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We may be evolving into a value-based healthcare economy, but how we define value is still a moving target. The perceived value of a drug or treatment can differ based on the decision maker and the desired outcome, as well as level of measurement – individual patients or populations. Where one stakeholder may place greater value on affordability, another may be more concerned about  risks, or effectiveness in comparison to other products. These differing views have made it difficult to clearly define the value of any product, or to compare like products. Multiplicity of benefits and risks occurring along heterogeneous timelines compounds this measurement challenge.

The value conundrum is particularly challenging in oncology, where innovative, precision-guided treatments are being developed every day that promise extended survival rates and improved quality of life -- but at a very high cost. In the ‘real-world’ when we evaluate costs that may lead to downstream benefits, we view acquisition costs through an investment lens.  If we are going to accurately and reliably appraise a drug’s potential return on investment in acquisition, we need clear and meaningful metrics to quantify process, outcomes, and risks associated with their use.

One potential solution are evidence-based value frameworks. Over the past few years, several healthcare organizations have developed frameworks to assess the value of oncology and other treatments. These tools provide regulators, payers, policymakers, physicians and, most importantly, patients with an objective valuation of treatment options based on a variety of measures. They are sub-optimal today yet provide a useful jumping off point to dive into a methodological discussion of what is the equivalent of Good Accounting Practice for oncology product valuation. How can we improve at standardizing and harmonizing a valuation process that appraises clinical, humanistic, and social evidence of product performance that truly reflects the patient perspective?

My team and I have been closely following the value framework trend, and in recognition of World Cancer Day on February 4, I’d like to share our perspective on the impact these tools can have on the value conversation around oncology drugs.

Four options

Value frameworks assess multiple value-based features of treatments and medical devices then use those results to generate scores, grades or ratings that can be compared across product categories. At last year’s International Society for Pharmacoeconomics and Outcomes Research (ISPOR) meeting, we presented a workshop analyzing the strengths, limitations, and heterogeneity of four of these frameworks. Each tool attempts to unify measures of value for treatments and medical devices, and our review found that each one has benefits and shortcomings, which we highlighted in the workshop. Here’s what we found:

The American Society of Clinical Oncology’s (ASCO) Value Framework defines value as a combination of clinical benefit, side effects, and improvement in patient symptoms or quality of life in the context of cost. Outcomes and costs are combined into a single score and is intended to be the basis for a software tool that doctors can use in shared decision-making with patients. 

  • Pros: ASCO’s value framework is simplified for patients so that they can evaluate outcomes represented as a single score and costs. ASCO has also modified its net health benefit scoring, which is a weighted measure of a treatment’s benefits and side effects, to better reflect true differences between treatments.
  • Cons: The updated version only evaluates treatments that were studied head-to-head in clinical trials; patient-reported outcomes (PROs) are not currently included.

The National Comprehensive Cancer Network’s (NCCN) Evidence Blocks provide a straight-forward visual summary of different therapies for a single indication using five key measures of value. The Evidence Blocks are meant to help users understand the clinical, scientific and economic rationale for specific treatments, and to offer a starting point for shared decision-making.

  • Pros: The Evidence Blocks rank five value features -- overall survival, progression-free survival, response rate, symptom palliation, time off treatment, and quality of life, along with comparative toxicity of treatment regimens, so users can compare multiple aspects of value.
  • Cons: The 1-5 scoring system narrows the degree of distinction between drugs, there is limited information on scoring procedures, and it over-weights trial design compared to the other frameworks/value tools.

Memorial Sloan Kettering Cancer Center’s (MSKCC) DrugAbacus assesses oncology drugs using six criteria to determine an appropriate price, then compares that price with the drug’s actual price. It is intended to help patients to see if a certain drug is a “good value” based on individually customizable needs. The tool currently includes 54 oncology drugs introduced to the market since 2001.

  • Pros: DrugAbacus allows patients to adjust emphasis-levels of value measures based on those factors they deem most important.
  • Cons: It is a patient-specific tool with limited application for biopharma companies or payers covering populations of heterogeneous individuals; there are no procedures in place to add new oncology drugs or to adjust existing drugs; and it is difficult to compare drugs head-to-head.

The Institute for Clinical and Economic Review’s (ICER) Value Assessment Framework calculates value-based price benchmarks to help drug companies, payers and providers better link prices to patient benefit. The benchmarks represent the price at which patients could be treated with reasonable long-term value and reasonable short-term costs. It is intended to improve the reliability and transparency of value determinations of insurers in the US, by assessing the comparative clinical effectiveness and value of health care tests, treatments, and delivery systems.

  • Pros: The Value Assessment Framework was designed to address the need for more transparency around how value determinations are made by payers and HTA bodies.
  • Cons: Its limited time horizon may undervalue the long-term benefits and cost-offsets of therapies for chronic diseases, and economic benefits such as improvements in worker productivity are not taken into consideration. NOTE: In July, ICER put out a call for suggestions on how to improve the value assessment framework as part of a planned 2017 update to the methods that underpin its evidence reports on new drugs and interventions.

Conclusion

Despite the limitations of today’s value frameworks, they represent an important step forward in the conversation about value, and provide industry stakeholders with useful tools in quantifying value on a number of levels.  The frameworks appear to be laser-focused on clinical attributes of products, which is understandable given the wealth of somewhat standardized clinical data originating from RCTs. The lack of consideration for the humanistic and social attributes of an oncologic is noteworthy however. These qualitative elements are by default difficult to quantify but should be studied carefully nonetheless. A patient may argue the importance of these variables actually govern their preferences for treatment options.

A related limitation of the frameworks that is common to all, is a lack of focus on real-world evidence. Using RCT data solely for valuation is akin to looking back and evaluating a performance through a series of snapshots versus looking forward using longitudinal observational data to see the motion picture version of the value story. The 21st Century Cures Act catalyzes regulatory consideration of real-world evidence. The same mandate should hold for value framework developers. RWE will be instrumental in elucidating how a product performs over extended periods of time under heterogeneous conditions including variability in the provision and adoption of care by all stakeholders. These insights should ultimately govern patient-provider decision-making.

Along with helping patients and physicians make better decisions about treatment options, these frameworks could significantly influence the design of clinical trials. Developers can use these tools to identify which outcomes are most valued, understand how payers will use value measures in their coverage decisions, and choose the right endpoints and trial comparator to demonstrate the value in their products. As these frameworks are harmonized, target endpoints and other aspects of clinical trial design will come into clearer focus for manufacturers. A pre-requisite of harmonizing will be establishing a gold standard for methodology, such as GAAP accounting for a company’s financial appraisal. 

These are important benefits as physicians, payers and regulators are increasingly incorporating value-focused data in their decision making processes. Manufacturers will need to be prepared to discuss the cost implications of their treatments with physicians and payers, and to demonstrate the value proposition for how patients will be able to gain access to these treatments. Using these frameworks as a guidepost in the development process will help them make that case and tell their story in a compelling and sustainable way, like the memorable movies do year after year.

Topics in this blog post: Biopharma, Healthcare Cost, Market Access, Payers, Oncology