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.

Date of review: September 2020

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Final Documents

Below you will find the final documents from the policy paper review process:


“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.”

“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.”

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.