Current view on novel vaccine technologies to combat human infectious diseases

Trials and tribulations of antibiotic development

Overcoming resistance.

The EMA Framework of Collaboration With Academic Stakeholders.

BackgroundBoth FDA and EMA reward/demand pediatric oncology studies. Do they advance pediatric cancer care?MethodsWe analysed publications of FDA representatives,1–3 FDA-triggered pediatric oncology studies in the literature and in www.clinicaltrials.gov,4 and...

Background:The authors performed gap analyses on the expedited programs associated with regenerative medicine therapies (RMTs), offered by Food and Drug Administration (FDA), European Medicines Agency (EMA) and Ministry of Food and Drug Safety (MFDS).Methods: The regulations and guidelines on expedited programs (e.g., Regenerative Medicine Advanced Therapy [RMAT] designation) associated with RMTs, and regulatory reviews on approved RMTs were retrieved from the websites of FDA, EMA and MFDS.Based on analyses of the retrieved materials, gap analyses were performed on the qualifying criteria, features and operational frameworks.Included were the comparisons of the expedited programs granted for tisagenlecleucel among the agencies.Results: FDA offers RMAT, Fast Track, Breakthrough Therapy, Priority Review designations, and Accelerated Approval.EMA's programs include PRIority MEdicines (PRIME), Accelerated Assessment, Exceptional Circumstances, and Conditional Marketing Authorisation.MFDS implements a single Fast-Track Processing program.RMAT designation is specific to RMTs and can be utilized with FDA's other expedited programs.PRIME applies to all medicinal products and can be used with EMA's other programs.Fast-Track Processing program is specific to RMTs but combines all expedited features into a single track.Tisagenlecleucel received RMAT, Priority Review and Accelerated Approval for follicular lymphoma from FDA, PRIME for acute lymphocytic leukemia from EMA, and Fast-Track Processing for follicular lymphoma from MFDS.Conclusion: FDA offers the RMAT designation exclusive to expediting the development, review and approval of RMTs in addition to other expedited programs, whereas EMA does RMT approvals under expedited drug approval programs.MFDS implements an exclusive, single-track program for RMTs.

IntroductionThe efficacy of pharmaceuticals is most often demonstrated by randomised controlled trials (RCTs); however, in some cases, regulatory applications lack RCT evidence.ObjectiveTo investigate the number and type of these approvals...

How do the EMA and FDA decide which anticancer drugs make it to the market? A comparative qualitative study on decision makers’ views

Background: Many patients with relapsing-remitting MS (RRMS) show a suboptimal response to first-line disease-modifying drugs. In these patients treatment with natalizumab is highly effective, however its use has been limited due to safety concerns. Objective: To evaluate the consistency between the eligibility to natalizumab according to Italian Drug Agency (AIFA), European Medicines Agency (EMA) and Food and Drug Administration (FDA) criteria, and its use in clinical practice. Methods: Medical records of 402 patients from four Italian MS Centres were reviewed to identify patients eligible to natalizumab according to AIFA, EMA, and FDA criteria and verify how many of them were currently treated or had been previously treated in practice. Results: Of 316 RRMS patients, 13.3% were currently or had been previously treated with natalizumab, while additional 7.0%, 14.2%, and 27.2% were not receiving the drug although they were eligible according to AIFA, EMA, and FDA criteria, respectively. Compared to patients treated with natalizumab, subject who were eligible but remained untreated were older and with shorter education. Conclusion: In a cohort of RRMS patients, 20.3%, 27.5%, and 40.5% were eligible to natalizumab according to AIFA, EMA, and FDA, respectively, although only part of them were actually treated.

Characteristics of professional society oncological drug evaluation in the Netherlands from 2016 to 2020: A retrospective analysis.

ObjectivesNon-randomised clinical trial designs involving comparisons against external controls or specific standards can be used to support regulatory submissions for indications in diseases that are rare, with high unmet need,...

Biosimilars, a class of biologic medications that are highly similar to reference biologics, have emerged as cost-effective alternatives to their expensive originator counterparts. Due to their complex nature and manufacturing processes, biosimilars differ significantly from small molecule generics and must undergo a rigorous assessment to ensure safety, efficacy, and accessibility. This review explores the regulatory landscape surrounding biosimilars across key markets such as the United States, Europe, and India, with a focus on approval processes and post-marketing pharmacovigilance for patient safety. The study conducted a detailed review of regulatory guidelines, approval frameworks, and post-marketing requirements for biosimilars across various countries. Data was collected from official sources such as the European Medicines Agency (EMA), the United States Food and Drug Administration (FDA), and relevant Indian regulatory bodies. The research also analysed guidelines focusing on pharmacovigilance practices, particularly for vulnerable populations like paediatric and geriatric patients. The analysis found that while regulatory agencies such as the EMA and FDA have established stringent biosimilar approval pathways, India's regulatory framework, though promising, still lacks comprehensive pharmacovigilance guidelines. The harmonization of global biosimilar guidelines has contributed to their widespread adoption in new therapeutic areas and emerging markets, driving market expansion. The study highlights the importance of postmarketing monitoring to ensure continued safety, with particular emphasis on vulnerable populations. The regulatory landscape for biosimilars is evolving, with increasing global collaboration fostering the harmonization of guidelines. Regulatory agencies such as the European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) have established rigorous approval frameworks, ensuring biosimilars meet the necessary standards for safety and efficacy. In emerging markets like India, the biosimilar sector is poised for significant growth, though the regulatory framework is still maturing. Strengthening regulatory infrastructure, particularly in areas such as approval processes and quality control, will be crucial in supporting this expansion. The review emphasizes the importance of robust and clear regulations to facilitate the safe and effective integration of biosimilars into global healthcare systems, ensuring greater accessibility for patients without compromising on quality.

To clarify the rationales of delay or difference in the review of new drug applications among regulatory authorities for new drugs, those first approved in the world being in Japan. Among 80 new drugs first approved in Japan from 2008 to 2019, we identified those subsequently approved in the USA or Europe. Significant delays in approval time (boxplot outliers) and the rationales for the delays were assessed among the Pharmaceuticals and Medical Devices Agency (PMDA), the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Of the 80 Japan-first approvals, 25 and 24 were approved in the USA and Europe, respectively, and their median approval times in Japan, the USA and Europe were 285, 334 and 477 days, respectively. Significant delays were identified for pirfenidone (1806 days, FDA), alogliptin benzoate (1856 days, FDA), insulin degludec (1457 days, FDA) and romosozumab (750 days, PMDA; 994 days, FDA; 748 days, EMA). Due to concerns about cardiovascular risk, alogliptin benzoate and insulin degludec were requested for additional clinical trials by the FDA, and romosozumab required a much longer review period than the standard approval time in all three regions. Among the new drugs significantly delayed in approval time in Japan, the USA or Europe, there were some differences in the requirements, the participating regions and the assessment of clinical trials. The regulatory views on the cardiovascular risk also differed among the three regions. These divergences may be associated with the differences in approval histories.

Reporting of older subgroups in registration trials of FDA and EMA approvals between 2017 and 2023 for diffuse large B-cell lymphoma: A systematic review

Improving oncology biosimilar launches in the EU, the USA, and Japan: an updated Policy Review from the Southern Network on Adverse Reactions.

Ophthalmic eye drops present one of the most demanding scenarios in bioequivalence (BE) science. Drug concentrations at the local site of action — the cornea, aqueous humor, and uveal tissues — are largely inaccessible through conventional plasma-based pharmacokinetic (PK) sampling. In response, the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have developed product-specific, graduated regulatory frameworks that deploy three classes of in vivo evidence for ophthalmic BE: aqueous humor PK studies, pharmacodynamic (PD) endpoint studies using intraocular pressure (IOP) or clinical outcome measures, and plasma PK studies for systemically absorbed drugs. These approaches are operationalized through the FDA’s Product-Specific Guidance (PSG) system and the EMA’s product-specific bioequivalence guideline (PSBGL) framework. This article presents a rigorous, guideline-anchored review of how PK and PD study methodologies are applied to demonstrate ophthalmic BE, using multiple drug class examples including prostaglandin analogues (latanoprost), alpha-2 agonists (brimonidine), corticosteroids (loteprednol etabonate, prednisolone acetate), and anti-infectives. We further describe how Model-Informed Drug Development (MIDD) — particularly physiologically-based ocular PK (PBOPK) modeling and population PK analysis — functions as a scientifically enabling tool within, not instead of, the existing regulatory BE framework. An optimal study design and regulatory submission strategy aligned with current FDA and EMA requirements is proposed, with explicit guidance on when PK versus PD endpoints are appropriate, how BE statistical criteria are applied in each context, and what MIDD can contribute at each stage.