The ACR recently contributed comments to the Institute for Clinical & Economic Review (ICER) to inform the initial drafting of an assessment of the efficacy and cost effectiveness of medical therapies for lupus nephritis. In a Sept. 21 letter, the ACR encouraged ICER to consider a number of issues essential for the appropriate treatment of lupus nephritis, highlighted limitations in analyses to date and provided current references about lupus nephritis treatment and outcomes.
Founded in 2006, ICER is an independent research organization that evaluates the clinical and economic value of medical treatments. It has published numerous assessments of the cost effectiveness of prescription drugs in various clinical areas using a systematic methodology that estimates the amount of money per year available for spending on new drugs in the U.S. and dividing that amount by the number of expected approvals by the U.S Food & Drug Administration.
Two previous ICER projects also focused on therapies for rheumatic conditions: In 2017, ICER reviewed the cost effectiveness of biologic drugs and in 2019 conducted a more targeted 2019 review of the cost effectiveness of Janus kinase inhibitors (JAKs).
In 2017, The Rheumatologist reported that then ACR President Sharad Lakhanpal, MD, MBBS, had expressed concern over the potential for access limitations to appropriate care based on the report’s findings. While acknowledging the importance of comparative effectiveness research, Dr. Lakhanpal stressed the importance of not limiting treatment options for patients. He said, “Ideally, [comparative effectiveness research] would highlight the need for multiple treatment options to address heterogeneous groups of patients with individual and unique co-morbidities. The ACR is concerned that the ICER report, while based on a commonly used method to assess cost effectiveness, provides insufficient information on model structure and validation.”
According to Chris Phillips, MD, a rheumatologist on the ACR’s Committee on Rheumatologic Care (CORC) and the ACR’s representative to ICER, ACR involvement has grown with each project, reflecting the importance the ACR places on working closely with ICER to ensure its assessments accurately address the essential issues of treating patients with rheumatic conditions.
“We recognize that ICER’s footprint in the economic discussions regarding drug pricing is growing, and we are glad to be able to have a good working relationship with them,” says Dr. Phillips.
Meaningful Discussions
To develop an assessment, Dr. Phillips explains, ICER first chooses a scope for its review then performs an extensive literature review to gather relevant data. For rheumatoid arthritis, for example, that data would focus on drug efficacy. Then ICER uses validated cross-walks between outcome measures (such as ACR 20/50/70 responses) and quality-adjusted life years (QALY) gained as inputs for drug pricing and economic models to try to measure cost per QALY.
The 2017 assessment was the ICER’s first foray into rheumatology. The ACR provided written comments during public comment periods and attended and commented during a ICER panel meeting.
Since then, however, the ACR has slowly increased its participation with ICER. In 2019, during the ICR assessment of JAK inhibitors, the ACR established a relationship with ICER early in the process and was able to provide input throughout the drafting of the assessment. The ACR also was allowed a seat at the table when ICER presented its draft assessment at its policy roundtable meeting, where a panel of health economists and physicians voted on various cost-effectiveness questions. ACR representatives (Dr. Phillips and Doug White, MD, ACR treasurer) provided further input at subsequent ICER policy roundtables, along with representatives from some patient groups and pharmaceutical companies.
Saying these discussions focus on fair drug coverage, Dr. White commented on how remarkable the discussions are for their deep dives into the issue at hand. “Participants who have a highly specialized understanding of the workings of the U.S. drug market and who represent organizations with conflicting interests share insights and hear each other in a way that I’ve not witnessed before,” he says. “I don’t know if it will be enough to break the log jam, but I do think these types of conversations are an essential component to any rational policy changes in the future.”
Evolving Relationship
Amanda Grimm Wiegrefe, the ACR’s director of regulatory affairs, emphasizes the ACR has slowly increased participation with ICER over the past few years and aims for further involvement. “We know that healthcare costs will continue to be a focus of policymakers and public and private payers,” she says. “The ACR must increase our level of participation in ICER’s analysis, [and] I hope we will have the opportunity to expand our presence with the staff and begin communications at the inception of a project before the scoping document is drafted.”
Early participation in the process is essential to ensure the perspectives of rheumatologists and rheumatology health professionals are adequately represented throughout the entire process, explains Rachel Myslinski, the ACR’s vice president of practice, advocacy & quality.
Ms. Myslinski notes the ACR doesn’t always agree with everything in the final ICER assessments and the methodology used is not able to fully account for all the issues clinicians face in real-world medical practice. However, she emphasizes that ICER is fulfilling its mission and focus to develop cost effectiveness assessments. The ACR’s role, she says, is to actively participate by providing input on the implications of these assessments on practices and practitioners.
For example, in the lupus nephritis assessment currently underway, ICER is evaluating the clinical and economic value of two medical therapies not yet approved by the FDA: voclosporin and belimumab. In its September letter to ICER, the ACR raised concerns about the lack of head-to-head or real-world data comparisons to these new therapies. To better assess the efficacy of these new therapies, the ACR urged ICER to consider trends seen in the standard-of-care arms of clinical trials of the new therapies and incorporate data on flares, end-stage renal disease, need for steroid rescue, dropouts or treatment failures and deaths.
The ACR has a good relationship with ICER despite questions about the methodology used, Dr. Phillips stresses. However, he expresses ongoing concerns about the ramifications of ICER’s conclusions, such as whether patients may lose access to treatment if insurance decides not to cover a drug that doesn’t meet the formal cost/QALY threshold used in an ICER assessment.
“We have found ICER to be fairly even-handed in [its] assessment of the data, and [it has] a genuine interest in helping to drive drug pricing to more rational levels,” Dr. Phillips says. “While we continue to be wary of whether the conclusions may be used by payers to be more restrictive to drug access, we have found [ICER] receptive to our input.”
Mary Beth Nierengarten is a freelance medical journalist based in Minneapolis.
