There are a number of laws, regulations, and guidance documents ruling the pharmaceutical and biotechnology industries. Laws are passed by Congress and must be followed by the FDA. The Agency then publishes or issues regulations and publishes guidance documents. So what is the difference between the two?
Although guidance documents are not legally binding, it is extremely important that they are not ignored. The FDA tends to rely on guidance documents as a source of informal policy making. As such, there are a number of extremely important guidance documents that we feel everyone involved in the industry should know, which include:
Published about seven years ago in October 2009, this guidance document summarizes the responsibilities of investigators, which is defined as “a person who conducts a clinical investigation of a drug, biological product, or medical device.”
This guidance document made our list of most important guidance documents because it clarifies FDA’s expectations regarding investigators’ responsibilities and helps investigators to better fulfill these responsibilities and meet the Agency’s expectations.
This guidance was issued as a revised guidance document in the Spring of 2004, and provides recommendations for NDA and ANDA holders trying to make postapproval changes to their application. The document explains what FDA considers to be a major, moderate, and minor change, and is extremely helpful to all sponsors looking to alter any part of an approved NDA or ANDA – which is why it made our list of go-to guidance documents.
Initially released in 2002, the FDA issued an updated version of this draft guidance in May 2016, which provides detailed information on the procedures and general policies that have been adopted by both CDER and CBER for special protocol assessment (SPA).
This draft guidance made our list of most important guidance documents because it provides a number of useful recommendations and some valuable insights regarding meetings with the FDA that are held to discuss the various aspects of the sponsor’s clinical trial plans. Details on this must-know document are critical for anyone preparing to go through the SPA process.
Published to help manufacturers with the implementation of modern quality systems and risk management approaches to meet the FDA’s GMP requirements, this guidance document “describes a comprehensive quality systems (QS) model, highlighting the model's consistency with the CGMP regulatory requirements for manufacturing human and veterinary drugs, including biological drug products.”
Although it is more than ten years old, this guidance document remains one of the most important that FDA has issued and is considered a necessity for anyone in the quality and compliance field.
At about two and a half years old, this guidance document helps facilitate and expedite the review of new drugs addressing an unmet medical need for a serious or life-threatening conditions. The document provides “a single resource for information on FDA’s policies and procedures for these four programs.”
This draft guidance was released by the Agency in September 2005 in order to provide sponsors with recommendations regarding the interpretation of the Pediatric Research Equity Act (PREA) and the pediatric study requirements that accompany it. Under PREA, all sponsors or applicants of new drug applications (NDAs) and biologics licensing applications (BLAs) for a new active ingredient, new indication, new dosage form, new dosing regimen, or new route of administration are required to include a pediatric assessment in their submission.
The draft guidance is of utmost importance to the applicable sponsors or applicants because it clarifies how these individuals can ensure compliance with all of PREA’s requirements, including the pediatric assessment, the pediatric plan (see section V.A), waivers and deferrals, compliance issues, and pediatric exclusivity provisions.
This document was issued as a draft in March of this year, and provides sponsors with information regarding the submission of initial pediatric study plans (iPSPs) as well as any amendments to an iPSP. With this draft guidance, FDA addresses the following topics:
In addition, the document provides a template that should be used when developing an iPSP submission. This, in conjunction with all of the other extremely valuable information provided in the draft guidance, make this a “must have” on the desk of many drug makers involved in the preparation of an iPSP.
This guidance document “provides recommendations for the nonclinical evaluation of previously approved drug substances when a new formulation or a new route of administration for a previously approved formulation is proposed by the sponsor.”
Finalized by the FDA just over a year ago in October 2015, this guidance document is critical for all individuals involved in the development and review of new formulations of previously approved drug substances, making it a must-have on our list of important guidances.
FDA finalized this guidance document in February 2013 with the intention of assisting applicants in “complying with the content and format requirements of labeling for human prescription drug and biological products under 21 CFR 201.56(d) and 201.57.”
The recommendations included in the guidance apply to applicants who are developing labeling for new prescription drugs or revising existing labeling for prescription drugs that have already received FDA approval. In addition, recommendations for developing Highlights of Prescribing Information, formatting labeling, and procedural information are also included in the guidance.
With all of this helpful information, when it came time to decide what to include in our list of most important guidance documents, this one was a no brainer.
And last but not least, issued by the FDA on March 10, 2015, this draft guidance is of particular importance to anyone planning a meeting with FDA related to the development and review of a drug or biological product that is regulated by the Agency’s Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER). However, it is important to note that the recommendations contained in the draft guidance do not apply to sponsors of abbreviated new drug applications (ANDAs), biosimilar biological product submissions, or medical device submissions.
This document is important because it provides recommendations regarding the principles of good meeting management practices (GMMPs) and describes how sponsors or applicants should go about requesting, preparing, scheduling, conducting, and documenting such formal meetings with the FDA.
While an overview can sometimes be great, we feel that these guidance documents are so important that they deserve more details. As such, we have individual blog posts dedicated to each of these guidance documents coming over the course of the coming weeks. Stay tuned, you won’t want to miss out on any of this vital information.
January 24, 2017
On March 8, 2016, the FDA announced availability of a revised draft guidance for industry regarding pediatric study plans, entitled “Pediatric Study Plans: Content of and Process for Submitting...
May 15, 2020
In March 2020, the FDA published a guidance entitled “Conduct of Clinical Trials of Medical Products during COVID-19 Public Health Emergency - Guidance for Industry, Investigators, and Institutional...
July 17, 2018
On Wednesday, July 11th, FDA released six draft guidances to aid in the development of new gene therapies. Of the six guidances, three were disease specific and three were related to manufacturing....