Documentation Requirements  

Regulatory Documentation Requirements 

Investigational New Drug (IND) Application: This application is filed with the FDA prior to the initiation of human trials and includes the proposed clinical protocols, animal pharmacology and toxicology studies, and manufacturing information. After submission, the sponsor must wait 30 days before initiation in human trials. During this time the FDA has the opportunity to review the IND for safety.61 

New Drug Application (NDA): As discussed in the pharmaceudicals section, an NDA is filed with the FDA to seek approval to market a pharmaceutical drug in the United States. The goals of an NDA are to convey information regarding safety and effectiveness in its proposed uses, whether the drugs proposed labeling is appropriate, and the methods used to manufacture the product are adequate to preserve strength, quality, and purity.27 

Biologics License Application (BLA): A request for permission to introduce a biologic pharmaceutical product into the United States. This application is similar to an NDA for small molecule drugs.29 

Premarket Notification (510(k)): This is a required document that must be sent to the FDA stating the intent to market a medical device or diagnostic at least 90 days in advance of marketing. This process is discussed further in the Medical Devices and Diagnostics section.62 

Premarket Approval (PMA): A Premarket Approval is the FDA process of regulatory review to evaluate the safety and effectiveness of Class III medical devices. This process is discussed further in the Medical Devices and Diagnostics section.63 

Formulary Decision Making Documentation Requirements 

Academy of Managed Care Pharmacy Dossier: The Academy of Managed Care Pharmacy Dossier (AMCP Dossier or The Format) is the standardized formulary submission document for pharmaceutical manufacturers when preparing clinical and economic information for formulary review by payers. AMCP hosts these dossiers, allowing for unsolicited requests for product information. The AMCP Dossier was last updated in 2020 to “The AMCP Format for Formulary Submissions Version 4.1.” Below is the outline for the major sections of the AMCP Dossier38: 

  2. 0 - Product Information and Disease Description: A description of the product, mechanism of action, epidemiology of the disease state, current treatment guidelines, and where the product fits in to treatment. 
  3. 0 - Clinical Evidence: A summary of clinical trials for the product, including ongoing trials and those trials which may support a different indication. The goal of this section is to outline the safety and effectiveness of the product. 
  4. 0 - Economic Value and Modeling Report: A summary of the economic information around the product and role in therapy. This section usually contains a budget impact model (BIM) and cost-effectiveness analysis (CEA) as part of the economic information. 
  5. 0 - Additional Supporting Evidence: Contains any additional evidence that may need to be provided to payers, including health technology assessment (HTA) analyses, additional economic models outside of the BIM and CEA, or clinical practice guidelines to help guide formulary decisions. 
  6. 0 - Dossier Appendices: Include any references cited, economic models, product prescribing information from regulatory labeling, patient information, and material safety data sheets. 

Communication of Healthcare Economic Information Prior to FDA Approval 

FDA guidance from June 2018 was a recent clarification by the regulator regarding the preapproval communication of HCEI between industry sponsors and payers related to drugs and devices, originally described in the 21st Century Cures Act in 2016. HCEI may include analysis of the economic consequences of a drug’s or device’s clinical outcomes to alternative options or no intervention. Additionally, HCEI may be presented in a variety of ways, including evidence dossiers, peer-reviewed publications, software-based models, or slide presentations. With this guidance, the FDA takes steps to define the appropriate audience for preapproval HCEI, how unapproved information should be labeled and presented, and what is inappropriate to present for unapproved products.64 The overall goal of this guidance is to hasten the time from drug approval to patient access by allowing for communications between manufacturers and payers prior to approval. This should, in theory, decrease the time from approval to formulary review and placement.  

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