• AMCP Format Expands Evidence Requirements for U.S. Payers

    In April of 2016, the Academy of Managed Care Pharmacy (AMCP) released a new Format for Formulary Submissions with revised requirements for manufacturers seeking coverage, reimbursement, and/or formulary placement of new drugs, tests, or devices.

    The last major update to the Format made in December of 2012 (version 3.1) added new information about comparative effectiveness research and specified evidence requirements for specialty pharmaceuticals and companion diagnostic tests. The updated Format (version 4.0) now includes numerous changes that reflect the increasing attention paid to value for money of new health technologies and greater rigor in coverage and reimbursement determinations in the U.S.

    Important changes in the new Format include:

    Guidelines for pre-approval dossiers:

    The new Format provides guidance on evidence that a manufacturer may provide to payers prior to FDA approval. Pre-approval dossiers may include published clinical studies, status of ongoing clinical trials, product data (product description and mechanism of action), and information about disease, treatment, and unmet needs (epidemiology, burden of disease, treatment options, clinical guidelines, etc.). This pre-approval evidence is seen as part of a continuing dialogue between manufacturers and payers to support payers’ planning needs with information about technologies on the horizon that have the potential to impact medical care and health plan budgets.

    New standards for devices and diagnostics:

    The new Format establishes standards for reporting evidence about the value of diagnostic tests and medical devices, particularly those related to the use of a drug, including companion diagnostics, implantable drug delivery devices, blood glucose measuring devices, inhalation devices, and devices or diagnostics to assess health status, diagnosis, prognosis, etc. The new standards include evidence most relevant for coverage and reimbursement determination that are designed to support assessments that span multiple related health technologies and evaluate overall health system impact.

    New, more complex requirements for evidence review:

    The new Format includes numerous additions that effectively raise the bar for evidence review by U.S. health plans, payers, pharmacy benefit managers, and other decision makers. Examples of new evidence requirements include information about how the new technology affects quality measures (such as HEDIS scores, CMS Star ratings, etc.) related to the new technology and inclusion of published ex-U.S. clinical guidelines and health technology assessments (HTA). Overall, the AMCP Format is evolving to more closely resemble the more stringent reimbursement submission requirements for HTA conducted by national health systems outside the U.S.


    Updated AMCP Format Evidence Requirements for U.S. Payers

    What happened: A new version of the AMCP Format was released

    Who is affected: Manufacturers of pharmaceuticals, medical devices, and value-priced diagnostic tests

    Why are the changes important: Standards for evidence review are a key part of the continuing debate about the value of new health technologies

    Key changes to the AMCP Format for Formulary Submissions, version 4.0

    Product Information and Disease Description
    • Product Information now includes biosimilar comparators and quality measures (e.g., HEDIS scores)
    • Comparator information is now a separate section including comparative efficacy as well as safety based on drug labels
    • Approaches to Treatment now includes care settings and information about the heterogeneity of treatment effect and post-approval monitoring of drug safety
    • A new section includes evidence about companion diagnostic tests, including place of the test in therapy, clinical data for the test, NPV, test price, etc.
    Clinical Evidence
    • The scope of clinical studies potentially relevant for payers was expanded to include additional study designs
    • Retrospective studies of the product and indirect treatment comparisons were added alongside data from clinical trials, signaling the relative importance of real-world data and analytic methods for determining comparative effectiveness
    • An option now exists to include comparator studies (i.e., those not including the reference product)
    Economic Value
    • Requires adherence to budget impact analysis best practices1
    • The modeling report format structure now must adhere to the CHEERS guidance2
    Additional Supporting Evidence
    • Now includes clinical guidelines, HTAs, and compendia 
    • Economic models and pharmacoeconomic studies not otherwise included in Section 4 now are included in Section 5 as Other Economic or Outcomes Evidence
    • A new section, Impact on Quality, includes evidence of the impact of the product on quality measures
    Supporting Information
    • Now includes patient information and material safety data sheets

    What was not included in this version of the Format?

    Frameworks for measuring value

    Medical professional societies have recently developed a number of frameworks or scoring algorithms to assess the value of new health technologies, including methods described by the American College of Cardiology and American Heart Association (ACC-AHA), the Memorial Sloan Kettering Cancer Center (MSKCC), the National Comprehensive Cancer Network (NCCN), and the American Society of Clinical Oncology (ASCO).3 Future dossier formats may specifically recognize particular value-scoring methods, or otherwise provide guidance on how manufacturers might prepare and communicate evidence in anticipation of such value assessments.

    Economic modeling requirements in response to ICER

    While cost-effectiveness analysis has been a central part of HTA by national health authorities outside the U.S., managed care decision makers have not typically requested evidence of cost-effectiveness or used cost-effectiveness criteria as a major component in coverage and reimbursement determinations. The U.S. Institute for Clinical and Economic Review (ICER) has recently published assessments of several emerging therapies, in which they applied new methods for determining economic value, including a value-priced benchmark that considers both budget impact and cost-effectiveness. Future dossier formats may provide guidance for preparation of pharmacoeconomic analyses using standard approaches in order to support direct comparison with ICER or other third-party assessments.

    Requirements for systematic literature reviews (SLR)

    Changes to the new Format include new guidance on objective criteria for inclusion and exclusion of clinical studies to be summarized or otherwise listed in the dossier. While this requirement does address challenges in selecting evidence relevant for consideration by payers without the appearance of bias, it falls short of requirements of HTA authorities for SLRs to identify clinical or economic evidence relevant to coverage and reimbursement determinations.

    Notes & Sources

    1. Sullivan SD, Mauskopf JA, et al. Budget Impact Analysis-Principles of Good Practice: Report of the ISPOR 2012 Budget Impact Analysis Good Practice II Task Force. Value Health. 2014 Jan-Feb;17(1):5-14
    2. Husereau D, Drummond M, et al. Consolidated Health Economic Evaluation Reporting Standards (CHEERS) Statement. Value Health. 2013 Mar-Apr;16(2):e1-5
    3. Neumann PJ and Cohen JT., Measuring the Value of Prescription Drugs. NEJM 373;27. 2015
    Notes & Sources


    Read the AMCP Format Version 4.0

    Dave Nellesen is a vice president in Analysis Group’s Menlo Park office who specializes in developing dossiers.