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Patient Perspectives Must Meaningfully Inform Healthcare Value Measurement


Despite the increasing focus on the concept of “value” in healthcare, there is lack of consensus on a definition of value. While patient-centricity has been a steady refrain in healthcare, we question whether the values, needs, and preferences of patients have been meaningfully incorporated into value assessments thus far.

The United States has the most expensive healthcare system in the world as well as a growing number of patients living with chronic illnesses.1 Further, there are an ever-expanding number of available treatment options, many with limited differences in efficacy and toxicity.2 In addition to rising societal costs, many patients are increasingly experiencing financial toxicity.3 High out-of-pocket (OOP) costs associated with healthcare can put patients at physical, emotional, and financial risk as patients skip doses, lose their savings, and/or face bankruptcy.4 As a result, there has been an increasing focus on the concept of “value” in healthcare. Yet, there is lack of consensus on a definition of value.5 While patient-centricity has been a steady refrain in healthcare, we question whether the values, needs, and preferences of patients have been meaningfully incorporated into value assessments thus far.

Perfunctory statements are often made regarding the consideration of patient viewpoints in value assessment, but meaningful action is another matter. There is not yet agreement—or even substantial serious conversations—about processes for measuring what patients truly value in healthcare. Without collaboration and routine efforts focused on how to build patient perspectives into such assessments, we will continue to engage in sporadic and superficial conversations with patients instead of capturing meaningful data that can contribute to a healthcare system which truly places them at the center.

There are encouraging trends that patient viewpoints and participation are beginning to be prioritized. The FDA is now conducting patient-focused drug development in order to systematically obtain patient perspective on diseases and their treatments.6 In a survey of pharmaceutical industry representatives last year, 77% said embracing patient-centricity is extremely important to their company.7 And in a recent report by the Center for Workforce Health and Performance examining employer use of research-based evidence in health, the authors recommended that employers look beyond cost and use evidence-based research to assess what’s most important to their employees.8

When patient perspectives are sought as part of value assessment, a vital understanding regarding what is most important to patients (such as productivity, OOP spending, convenience, and the promise of hope among many other concerns) is unlocked and can be considered in value calculations. This gives healthcare decision makers—from clinical trial designers to employee benefit designers—more insight into factors affecting choice and adherence to treatment, which has an impact on outcomes. Moreover, these stakeholders gain the advantage of having more precise tools to assess how diagnostics and therapies may work for individuals, rather than being limited to population-based averages, which may overlook crucial differences in patient response. In the decentralized, and increasingly value-based US healthcare marketplace, data, analysis, and a real-world understanding of the covered population is imperative.

Patients and patient advocates have long understood that value means more than just efficacy and cost, and it differs according to the patient being treated. Oncology is a perfect example. Oncology care is more expensive than any other disease with a rapidly increasing array of treatment and imaging options.9,10 With numerous therapy options for many patients, there are a host of factors and trade-off decisions that influence patient choice. Cancer is a disease-area that demonstrates how value factors can go beyond a therapy’s cost.

Government agencies like the FDA are increasingly interested in patient-centered drug design and incorporation of patient-centered measures in clinical trials, but these efforts remain in their infancy. Several organizations—including the National Health Council, Pharmaceutical Research and Manufacturers of America, Avalere/FasterCures, and National Pharmaceutical Council—have called for patient engagement in developing value frameworks, but significant room for improvement exists in order to incorporate ongoing, meaningful and systematic patient feedback.

Existing frameworks tend to focus on clinical and economic outcomes, overlooking key concepts of importance to patients. Research has found that the value to the individual patient is considered by many to be the most important factor in any assessment, yet individual patient disease characteristics are not considered by many of the frameworks.11 Many frameworks overlook concepts such as quality of life, severity of disease, and daily functioning.12 Simply put, value is not comprehensively assessed when patients are not partners in the process.

But still the question persists: how should patient information be collected and incorporated into value assessment?

The Innovation and Value Initiative (IVI) makes incorporation of patients’ perspectives a priority in developing open-source, transparent value models, with multiple opportunities for patients to participate in model development and provide feedback.

While incorporating these perspectives into IVI’s mathematical models is often challenged by a lack of detailed data (though increasing use of patient-reported outcomes and patient experience metrics in trials and studies could begin to change this) IVI has demonstrated how robust qualitative research utilizing focus groups and in-depth, structured interviews, leads to the collection and incorporation of patient experiences and perspectives.

IVI recently released qualitative research with metastatic non-squamous non-small cell lung cancer (NSCLC) patients who had received systemic therapy within the past 5 years that explored drivers of value, preferences in treatment, and other key contextual questions, which helped inform the structure and content of IVI’s Open-Source Value Platform (OSVP) decision models for assessing the relative value of sequential treatments for epidermal growth factor receptor—positive NSCLC.

According to the research, patients reported valuing treatments that would help increase overall or progression-free survival, help stop or slow progression of their disease, as well as the degree to which treatment efficacy would allow them to maintain functional ability and their quality of life with minimal side effects.

Incorporating these findings into IVI’s OSVP model from the onset is an important development for the science and implementation of value assessment and a step those involved in assessing risk, value, and cost should analyze.

Still, the fact that best practices to collect real-world patient experience and preference data are not defined is evidence enough that we must prioritize ways to systematically capture and measure what is most meaningful to patients. It is no longer acceptable to say that this data is “messy” or challenging to quantify. If we are assessing a treatment or test specific to a patient population, it is our responsibility to collaborate with one another to better understand what the patients who are being impacted by these assessments have experienced and what they most value.

Value in not just the catch phrase of the moment in healthcare. It has a direct impact on the lives of patients. All stakeholders need to step forward and embrace the challenge to find a way to routinely measure and include patient experiences and perspectives in treatment development and surveillance, and ultimately in value assessment. Accelerating action will ensure we can deliver better results for employers, plans, providers, and, most importantly, patients.


1. Waters H, Graf M; Milken Institute. The cost of chronic diseases in the U.S. Milken Institute website. milkeninstitute.org/publications/view/910. Published May 25, 2018. Accessed November 2018.

2. Schnipper LE, Davidson NE, Wollins DS, et al. Updating the American Society of Clinical Oncology value framework: revisions and reflections in response to comments received. J Clin Oncol. 2016;34(24):2925-2934. doi: 10.1200/JCO.2016.68.2518.

3. Zafar SY. Financial Toxicity of cancer care: it's time to intervene. J Natl Cancer Inst. 2016;108(5). doi: 10.1093/jnci/djv370.

4. Bach PB, Pearson SD. Payer and Policy maker steps to support value-based pricing for drugs. JAMA. 2015;314(23):2503-2504. doi: 10.1001/jama.2015.16843.

5. Neumann PJ, Cohen JT. Measuring the value of prescription drugs. N Engl J Med. 2015;373(27):2595-2597. doi: 10.1056/NEJMp1512009.

6. Food and Drug Administration. Patient focused drug development: disease area meetings held in fiscal years 2013-2017. FDA website. fda.gov/forindustry/userfees/prescriptiondruguserfee/ucm347317.htm. Updated March 23, 2018. Accessed November 2018.

7. Miseta E. Is patient centricity just a buzzword? Clinical Leader website. clinicalleader.com/doc/is-patient-centricity-just-a-buzzword-0001. Published September 18, 2017. Accessed November 2018.

8. Parry T, Jinnett K; The Center for Workforce Health and Performance. The employer's role in using research-based healthcare evidence. CWHP website. tcwhp.org/how-employers-use-evidence-make-employee-health-investment-decisions. Published June 2018. Accessed November 2018.

9. Adelson KB, Velji S, Patel K, Chaudhry B, Lyons C, Lilenbaum R. Preparing for value-based payment: a stepwise approach for cancer centers. J Onc Pract. 2016;12(10):e924-e932.

10. Ganz PA. Improving the quality and value of cancer care: a work in progress—The 2016 Joseph V. Simone Award and Lecture. J Onc Pract. 2016;12(10):876-879.

11. Slomiany M, Madhavan P, Kuehn M, Richardson S. Value frameworks in oncology: comparative analysis and implications to the pharmaceutical industry. Am Health Drug Benefits. 2017;10(5):253-260.

12. Sorenson C, Lavezzari G, Daniel G, et al. Advancing value assessment in the United States: a multistakeholder perspective. Value Health. 2017;20(2):299-307. doi: 10.1016/j.jval.2016.11.030.

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