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.
REGULATIONS & ICH GUIDELINES
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 (europa.eu)
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 (europa.eu)
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 (europa.eu)
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 (europa.eu)
EU GUIDANCE & REPORTS
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 (europa.eu) and prime-analysis-first-5-years-experience_en.pdf (europa.eu)
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 (europa.eu)
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 (europa.eu)
UK GUIDANCE & BLOGS
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:
- Stratification of trials according to the risk of the Investigational Medicinal Product (IMP)
- Trial-specific risk assessment to identify particular vulnerabilities Risk-Adapted Approach to clinical trials and Risk Assessments – GOV.UK (www.gov.uk)
(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 (www.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
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 (www.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 (abpi.org.uk)
US GUIDANCE
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 (fda.gov)
New FDA Guidance on Bioavailability Studies
General considerations, guidance for industry (April 2022): Bioavailability Studies Submitted in NDAs or INDs – General Considerations (fda.gov)
New FDA Guidance on Population Pharmacokinetics
Guidance for industry (February 2022): Guidance for Industry (fda.gov)
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 (fda.gov)
REAL WORLD DATA (RWD) & REAL WORLD EVIDENCE (RWE)
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 (europa.eu)
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?
ONCOLOGY
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 (fda.gov)
(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 (fda.gov)
(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 (fda.gov)
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 (sapea.info) and Improving cancer screening to help beating cancer (efpia.eu)
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 (europa.eu)
MISCELLANEOUS
Advice to Sponsors on Managing Clinical Trials in Ukraine
EU Heads of Medicine Agencies working group – AGES Standardvorlage englisch (hma.eu)
UK NHS HRA – Fast-track research review for studies affected by the war in Ukraine – Health Research Authority (hra.nhs.uk)
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 (europa.eu)
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 (europa.eu)
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 – Canada.ca