— Informed by expert input from patient groups, clinician specialty societies, payers, and life science companies, white paper provides recommendations for appropriate design and implementation of policies that determine patient access to prescription drugs —

— ICER has formed a multi-stakeholder Working Group to help guide assessment of major payer policies; by mid-2021, ICER will publish first annual report on how those policies align with fair access criteria —

BOSTON, September 28, 2020 — The Institute for Clinical and Economic Review (ICER), in collaboration with researchers at the Office of Health Economics (OHE), has published a new white paper: “Cornerstones of ‘Fair’ Drug Coverage: Appropriate Cost-Sharing and Utilization Management Policies for Pharmaceuticals.” The purpose of the white paper is to analyze the ethical and practical dimensions of insurance coverage policy, while presenting a corresponding set of criteria that will support a more transparent discussion among all health care stakeholders about whether specific cost sharing and utilization management policies are delivering “fair” patient access to prescription drugs.

In conjunction with the publication of this white paper, ICER is launching a new initiative to assess the degree to which coverage policies among leading US insurers and pharmacy benefit managers meet the criteria for appropriate policy design and implementation.

“With the growing attention being paid to fair pricing of drugs, it is equally important that policy makers be just as clear about what fair access looks like, and work to make sure that it is achieved throughout the US health care system,” said Steven D. Pearson, MD, MSc, President of ICER. “This white paper gets right at questions that are critically important for patients — questions that should drive the way plan sponsors, payers, and drug makers develop policies that determine patient access to drugs. Is it fair to have patients pay at the highest cost-sharing level when there is only a single drug available in a drug class? What are the circumstances in which step therapy is a reasonable approach to prescription drug coverage? When is it appropriate for the clinical criteria required for coverage to be narrower than the FDA-labeled indication? And how should the pricing of a drug, whether it is deemed reasonable or not, factor in to whether certain strategies to limit or steer patient access are appropriate? We intend for the criteria proposed in the white paper to address these questions and provide guidance for all participants in the health system — from plan sponsors designing their health insurance cost sharing tiers for their employees, to payers creating prior authorization policies, to drug makers negotiating coverage terms, to clinical specialty societies and patient groups who want to hold all of the above accountable. We hope all parties can find in this white paper a guide to a common understanding of what is reasonable and what is not.”

Sarah K. Emond, MPP, ICER’s Executive Vice President and Chief Operating Officer, added: “To kickstart these efforts, we are launching our own initiative to evaluate the formularies of leading payers and, using the criteria from the white paper, assess whether we find concordance across the coverage policies of leading US health insurers. We look forward to this research effort and will publish an annual report on what we find — a report on whether insurance coverage for key drugs that we have assessed as being fairly priced is meeting the commensurate standards that patients should expect for fair access.”

Cornerstones of “Fair” Drug Coverage

In establishing principles and criteria for fair access, ICER’s white paper focuses on the areas that prompt the most controversy and where payers have significant discretion. It provides a conceptual analysis of the ethical and practical tradeoffs that payers must navigate, a description of the contextual considerations that are critical to striking a reasonable balance between cost control and broad access, specific criteria for assessing the fairness of benefit designs, as well as specific criteria for implementing appropriate cost-sharing and utilization management strategies.

Key recommendations include:

  • Cost Sharing:
    • Patient cost sharing should be based on the net price to the plan sponsor, not the unnegotiated list price.
    • At least one drug in every class should be covered at the lowest relevant cost-sharing level unless all drugs are priced higher than an established fair value threshold.
    • If all drugs in a class are priced so that there is not a single drug that represents a fair value as determined through value assessment, it is reasonable for payers to have all drugs on a higher cost-sharing level.
  • Eligibility Criteria:
    • Clinical eligibility criteria should be developed with explicit mechanisms that require payer staff to document that they have 1) considered limitations of evidence due to systemic under-representation of minority populations; 2) sought input from clinical experts on whether there are distinctive benefits or harms of treatment that may arise for biological, cultural, or social reasons across different communities; and 3) confirmed that clinical eligibility criteria have not gone beyond reasonable use of clinical trial inclusion/exclusion criteria to interpret or narrow the FDA label language in a way that disadvantages patients with underlying disabilities unrelated to the condition being treated.
    • For reasonably priced drugs, clinical eligibility criteria should not deviate from the FDA label in a manner that would narrow coverage.
    • For drugs deemed to be priced unreasonably, criteria may narrow coverage to the eligibility criteria from the pivotal trials if implemented with reasonable flexibility and supported by robust appeals procedures.
  • Step Therapy:
    • In order to justify economic step therapy policies as appropriate, payers should explicitly affirm or present evidence that 1) use of the first-step therapy reduces overall health care spending, not just drug spending; 2) the first-step therapy is clinically appropriate for all or nearly all patients and does not pose a greater risk of any significant side effect or harm; patients will have a reasonable chance to meet their clinical goals with first-step therapy; 3) failure of the first-step drug and the resulting delay in beginning the second-step agent will not lead to long-term harm for patients; and 4) patients are not required to retry a first-line drug with which they have previously had adverse side effects or an inadequate response at a reasonable dose and duration.
  • Prescriber Restrictions:
    • Restrictions of coverage to specialty prescribers are reasonable when payers explicitly affirm that 1) accurate diagnosis and prescription require specialist training, with the risk that non-specialist clinicians would prescribe the medication for patients who may suffer harm or be unlikely to benefit; 2) determination of the risks and benefits of treatment for individual patients requires specialist training due to potential for serious side effects of therapy; or 3) dosing, monitoring for side effects, and overall care coordination require specialist training to ensure safe and effective use of the medication.

The complete list of fair access criteria is included within the white paper.

The white paper was co-authored by leading researchers at the OHE, a not-for-profit organization based in the UK that conducts research on the economics of innovation and the life sciences industry, the organization and financing of health care, and the role for outcomes research and health technology assessment.

This paper was developed following ICER’s Policy Summit in December 2019, where representatives from patient organizations and clinical societies joined ICER’s Policy Leadership Forum – comprising senior policy leaders from 25 payer and life science companies – to discuss a preliminary draft of this paper, debate the concept of fair access, and provide suggestions for revisions to the paper. None of these individuals, nor their organizations, is responsible for the final contents of this report or should be assumed to support any part of this report, which is solely the work of the ICER team and its affiliated researchers.

Annual Assessment of Insurance Coverage

In conjunction with this white paper, ICER is initiating a review of current coverage policies of US insurers and pharmacy benefit managers, with plans to publish the first annual report on concordance of policies with fair access criteria by the middle of 2021.

While the guiding principles of this forthcoming assessment will derive from those laid out within this white paper, ICER will be publishing a specific review protocol in November. This protocol will be informed by ongoing discussions with a cross-stakeholder Working Group, which includes the following individuals:

  • Cat Davis Ahmed, MBA, Vice President of Policy and Outreach, Familial Hypercholesterolemia Foundation
  • Alan Balch, PhD, Chief Executive Officer, Patient Advocate Foundation
  • Robert W. Dubois, MD, PhD, Interim President and Chief Executive Officer, Chief Science Officer, National Pharmaceutical Council
  • Patrick Gleason, PharmD, Assistant Vice President of Health Outcomes, Prime Therapeutics
  • Barbara Henry, Manager, Clinical Pharmacy Services, Harvard Pilgrim Health Care
  • Leah Howard, JD, Chief Operating Officer, National Psoriasis Foundation
  • Cliff Hudis, MD, FACP, FASCO, Chief Executive Officer, American Society of Clinical Oncology
  • Anna Hyde, Vice President of Advocacy and Access, Arthritis Foundation
  • Rebecca Kirch, JD, Executive Vice President, National Patient Advocate Foundation
  • Eleanor Perfetto, PhD, MS, Executive Vice President, National Health Council
  • Carl Schmid, Executive Director, HIV+Hepatitis Policy Institute
  • Bari Talente, Executive Vice President, Advocacy, National Multiple Sclerosis Society
  • Douglas White, MD, PhD, Treasurer, American College of Rheumatology

Although this Working Group will provide helpful input that will influence the decisions ICER makes in establishing the assessment protocol, none of these individuals or their organizations is responsible for the final protocol. Further, this Working Group will not be involved in the actual review of payer policies or the development of the final report. It should not be assumed that these individuals or their organizations endorse any specific part of the protocol or the ultimate report, as ICER is solely responsible for both documents.

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.