Regulatory Affairs Resource Library

Bring your molecule to market with insights to advance your program along the relevant regulatory affairs pathway. Our expert consulting team dives into a variety of strategies in the resources below to provide you with the full scope of possibilities ahead. For more information about our services, reach out to a member of our team to discuss the offerings most relevant to you.

Feature Webinar

Overview of FDA’s Expedited Programs for Serious Conditions

Serious or life-threatening diseases remain a large unmet medical need in the United States, as many do not currently have effective therapies. As innovative therapies are increasingly researched and developed, including regenerative medicine therapies, it is important to expedite such treatments to ensure patients are treated as quickly as possible. Thus, the FDA expedited review programs, such as the Fast Track Designation (FTD), Breakthrough Therapy Designation (BTD), and Regenerative Medicine Advanced Therapy (RMAT) Designation, are vital for increasing the likelihood for advancing an investigational product to market. This presentation will focus on what these expedited review programs are, the incentives for obtaining such designations, and the required information/data to better position your investigational product for success.

On-Demand webinar

Navigating the Investigational New Drug Application Process

The investigational new drug (IND) application is an important milestone in drug development. The aim of an IND is to demonstrate every aspect of the drug is safe for human exposure. It is important for sponsors to evaluate IND readiness to ensure they have enough data to support initiation of clinical trials. Submission of an incomplete IND lacking important studies or data can lead to a clinical hold, thus delaying clinical trials and increasing costs for the sponsor. This presentation will explore the drug development process with specific insights on FDA’s regulations, including an overview of the required content for a successful IND application and recommended interactions with the Agency to ensure success.

Orphan Drug Designations (ODD) in the US & the EU

Orphan drugs are therapeutic agents developed to treat rare diseases. In the US, the Food and Drug Administration (FDA) determines rare medical conditions are those with a prevalence below 200,000 people for a potential drug to be eligible for designation. In the EU, the European Medicines Agency (EMA) designated rare medical conditions as those with a prevalence below 5 in 10,000 people within the EU. Orphan designation programs at the FDA and the EMA offer incentives for sponsors to stimulate drug development for rare diseases, which otherwise would not be profitable due to small patient populations. Additionally, the FDA offers rare pediatric disease (RPD) designation and priority review voucher programs to further incentivize drug development for rare diseases that primarily affect the pediatric population. This presentation will focus on what the orphan designation and RPD programs are, the incentives for obtaining such designations, and the required data needed to position your drug for regulatory success.

FDA eCTD Submissions: Compilation and Publishing

Making an electronic common technical document (eCTD)-compliant submission to the FDA requires complex coordination of stakeholders and a detailed culmination of product information, providing the necessary information to FDA while also optimizing the submission package for ease of review. This presentation will cover the anatomy of an eCTD submission, starting with the structure of the eCTD backbone and its 5 modules, various applications of the eCTD format, the compilation process, validation, and submission to FDA using their electronic submissions gateway (ESG) WebTrader service. Additionally, the talk will cover common challenges, including information requests, validation errors, digital dataset files, submission navigation and readability, and document optimization.

whitepapers

eCTD-Compliant Publishing & Submissions

The common technical document (CTD) is a globally accepted submission format developed by the International Council for Harmonization (ICH). In this whitepaper, we discuss the structure and different modules used, formatting when writing applications, and additional considerations.

How Consultants Can Assist Throughout the IND Application Process

The Investigational New Drug (IND) application is one of the most important applications in a drug’s life cycle, as it is a prerequisite for performing clinical trials in humans. In this whitepaper, we discuss how consultants can offer additional support and expertise throughout the IND application process.

The FDA’s Orphan Drug Designation

The Orphan Drug Act of 1983 created an FDA application program known as Orphan Drug Designation (ODD), which supports innovation through incentivizing the research and development of products for orphan diseases. In this whitepaper, we discuss crafting a successful ODD, reviewing the application and maintaining designation, and the benefits of ODD.

The FDA’s Regenerative Medicine Advanced Therapy (RMAT) Program

The Food and Drug Administration (FDA) offers a program called the Regenerative Medicine Advanced Therapy (RMAT) designation to expedite the availability of promising therapies for patients. In this whitepaper, we discuss the growing field of regenerative medicine, the FDA’s expedited review program, how to prepare a successful RMAT designation request, and the role of a regulatory affairs consulting firm.

The FDA’s Breakthrough Therapy Designation

The Breakthrough Therapy Designation (BTD) is a unique expedited drug development program created by the US Food and Drug Administration (FDA) in 2012 through the Food and Drug Administration Safety and Innovation Act (FDASIA) Section 902. In this whitepaper, our experts discuss crafting a successful Breakthrough Therapy Designation, reviewing your application, maintaining designation, and leveraging the benefits of BTD.

The EMA’s Orphan Drug Designation

Following the FDA’s successful implementation of the Orphan Drug Designation (ODD) program in the US in 1983, the European Medicines Agency (EMA) introduced its own version in 2000 to support the development of treatments for rare diseases across the EU. In this whitepaper, our experts explain EMA ODDs, discuss the incentives of pursuing this designation, and provide insight into the application process.

The FDA’s Rare Pediatric Disease Designation

The Rare Pediatric Disease (RPD) Designation Priority Review Voucher (PRV) program was created to further improve novel drug development for pediatrics by offering sponsors additional benefits. In this whitepaper, our expert reviews the origin of the RPD designation program, key definitions, the application process, and the benefits and responsibilities of obtaining designation.

The FDA’s Qualified Infectious Disease Product (QIDP) Designation

The GAIN Act encourages the development of products to treat antibiotic resistant infections and provides incentives and additional guidance to pharmaceutical companies to develop Qualified Infectious Disease Products (QIDPs). In this whitepaper, our expert outlines how to craft a successful QIDP designation application, what advantages designation offers, and why it’s worth considering specialized support.

The FDA’s Fast Track Designation

In 1997, the Food and Drug Administration (FDA) created the Fast Track Designation (FTD) program through the Food and Drug Administration Modernization Act. This designation facilitates faster drug development for serious diseases or conditions that are debilitating or life-threatening by enabling more direct communication with the FDA during the drug development process. In this whitepaper, our experts cover the qualifications necessary for applying, the application process, and the key opportunities to take advantage of once designation is granted.

About Pace® Life Sciences

Pace® makes the world a safer, healthier place. Pace® people are committed to advancing the science of our customers in the pharmaceutical and biopharmaceutical industries. The therapies our customers develop improve patient lives and we are committed to supporting them through all phases of development.

Through our nationwide network of world-class CDMO and CRO sites, Pace® supports customers from early-phase R&D to clinical trial materials production and ongoing commercial product GMP laboratory support. For our customers with manufacturing facilities and in-house labs, Pace® provides a wide range of professional services to keep their operations moving forward.