drugs 1 year post approval.

OBJECTIVES: To examine US commercial health plans' adoption of 2018 FDA-approved drugs. STUDY DESIGN: Database analysis. METHODS: We identified novel drugs that the FDA approved in 2018 and categorized them as follows: cancer treatment, orphan drug, included in an expedited review program, and biosimilar. Using a data set of 17 large health plans' drug coverage policies and formularies, we examined coverage 1 year following FDA approval. RESULTS: The FDA approved 66 drugs in 2018 (5 were not yet marketed 1 year following approval). For 60 of 61 drugs, some plans issued coverage policies whereas other plans included the drug in their formularies. Plans imposed restrictions (eg, step therapy) in 37% (275/742) of coverage policies. Plans covered biosimilars, orphan drugs, and cancer treatments more generously than drugs not in those categories (P < .05). Plans imposed restrictions in their policies with different frequencies (range, 7%-52%). Plans imposed utilization management (UM) in 82% (3837/4697) of formulary entries. Of those entries, plans required prior authorizations in 98%, included drugs on the highest patient co-payment tier in 70%, and imposed step therapy in 3%. Plans most often placed orphan drugs and cancer treatments on the highest cost-sharing formulary tiers (68% and 64% of the time, respectively). Plans imposed UM in their formularies with different frequencies (range, 62%-100% of entries). CONCLUSIONS: Health plans imposed fewer coverage restrictions on cancer treatments, orphan drugs, and biosimilars than on drugs not in those categories. Some plans covered 2018 FDA-approved drugs more generously than others, which has implications for patients' access to innovative therapies.

Patients' access to rare neuromuscular disease therapies varies across US private insurers.

BACKGROUND: The extent to which different US private insurers require their enrollees to meet the same coverage criteria before gaining access to treatment is unclear. Our objective was to scrutinize the patient access criteria imposed by US private insurers for a set of rare neuromuscular disease (NMD) disease-modifying therapies (DMTs). METHODS: We examined coverage policies issued by 17 large US private insurers for the following NMD treatments: nusinersen and onasemnogene abeparvovec for spinal muscular atrophy, edaravone for amyotrophic lateral sclerosis, and eteplirsen for Duchenne muscular dystrophy. We reviewed the plans' coverage policies and identified the patient access criteria, including clinical prerequisites, step therapy protocols, and prescriber requirements. We compared the plans' patient access criteria with the therapies' US Food and Drug Administration (FDA)-labeled indications. RESULTS: The included insurers issued 65 coverage policies for the included therapies. Plans imposed coverage restrictions beyond the FDA-approved indications in 60 coverage policies; plans did not cover eteplirsen in five policies. No therapy was covered the same way by all insurers. Plans applied clinical criteria beyond the FDA label indication in 56 policies and step therapy protocols in three policies. Plans required that a neurologist prescribe the therapy in 37 policies, 22 of which required the neurologist to have expertise in the particular disease. Plans often required patients to suffer from symptoms of particular severity; e.g. for eteplirsen, plans differed in their 6-min walk test requirements; for edaravone, some plans required that patients had normal respiratory function, while others required only that patients did not require ventilation; for nusinersen and onasemnogene abeparvovec, plans differed in the number of SMN2 gene copies they required patients to have (SMN2 copy number is correlated with disease severity). CONCLUSIONS: The evaluated large US private insurers tended to impose coverage restrictions beyond the FDA label indication for the included set of rare NMD DMTs. Plans rarely applied the same patient access criteria in their coverage policies for the same products. Inconsistent coverage criteria mean that patients with different insurers have variable access to the same therapies across insurers.

Is an Orphan Drug's Cost-Effectiveness Associated with US Health Plan Coverage Restrictiveness?

BACKGROUND AND OBJECTIVES: Orphan drugs' high prices raise questions about whether their costs are worth their benefits. We examined the association between an orphan drug's cost-effectiveness and health plan coverage restrictiveness. METHODS: We analyzed a dataset of US commercial health plan coverage decisions (information current as of 2019) for orphan drugs (n = 3298). We used multi-level random-effect logistic regression to examine the association between orphan drug cost-effectiveness and coverage restrictiveness. We identified cost-effectiveness estimates from the Tufts Medical Center Cost-Effectiveness Analysis Registry, and from the Institute for Clinical and Economic Review's value assessments. We included only cost-effectiveness studies not funded by product manufacturers. We included the following independent variables: cancer indication, availability of alternatives, pediatric population, number of years since US Food and Drug Administration (FDA) approval, disease prevalence, annual cost, additional non-orphan indication, safety, and inclusion in a FDA expedited review program. RESULTS: Plans restricted drug coverage in 29.7% (n = 981) of decisions. Plans were more likely to restrict drugs with incremental cost-effectiveness ratios of $50,000-$175,000 per quality-adjusted life-year [QALY] (odds ratio = 1.855, p < 0.05), $175,000-$500,000 per QALY (odds ratio = 1.859, p < 0.05), and >$500,000 per QALY/dominated (odds ratio = 2.032, p < 0.01), compared to drugs with incremental cost-effectiveness ratios <$50,000 per QALY. Plans more often restricted drugs with non-cancer indications, having available alternatives, with more recent approval, in an FDA expedited review program, and for which the FDA additionally issued approval for a non-orphan disease. Plans more often restricted drugs with higher annual costs, and drugs indicated for higher prevalence diseases. All findings p < 0.05. CONCLUSIONS: Among other factors, an orphan drug's cost-effectiveness was associated with health plan drug coverage restrictiveness.

Are Medical Devices Cost-Effective?

OBJECTIVE: Medical devices can offer important therapeutic advances but, as for any medical interventions, there are questions about their costs and benefits. We examined health benefits and costs for pre-market approved (PMA) devices approved by the US Food and Drug Administration (FDA) (1999-2015), grouping them by generic category (e.g., drug-eluting stents) and indication. METHODS: We searched PubMed for incremental health gain estimates [measured in quality-adjusted life-years (QALYs)] and incremental costs for each device category compared to previously available treatments. We calculated incremental cost-effectiveness ratios by dividing the average incremental costs by the average incremental QALY gains. In sensitivity analysis, we repeated the analysis when excluding industry-funded studies. RESULTS: We identified at least one relevant cost-utility or comparative-effectiveness study for 88 devices (15.9% of non-cosmetic devices approved from 1999 to 2015), and at least one device across 53 (26.2%) generic categories. The median (mean) incremental cost across generic device categories was $1701 ($13,320). The median (mean) incremental health gain across generic device categories was 0.13 (0.46) QALYs. We found that cost-effectiveness ratios for 36 of 53 (68%) and 43 of 53 (81%) device categories fell below (were more favorable than) $50,000 and $150,000 per QALY, respectively. Results were roughly similar when we excluded industry-funded studies. CONCLUSIONS: We found that roughly one-quarter of the major PMA medical device categories have published cost-effectiveness evidence accessible through a large, publicly available database. Available evidence suggests that devices generally offer good value, as judged relative to established cost-effectiveness benchmarks.