Cost and Value in Contemporary Heart Failure Clinical Guidance Documents

John W. Ostrominski, MD; Sameer Hirji, MD, MPH; Ankeet S. Bhatt, MD, MBA; Javed Butler, MD, MPH, MBA; Mona Fiuzat, PHARMD; Gregg C. Fonarow, MD; Paul A. Heidenreich, MD, MS; James L. Januzzi, JR, MD; Carolyn S.P. Lam, MBBS, PHD; Thomas M. Maddox, MD, MS; Christopher M. O'Connor, MD; Muthiah Vaduganathan, MD, MPH

Disclosures

JACC Heart Fail. 2022;10(1):1-11. 

In This Article

Discussion

Despite their central role in guiding clinical decision making and use of health care resources, few cost/value statements were observed in contemporary clinical practice guidelines and appropriate use criteria. When included, the high economic impact of HF and HF-related care is frequently mentioned in contemporary HF CGDs; however, explicit integration of cost/value in clinical guidance recommendations remains infrequent. Over time, patterns of inclusion of statements and citations of cost/value have been largely stable.

Historically, cardiovascular clinical practice guidelines have included cost/value considerations only implicitly and instead have focused on safety and efficacy of recommended interventions.[12–14] To address the increasing demand for explicit cost/value considerations to manage cost at the societal level, a 2014 ACC/AHA Task Force recommended several modifications to existing guideline development protocols, most notably the statement that a level of value should accompany conventional recommendations on the basis of clinical data (eg, level of recommendation) when high-quality economic data are available.[12] Levels of value have since been published in multiple non-HF clinical practice guidelines.[15,16] For instance, in the 2018 ACC/AHA guideline for the management of blood cholesterol, proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors received a designation of low value (>$150,000 per quality-adjusted life-year) on the basis of concomitant list prices.[15] Levels of value can serve as a relatively stable metric across different countries and health care coverage schema. Unfortunately, in many cases, independently conducted cost-effectiveness and value assessments are lacking. The generation of high-quality data on cost-effectiveness and value for all established and emerging interventions should be prioritized.

We observe low or variable adherence to the foregoing recommendations in HF clinical practice guidelines, including the 2016 and 2017 ACC/AHA/Heart Failure Society of America focused updates, for which no levels of value or explicit appraisals of available economic data were reported.[17,18] When coupled with the observation that the inclusion of cost/value statements and citations did not change over time, it does not appear that the 2014 ACC/AHA Task Force on cost/value methodology has significantly influenced the development of HF-related CGDs. Just as it takes time for guideline recommendations for therapeutics to translate into practice, it may take years before documents incorporate cost/value more regularly and explicitly. Additionally, although the 2014 Task Force provided overarching guidance, it may be helpful for individual guideline leadership to provide writers with an explicit framework for consideration and integration of these issues, as well as regularly including health economists in guideline development. Finally, the lack of an apparent change in guideline practice may reflect a persistent lack of high-quality cost/value data, as highlighted earlier. To tailor the recommendations of the 2014 ACC/AHA Task Force to HF content, we have provided a list of potential opportunities for future HF CGDs (Table 1).

Appropriate use criteria documents, which typically have been constructed to provide practical, scenario-based guidance in use of cardiovascular procedures or testing, may have unique considerations for the integration of cost/value. Writers of appropriate use criteria documents are encouraged to incorporate cost/value in each recommendation, which may explain the relatively lower frequency of detailed cost/value discussions in these documents. However, a methodology update in 2018 highlighted the goal of incorporating more transparent discussion of available cost/value studies in future appropriate use criteria documents, as well as longer-term goals of integrating scarcity and opportunity costs, cost-effectiveness analyses, and value-based recommendations for health systems.[19]

The high burden of HF-related costs is borne not only at the societal level, but also at the level of patients and caregivers. Accelerating levels of cost sharing have been associated with worse HF outcomes and have broader health and health equity implications.[5,20,21] We did observe an increasing focus on the management of the patient-level cost of HF and HF-related care, driven largely by the 2017 and 2021 ACC expert consensus documents for the optimization of HF treatment.[1,22] These documents provided new and explicit guidance for managing patient-level costs, largely related to cost reduction and medication access measures. Although they are a significant step forward, numerous opportunities remain.

In addition to potentially supporting high-value health care decision making, price transparency has been cited as a central goal to improve access to cardiovascular therapies.[6,23,24] In this analysis, however, no document reported estimated out-of-pocket costs for recommended HF drugs. Despite the challenges associated with this strategy, including the opacity of prescription drug costs[25] and the wide variation by geographic location,[26] pharmacy, or payer contract, other guideline-producing societies have implemented drug price transparency efforts. To encourage clinical cost consideration, the American Diabetes Association (ADA) reports ranges of average monthly costs of recommended antihyperglycemic therapies, on the basis of average wholesale prices and National Average Drug Acquisition Costs.[27–29] The ADA has also developed explicit diabetes management pathways for clinical situations in which costs are limiting.[27] Crucially, recent studies have shown that patients with HF are highly receptive to medication-related cost discussions,[30,31] and greater price transparency may help to inform cost-sensitive and value-focused care efforts. As an example of how the ADA's price transparency approach may be applied in HF CGDs, median monthly costs for selected HF drugs, expressed as average wholesale prices and National Average Drug Acquisition Costs with estimated Medicare Part D copayment ranges, are displayed in Table 2. Even with these estimates, given variability in clinical practice related to conveying cost information to patients, standardized cost education and decision-making tools are needed for their implementation.

Importantly, because of the large variation in health systems and regulatory policies, as well as the opacity of drug pricing policies for different regions of the world, multiple issues relating to cost/value considerations and drug price transparency become more complicated on consideration of the global perspective. Purchasing power parity, or the relative purchasing power of different countries' currencies as illustrated by the Big Mac Index, adds yet another layer of complexity.[32] Notably, the impact of purchasing power parity on the region- and country-level cost of HF therapies remains underexplored. Although CGDs developed by professional societies in the United States and Europe are often applied in lower- to middle-income countries, these issues highlight the need for practice guidelines, including cost/value considerations, to be tailored to the population for which they are intended. Otherwise, the sustainability of under-resourced health systems may be compromised.

Although we believe in the potential for care optimization with the inclusion of cost and value considerations in CGDs, we also recognize that decisions regarding their integration and use are complex. Guideline writers already carry enormous responsibility in evaluating the safety and efficacy of available therapies. Whether explicit and transparent consideration of cost and value in guidelines may promote appropriate and equitable access to therapies is not entirely certain. CGDs are currently used to facilitate access by justifying coverage to payers for expensive but effective therapies. Conversely, if cost and value are introduced more routinely into CGD development, there is potential for payers to leverage these statements adversely and deny coverage of these higher-cost therapies. In some cases, lower-cost interventions may not have sufficient evidence to support routine use, or best available evidence-based therapies for individual patients may run counter to value-based strategies to optimize societal health. Ultimately, these concerns illustrate the need for value considerations to achieve balance between providing an added layer of information and being too prescriptive.[12]

Study Limitations

The main limitations of this study include qualitative review and exclusion of other document types, such as performance measures. Furthermore, we focused on U.S. and European documents given that these documents are commonly viewed as international guidelines and are applied in countries outside the United States and Europe, but we recognize that other country- and region-specific guidance documents are increasingly available.

processing....