• New AMCP Format for Formulary Submissions Version 4.1 recommends including preliminary product pricing in pre-approval and unapproved indication dossiers for payers

    On December 23, 2019, the Academy of Managed Care Pharmacy (AMCP) introduced a new Format for Formulary Submissions. Format Version 4.1 was originally released as a draft in May 2019. The most salient change introduced in this version of the Format has immediate implications for both manufacturers and payers. Specifically, manufacturers are often uncertain how to communicate information about investigational or off-label uses of their products. The new Format offers additional guidance on communicating clinical and health care economic information about unapproved products and unapproved uses for approved products to payers.

    However, the new Format stops short of directing manufacturers to proactively communicate information about unapproved products and unapproved uses for approved products. Instead, the new Format encourages manufacturers to follow their own policies and procedures regarding how to convey this off-label information. Note that the guidance is unchanged regarding approved product dossiers, which should still be provided only in response to an unsolicited request.

    Why was the Format updated?

    Payers have increasingly requested more timely delivery of health care economic information about pipeline products to meet budgetary and forecasting needs.

    In 2016, the 21st Century Cures Act expanded the provisions in Section 114 of the Food and Drug Administration Modernization Act (FDAMA), which regulates the communication of health care economic information (HCEI) between pharmaceutical manufacturers and US health plans. In 2018, the FDA released final guidance on communication of competent and reliable scientific evidence, including health care economic information, to payers.

    Because AMCP dossiers are key to the effective provision of this information to payers, it was necessary to update the Format to align with the new legislation and FDA guidance.

    What information should be included in the pre-approval and unapproved indication dossiers according to Format Version 4.1?

    Given that much of the clinical and economic information about products may not be finalized until FDA approval, some specific considerations apply to communicating information about unapproved products (in pre-approval dossiers) and unapproved uses of approved products (in unapproved indication dossiers). Below is a quick guide to the new Format’s recommendations surrounding development of pre-approval and unapproved indication dossiers.


    Section Key Recommendations
    1. Highlights and Overview

    A table including the following information:

    • Product name and product information
    • Disease or anticipated indication
    • Special FDA considerations and review process timeline
    • Potential launch date and distribution strategy
    • Tentative population size
    • Clinical trial evidence
    • Anticipated annual cost per patient (e.g., in ranges)
    • Anticipated patient support programs
    2. Product Information and Disease Description

    In addition to the elements typically provided in product dossiers, include:

    • A disclaimer that the product has not yet been approved by the FDA for the proposed indication
    • For unapproved use of an approved product, the approved indication and most current FDA-approved prescribing information
    • The status of product development and anticipated timeline for FDA submission/approval
    • Patient utilization projections
    • For unapproved indication dossiers, the product description must be included in the product comparison table; however, this is optional for pre-approval dossiers
    3. Clinical Evidence

    Include a factual presentation of clinical evidence for the product that is available at the time of communication; it should be in the form of study summaries and/or evidence tables (per manufacturer discretion)

    4. Economic Information

    For unapproved products, information could be provided in a variety of ways, which may include but are not limited to:

    • Anticipated product price (or range of treatment cost)
    • Potential cost offsets, avoidances, and savings
    • Comparisons to current treatment options
    • Anticipated budget impact

    What economic evidence (in Section 4) could be included in a pre-approval or an unapproved indication dossier?

    The Format acknowledges the challenges associated with providing economic modeling results months in advance of the product approval. Here are a few ways to communicate this information while maintaining confidentiality and flexibility for future updates:

    • Consider how to provide product pricing information in a pre-approval dossier. Instead of a fixed price per dose or unit (e.g., a vial), consider including a range of annual per-patient costs of treatment, potentially combined with any other applicable costs (e.g., costs of adverse event [AE] treatments). Alternatively, a price benchmark (i.e., a similar, recently approved comparator) could be considered. Of course, careful consideration should be given to these assumptions so that the estimates provided in the pre-approval dossier are not substantially different from the launch price.
    • Include a population or patient flow model along with information about the intended modeling framework. Even if other elements of the model change over time, epidemiological inputs and calculations surrounding the target population, overall modeling framework, and key assumptions will most likely remain unchanged.
    • Consider including preliminary budget impact models (BIMs), with a range of market shares and estimated per-member per-month (PMPM) budget impacts for at least the first year after market entry. Estimates of the PMPM budget impact, along with an expected market share during the first year after launch of a new product, are important considerations for decision makers planning to provide coverage for a new treatment.
    • Include scenario/sensitivity analyses. Give payers information about the expected range of the economic impact.

    In conclusion

    The AMCP Format Version 4.1 provides manufacturers with additional guidance to communicate evidence about potential new products or new uses to payers in a compliant fashion, allowing manufacturers to meet the needs of payers for the information they require to facilitate budgetary and forecasting decisions when they need it most. These recommendations pose a number of challenges – particularly around proactive dissemination of information and the development of preliminary economic models prior to launch – that may necessitate changes to manufacturers’ policies and procedures.

    Our team is prepared to help you navigate these issues in the future. For more information, please contact: