November 17, 2023 BioPharma - Regulatory


FDA Publishes Two Final Guidances on Technical Specifications for Clinical Data

The FDA published a pair of final guidances Friday meant to assist drugmakers by providing technical specifications for submitting general clinical outcome assessment (COA) data and patient-reported outcome (PRO) data collected from cancer studies, respectively.

FDA Needs to Take Direct Action to Combat Effects of Medical Misinformation

Seeking to address the growing problem of how misinformation undermines confidence in both science and the FDA, the Regan-Udall Foundation recently released a report focusing on how the FDA can deliver clear messaging to deliver improved understanding of the FDA and the products it regulates.

Form 483 Responses Must Include Adequate Corrective Actions, Says FDA Official

Though there is no regulation requiring they do so, companies that receive an FDA Form 483 should always provide a response in writing that is well-organized, comprehensive and includes adequate corrective actions to addresses deficiencies, says Rebecca Asente, an FDA compliance officer with the Office of Regulatory Affairs. “I’ve received responses that were out of order such that I had to contact the firm for a corrected, organized response package,” Asente remarked during an FDA webinar, What to Expect after an Inspection: 483s, Responses and Beyond, posted to the agency’s YouTube channel.

Drug Facility Remote Inspections is Focus of Draft Guidance

In a new draft guidance, the FDA describes how it requests and conducts voluntary remote regulatory assessments (RRA), an evaluation the agency says it may conduct in lieu of or in support of an inspection.

Communications on Unapproved Use of Approved Products is Focus of Draft Guidance

The FDA has issued a draft guidance intended to strike a balance between health care providers’ interest in scientific information on unapproved uses of approved/cleared medical products and mitigating the FDA’s interest in prohibiting use of a product for an unapproved indication.

FDA Clarifies Risk-Benefit Assessment During Pre- and Postmarket Considerations

The FDA provides clarity for drugmakers in a final guidance on how the agency’s considerations about a drug’s risks and benefits factor into NDA and BLA premarket and postmarket decisions.

FDA Shares Clinical Trial Considerations for Diabetic Foot Infection Drugs

The FDA’s new draft guidance provides recommendations on overall development programs for drugs to treat diabetic foot infections (DFI) as well as clinical trial designs.

FDA Guidance Shares Quality Considerations for Ophthalmic Drugs

The FDA’s newest draft guidance discusses quality considerations for ophthalmic drugs, including ways to assess impurities, evaluate visible contaminants, design containers and conduct stability studies. The 15-page draft guidance shares ways to evaluate visible particulate matter, extractables and leachables from closed container systems as well as the use of in vitro drug release and dissolution testing for quality control.

Data-Sharing, AI, Collaboration Prioritized in FDA IT Modernization Plan

The FDA’s plan to modernize its IT infrastructure features several improvements that will benefit clinical trials and sponsors, including upgrades to data-sharing and electronic submissions, support for responsible use of AI/machine learning (ML) and increased collaboration between the agency and industry. The strategy for FY 2024 to 2027 acknowledges the growing importance of technology in drug and device development, as well as the challenges that accompany fast-paced scientific progress and innovation. IT-focused upgrades will be needed to keep up with ever-evolving technology and therapeutics, says Vid Desai, chief information officer for the agency’s Office of Digital Transformation.

Speedier Approval is Goal of FDA Revision of MAPP for ANDA Assessment Practices

The FDA has revised the Manual of Policies and Procedures (MAPP) for ANDA assessment practices with changes intended to decrease the number of review cycles needed for approval.



First electronic product information (ePI) published for selected human medicines

The Heads of Medicines Agencies (HMA), the European Commission (EC) and EMA have published for the first time electronic product information (ePI) for selected human medicines harmonised across the European Union (EU). The product information of a medicine includes its summary of product characteristics, labelling and package leaflet. These documents accompany every medicine authorised in the EU and explain how they should be prescribed and used. They can all be found, often as a PDF document, on the websites of EU regulators, with a printed package leaflet also provided in the medicine’s box. Digital platforms open new possibilities to share this information electronically, keep it constantly updated and make it more accessible to end users such as healthcare professionals and patients.

How to create, submit and withdraw a CTA CTIS Training Programme – Module 10 is updated

The European Medicines Agency developed this training material to enhance public access to information on the Clinical Trial Information System (CTIS). This material describes a preliminary version of CTIS and may therefore not entirely describe the system as it is at the time of use of this material. The Agency does not warrant or accept any liability in relation to the use (in part or in whole) or the interpretation of the information contained in this training material by third parties.

EMA encourages companies to submit type I variations for 2023 in November 2023

EMA is advising marketing authorisation holders to submit type IA and type IAIN variations for 2023 no later than Thursday, 30 November 2023. This will enable EMA to acknowledge the validity of the submissions before the Agency’s closure between 25 December 2023 and 3 January 2024 and within the 30-day timeframe set out in Article 14 of Commission Regulation (EC) No 1234/2008. Marketing authorisation holders are advised to submit any type IB variations or groupings of type IBs and type IAs by 30 November 2023 for a start of the procedure in 2023. For submissions received on or after 1 December 2023, the procedure may not start until January 2024.

Monitoring of products originating from Japan for the possibility of radioactivity

EMA and EU national competent authorities are no longer requiring the monitoring of medicines manufactured in Japan for possible risks of radioactive contamination following the 2011 accident at the Fukushima nuclear power plant. This is in line with the withdrawal of the Commission Implementing Regulation (EU) No 297/2011 and the subsequent revisions. The European Medicines Agency is working with its European and international regulatory partners to monitor and evaluate the possible risk of radioactive contamination of medicines manufactured in Japan following the radiation leak from the Fukushima Daiichi nuclear power plant.

Revised transparency rules for the EU Clinical Trials Information System (CTIS)

EMA has adopted PDF icon revised transparency rules for the publication of information on clinical trials submitted through the Clinical Trials Information System. The simplifications introduced will give access to clinical trial information to stakeholders including patients and healthcare professionals in a faster and more efficient way. One of the key changes of the revised rules is the removal of the deferral mechanism, which allowed sponsors to delay the publication of certain data and documents for up to seven years after the end of the trial to protect personal data and commercially confidential information (CCI).

Fee Application Form for Human Products in Ireland

HPRA published Fee Application Form for Human Products fin-f0018. This includes application form, fee codes, fee for different types.



HSA Singapore Designated as WHO-Listed Authority for its Medicines Regulatory System

The Health Sciences Authority (HSA) has been designated by the World Health Organization (WHO) as a WHO-Listed Authority (WLA) on 26 October 2023 for its medicines regulatory system. This means that HSA is now recognised globally as operating at an advanced level of regulatory performance, ensuring that medicines in Singapore meet high safety, efficacy and quality standards. In January 2022, HSA had attained the WHO Maturity Level 4[1] based on our operational efficiency. The subsequent assessment for WLA included a thorough review of the quality of regulatory assessments, the scientific soundness of the decision-making and their consistent implementation. WHO’s comprehensive assessment of HSA’s core functions and operations, conducted by a team of 10 international assessors and WHO officials, took place from October 2022 to March 2023.

DRAP invites Comments on Draft Guideline for Storage and Destruction of Substandard-Falsified (SF )and recalled Therapeutic Goods

The Drug Regulatory Authority of Pakistan (DRAP) has drafted a guideline on the storage and destruction of recalled therapeutic goods, which are medicines, medical devices, and health products that do not meet the quality standards or are deliberately misrepresented. The guideline aims to provide a framework for the safe and effective management of such goods, from their identification and reporting to their disposal and documentation.

DRAP Added New eService for Online Data Submission for Registration of Pharmaceutical and Biological Product

The Drug Regulatory Authority of Pakistan (DRAP) is pleased to announce the addition of a new e-service for online regulatory data submission registration/marketing authorization of human drugs for local manufacturing, import and export. The Licensing Operations of all [pharmaceutical and biological manufacturers are already enabled through the same platform. This e-service is part of the transformation strategy of the Drug Regulatory Authority of Pakistan which aim to ensure the quality, safety and efficacy of therapeutic goods in Pakistan. The e-service requires applicants to submit their data in a standardized format.

NMPA becomes the applicant for PIC/S

In late September 2023, the National Medical Products Administration (NMPA) submitted the formal application to the Pharmaceutical Inspection Co-operation Scheme (PIC/S). On Nov 8, 2023, PIC/S sent a letter to NMPA confirming the applicant status. Following this confirmation, NMPA will strengthen communication and cooperation with the PIC/S, promote itself to become a full member of PIC/S, and take this opportunity to continuously improve the country’s drug inspection system and standards, to constantly enhance its drug inspection quality management system and to steadily promote the development of its inspectors’ team, so as to modernize the national drug regulation in China.