Regulatory Update

Our Industry Update Newsletter returns for this second update of 2022 and focuses on Regulations & ICH Guidelines; EU, UK & US Guidance; 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.


Regulation on Crisis Preparedness – (EU) 2022/123

This regulation which became applicable 1st March, reinforces European Medicines Agency’s (EMA’s) role in crisis preparedness and management for medicinal products and medical devices. Regulation on EMA’s extended mandate becomes applicable | European Medicines Agency (

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

The European Commission (EC) has updated the questions and answers (Q&As) document.

On the 1st February, the Q&A document was updated to version 5.0 and included substantial amendments (12 new questions plus updates to many others).

Version 6.0, which became effective on 12th April, includes three new questions. The complete document is now 143 pages long. regulation5362014_qa_en.pdf (

Regulation on Health Technology Assessment – (EU) 2021/228

The EMA and the European Network for Health Technology Assessment EUnetHTA) 21 consortium published a joint workplan to focus on the preparation of this forthcoming Regulation.

(EU) 2021/228 will become effective in January 2025, following a three year implementation period. EMA and the EUnetHTA 21 consortium set priorities for their collaboration | European Medicines Agency (

ICH Guideline E8(R1) on General Considerations for Clinical Studies

This revision was published in October last year and the effective date in 14th April: ICH guideline E8(R1) Step 2b on general considerations for clinical studies (


EMA’s PRIority MEdicines (PRIME) Scheme

The PRIME scheme was launched in March 2016 to provide early and enhanced scientific and regulatory support to medicines that have the potential to significantly address patients’ unmet medical needs. The EMA has issued a detailed report on the first 5 years’ experience of PRIME. prime- 5-years-experience_en.pdf ( and prime-analysis-first-5-years-experience_en.pdf (

EMA Human Medicines Highlights 2021

The EMA has published an overview of the key recommendations made last year on the authorisation and safety monitoring of medicines. Human Medicines Highlights 2021 (

Electronic Product Information (ePI)

In February, the European Medicines Regulatory Network adopted a new Common Standard for the EU. European medicines regulatory

Updated Integrated Research Information System (IRIS) Guide for Applicants

The EMA published version 2.10 on 16th May: iris-guide-applicants-how-create-submit-scientific- applications-industry-individual-applicants_en.pdf (


Two New Medicines and Healthcare product Regulatory Agency (MHRA) Guidance Documents Published at the end of January 2022

(1) Risk-Adapted Approach to Clinical Trials

This new guidance supports a risk-based approach to clinical trials. There is a dual strategy:

(2) Oversight and Monitoring Activities

The MHRA now supports a proportionate approach to the management and monitoring of clinical trials, incorporating the risk assessments detailed above. This is a move away from detailed protocol compliance assessments and 100% source data verification. Oversight and monitoring activities – GOV.UK (

Two New Templates for Commercially Sponsored Studies

The National Health Service (NHS) and HRA have launched two new model templates:

  • Confidentiality Disclosure Agreement
  • Non-Interventional Study Agreement

Reducing the time to set up commercially sponsored studies in the UK – Health Research Authority (

UK to Improve Public Involvement in Research

Agencies, including MHRA, Health Research Authority (HRA), National Institute for Health Research and Association of British Pharmaceutical Industry (ABPI), are working together to improve public involvement in research. Health and social care leaders unite to improve public involvement in research – GOV.UK (

UK to Improve the Lives of People with Rare Diseases

The ABPI has published a series of blog articles relating to rare diseases By working together we can improve the lives of people with rare diseases (


New Food and Drug Administration (FDA) Guidance – ‘Patient-Focused Drug Development: Methods to Identify What is Important to Patients’

Guidance for industry, FDA staff and other stakeholders (February 2022): Final Guidance: Patient- Focused Drug Development: Methods to Identify What is Important to Patients (

New FDA Guidance on Bioavailability Studies

General considerations, guidance for industry (April 2022): Bioavailability Studies Submitted in NDAs or INDs – General Considerations (

New FDA Guidance on Population Pharmacokinetics

Guidance for industry (February 2022): Guidance for Industry (

FDA Draft Guidance for Industry – ‘Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials’

Comments should be submitted by 13 June: Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials Guidance for Industry (


New European Coordination Centre for Data Analysis and Real World Interrogation Network – DARWIN EU

At the beginning of February, the EMA established a network of RWD sources across the EU. The aim is to provide the EMA and EU Competent Authorities with valid and trustworthy RWE. Data Analysis and Real World Interrogation Network (DARWIN EU) | European Medicines Agency (

Using RWE in EU Medicines Regulation

Regulators in Europe have published two articles outlining their vision for how RWE will complement randomised clinical trials data to answer regulatory questions and support decision making. Real-World Evidence in EU Medicines Regulation: and What was the Contribution of Real-World Evidence?


Several New FDA Guidance on Advancing Drug Development in Oncology

(1) Inclusion of older adults in cancer clinical trials: Inclusion of Older Adults in Cancer Clinical Trials (

(2) Expansion Cohorts: Use in first-in-human clinical trials to expedite development of oncology drugs and biologics Expansion Cohorts: Use in First-in-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics (

(3) Master Protocols: Efficient clinical trial design strategies to expedite development of oncology drugs and biologics Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics (

EU – Cancer Screening

The Science Advice for Policy by European Academies has published a detailed report promoting the improvement of cancer screening in the EU. For cancer patients, early diagnosis is a key determinant of timely treatment and survival outcome. cancer-screening-report.pdf ( and Improving cancer screening to help beating cancer (

EU – Cancer Medicines Forum

The EMA in collaboration with the European Organisation for Research and Treatment of Cancer, has launched a forum aiming to optimise cancer treatment by bringing together representatives from the European medicines regulatory network as well as academia. EMA establishes Cancer Medicines Forum with academia to optimise cancer treatments in clinical practice | European Medicines Agency (


Advice to Sponsors on Managing Clinical Trials in Ukraine

EMA – Advice to sponsors on managing the impact of the war in Ukraine on clinical trials | European Medicines Agency (

EU Heads of Medicine Agencies working group – AGES Standardvorlage englisch (

UK NHS HRA – Fast-track research review for studies affected by the war in Ukraine – Health Research Authority (

Accelerated Clinical Trials in the EU “ACT EU”

At the beginning of the year, the European Federation of Pharmaceutical Industries and Associations (EFPIA) issued a statement welcoming the ACT EU initiative, which was launched by EC, EMA and HMA (see Q1 Newsletter for further information). EFPIA statement on EMA’s “Accelerating Clinical Trials in the EU (ACT EU)” initiative

Clinical Trial Information System (CTIS)

The EMA has renamed the CTIS Highlights newsletter to Clinical Trials Highlights. The newsletter will continue to provide details of CTIS, but will now also include topics such as ACT EU, see above. clinical- trials-highlights-march-2022_en.pdf (

The EMA has issued a draft guidance document relating to the protection of personal data and commercially confidential information uploaded and published in CTIS. Draft_ Guidance document on protection of personal data and commercially confidential information (CCI) in CTIS (

Draft ICH Guideline on Paediatric Extrapolation

This guideline is currently under public consultation: ICH_E11A_Document_Step2_Guideline_2022_0404_0.pdf

Draft ICH Guidelines on ‘Analytical Procedure Development Q14’ and ‘Validation of Analytical Procedures Q2(R2)’

Guidelines are currently under public consultations: ICH_Q14_Document_Step2_Guideline_2022_0324.pdf and ICH_Q2- R2_Document_Step2_Guideline_2022_0324.pdf

Record Retention

Health Canada has reduced the retention period for clinical trial records from 25 to 15 years. Notice: Period reduced for keeping clinical trial records for drugs and natural health products –

If you require support or advice with any issues raised by this latest industry and Clinical Trials update, please contact us here.