On July 17, the Partnership to Improve Patient Care (PIPC) and Everylife Foundation for Rare Diseases held a webinar about value assessments, their potential to limit access and innovation for rare disease treatments.
The webinar featured Sara van Geertruyden, Executive Director, Partnership to Improve Patient Care; Ari Ne’eman, Disability Advocate, Partnership to Improve Patient Care; Siri Vaeth, Executive Director, Cystic Fibrosis Research, Inc.; and Christina Hartman, Senior Director of Policy and Advocacy, Everylife Foundation for Rare Diseases.
The panelists highlighted their concern about the value assessments — like the quality-adjusted life year (QALY) and equal value life-year gained (evLYG) – because the metrics they use treat patients as averages. They noted that cost-effectiveness assessments are particularly concerning when it comes to treatments developed for rare diseases, as they have the potential to impact both patient access and disincentivize investment in rare disease research and development.
Mr. Ne’eman explained that metrics for measuring the “cost effectiveness” of treatments often relies on discriminatory methods like the QALY, which values the lives of people with disabilities and serious chronic conditions as worth less than those of non-disabled people. For example, Mr. Ne’eman said that under one QALY measure, one “year in a life with moderate multiple sclerosis is worth 54 percent of the year in the life of a hypothetical non-disabled person.” Mr. Ne’eman went on to highlight the issues that materialize when using the QALY to value medical treatments, particularly for those with rare conditions that have no curative treatment options.
Ms. van Geertuyden gave an overview of the organization that is the most pervasive in championing value assessments based on cost-effectiveness — The Institute for Clinical and Economic Review (ICER). She explained that, despite advocates having long pushed back on ICER for using a flawed and discriminatory QALY-based methodology, a concerning number of payers and policymakers are viewing ICER’s work as credible. She highlighted that ICER has a new emphasis on first-in-class therapies, which is particularly concerning in relation to treatments for rare diseases. Sara explained that ICER’s methodology not only has the potential to limit access, but also future innovation in rare treatments.
Ms. Vaeth elaborated on Ms. van Geertruyden’s depiction of ICER by sharing her personal experience engaging with ICER on their 2017 Cystic Fibrosis assessment. She focused heavily on the fact that ICER claims to engage patients but did not actually seek or include the input of her organization (CFRI) and other Cystic Fibrosis groups. Ms. Vaeth imparted advice to other organizations that will be going through an ICER review, encouraging organization to be pay close attention to ICER and be ready to quickly take policy positions related to their value assessments. She also stressed that ignoring ICER is not going to help. “We want the attention to be on them, because we want people to understand how flawed this methodology is,” stated Ms. Vaeth.
Ms. Hartman gave thoughtful remarks from her perspective as a rare disease advocate and parent of a child with a rare disease. She focused on the importance of including the patient voice and her concern that ICER does not meaningfully engage with patients.
As rare disease patient advocates deal with value assessments and face ICER reports, PIPC and the Everylife Foundation hopes this webinar can serve as a helpful resource. Please do not hesitate to follow up with Sara van Geertruyden, firstname.lastname@example.org, for additional information.