Regulatory Documentation Requirements
Investigational New Drug (IND) Application
This application is filed with the US Food and Drug Administration (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.67
New Drug Application (NDA)
As discussed in the Pharmaceuticals 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 drug’s proposed labeling is appropriate, and the methods used to manufacture the product
are adequate to preserve strength, quality, and purity.31
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.33 (BLA)
Formulary Decision Making Documentation Requirements
Role of the AMCP Format and dossier in the reimbursement process47
Academy of Managed Care Pharmacy (AMCP) Format aims to identify comprehensive evidence and information for
healthcare decision makers (HCDMs). It encourages sharing of objective and credible information to seek important goals
for reimbursement and distribution of products to HCDMs.
The AMCP format improves the timeliness, scope, quality, and relevance of clinical and economic evidence by furnishing
manufacturers with recommendations and guidelines on the nature of the evidence expected for a product.
It streamlines the evidence and acquisition information for HCDMs’ review process. The manufacturer may be a valuable
source for reimbursement evidence, but the AMCP dossier and format allows standardized presentation and formal
evaluation of distribution and reimbursement of a product.
There are 3 types of evidence dossiers used in product reimbursement and dossiers:
Unapproved Product Dossier
- Product approval by FDA is pending
- It is used by manufacturers
to communicate information
with HCDMs before FDA
approval process
Approved Product Dossier
Contains information on FDA-
approved products disseminated
to HCDMs by the manufacturers
Unapproved Use Dossier
Contains information on the
product’s off-label use where
the manufacturer is seeking
FDA approval
Communication of Healthcare Economic Information (HCEI) 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.68 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.