Patient Perspective on involvement in regulatory decision making and ...

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Jun 11, 2015 - Joint BIA MHRA Conference. 11th June ... Outreach to over 1800 patient groups .... EURORDIS Call: Release
Patient Perspective on involvement in regulatory decision making and Drug Development

Lesley Greene Vice Chair COMP/ Patient Advocate EURORDIS Pathway to Innovation from Research to Patients. Joint BIA MHRA Conference 11th June 2015

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EURORDIS   

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a collective Patient Voice for the Rare Disease Community

Founded in 1997 667 member patient organisations 61 countries (26 EU countries) 36 National Alliances of RD Patients Organisations 49 European Federations of specific rare diseases Outreach to over 1800 patient groups 30 staff, offices in Paris, Brussels, London, Barcelona, Geneva Over 320 Volunteers: +75 patient advocates and +250 moderators 4 Million € Budget

OBJECTIVE :To achieve the quickest access to as many safe, efficient and affordable medicines with a real therapeutic added value, for all rare disease patients in the European Union.

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Patients in EMA Committees: Impact and Added Value  Bringing a unique and critical “patient perspective”, based on real-life experience of the disease, current therapeutic environment and/or identifying patients with experience of the disease when necessary, on behalf of those directly affected by regulatory decisions  Contributing to patient information and communication related to medicines to ensure stakeholders can access useful and understandable information  Increasing transparency in the regulatory process ; building confidence  Disseminating committees’ outcomes when public to patients and patients’ organisations.  Advising and supporting regulators in dialogue with industry and other stakeholders when identifying areas of medical need for target research.  BUT take care not to lose touch-too educated/ no longer a “typical/naive” patient perspective “If we really want to involve desperate patients in discussion meetings, and if we have to crash all their hope in the process, maybe we should have some sort of a support system in place?” Clinician COMP member viewpoint

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Empowerment and Training Patient Representatives

EURORDIS Summer School (2008) Clinical Trials & Medicines Development EU Regulatory Processes & EMA HTA, Reimbursement, Patient Access Translational & International Research 227 patient advocates trained from 34 countries, over 70 diseases Alumni involved in regulatory processes at the EMA and/or in collaboration with sponsors and/or as EURORDIS’ Volunteers

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Empowerment and Training Patient Representatives

European Patients’ Academy on Therapeutic Innovation project (EUPATI) • provides scientifically reliable, objective, comprehensive information to patients on the research and development process of medicines.

• increases the capacity of patients and the lay public to be effective advocates and advisors in clinical trials, with regulatory authorities and in ethics committees. • EURORDIS is a partner in the EUPATI project

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IMPACT: Involvement in EMA Committees COMP: Committee for Orphan Medicinal Products (2000) 2 EURORDIS representatives (Vice-Chair +1 full member) + 1 Observer PDCO: Paediatric Committee (2006) 2 EURORDIS representatives (full member & alternate)

CAT: Committee for Advanced Therapies (2007) 2 EURORDIS representatives (full member & one expert) PRAC: Pharmacovigilance and Risk Assessment Committee (2012) Scientific Advice & Protocol Assistance (since 2005/2013) CHMP: Committee for Human Medicinal Products (new pilot since September 2014)



PCWP: Patients' and Consumers' Working Party

2 EURORDIS representatives (full members) “In essence I think the decision making is enriched and better by having patient reps involved” Regulator viewpoint eurordis.org

Package Leaflets (PL) EPAR summaries

Committee s and Working Parties

Patient input

Public Summaries of Opinion (PSO) Patient

Patient input

input

Regulatory Procedure

Documents for the Public

IMPACT: Where the EU Regulatory Process involves Patient representatives

Orphan Designation

CHMPSAWP

COMP Patient input

Paediatric Investigation Plan

Scientific Advice Protocol assistance

Risk Management Plan summaries (tentative)

Marketing Authorisation Application Evaluation

PDCO

Patient input

Patient input

Patient input

CAT

Molecule Identification

Preclinical tests Patient input

Patient input

Patient input

Post Marketing Authorisation

CHMP PRAC Patient input

SAG Patient input

Clinical Development Phase IV Phase I Patient input

Phase II Patient input

Safety Communications Patient input

SAG

Patient input

Drug Discovery

CHMP CAT PRAC

Package Leaflets (PL) (renewal)

Phase III Patient input

EXAMPLE: COMP responsibilities - designation criteria

Orphan Regulation: Designation criteria • Rarity (prevalence) / Return of Investment (Art 3.1 (a) of 141/2000) • Medical condition affecting not more than 5 in 10,000 in the Community (around 250,000 people) • Without incentives it is unlikely that the marketing of the product would generate sufficient return to justify the necessary investment • Seriousness Life –threatening or chronically debilitating • Alternative methods authorised (Art 3.1(b)of 141/2000) • If satisfactory methods exist the sponsor should establish that the product will be of significant benefit (exclusive to EU)

COMP responsibilities • Provide opinions on suitability of granting orphan designation status • Advise Commission on establishment and development of a policy on Orphan Medicinal products (OMPs) • Assist Commission on international liaison and drawing up relevant guidelines • Contribute to Protocol Assistance (especially Significant Benefit) • identifying experts, both patients and scientists

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IMPACT: Patients at the Heart of Drug Development Examples of projects currently in progress initiated by a patient group • ENRAH-SME-Laying the ground for breakthrough in Alternating Hemiplegia in Childhood (AHC)

• AKUre: Assessing nitisinone as potential orphan treatment for Alkaptonuria

• AAVrh10.hSGSH:Adenovirus Associated Viral vector serotype 10 carrying the human Nsulfoglycosamine sulphohydrolase San Filippo A

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IMPACT : Clinical Trial Design and PROs •

Making clinical development “patient-relevant”: design, endpoints, measurement of endpoints, informed consent, selection of centres, recruitment, interpretation of data, dissemination of results



Ensuring that new medicines respond to patients’ needs

“ A patient representative is helpful judging the burden of side effects e.g nausea is considered more of a burden than vomiting.” (Clinical researcher viewpoint) • Emphasising Patients Relevant Outcomes and Patients Reported Outcomes • How much harm or risk are patients willing to accept for a given benefit? • Providing evidence based opinion, quantitative as well as qualitative, in an independent manner Health Related Quality of Life • “the effect of an illness and its therapy upon a patient’s physical, psychological and social wellbeing, as perceived by the patients themselves.” • In clinical research, quality of life measures can provide a means of capturing the personal and social context of the disease experience…and effect on activities of daily living •

Preferred treatment options e.g inhalation devices, infusions, injections etc

“The practicalities of daily life seem more important to patients which is something regulators hardly ever take into account”(Regulator’s viewpoint)

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CONCERNS: Patient involvement in benefit/risk at CHMP: First case from pilot study 2014 “by involving patients representatives it has turned out that they had very different views of what is important. ..My experience is also that patient representatives have been willing to take greater risks than the regulators would like them to do” Regulators viewpoint •

One case in 8 months ‒ Main feedback from Task Force: Pressure on CHMP: no freedom of discussion when patients present ‒ No need for personal story; interest of getting personal experience ‒ Are patients independent? Patients not previously treated by the product should also be invited ‒ Need for induction of CHMP members



Main feedback from patients: ‒ Overall positive ‒ Clarify confidentiality aspects and role of the “mentor” ‒ Not clear when patients have to leave the room

Patient Representative conclusion “A more structured approach for involvement in B/R is needed that embeds the value patient perspective in the evaluation process for MA “ eurordis.org

CONCERNS : Patient Involvement Establishing Evidence in Clinical Trials with Small Populations Patient/carer viewpoint: the need for a greater flexibility in the methodology of clinical trials for these patient populations.. a greater acceptance of the role which uncertainty plays, allowing patients …. to access new therapies faster •

a special approach such as exceptional circumstances or conditional licencing?



Need to facilitate the collaboration and interaction with patient organisations



Help to identify bottlenecks and status-quo of methodological issues for small population research



Involve in proposing new & improved statistical design methodologies

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Where Patient advocates currently contribute to research and clinical trials in small populations •

EU funded international, multidisciplinary research consortia aiming at the efficient assessment of the safety and/or efficacy of a treatment for small population groups.



ASTERIX (Advances in Small Trials dEsign for Regulatory Innovation and eXcellence):



IDEAL (Integrated Design and AnaLysis of small population group trials)



InSPiRe (Innovative methodology for small population research): FP7 HEALTH 2013 – 602144

All three involve a network of clinical trial experts from all relevant stakeholders including academia, industry, regulators, and patient advocacy groups

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CONCERNS: Equable access to Medicines: 5 key concepts* • RD Treatments Evidence Generation is a Continuum

• Medicine Adaptive Pathways to Patients and Flexibility of Regulators & HTA & Payers should become an Official Policy • Focus on Effectiveness beyond Quality, Safety and Efficacy

• Bridging the Gap Between EU Centralised Regulatory Decision and National Decisions on Pricing & Reimbursement in a seamless process • Enhancing the Dialogue Between all Stakeholders all Along the Product Development & Life Cycle *EURORDIS Position Paper 2009; EU Pharma Forum on Improving Access to Orphan Medicinal Products 2009; Commission Expert Group on Rare Diseases, Recommendation on the Clinical Added Value of Orphan Medicinal Products (CAVOMP) in 2012 ; Process on Corporate Social Responsibility in the Field of Pharmaceuticals Platform on Access to Medicines in Europe, Working Group on Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA-OMP) in 2013.

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EURORDIS Call: Released May 2015 to establish a new “Table for Price Negotiations” with a core group of volunteer EU MS

Championed by Belgium (INAMI) together with the Netherlands, Austria. 

Value-based pricing discussion linked to the volume of patients and to postmarketing evidence generation



Certain level of transparency on cost is expected



Price: 

a) Price will have to fluctuate over time based on additional generated evidence,



b) conditional pricing, managed entry agreements, payment on outcomes, and other innovative pricing schemes,



c) a reference for basket of price

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Added value of patient involvement in regulatory decision making and drug development IMPACT Assessment of applications; promotion of research; Involvement in clinical study design CONCERNS Need for greater involvement in risk/benefit; clinical trials in small populations; access to medicines  Essential that training/education ensures appropriate involvement  Patients are often the instigators, drivers and funders of new research  Empowered and trained patients who have advocated for therapy for rare diseases for decades, bringing passion and real life experience to scientific committees The contribution of the patient is one that stimulates and challenges but also enriches and instructs the process all along the regulatory and development pathways eurordis.org

Thank You for listening!

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