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Global Guidance Dr Lembi bit Rgo Rgo Secretary-General Council for on patient involvement International Organizations of Medical Sciences (CIOMS) Joint PCWP-HCPWP Meeting, EMA, Amsterdam, 4 March 2020 Geneva, Switzerland

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Global Guidance

  • n patient involvement

Joint PCWP-HCPWP Meeting, EMA, Amsterdam, 4 March 2020

Dr Lembi bit Rägo Rägo Secretary-General Council for International Organizations of Medical Sciences (CIOMS) Geneva, Switzerland ragol@cioms.ch

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Content

2

What is CIOMS?

 Organization, links with other international organizations  Areas of work and Working Groups

CIOMS Working Group XI: Patient Involvement in Drug Development and Safe Use  Background and scope  WG processes, progress  Draft content  Examples of selected chapters  Plans for future Conclusions

EMA 4 March 2020

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What is CIOMS?

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Acronym & Logo

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Council for International Organizations of Medical Sciences Founded in 1949 by WHO and UNESCO In official relations with WHO and UNESCO associated partner ICH Observer since 2016

Mis ission ion Statem tement ent CIOMS mission is to advance public health through guidance on health research including ethics, medical product development and safety

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CIOMS in short

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Organization located in Geneva :

  • A. International
  • B. Nongovernmental
  • C. Not-for-Profit

Forum for discussion and neutral platform to elaborate new ideas in medical product development, pharmacovigilance and research ethics (bioethics) ... for WHO, health authorities, academic organizations, pharmaceutical industry and other concerned stakeholders CIOMS: an umbrella organization of medical science organizations

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Programs, Activities

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Core of activities: Technical Working Groups Run-time: Mostly 2-4 years, or even more than 10 years (SMQs) Impact: Legally not binding, yet significant influence on healthcare community (including decision makers and other organizations with impact); can also be transformed to be legally binding when embodied in regional/national legislation

  • Since 1967; 1. CIOMS

Round Table Conference “Biomedical Science and the dilemma of Human Experimentation”) Issuance of significant guidelines; latest revision 2016

  • Focus on "low and

middle income countries“; translated into many languages including Chinese, Spanish, Japanese, Russian

Bioethics

  • 1986 first

PV Working Group, 13 more working group reports until today

  • Several

ICH Guidelines are based on results of CIOMS Working Groups

Pharmaco

  • vigilance
  • Trends and

Prospects in Drug Research and Development, Proceedings of the 11th CIOMS Round Table Conference, Geneva, Switzerland, 8-9 December 1977.

  • Ed. Z. Bankowski,

J.F. Dunne, published by Scrip World Pharmaceutical News, London, 1978.

Product Develop- ment

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CIOMS communication Website: https://cioms.ch

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Members, links with other international organizations

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CIOMS membership

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Recent new members (2018)

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New international member of CIOMS

The International Federation of Associations

  • f Pharmaceutical Physicians and

Pharmaceutical Medicine (IFAPP)

New national member of CIOMS

Bangladesh Medical Research Council

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CIOMS and ICH (2)

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CIOMS is ICH Observer since 2016

MEMBERS

Foun undin ing g Reg egula lato tory y Membe bers

  • EC, Europe
  • FDA, US
  • MHLW/PMDA, Japan

Foundin ing Indust stry ry Members

  • EFPIA
  • JPMA
  • PhRMA

Standin ing Regula lato tory ry Members rs

  • Health Canada, Canada
  • Swissmedic, Switzerland

Regula lato tory ry Members rs

  • ANVISA, Brazil
  • CFDA, China
  • HSA, Singapore
  • MFDS, Republic of Korea

Industry stry Members rs

  • BIO
  • IGBA
  • WSMI

OBSERVERS

Stan

andin ing Obse servers vers

  • IFPMA
  • WHO

Legisl islati tive ve or Admin inist istrati rative ve Auth thorit itie ies

  • CDSCO, India
  • CECMED, Cuba
  • COFEPRIS, Mexico
  • INVIMA, Colombia
  • MCC, South Africa
  • National Center, Kazakhstan
  • Roszdravnadzor, Russia
  • TFDA, Chinese Taipei
  • TGA, Australia

Reg egio iona nal l Harmon

  • nisa

isatio tion Init itia iative ives s (RHIs) Is)

  • APEC
  • ASEAN
  • EAC
  • GHC
  • PANDRH
  • SADC

Inte tern rnati tional l Pharm rmace ceutica ical l Industry stry Organisa isatio ion

  • APIC

Inte tern rnati tional l Organisa isati tion regula late ted

  • r affe

fecte cted by ICH Guid ideli line(s) (s)

  • Bill & Melinda Gates Foundation
  • CIOMS
  • EDQM
  • IPEC
  • PIC/S
  • USP
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CIOMS impact on ICH* Guidelines

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CIOMS pharmacovigilance guidelines served as a basis for several ICH guidelines. Some examples:

*ICH - The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use Work rking ing Group

  • up

ICH Guideline CIOMS WG I und II Reports (1990, 1992) ICH-E2A (1994): Clinical Safety Data Management – Definitions and Standards for Expedited Reporting CIOMS IA Report (1992) ICH E2B: Clinical Safety Data Management – Data elements for transmission of individual case safety reports CIOMS WGs II und III Reports (1992, 1995) ICH-E2C (1996): Clinical Safety Data Management – Periodic Benefit-Risk Evaluation Reports (PBRER) CIOMS WG V Report (2001) ICH-E2D (2003): Post-Approval Safety Management – Definitions and Standards for Expedited CIOMS WG VIII Report (2006) ICH-E2F (2010): Development Safety Update Reports

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Cooperation with ICH

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Extract from MedDRA MC to ICH Assembly

November 2019, Singapore

Med = Medical D = Dictionary for R = Regulatory A = Activities

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Areas of Work and Working Groups

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Pharmacovigilance: Working Groups

Overview of CIOMS activities, including Working Group XI 15

Working ng Group Period (some examples es) Report / Year CIOMS I

  • International Reporting of Adverse Drug Reactions (1990)

CIOMS II

  • International Reporting of Periodic Drug Safety Update Summaries (1992)

CIOMS III

  • Guidelines for Preparing Core Clinical Safety Information on Drugs (1995)

CIOMS IV 01/1995 – 07/1997 Benefit-risk balance for marketed drugs (1998) CIOMS V 04/1997 – 08/2000 Current Challenges in Pharmacovigilance: Pragmatic Approaches (1999) CIOMS WG on SMQs 05/2002 - Development and Rational Use of Standardized MedDRA Queries (SMQs): Retrieving Adverse Drug Reactions with MedDRA (2004) CIOMS VI 03/2001 -10/2004 Management of Safety Information from Clinical Trials (2005) CIOMS VII

  • Development Safety Update Reports (DSUR): Harmonizing the Format and Content for Periodic Safety

Report during Clinical Trials (2006) CIOMS VIII

  • Practical Aspects of Signal Detection in Pharmacovigilance (2010)

CIOMS/WHO WG 11/2005 – 10/2010 Definition and Application of Terms for Vaccine Pharmacovigilance (2012) CIOMS IX

  • Practical Approaches to Risk Minimisation for Medicinal Products (2014)

CIOMS X 06/2011 – 07/2015 Evidence Synthesis and Meta-Analysis for Drug Safety (2016) CIOMS SMQ Implementation WG (05/2002) – 2019 Development and Rational Use of Standardised MedDRA Queries (SMQs): Retrieving Adverse Drug Reactions with MedDRA (2016) CIOMS WG to Vaccine Safety 2013 -2016 CIOMS Guide to Active Vaccine Safety Surveillance (2017) CIOMS Guide to Vaccine Safety Communication (2018) CIOMS WG on DILI 2017 – ongoing

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Pharmacovigilance: Recent Safety Publications

Shanghai, December 2019 16

https://cioms.ch/shop/product-category/recently-published/

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EMA 4 March 2020 17 Geneva, December 2019I 17

CIOMS reaches out in many languages

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E-training course

https://cioms.blendleren.nl/Navigating-the-2016-CIOMS-International-Ethical-Guidelines

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Ongoing Working Groups today

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Five WGs ongoing:

  • CIOMS Working Group on Drug Induced Liver Injury (DILI) (started 2017)
  • CIOMS Working Group on Clinical Research in Resource Limited Settings

(November 2017)

  • CIOMS Working Group on Patient Involvement in Development and Safe Use of

Medicines (April 2018) – WG XI

  • CIOMS Expert Working Group on MedDRA Labeling Groupings (MLGs) (April

2019)

  • Revision of CIOMS IV : Benefit-Risk Balance for Marketed Drugs: Evaluating

Safety Signals (September 2019) – WG XII New WGs 2020:

  • CIOMS Working Group on Real World Evidence and Real World Data in

Regulatory Decision (March 2020) – WG XIII

  • CIOMS Working Group on Severe Cutaneous Adverse Reactions (SCARS) – Q3

2020

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CIOMS Working Group on Patient Involvement in Development and Safe Use of Medicines (April 2018) – WG XI

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When CIOMS WG started a lot was already ongoing…

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Source: Marc M. Boutin , DIA San Diego June 2019

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Working Group objectives

The CIOMS Working Group XI on Patient Involvement in the Development and Safe Use of Medicines was launched in April 2018 with an ambition to cover the whole product life-cycle – from early development until retirement from the market. It includes participants from patient

  • rganizations, industry, regulators, academia and the World Medical Association. All F2F meeting

minutes are made public

Progress

1st meeting held on 19-20 April 2018, Geneva, Switzerland (minutes available) 2nd meeting held on 23–24 October 2018, Berlin, Germany (minutes available) 3rd meeting held on 1-2 May 2019, Geneva, Switzerland (minutes availble) ♦ Open meeting held on 30 April 2019, Geneva, Switzerland 4th meeting held on 16-17 October 2019, Basel, Switzerland (minutes available) 5th meeting to be held on 1-2 April 2020, Utrecht, Netherlands 6th meeting to be held in October 2020, Amsterdam, Netherlands

CIOMS Working: Patient Involvement in Development and Safe Use of Medicines

https://cioms.ch/working_groups/working-group-xi-patient-involvement/

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CIOMS Working Group on Patient Involvement in the Development and Safe Use of Medicines (since April 2018)

The CIOMS Working Group on Patient Involvement 4th Meeting in Basel, Switzerland.

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CIOMS Working Group on Patient Involvement in Development and Safe Use of Medicines

Draft content for the future guidance (1) Chapters 1.Introduction; 2.Landscape of patient involvement in the development and safe use of medicines; 3.Guiding principles and considerations for patient engagement; 4.Patient involvement in advancing treatments for their disease; 5.Patient involvement in patient product labelling; 6.Opportunities for patient involvement in additional risk minimization measures; 7.Patient participation in the generation and utilization of safety and effectiveness data; 8.Patient input in developing safety issue communications regarding medicinal products; 9.Challenges and opportunities in patient involvement in resource-limited settings; 10.Guiding principles for patient participation in therapeutic decision-making.

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CIOMS Working Group on Patient Involvement in Development and Safe Use of Medicines

Draft content for the future guidance (2a)

Chapter 6 Opportunities for Patient Involvement in Additional Risk Minimisation

Description of Risk Minimisation Purpose:

  • Optimise a product’s benefit-risk profile
  • Enhance safe use
  • Preserve patient access

Types: Regulatory Aspects of Additional Risk Minimisation Current regulatory landscape for EU, USA, and Japan Case examples of patient involvement with regulators for additional risk minimisation planning Determining the Need for Additional Risk Minimisation Description of pharma adaptation of Failure Mode and Effects Analysis (FMEA) and opportunities for including patient perspective

Routine Additional Adequate for most products Reserved for specific products / risks

  • Product Label
  • Packaging
  • Prescription Status
  • Communication / Educational

Measures

  • Controlled Product Distribution and Use
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CIOMS Working Group on Patient Involvement in Development and Safe Use of Medicines

Draft content for the future guidance (2b)

Chapter 6. Opportunities for Patient Involvement in Additional Risk Minimisation

Patient Involvement in the Design, Implementation and Effectiveness of Additional Risk Minimisation Measures (aRMMs)

Additional Risk Minimisation Process Steps

Patient input can inform design of aRMMs to promote behaviors /actions to minimize risk. Patients’ ideas or feedback can

  • ptimise tool

prototypes and enhance health literacy of content. Patient input can help customise implementation on country level and lessen burden on healthcare setting. Patients can provide input on design and conduct of aRMMs effectiveness evaluations and participate in evaluation activities.

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CIOMS Working Group on Patient Involvement in Development and Safe Use of Medicines

Draft content for the future guidance (3a)

Chapter 7. Patient participation in the generation and utilization of safety and

effectiveness data Overview

Sources of data, entities with which patients interact, and the existing rules of engagement are

  • reviewed. Followed by critical appraisal of gaps, needs, and opportunities. Specific challenges and

promising future directions are highlighted, followed by a set of recommendations.

Current environment

Sources of data

  • Adverse Event Spontaneous reporting
  • Real-World Evidence: Post-authorisation safety studies (PASS), Health Economics

and Outcomes Research (HEOR) Studies, Patient Surveys, Risk Management Registries

  • Expanded access programs and compassionate use programs
  • Qualitative Studies (Structured Interviews and Focus Groups)
  • Industry Medical Information Systems
  • Internet Posts/Social media
  • Personal sensors and wearable devices (new forms of data)
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CIOMS Working Group on Patient Involvement in Development and Safe Use of Medicines

Draft content for the future guidance (3b)

Chapter 7. Patient participation in the generation and utilization of safety and effectiveness data

… … Data linkage Sharing of data and rules of engagement

  • Patients–regulatory authorities, –industry, –healthcare professionals, –patient
  • rganizations, –academics.

The patient’s perspective Gaps, needs, and untapped opportunities

Mobilizing patients, caregivers, and patient families as advocates for open access to patient benefit- risk data. Increasing patient engagement in regards to providing their data.

Challenges

Legal requirements, Global Environment on Data Privacy, large volume data, societal attitudes towards industry and regulators/government partnering with patients

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CIOMS Working Group on Patient Involvement in Development and Safe Use of Medicines

Draft content for the future guidance (4)

Chapter 8. Patient input in developing safety issue communications regarding medicinal

products Scope - Definition of An Urgent Communication Patient involvement Content Type of issues

Crisis Communication in Clinical Trials Crisis Communications for Marketed Products Crisis Communications for Generics

Type of distribution Type of communication

Clarification Additional communication Educational Contact for information Links for communication Patient organisations to assist in communication Vulnerable patients

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CIOMS Working Group on Patient Involvement in Development and Safe Use of Medicines

Draft content for the future guidance (5)

Gaps / additional topics identified

 Resource-Limited Settings challenges and opportunities

  • CHAIN Uganda workshop
  • Resource-Limited Settings subgroup: India, Iraq, Cameroon, Ghana, Malawi,

Argentina, and Brazil  Therapeutic decision-making contributions  Bioethicists subgroup  Case studies worldwide to broaden scope  Considerations for special populations such as those with rare diseases, disabilities, and multiple co-morbidities, as well as various pediatric patients  Patient involvement in benefit-risk considerations

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CIOMS Working Group on Patient Involvement in Development and Safe Use of Medicines

Draft content for the future guidance (6) Considerations for communication and distribution Glossary – Most pertinent terms from the stakeholder perspectives Easy to read – Content structure, flow, navigation, infographic, high-level recommendations Implementation strategy – Reach target readership Differentiators of CIOMS’ approach Comprehensively combines all existing initiatives; Capitalises on the outstanding work completed to date. Feedback from the field Real sense of excitement from for example:

CIOMS Open Meeting on Patient Involvement in Development and Safe Use of Medicines DIA 2019 Global Annual Meeting; DIA 2019 Annual Canadian Meeting; European Medicines Agency (EMA) Patients’ and Consumers’ Working Party (PCWP) 22 European organisations representing patients DIA 2020 Euro meeting … public consultation (early 2021?) before finalisation

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Conclusion

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Conclusion

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A lot of progress has been made in learning how the best to benefit from patient involvement in the process of drug development and safe use However, working for public health has a problem:

No matter r how good d you are you can alw lways ays do better ter!