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reviews the two most salient changes in Academy of Managed Care Pharmacy's new Format for Formulary Submissions
On October 31, 2019, the Academy of Managed Care Pharmacy introduced a new Format for Formulary Submissions at the Nexus meeting in National Harbor, MD. The new Format (version 4.1), was originally released as a final draft in September 2019. The two most salient changes introduced in this version of the Format have meaningful implications for both manufacturers and payers, and include the following:
Communications between manufacturers and payers are strictly regulated by the FDA. In years past, the FDA directed manufacturers to provide clinical and economic information about a product only in response to unsolicited payer requests. However, payers have increasingly requested more timely delivery of healthcare economic information about pipeline products to meet budgetary and forecasting needs. In 2016, the 21stCentury Cures Act was passed, which expanded the provisions in FDAMA Section 114 to allow for dialogue between payers and manufacturers to occur earlier. Consequently, in 2018, the FDA released Final Guidance on proactive communication of competent and reliable scientific as well as economic information of unapproved products to payers.
Because product dossiers are a cornerstone for these discussions, it was therefore necessary to update the AMCP Format to incorporate practical guidance on communicating information about unapproved products and unapproved uses for approved products to payers.
Given that much of the clinical and economic information about the products may not be finalized until FDA approval, some specific considerations apply to communicating information about unapproved products (in pre-approval dossier) and unapproved uses of approved products (unapproved indication dossier). Below is a quick guide to the Format’s recommendations surrounding development of pre-approval and unapproved indication dossiers:
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
• Tentative population size
• Clinical trial evidence
• Anticipated annual cost per patient
In addition to the elements typically included in Product Dossiers, include:
• A disclaimer that the product has not yet been approved by the FDA
• For unapproved use of an approved product, the approved indication and most current FDA-approved prescribing information
• Status of product development and anticipated timeline for FDA submission/approval
• Patient utilization projections
• For unapproved indication dossier, the product description must be included in the product comparison table; however, this is optional for pre-approval dossiers
Factual presentation of clinical evidence for the product that is available at the time of communication in a format similar to post-approval product dossiers
Information may be provided in a variety of ways, which may include but not limited to:
• Anticipated product price (or range of treatment cost)
• Preliminary cost-effectiveness and budget impact models
• Potential cost offsets, avoidances, and savings
• Comparison to current treatment options
• Anticipated budget impact
Similar to Section 3, include as much information as available to support the product’s value proposition, in a format similar to post-approval product dossiers
To the extent possible, include draft label, patient information sheet, MSDS, and other models
The Format acknowledges the challenges associated with providing economic modeling results several months ahead of the product approval. At the same time, not including any economic evidence in the pre-approval dossier undercuts the primary intent of allowing proactive communication with payers regarding these products. Here are a few ways you can communicate this information while maintaining confidentiality and flexibility for future updates:
• Include a population model and the modeling framework-even if other elements of the model change over time, the epidemiology and overall modeling framework will most likely remain the same
• If applicable, include most recent clinical trial results-potentially together with specifying the data cut-off date
• For BIMs, at minimum include a range of market shares and estimated PMPM for the first year after market entry-this is often the most important data point for decision makers with respect to a potential new treatment
• Product pricing-instead of price per 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., due to AE treatments). Alternatively, benchmarking the price against a similar, recently approved, comparator could also be considered.
Of course, careful consideration should be given to these assumptions so that the estimates provided in the pre-approval dossier are not vastly different from the actual price at the time of product launch
• Include preliminary scenario/sensitivity analyses-to give payers confidence about the estimated economic impact
Format 4.1 provides manufacturers with an additional means to proactively communicate clinical and economic evidence about potential new products to payers in a compliant fashion, allowing manufacturers to meet evolving payer needs for earlier communication to facilitate budgetary and forecasting decisions. Although it is a step in the right direction, there are several challenges in following these recommendations in practice -particularly around developing the economic models, which often form the basis of formulary placement and coverage decisions.
Dave Nelleson is Principal at Analysis Group.