Drug Coverage Decisions Research Articles

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.

Appraising Drugs Based on Cost-effectiveness and Severity of Disease in Norwegian Drug Coverage Decisions

Rising health care costs are a major health policy challenge globally. Norway has implemented a priority-setting system intended to balance cost-effectiveness and concerns for fair distribution, but little is known about this strategy and whether it works in practice. To present and evaluate a systematic drug appraisal method that uses the severity of disease to account for a fair distribution of health in cost-effectiveness analysis, forming the basis for price negotiations and coverage decisions. This cross-sectional study uses confidential drug price information and publicly available data from health technology assessments and logistic and linear regression analyses to evaluate drug coverage decisions for the Norwegian specialized health care sector from 2014 to 2019. Drug coverage decisions by Norwegian authorities and incremental cost-effectiveness and severity of disease measured as absolute shortfall of quality adjusted life years. Between 2014 and 2019, a total of 188 drugs were appraised, of which 113 were cancer drugs. The overall coverage rate was 73% (138 of 188). The number of annual appraisals increased during the observation period. Based on 83 chosen decisions, regression analysis showed that incremental cost-effectiveness ratios (ICER) based on negotiated drug prices, adjusted for severity-differentiated cost-effectiveness thresholds, was the variable that best projected drug approvals (OR, 0.60; 95% CI, 0.42-0.86). An increase in the ICER by $10 000 was associated with a reduction in the odds for approval of 40% for drugs assessed from 2018 to 2019. This cross-sectional study demonstrated how concerns for efficiency and fair distribution of health can be implemented systematically into drug appraisals and reimbursement decisions. New, expensive drugs are expected to escalate health care costs in the years to come, and it may be feasible to control costs by negotiating the prices of new drugs while appraising both their cost-effectiveness and how their health benefits are distributed.

Use of prescriber requirements among US commercial health plans.

BACKGROUND: Prescriber requirements are a form of utilization management (UM) in which health plans require that a certain type of physician (eg, a rheumatologist) prescribe a drug. OBJECTIVE: To examine how a set of US commercial health plans impose prescriber requirements in their specialty drug coverage decisions. METHODS: We identified specialty drug coverage decisions from the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) Database. SPEC includes coverage information issued by 17 large US commercial health plans. We categorized prescriber requirements as the following: (1) the drug must be prescribed in consultation, supervision, or coordination with a specialist; (2) the drug must be prescribed by a specialist (eg, a neurologist); or (3) the drug must be prescribed by a specialist with particular expertise (eg, a neurologist with expertise in spinal muscular atrophy). First, we examined how often each plan imposed prescriber requirements. Second, we determined the degree of specialization that plans required prescribing physicians to have. Third, we used Pearson's chi-square tests to examine the association between plans' use of prescriber requirements and the following drug characteristics: cancer treatment, orphan indication, pediatric indication, drug approved in the last year, black box warning, and self-administered formulation. RESULTS: Overall, health plans imposed prescriber requirements in 22.0% (1,844/8,383) of their coverage decisions, although the frequency that they did so varied (range: 0.8%-86.0%). Of prescriber requirements, 79.1% (1,459/1,844) required that the drug be prescribed in consultation, supervision, or coordination with a specialist; 18.3% (338/1,844) required that the drug be prescribed by a specialist; and 2.6% (47/1,844) required that the drug be prescribed by a specialist with particular expertise. Plans were more likely to impose prescriber requirements for drugs with the following characteristics: indicated for a pediatric population, black box warning, self-administered, and noncancer treatments (all P < 0.001). CONCLUSIONS: Health plans varied in the frequency that they imposed prescriber requirements in their specialty drug coverage decisions and with respect to the degree of specialization they required prescribing physicians to have. DISCLOSURES: This study was funded by the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center. The center receives funding from a variety of sources, including government agencies, foundations, and pharmaceutical and device companies. The SPEC Database, which provided data for this study, is funded in part through a data subscription program to which a number of pharmaceutical companies subscribe. All authors are employed by the Center for the Evaluation of Value and Risk in Health. In addition, Chambers reports speaker fees from Astellas and consulting fees from Biogen and Lundbeck. The other authors have nothing to disclose. An earlier version of this study was presented as a poster at the AMCP 2021 Virtual Meeting, April 12-16, 2021.

PMU34 The Influence of Expedited Approval Designations on Health PLAN Coverage: An Empirical Evaluation

FDA expedites the review of drugs that address unmet clinical needs through four programs: priority review, accelerated approval, breakthrough therapy, and fast-track. These programs aim to speed patient access to substantial clinical advances. We examined whether health plans prioritize access to FDA expedited drugs. We used the Tufts Medical Center Specialty Drug and Evidence Coverage database, which contains information on specialty drug coverage decisions issued by 17 large US commercial health plans and details coverage restrictions (e.g., patient subgroups, step therapy protocols). We included drugs that FDA approved after January 1, 2013 to coincide with implementation of the breakthrough therapy designation program. We categorized drugs as included in no FDA expedited program, one program, or multiple programs. Our dichotomous dependent variable was: coverage with no restrictions vs. coverage with restrictions. We controlled for these drug characteristics: cancer treatment; drug type (biosimilar or otherwise); availability of alternatives; pediatric indication; years since FDA approval; administration (self- or physician-administered); annual cost; and disease prevalence. We analyzed the data using univariate analysis and multivariable logistic regression. We included 3,733 coverage decisions (current as of August, 2020). Univariate analysis found that plans were less likely to restrict coverage of drugs included in an expedited program (one program; OR=0.54, p<.01; multiple programs OR=0.46, p<.01). Multivariable regression analysis found that plans were neither more nor less likely to restrict coverage of drugs included in an expedited program (one program; OR=1.11, p=0.43; multiple programs OR=0.95, p=0.63). Plans were more likely to restrict a drug’s coverage (p<0.05) if it was not a cancer treatment, not a biosimilar, more recently approved, physician administered, had a higher annual cost, or treated a highly prevalent disease. When controlling for other characteristics, we found no association between a drug’s inclusion in an FDA expedited program and health plan coverage restrictiveness.

PNS52 Examining US Commercial Health Plans' Use of Step Therapy Protocols for Specialty Drugs: An Empirical Analysis

Step therapy protocols require patients to first fail an alternative cheaper treatment before gaining access to a more expensive drug. Health plans use step therapy protocols to optimize prescribing, but there is a risk that their inappropriate application can impede patients’ access to much needed treatments. We assessed a set of US commercial health plans’ use of step therapy protocols in their specialty drug coverage decisions.

Cost-effectiveness of cancer drugs: Comparative analysis of the United States and England.

BackgroundEngland's National Institute for Health and Care Excellence (NICE) and the US’ Institute for Clinical and Economic Review (ICER) both conduct cost-effectiveness evaluations for new cancer drugs to help payers make drug coverage decisions. However, NICE and ICER assessments have been noted to reach different conclusions. We aim to better understand the degree to which their recommendations diverge and what drives these apparent differences.MethodsWe compared the methods and results of publicly available cost-effectiveness evaluations performed by ICER and NICE of similarly assessed cancer drugs. Assessments were compared based on incremental cost-effectiveness ratio, comparator treatment, price, recommendation, and the design of the economic evaluation.FindingsAmong 11 commonly assessed cancer drugs, ICER and NICE were in concordance for 7 evaluations and in discordance on the cost-effectiveness and coverage decisions for 4 drugs. Most new cancer drugs were not cost-effective in either the US (7/11) or England (7/11). Furthermore, NICE's capacity to negotiate price discounts and access schemes result in much lower cost per QALY valuations and more favourable recommendations than those of ICER for similarly assessed cancer drugs.InterpretationsNICE and ICER employ similar health technology assessment (HTA) methodologies and were aligned with most recommendations, finding that many new and expensive cancer drugs are cost ineffective. Growing use of ICER assessments will continue to send stronger price signals to manufacturers that cancer drugs with low value for money will be viewed less favourably by private insurers. NICE provides an important reminder of how much lower other countries pay for drugs when comparative effectiveness and value-based pricing are integrated into public drug coverage decisions.

Ethical and Social Values for Paediatric Health Technology Assessment and Drug Policy.

Background: Public policy approaches to funding paediatric medicines in advanced health systems remain understudied. In particular, the ethical and social values dimensions of health technology assessment (HTA) and drug coverage decisions for children have received almost no attention in research or policy. Methods: To elicit and understand the social values that influence decision-making for public funding of paediatric drugs, we undertook a series of in-depth, semi-structured interviews with a stratified purposive sample (n = 22) of stakeholders involved with or affected by drug funding decisions for children at the provincial (Ontario) and national levels in Canada. Constructivist grounded theory methodology guided data collection and thematic analysis. Results: Our study provides empirical evidence about the unique ethical and social values dimensions of HTA for children, and describes a novel social values typology for paediatric drug policy decision-making. Three principal categories of values emerged from stakeholder reflections on HTA and drug policy-making for children: procedural values, structural values, and sociocultural values. Key findings include the importance of attention to the procedural legitimacy of HTA for children, with emphasis on the inclusion of child health voices in processes of technology appraisal and policy uptake; a role for HTA institutions to consider the equity impacts of technologies, both in setting review priorities and in assessing the value of technologies for public coverage; and the potential benefits of a distinct national framework to guide drug policy for children. Conclusion: Current approaches to HTA are not well designed for the realities of child health and illness, nor the societal priorities regarding children that our study identified. This research generates new knowledge to inform decision-making on paediatric drugs by HTA institutions and government payers in Canada and other publicly-funded health systems, through insights into the relevant social values for child drug funding decisions from varied stakeholder groups.

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

To examine the RWE U.S. commercial health plans cite in their specialty drug coverage decisions. 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. 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. 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.

Variation in health plan specialty drug coverage: an empirical analysis

Background: Patients’ access to specialty drugs is determined in part by their health plan. Because health plans develop their own coverage policies, coverage of specialty drugs can vary, which in turn may affect their members’ access to care. Aims: Our objective was to examine variation in how commercial health plans cover specialty drugs. Methods: We used the Specialty Drug Evidence and Coverage (SPEC) Database for this research. SPEC contains 6,139 specialty drug coverage decisions current as of December 2018 issued by 17 of the largest US commercial health plans. SPEC includes 239 drugs and 484 drug-indication pairs (when a drug is approved for multiple indications, each drug-indication pair is represented separately in the database). We compared each plan’s publicly available coverage policies with the drugs’ FDA labels and categorized coverage as follows: (i) broader coverage than the FDA label (the plan provided coverage for populations not included in the FDA label indication), (ii) coverage with no restrictions, (iii) coverage with restrictions (the plan applies restrictions beyond the FDA label to their coverage decision, e.g., step edits or patient subgroup restrictions), (iv) mixed restrictiveness (coverage was more restrictive than the FDA label in one way but broader in another) (v), or not covered. We examined the consistency in coverage of the drug-indication pairs across the included health plans. Results: Overall, 10% of coverage decisions were broader than the FDA label, 43% covered with no restrictions, 40% covered with restrictions, 5% “mixed” restrictiveness, and 3% not covered. We found substantial variation between health plans, with the proportion of the included plans’ coverage decisions including restrictions ranging from 14% to 85%. We also found notable variation in how the included health plans covered the included drug-indication pairs, with only 5% of drug-indication pairs covered the same way by all included plans. Conclusions: Health plans applied restrictions in roughly half of their coverage policies. We found notable inconsistency between plans, with the proportion of restricted coverage decisions varying widely. All plans covered few drug-indication pairs the same way, indicating that a patient’s plan may have a large influence on their access to specialty medications.

PRO42 ARE HEALTH PLANS MORE LIKELY TO RESTRICT COVERAGE OF ORPHAN DRUGS WITH LARGER BUDGET IMPACTS?

The high costs of orphan drugs, along with their rising numbers, have raised concerns among health plans regarding their affordability. We examine whether US commercial health plans are more likely to restrict coverage of orphan drugs with larger budget impacts. We identified orphan drug coverage decisions from the Tufts Medical Center Specialty Drug and Evidence Coverage (SPEC) database, which includes information on specialty drug coverage decisions issued by 17 of the largest 20 US commercial health plans. We used multivariate logistic regression to assess the association between a drug’s budget impact and coverage restrictiveness. We considered restrictive coverage decisions to include at least one of the following; (i) patient subgroup restriction, (ii) step therapy protocol, (iii) prescriber restriction (a certain type of physician, e.g., a rheumatologist, must prescribe the drug), or (iv) other restriction (any other condition on coverage that reduces the eligible patient population size). We estimated a drug’s budget impact by multiplying a drug’s annual cost and the US prevalence of the indicated disease. We controlled for other relevant factors by including the following variables: cancer treatment; availability of alternative therapies; pediatric indication; number of years since FDA approval; self-administered; and an additional indication for a non-orphan indication. We included 2,168 orphan drug coverage decisions (current as of March 2018). We found that the odds of restricted coverage were 1.5 times larger for orphan drugs with the largest budget impacts compared to the smallest budget impacts (OR: 1.512, CI 1.119-2.042). We found the following controlling variables were also associated with an increased likelihood of restrictive coverage (p<0.05): non-cancer treatment; availability of alternative therapies; number of years since FDA approval; self-administered. We found that, when controlling for other factors, health plans are more likely to restrict coverage of orphan drugs with larger budget impacts.

What do Canadians value most for drug coverage decisions?

What do Canadians value most for drug coverage decisions?

A framework to guide the optimal development and use of real-world evidence for drug coverage and formulary decisions.

To provide a framework for optimizing the development and use of real-world evidence (RWE) in drug coverage decisions. Materials& methods: The Institute for Clinical and Economic Review convened a Policy Summit with representatives from 23 payer and life science companies that compose the Institute for Clinical and Economic Review membership. Summit participants helped refine a new conceptual framework that emphasizes the central role of contextual considerations and the evidentiary argument that the RWE is intended to support in designing the process for the development and interpretation of RWE. This framework may provide a structured way for pharmaceutical manufacturers and payers to develop a shared understanding of the best way to develop RWE that will ultimately be useful in informing coverage and formulary decisions.

Prices For Common Cardiovascular Drugs In The US Are Not Consistently Aligned With Value.

Health care reimbursement agencies in countries other than the US often rely on cost-effectiveness evidence for drug coverage decisions, signaling to drug manufacturers their expectations for value-based pricing. To see whether drug prices in the US are influenced by value, we estimated the range of cost-effectiveness for thirty frequently prescribed cardiovascular drugs. We extrapolated evidence from randomized controlled trials to determine average lifetime quality-adjusted life-years (QALYs) and payer-related costs and to calculate incremental cost-effectiveness ratios (ICERs), the principal metric of cost-effectiveness studies. Across the thirty drugs, the ICERs ranged from cost-saving with increased QALYs to more costly with decreased QALYs. This range suggests that drug pricing is not consistently influenced by value, or that such influence is masked by inaccessible factors, such as price discounts. Our findings highlight the need to debate how to define and use value-based evidence to inform US coverage and reimbursement decision making.

Specialty Drug Coverage Varies Across Commercial Health Plans In The US.

We analyzed specialty drug coverage decisions issued by the largest US commercial health plans to examine variation in coverage and the consistency of those decisions with indications approved by the Food and Drug Administration (FDA). Across 3,417 decisions, 16percent of the 302 drug-indication pairs were covered the same way by all of the health plans, and 48percent were covered the same way by 75percent of the plans. Specifically, 52percent of the decisions were consistent with the FDA label, 9percent less restrictive, 2percent mixed (less restrictive in some ways but more restrictive in others), and 33percent more restrictive, while 5percent of the pairs were not covered. Health plans restricted coverage of drugs indicated for cancer less often than they did coverage of drugs indicated for other diseases. Using multivariate regression, we found that several drug-related factors were associated with less restrictive coverage, including indications for orphan diseases or pediatric populations, absence of safety warnings, time on the market, lack of alternatives, and expedited FDA review. Variations in coverage have implications for patients' access to treatment and health system costs.

PSS35 - The Role of Patient-Reported Outcomes in Drug-Coverage Decisions for New Agents with Advanced Melanoma Indication

PSS35 - The Role of Patient-Reported Outcomes in Drug-Coverage Decisions for New Agents with Advanced Melanoma Indication

PHP177 - Scandinavian Drug Reimbursement and Coverage Decisions: The Role of HTA

PHP177 - Scandinavian Drug Reimbursement and Coverage Decisions: The Role of HTA

PCN174 - Cancer Patients May Have a Different Point of View on Oncology Drug Coverage Decisions Than Non-Patients

PCN174 - Cancer Patients May Have a Different Point of View on Oncology Drug Coverage Decisions Than Non-Patients

Anticancer drug coverage decisions evaluated

Anticancer drug coverage decisions evaluated

Comparison of Anticancer Drug Coverage Decisions in the United States and United Kingdom: Does the Evidence Support the Rhetoric?

In contrast to the United States, several European countries have health technology assessment programs for drugs, many of which assess cost effectiveness. Coverage decisions that consider cost effectiveness may lead to restrictions in access. For a purposive sample of five decision-making bodies, we analyzed US and United Kingdom coverage decisions on all anticancer drugs approved by the US Food and Drug Administration (FDA) from 2004 to 2008. Data sources for the timing and outcome of licensing and coverage decisions included published and unpublished documentation, Web sites, and personal communication. The FDA approved 59 anticancer drugs over the study period, of which 46 were also approved by the European Medicines Agency. In the United States, 100% of drugs were covered, mostly without restriction. However, the United Kingdom bodies made positive coverage decisions for less than half of licensed drugs (National Institute for Health and Clinical Excellence [NICE]: 39%; Scottish Medicines Consortium [SMC]: 43%). Whereas the Centers for Medicare and Medicaid Services (CMS) and the Department of Veterans Affairs (VA) covered all 59 drugs from the FDA license date, delays were evident for some Regence Group decisions that were informed by cost effectiveness (median, 0 days; semi-interquartile range [SIQR], 122 days; n = 22). Relative to the European Medicines Agency license date, median time to coverage was 783 days (SIQR, 170 days) for NICE and 231 days (SIQR, 129 days) for the SMC. Anticancer drug coverage decisions that consider cost effectiveness are associated with greater restrictions and slower time to coverage. However, this approach may represent an explicit alternative to rationing achieved through the use of patient copayments.

Using Effectiveness and Cost-effectiveness to Make Drug Coverage Decisions

National public insurance for drugs is often based on evidence of comparative effectiveness and cost-effectiveness. This study describes how that evidence has been used across 3 jurisdictions (Australia, Canada, and Britain) that have been at the forefront of evidence-based coverage internationally. To describe how clinical and cost-effectiveness evidence is used in coverage decisions both within and across jurisdictions and to identify common issues in the process of evidence-based coverage. Descriptive analysis of retrospective data from the Common Drug Review (CDR) of Canada, National Institute for Health and Clinical Excellence (NICE) in Britain, and Pharmaceutical Benefits Advisory Committee (PBAC) of Australia. All publicly available information as of December 31, 2008, was gathered from each committee's Web site (data set begins in January 2004 [CDR], February 2001 [NICE], and July 2005 [PBAC]). Listing recommendations for each drug by disease indication. NICE recommended 87.4% (174/199) of submissions for listing compared with a listing rate of 49.6% (60/121) and 54.3% (153/282) for the CDR and PBAC, respectively. Significant uncertainty around clinical effectiveness, typically resulting from inadequate study design or the use of inappropriate comparators and unvalidated surrogate end points, was identified as a key issue in coverage decisions. Recommendations varied considerably across countries, possibly because of differences in the medications reviewed; different agency processes, including the willingness to negotiate on price; and the approach to "me too" drugs. The data suggest that the 3 agencies make recommendations that are consistent with evidence on effectiveness and cost-effectiveness but that other factors are often important. NICE, PBAC, and CDR face common issues with respect to the quality and strength of the experimental evidence in support of a clinically meaningful effect. However, comparative effectiveness and cost-effectiveness, along with other relevant factors, can be used by national agencies to support drug decision making. The results of the evaluation process in different countries are influenced by the context, agency processes, ability to engage in price negotiation, and perhaps differences in social values.