Medicines and Vaccines
CTR and CTIS
- The EMA CTIS Webinar summary on 6 months of CTIS and looking forward is available now.
- Clinical Trials Information System (CTIS) bitesize talk: Deferral rules and Public website held on 20 July 2022. A video recording is made available after the event. Processing and publication of the video recording takes up to 60 days.
- The European Commission, EMA, HMA, and the Clinical Trials Coordination Group (CTCG) rolled out a survey on the implementation of the Clinical Trials Regulation (EU) No 536/2014 and the new IT platform, CTIS. The objectives of the survey are to understand the overarching hurdles that hamper a smooth implementation of the CTR and to capture how clear the requirements of the CTR are to the stakeholders. The deadline for responses is 09 September 2022 at midnight CET.
- The July 2022 issue of the “Clinical Trials Highlights” newsletter is now released.
- EMA’s Eudralex Volume 10 ‘Clinical Trials Guidelines’ has a very useful new section titled ‘Latest updates’. Check it out.
UK and MHRA News
- The UK government published the Life science competitiveness indicators which revealed that ‘combined review’ considerably reduces the length of time taken to approve clinical trials. From 2022, all UK trials will be reviewed through the ‘combined review’. Combined review is an approval process for new Clinical Trials of Investigational Medicinal Products (CTIMPs) and combined medicine and device trials. A short explainer video to ‘combined review’ is also available for newcomers.
FDA Guidance and News
- June 2022 Final FDA Guidance: Assessing the Effects of Food on Drugs in INDs and NDAs – Clinical Pharmacology Considerations.
- June 2022 Final FDA Guidance: Providing Regulatory Submissions in Alternate Electronic Format Guidance for Industry.
- June 2022 Draft FDA Guidance: Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments. See below 'Development Strategy News' section point 2 for a summary of this guidance.
- June 2022 Draft FDA Guidance: Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics. This guidance provides recommendations for evaluations including pharmacokinetic, pharmacodynamic, and safety assessments during oligonucleotide therapeutic development.
- 14 July 2022: FDA released final guidance on the content and format of Instructions for Use (IFU) labelling written for patients (or caregivers) and covers human prescription drug and biological products submitted under a new drug application (NDA) or a biologics license application (BLA). The recommendations are designed to help pharmaceutical manufacturers provide consumers with easily understood information for the safe and effective use of their products.
EMA Guidance and News
- EMA launched the pilot on using raw data in medicine evaluation in July 2022. Selected applicants can submit 'raw data' to EMA as part of their initial and post-authorisation marketing authorisation applications. The EMA also released ‘Information about the raw data proof-of-concept pilot for industry’ to provide an overview of the submission and analysis of ‘raw data’ from clinical studies.
- Users of CTIS and the EMA systems should take note of the planned outage/interruptions to these websites in order to plan for workarounds.
- The EMA’s Big Data Steering Group and the Heads of Medicines Agencies (HMA) has published its third workplan that sets key actions to be delivered between 2022–2025. The new workplan will further enhance the efficient integration of data analysis to improve decision making. Evidence generated from big data is expected to accelerate medicine development, improve treatment outcomes and facilitate earlier patient access to new treatments.
- EMA has released on 28 July 2022 ‘Key performance indicators (KPIs) to monitor the European clinical trials environment’ that includes metrics on the CTR and CTD from 1-30 June 2022.
- The workshop on COVID-19 real-world evidence observational studies was held by the International Coalition of Medicines Regulatory Authorities (ICMRA) on 20 May 2022. The workshop minutes are available on the ICMRA website.
- The EMA has endorsed a joint statement by ICMRA calling for international collaboration to enable the generation and use of real-world evidence for regulatory decision-making. To achieve this, the ICMRA members have identified the focus areas of opportunities for regulatory collaboration.
Transparency and Disclosure Resources and News
- Phuse Data Transparency Summer Event was held 28-30 June 2022. Presentations over the 3 days covered EU CTR Regulations (Day1), Patient Engagement Side (Day 2) and Anonymisation and Data Sharing (Day3). Recordings from the 12 presentations are available to access for free from the PHUSE Engagement Hub.
- This 12 July 2022 BMJ news article describes that for ‘Covid-19: Researchers face wait for patient level data from Pfizer and Moderna vaccine trials’.
- Compliance of authors who published in open access journals with mandatory data availability statements (DAS) was reported as similar to those publishing in journals that did not provide DAS. (Note journal article behind a paywall).
- An update to the State of Open Data Survey has been released. This is a longitudinal survey and analysis on open data which has been running for over 6 years.
- Pfizer has created a portal to host publication-related outputs covering Publication Plain Language Summaries (PLS), supplemental data associated with published manuscripts (research data, tables, images, videos, software) and Open Access (OA) manuscripts.
Development Strategy News
- Beckman et al published a comment on ‘Advancing innovative clinical trials to efficiently deliver medicines to patients’. The article itself provides an informative overview on complex innovative designs - helpful for anyone looking to get an understanding of CIDs. Supplementary information details the 2020 DIA/FDA conference which include lessons learned on master protocol studies addressing chronic pain and Ebola (PREVAIL II & PALM), as well as case-study that included CID design considerations from the FDA. Sessions on efficiency and Type I error control for a confirmatory basket trial design, adaptive design, and Bayesian modelling also provide some interesting reading material.
- The FDA released its third draft guidance in a series of four methodological patient-focused drug development documents: Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments. The guidance covers each of the four COAs - PROs, ObsROs, ClinROs, and PerfOs, deciding whether the COA is fit for purpose, and gives a roadmap for COAs in clinical studies (summarized in Figure 2). Overall, a great resource for walking through COA assessments prior to protocol development. Comments by 28 Sep 2022.
- Treweek et al published a study on Getting it wrong most of the time? Comparing trialists’ choice of primary outcome with what patients and health professionals want. Although it has been long-understood that studies don't necessarily match primary outcomes with patient preferences, there is limited quantitative evidence that assesses this delta. In this study - part of the Trial Forge initiative - the authors extracted key outcomes from a selection of 44 studies and asked 42 patients and HCPs to rank the importance of the outcomes. In total, only 28% (13/46) of the primary outcomes were successfully identified as the most important outcome - indicating that there is significant room for improvement when designing the study and seeking participant input.
News from Asia Regulators
- In February 2022, CDE of NMPA China released an interim Technical Guidance for Clinical Trials of New Antiviral Drugs for COVID-19 (in Mandarin) which provides recommendations on the study design, conduct, and assessments of antiviral drugs for COVID-19 in China.
Medical devices information is kindly compiled by Raquel Billiones.
- Press release from the UK Government: UK to strengthen regulation of medical devices to protect patients. in this regard, the UK Government released the response to consultation on the future regulation of medical devices in the UK.
- The UKCA (UK Conformity Assessed) marking is a new UK product marking that is used for goods being placed on the market in Great Britain (England, Wales and Scotland). It covers most goods which previously required the CE marking, known as ‘new approach’ goods. The UKCA marking came into effect on 1 January 2021. However, to allow businesses time to adjust to the new requirements, you will still be able to use the CE marking until 1 January 2023 in most cases.
- EU Updates on Companion Diagnostics
A companion diagnostic is a medical device, often an in vitro device, which provides information that is essential for the safe and effective use of a corresponding drug or biological product. Below are recent updates on guidance documents for companion diagnostics in the EU regulated under the IVDR.
Updated: Guidance on the procedural aspects for the consultation to the European Medicines Agency by a notified body on companion diagnostics Updated: Q&A document - Practical arrangements on the companion diagnostics consultation procedure to the European Medicines Agency by notified bodies has been updated. The accompanying forms and templates have also been updated, as follows:
Updated: Template for the assessment report on Consultation on companion diagnostic
Updated: Application form for initial consultation by a notified body on a companion diagnostic
New: Application form for a follow-up consultation procedure by a notified body on a companion diagnostic
4. EU Medical Devices Newsletter June 2022
All these updates are summarized in the June 2022 issue of Medical Devices news.
- Updates from TGA Australia
The Australian Therapeutic Goods Administration released in June 2022 an update to the Clinical evidence guidelines for medical devices (version 3.1).
- From early 2022, Taiwan FDA has implemented a new online Medical Device Electronic Pre-market Application Platform for Class II and III medical devices. Through the e-Submission system, medical device manufacturers could apply for Class II and III medical device registration, update the registration, or extend licensing.
- EUDAMED Delay
The European Commission issued a new update on the European Database for Medical Devices (EUDAMED) timelines in June, indicating a delay of approximately a year. The EUDAMED is now expected to be fully functional in Q2 2024 and will be mandatory in Q2 2026 following a 2-year transition period.
- MDCG-2022-12 on IVD in EUDAMED
In July, the MDCG released a guidance on harmonised administrative practices and alternative technical solutions until Eudamed is fully functional in relation to the EU Regulation 2017/746 on in vitro diagnostic medical devices (IVDR).
- MHRA Updates
Last year, MHRA consulted on the new UK regulatory framework for medical devices post-Brexit. The full outcome of the consultation has been released.
- Updates from China
The NMPA released the “Guideline for Fundamental Principles Compliance of Medical Device Safety and Performance” which describes the general requirements for all medical devices and its risk-based approach for registration review.
- An article on “China’s NMPA: The evolution of medical device regulation” was published in the June 2022 issue of RAPS RF Quarterly (RAPS membership is needed to access the article).