–Final Report and Meeting Summary reviews evidence on 11 targeted immunomodulator drugs, including two currently under FDA review: sarilumab and baricitinib; highlights actions for payers, manufacturers, clinical societies, and other stakeholders to improve patient care while controlling costs–

Boston, Mass., April 7, 2017– The Institute for Clinical and Economic Review (ICER) has released a Final Evidence Report and Meeting Summary assessing the comparative clinical effectiveness and value of targeted immune modulators (TIMs) for treatment of moderately-to-severely active rheumatoid arthritis (RA). TIMs were evaluated alone or in combination with conventional disease-modifying anti-rheumatic drugs (DMARDs), as compared head-to-head or to conventional DMARDs alone. The review includes 11 TIMs, including two agents currently under regulatory review: the IL-6 inhibitor sarilumab (Kevzara™, Sanofi and Regeneron) and the JAK inhibitor baricitinib (Olumiant™, Eli Lilly and Co.). The report, along with an accompanying Report-at-a-Glance, is available on the ICER website.

“Since the late 1990s, TIMs have contributed to a revolutionary shift in how RA is treated. Evidence suggests these drugs are highly effective, and patients attest to their life-changing effects,” noted Steven D. Pearson, MD, MSc, ICER’s President. “Their high costs, however, lead payers to institute step therapy and prior authorization requirements that are often based on the level of rebate provided and are difficult for both patients and physicians to navigate. Too many patients struggle to access the therapies they need and miss out on the advances in treatment that they provide. Our hope is that our report and public meeting will bring new perspectives to manufacturers, payers, and other stakeholders who can work towards a system in which we can reward innovation while ensuring that patients are able to access the treatment most clinically appropriate for their needs.”

Comparative Clinical Effectiveness and Value
Voting questions focused on the two investigational agents as well as the other approved TIMs in their class. While all TIMs were found to provide substantial clinical benefits over conventional DMARDs alone, their prices also generated cost-effectiveness estimates that exceeded commonly-cited thresholds. Voting questions therefore focused on available head-to-head comparisons with the current TIM market leader, the TNFα inhibitor adalimumab (Humira®, AbbVie). The Council unanimously voted that evidence is adequate to suggest that monotherapy with the two IL-6 inhibitors tocilizumab (Actemra®, Genentech) and sarilumab offer added net health benefits compared to monotherapy with adalimumab. A majority of the Council also voted that tocilizumab represents a high long-term value for money versus adalimumab, a measure which considers both clinical and cost-effectiveness, along with other benefits, disadvantages, and contextual considerations. When comparing TIMs used as monotherapy against each other, the Council found evidence to be insufficient to make a meaningful comparison.

When looking at the drugs used in combination therapy, the Council was split on whether evidence is sufficient to show the JAK inhibitor baricitinib used in combination with conventional DMARDs offers superior net health benefit to adalimumab in combination with conventional DMARDs. The Council voted that evidence was insufficient to distinguish benefit in any other comparisons of combination therapies.

Policy Implications
During the meeting, the Council discussed policy implications of the evidence votes with a panel of subject-matter experts, including a patient and patient advocate, clinical experts, drug manufacturer representatives, and public and private payer representatives. Among the key implications are the following:

  • Payers and pharmacy benefit managers should focus on streamlining step therapy and prior authorization protocols, and consider removing step therapy requirements if drug prices fall into better alignment with the benefit they bring to patients.
  • Payers should also work to negotiate better rebates and share the savings with patients, and be more transparent regarding the role of discounting and rebates in formulary design.
  • Payers should design innovative risk-sharing payment agreements, including pay-for-performance contracts, value-based contracting, and indication-specific pricing.
  • Providers, clinical societies, and payers should develop clinical guidelines and coverage policies that align with the evidence on outcomes of patients stratified by prognosis, allowing for earlier use of TIM therapy in patients with poor prognostic factors.
  • Clinical societies and manufacturers should establish standardized assessments to allow for rigorous direct and indirect comparisons of evidence across studies and therapeutic alternatives.
  • Policy makers may need to consider regulatory intervention to ensure that drug prices and price increases do not continue to increase, taking them further from reasonable alignment with the added benefits to patients.

Further discussion of the policy recommendations is available in the full report. The report and other materials, including a Report-at-a-Glance, can be found on ICER’s website.

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.