Therefore, PIPC engaged Avalere to review a cross section of ICER assessments to better understand how they use the information provided from patients, providers and researchers related to the outcomes that matter most to patients. Specifically, the review included four disease areas—spinal muscular atrophy, hypertrophic cardiomyopathy, myasthenia gravis, and amyotrophic lateral sclerosis. Some highlights from the report include:
- The use of patient input, especially around scoping inclusion of relevant patient-centered outcomes, does not quantitatively impact the results of ICER’s cost-effectiveness modeling and health benefit price benchmarks. Only one of the four assessed reports included quantitative use of PCOs to model value.
- ICER’s methodology often relies on standardized patient quality of life scales across different disease states and patient populations. This may limit its ability to consistently incorporate the outcomes that matter to patients across subpopulations and people with different diseases. PCOs were only used to define health states for 2 of the 4 reports, ALS and hypertrophic cardiomyopathy. The disease-agnostic EQ-5D was used for health state utility estimates to model clinical benefit, even when PCO’s were used to define health states.
- ICER’s policy recommendations for 2 of the 4 assessments included patient-centered work to strengthen future assessments.
In addition to the above highlights from Avalere, PIPC’s conclusions from this analysis include:
- PIPC is concerned that the use of the disease-agnostic EQ-5D potentially limits the patient-centered and disease-specific perspective of clinical benefit. PIPC, the National Council on Disability, an independent federal agency advising Congress and the administration on disability policy, and others have long expressed concerns about the lack of validity and the inherent bias of the EQ-5D used to measure clinical benefit.
- PIPC emphasizes that even ICER frames the lack of inclusion of PCOs as a limitation of its findings. In order to be considered “patient centered,” PIPC recommends ICER do more than give “token” consideration to the real burdens and costs experienced by patients.
PIPC believes our opportunity to provide this feedback is now. Every three years, ICER creates a new value framework to guide its value assessment work. ICER released its new proposed value framework for 2023–2026, with comments due June 30, 2023. In it, we see ICER struggling to respond to the criticism from patients and people with disabilities that their work fails to meaningfully incorporate a patient perspective of value. Despite this ongoing shortcoming in its value assessments, ICER states that after “ongoing considerations” it still will not include additional dimensions of value citing issues related to double counting. It is disheartening that ICER is unwilling to make this change, even as others in the value assessment ecosystem, like ISPOR are acknowledging the need for it. At best, ICER recognizes the problem. At worst, ICER concludes it’s just too hard to do.
To be clear, PIPC has found the methods to incorporate patient-centered outcomes into research are not as elusive as ICER may claim them to be. Just recently, the FDA called for comments to update its guidance on conducting patient preference information studies for devices that recognized how much progress has been made on methods that capture input from patients and the translation of that information into clinical trials. Similarly, the FDA has invested in Patient-Focused Drug Development (PFDD), which is a systematic approach to ensure that patient experiences and perspectives are captured and meaningfully incorporated into drug development and evaluation.
ICER’s unwillingness to take similar steps and meaningfully incorporate patient input shows its complete indifference to the patient populations it claims to serve. ICER’s assessments have real world ramifications on patient access. The least ICER could do is make a sincere effort to ensure the patient perspective is clearly incorporated in its economic models.