(1 of 4) Transitioning from Clinical Research to FDA Review: Preparing Submission Deliverables

The process of developing new pharmaceuticals and biologics is long-drawn, pricey, and heavily regulated. After drug discovery and years of pre-clinical and clinical research, a regulatory authority, such as the U.S. Food and Drug Administration (FDA), must review all animal and human data regarding a product’s safety, efficacy, and biological mechanisms. Successfully achieving market approval requires not only a safe and effectual therapy but also accurately presenting all of the evidence to prove it works and is safe for use. In this blog series, we will be reviewing some of the most important components necessary for filing a New Drug Application (NDA) or a Biologics License Application (BLA): the deliverables, the people, the timeline, and the budget. Today, let’s start with the deliverables.

Filing an NDA or BLA calls for extensive documentation of the clinical, pharmacological, toxicological, and statistical data collected on a new drug or biologic. All of this information makes for one complex and long-winded application packet that can be delayed for months if anything is missing. With an estimated price tag of $1 million per day that an application is delayed, it is absolutely vital to be sure your NDA or BLA is complete, accurate, and standardized. As your neighborhood biostatistical experts, we at Princeton Pharmatech are here to help you prepare your statistical deliverables for FDA approval.

When it comes time to prepare data files for a regulatory application packet, there are three general rules-of-thumb you can follow to keep your project smoothly sailing through the approval process:

1. ALL data collected during pre-clinical and clinical trials must be submitted.

In the course of researching the biological safety and effects of new pharmaceuticals and biologics, participant data is collected for many variables. Even data that may seem innocuous or unimportant must be provided to the FDA to build the most complete picture of your new product. Additionally, leaving out any relevant safety or efficacy data is a violation of federal patent law and could result in a penalty under perjury. Yikes!

2. Data must be submitted in the standardized format required by the FDA.

Regulatory agencies tend to maintain humongous databases that include all of the information submitted through clinical trials. Requiring a standardized method of structuring this data enables FDA scientists and reviewers to more quickly and efficiently process, review, and store this data. They can also explore new research questions by combining results from multiple studies. All data must be structured in accordance with the standards set forth by the Clinical Data Interchange Standards Consortium, better-known as CDISC. These standards include the Standard Data Tabulation Model (SDTM), which specifies how raw data tables must be formatted, and the Analysis Data Model (ADaM), which describes how data derived from SDTM sets for analytical purposes must be structured.

3. Do NOT hesitate to contact your Regulatory Project Manager (RPM) with any questions or concerns.

The approval process is convoluted and it is nearly impossible for a person to know all of the pertinent regulations. Despite this complexity, the FDA is committed to providing drug sponsors with guidance and advice to facilitate more efficient and robust development programs. Rather than making assumptions about statistical reports and risking the delay of your application, reach out to those who have the answers you need.

There are a lot of moving parts in clinical development and any small hiccup can delay the approval of your therapeutic for weeks. These delays can be avoided by using statistical professionals with extensive experience in regulatory submissions and the application process. Luckily, Princeton Pharmatech has some of the foremost experts in biostatistics, clinical trial design, and data structuring. Contact us today to set up your consultation.

1. ALL data collected during pre-clinical and clinical trials must be submitted.

2. Data must be submitted in the standardized format required by the FDA.

3. Do NOT hesitate to contact your Regulatory Project Manager (RPM) with any questions or concerns.

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