EU Clinical Trials Regulation - IPPOSI perspective
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IPPOSI CEO, Dr Derick Mitchell delivered a presentation on the EU Clinical Trials Regulation from the patients' perspective at the 20th International Conference on Pharmaceutical Medicine, Athens, Greece.
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EU Clinical Trials Regulation - IPPOSI perspective
- 1. The EU Clinical Trials Regulation from a patients’ perspective Derick Mitchell IPPOSI ICPM, October 19th, 2022
- 2. Overview Who are IPPOSI? Setting the standard for greater diversity in CTs The power of publicly-accessible information Educated patients in European countries
- 3. Who are IPPOSI? We are a patient-led organisation that works with patients, science & industry to put patients at the heart of health innovation. www.ipposi.ie info@ipposi.ie @IPPOSI
- 4. What do we do, and how? • Digital discussions • In-person conferences • Training programmes • Matchmaking • Member resources • Coalitions and networks • Citizens’ juries EDUCATION ADVOCACY INFORMATION 50/50 public : private funding
- 5. IPPOSI and Clinical Trials? CTReg an opportunity for EU to act as a role model for patient engagement, stirring culture change globally! Patient voices must be recognised and put first!
- 6. Transitionary period – where are we now? • Effectiveness of CT regulatory framework is of fundamental importance for our patient members • Policy environment in Europe is switching towards patient focus, engagement, and transparency • Patients have the right to be involved in the way clinical trials are developed, managed and evaluated • Analysis of CT applications continues to show preponderance of small, single member state studies • Effective, Proportionate, and Patient-centred CT is the goal but has that been the reality so far?
- 7. Fundamental Questions... • Is making the EU ‘attractive/competitive’ for CTs different from making EU a ‘role model’ to influence global standards? • Are the envisaged efficiencies in some areas being offset by increased workloads in others? • Are the lessons from COVID-19 being learned?
- 8. Improving Diversity in Clinical Trial Participants • Diversity a key focus of CTReg • Informed Consent processes must be transparent, honest and helpful • CTReg has clearly defined informed consent and trial conditions for vulnerable populations: • broad consent • simplified consent for cluster trials • consent for minors or incapacitated people • consent for pregnant or breastfeeding women • consent for other vulnerable populations • consent during trials in emergency settings • updates the definition of a legally designated representative • Will these encourage more CTs in the EU in indications (to date) hardly covered?
- 9. Maybe not on their own...but in combination with greater levels of... • Understanding health disparities + experiences + motivations • Increased use of patient preference studies AWARENESS • Both internal + external - define what ‘engagement’ means • Tailored approaches – with/without technology ENGAGEMENT/INVOLVEMENT • For sponsors – exclusivity periods? • For investigators – training on meeting information needs INCENTIVES/SUPPORTS
- 10. What information empowers patients? Systematic provision of RELIABLE and ACCESSIBLE information for a broad audience CTReg includes both legal requirement for lay summaries as well as a publicly accessible portal
- 11. EU Legal requirement for (timely) Lay Summaries • Develops and reinforces public/patient trust & confidence in clinical trial processes • Combining scientific rigour with readability a significant challenge & needs training & support • Recognition that patient involvement in the process of creating lay summaries brings added value and better results • Should become the norm for everyone in clinical research, not just trials Oct ’21: EC publishes “Good Lay Summary Practice Guideline” - EudraLex Volume 10
- 12. Public portal/workspace? • CTIS will have two restricted, secured workspaces (for sponsors and authorities) and a public website • Overview of clinical trial statistics • Download of data and documents • Will the public website deliver empowering information that is easily accessible?
- 13. Promoting a culture of transparency • Transparency requirements require new ways of working with all key stakeholders to ensure personal / confidential information is protected • Informed consent involves a full and frank discussion on data protection, privacy and possible sharing of patients’ data for further research • Patient expectations for reliable CT results when wanted are very real – COVID-19 ushered a quantum leap in public interest in clinical trials
- 14. EUPATI Patient Engagement Roadmap Improving Patient Involvement in Medicines Research and Development: A Practical Roadmap. Geissler, Ryll, Leto, Uhlenhopp, Therapeutic Innovation & Regulatory Science (2017), doi: 10.1177/2168479017706405, and at www.eupati.eu Setting research priorities Protocol synopsis Protocol design Trial Steering Committee Information to participants Data & Safety Monitoring Board Investigator meetings Fundraising for research Practical Considerations Patient Information Informed Consent Ethics Review Study Reporting Post-Study Communication Regulatory Affairs Health Technology Assessment Research Design and Planning Research Priorities Research Conduct and Operations Market Authorization and Post-approval • gap analysis • early horizon scanning • matching unmet needs with research • defining patient- relevant added value and outcomes • design • target population • relevant endpoints, • PRO • in-/exclusion criteria • diagnostic procedures • patient-reported outcomes/QoL measures, • benefit/risk balance • ethical issues, • mobility issues/logistics, • data protection • protocol follow up • improving access • adherence • protocol amendments • new safety information • trial design • recruitment • challenges, • opportunities, • can trigger amendments • benefit/risk • drop-out issues • amendments • MAA evaluation • EPAR summaries • package leaflets • updated safety communications • lay summary of results • contribution to publications • dissemination of research results to patient community / professionals • contribution to publications • dissemination of research results to patient community / professionals • summary of interim results • dissemination to patient community • content • visual design • readability • language High expertise in disease area required Medium expertise in disease area required • content • visual design • readability • language • dissemination • contractual issues • travel expenses • support for family members • mobility
- 15. EUPATI OPEN CLASSROOM Patient Expert Training Programme • Expert-level training on medicines R&D • Non-disease specific • Online + In-person/live streaming • 12-14 months • Continuous enrollment • 200+ graduates = EUPATI Fellows learning.eupati.eu
- 16. EUPATI Impact EUPATI Fellows before training… EUPATI Fellows after training… 1. Leadership roles in patient organisations 59% 72% 2. Advising pharmaceutical companies 20% 54% 3. Advising a regulatory agency 17% 48% 4. Advising a reimbursement / HTA body 5% 12% Source: Internal EUPATI Cohort 1-3 Surveys, 144 respondents
- 17. EUPATICONNECT Enhancing collaboration between patients & researchers • Matchmaking platform • Bringing together EUPATI Patient Experts with researchers • Enhancing patient involvement (e.g. protocol review, focus groups, speaking opportunities, patient advisory boards, ethical committees, regulatory processes) connect.eupati.eu
- 18. Implementation of CTReg If sponsors can answer the following questions, they should be able to see the relative potential of embracing the EU as a place to conduct CTs 1. What motivates participants and how do they deal with everyday challenges of their lives? 2. What are the administrative and specific regulatory requirements that empower or challenge the adoption of new solutions? 3. What are the (in-)surmountable barriers in the CT that are defined by the protocol? 4. How can I include educated patient experts through meaningful patient engagement to increase the quality + relevance of the CT? 5. How can we maximise the value of patient engagement for improving research quality and transparency in a post-COVID-19 world?
- 19. www.ipposi.ie info@ipposi.ie @ipposi Irish Platform for Patient Organisations, Science and Industry
Editor's Notes
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- 2019 – conference on EHRs 2021 – three digital discussions on digital health: Ireland’s preparedness, European commitments, Citizen/patient involvement – we also hosted three podcasts around the topic 2021 – coalition around COVID-19 vaccines. 2021 – citizens’ jury on access to health information for secondary purposes 2022 – citizens’ jury on the future use of genomics in healthcare and research in Ireland Ongoing – modules on clinical trials, medicines regulation, health technology assessment – and soon research ethics – and digital health Ongoing – match patients completing our training with available PPI opportunities Ongoing – prepare resources to help members with their own education or advocacy – e.g. PPI mapping, national and EU consultation responses, toolkits and briefing notes Ongoing – campaign to raise awareness about the importance of building a digital health future Ongoing – network of graduates and ENP linked to EUPATI
- Massive opportunity to influence health research - beyond the CTReg and beyond medicines regulation Key elements for IPPOSI are the recommendations to include patient engagement throughout the process of development and dissemination of Lay Summaries for adult and paediatric clinical studies.
- Ultimately impacts patients’ access to new therapies throughout the EU Sponsors can still choose to submit under CTD until Jan 2023 There are still many unanswered questions about how the common objectives are to be fully and efficiently achieved
- Many ‘firsts’ in the CTRegs, internationally, COVID-19: de-centralization of CTs to promote a more responsive research environment? Jan 2022: Accelerating Clinical Trials in Europe (ACT-EU) initiative, aims to "engage all stakeholders to deliver inclusive patient-oriented medicines development and delivery across populations."
- For clinical studies to be truly patient-centric and for medicines development to be effective for the highest number of patients,
- Differences in age, gender, and racial background can lead to different effects from the same treatment, and some groups may experience completely different results from the majority. To make clinical trials/research more diverse, we need to: Engage with communities – bringing clinical trial knowledge and expertise into the places people are seeking care. Armed with this knowledge, primary care physicians and consultants can have conversations with eligible patients and make recommendations of clinical trials which are accessible and beneficial to them. Increase diversity amongst researchers – patients are more likely to trust and participate in clinical research when they can relate to those involved with the studies Invest in targeted outreach – CROs and sponsors should co-create clinical trial communications with patient groups and minority communities, and work together on outreach programmes. However, when it comes to making vulnerable groups rights real in their daily lives, the Member State’s duty is to lead, not impede. Vulnerable groups are overlooked in many child- and disability-focused laws, policies and programmes. Vulnerable groups are not actively or effectively involved in consultations on many existing laws, policies and programmes affecting them. Vulnerable groups are relatively invisible in data. How can we maximise the value of patient engagement for improving research quality and transparency in a post-COVID-19 world?
- Lay summaries give researchers a channel to better communicate results and impact but also has a positive knock-on effect in the overall EU regulatory system for medicines Dispells public concerns that trials are part of an opaque black-box procedure Contributes to a more conducive environment for continued research Oct ’21: EC publishes “Good Lay Summary Practice Guideline” in EudraLex Volume 10. Patient expectations for reliable CT results when wanted are very real – patients need access – COVID-19 ushered a quantum leap in public interest in clinical research To ease funding constraints, particularly for academic studies, need to change the current concept of eligible research costs in research grants, so that they allow for LS costs out of time
- Legal requirement for lay summaries within a short period of time after last patient-last visit Gives researchers a channel to better communicate results and impact Has a positive knock-on effect in the overall EU regulatory system for medicines Dispells public concerns that trials are part of an opaque black-box procedure Contributes to a more conducive environment for continued research A good lay summary is an ethical imperative and a good thing - for patients, for public, and ultimately for regulation and for research. • Producing a good lay summary is very demanding, and requires skills, time and resources – and clear understanding of what GLSP really means in practical terms. • Patient engagement will be critical throughout. To ease funding constraints, particularly for academic studies, it is essential to win changes to the current concept of eligible research costs in research grants, so that they allow for GLSP costs out of time
- DPOs, experts in data de-identification, etc. Establishing disclosure/transparency policies, procedures, strategies & incorporating into clinical development plan + all clinical trial activities Will regulatory confidentiality ‘trump’ public disclosure?
- EUPATIConnect is a fee-for-service matchmaking platform that connects EUPATI Patients Experts (EUPATI Fellows & Open Classroom learners) and in some instances, lay patients with researchers looking to involve patients in various projects (e.g. protocol review, focus groups, speaking opportunities, patient advisory boards, ethical committees, regulatory processes). EUPATIConnect does not provide matchmaking for participation in clinical trials. The purpose is to facilitate matchmaking on both sides. By being part of the platform, EUPATI Patient Experts will be able to indicate their availability to participate in various projects, and on the other hand, researchers will be able to submit their requests to find patient partners for their projects. The EUPATI Secretariat, via EUPATIConnect, will connect both parties.