The Health Benefits, Costs, and Cost-Effectiveness of Ultraorphan Drugs.

OBJECTIVES: To examine ultraorphan drugs in terms of incremental health, costs, and cost-effectiveness compared with more prevalent disease drugs. METHODS: We identified Food and Drug Administration drug approvals from 1999 to 2019. For drugs approved for multiple indications, we considered each drug-indication pair separately. Utilizing Food and Drug Administration's orphan drug designation and US disease prevalence, we categorized drug-indication pairs as: ultraorphan (<10 000 patients), "other" orphan (≥10 000 and <200 000), and nonorphan (≥200 000). We searched the PubMed database for cost-effectiveness analyses and comparative effectiveness studies. We excluded manufacturer-funded studies. We extracted estimates of incremental health gains in terms of quality-adjusted life-years (QALYs) and incremental costs associated with drug-indication pairs compared with the standard of care at the time of their approval. We compared QALY gains, added costs, and incremental cost-effectiveness ratios (ICERs) using the Kruskal-Wallis, Mann-Whitney U (MWU), and Kolmogorov-Smirnov (KS) tests. RESULTS: Median incremental QALYs, costs, and ICERs differed across nonorphan, "other" orphan, and ultraorphan categories (Kruskal-Wallis P < .01). Compared with nonorphan drugs, ultraorphan drugs had larger QALY gains (0.700 vs 0.050, MWU P < .01, KS P < .01), larger costs ($172 231 vs $3360, MWU P < .01, KS P < .01), and larger ICERs ($1 216 184/QALY vs $114 061/QALY, MWU P < .01, KS P <.01). Compared with "other" orphan drugs, ultraorphan drugs had larger QALY gains (0.700 vs 0.310, MWU P =.65, KS P =.32), larger costs ($172 231 vs $69 308, MWU P = .03, KS P = .03), and larger ICERs ($1 216 184/QALY vs $223 472/QALY, MWU P <.01, KS P <.01). CONCLUSIONS: Novel ultraorphan drugs typically offer larger incremental health gains than drugs for more prevalent diseases, but because of their substantial added costs, are typically less cost-effective.

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.

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.

An Evidence Review of Low-Value Care Recommendations: Inconsistency and Lack of Economic Evidence Considered.

BACKGROUND: Low-value care, typically defined as health services that provide little or no benefit, has potential to cause harm, incur unnecessary costs, and waste limited resources. Although evidence-based guidelines identifying low-value care have increased, the guidelines differ in the type of evidence they cite to support recommendations against its routine use. OBJECTIVE: We examined the evidentiary rationale underlying recommendations against low-value interventions. DESIGN: We identified 1167 "low-value care" recommendations across five US organizations: the US Preventive Services Task Force (USPSTF), the "Choosing Wisely" Initiative, American College of Physicians (ACP), American College of Cardiology/American Heart Association (ACC/AHA), and American Society of Clinical Oncology (ASCO). For each recommendation, we classified the reported evidentiary rationale into five groups: (1) low economic value; (2) no net clinical benefit; (3) little or no absolute clinical benefit; (4) insufficient evidence; (5) no reason mentioned. We further investigated whether any cited or otherwise available cost-effectiveness evidence was consistent with conventional low economic value benchmarks (e.g., exceeding $100,000 per quality-adjusted life-year). RESULTS: Of the identified low-value care recommendations, Choosing Wisely contributed the most (N=582, 50%), followed by ACC/AHA (N=250, 21%). The services deemed "low value" differed substantially across organizations. "No net clinical benefit" (N=428, 37%) and "little or no clinical benefit" (N=296, 25%) were the most commonly reported reasons for classifying an intervention as low value. Consideration of economic value was less frequently reported (N=171, 15%). When relevant cost-effectiveness studies were available, their results were mostly consistent with low-value care recommendations. CONCLUSIONS: Our study found that evidentiary rationales for low-value care vary substantially, with most recommendations relying on clinical evidence. Broadening the evidence base to incorporate cost-effectiveness evidence can help refine the definition of "low-value" care to reflect whether an intervention's costs are worth the benefits. Developing a consensus grading structure on the strength and evidentiary rationale may help improve de-implementation efforts for low-value care.

Orphan Drugs Offer Larger Health Gains but Less Favorable Cost-effectiveness than Non-orphan Drugs.

BACKGROUND: Orphan drugs offer important therapeutic options to patients suffering from rare conditions, but are typically considerably more expensive than non-orphan drugs, leading to questions about their cost-effectiveness. OBJECTIVE: To compare the value of orphan and non-orphan drugs approved by the FDA from 1999 through 2015. DESIGN: We searched the PubMed database to identify estimates of incremental health gains (measured in quality-adjusted life-years, or QALYs) and incremental costs that were associated with orphan and non-orphan drugs compared with preexisting care. We excluded pharmaceutical industry-funded studies from the dataset. When a drug was approved for multiple indications, we considered each drug-indication pair separately. We then compared incremental QALY gains, incremental costs, and incremental cost-effectiveness ratios for orphan and non-orphan drugs using the Mann-Whitney U (MWU) test (to compare median values of the different distributions) and the Kolmogorov-Smirnov (KS) test (to compare the shape of different distributions). RESULTS: We identified estimates for 49 orphan drug-indication pairs, and for 169 non-orphan drug-indication pairs. We found that orphan drug-indication pairs offered larger median incremental health gains than non-orphan drug-indication pairs (0.25 vs. 0.05 QALYs; MWU p = 0.0093, KS p = 0.02), but were associated with substantially higher costs ($47,652 vs. $2870; MWU p < 0.001, KS p < 0.001) and less favorable cost-effectiveness ($276,288 vs. $100,360 per QALY gained; MWU p = 0.0068, KS p = 0.009). CONCLUSIONS: Our study suggests that orphan drugs often offer larger health gains than non-orphan drugs, but due to their substantially higher costs they tend to be less cost-effective than non-orphan drugs. Our findings highlight the challenge faced by health care payers to provide patients appropriate access to orphan drugs while achieving value from drug spending.

What types of real-world evidence studies do U.S. commercial health plans cite in their specialty drug coverage decisions?

PURPOSE: To examine the RWE U.S. commercial health plans cite in their specialty drug coverage decisions. METHODS: We used the Tufts Medical Center Specialty Drug Evidence and Coverage Database to identify specialty drug coverage decisions (n = 7267) issued by 17 large commercial health plans. We categorized the clinical evidence plans cited in these coverage decisions (n = 5227) as randomized controlled trials (RCTs), RWE studies, and other clinical studies (studies other than RCT or RWE study). We categorized RWE studies with respect to study type, for example, case series, studies based on medical records, and so on. We compared the frequency that plans cited different categories of RWE, cited RWE for different diseases, and cited RWE for drugs on the market for different time periods. RESULTS: RWE comprised 16% of cited clinical studies. Health plans cited RWE with different frequencies (5%-31% of the cited clinical evidence). Overall, plans cited RWE categorized as medical records most often (26% of cited RWE studies). Plans varied in the frequency they cited different RWE categories. Plans most frequently cited RWE for gastroenterological diseases (35% of clinical study citations) and least frequently for respiratory diseases (11% of clinical study citations). Plans cited RWE more for drugs that have long been on the market. CONCLUSIONS: Health plans varied with respect to the number and types of RWE studies they cited in their specialty drug coverage decisions. Plans cited RWE more often for some diseases than others, and cited more RWE for older drugs.

A Review of Empirical Analyses of Disinvestment Initiatives.

BACKGROUND: Disinvesting in low-value health care services provides opportunities for investment in higher value care and thus an increase in health care efficiency. OBJECTIVES: To identify international experience with disinvestment initiatives and to review empirical analyses of disinvestment initiatives. METHODS: We performed a literature search using the PubMed database to identify international experience with disinvestment initiatives. We also reviewed empirical analyses of disinvestment initiatives. RESULTS: We identified 26 unique disinvestment initiatives implemented across 11 countries. Nineteen addressed multiple intervention types, six addressed only drugs, and one addressed only devices. We reviewed 18 empirical analyses of disinvestment initiatives: 7 reported that the initiative was successful, 8 reported that the initiative was unsuccessful, and 3 reported that findings were mixed; that is, the study considered multiple services and reported a decrease in the use of some but not others. Thirty-seven low-value services were evaluated across the 18 empirical analyses, for 14 (38%) of which the disinvestment initiative led to a decline in use. Six of the seven studies that reported the disinvestment initiative to be successful included an attempt to promote the disinvestment initiative among participating clinicians. CONCLUSIONS: The success of disinvestment initiatives has been mixed, with fewer than half the identified empirical studies reporting that use of the low-value service was reduced. Our findings suggest that promotion of the disinvestment initiative among clinicians is a key component to the success of the disinvestment initiative.

EXAMINING EVIDENCE IN U.S. PAYER COVERAGE POLICIES FOR MULTI-GENE PANELS AND SEQUENCING TESTS.

OBJECTIVES: The aim of this study was to examine the evidence payers cited in their coverage policies for multi-gene panels and sequencing tests (panels), and to compare these findings with the evidence payers cited in their coverage policies for other types of medical interventions. METHODS: We used the University of California at San Francisco TRANSPERS Payer Coverage Registry to identify coverage policies for panels issued by five of the largest US private payers. We reviewed each policy and categorized the evidence cited within as: clinical studies, systematic reviews, technology assessments, cost-effectiveness analyses (CEAs), budget impact studies, and clinical guidelines. We compared the evidence cited in these coverage policies for panels with the evidence cited in policies for other intervention types (pharmaceuticals, medical devices, diagnostic tests and imaging, and surgical interventions) as reported in a previous study. RESULTS: Fifty-five coverage policies for panels were included. On average, payers cited clinical guidelines in 84 percent of their coverage policies (range, 73-100 percent), clinical studies in 69 percent (50-87 percent), technology assessments 47 percent (33-86 percent), systematic reviews or meta-analyses 31 percent (7-71 percent), and CEAs 5 percent (0-7 percent). No payers cited budget impact studies in their policies. Payers less often cited clinical studies, systematic reviews, technology assessments, and CEAs in their coverage policies for panels than in their policies for other intervention types. Payers cited clinical guidelines in a comparable proportion of policies for panels and other technology types. CONCLUSIONS: Payers in our sample less often cited clinical studies and other evidence types in their coverage policies for panels than they did in their coverage policies for other types of medical interventions.

Thirty Years of Media Coverage on High Drug Prices in the United States--A Never-Ending Story or a Time for Change?

In recent years drug prices have increasingly become a topic of debate for patients, providers, payers and policy makers. To place the current drug price debate into historical context, we searched the New York Times and Wall Street Journal from 1985 - 2015 and found that concerns about drug prices have commonly featured in the press over the study period with recently stronger calls for change. Price levels, types of innovations, stakeholder responses, and strategies to address high prices discussed in the media suggest that concerted efforts are required to enable affordable and high-value innovations.

Medicare's enduring struggle to define "reasonable and necessary" care.

Medicare legislation mandates that the program not pay for items and services that are not "reasonable and necessary" - terms that are open to interpretation and easily politicized. In today's fiscal environment, "reasonable and necessary" warrants a closer look.

CHANGING FACE OF MEDICARE'S NATIONAL COVERAGE DETERMINATIONS FOR TECHNOLOGY.

OBJECTIVES: The Centers for Medicare and Medicaid Services (CMS) issues National Coverage Determinations (NCDs) for medical interventions expected to have a significant impact on Medicare, the health insurance program for US citizens aged 65 years and older and certain people with disabilities under the age of 65 years. The objective of this study was to evaluate NCDs issued from 1999 to 2013 to identify key trends, and to discuss implications for future CMS policy. METHODS: We used the Tufts Medical Center Medicare National Coverage Determination Database to examine characteristics of NCDs from 1999 through 2013. We examined various characteristics of NCDs, including: whether the intervention under review is used for prevention or treatment of disease, the type of intervention considered, evidence limitations cited by CMS, and coverage determination outcome. We evaluated longitudinal trends in categorical and continuous variables in the database, using Cochran-Armitage trend tests and linear regression, respectively. RESULTS: We found that NCDs increasingly focus on preventive care (p = 0.072), pertain to diagnostic imaging (p = 0.033), and evaluate health education/behavioral therapy interventions (p = 0.051). CMS increasingly cites the lack of relevant outcomes (p = 0.019) and the lack of applicability of study results to the Medicare population (p < 0.001) as evidence limitations. CMS less often restricts coverage to certain population subgroups in NCDs (p < 0.001), but increasingly applies coverage with evidence development policies (p < 0.001). CONCLUSIONS: Identified trends reflect broader changes in Medicare as CMS shifts its focus from treatment to prevention of disease, addresses potentially overutilized technologies, and attempts to issue flexible coverage policies.

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 Plans Increase Choice Of Biosimilar And Originator Products; Market Net Prices Decrease.

Biosimilars drugs are almost identical copies of original biologic products. Early biosimilars had slower adoption and savings than expected; however, biosimilars launched in recent years have had more success. With several biosimilar launches planned in the next few years, it is important to understand how the state of the market might foretell significant market savings in the future. To do so, we explored how the introduction of biosimilars affected originator-biosimilar markets during the period 2017-22. We found that after biosimilar availability, payers increasingly allowed choice of preferred products. By 2022, 76 percent of commercial payers' coverage policies listed two or more products (originator or biosimilar) as first-line options. Biosimilar market shares exceeded those of originators a mean of three years after first biosimilar launch, and originator-biosimilar market average sales price declined substantially. Taken together, these findings provide evidence of a functioning competitive market.

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.

Are Drug Novelty Characteristics Associated With Greater Health Benefits?

OBJECTIVE: The aim of this study was to examine the association between characteristics of novel drugs and incremental health gains relative to standard of care, in terms of quality-adjusted life-years (QALYs). METHODS: This study's unit of analysis is the drug-indication pair. For pairs approved by the US FDA from 1999 to 2018, we quantified incremental health gains using QALYs from the published literature and characterized each pair's novelty in terms of a series of six binary (yes/no) characteristics of novel drugs given special consideration by Health Technology Assessment agencies: Novel mechanism of action, Indicated for a rare disease, Indicated for a pediatric population, Treats a serious condition, Offers meaningful improvement over available therapies, and Potential to address unmet clinical needs. We analyzed measures of bivariate association (Mann-Whitney U and Kolmogorov-Smirnov tests) and multivariable regression, accounting for the influence of multiple novelty characteristics simultaneously. RESULTS: Our sample of 146 drugs represents 21% of drugs approved the FDA in the time period (1999-2018). Median and mean QALY gains for 'novel' drug-indication pairs exceeded corresponding QALY gains for non-novel drug-indication pairs. For most comparisons, the bivariate relationships between QALY gains and novelty characteristics were significant at p < 0.05 except for novel mechanism of action (Kolmogorov-Smirnov test) and pediatric indication (both bivariate tests). Multivariable models revealed an independent association between novelty characteristics and QALY gain except for unmet clinical need and indicated for a rare disease. CONCLUSIONS: Drugs with novelty characteristics conferred larger health gains than drugs without these characteristics in bivariate analysis, multivariable models, or both. Future research should examine other aspects of drug novelty, such as patient and health system costs and equitable access.

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.

Illustrating potential efficiency gains from using cost-effectiveness evidence to reallocate Medicare expenditures.

OBJECTIVES: The Centers for Medicare & Medicaid Services does not explicitly use cost-effectiveness information in national coverage determinations. The objective of this study was to illustrate potential efficiency gains from reallocating Medicare expenditures by using cost-effectiveness information, and the consequences for health gains among Medicare beneficiaries. METHODS: We included national coverage determinations from 1999 through 2007. Estimates of cost-effectiveness were identified through a literature review. For coverage decisions with an associated cost-effectiveness estimate, we estimated utilization and size of the "unserved" eligible population by using a Medicare claims database (2007) and diagnostic and reimbursement codes. Technology costs originated from the cost-effectiveness literature or were estimated by using reimbursement codes. We illustrated potential aggregate health gains from increasing utilization of dominant interventions (i.e., cost saving and health increasing) and from reallocating expenditures by decreasing investment in cost-ineffective interventions and increasing investment in relatively cost-effective interventions. RESULTS: Complete information was available for 36 interventions. Increasing investment in dominant interventions alone led to an increase of 270,000 quality-adjusted life-years (QALYs) and savings of $12.9 billion. Reallocation of a broader array of interventions yielded an additional 1.8 million QALYs, approximately 0.17 QALYs per affected Medicare beneficiary. Compared with the distribution of resources prior to reallocation, following reallocation a greater proportion was directed to oncology, diagnostic imaging/tests, and the most prevalent diseases. A smaller proportion of resources went to cardiology, treatments (including drugs, surgeries, and medical devices, as opposed to nontreatments such as preventive services), and the least prevalent diseases. CONCLUSIONS: Using cost-effectiveness information has the potential to increase the aggregate health of Medicare beneficiaries while maintaining existing spending levels.

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.

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.