Introduction

The United States FDA regulates pharma drug products manufactured or marketed in the United States. There are many different functions of the FDA, and one of them is devising new regulations. These new regulations come into existence in response to various factors, such as

· Feedback from different stakeholders

· Complains

· Innovation & development in the pharma industry.

For continuous operations, it is necessary for every pharma manufacturer to comply with these regulations They can face warning, fines or even shutting down of their operations in an event of non-compliance.

These regulations are made available on its website for public view.

Some guidance documents in the year 2024

Let’s take a brief overview of the regulations made in the year – 2024

Advanced Manufacturing Technologies Designation Program

This guidance is about advanced manufacturing technologies in the pharma industry. The FDA acknowledges the importance of these technologies towards the reliability of the process, product quality, and reduction of development time, especially for life-supporting drugs.

This guidance provides recommendations for individuals or organizations to participate in the FDA’s Advanced Manufacturing Technologies Designation Program, which facilitates the implementation of TMA.

M15 General Principles for Model-Informed Drug Development

This FDA guidance provides guidelines related to Model Informed Drug Development – MIDD. This development program uses different quantitative methods to reduce risks during the development program.

This guidance is prepared under the auspices of ICH and discusses different areas of MIDD, such as planning, model evaluation and evidence documentation.

E11A Pediatric Extrapolation

This guidance is also prepared under the auspices of ICH and focuses on pediatric extrapolation during drug development. It contains approaches to study design and statistical methodologies, such as modelling & simulation.

This guidance intends to increase the efficiency of pediatric drug development and enhance safe & effective pediatric drug availability.

Protocol Deviations for Clinical Investigations of Drugs, Biological Products, and Devices

This guidance is intended for sponsors, clinical investigators, and institutional review boards to define, identify, and report protocol deviation during clinical investigation.

It includes definition of protocol deviation, recommendations for sponsors to report deviations to the FDA, types of deviations that investigators should report to sponsors & IRBs, and for IRBs when evaluating protocol deviation

Accelerated Approval – Expedited Program for Serious Conditions

The purpose of these guidelines is to help provide FDA policies about accelerated approval, which is an expedited program of the FDA to facilitate the development of new drugs with high and urgent needs.

This guidance also covers the expedited withdrawal of a product approved under an accelerated approval program and revisions Congress made through the Consolidated Appropriations Act, 2023 (Public Law 117-328)

Requests for Reconsideration at the Division Level Under GDUFA

This guidance provides guidance related to the applicants of abbreviated new drug applications (ANDAs) who wish to pursue reconciliation within the review discipline at the division level or original signatory authority.

Temporary Policies for Compounding Certain Parenteral Drug Products

This guideline describes the FDA's thinking on compounding certain parenteral drug products by outsourcing facilities, State-licensed pharmacies, and non–registered outsourcing FDA Federal facilities.

Electronic Systems, Electronic Records, and Electronic Signatures in Clinical Investigations: Questions and Answers

This guidance is for sponsors, clinical investigators, contract research organizations, and others who use electronic systems, records, and signatures for foods, medical, tobacco, and new animal drugs.

It depicts the FDA requirement for the above-mentioned electronic systems, records & signatures to remain trustworthy, reliable, and generally equivalent to paper records and handwritten signatures executed on paper.

Conducting Clinical Trials with Decentralized Elements

Decentralized trials allow trial activities to be performed at remote locations, including

· Tele health visits with trial personnel

· In-home visits with remote trial personnel, or

· Visits with local healthcare providers.

This guidance helps sponsors, investigators, and others related in  using and applying decentralized elements in clinical trials.

Providing Regulatory Submissions in Electronic Format — Certain Human Pharmaceutical Product Applications and Related Submissions Using the eCTD Specifications Guidance for Industry

This guidance concerns the sponsors and investigators about the format and organization of content submitted to the agency electronically under section 745A(a) of the FD&C Act, which requires the submission of electronic formats of submissions.

Providing Over-the-Counter Monograph Submissions in Electronic Format

This guidance describes the electronic over-the-counter (OTC) monograph submissions required in the Federal Food, Drug, and Cosmetic Act (FD&C Act). It also recommends sending OTC monograph submissions to the FDA in electronic format. 

Post approval Manufacturing Changes to Biosimilar and Interchangeable Biosimilar Products Questions and Answers

This guidance answers commonly asked questions from all interested parties for post-approval manufacturing changes made to licensed and interchangeable biosimilars.

This guidance aims to help applicants provide all relevant information supporting manufacturing changes to licensed biosimilars and interchangeable biosimilars.

Purpose and Content of Use-Related Risk Analyses for Drugs, Biological Products, and Combination Products

This guidance is intended for the industry and the FDA staff. It provides advice regarding use-related risk analysis and how it can be used along with other information to determine human factors data needed during product development.

M14 General Principles on Plan, Design, and Analysis of Pharmacoepidemiological Studies That Utilize Real-World Data for Safety Assessment of Medicines

Prepared under the auspices of ICH, and outlines general principles related to  planning, designing, and analyzing observational (noninterventional) pharmacoepidemiological studies that utilize fit-for-purpose data for safety assessment of medicines

It includes high-level best practices for conducting these studies, aims to streamline the development and regulatory assessment of postmarketing pharmacoepidemiological safety studies 

Facility Readiness: Goal Date Decisions Under GDUFA

This guidance informs applicants of assigning a goal date based on the facility’s readiness for inspection as certified on Form FDA 356h.

It also explains how the FDA incorporates program enhancement, as agreed by the FDA and industry during the negotiations relating to the reauthorization of the Generic Drug User Fee Amendments (GDUFA).

Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics

This guidance recommends developing oligonucleotide therapeutics under section 505 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355) and 21 CFR parts 312 and 314.

Special attention is made to certain evaluations, including

· Characterizing the potential for QTC interval prolongation

· Performing an immunogenicity risk assessment

· Characterizing the impact of hepatic and renal impairment

· Assessing the potential for drug-drug interactions. 

This guidance also helps  in determining when to conduct a suitable assessment to address the above-mentioned areas

Content and Format of Composition Statement and Corresponding Statement of Ingredients in Labeling in NDAs and ANDAs

This guidance assists new drug applications and abbreviated ones in submitting complete and accurate composition statements and statements of ingredients in labelling. It can help applicants minimize the assessment cycle, and communicate for approval by providing the full information.

Data Integrity for In Vivo Bioavailability and Bioequivalence Studies

This guidance is a recommendation for applicants for data integrity for the following.

· Clinical and bioanalytical portions of bioavailability (BA) and bioequivalence (BE) studies submitted in support of investigational new drug applications (INDs)

· New drug applications (NDAs)

· Abbreviated new drug applications (ANDAs)

· Bioanalytical portion of clinical pharmacologic studies supporting CDER-regulated biologic license applications (BLAs)

· Amendments and supplements to these applications

Real-World Evidence: Considerations Regarding Non-Interventional Studies for Drug and Biological Products

This guidance is a part of the Real World Evidence – RWE program about using RWE in regulatory decision-making. It provides guidance to sponsors considering submitting a non-interventional study to demonstrate the evidence of the drug's effectiveness and/or safety.

Controlled Correspondence Related to Generic Drug Development

This guidance is for the industry when submitting generic drug development information requests. It can also be used to follow a process by industry to clarify ambiguities in the FDA’s controlled correspondence response and the process for responding to those requests. 

Annual Reportable Labeling Changes for New Drug Applications and Abbreviated New Drug Applications for Nonprescription Drug Products

This guidance is for applicants of New Drug Applications – NDA and Abbreviated New Drug Applications – ANDAs. It provides recommendations for nonprescription drug products for documenting minor changes in their labelling, submitted in the annual report.

In this guidance, the FDA addresses the label changes appropriate for submission so that consumes have up-to-date information for nonprescription drug product’s safe and effective use

Q2(R2) Validation of Analytical Procedures

This guidance is prepared under the auspices of ICH and provides a framework for analytical procedure validation principles. This guidance is developed to facilitate regulatory evaluations and flexibility in post–approval change management of analytical procedures.

This guidance replaces the previously developed draft guidance “Q2(R2) Validation of Analytical Procedures” and” Q14 Analytical Procedure Development” issued on August 29, 2022.

Notifying FDA of a Discontinuance or Interruption in Manufacturing of Finished Products or Active Pharmaceutical Ingredients Under Section 506C of the FD&C Act

This guidance is in its draft form and is about informing the FDA about the changes in the production of certain finished products, including biological products and APIs, that may help the FDA prevent shortages.

Requests for Reconsideration at the Division Level Under GDUFA

This guidance provides recommendations about the procedures for applicants of abbreviated new drug applications – ANDA who wish to request reconsideration within the review discipline at the division level or original signatory authority.

This guidance replaces the draft guidance for industry of the same title issued on January 11, 2024.

About the Author:

Muhammad Asim Niazi

Muhammad Asim Niazi has a vast experience of about 11 years in a Pharmaceutical company. During his tenure he worked in their different departments and had been part of many initiatives within the company. He now uses his experience and skill to write interested content for audiences at PharmaSources.com.