ICER Publishes Evidence Report on Targeted Immune Modulator Therapies for Ulcerative Colitis

— All assessed therapies provide significant health benefits, but even after rebates, all are priced at levels that exceed common thresholds for cost-effectiveness —

— At the upcoming September 24 virtual meeting, the California Technology Assessment Forum will review the evidence, hear further testimony from stakeholders, and deliberate over these treatments’ comparative clinical effectiveness, other potential benefits, and long-term value for money —

BOSTON, September 11, 2020 – The Institute for Clinical and Economic Review (ICER) today released an Evidence Report assessing the comparative clinical effectiveness and value of several targeted immune modulators (TIMs) that treat ulcerative colitis, including:

  • Adalimumab (Humira®, AbbVie)
  • Golimumab (Simponi®, Janssen)
  • Infliximab (Remicade®, Janssen)
  • Infliximab-abda (Renflexis®, Merck)
  • Infliximab-dyyb (Inflectra®, Pfizer)
  • Tofacitinib (Xeljanz®, Pfizer)
  • Ustekinumab (Stelara®, Janssen)
  • Vedolizumab (Entyvio®, Takeda)

“Ulcerative colitis poses a significant lifetime burden for patients and caregivers, many of whom need new treatments to help with symptoms and avoid major surgical intervention,” said Pamela Bradt, MD, MPH, ICER’s Chief Scientific Officer. “The evidence clearly demonstrates that targeted immune modulators often produce significant and durable periods of clinical remission, allowing patients to resume normal activities. Unfortunately, although patients now have multiple drug options using different mechanisms of action, we still lack head-to-head clinical trial data for most of these treatments, and there remain important gaps in knowledge about their comparative benefits and safety for different kinds of patients. Finally, the costs of these drugs is substantial, especially over time, and the prices for all of these therapies remain above reasonable levels for the benefits patients receive. It is encouraging to see the price for infliximab come down over the past couple years, likely as a result of competition with its biosimilar versions that have now entered the market, but even for this set of drugs the pricing would need to come down further to hit that sweet spot at which we are richly rewarding the benefits for patients and encouraging further innovation without adversely contributing to greater harms through the negative effects of rising health care costs for patients and families.”

This Evidence Report will be reviewed at a virtual public meeting of the California Technology Assessment Forum (CTAF) on September 24, 2020. The CTAF is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.

Register here to watch the live webcast of the CTAF meeting.

A draft version of this report was previously open for a two-month public comment period. The updated Evidence Report and voting questions reflect changes made based on comments received from patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.

Key Clinical Findings

All agents in this review had evidence clearly demonstrating their superiority to placebo. The evidence was quite limited in helping to distinguish among the different treatments, but in one head-to-head trial, supported by indirect network meta-analysis, vedolizumab was found to produce greater rates of clinical response and remission over adalimumab, the market leader, in both biologic-naïve and biologic-experienced patients. All the other TIMs were found to produce net health benefits at least comparable to adalimumab, with no clear differences among them.

Key Cost-Effectiveness Findings

For the indication of ulcerative colitis, ICER’s recommended health-benefit price benchmark (HBPB) ranges are relatively broad given that the relative effectiveness of TIMs varies between induction and maintenance phases of treatment and across populations of patients who have and haven’t previously taken biologics. There are also small differences in point estimates of clinical effectiveness that drive relatively large differences in the price benchmarks.

The HBPB ranges are $5,800-$6,900 per year for adalimumab; $6,300-$7,600 for golimumab; $8,800-$10,900 for infliximab and its biosimilars; $12,600-$15,300 for tofacitinib; $9,000-$17,200 for ustekinumab; and $9,200-$12,000 for vedolizumab. Among these therapies, the prices net of rebates for infliximab and its biosimilars come the closest to meeting its HBPB, requiring an additional 25% discount to reach the top end of the recommended price range. The other TIMs require much deeper discounts in addition to their current estimated rebates to reach their respective HBPB ranges (e.g., 85% for adalimumab and 82% for ustekinumab). The relatively better cost-effectiveness for infliximab and its biosimilars reflects reductions in net pricing for infliximab seen over the past several years that may represent the effects of biosimilar competition.

ICER’s HBPB is intended to suggest the highest US price a manufacturer should charge for a treatment, based on the amount of improvement in overall health patients receive from that treatment, when a higher price would cause disproportionately greater losses in health among other patients in the health system due to rising overall costs of health care and health insurance. In short, it is the top price range at which a health system can reward innovation and better health for patients without doing more harm than good.

About ICER

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.