An agency of the European Union

Interaction with patients and consumers

Nathalie Bere, Patients and Healthcare professionals Dept, Stakeholders and Communication Division

Overview of involvement in EMA activities during 2015

1

2000 Patients join COMP as full members

2003 Working group with patients created

2005 Framework of interaction with patient and consumer organisations

2006 Patients and Consumers Working Party (PCWP)created

2014 • Dedicated Patients and Healthcare Professionals Department created

• Revised Framework

Interaction with patients: the EMA journey… so far

Ongoing… Systematic inclusion of real life experience in EMA regulatory outputs

1996 Dialogue with HIV patients 1995

EMA created

2

Patient and consumer involvement over the years..

76

167 213

307

423

525 551

633

742

0

100

200

300

400

500

600

700

800

2007 2008 2009 2010 2011 2012 2013 2014 2015

3

CHMP CAT

PRAC COMP

CHMP- SAWP

CHMP PRAC

Orphan Designation

Scientific Advice Protocol assistance

Paediatric Investigation

Plan

Post Marketing Authorisation

Marketing Authorisation Application Evaluation

COMP PDCO

SAG SAG

Patient input

Patient input

Patient input

Public Summaries of Opinion

Patient input

Package Leaflets (PL) EPAR summaries

Safety Communications

Patient input

Patient input

Patient input

Patient input

Patient input

Package Leaflets (PL) (renewal)

Patient input

Patient input

Opportunities for involvement along the medicine lifecycle at EMA

Categories of patient participation:

• Management Board

• EMA Scientific Committee(s)

Patients representing patients’ organisations

• Patients’ and Consumers’ Working Party (PCWP)

• EMA consultations

• Workshops

Patients representing their organisations

• Scientific Advice / Protocol Assistance Procedures • Scientific Advisory / ad hoc expert Groups • Scientific committee consultations • Review of documents

Patients as individual experts

5

26 33 35 46 47 44 55 55 53

22

87 108

175 200

240

333 336 331

28 47

70 86

176

241

163

242

358

0

50

100

150

200

250

300

350

400

2007 2008 2009 2010 2011 2012 2013 2014 2015

Comparison of involvement as committee/WP members, experts and representatives of organisations

2007–2015

MB/ Committee/WP members Individual experts Representatives of their organisations

6

3 3 3 2 3

13 14 8 11

5 13

22

7

27 18 19

36

13

24

44

28 33

10

39

87

37 35

25

44

104

76

23 24 19

115

0102030405060708090

100110120

SA/PA procedures SAG/ad hoc expertmeetings

Committeeconsultations

Safety communications Workshops

2009 2010 2011 2012 2013 2014 2015

Comparison of involvement across different activities

The revised framework of interaction ; Action Plan

• Maintain the network of patients and consumers organisations • Establish a pool of experts:

– Continue to identify patients/consumers through network – Identify gaps in expertise – Publish a call for expression of interests

• Promote participation at key milestones during lifecycle of medicines:

– Ensure early involvement in development of medicines – Patients involvement in benefit/risk evaluation at CHMP; develop

processes to capture patients’ input

• Build capacity: – Explore means to increase awareness on medicines evaluation – Gather feedback and streamline provision of EMA training – Conduct a reflection on providing further support to enable patient involvement

PCWP

• Monitor and increase transparency on involvement of patients, consumers and organisations in EMA:

– Establish system for regular collection of quantitative and qualitative data for monitoring and reporting purposes

– Explore methodologies to measure the impact of patients’ involvement on regulatory outcomes – Acknowledge and promote visibility of patient/consumer organisations input provided in Agency

activities

Eligible organisations: patients/consumers

Patients representing patients’ organisations

Members of :

• EMA Management Board (MB)

• Committee for Orphan medicines (COMP)

• Committee for paediatric medicines (PDCO)

• Committee for advanced therapies (CAT)

• Pharmacovigilance Risk Assessment Committee (PRAC)

9

Patients representing their organisations

EMA Working Party with Patients & Consumers Organisations (PCWP)

• 19 members and 16 alternates representing PCOs;

• 6 members from the EMA Scientific Committees;

• 1 member from the EMA secretariat;

• Observers from the CMD-h, HCPWP and MB.

10

Five PCWP meetings; • one plenary meeting, • two joint meetings with the HCPWP

(including a dedicated session on “Biosimilars” and a workshop on “Risk minimisation tools”)

• one with all eligible organisations • one-day training session

11

Patients also contribute to EU-wide initiatives where EMA is involved such as:

• Enpr-EMA - European Network of Paediatric Research at the European Medicines Agency

• ENCePP - European Network of Centres for Pharmacoepidemiology and Pharmacovigilance

• WEB-RADR - Recognising Adverse Drug Reactions

Patients representing their organisations (Cont.)

Examples during 2015: • Committee/EMA consultations

• Pharmacovigilance legislation forum

• Patient registries

• EMA policy on proactive publication of and access to clinical-trial data

• Pandemic preparedness

• WEB-RADR stakeholders survey

• Ad-hoc observers attending PCWP meetings

• Working groups

• Workshops

• Workshop on biosimilars

• Science, Medicines, Health: Patients at the heart of future innovation conference

• Duchene (DMD) workshop

• Workshop on chordoma as a model for very rare cancers

• Webinar: Implementation of EMA policy on publication of clinical data

• EMA workshop on the development of new medicines for the treatment of ulcerative colitis and Crohn’s disease

• Workshop on haemophilia registries

• Implement. policy on access to clinical data

• Follow-up stakeholder meeting on the implementation of EMA policy on publication of clinical data

• 9th Stakeholder Platform meeting

• Joint EFGCP-DIA-EMA paediatric conference

• Lunchtime talk and debate: 'Involving young people in the evaluation of medicines for children‘

• EMA workshop on shortages

• Anticoagulants workshop

• Workshop to clarify concept/demonstrating of significant benefit of orphan medicines

• EMA 20th anniversary event: The view from the sharp end: what patients and healthcare professionals can do for us

• IMI ADVANCE project

12

Involvement in EMA workshops/conferences

Medicines’ development:

• Participation in scientific advice/protocol assistance procedures

Benefit/risk evaluations:

• Participation in scientific advisory / ad-hoc expert group meetings (SAGs) convened by CHMP or PRAC

• Respond to ad-hoc consultations on assessment of medicines from all Committees

• Review information on medicines: Package leaflets, EPAR summaries, safety communications (Q&As) and soon herbal summaries

Patients as individual experts

14

32 39

46

18

46 35

48 60 57

11

43

75 62

79

102 110

89

108

0

20

40

60

80

100

120

2007 2008 2009 2010 2011 2012 2013 2014 2015

Package leaflets and EPAR summaries sent for review

EPAR Summaries Package leaflets

84% 82% 78% 78% 83%

100%

81% 80% 82% 82% 91%

44%

66%

89% 89% 86% 90%

66%

0%10%20%30%40%50%60%70%80%90%

100%

2007 2008 2009 2010 2011 2012 2013 2014 2015

Percentage of package leaflets and EPAR summaries reviewed

EPAR Summaries Package leaflets

Feedback on comments received; EPAR summaries

15

62% of EPARs adjusted due to feedback by patients

47

33

05

101520253035404550

Total amount EPARschecked

Total amount EPARschanged by patient

feedback

Ongoing initiatives

• Pilot project to involve patients in CHMP plenary discussions

3 cases so far; ongoing

• Elicitation of B/R preferences

Pilot study with melanoma / myeloma patients

Expanded study 2016

• Enhanced training materials

‘EMA basics’ short videos

Info-sheets

Improvements to webpages dedicated to patients

• Topic groups

Measure impact/value of patient involvement

Acknowledge and promote visibility of patient input in the Agency’s activities

Training and support to patients

Social media

Involvement of young people

16

Continued to raise awareness

Participation in conferences

17

• EATG stakeholders meeting, Brussels • Giving patients a voice - Federal Joint Committee,

Berlin • PROTECT Final Symposium, EMA • Eurordis rare disease day media event • LSE conference - Pharmaceutical Policy: Pricing,

access and reimbursement, London • EFPIA 50 year anniversary of EU pharmaceutical

legislation, Brussels • Newcastle University – Workshop: Participants not

subjects – engaging patients and families in paediatric clinical research

• DIA Euromeeting-Paris • Melanoma (MPNE) 2015 conference - Brussels • Myeloma Patients Europe annual meeting -

Dubrovnik

• University of Copenhagen: Patient Involvement in medicines development and approvals

• Eurordis summer school - Barcelona • EUPATI Advisory Board meeting - Berlin • CIRS patient Engagement workshop - UK • EUPATI training course for patients - Barcelona • WONCA conference - Istanbul • ISPOR roundtable - Milan • FT global pharmaceutical and Biotechnology

conference - London • EUnetHTA-EMA meeting - Copenhagen • Invitation to participate and advise on workshop

on stem cell therapies and gene therapies (Genetic Alliance UK/Wellcome Trust - Medical Research Council Cambridge Stem Cell Institute)

• Joint EMA-EBE (European Biopharmaceutical Enterprises) seminar

Conclusion

• The involvement of PCOs continues to be extremely beneficial;

• Patients are a recognised and integral part of the Agency’s work with opportunities for input along the lifecycle of the medicines development

• With the passing years, their involvement not only expands, but evolves to ensure it occurs in the most optimal manner possible

• We will continue to look to enhance and improve involvement wherever feasible

• We look forward to a continued mutually beneficial collaboration during 2016!

18