November 2021

Medicines and Vaccines

EMA Publish Statements and Guidances

EMA published their final guideline on registry-based studies on 26 October 2021.

CTIS News: Supporting materials from DIA

Drug Information Associates (DIA) have prepared some materials to support the Clinical Trials Information System (CTIS): Virtual information day held on 11 November 2021:

  1. CTIS The future user perspective: Welcome - It includes a brief introduction to CTIS, information on its workspace and public portal, its go-live timeline, purpose, and steps required for its preparation.
  2. CTR & CTIS: Key aspects for users to consider when preparing for CTIS - It includes milestones for Clinical Trial Regulation 563/2014 (CTR), the process of CTR assessment, CTIS roles and actions, and CTIS high-level structure. It also includes information on a harmonised dossier, transparency in the CTR, and requirements for CTR submission.
  3. How to prepare for CTIS: a user perspective - It explains the process of registration, user management, an overview of CTIS user personas, notices, alerts, and requests for information (RFIs).
  4. How to access CTIS training materials and support - It provides links to all the CTIS training modules, sponsor handbook for clinical trial sponsors, reference material, and recording of previous events. It also presents the three phases of CTIS Sandbox.
  5. CTIS The future user perspective: Closing - It includes a diagrammatic representation for preparation of CTIS and the countdown for CTIS go-live.

FDA Guidances and News

  1. FDA, NIH, and 15 private organizations join forces to increase effective gene therapies for rare diseases. Read the Press Release which states “A clinical component of BGTC-funded research will support between four and six clinical trials, each focused on a different rare disease.”

AMA Manual of Style Commentary to Support Guideline Changes

This American Medical Association’s ‘Style Insider’ article ‘Updated Guidance on Reporting Race and Ethnicity: Let’s Start With the Why’ emphasizes that the updated AMA Manual of Style 11th Edition guidance on race and ethnicity is important to help safeguard against unconscious bias.

Real World Evidence

The following two articles are relevant to our understanding of how agencies can use real world data post approval, to support clinical trial findings:

EMA have released a new framework for gathering patient perspectives in Europe. The Innovative Medicines Initiative (IMI) is developing a clear practical framework for their measurement, called the PREFER framework, which is now open for public consultation in the form of an EMA Draft Qualification Opinion that explains how patient preference studies could be used to help inform decisions about medicines approval and their subsequent availability to patients.

Updated TransCelerate Template Assets 2021

In the October 2021 updates of TransCelerate's assets, the Common Protocol Template (CPT), Statistical Analysis Plan (SAP) template, and Clinical Study Report (CSR) template have been updated to reflect estimand considerations, amongst other points. The 'summary of changes' slide deck calls out the changes since the last version assets, and all new assets, as well as a host of other supporting materials are downloadable from the ‘Clinical Content and Reuse Solutions’ page.

Transparency and Disclosure Resources and News

  1. The UK NHS’s Health Research Authority has published its Make it Public Annual Report following its conference held on 03 and 04 November 2021. This is “The first Make it Public annual report has been published, telling a story of progress towards trusted information about all health and social care studies being publicly available for the benefit of all.” Note the ‘Reporting Results’ section.
  2. This TranspariMED article sets out the key milestones and delivery dates associated – including those for reporting results - for the UK’s clinical trial transparency system, which will go live in 2022.
  3. The UK’s MHRA has published its Patient Involvement Strategy 2021 to 2025. This first strategy document explains how MHRA will “…involve the public and patients at every step of the regulatory journey.”
  4. This ISMPP article, published in August 2021, describes Pfizer’s approach to Plain Language Summary development.
  5. As part of the collaboration between EMWA’s Regulatory Public Disclosure Special Interest Group (RPD SIG) with the Statisticians in the Pharmaceutical Industry (PSI) Data Transparency SIG, they kindly permitted the sharing of an article on secondary use of data which was published in a PSI ‘members only newsletter’ earlier in 2021. The article titled ‘Secondary use of data - Unleashing Data Assets to Create Value’ is published on the EMWA website.
  6. The UK’s National Institute for Health Research (NIHR) has published its Open Access policy - for publications submitted on or after 1 June 2022. This policy makes all NIHR-funded research findings published in academic peer-reviewed journals freely available.
  7. This article from the Publication Plan asks ‘What’s stopping patients from publishing?’ and suggests biomedical research industry should support greater patient participation through encouraging ethical patient authorship.


Eudamed is officially in place in the EU

On 26 Nov 2021, the European Commission published the final document laying down rules for the European Database on Medical Devices (Eudamed). The Eudamed is the electronic system that contains data on medical devices with respect to clinical trials, market access, and post-market surveillance.
Some key information highly relevant to data transparency and public disclosure are provided below:

  • Eudamed is accessible via a restricted website for authorized users and a public website for non-identified users. In addition, Eudamed will also be accessible through machine-to-machine data exchange services (Article 2 Modes of Access).
  • To facilitate traceability, authorized users have free access to the European Medical Device Nomenclature (EMDN; Article 4 Nomenclature).
  • Personal data will be processed to comply with the MDR and the IVDR. The European Data Protection Supervisor has been consulted (Article 6 Ownership and Processing of Personal Data).
  • The Commission commits to providing some sort of sandbox environment to train Eudamed users (Article 9 Websites for Testing and Training Purposes).

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