Advisor 462

CR & CQA Advisor issue 462

ISSN 2041-3661

1 J UNE 2 0 2 0

New European pharmacovigilance inspection follow-up procedures The European Medicines Agency has published a new guideline outlining the expected follow-up procedures for pharmacovigilance inspections performed in the EU. The guideline, which came into effect on 1 May 2020, supports harmonisation for the mutual recognition of pharmacovigilance inspections and facilitates administrative collaboration and the exchange of inspection-related information. It also includes new sections on managing unacceptable responses to inspection findings, sharing inspection reports, periodic progress reports and post-inspection meetings, plus timelines for a number of key activities. More on page 5 u EC issues guidance to mitigate EU clinical trial disruption during the pandemic The European Commission (EC) has published further guidance on the conduct of clinical trials in Europe during the current pandemic. Issued on 28 April, Version 3 of the guidance aims to mitigate the disruption of clinical research in Europe in an effort to avoid further negative effects of the pandemic, without compromising on data quality and participant safety. It is hoped that the temporary recommendations – which will be revoked once the current crisis is over – will contribute to the overall strategy for finding safe and effective treatments and developing a vaccine to protect citizens against the virus. See page 4 u

Frequency of MHRA GPvP inspection findings unchanged The Medicines and Healthcare products Regulatory Agency’s (MHRA’s) Good Pharmacovigilance Practice (GPvP) Inspectorate has issued its annual metrics report for pharmacovigilance inspections completed during the 12 months to 31 March 2019. Across the 18 inspections performed, just two were triggered by critical findings from previous inspections. A total of 120 inspection findings were reported, with four critical, 78 major and 38 minor inspection findings. Overall, the average number of inspection findings per inspection has remained fairly constant over time and currently stands at 6.7. Learn more on page 3 u

Korean device company’s CAPA procedures and responses fail to impress the FDA A medical device company in South Korea has received a second FDA warning letter. The letter refers back to an earlier inspection and notes that the company’s corrective and preventive action (CAPA) procedures did not allow for the full investigation or resolution of issues identified in the first inspection. Device manufacturers should make sure that their CAPA documentation demonstrates to the FDA that their quality system is effective, and that it allows them to quickly identify problems and to implement effective CAPAs, as needed. Details on page 8 u

EMA addresses validation and qualification of computerised systems used in trials The European Medicines Agency (EMA) has issued a new Notice for sponsors that highlights how the inadequate validation and qualification of computerised clinical trial systems can impact the integrity of clinical trial data. The agency refers to recent inspection findings that include poor documentation of computer system validation and qualification, lack of inspector access and inadequate contractual arrangements. Find out more on page 6 u

Issue 462

CLINICAL RESEARCH • CLINICAL QUALITY ASSURANCE

STOP PRESS – global news

EMA starts rolling review of a potential COVID-19 treatment The European Medicines Agency (EMA) has begun a ‘rolling review’ of data on the use of the investigational antiviral medicine remdesivir for the treatment of coronavirus disease (COVID-19). The decision by the Committee for Medicinal Products for Human Use (CHMP) to start the rolling review was based on preliminary study results that suggest a beneficial effect in hospitalised patients with mild-to-moderate or severe COVID-19. Announcing the decision on 30 April 2020, the EMA explained that a rolling review is a regulatory tool that can be used to speed up the assessment of a promising investigational medicine during a public health emergency, such as the ongoing pandemic. Commencing a rolling review does not imply that a drug’s benefits outweigh its risks. In a rolling review, CHMP Rapporteurs are appointed while development is ongoing, and data are reviewed as they become available. Several rolling review cycles can be performed during the drug’s evaluation as data emerge, with each cycle lasting around 2 weeks. Once the data package is complete, the developer can submit a formal marketing authorisation application, which is then processed under a shortened timetable. While the overall review timeline for remdesivir is currently unknown, the EMA should be able to complete its assessment significantly more quickly than usual, while still ensuring that German guidance on clinical trials during the pandemic Although the German Federal Institute for Drugs and Medical Devices (BfArM) participated in compiling the European Commission’s guidance (Version 3.0) on the management of clinical trials during the current pandemic, BfArM has also issued supplementary local guidance entitled ‘Supplementary Recommendations to the Document European Guidance on the Management of Clinical Trials during the COVID-19 (Coronavirus) Pandemic ’. Within its supplementary guidance (issued on 26 March 2020), BfArM highlights the need to consider specific national laws and guidelines in individual Member States on key clinical trial topics, which may take precedence over European recommendations. The guidance specifically addresses remote data monitoring and the shipment of investigational medicinal product to trial subjects. Source: a robust scientific opinion is reached. Source:

Changing face of FDA drug approvals

A study published in JAMA Network Open on 21 April 2020 evaluates whether the number and characteristics of pivotal efficacy trials supporting the US approval of new drugs and biological products have changed over the past 3 decades. Looking at 273 new drugs and biological products approved by the FDA for 339 indications in three periods (1995–1997, 2005–2007 and 2015–2017), the study found that more recent approvals increasingly used special regulatory programmes and were based on fewer pivotal trials than older approvals. When aggregated by indication, these trials had less rigorous designs but longer durations. The US authors suggest that ongoing post-approval evaluation of therapeutic safety and efficacy is needed. Source: Communicating results of COVID-19 trials On 4 May 2020, the Editors of JAMA highlighted concerns about the reporting of results from COVID-19 clinical trials. At the time of writing, more than 600 interventional studies and randomised clinical trials relating to COVID-19 were registered on ClinicalTrials.gov, with data likely to become available in the coming weeks and months. Presenting and interpreting these results clearly – and communicating findings appropriately to clinicians, the public and policy makers – are critically important. Investigators, journals and the media have an obligation to report the results, and what they mean for individuals and population health, accurately and responsibly. Source: New FDA guidance on non-binding feedback after device inspections On 21 April 2020, the FDA issued final guidance on non-binding feedback after certain FDA inspections of device establishments. The guidance explains how a device establishment may submit a request for non-binding feedback about the corrective actions it proposes to take to address certain inspectional observations documented by the agency during an inspection. The guidance identifies a standardised communication method, and describes how the FDA

evaluates and responds to requests. Source:

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Frequency of MHRA GPvP inspection findings unchanged Annual inspection metrics from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) Good Pharmacovigilance Practice (GPvP) Inspectorate suggest that the overall number of inspection findings per inspection continues to plateau.

Types of inspection The 18 inspections comprised • two inspections triggered by critical findings from previous inspections • 16 inspections scheduled and conducted in line with the routine national or EMA inspection schedule – nine were of MAHs that had never been inspected by the MHRA before, and seven were routine re-inspections of MAHs. Nine inspections included a remote inspection element. For four of these, office-based inspection days were planned and conducted prior to the on-site inspection. For six inspections, ad hoc office-based inspection days were required after the on-site inspection (eg. to review additional documentation for significant inspection findings or documentation that was not readily available during the on-site inspection). One inspection was conducted entirely remotely as part of a pilot project on office-based GPvP inspections. Overview of findings Four critical, 78 major and 38 minor findings were identified (Table 1). Overall, the average number of findings per inspection (all grades) for the 12 months to 31 March 2019 has remained relatively stable when compared with previous reporting periods and currently stands at 6.7. However, the average number of critical and major findings per inspection increased slightly from the previous 12-month period, while the average number of minor inspection findings fell from 3.14 to 2.11. The report includes a graph of the average number of inspection findings by type reported per inspection over time, going back as far as 2006. It indicates that the average number of critical findings has remained relatively constant throughout the

On 15 April 2020, the MHRA GPvP Inspectorate published its latest annual Pharmacovigilance Inspection Metrics

Report. A total of 18 inspections of marketing authorisation holders (MAHs) were conducted by the Inspectorate from April 2018 to March 2019, compared with 22 in the previous 12-month period. The purpose of the inspections was to examine compliance with existing EU and national pharmacovigilance regulations and guidelines; the metrics published by the Inspectorate relate to all 18 inspections. As in previous years, MAHs were selected for inspection using a risk-based methodology, in accordance with Good Pharmacovigilance Practice Module III. The national inspection programme for this reporting year also took into account the European Medicines Agency’s (EMA’s) programme of routine pharmacovigilance inspections of organisations with centrally authorised products. Factors considered in the risk-based approach include • product-specific risks (eg. new active substances or new biological products) • the complexity of the pharmacovigilance system • the complexity and size of the organisation(s) involved in the pharmacovigilance system, including service providers • the compliance and inspection history of an organisation. Findings identified during inspections were graded as critical, major or minor (definitions of these terms are provided in Appendix 1 of the metrics report). Below we summarise the number of inspection findings; in future articles we will examine the nature of these findings.

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Table 1 . Findings by inspection type (1 April 2018 – 31 March 2019).

Number of inspection findings a

Number of inspections

Critical

Major

Minor

Total

Inspection type

Routine initial inspection Routine re-inspection Previous critical finding

9 7 2

0 3 1

41 31 6

23 12 3

64 46 10

Total (average/inspection)

18

4 (0.22)

78 (4.33)

38 (2.11)

120 (6.67)

a Numbers estimated from bar charts in the report. period, while the average number of major findings per inspection has increased from a low of two in the reporting period January 2009 – March 2010 to just over four in the most recent period. The average number of minor findings reported per inspection

has also fluctuated over time. However, there has been an overall decrease from approximately 3.8 in the 12-month period to December 2006 to 2.1 in the most recent period. Source:

EC issues guidance to mitigate EU clinical trial disruption during the pandemic

The European Commission (EC) has published Version 3 of its guidance on managing clinical trials during the COVID-19 pandemic.

The aim of the guidance (released on 28 April 2020) is to mitigate the disruption of clinical research in Europe and avoid further negative effects of the pandemic, without compromising on quality and safety. The recommendations are an important part of the overall strategy for finding treatments and a vaccine to protect citizens against coronavirus. The original guidance was published on 20 March 2020 by the Clinical Trials Expert Group, supported by the European Medicines Agency (EMA), the Clinical Trials Facilitation and Coordination Group of the Heads of Medicines Agencies, and the EMA’s GCP Inspectors’ Working Group. Version 2 followed on 27 March 2020. With more than 200 coronavirus clinical trials now registered on the EU’s EudraCT database, the guidance offers recommendations for simple and flexible measures to respond to the current situation, and to ensure that patients participating in clinical trials across the EU can continue to receive their medicines.

Commissioner Stella Kyriakides said, “We are in the midst of the worst pandemic in recent memory and it is absolutely crucial that we show flexibility in our rules to maintain research on critical treatments, including chronic and rare diseases, through clinical trials. Developing and deploying effective diagnostics, treatments and a vaccine will also undoubtedly be the most important breakthrough to stop the coronavirus. On 4 May, our international pledging conference will kick-start global cooperation and support for this work, with the aim to raise €7.5 billion in funding to the benefit of the global community. Together in solidarity, we will prevail.” Key recommendations in the 21-page guidance document include the following. • Distribution of medicines to patients in clinical trials: the purpose is to protect the safety and well-being of trial participants and the integrity of clinical trials. The recommendation takes into account social distancing measures

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and possible limitations in trial site and hospital resources. • Remote source data verification (SDV): the verification of raw data in hospitals can be extremely difficult during the pandemic due to safety measures such as social distancing. Remote SDV to conclude a trial could facilitate the marketing authorisation of coronavirus treatments and life-saving medicines. • Communication to authorities: urgent actions to protect trial participants against any immediate hazard or other changes with an effect on patient safety or data robustness might become necessary to mitigate disruptions during the ongoing public health crisis. The guidance clarifies the classification and notification of these actions.

All these measures apply exclusively during the coronavirus pandemic, and will be revoked once the current health crisis in the EU/European Economic Area is over. Member States are encouraged to implement the harmonised guidance to the maximum possible extent to mitigate and slow down the disruption of clinical research in Europe during the public health crisis. At the same time, sponsors and investigators need to take into account that national legislation and derogations cannot be superseded. Member States should provide additional clarity on specific national legal requirements and derogations, as appropriate.

Source:

New European PV inspection follow-up procedures The European Medicines Agency (EMA) has issued a new guideline that explains follow-up procedures for pharmacovigilance (PV) inspections in the EU.

The 15-page guideline came into effect on 1 May 2020 and supersedes a previous document from 2014 (EMA/INS/PhV/192231/2014). It includes several new sections and provides a more detailed timeframe for post- pharmacovigilance inspection activities. After every inspection, the competent authority is required to report on whether the marketing authorisation holder (MAH) complies with the requirements laid down in Directive 2001/83/EC. According to Article 111(8) of the Directive, if the pharmacovigilance inspection finds that the MAH does not comply with the pharmacovigilance system as described in the Pharmacovigilance System Master File and with Title IX of the Directive, the competent authority of the Member State concerned has to inform the MAH of the deficiencies and give them an opportunity to submit comments. Other Member States, the EMA and the European Commission also have to be informed.

Any identified non-compliance has to be rectified by the MAH in a timely manner through the implementation of a corrective and preventive action (CAPA) plan. Inevitably, depending on the nature of the findings, some pharmacovigilance inspections will require significant follow-up and management. The new guideline defines the steps in the follow-up of pharmacovigilance inspections and the responsibilities of the various parties involved, including the following: • the process for requesting a CAPA plan from the MAH – when an inspection report has been issued to the MAH (or other inspected organisation), the lead inspector should propose date(s) for the receipt of responses to the findings listed in the inspection report; this is generally within 30 working days following receipt of the report, unless there are specific national requirements for provision of responses or the lead inspector determines that an expedited response should

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are fully investigated and that an appropriate and timely CAPA plan is implemented to address the findings, with appropriate prioritisation of critical and/or major findings • inform the EMA lead inspector if timelines for agreed CAPA deliverables change • ensure the timely evaluation of any new safety data identified through inspection • ensure timely communication about safety concerns to the competent authorities, patients and healthcare professionals, in particular notifying changes to the benefit-risk balance of concerned medicinal product(s) according to the urgency required (including the implementation of variations to marketing authorisations for safety reasons) • respond to requests from competent authorities, including the provision of correct and complete information.

be provided. The responses should address non- compliance and be provided in the form of a CAPA plan. • CAPA plan review and approval by the inspectors, to determine whether it is adequate – this should usually occur within 30 working days following receipt of the responses; a shorter review deadline (10 working days) applies for responses to inspections requested by the Committee for Medicinal Products for Human Use. • routine interaction within and between Member States and the EMA. • actions to be taken following the identification of inspection findings that may impact the robustness of the benefit-risk profile of medicinal product(s). • re-inspection planning. Sponsor responsibilities The main responsibilities of the MAH during the post-inspection period are to • ensure that findings identified during an inspection

Source:

EMA addresses validation and qualification of computerised systems used in trials

The European Medicines Agency (EMA) has issued a short Notice to sponsors on the validation and qualification of computerised systems used in clinical trials.

Recent inspection findings have included issues with the integrity, reliability, robustness and acceptability of data submitted within marketing authorisation applications (MAAs). This has led the GCP Inspectors Working Group – in cooperation with the Committee for Medicinal Products for Human Use (CHMP) – to issue a Notice (dated 7 April 2020) that highlights the requirements for sponsors/vendors providing computerised systems or services that are used to manage clinical trial data. It notes that European regulations require the information generated in a clinical trial to be recorded, handled and stored adequately for the purpose of ensuring effective regulatory inspection.

The International Council for Harmonisation E6(R2) Guideline requires sponsors to operate computerised trial data handling or computerised data systems, and to • validate these systems • maintain an audit trail for the initial entry of data and any subsequent changes • maintain a security system to protect against unauthorised access • maintain a list of individuals authorised to create, access, modify or delete data. Data integrity, reliability and robustness will depend on the design and validation status of the computerised systems used. Failure to document and therefore to demonstrate the validated

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state of a computerised system is likely to pose a risk to data integrity, reliability and robustness. Depending on the criticality of the affected data, this may result in GCP inspectors recommending that the CHMP does not use the data to support a MAA. Lack of documentation Recent inspection findings relating to the qualification and validation of computerised systems indicate that some sponsors have not been able to provide adequate documentation of the required qualification and validation activities for computerised data collection tools/ software during inspections. Computerised systems used in clinical trials may be built by the sponsor but are more likely to be purchased from a vendor, either under licence or as part of a service. Qualification activities might therefore be performed by the vendor, by the sponsor or both. The sponsor is ultimately responsible for the validation of the computerised system and for providing adequate documented evidence on the validation process. Sponsors must be able to provide GCP inspectors with access to the requested documentation, regardless of who performed the activities. The sponsor may rely on qualification documentation provided by the vendor, if the qualification activities performed by the vendor have been assessed as adequate. However, the sponsor may also have to perform additional qualification (and validation) activities based on a documented risk assessment. Further information on the conditions necessary for a sponsor to rely on a vendor’s qualification documentation are described in Question 9 of the EMA’s online questions and answers (Q&A) document on ‘GCP Matters’, which was also updated in April 2020. If the sponsor choses to perform its own full qualification of a system purchased from a vendor, the sponsor should have access to the vendor’s system requirement specifications, as otherwise the sponsor would not know all the built-in system functionalities and would consequently risk

unknown functionalities/actions with unknown impact on data.

Inadequate contractual arrangements Clear written agreements should be in place to document any arrangements between the sponsor and the vendor on qualification and validation. The sponsor remains responsible for ensuring that the conduct of the trial, the final data and data that are submitted within an MAA comply with the relevant legislation. Further information on potential pitfalls relating to contractual arrangements is provided in Question 8 of the EMA Q&A document. IT vendors may be inspected when they contractually assume clinical trial sponsor-related duties/activities and/or the contract between the sponsor and the vendor contains provisions for inspections/audits of duties/functions performed by the vendor. Inspectors should be able to inspect third parties who have trial-specific relevant documentation. However, as qualification documentation of generic software (ie. non-trial-specific software) does not necessarily fall into this category, the sponsor should ensure access for GCP inspectors in case any such activities are delegated to the vendor. The Notice points out that it is not acceptable to use computerised systems in clinical trials for which the validation status is not confirmed or for which appropriate confirmatory documentation cannot be supplied to GCP inspectors. If appropriate contracts cannot be put in place, vendor systems should not be used in clinical trials (regardless of whether sponsors have used them in the past), because serious GCP non-compliances and risks to data integrity, reliability and robustness could exist and go unnoticed by auditors and GCP inspectors if they are not allowed access. In addition, potential serious breaches may not be escalated appropriately by the vendor. Source: ,

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Korean device company’s CAPA procedures fail to impress FDA

Principal Author & Editor: Prof David Hutchinson Senior Contributor: Jane Baguley Production Editor/Writer: Sharon Jordan Senior Correspondents: Fabio Camarri, Mark Elsley, Hideki Fujiwara, Lisbeth Tofte Hemmingsen, Peter Marks, Stuart McCully, ColinWilsher Aim To provide news and information to allow clinical research and quality assurance professionals, trainers, regulators, academics and members of ethics committees to stay up to date with clinical research and good practice developments. Scope Executive summaries of key laws and guidelines relating to clinical research in the ICH regions. Summaries of relevant articles and information in other publications, press releases and information on the Internet. Information on: • changes in regulations, codes of practice, guidelines and new clinical research procedures • news from important meetings and conferences • ICH developments and progress • news, views and opinions about ICH GCP implementation • solutions to compliance-related problems • inspection findings and lessons to be learnt • clinical research methodology, statistical and legal issues • quality assurance issues and procedures • self- and independent audit practice • training courses, jobs and other opportunities. Sources of information • We gather news from correspondents and other sources around the world. • We gather intelligence from those actively involved in the regulatory process. • We review the major medical, clinical research and QA journals. • We search the web and regularly visit the websites of the major regulatory authorities in Europe, the USA and Japan, pharmaceutical industry and professional associations, major academic organisations and health associations. • Sources of information, current at the time of publication, are usually quoted at the end of each article. Published by: Brookwood Global (Canary Ltd), 5 Studley Court Mews, Chobham, Surrey GU24 8EB, UK Telephone: +44 1483 811383; Fax: +44 1483 812163 Email: info@brookwood-global.com; website © Canary Ltd 2020 All rights reserved. No part of this publication may be copied, transmitted, reproduced in any way without the written permission of the publisher. Disclaimer: Whilst we try to ensure that information published is correct, the Editors, Advisors or publishers accept no liability for losses or damages arising. You should always seek a second opinion before acting on any information provided.

The FDA has issued a warning letter to a South Korean medical device company detailing multiple deficiencies, including several relating to its corrective and preventive action (CAPA) procedures. The medical device company was initially inspected by the FDA in October 2015 and a first warning letter was issued in December 2016. A follow- up inspection took place in April 2019 and resulted in a second warning letter, which states that the device company had failed to adequately establish and maintain procedures for implementing CAPAs, as required by the federal regulations (21 CFR 820). The letter refers back to the original inspection, and indicates that the company’s CAPA procedures were inadequate as they had not fully investigated the root cause of all the previously identified inspection deficiencies. The deficiencies included findings relating to the design validation procedure, medical device reporting procedure, complaint handling procedure and device history record procedure, and other quality system deficiencies. These were all documented in an FDA Form 483 issued on 15 October 2015. The company responded that it had revised its guidelines in line with the FDA’s requirements for CAPA procedures, and provided a revised CAPA report for the agency’s input and evaluation. Other CAPA reports generated to address the observations from the second inspection were also provided, along with a CAPA Report for Observation from the October 2015 inspection, which included information on verification and validation performed for those particular CAPA procedures. However, in the second warning letter the FDA explained that it found the company’s response to be inadequate. Specifically, the company did not • provide evidence that it had investigated all the deficiencies identified in 2015 and conducted verification or validation to ensure that any actions taken were effective • address any plans to conduct a retrospective analysis of other potential non-conformities associated with each respective observation from the previous inspection. In response to these and several other findings described in the warning letter, the FDA has taken steps to prevent the company’s medical devices from entering the USA. Source:

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