Current Awareness - Pharmacy: Pharmacy - Current Awareness

Recenti Prog Med. 2025 Jun;116(6):371-379. doi: 10.1701/4518.45175.

ABSTRACT

INTRODUCTION: Rapid access to safe and effective oncological therapies is crucial. In recent years, many expensive drugs have been commercialized, making it essential to prioritize those with significant clinical benefits. For this purpose, the European Society for Medical Oncology (ESMO) introduced the ESMO-MCBS (Magnitude of Clinical Benefit Scale), which assigns a score to quantify the clinical benefit of treatments. This study aims to evaluate whether there is a correlation between the ESMO-MCBS score and drug access times in Italy.

MATERIALS AND METHODS: The clinical benefit was directly extracted from the official ESMO website, considering registrational trials. Data on drug access times included: the date of a positive opinion from the Committee for Medicinal Products for Human Use (CHMP), the date of authorization published in the European Official Journal (EU OJ), the date of submission of the drug dossier to the Italian Medicines Agency (AIFA) by the company, the date of opinions issued by AIFA's Technical Scientific Committee and pricing and reimbursement committee, and the date of publication in the Italian Official Journal (OJ). Access time was calculated as the difference in days between the publication dates in the Italian OJ and EU OJ. Subgroup analyses also considered EMA authorization procedures (accelerated approval, conditional marketing), drug classification as "orphan drug," and the innovativeness requirement.

RESULTS: The analysis included 48 drugs, corresponding to 57 registrational trials, of which 4 referred to drugs not yet commercialized by AIFA, and 7 did not lead to reimbursement. The evaluation focused on 46 registrational trials. The average access time was 488 days: 480 days for drugs with significant benefit and 499 days for those without significant benefit. For orphan drugs, the average access time was 499 days. Drugs with EMA accelerated assessment showed shorter access times (440 days), while those with conditional marketing approval had longer access times (556 days). Drugs evaluated by AIFA for innovativeness were authorized faster on average.

DISCUSSION AND CONCLUSIONS: No significant difference in access times was observed between drugs with significant and non-significant clinical benefits. Among the analyzed subgroups, access times are better for drugs with accelerated approval and for which AIFA has expressed an opinion of innovativeness, comparable for drugs designated as "orphan drugs", worse when the EMA authorization occurred as conditional marketing.

PMID:40491402 | DOI:10.1701/4518.45175

AAPS J. 2025 Jun 4;27(4):105. doi: 10.1208/s12248-025-01091-0.

ABSTRACT

The Pharmaceuticals and Medical Devices Agency (PMDA) is a Japanese authority responsible for reviewing the quality, efficacy, and safety of drugs and medical devices to be marketed for public health protection. This study investigated the approval numbers of monoclonal antibodies (mAbs). In addition, it classified canonical and modified antibodies, such as antibody-drug conjugates (ADCs), antibody fragments, and bispecific antibodies (bsAb), excluding the biosimilar versions of mAbs in each fiscal year (FY) based on the PMDA website (1). Moreover, the approval FY data, brand names, international nonproprietary names, cell substrates, targets, disease areas at initial approval, and regulatory pathways were compiled (Supplement Document 1).

PMID:40468128 | DOI:10.1208/s12248-025-01091-0

J Drugs Dermatol. 2025 Jun 1;24(6):586-589. doi: 10.36849/JDD.8840.

ABSTRACT

Benzyl peroxide (BPO) has been an important component of many acne treatment regimens. However, an independent testing laboratory, Valisure, filed a Citizen’s Petition with the US Food and Drug Administration (FDA) requesting the removal of BPO products from the market. Their testing demonstrated that when exposed to elevated temperatures which could occur during shipping, marketed BPO products can form over 800 times the FDA concentration limits of benzene, a class 1 carcinogen. In this review, we aim to describe what a post-BPO removal acne treatment regimen might look like based on currently approved products and products in late-stage development. Citation: Dhawan S, Nardo CJ. The impact on acne treatment regimens if benzoyl peroxide-containing products are removed from the market. J Drugs Dermatol. 2025;24(6):586-589. doi:10.36849/JDD.8840R1.

PMID:40465500 | DOI:10.36849/JDD.8840

Pharm Res. 2025 May;42(5):731-735. doi: 10.1007/s11095-025-03861-z. Epub 2025 Jun 3.

ABSTRACT

This overview summarizes the history and advancements of the modeling and simulation programs utilized in drug development and regulatory assessment, including the FDA's Model-Informed Drug Development (MIDD) Paired Meeting Program and the Model-Integrated Evidence (MIE) Meeting Pilot Between FDA and Generic Drug Applicants. The U.S. Food and Drug Administration's (FDA) recent notice concerning the use of the Type V Drug Master File (DMF) for Model Master File (MMF) submissions to support abbreviated new drug applications (ANDAs) encourages and facilitates model-sharing and model-reusability in drug development, supporting MIE programs using a broad range of quantitative models, including, but not limited to physiologically based pharmacokinetic (PBPK), population pharmacokinetics (PPK) and computational fluid dynamics (CFD) modeling. This overview also introduces the considerations and representative mock examples of MMFs discussed in the workshop titled "Considerations and Potential Regulatory Applications for a Model Master File (MMF)" co-hosted by the U.S. Food and Drug Administration (FDA) and the Center for Research on Complex Generics (CRCG) on May 2-3, 2024. MMFs promote modeling and simulation approaches by reducing the burden of resources in developing this type of approaches for the pharmaceutical industry while increasing consistency and efficiency in regulatory assessments.

PMID:40461748 | DOI:10.1007/s11095-025-03861-z

Orphanet J Rare Dis. 2025 Jun 2;20(1):266. doi: 10.1186/s13023-025-03756-7.

ABSTRACT

BACKGROUND: In the European Union (EU), the orphan legislation, aiming to increase the number of pharmacotherapies available for rare diseases, came into force in April 2000. This study examined the development of the selection of orphan medicines granted marketing authorisation, their approved indications, and the number of orphan medicines developed for paediatric use in EU during 2000-2022. This study also examined the availability of the orphan medicines with a marketing authorisation in the Finnish market in order to demonstrate their country level uptake in a single member state.

METHODS: The material on orphan medicines' marketing authorisations and their introduction were collected from the European Commission's Community Registers in June 2022 and analysed with a qualitative document analysis. This study covered the period 2000-2022 since the introduction of the orphan legislation, and comparisons were made in 10-year periods of, 2001-2010 and 2011-2020.

RESULTS: By May 2022, there were 213 novel orphan medicines approved in Europe during the observation period. Of them, 67% (n = 142) were on the market in Finland in May 2024. The number of new orphan medicines approved in Europe doubled from 63 products in 2001-2010 to 127 products in 2011-2020. Several orphan medicines were developed for certain type of rare diseases, such as haematological cancers. The proportion of orphan medicines approved for paediatric use decreased from 55% in 2001-2010 to 42% in 2011-2020.

CONCLUSION: The number of orphan medicines available within EU increased significantly after the orphan legislation came into force. The development of orphan medicines seemed to often focus on diseases or disease groups that already have available treatment options, while several rare diseases remain without available treatment. Even though rare diseases are more common in children, orphan medicines have not been developed for paediatric use in the same proportion.

PMID:40457478 | PMC:PMC12131831 | DOI:10.1186/s13023-025-03756-7