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Report of a one-day convening on regulatory science, practices, and innovative approaches to facilitate approval of novel combination vaccines.

Combination vaccine formulations contain distinct components targeting multiple strains of a single pathogen or multiple pathogens. By minimizing the number of separate vaccine administrations required, combination vaccines have been critical in allowing the broad expansion of the number and range of diseases that can now be prevented by immunization. Recent advances in vaccine development and our understanding of the immune system now make it possible to envision how new combination vaccines could play a major role in helping immunization programs address a much wider range of emerging or sti

Efforts of the Pharmaceuticals and Medical Devices Agency of Japanese regulatory agency in supporting biosimilar development and disseminate information.

Information on biosimilars was rarely disseminated on the Pharmaceuticals and Medical Devices Agency (PMDA) website of the Japanese Regulatory Agency until the fiscal year (FY) 2022. Therefore, the PMDA website for biosimilars was created in FY 2023. This study confirmed the PMDA website for biosimilars and surveyed information about the approval fiscal year, brand name, indications at the initial approval, type of biopharmaceuticals, and disease area at the initial approval based on review reports and the "List of Approved Products." The PMDA website for biosimilars provides information on th

Gaps, tensions, and conflicts in the FDA approval process: implications for clinical practice.

Despite many successes, drug approval at the Food and Drug Administration (FDA) is subject to gaps, internal tensions, and conflicts of interest. Recalls of drugs and devices and studies demonstrating advantages of older drugs over newer ones highlight the importance of these limitations. The FDA does not compare competing drugs and rarely requires tests of clinical efficacy for new devices. It does not review advertisements before use, assess cost-effectiveness, or regulate surgery (except for devices). Many believe postmarketing surveillance of drugs and devices is inadequate. A source of te…

The year's new drugs & biologics - 2008.

This annual article presents new drugs and biologics that were launched or approved for the first time during the previous year. In 2008, 31 new medicines-this figure includes both drugs and biologics for therapeutic use as well as new diagnostic agents-reached their first markets. Line extensions (new indications, new formulations and new combinations of previously marketed products) accounted for more than one-third of the new medicines launched in 2008. In addition to providing an overview of all drugs and biologics launched or approved for the first time ever in the previous year, this art

Development Timelines of Innovative Drugs in China: A 15-Year Cross-Sectional Analysis of Characteristics and Influencing Factors.

Timely development and approval of innovative drugs are essential for patient access and public health. China has implemented regulatory reforms, including priority review and conditional approval, to accelerate drug development. We conducted a cross-sectional study of 232 innovative drugs, comprising chemical drugs, biologics, and traditional Chinese medicines (TCMs), approved by the National Medical Products Administration during 2010-2025. Development timelines for 227 drugs were analyzed using generalized linear models with a gamma distribution and log link to assess associations with drug

Unapproved prescription cough, cold, and allergy drug products: recent US Food and Drug Administration regulatory action on unapproved cough, cold, and allergy medications.

The US Food and Drug Administration (FDA) drug approval and over-the-counter drug monograph processes play an essential role in ensuring that all drugs are both safe and effective for their intended uses. Manufacturers of drugs that lack required approval have not provided the FDA with evidence demonstrating that their products are safe and effective. Some of these prescription drugs have been marketed for many years and have remained on the market despite changes to the Federal Food, Drug, and Cosmetic Act, which requires approval for safety and efficacy purposes. Many health-care providers m…

Expanding Quantitative Medicines' Reach: Systems and Methods to Enhance R&D Productivity and Decision-Making in the Agentic-Era.

Despite integration of artificial intelligence (AI) into drug discovery and an expanding global medicines pipeline, new drug approvals have declined, highlighting a paradox in biopharma: More drugs discovered, but less approvals, higher costs, and longer timelines. Reasons for the decreased research and development (R&D) efficiency are multifactorial, in part driven by the complexity of new modalities, difficult targets and indications, and persistence of cognitive biases in clinical decision-making. One proposed solution to address R&D productivity challenges includes the adoption of organiza…

[Pharmacotherapeutical possibilities in mental disorders: current status in Serbia].

The new therapeutical approaches have direct implications on living in order to accomplish remission, stop further progression of illnesses or improve the quality of life. The life expectancy has been increased up to 10 years in the last 55 years, probably owing to the innovative drugs. The innovative drugs application in our everyday clinical practice should be analyzed, related to economical aspects and to the transitional status of a country, but also in the context of fears and misconceptions of therapists. The cost of therapy is not equal to the cost of prescribed drugs, however it also i

Navigating the Prescription Drug Information System.

Information on new prescription drugs is increasingly complex and fragmented, posing challenges for healthcare professionals, patients, and … advertising, which coexist with regulated content. Against a backdrop of increasing uncertainty of drug approval evidence, we identify documented principles that regulators can adopt to meet drug information needs. Recommendations for improvements aim to better serve patients, support clinical decision making, and promote ethical healthcare resource allocation …

Tavaborole: first global approval.

Tavaborole is a novel, low-molecular weight oxaborole antifungal drug under development by Anacor Pharmaceuticals Inc. for the topical treatment of onychomycosis of the toenail. The drug has received its first global approval for this indication in the US. This article summarizes the milestones in the development of tavaborole leading to this first approval for onychomycosis of the toenails.

An appraisal of drug development timelines in the Era of precision oncology.

The effects of incorporating a biomarker-based (personalized or precision) selection strategy on drug development timelines for new oncology drugs merit investigation. Here we accessed documents from the Food and Drug Administration (FDA) database for anticancer agents approved between 09/1998 and 07/2014 to compare drugs developed with and without a personalized strategy. Sixty-three drugs were included (28 [44%] personalized and 35 [56%] non-personalized). No differences in access to FDA-expedited programs were observed between personalized and non-personalized drugs. A personalized approach

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… Incorporating biomarkers for treatment selection in cancer therapy holds the promise of enhancing efficacy of new treatments and improving the success of drug approvals [ 6 ]. … Information about drug approvals and access to FDA expedited programs were also compared with contemporary literature [ 29 ]. …

Expanded Patient Access to Investigational New Devices: Review of Emergency and Nonemergency Expanded Use, Custom, and 3D-Printed Devices.

U.S. Food and Drug Administration (FDA) approval of Class III medical devices can take from 3 to 7 years. Although this is shorter than times for drug approvals, patients with serious or life-threatening diseases and disorders may not have time to wait for device approval to access needed treatments. The FDA has a number of pathways, similar to drug approval processes, for expanded use of unapproved medical devices in patients for whom no reasonable alternative therapy is available. Additionally, the FDA regulates the manufacture and use of "custom" medical devices-those made for use by 1 spec…

Scoping review of the role of pharmacometrics in model-informed drug development.

Model-informed drug development (MIDD) is a framework that utilizes quantitative modeling and simulation methods to integrate nonclinical and clinical data, as well as prior knowledge, to inform drug development and regulatory decision-making. This scoping review examines the current MIDD landscape with a focus on pharmacometrics. It will clarify essential MIDD concepts and provide a taxonomy of terms. Additionally, it will evaluate the implications of the International Council for Harmonisation (ICH) M15 MIDD draft guidelines and summarize case studies that illustrate the use of MIDD in vario

Golodirsen: First Approval.

Golodirsen (Vyondys 53™), an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO) subclass designed to induce exon 53 skipping, has been developed by Sarepta Therapeutics for the treatment of Duchenne muscular dystrophy (DMD). In December 2019, intravenous golodirsen received its first global approval in the USA for the treatment of DMD in patients with a confirmed mutation of the DMD gene that is amenable to exon 53 skipping, based on positive results from a phase I/II clinical trial. Golodirsen is in phase III clinical development for the treatment of DMD worldwide. T

Helsinn: 20 years in primary cancer supportive care.

Sergio Cantoreggi speaks to Henry Ireland, Commissioning Editor: Sergio Cantoreggi, PhD, is the Chief Scientific Officer and Global Head of Research and Development of the Helsinn Group, a mid-sized pharmaceutical company headquartered in Lugano, Switzerland, and focused on providing cancer supportive care solutions to oncology patients worldwide. Dr Cantoreggi has overall responsibility for all R&D activities of the Helsinn Group and has contributed to six major regulatory approvals of cancer supportive care agents in the USA, Europe and Japan. Dr Cantoreggi joined Helsinn Healthcare in 2000