![]() In some cases, a developer may feel that patient number projections are too preliminary to be included in the early dossiers. In cases where a product targets a specific patient population or subgroup, it may be useful to develop a funnel diagram to help the payer understand the size of the population to be treated. The overall patient population can be described using epidemiological data. ![]() ![]() ![]() While early dossiers will not contain economic models, manufacturers can include a description of the target patient population along with any patient utilization projections and patient funnel. Takeaway 4: Provide patient utilization projections Manufacturers may choose to present these data succinctly (as evidence tables) or more comprehensively (as study summaries and evidence tables). Manufacturers should determine the appropriate approach for the release of specific clinical data early in the medical dossier development process to ensure that dossier development is not delayed.Ī principal element of the decision-making process is determining the format for presentation of the data. How will the inclusion of clinical data affect planned or pending publications?.What are the critical studies to be introduced in early discussions, and which data from these studies is essential to include?.Manufacturers must determine the level of detail to provide, keeping the following considerations in mind: There may be differences in marketing or launch strategies regarding which data can be discussed publicly before regulatory approval and launch. There are often regulatory or internal legal constraints regarding the data that can be shared. All internal stakeholders should agree on the data to be made publicly available. Takeaway 3: Decide on clinical data to be sharedĬonsiderable discussion among internal stakeholders regarding content is generally related to Section 3 of the AMCP dossier format, which contains clinical evidence in support of a product. This may be because manufacturers can include information on a potential new indication for an already approved product in the off-label section of an AMCP dossier for an approved product. In our experience, companies are more likely to develop a UPD as opposed to a UUD. These objectives may also differ depending on the product or therapeutic area. Another may view it as an opportunity to present as much clinical data as early as possible. One manufacturer may consider it an opportunity to introduce the disease and unmet need, with minimal focus on ongoing or planned studies. While this may seem straightforward, manufacturers can have different objectives for developing an early dossier. Takeaway 2: Decide on the objective for creating an unapproved product dossier or unapproved use dossier It is critical to involve all stakeholders in preliminary discussions regarding content and plans for the dossier. I t is essential to determine key stakeholders and internal review committee members before beginning work on a UPD or UUD. Takeaway 1: Determine your internal stakeholders for pre-approval dossiers The purpose of this article is to share insights from our experiences developing these dossiers, with key considerations for manufacturers planning to develop early dossiers for their products. Since the new guidance release, RTI Health Solutions (RTI-HS) has prepared numerous unapproved product dossiers (UPDs) and unapproved use dossiers (UUDs) in various therapeutic areas, including rare diseases, oncology, and women’s health. The updated guideline provides guidance to manufacturers regarding communicating clinical and economic evidence to support new products or new uses of existing products before gaining regulatory approval by the US Food and Drug Administration to benefit eventual market access. In December of 2019, the Academy of Managed Care Pharmacy (AMCP) released Version 4.1 of the AMCP Format for Formulary Submissions, which provides guidance for formulary submission dossier development. Insights from Working with Newest AMCP Format Guidance During 2020
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