Incorporating the Patient Voice in Value Assessments: The Evolution of ICER’s Approach to Patient Engagement

By Xcenda

As the use of value assessment frameworks to evaluate the cost-effectiveness of healthcare interventions in the United States continues to grow and evolve, the narrative around how, and to what extent, these frameworks incorporate patient perspectives has attracted considerable attention. While the Institute for Clinical and Economic Review has historically been deficient in this area, the organization has recently taken additional steps to improve its approach to patient engagement. 


Incorporating the Patient Voice in Value Assessments: The Evolution of ICER’s Approach to Patient Engagement

By: Anne Loos, MA; Robin Tan, PhD


As the unofficial drug pricing watchdog in the United States (US), the Institute for Clinical Economic Review (ICER) is no stranger to public disapproval of its approach to value assessment. One of the most common criticisms in recent years is related to ICER’s approach (or lack thereof) to patient engagement. Several patient advocacy groups, including the Institute for Patient Access and Patients Rising Now, have been particularly critical of ICER’s methods, with some organizations calling ICER’s work “discriminatory.” However, ICER has recently taken more definitive actions to include the patient voice in its work. When ICER released its updated Value Assessment Framework (VAF) for 2020–2023 in January of this year, it included several important modifications to its approach to engaging with patients. Specifically, ICER stated that it would refocus its efforts to ensure that patient perspectives are at the center of all its assessments by: 1) consolidating and updating existing patient engagement materials (eg, the Patient Participation Guide), and 2) formally creating a Patient Engagement Program. ICER also added additional points of contact for patients to collaborate during the assessment process; an overview of these touchpoints is presented in Table 1.

Table 1. Key Patient Input Opportunities
Key Patient Input Opportunities

Notably, in April 2020, ICER announced the appointment of Yvette Venable, the organization’s first-ever Vice President of Patient Engagement. In this role, Ms. Venable will manage the Patient Engagement Program, collaborate with patients and patient organizations during every assessment, and ensure that patient perspectives are included in ICER’s processes and decisions. This new position, along with the other VAF changes, represents ICER’s latest commitment to ensuring the interests of patients, families, and caregivers are included throughout ICER’s entire review process going forward. 

Implementation of New and Enhanced Patient Engagement Methodology

Figure 1 summarizes the 5 key approaches ICER identified as new and enhanced patient engagement initiatives in its 2020–2023 VAF. Considering these recent proposed modifications, it is important for manufacturers to understand how ICER’s overall approach to patient engagement has evolved, to what extent these new developments are being incorporated in ICER’s most recent assessments, and where there are remaining opportunities for improvement.  

Figure 1. Key Approaches of ICER’s Enhanced Patient Engagement ProgramKey Approaches of ICER’s Enhanced Patient Engagement Program

Dedicated Chapter on Patient Experience

Although ICER has traditionally sought patient input to inform its assessments, the process surrounding these communications was not well-defined, and the degree to which patient preferences influenced its assessments was unclear. As part of its 2017–2019 VAF, ICER began formally including patient feedback in its reports as a subsection of the Introduction titled “Insights gained from discussions with patients and patient groups.” The section is brief, usually about 1 page, highlighting any major themes from conversations with patients and patient groups. 

In line with the increased focus on including patient perspectives, the 2020 sickle cell disease (SCD) report included a dedicated section on patient perspectives, which was over 6 pages long and described, in detail, many of the challenges surrounding the management of SCD, including the biopsychosocial impact of the disease, stigma, racial bias, lack of investment in research, insufficient coverage and medication inventory, and increased anxiety and depression. ICER also reviewed and summarized the available literature on the patient experience in SCD. Similarly, whereas the section on patient perspectives in the 2018 cystic fibrosis (CF) report was about 1 page, the 2020 reassessment included about 6 pages describing ICER’s discussions with patients and patient advocacy organizations, comments received during the stakeholder comment period, and responses to ICER’s Patient Input Questionnaire, which was developed as another opportunity for patients to provide input that would guide ICER throughout the review process. 

Outcome Measures Informed by Patients

While earlier ICER reports published under the previous VAF period state that the list of interventions was developed with feedback from patient organizations, it was not clear what outcomes, if any, were recommended by patients and ultimately included in the assessment. In the more recent 2020 assessments (ie, SCD and CF), there is additional clarity around which outcomes were recommended by patients and patient groups. In fact, the reassessment of CF lists those outcomes that were added based on this input, including health-related quality of life, pill burden, and serious adverse events, while also highlighting the specific symptoms that have the greatest impact on quality of life for patients with CF. Nevertheless, some of these issues, such as the impact of CF on mood (eg, anxiety and depression), were not further discussed, even though several included studies measured these outcomes.

Formal Patient Survey

Although ICER had conducted patient surveys for some past assessments (eg, multiple sclerosis), this more formalized approach to addressing limitations in the literature had not been consistently implemented. The SCD report is the first example of ICER conducting a de novo patient and caregiver survey under its 2020–2023 VAF, which was specifically intended to capture “data not adequately addressed in the literature on the impact of SCD and its complications on ability to work, go to school, or perform usual activities, as well as out-of-pocket costs for treatments and supportive care.” ICER worked closely with Sick Cells, a patient advocacy organization that represents the SCD patient community, to develop and implement the web-based survey, which was distributed to members of the Sickle Cell Disease Association of America. The information captured by the survey, specifically the duration of pain crises and productivity losses, were used to inform health utilities and inputs related to the societal perspective model analysis.

However, ICER is not yet consistently implementing patient surveys to fill data gaps. For example, the 2020 CF report, which was released after the SCD report, did not include a de novo patient survey, and no survey data were used to inform the cost-effectiveness model of CF transmembrane conductance regulator (CFTR) gene modulators. Additionally, ICER noted that “limited evidence exists about the CFTR modulators’ impact on individuals’ ability to work or attend school, or the degree to which caregiver burden is reduced by CFTR modulator treatment.” It is unclear why ICER did not conduct a survey to fill these knowledge gaps.

Expert Reviewers Include a Patient Representative

All of ICER’s reports in the last several years have included at least 1 expert reviewer to validate its interpretation of the evidence; these reviewers are typically clinical experts in the specific disease area with experience in academia. In an effort to incorporate the patient experience, ICER has committed to including at least 1 patient representative as an expert reviewer in all of its reports. Presently, the SCD report is the only example of this, as it was reviewed by the co-founder of Sick Cells. Interestingly, the 2020 CF report lists the Cystic Fibrosis Foundation (CFF) as an expert reviewer, though no specific person is listed. It is unclear if this represents a consistent change in ICER’s approach, as CFF was also listed under the list of expert reviewers in the 2018 CF report. Furthermore, as these individuals review a pre-published version of the reports, their input is not publicly available. 

Dedicated Patient Representation on the Policy Roundtable

While the inclusion of patients in the policy roundtable discussion during ICER’s public meetings is not a new approach, the pledge to include 2 patient representatives on every roundtable will help ensure that patient voices are consistently heard and considered along with the discussions on how to apply clinical and economic evidence in policy and practice. In reviewing earlier reports, the policy roundtable included at least 1 patient or patient advocate. For the 2020 reports that are currently available (SCD and 2020 CF), the policy roundtable participants have not yet been identified as the public meetings were postponed indefinitely due to the COVID-19 pandemic. 


Overall, ICER has made some important modifications to its approach to engaging with patients, with the most noteworthy changes being related to the inclusion of an entire report chapter on the patient experience and the use of de novo evidence generation through a formal patient survey. These 2 activities alone represent considerable progress toward including patients in a meaningful way as part of ICER’s assessments. Additionally, as Hemophilia A was intended to be the first topic to be completed under the 2020–2023 VAF (ie, the first topic initiated in 2020), the fact that ICER has already started implementing these changes to patient engagement should be recognized as a step in the right direction. 

However, there are still some areas for improvement. First, while ICER has started to incorporate patient perspectives around the appropriate outcomes to include in both the clinical effectiveness and cost-effectiveness sections, some of these suggested outcomes were not explored or analyzed further even where some data were available from included studies. Secondly, it is unclear under what circumstances ICER will conduct a de novo patient survey, as this was incorporated in the SCD review but not the 2020 CF report despite there being similar data limitations. It is possible that ICER may not conduct these surveys for reassessments, though no specific rationale was provided for this decision.

Evaluating ICER’s implementation of its enhanced patient engagement initiatives through examination of recently released Evidence Reports has some limitations that should be considered in the context of ICER engagement planning. As previously mentioned, Hemophilia A was intended to be the first topic to be reviewed under ICER’s 2020–2023 VAF, but this assessment has been delayed and is currently scheduled to be completed in November 2020. Thus, ICER has not yet fully incorporated its new and enhanced patient engagement approaches in any currently available report. Additionally, the 2020 evaluations that are available at this time (SCD and CF) are not the final versions, which are published after ICER’s public meetings. As previously mentioned, these meetings have not yet taken place due to the COVID-19 pandemic, which means we have yet to see whether ICER’s commitment to reserving 2 seats for patient representatives at the policy roundtable will be observed in practice.  

As ICER’s methodology continues to evolve, it is clear that the organization has made considerable improvements to its patient engagement approach in an effort to be more inclusive of patient voices in discussions about value. Recent market research suggests that payers are increasingly using ICER’s recommendations in their decision making, so it is important for manufacturers to understand how these changes could impact an upcoming review.  


The article should be referenced as follows: 

Loos A, Tan R. Incorporating the patient voice in value assessments: the evolution of ICER’s approach to patient engagement. HTA Quarterly. Fall 2020.