The evolution of payer management of oncology drugs in the United States between 2017 and 2022

Abstract

BACKGROUND: Research conducted in 2017 by Runyan et al concerning the current and future management of oncology drugs in the United States formed the basis for this research. The authors concluded that despite the high cost of oncology drugs, US payers relied on traditional management tools to manage the category, although these tools were ineffective at controlling costs. Innovative tools were not common in 2017.

OBJECTIVE: To compare findings from the 2017 research with findings from a 2022 payer survey to understand how payer management of oncology drugs changed over 5 years. The study evaluates changing trends in oncology drug management.

METHODS: The survey that informed the publication by Runyan et al in 2017 was reviewed, updated, and completed by 21 pharmacy and medical directors across 18 organizations representing 121.9 million covered lives. Both surveys included questions about management tools being employed in oncology and challenges to managing oncology. They used case studies in non–small cell lung cancer and chronic lymphocytic leukemia. These disease areas were chosen again in 2022 because they were included in the 2017 survey and because of the increase in competition in both categories from 2017 to 2022. The payer sample was designed to match the 2017 sample. The research was fielded from March to May 2022. The results were analyzed in Microsoft Excel; basic statistical analysis was conducted. Payers’ responses for each question were weighted by the number of reported covered lives at their organization so that the organization’s site was represented.

RESULTS: On average, payers rated the management priority of oncology as a 5.3 and the budget impact as a 6.3 on a scale of 1 to 7, where 1 was low and 7 was high. Traditional tools remain dominant in this therapeutic area. However, there has been an increase in use of innovative tools. Pathways of care are trending upward since the initial survey in 2017. The Institute for Clinical and Economic Review (ICER) also influences payers’ decision-making in oncology more than it did 5 years ago. Despite these shifts, most payers allow for unrestricted access of targeted therapies in non–small cell lung cancer and chronic lymphocytic leukemia, in line with each drug’s US Food and Drug Administration–approved label.

CONCLUSIONS: The increased use of pathways of care, shifting financial risk to providers, and the influence of ICER should continue to be monitored. Future research should focus on the role of pathways of care, comprehensive, evidence-based treatment protocols, in influencing prescribing decisions of hematologists and oncologists.

DISCLOSURES: The authors work for Envision Pharma Group (formerly Two Labs), a company that provides consulting services to the pharmaceutical and biotech industries. As such, clients in these industries pay Envision Pharma Group for their services. This study was funded independently by Envision Pharma Group.

Plain language summary

In 2017, health insurance companies in the United States did not restrict patient access to cancer drugs. Since 2017, the cost of cancer drugs has gone up and there is more competition. This led to the question of whether any changes since then have occurred. This research addresses that question. It identifies some changes, and it concludes that it is important to continue to track these changes.

Implications for managed care pharmacy

This study finds that despite the high and increasing cost of oncology drugs, and greater competition in some indications, payers in the United States have not significantly increased their level of management in oncology over the past 5 years. However, it finds that some value-based tools are becoming more important, specifically pathways of care and the Institute of Clinical and Economic Research reviews.

Oncology is the top commercial and Medicare managed care spending category in the United States, and spending on oncology drugs continues to increase. In 2022, the per-member-per-month (PMPM) spending on oncology drugs increased 5.1% compared with 2021.¹ Overall, spending on oncology drugs and oncology supportive care drugs amounted to almost 50% of PMPM spending in 2022.¹ In 2020, 6 of the top 10 highest-cost physician-administered drugs, covered under the medical benefit, were oncology drugs.² Additionally, research and development in oncology continues to grow; for example, oncology clinical trial starts increased 56% between 2016 and 2021.³ It is expected that global oncology spending will reach $375 billion by 2027.⁴ However, the cost is not always associated with improved patient outcomes in the United States.⁵

Research conducted in 2017 found that despite the cost burden of oncology drugs, payers rely on traditional management tools such as quantity limits, prior authorization, and reauthorizations, which are ineffective at controlling cost.⁶ The research showed that more innovative tools such as pathways of care, buy-and-bill incentives that influence preference toward certain preferred drugs, oncology-specific models, such as the Oncology Care Model, bundled payments, and restructuring provider networks based on quality and cost management were not commonly used.⁶ Definitions of the specific management tools explored in 2017 and in 2022 can be found in Table 1 of Runyan et al⁶ and in Supplementary Table 1 (available in online article).

TABLE 1.

Payer Use of Management Tools in Oncology: 2017 vs 2022

Management tool	2017		2022		Change
	Number of payers (N = 21)	Lives (in millions)	Number of payers (N = 21)	Lives (in millions)	Change count, %	Change lives, %
Manage to label	19	109.2	19	113.4	0.0	3.8
Quantity limits	19	118.3	18	107.9	−5.3	−8.8
Split fills	12	75.8	9	49.5	−25.0	−34.6
Reauthorization	14	74.0	15	98.5	7.1	33.2
Preferred through tiering	4	27.5	7	47.3	75.0	72.1
Step therapy for products recommended in the same line of therapy by NCCN compendia	6	43.4	8	61.1	33.3	40.9
Step therapy independent of NCCN guidelines	4	19.0	3	20.1	−25.0	6.0
Blocking agents and only having them available by medical exception	3	29.1	4	30.9	33.3	6.2
Pathways of care without risk for providers (eg, pathways where financial risk is not shared with providers, but they are incentivized through bonuses or other upside payments)	6	35.7	8	51.9	33.3	45.5
Pathways of care with risk for providers (eg, pathways where the savings or costs are shared with providers)	1	9.0	3	24.8	200.0	175.6
Buy-and-bill incentives to influence preference toward branded agents	3	26.7	1	9.0	−66.7	−66.3
Buy-and-bill incentives to influence preference toward generic agents	1	9.0	4	17.0	300.0	89.9
Shifting of financial risk to providers through payment system (eg, bundled payments)	3	24.1	4	24.7	33.3	2.3
Pursuing oncology-specific models, such as oncology care models	4	24.5	2	15.2	−50.0	−38.0
Restructuring provider networks based on their ability to reduce oncology cost	1	9.0	2	10.1	100.0	11.7

This table reflects responses from 21 payers representing 121.9 million lives. Operational definitions of each tool can be found in Table 1 of the publication by Runyan et al.⁵

NCCN = National Comprehensive Cancer Network.

This study aims to identify whether any changes in US payer management in oncology have occurred since 2017 and to assess whether the United States is moving toward value-based care in oncology.

Methods

First, the 2017 survey and research findings were reviewed. Next, a literature search was conducted to identify changes in US payer cost management of oncology drugs between 2017 and 2022. The search was conducted using PubMed and Google Scholar databases to find published articles from 2017 to 2022. Several search terms such as “oncology management in the United States,” “oncology drug cost in the United States,” “United States payer management tools in oncology,” “value-based care in oncology in the United States,” “pathways of care in oncology,” and “oncology value frameworks” were used to find oncology management and cost publications published since 2017. The search was designed to identify notable contributions to the literature over the past 5 years and was not a formal review. Findings from this search were used to update the 2017 survey. The updated 2022 survey was programmed in an online survey tool, contained 91 questions, and was sent to payer respondents via an email web link. The full survey can be found in Supplementary Appendix 1. Survey responses were collected between March and May 2022. Payers could only advance in the survey by providing a response to the previous question, and only completed surveys were accepted. Payers were able to provide commentary on responses.

In both 2017 and 2022, the survey began with general questions about each payer’s organization. Next, payers were asked about their organization’s current management of oncology in general. Then, payers were asked about the use of specific management tools today. Management tools were segmented into 3 categories: traditional tools (those used across all therapeutic categories), oncology-specific tools (those used in oncology but typically not used in other categories), and systemic tools (non–product-specific tools, but that affect the way services are provided and funded). A summary of the tools tested, and their operational definitions, can be found in Supplementary Table 1. Next, payers were asked to identify challenges their organization faced in managing oncology. Payers were then asked about the expected use of each of the specific management tools in the next 3-5 years.

Finally, 2 case studies, non–small cell lung cancer (NSCLC) and chronic lymphocytic leukemia (CLL), were explored. These areas were chosen to specifically understand management decisions in oncology. They were the same examples studied in 2017 and remained relevant because there has been a notable increase in competition among branded targeted therapies in both areas over the past 5 years. Payers were asked about the level of therapeutic improvement, budget impact, and level of management of specific, branded, targeted therapies in these categories. Management of the specific drugs in the commercial and Medicare books of business was explored. Several 2017 survey questions were updated to include additional value frameworks such as those established by Memorial Sloan Kettering Cancer Center and the European Society for Medical Oncology, as well as to include additional branded agents and combination therapies that were approved for NSCLC and CLL since 2017. National Comprehensive Cancer Network (NCCN) treatment guidelines shown in the survey for NSCLC and CLL were also updated in 2022. A copy of the full survey can be found in Supplementary Appendix 1.

Respondents were recruited through a panel of payers provided by Envision Pharma (previously MKO Global Partners), a life science consulting firm, and were contacted via email. The panel has been built over the past 8 years and includes pharmacy directors and medical directors who are responsible for formulary decision-making at their organizations. The 21 payers who responded to the 2017 email were identified first. Individuals who remained in the same role within their organization, or in a similar role at a similarly structured organization, were recontacted. If an individual had moved to a different organization, another individual at their organization was recruited. If an individual at that organization could not be recruited, someone at a similar organization was recruited. A total of 34 individuals were contacted, of whom 21 responded.

Survey responses were gathered unblinded but kept confidential. Responses were made anonymous and aggregated before data analysis was conducted. The survey data were analyzed in July 2022 using Microsoft Excel (Microsoft Corporation). All reported averages were weighted averages by covered lives. Results were expressed as descriptive statistics (eg, mean, minimum, maximum, median, SD, range, and sum).

Results

SAMPLE OVERVIEW

A total of 21 payers representing 121.9 million covered lives across 17 organizations participated in the survey. The sample represented 74% commercial lives, 12% Medicare lives, 10% Medicaid lives, and 4% other lives, which primarily consisted of health care exchange lives. There is an unmet need in NSCLC across all lines of business, and research has shown that patients without access to health care insurance have worse outcomes in NSCLC.⁷ Similarly, there is still an unmet medical need in CLL.⁸ Research has also shown that patient outcomes are improved in patients with leukemia who have private insurance.⁹ Therefore, the sample aimed to match the breakdown of insured lives in the United States as well as the breakdown in the 2017 sample. In 2021, there were 295.3 million insured lives in the United States, with 60% covered by employer or nongroup commercial coverage, 16% covered by Medicare, and 23% covered by Medicaid. Medicaid-covered lives were underrepresented in our sample.¹⁰ The sample included 14 pharmacy directors representing 76.9 million lives and 7 medical directors representing 45.0 million lives. Included were 4 regional managed care organizations (MCOs), 8 national MCOs, 5 pharmacy benefit managers (PBMs), and 4 integrated delivery networks. On average, respondents took 30 minutes to complete the survey.

There were no substantial differences in the sample composition in 2022 compared with 2017. The 2017 sample represented 75% commercial lives, 16% Medicare lives, 8.5% Medicaid lives, and 0.5% other lives. The 2017 sample included 14 pharmacy directors (83.8 million lives) and 7 medical directors (37.7 million lives). Included in 2017 were 5 regional MCOs, 6 national MCOs, 6 PBMs, and 1 integrated delivery network. The original 21 payers from the 2017 survey were initially invited to participate. Sixteen individuals who responded to the 2017 survey also responded in 2022 because they were still in the same or a similar role at the same organization or a similarly structured organization. Five individuals who took the 2017 survey were unable to respond in 2022, and replacements at the same or similar organization were then targeted. In 2 cases, another individual at the same organization responded to the survey. In 3 cases, individuals at a similarly sized and structured organization were recruited for the 2022 sample. In total, 34 payers were invited to participate and 21 complete responses were received.

MANAGEMENT AND BUDGET IMPACT OF ONCOLOGY

In the 2017 and 2022 surveys, each payer was asked to rate the budget impact and management priority of the oncology category at their organization on a scale from 1 to 7, where 1 was low and 7 was high. In 2017, the weighted average payer rating of budget impact of oncology was 6.4 (minimum of 3.0, maximum of 7.0, SD of 0.7, and median of 6.0) and of management priority was 5.1 (minimum of 2.0, maximum of 7, SD of 1.7, and median of 4.0). In 2022, the weighted average budget impact rating was 6.3 (minimum of 4.0, maximum of 7.0, SD of 0.9, and median of 7.0) and management priority was 5.3 (minimum of 2.0, maximum of 7.0, SD of 1.5, and median of 5.0).

CHALLENGES TO MANAGING ONCOLOGY

According to sampled payers, the top challenge to managing the oncology therapeutic area in general in 2022 and 2017 was the difficulty in comparing products and selecting a preferred one. Other top challenges in 2017 and 2022 included managing a complex patient population, lack of mature evidence, government regulations, and physician pushback on additional management.

MANAGEMENT TOOLS IMPLEMENTED IN ONCOLOGY

Most payers managed products to their US Food and Drug Administration (FDA)–approved indication. Prior authorizations and quantity limits were common in 2017 and 2022 (Table 1).

Some management tools were uncommon in both 2017 and 2022: step therapy independent of the NCCN guidelines (4 payers in 2017 and 3 payers in 2022), blocking agents (eg, with an electronic National Drug Code block) and only having them available through medical exceptions (3 payers in 2017 and 4 payers in 2022), shifting financial risk to providers through a payment system (3 payers in 2017 and 4 payers in 2022), and restructuring provider networks based on their ability to reduce oncology cost (1 payer in 2017 and 2 payers in 2022) (Table 1).

Some management tools were used less often in 2022 compared with 2017: split fills (12 payers in 2017 and 9 payers in 2022), buy-and-bill incentives to influence preference toward a specific preferred branded agent by providing higher reimbursement for the preferred agent (3 payers in 2017 and 1 payer in 2022), and pursuing oncology-specific models such as the Oncology Care Model (4 payers in 2017 and 2 payers in 2022) (Table 1).

Some management tools were more common in 2022 vs 2017: use of tiering differentials to drive patient preference by offering a lower drug copay for preferred products (4 payers in 2017 and 7 payers in 2022), step therapy for products recommended in the same line of therapy by NCCN compendia (6 payers in 2017 and 8 payers in 2022), buy-and-bill incentives to influence physician prescribing preference toward generic agents (1 payer in 2017 and 4 payers in 2022), and pathways of care with and without risk for providers (1 payer in 2017 and 3 payers in 2022 used pathways with risk, and 6 payers in 2017 and 8 payers in 2022 used pathways without risk).

PATHWAYS OF CARE

For the purposes of this research, pathways of care without risk for providers were defined as pathways in which financial risk is not shared with providers, but providers are incentivized through financial bonuses or positive performance ratings.

Pathways of care with risk for providers were defined as pathways in which the financial savings or costs are shared with providers. As described above, this tool was more common in 2022 than it was in 2017 but was only used by 3 out of 21 payers in 2022.

In 2022, pathways of care covered more disease areas than they did in 2017. Pathways of care without risk increased by 75% in hematological malignancies (4 payers in 2017 to 7 payers in 2022) between 2017 and 2022 and 300% in orphan oncology (0 payers in 2017 to 3 payers in 2022) between 2017 and 2022. Pathways of care with risk increased by 200% in hematological malignancies (0 payers in 2017 to 2 payers in 2022) and 100% in orphan oncology (0 payers in 2017 to 1 payer in 2022) (Figure 1). Pathways also became more restrictive. The use of pathways of care without risk that were more restrictive than NCCN guidelines increased 300% (1 payer in 2017 to 4 payers in 2022) between 2017 and 2022, and the use of pathways of care with risk that were more restrictive than NCCN guidelines increased 100% (1 payer in 2017 to 2 payers in 2022). The use of pathways without risk that recommend against combination therapies increased by 200% (0 payers in 2017 to 2 payers in 2022) between 2017 and 2022. In 2017, no payers implemented pathways where providers face negative incentives for noncompliance, but by 2022, 1 payer implemented this type of pathway (Figure 2).

FIGURE 1.

Use of Pathways of Care Across Oncology Tumor Types

FIGURE 2.

Restrictiveness of Pathways of Care

THE INFLUENCE OF ICER

The Institute for Clinical and Economic Research (ICER) developed its methods in 2006 and gained national prominence between 2014 and 2016.¹¹ ICER uses a value-assessment framework to review drugs and provide recommendations on whether the price of drugs is aligned with their clinical benefit. In 2017, ICER had a low influence on payer decisionmaking on oncology drug coverage (average of 2.4 on a 1-7 scale, where 1 is low and 7 is high, with a minimum of 1.0, maximum of 6.0, SD of 1.9, and median of 3.0). By 2022, the influence of ICER moderately increased (average of 3.2 on the same 1-7 scale, with a minimum of 1.0, maximum of 6.0, SD of 1.9, and median of 3.0). In the 2022 survey, payers indicated that they expected the influence of ICER to increase in the next 3-5 years.

BUDGET IMPACT AND MANAGEMENT OF NSCLC AND CLL

Competition in NSCLC and CLL increased between 2017 and 2022. In NSCLC, 2 programmed death protein (ligand) 1–blocking antibodies were introduced to the market between 2017 and 2022 (durvalumab and cemiplimab). In CLL, 3 new kinase inhibitors were introduced between 2017 and 2022 (acalabrutinib, zanubrutinib, and duvelisib). Combination therapies were also approved in CLL between 2017 and 2022. In addition, biosimilars of rituximab were approved in 2022.

In NSCLC, payers reported a higher budget impact for most branded therapies in 2022 compared with 2017. Each payer was asked to rate the budget impact of specific branded NSCLC therapies on a scale from 1 to 7, where 1 is no budget impact and 7 is very high budget impact. For pembrolizumab, the weighted average budget impact rating, across the 21 payers, was 5.5 (minimum of 3.0, maximum of 7.0, SD of 0.9, and median of 6.0) in 2017 vs 6.1 (minimum of 5.0, maximum of 7.0, SD of 0.8, and median of 6.0) in 2022. For nivolumab plus ipilimumab, the weighted average budget impact was 4.2 (minimum of 2.0, maximum of 6.0, SD of 1.1, and median of 4.0) in 2017 vs 6.2 (minimum of 2.0, maximum of 7.0, SD of 1.4, and median of 7.0) in 2022. For ramucirumab, the weighted average budget impact was 4.3 (minimum of 2.0, maximum of 7.0, SD of 1.3, and median of 4.0) in 2017 vs 4.5 in 2022 (minimum of maximum of 7.0, SD of 1.4, and median of 5.0) (Table 2).

TABLE 2.

Payer Ratings for the Budget Impact of Branded NSCLC and CLL Therapies

Budget impact of NSCLC and CLL therapies	2017					2022
	Weighted averagea	Minimum	Maximum	SD	Median	Weighted averagea	Minimum	Maximum	SD	Median	Change in weighted average (2017-2022), %
NSCLC
Pembrolizumab	5.5	3	7	0.9	6	6.1	5	7	0.8	6	10.9
Nivolumab + ipilimumab	4.2	2	6	1.1	4	6.2	2	7	1.4	7	47.7
Ramucirumab	4.3	2	7	1.3	4	4.5	1	7	1.4	5	4.7
Atezolizumab	5.4	3	7	1.2	5	4.8	3	7	1.4	5	−11.1
Durvalumab	N/A	N/A	N/A	N/A	N/A	4.5	2	7	1.6	5	N/A
Cemiplimab	N/A	N/A	N/A	N/A	N/A	4.5	2	7	1.8	5	N/A
Nivolumab	N/A	N/A	N/A	N/A	N/A	5.6	3	7	1.0	6	N/A
Atezolizumab + bevacizumab	N/A	N/A	N/A	N/A	N/A	5.7	2	7	1.4	6	N/A
CLL
Venetoclax	3.8	2	6	1.5	4	4.9	3	7	2.0	5	28.9
Ibrutinib	4.8	2	7	1.0	5	5.5	2	7	2.5	5	14.5
Ofatumumab	3.8	2	6	1.2	4	4.7	1	7	1.6	5	23.7
Idelalisib	4.3	2	7	1.3	4	4.7	3	7	2.0	5	9.3
Obinutuzumab	4.4	2	7	1.1	4	4.8	2	7	1.7	5	9.1
Rituximab	5.0	3	7	1.1	4	4.1	1	7	1.3	4	−18.0
Acalabrutinib	N/A	N/A	N/A	N/A	N/A	5.0	3	7	N/A	5	N/A
Zanubrutinib	N/A	N/A	N/A	N/A	N/A	4.4	2	7	N/A	5	N/A
Duvelisib	N/A	N/A	N/A	N/A	N/A	4.4	2	7	N/A	5	N/A
Venetoclax + obinutuzumab	N/A	N/A	N/A	N/A	N/A	5.6	3	7	N/A	6	N/A
Idelalisib + rituximab	N/A	N/A	N/A	N/A	N/A	5.3	2	7	N/A	5	N/A
Acalabrutinib + obinutuzumab	N/A	N/A	N/A	N/A	N/A	5.5	3	7	N/A	6	N/A
Rituximab biosimilars	N/A	N/A	N/A	N/A	N/A	3.6	1	7	N/A	3	N/A

^aWeighted averages were based on a rating scale of 1-7, where 1 is low budget impact and 7 is high budget impact. Each payer response was weighted by the number of covered lives at their organization, and a weighted average was calculated based on each payer’s reported covered lives and their 1-7 rating.

CLL = chronic lymphocytic leukemia; N/A = not applicable; NSCLC = non–small cell lung cancer.

In CLL, payers reported a higher budget impact for all branded therapies in 2022 compared with 2017. Each payer was asked to rate the budget impact of specific branded CLL therapies on a scale of from 1 to 7, where 1 is no budget impact and 7 is very high budget impact. The weighted average budget impact across the 21 payers rated by payers on the same scale from 1 to 7 for CLL was 3.8 (minimum of 2.0, maximum of 6.0, SD of 1.5, and median of 4.0) in 2017 vs 4.9 (minimum of 3.0, maximum of 7.0, SD of 2.0, and median of 5.0) in 2022 for venetoclax, 4.8 (minimum of 2.0, maximum of 7.0, SD of 1.0, and median of 5.0) in 2017 vs 5.5 (minimum of 2.0, maximum of 7.0, SD of 2.5, and median of 5.0) in 2022 for ibrutinib, 3.8 U(minimum of 2.0 maximum of 6.0, SD of 1.2, and median of 4.0) in 2017 vs 4.7 (minimum of 1.0, maximum of 7.0, SD of 1.6, and median of 5.0) in 2022 for ofatumumab, 4.3 (minimum of 2.0, maximum of 7.0, SD of 1.3, and median of 4.0) in 2017 vs 4.7 (minimum of 3.0, maximum of 7.0, SD of 2.0, and median of 5.0) in 2022 for idelalisib, and 4.4 (minimum of 2.0, maximum of 7.0, SD of 1.1, and median of 4.0) in 2017 vs 4.8 (minimum of 2.0 maximum of 7.0, SD of 1.7, and median of 5.0) in 2022 for Obinutuzumab. However, the budget impact of rituximab decreased, going from an average budget impact rating of 5.0 (minimum of 3.0, maximum of 7.0, SD of 1.1, and median of 4.0) in 2017 to 4.1 (minimum of 1.0, maximum of 7.0, SD of 1.3, and median of 4.0) in 2022 (Table 2).

Despite the increased level of competition and increased budget impact in NSCLC and CLL, the use of specific management tools for branded products in these 2 categories was limited in 2017 and remained limited in 2022 (Table 3).

TABLE 3.

Payer Use of Management Tools in NSCLC and CLL Commercial and Medicare Management: 2017 vs 2022

NSCLC and CLL payer management	2017–Actual		2022–Actual		Change
	Number of payers (N = 21)	Lives (in millions)	Number of payers (N = 21)	Lives (in millions)	Number of payers, %	Lives, %
NSCLC—commercial management
Manage more restrictively than label	0	0	2	7.7	0	0
Not providing coverage for off-label use (not FDA approved) despite NCCN compendia	0	0	0	0	0	0
Drive utilization through tiering differentials for like products (same line of therapy or MOA)	0	0	1	0.2	0	0
Use management tools such as split fills	0	0	0	0	0	0
Implement step edits that require use of one product over another in the same line of therapy per guidelines	0	0	1	0.2	0	0
Excluded an agent from formulary/coverage	0	0	0	0	0	0
Use provider-developed pathways to manage	1	9.0	1	0.2	0.0	−97.4
Clinical pathways for providers, without risk sharing	0	0	2	7.7	0	0
Enter in risk-sharing agreements with providers using pathways (narrower than NCCN guidelines) to manage	0	0	0	0	0	0
Use of value frameworks (eg, ASCO or ICER)	0	0	1	0.2	0	0
Buy-and-bill incentives to influence preference toward branded agents	0	0	0	0	0	0
Buy-and-bill incentives to influence preference toward generic agents	0	0	0	0	0	0
CLL—commercial management
Manage more restrictively than label	0	0	0	0	0	0
Not providing coverage for off-label use (not FDA approved) despite NCCN compendia	1	4.8	0	0	−100.0	−100.0
Drive utilization through tiering differentials for like products (same line of therapy or MOA)	0	0	2	12.6	0	0
Use management tools such as split fills	1	4.8	0	0	−100.0	−100.0
Implement step edits that require use of one product over another in the same line of therapy per guidelines	0	0	3	13.1	0	0
Excluded an agent from formulary/coverage	1	4.8	3	34.9	200.0	627.1
Use provider-developed pathways to manage	2	13.8	2	12.6	0.0	−8.4
Clinical pathways for providers, without risk sharing	0	0	2	12.6	0	0
Enter in risk-sharing agreements with providers using pathways (narrower than NCCN guidelines) to manage	0	0	0	0	0	0
Use of value frameworks (eg, ASCO or ICER)	0	0	0	0	0	0
Buy-and-bill incentives to influence preference toward branded agents	0	0	0	0	0	0
Buy-and-bill incentives to influence preference toward generic agents	0	0	0	0	0	0
NSCLC Medicare management
Manage more restrictively than label	0	0	2	7.7	0	0
Not providing coverage for off-label use (not FDA approved) despite NCCN compendia	0	0	0	0	0	0
Drive utilization through tiering differentials for like products (same line of therapy or MOA)	0	0	1	0.2	0	0
Use management tools such as split fills	0	0	0	0	0	0
Implement step edits that require use of one product over another in the same line of therapy per guidelines	0	0	1	0.2	0	0
Excluded an agent from formulary/coverage	0	0	0	0	0	0
Use provider-developed pathways to manage	1	9.0	1	0.2	0.0	−97.4
Clinical pathways for providers, without risk sharing	0	0	2	7.7	0	0
Enter in risk-sharing agreements with providers using pathways (narrower than NCCN guidelines) to manage	0	0	0	0	0	0
Use of value frameworks (eg, ASCO or ICER)	0	0	1	0.2	0	0
Buy-and-bill incentives to influence preference toward branded agents	0	0	0	0	0	0
Buy-and-bill incentives to influence preference toward generic agents	0	0	0	0	0	0
CLL Medicare management
Manage more restrictively than label	0	0	0	0	0	0
Not providing coverage for off-label use (not FDA approved) despite NCCN compendia	1	4.8	0	0	−100.0	−100.0
Drive utilization through tiering differentials for like products (same line of therapy or MOA)	0	0	2	12.6	0	0
Use management tools such as split fills	1	4.8	0	0	−100.0	−100.0
Implement step edits that require use of one product over another in the same line of therapy per guidelines	0	0	3	13.1	0	0
Excluded an agent from formulary/coverage	1	4.8	3	34.9	200.0	627.1
Use provider-developed pathways to manage	2	13.8	2	12.6	0.0	−8.4
Clinical pathways for providers, without risk sharing	0	0	2	12.6	N/A	N/A
Enter in risk-sharing agreements with providers using pathways (narrower than NCCN guidelines) to manage	0	0	0	0	0	0
Use of value frameworks (eg, ASCO or ICER)	0	0	0	0	0	0
Buy-and-bill incentives to influence preference toward branded agents	0	0	0	0	0	0
Buy-and-bill incentives to influence preference toward generic agents	0	0	0	0	0	0

ASCO = American Society of Clinical Oncology; CLL = chronic lymphocytic leukemia; FDA = US Food and Drug Administration; ICER = Institute for Clinical and Economic Review; MOA = mechanism of action; NSCLC = non–small cell lung cancer.

The only notable increases in management were observed in CLL, and this was limited to restrictions on rituximab and nonpreferred rituximab biosimilars (Table 3).

Discussion

The challenges that US payers faced in managing oncology drugs in 2017 persist in 2022, with payers still primarily relying on traditional management tools. This is partially a result of limited pharmacy benefit manager/manufacturer contracts for medical benefit drugs in oncology today. Oftentimes, more restrictive utilization management tools imposed on one branded product over another in the category are often a result of contracting between payers and manufacturers. However, one study found that 46% of payers expect that by the end of 2023, most medical benefit oncology drugs will have contracts in place.¹² As contracting for medical benefit products increases in the future, it will be important to monitor how payer management of branded oncology drugs evolves, especially in areas with a high level of competition.

As the cost of treating oncology increases in the United States, it will become important for payer management to evolve. The Centers for Disease Control and Prevention estimates that the total number of cancer cases in the United States will increase by 49% in 2050, compared with 2015 levels.¹³ At the same time, patients with cancer are expected to live longer. The National Cancer Institute estimated that in 2022 there were 18.1 million cancer survivors in the United States and that the number of cancer survivors would increase by 24.4% by 2032. In 2022, there were 623,405 Americans living with metastatic melanoma, metastatic breast cancer, bladder cancer, lung cancer, prostate cancer, or colorectal cancer, and this is expected to reach 693,452 by 2025.¹⁴ As more patients live for a greater amount of years while on costly cancer drugs, the cost of drug treatment will continue to increase. This cost will be further driven by increases in innovation. Research in cell and gene therapy continues to increase, and it is estimated that chimeric antigen receptor cell therapy revenues could surpass $6 billion by 2024. Investment in precision medicine also continues; in 2020, more than 90% of pivotal trials in oncology were against specific molecular targets. In addition, there is an increased investment in combination therapies, with more than 200 combinations being investigated in 2020.¹⁵ These therapies have high research and development cost and will come with significant price tags.

Other stakeholders may aid the shift toward value-based care in oncology. For example, policymakers in the United States have begun to promote value-based care and other mechanisms to decrease costs.¹⁶

In 2016, the Centers for Medicare & Medicaid Services (CMS) introduced the Oncology Care Model (OCM), which is aimed at improving the quality of cancer care while reducing costs.¹⁷ The OCM resulted in moderate cost reductions. One analysis found no statistically significant improvements in health care services utilization, quality of care, or patient experience as a result of the OCM.¹⁸ Another analysis showed that the OCM did not significantly impact health care spending, hospitalizations, or emergency department visits.¹⁹ In 2022, CMS introduced the Enhancing Oncology Model, which aims to address issues that the OCM faced.²⁰ Furthermore, the newly signed Inflation Act (IRA) will play a role by capping Medicare Part D patient out-of-pocket drug spending at $2,000 per year and limiting drug price increases due to inflation. It will also allow CMS to negotiate drug prices. By 2029, CMS will be able to negotiate prices for 20 Medicare Part D and Part B drugs. It is estimated that 4 Part D and 10 Part B oncology drugs could be affected. Allowing Medicare to negotiate drug prices could put negative pricing pressure on oncology drugs, but there is still some uncertainty about implementation.²¹ The impact of the IRA should continue to be tracked.

In addition, physicians perceive increased financial pressure compared with 5 years ago and are beginning to take cost-effectiveness into account when making prescribing decisions.²² Future research should continue to monitor this trend.

LIMITATIONS

Although the payer sample aimed to match the sample from 2017, some individuals had changed organizations, and another individual at that organization was not available to participate in this market research.

Another limitation was that most lives covered by payer respondents were commercial lives. This was intentional, to match the 2017 sample and to represent the total insured lives in the United States. However, the incidence and prevalence of cancers such as CLL and NSCLC are higher in older populations who are likely covered by Medicare organizations. Future research should target Medicare payers specifically.

Additionally, the sample was not powered to support interdemographic analysis.

Finally, there were concerns of survey fatigue, which limited survey content to 2 case studies. Future research should consider case studies in chimeric antigen receptor cell therapy and orphan oncology.

Conclusions

Despite the high and increasing cost of oncology drugs, the high budget impact they impose on health care plans, and increases in competition over the last 5 years, US payer management of the category remains low to moderate.

The challenges that US payers faced in managing oncology in 2017 are still largely in place in 2022. Specifically, because of the way clinical trials are designed, it is difficult to directly compare products and limit access to one oncolytic over another. In addition, the complex and heterogeneous patient populations pose a challenge to payer management. There is still a lack of mature evidence for many products, and some government restrictions continue to limit payers’ ability to manage this category.

The traditional tools used to manage oncology in 2017, which were largely ineffective at containing cost, remain the most common tools used in 2022. Even in the 2 target categories explored in this research, NSCLC and CLL, where there has been an increase in competition over the past 5 years, most payers still do not restrict utilization of products beyond their FDA-approved label. This is true for both commercial and Medicare formularies.

However, there is an indication that shifts toward value-based care are occurring. For example, pathways of care with and without downside financial risk for providers are more common than they were 5 years ago. Pathways now cover more disease areas and in some cases are more restrictive. Furthermore, ICER influences payers’ decision-making in oncology more in 2022 compared with 2017, and its influence is expected to increase in the next 3-5 years.

Future research should explore how pathways of care and ICER ratings influence oncologists’ prescribing behavior.

In the next 3-5 years, it will also be important to monitor how policy changes in the United States impact value-based care in oncology. Specifically, the IRA, which will allow CMS to negotiate drug prices in Medicare,²¹ could have an important impact on oncology care in the United States.