Regulatory Focus™ > News Articles > Global Harmonization Initiatives, FDA's User Fee Programs, Pediatric Legislation, Conditional Approv

Global Harmonization Initiatives, FDA's User Fee Programs, Pediatric Legislation, Conditional Approvals, Quality Certificates and a Preview of June

Posted 06 June 2017 | By Gloria Hall 

Global Harmonization Initiatives, FDA's User Fee Programs, Pediatric Legislation, Conditional Approvals, Quality Certificates and a Preview of June

Feature articles over the past several weeks have highlighted various global harmonization initiatives, including the benefits of a new regulatory tool for improving operational flexibility when changes are introduced post-approval and progress with FDA's and EMA's pediatric regulatory activities. Other articles presented changes to and the industry impact of PDUFA, MDUFA and GDUFA, regulatory pathways to expedite the review of new drugs and the electronic Common Technical Document (eCTD) submission process for drugs marketed internationally.


Government bodies, non-governmental associations and trade organizations all play a role in regulatory harmonization and convergence in an effort to align regulatory requirements and adopt internationally recognized guidance documents, standards, technical and scientific principles, and best practices and procedures. In their article, "Regulatory Harmonization Initiatives," Kumar, Barras and Lau provide an overview of global regulatory harmonization initiatives and address how various regulatory and other organizations are working toward improving access to pharmaceuticals and medical devices and increasing quality through internationally harmonized inspection standards.

Over the past two decades, considerable progress has been made in developing drugs for pediatric populations and clinical trials for testing. Legislative requirements, as well as harmonization and convergence efforts between the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), have set the global stage for expanding this effort. Sanguedolce and Mulchan present pediatric regulatory activities conducted by FDA and EMA and highlight global harmonization efforts, collaborative discussions and regulations for pediatric clinical trials in their article, "Regulating Pediatric Studies: An EU and US Perspective."

The International Council for Harmonisation (ICH) developed a vision for harmonizing quality systems across the product lifecycle with an emphasis on an integrated approach to quality risk management and science. In their article, "ICH Q12 Post Approval Change Management Protocol: Advantages for Consumers, Regulators and Industry," Pazhayattil, Sayeed-Desta and Iyer discuss the latest draft of ICH Q12 and the significance of harmonizing regulatory requirements across regional borders. The authors explain how Post-Approval Change Management Protocol (PACMP) can be used as a tool to improve strategic change management and ensure supply chain reliability.

Policy and Guidance

Prior to the passing of the Prescription Drug User Fee Act (PDUFA), FDA experienced backlogs in its drug approval process with very long lead times to review and approve submissions. These backlogs kept companies around the world from launching approved drugs in the US market in a timely way. Both industry and FDA became frustrated with the slow drug approval. In the article, "FDA User Fee Programs," Gottfried, Maguire, Chooi, Eldridge and Gottfried provide an overview of upcoming important changes to FDA's major user fee programs (PDUFA VI, MDUFA IV and GDUFA II) for Fiscal 2018–2022 and discuss new performance goals, additional commitments FDA has agreed upon with industry and how these changes could impact various types of industry submission strategies.

The generic drug industry and the market for generic drugs have changed significantly over the last several decades with the increase in the number of Abbreviated New Drug Applications (ANDAs) and large number of foreign facilities currently manufacturing generic drugs. As a result, FDA's generic drug program is under-resourced and unable to keep up with new generic drug review and approval processes. In her article, "Generic Drug User Fee Act (GDUFA): Overview and Proposed Changes for 2018," Mark-Lantz explores aspects of the GDUFA enacted by Congress as part of the Food and Drug Administration Safety and Innovation Act (FDASIA), including fees, changes enabling FDA to speed up reviews, GDUFA goals and proposed changes and impacts as the first five–year phase concludes in 2017.

FDA reviews new medical device applications and regulates medical devices in the marketplace. With the passage of the Medical Device User Fee and Modernization Act in 2002, FDA was empowered to collects user fees from industry to help facilitate its medical device review, regulation activities, responsibilities and meet its commitment to Congress regarding the application of timely resources in the effort to get medical devices to the patients who need them. In the article,"Navigating the Medical Device User Fee Act (MDUFA)," Gottfried, et al, examine the benefits for both FDA and the medical device industry derived from "user fees" authorized by the passage of MDUFA and its subsequent reauthorizations.

Providing healthcare aid to civilians during war and crisis is a complicated task. The Syrian war has impacted the healthcare system inside Syria and the neighboring countries. Sahloul discusses the difficulties encountered by medical personnel working with refugees in Syria and delivering pharmaceutical products. She describes relevant regulatory policy and guidance for drug procurement and quality in her article, "Challenges Providing Pharmaceutical Products to Syrian Refugees."

Before a medicinal product for human use is authorized to enter the market, it must undergo extensive study to ensure its safety, quality and effectiveness. However, there is a growing call from both healthcare professionals and patients—many with life threatening and debilitating diseases—for earlier access to new medicines. In response, regulatory agencies have devised various pathways to expedite the review of new drugs and acquire Conditional Approvals (CAs). In her article, "Conditional Approvals for Early Access to New Medications" Arora sheds some light on Conditional Approvals (CAs) and Conditional Marketing Approvals (CMAs) as developed across several jurisdictions to potentially provide earlier access to new medicines. The author explains the specifics regarding regulatory requirements for obtaining CAs in the European Union (EU), Japan and the US.

From clinical trials to marketing authorization, regulatory dossiers for small molecule drug products are incomplete without the presentation of certain regulatory certificates, an important part of Module 1 of the electronic Common Technical Document (eCTD) submission process. In her article, "Quality Certificates for Small Molecule Drug Product Applications for International Regulatory Submissions," Jain provides a global overview of the general requirements and issues regulatory professionals may encounter while arranging for a variety of certificates for applications for small molecule drug products to be marketed internationally in the EU, US, Canada, Australia and Japan.

Regulatory Research

According to some sources, there is a growing threat of bacteria that can resist all or nearly all antibiotics. Today, microbes appear to be outpacing science's capacity to develop new human defenses. New antibiotics are desperately needed. In his article, "Medicine's Unsung Heroes," Sherman introduces readers to Boyd Woodruff, a legend in the field of microbiology, and discusses his groundbreaking achievements in antibiotic development and Merck's research laboratories.

Feature Articles Coming Throughout June

Feature articles throughout the month of June will present an overview of the New Drug Application (NDA) process in Egypt, various types of inspections conducted by FDA, IT approaches to support the gap analysis and implementation of the Identification of Medicinal Products (IDMP) standards, naming conventions for biological products and biosimilars, and challenges of risk management in the medical device industry. Look for these feature articles throughout June.

If you have a suggestion for a feature article, contact me at

Categories: RAPS' Latest

Regulatory Focus newsletters

All the biggest regulatory news and happenings.