Public comments


FasterCures’ Comments on Proposed Updates to the ICER Value Framework

April 3, 2017

Steven D. Pearson, MD, MSc
Institute for Clinical and Economic Review
Two Liberty Square, Ninth Floor
Boston, MA 02109

Dear Dr. Pearson:

FasterCures, a center of the Milken Institute, is a non-profit, non-partisan action tank driven by a singular goal -- to save lives by speeding up and improving the medical research system. We appreciate the opportunity to share this input, prepared and authored by FasterCures, on ICER’s proposed updates to its Value Assessment Framework.

At FasterCures, we believe it is critical for stakeholders to develop more systematic ways of capturing and integrating patient perspectives into all aspects of medical product development and delivery. Since its inception in 2003, FasterCures has been working to put patients forward as partners in the biomedical research enterprise. To that end, through our Patients Count program, we are focused on expanding opportunities for patients’ perspectives to shape the processes by which new therapies are discovered, developed and delivered. This includes ensuring that patient perspectives, including patients’ real-world experience living with, and undergoing treatment for, their conditions are considered when assessing the value of a therapy.

There has been growing concern, expressed by a variety of different stakeholders, about overall increased health care spending. ICER’s ongoing work to assess the value of medical treatments using its value assessment framework has been a significant part of this discussion. Concerns about value have spurred several different activities to explore appropriate ways to assess the value of treatment options, including our own initiative with Avalere Health to develop a Patient Perspective Value Framework (PPVF). We believe that value assessments must be informed by criteria that matter to patients. We are pleased that ICER’s recent updates begin to consider the patient perspective and urge ICER and other value framework developers to continue to explore how the PPVF could be used to make value assessments more patient-centered.   

We appreciate ICER’s outreach to solicit comments from stakeholders on its value framework, and its effort to address many of those comments in its most recent update. For example, we were pleased to see that the updated ICER framework includes a “complexity of regimen” measure, which is a key component of the PPVF. We are using this opportunity to highlight two areas where ICER can take important steps to achieve a more patient-centered approach to measuring value. 

Continue to create opportunities to solicit, respond to, and integrate meaningful patient input into the value assessment process.

ICER has made progress in responding to concerns that the patient voice is not meaningfully reflected in its value assessment process, and we think it’s critical that these efforts continue to move in a positive and productive direction.

While the patient engagement guide lays out the opportunities for the public to submit input to ICER, we encourage ICER to be proactive in affirmatively seeking out patient input at each stage of the value assessment process. Patient engagement must be an ongoing back-and-forth between ICER and affected patient populations – discrete submissions of input without opportunity for meaningful dialogue will limit the utility of patient-provided information.

Moreover, while it is encouraging to see that patient representatives and clinical experts will now be permitted to join the independent public appraisal committee for the entire meeting, it is not clear from the proposed updates how these representatives will be vetted and selected. We encourage ICER to adopt a transparent evaluation and selection process to give stakeholders comfort that representatives have the appropriate expertise and breadth of knowledge to represent patient perspectives. In many cases, this may necessitate having multiple representatives who can convey the full range of patient perspectives that may be implicated in a certain disease or condition.

Finally, ICER indicates that before voting on designated “value elements” that make up other benefits and disadvantages and contextual considerations, “further input” will be obtained from patient representatives, clinical experts and other stakeholders. It’s not clear how this input will be solicited or incorporated. Moreover, it doesn’t appear that patient representatives will have a vote in the assessment process. We encourage ICER to reevaluate this process with input from the patient and stakeholder community, to help ensure that such input is solicited and integrated into the assessment in a meaningful way.

ICER should look beyond Randomized Clinical Trial (RCT) data and incorporate more observational and patient-reported data in its assessments.

While we are heartened by ICER’s restatement of its goal to consider other data sources beyond RCTs to inform its value assessments, we encourage ICER to work proactively to integrate broader sources of evidence into its value assessments to ensure outcomes more grounded in what matters to patients in the real-world. As pointed out in the Avalere-FasterCures PPVF draft methodological report, RCTs are carried out in tightly controlled conditions, often using homogeneous populations, thus limiting their generalizability to populations that may actually be candidates for the treatment in the real-world setting. Including more observational data (e.g., patient-reported outcomes, real-world evidence, and registry data) provides an opportunity to incorporate more patient-centered outcomes that may not necessarily be reported in clinical trials and utilize some of the best evidence available to understand real-world implications of certain medical interventions.

Moreover, ICER notes that “whenever possible” it will look to available data on subpopulations to evaluate the heterogeneity of treatment effect among different groups of patients. We think this is a critical consideration for value assessments, and is a key component of the “Quality & Applicability of Evidence” domain in the Avalere-FasterCures PPVF. ICER should work with patient groups to identify evidence of heterogeneity of treatment effect and where such evidence is lacking, state so clearly to help identify priority areas for future evidence-gathering.

We recognize that it is not easy to accurately reflect and incorporate patient perspectives into value assessments. But the difficulty of the task, does not make it any less critical. At FasterCures, we believe patient-relevant outcomes can drive value. To pay for value, payers and other stakeholders need to understand how care is impacting patients’ functioning in the real world. Evaluating value from the perspective of the patient can have substantial benefit for all stakeholders.

We commend ICER for the steps it has taken to better incorporate patient perspectives into its methodology through its changes to the “Other Benefits or Disadvantages” and “Contextual Considerations” domains. However, we believe that this should only be the beginning. The PPVF demonstrates how patient preferences can drive the methodology of a value assessment framework rather than being considered on the periphery. To that end, we urge continued engagement with patients and other stakeholders to achieve more patient-centered value assessments. Indeed, we hope that the May 2017 release of version 1.0 of the Avalere-FasterCures PPVF, will be a useful resource to help ICER more fully integrate the patient perspective into its assessments. We appreciate the open dialogue we have had with ICER to date and look forward to continued engagement as both initiatives move forward. 

We appreciate your consideration of these comments and welcome the opportunity to explore these comments in greater detail with you or your staff.



anderson signature

Margaret Anderson
Executive Director
FasterCures, a Center of the Milken Institute

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