Latest Regulatory Guidance

Our Industry Update Newsletter returns for this third update of 2022 and focuses on Regulations & ICH Guidelines; Inspections; Initiatives across the World; Real World Data & Real World Evidence, and much more.

As always, this quarter’s update has been expertly compiled by Christina Hägglund, QA Manager.

You can download a pdf of this update here and sign up to receive our updates via email here.


Clinical Trial Regulation (CTR) (EU) 536/2014

Performance Metrics

Q2 Newsletter, miscellaneous section, refers to ACT EU (Accelerating Clinical Trials in the EU). One of the priority actions of ACT EU is to monitor the implementation of the CTR. The European Commission (EC), the European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) issues quarterly reports to provide an overview of Key Performance Indicators (KPIs) for the implementation of the CTR. ACT EU setup for HMA November 2021 – 221021-ESKB (

CTR – Q&A Document

The EC has provided minor updates to the questions and answers (Q&As) document. Microsoft Word – CTR QnA v6.1 27-05-2022 (

Regulation (EU) 2017/746 on in vitro Diagnostic Medical Devices (IVDR)

EFPIA Statement of Concern

European Federation of Pharmaceutical Industries and Associations (EFPIA) fully supports the IVDR in its aim to ensure a high level of public health and patient safety in Europe. EFPIA has however issued a statement detailing the challenges which need to be addressed. EFPIA statement on the concerning impact of the In Vitro Diagnostic Regulation

In vitro Diagnostic Medical Devices, Swissmedic Statement May 2022

Swissmedic, the regulatory agency in Switzerland has issued a statement: Clinical trials with in vitro diagnostic medical devices (IVD) will be regulated by the Ordinance on Clinical Trials with Medical Devices instead of the Ordinance on Clinical Trials, aiming to improve patient safety through stricter measures for conformity assessment and post-marketing surveillance.

Further information is provided on the implementation of the new requirements, the transition period from the previous rules, obligations for the registration of medical devices and safety reporting. New regulations applicable to in vitro diagnostic medical devices as of 26 May 2022 (

Australia’s Therapeutic Goods Administration (TGA) Updated Guidance

The updated guidance reflects TGA’s approach on how it assesses clinical evidence and at the same time provides a reference point for the requirements for medical devices, including IVDs. There is a supplementary document entitled ‘Clinical Evidence Guidelines Supplement: In Vitro Diagnostic (IVD) Medical Devices’ (Version 3.1, June 2022). Clinical evidence guidelines: Medical devices | Therapeutic Goods Administration (TGA)

UK Strengthening Regulation to Protect Patients

The Medicines and Healthcare product Regulatory Agency (MHRA) has published plans to strengthen the regulation of medical devices and IVDs in the UK. A package of reforms will apply and include, for example, X-ray machines and insulin pumps as well as new technologies such as smartphone apps and artificial intelligence. UK to strengthen regulation of medical devices to protect patients – GOV.UK (

New Q&A Document on the Interface Between CTR and IVDR

The Q&As were issued at the end of May by the EC’s Medical Device Coordination Group and Clinical Trial Expert Group and aim to clarify certain interfaces between the Regulations. mdcg_2022-10_en.pdf (

ICH Guideline

E8(R1) on General Considerations for Clinical Studies

Training presentation on E8(R1): Microsoft PowerPoint – ICHE8(R1)_Step4Presentation_2022_0408


EU and US Collaboration on GCP Inspections

New research reveals the extent to which the US Food and Drug Administration (FDA) and the EMA agree on GCP inspection findings. Continued collaboration aims for broader inspection coverage and avoidance of duplications, leading to more efficient use of resources. Descriptive Analysis of Good Clinical Practice Inspection Findings from U.S. Food and Drug Administration and European Medicines Agency (

Annual EMA Report

Although inspection activities remain an area of focus for the EMA, the number of GCP inspections carried out in 2021 decreased to 36, compared with 59 in 2020. The decrease can be explained by travel and safety restrictions caused by the pandemic. Annual reports and work programmes | European Medicines Agency (

FDA’s Bioresearch Monitoring (BIMO) Program Inspection Metrics

The BIOM program delivers on-site inspections and data audits designed to monitor all aspects of conduct and reporting of FDA’s regulated research.

In 2021, the total number of inspections were reduced significantly compared to the previous year. In March 2020, FDA paused on-site inspections and only conducted ‘critical’ ones. To maintain oversight, BIMO introduced Remote Regulatory Assessments (RRAs), which are voluntary remote evaluations of data and processes conducted via video teleconference. RRAs are however not equivalent to on-site inspection and do not result in classifications. PowerPoint Presentation (


Cancer Grand Challenges Program

The US National Cancer Institute and Cancer Research UK have partnered to launch this programme which aims to provide multiple rounds of funding for interdisciplinary research teams whose novel ideas have the greatest potential to advance cancer research and improve outcomes for the people affected. Four multinational, interdisciplinary teams selected to address major challenges in cancer | National Institutes of Health (NIH)

European Health Data Space

The aim of this initiative is for a connected data system across Europe in order to create more efficient and effective research and development as well as a better planning and delivery of patient-centred care. efpia-recommendations-on-a-connected-data-system-in-europe_final.pdf

Clinical Trials Transformation Initiative (CTTI)

Following a multi-stakeholder expert meeting in May, a statement has been published which recommends embedding elements of clinical trials into routine patient care. CTTI Holds Meeting to Discuss Embedding Clinical Trials in Health Care Settings – CTTI (

Innovative Medicines Funds (IMF)

In June, the UK government launched the IMF which aims to give NHS patients in England early access to new potentially life-saving and cutting-edge treatments. This includes rare diseases and cancer. Patients to have earlier access to cutting-edge treatments on NHS – GOV.UK (

New FDA Plan for rare Neurodegenerative Diseases

Five-year action plan, published June 2022: FDA Releases Action Plan for Rare Neurodegenerative Diseases, Including ALS | FDA


International Collaboration for Use of RWE

EMA has endorsed a joint statement calling for international collaboration to enable the generation and use of RWE for regulatory decision-making. Global regulators call for international collaboration to integrate real-world evidence into regulatory decision-making | European Medicines Agency (

COVID-19 RWE Observational Studies

A report published by the International Coalition of Medicines Regulatory Authorities (ICMRA) highlights how collaboration on observational research during the pandemic can be of use and how this may be extended beyond COVID-19. ICMRA Teleconference Minutes COVID-19 Real-World Evidence observational studies | International Coalition of Medicines Regulatory Authorities (ICMRA)

Guidance on Use of RWE

In July, Swissmedic issued a position paper providing guidance on marketing authorisation applications containing RWE. Swissmedic position paper on the use of real-world evidence


Decommission of eSUSAR

The MHRA is retiring the eSUSAR website in favour for Individual Case Safety Reports (ICSR) Submissions – providing users with a more robust, stringent, and transparent way of expediting Suspected Unexpected Serious Adverse drug Reactions (SUSARs) from Clinical Trials of Investigational Medicinal
Products. Decommission of eSUSAR – MHRA Inspectorate (

Impact of UK Combined Review

New data shows that the UK’s combined review for clinical trials considerably reduces the length of time taken to approve health research in the UK. New clinical trials data shows the impact of combined review – Health Research Authority (


New FDA Guidance – ‘Providing Submissions in Electronic Format – Postmarketing Safety Reports’

Guidance for Industry (April 2022): Guidance for Industry; Providing Submissions in Electronic Format — Postmarketing Safety Reports (

Instructions for Use – Patient Labeling for Human Prescription Drug and Biological Products – Content and Format

Guidance for Industry (July 2022): Instructions for Use-Patient Labeling for Human Prescription Drug and Biological Products-Content and Format-Guidance for Industry (

Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-For-Purpose Clinical Outcome Assessment

Draft guidance for Industry, FDA staff and other stakeholders (June 2022): Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments (

Evaluation of Therapeutic Equivalence

Draft guidance for Industry (July 2022): Draft Guidance for Industry: Evaluation of Therapeutic Equivalence (

Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics

Draft guidance for Industry (June 2022): Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics (

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