The FDA has announced details on its Split Real Time Application Review (STAR) pilot program 

In the prescription drug user fee amendments (PDUFA) VII commitment letter, the FDA has announced details on its STAR pilot, a program which aims to shorten the time from the date of complete submission to the action date, in order to allow earlier patient access to therapies that address an unmet medical need.

The STAR pilot program will apply to efficacy supplements across all therapeutic areas and review disciplines that meet specific criteria. Accepted STAR applications will be submitted in a “split” fashion, specifically in two parts with the components submitted approximately two months apart. FDA will begin to review the data once the agency receives the complete Part 1 Submission.

The PDUFA review clock will start once the agency receives the Part 2 Submission, which will include the final clinical study report(s), the Integrated Summary of Safety, and Integrated Summary of Effectiveness. The program applies to both drugs and biologics, collectively referred to as drug(s). The STAR program will be available in 2023. 

The FDA has issued final guidance on the inclusion of patients with incurable cancers in clinical trials for investigational therapies 

The FDA has issued final guidance on the inclusion of patients with incurable cancers in clinical trials for investigational therapies.

The guidance provides recommendations to clinical investigators and sponsors regarding the inclusion of patients who have not received available therapy (commonly referred to as existing treatment options) for their cancer in clinical trials of drugs and biological products for the treatment of cancer in the non-curative setting (i.e., when there is no potential for cure or prolonged/near normal survival). 

The FDA’s Centre for Drug Evaluation and Research (CDER) has launched a New Accelerating Rare disease Cures (ARC) Program  

The FDA’s CDER has announced the launch of the new ARC program to bolster the development of effective and safe treatment options addressing the unmet needs of patients with rare diseases.

In its first year, CDER’s ARC program will focus on strengthening internal and external partnerships with stakeholders and will engage with external experts to help identify solutions for the challenges in rare disease drug development. This is a CDER-wide effort with leadership represented from several offices throughout the centre. 

The FDA has published four draft guidances spelling out how it will consider real-world data (RWD) in regulatory decision-making  

The FDA has published four draft guidance spelling out how it will consider RWD in regulatory decision-making, from the use of electronic health records (EHRs) and registries to RWD in non-interventional studies.

The first draft guidance intends to provide sponsors, researchers, and other interested stakeholders with considerations when proposing to use EHRs or medical claims data in clinical studies to support a regulatory decision on effectiveness or safety.

The second draft guidance provides sponsors and other stakeholders with considerations when either proposing to design a registry or using an existing registry to support regulatory decision-making about a drug’s effectiveness or safety.

The third draft guidance addresses considerations for the use of data standards currently supported by FDA in applicable drug submissions containing study data derived from RWD sources.

The fourth draft guidance discusses the applicability of FDA’s investigational new drug (IND) application regulations under part 312 (21 CFR part 312) to various clinical study designs that utilise RWD. The guidance also clarifies the Agency’s expectations concerning clinical studies using RWD submitted to FDA in support of a regulatory decision regarding the effectiveness and safety of a drug.  

The EC has updated the Clinical Trials Regulation (EU) No 536/2014 Questions & Answers (Q&A) document to Version 6.2 

The document sets out frequently asked Q&As regarding the implementation of the rules on clinical trials. The document includes new and updated information on Section 1.24: “How are patient-facing documents expected to be submitted?”, Chapter 11: “Arrangements for the transitional period”, Q&A 3.8: “How should a sponsor proceed when a substantial modification is related to a document common to various clinical trials of the same sponsor and same IMP?”.  

The EMA has released a reflection paper outlining the criteria to be considered for the evaluation of new active substance (NAS) status of biological substances.

This reflection paper is intended to reflect the current experience of the Biologics Working Party of the Committee for Medicinal Products for Human Use, the Committee for Advanced Therapies, and the Co-ordination Group for Mutual Recognition and Decentralised Procedures-Human on NAS in the context of scientific advice and assessment of Marketing Authorisation Applications (MAA).

It applies to all types of procedures for submission of a MAA, i.e. Centralised Procedure, Mutual Recognition Procedure/Decentralised Procedure and purely national procedures for biological and biotechnology-derived medicinal products for human use. 

The EMA has published the 2022-2026 workplan of the initiative Accelerating Clinical Trials in the EU (ACT EU).

ACT EU seeks to transform how clinical trials are initiated, designed and run. The aim is to further develop the EU as a focal point for clinical research, promote the development of high-quality, safe, and effective medicines, and to better integrate clinical research in the European health system.

ACT EU will strengthen the European environment for clinical trials, whilst maintaining the high level of protection of trial participants, data robustness and transparency that EU citizens expect. The workplan highlights key focus areas such as innovation in clinical trials, robust methodologies and collaboration across stakeholders.

The ACT EU workplan is structured in line with the ten priority actions for ACT EU and has been prepared based on the recommendations of the European medicines agencies network strategy to 2025 and the European Commission’s Pharmaceutical Strategy for Europe. 

The MHRA has opened consultation on how it communicates with healthcare professionals to improve medicine safety 

The MHRA is reviewing its approach to engagement with healthcare professionals to improve the safety of medicines. This is to ensure that healthcare professionals are receiving actionable information and guidance on safe use of medicines that they can take into their working practice, providing timely advice to patients.

Participation in this consultation is anonymous and can be carried out here. The consultation closes on 18 January 2023.