“Value” in healthcare has been defined as outcomes relative to cost.1 Determining the value of a healthcare benefit remains difficult and complex for many stakeholders. With the increasing cost of cancer therapies, value has become an important topic of conversation for patients, healthcare providers, self-funded plan sponsors (employers), and payers.
Several national health organizations have designed value frameworks (guidelines or to determine value) to assist stakeholders in the decision-making process to ensure that high-quality care is provided.2 These organizations have developed criteria to determine which prescription drug therapy has the highest clinical value and is the most cost-effective under certain scenarios. Quantifying value for clinical assessment is a step in the right direction, but the details of each framework leave significant room for improvement in incorporating all important stakeholder perspectives, including those related to cancer.
The challenges in navigating these frameworks include understanding the methodology behind determining value, defining the strengths and limitations of the framework, and interpreting the results. Another challenge is defining the target audience for each framework.
Currently, the majority of frameworks aim to help payers with formulary decision-making and may leave out the patient perspective when deciding value. Some frameworks that assist payers and providers in determining value claim to incorporate benefits and shortcomings that are important to patients, but it is often unclear how this information is used in the final determination of value. Other frameworks are intended to assist patients in choosing therapy, but the terminology used in these frameworks can be confusing for patients.
This article discusses gaps within existing frameworks to define value from the patient and employer perspectives.
The consensus among the medical community is that instituting frameworks as guidance for determining the appropriateness of therapy on a case-by-case fashion is a step forward.2 Cancer drug prices are often deemed “unsustainable,” and determining their fair value is a major concern among stakeholders.3 The expense of bringing a drug to market is costly, so it is no surprise that pharmaceutical companies are seeking optimal pricing models that satisfy return on investment.3 However, justifying an expensive drug that can extend life by a few months has been challenging to sell to consumers and to some healthcare providers, and is a topic of much public debate.3
When a patient consents to undergo cancer treatment, a burden is placed on all major stakeholders in terms of cost and therapy management. Providers have to manage the patient’s disease and expectations, as well as work with the patient to select the most cost-effective treatment options.
Payers may push back on diagnostic and treatment selections based on hospital system and outpatient setting reimbursements. With increasing research and advances in drug development, the price of innovative cancer treatments has doubled in the past decade.3 In some instances, payers are charged an annual cost of $100,000 per treatment for breakthrough cancer drugs.3 Because issues related to pricing and affordability often overshadow the patient perspective, patient assessment and preference are lacking in the design of therapeutic value frameworks.
In a webinar hosted by the Academy of Managed Care Pharmacy, speakers from a health policy research organization discussed the clinical factors of various frameworks.4 Patient-centric metrics of frameworks were found to be excluded from current treatment guidelines, with the exception of those developed by the Institute for Clinical and Economic Review (ICER). Indirect benefits, unmet needs, and the burden of illness were other clinical factors that were found to be lacking from the framework design.4 The Figure shows some of the key factors that were included (and missing) from established value frameworks that are discussed in this article.
Several national organizations have designed frameworks, with variability in what factors each organization considers necessary for assigning value. For example, the National Comprehensive Cancer Network (NCCN) focuses on 5 key measures in its Evidence Blocks for the efficient interpretation of clinical treatment decision-making.5,6
Although the NCCN’s measures include assessments of efficacy, safety, affordability, quality, and consistency of evidence, there are no clear explanations for how these measurements assess disease-specific data.5,6 The NCCN panel experts incorporate clinical experience and knowledge of the landscape to rank each category on a scale from 1 to 5.7 However, the individual rankings and the methods used to reach consensus are not disclosed.
The NCCN characterizes the criteria for scores in qualitative measures, which can be difficult for patients to understand. For example, in terms of characterizing affordability, a score of 4 is classified as “inexpensive” and a 3 is classified as “moderately expensive.”7 These classifications are difficult to assess, because coverage and out-of-pocket expenses may vary widely based on individual insurance. In addition, the Evidence Blocks do not evaluate novel mechanisms of action of therapies or the current burden of disease.
Although the NCCN’s Evidence Blocks system is visually appealing and intuitive at face value for patients to understand, it is still lacking in practicality. Navigating the specifics and applying the Evidence Blocks can be confusing and overwhelming for patients individually. For example, multiple treatment regimens are listed for each cancer type, and Evidence Blocks are listed for each therapy, with no clear-cut recommendations on how to choose one therapy over another.
In addition, the size of the scale for each category is not defined, making distinguishing a therapy with an efficacy score of 3 of 5 versus a score of 4 of 5 mean very little to a patient who does not know how to quantify the difference. A patient may decide that a therapy with an affordability score of 4 of 5 and an efficacy score of 3 of 5 is a better option than a therapy with better efficacy and greater cost.
Is enough information provided with the Evidence Blocks for patients to make that decision? Furthermore, limited data are available on the time providers spend explaining these frameworks and extraneous data to patients to allow for joint decision-making.7,8
The value framework developed by the American Society of Clinical Oncology (ASCO) weighs individual components to determine a drug’s value.8 For example, clinical benefit and toxicity, as well as bonus points for palliation and treatment-free survival, are calculated, with a maximum score of 130 points, allowing clinicians to differentiate between the value of multiple therapy options.8 The scores in each category are determined according to a selection of quantified data, which clarifies how scores are defined and simplifies patient and provider decision-making.
One unique feature of the ASCO framework is that cost is scored separately from the clinical value of a therapy, although some experts are concerned that the cost of therapy is not properly scored. The National Pharmaceutical Council issued comments on ASCO’s framework, including on the proper communication of cost to patients, noting that the framework’s current inclusion of drug acquisition cost has many associated shortcomings, including not capturing additional treatment-related services (eg, diagnostics), markup pricing by hospitals and clinics, and variance of cost by site of care (ie, inpatient vs community clinics).9 Population-specific differences in treatment response are another important issue to patients who are considering individualized therapy options.9
Memorial Sloan Kettering Cancer Center (MSKCC)’s Drug Abacus is a value decision tool that incorporates a drug’s efficacy, toxicity, novelty, cost of development, rarity, and population burden.10 However, there is no clear target audience. Inputting information into the tool allows the user to adjust multipliers for each factor, including the cost per quality-adjusted life-year (QALY), but doing so limits the patient friendliness of the tool, because patients would not be able to set a cost per QALY threshold or determine how high a multiplier to set for novelty, rarity, and population burden.10
The ICER Value Assessment Framework uses all publicly available data to determine a drug’s value on the basis of its budget impact and cost per QALY. Modifications are made to account for clinical effectiveness, contextual factors, and other considerations; however, it is unclear how other benefits and disadvantages are weighted in the final budget impact calculation. Care value and health system value are determined from the previously mentioned factors, but these values do not reflect economic benefits, such as improvements in worker productivity or reductions in caregiver burden.11
Revisions to frameworks should be considered by their respective organizations to better account for the stakeholders that are most affected by them. Adding more weight to the patient and employer perspectives is necessary to properly incorporate a complete healthcare decision. This can be accomplished by readjusting scoring methods and by adding metrics to the current layouts, giving all stakeholders an outlook on treatment variables that matter most to them.
Universal areas to revise include increasing the transparency of the methodologies that are used to determine each value score, creating a closer link between cost and outcomes, and improving patients’ understanding of difficult concepts. These changes will enable value frameworks to be more patient-centered and will include the employer perspective, a stakeholder that is often overlooked in decision tools.
In addition to weaknesses in methodology addressing patients’ concerns regarding the role in decision-making, the opinion of the employer is often overlooked. Employers are key stakeholders when considering the burden of illness in cancer survivors. Data from the 2000 National Health Interview Survey, which was administered by the US Census Bureau, revealed that cancer survivors experienced increased rates of productivity loss compared with people without cancer when controlled for age, sex, and education.12
There were 5 statistically significant measures of productivity loss in the study, including the percent of people working in the past 12 months, the percent unable to work as a result of health problems, the percent limited in the amount or type of work they performed because of health problems, and the mean (reported and modified) number of days lost from work in the past 12 months. The study demonstrates the burden of illness of patients with cancer relevant to lost productivity that impacts employers and is not currently addressed in any of the existing frameworks.12
Because drug pricing in cancer therapy has been an intensely scrutinized issue, there is an increased focus on new value assessment tools. Healthcare organizations will face several challenges when creating future versions of value frameworks to better incorporate patient and employer perspectives. Although limited, this review could jump-start efforts to improve value frameworks, as well as increase their use and effectiveness in the marketplace. Because frameworks are still being instituted in common clinical practice, resolving their disparities will increase their utility and move closer to achieving value-based decision-making and high-quality cancer care.
