Final CHMP Workplan 2017 for publication - European Medicines ...

18 April 2017 EMA/CHMP/153123/2017 Inspections, Human Medicines Pharmacovigilance and Committees Division

CHMP work plan 2017

Adopted by the Committee on 23 February 2017

Table of Content

1. Evaluation activities for human medicines .............................................. 2 1.1. Pre-authorisation activities .............................................................................. 2 1.1.1. Multi-stakeholder consultations to facilitate optimisation of clinical evidence generation in drug development programmes ........................................................ 2 1.1.2. Accelerated Assessment ............................................................................... 3 1.2. Initial-evaluation activities .............................................................................. 4 1.2.1. Benefit/Risk methodology ........................................................................... 4 1.2.2. Patients involvement in assessment work .................................................... 5 1.2.3. Concepts of significant benefit ..................................................................... 6 1.2.4. Documenting medicines evaluation............................................................... 7 1.2.5. PRIME (Enhanced early dialogue to foster development and facilitate accelerated assessment) ........................................................................................ 8 1.2.6. Liaison with non-EU regulators ..................................................................... 9 1.3. Pharmacovigilance activities .......................................................................... 10 1.3.1. Registries – PRAC/CHMP Cross Committee liaison ...................................... 10 1.4. Other specialised areas and activities ............................................................ 11 1.4.1. Extrapolation .............................................................................................. 11 1.4.2. Quality assurance of SmPCs ........................................................................ 12 1.4.3. Geriatric medicines strategy ....................................................................... 13 1.4.4. The interface of precision medicines and diagnostics ................................. 14

2. Horizontal activities and other areas ..................................................... 15 2.1. Working parties ............................................................................................. 15 2.1.1. Monitoring of Working Parties (self-standing/temporary) and drafting groups taking into consideration recommendations from their review ................. 15 2.2. Collaboration with PDCO to address the needs of the paediatric population .. 16 2.3. CHMP preparedness and capacity building ..................................................... 17

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1. Evaluation activities for human medicines 1.1. Pre-authorisation activities 1.1.1. Multi-stakeholder consultations to facilitate optimisation of clinical evidence generation in drug development programmes Activity areas Clinical evidence generated during drug development generally serves decision making by different actors. Whilst scientific advice on evidence requirements for regulatory purpose is well established, in recent years the opportunities for engagement with additional stakeholders during such discussions have been increasingly recognised. Parallel regulatory/HTA advice has been developed as option to drug developers wishing to construct a drug development programme that is able to address the different needs of regulators and health technology bodies in the most efficient manner possible. Using the experience from this platform, engagement with other stakeholders involved in down-stream decision making should be considered in case beneficial for specific types of medicines developments. This includes payer organisations as well as so-called “switch bodies”. CHMP topic leader: Robert Hemmings

Key objectives •

To monitor the utility of the existing platform for parallel regulatory / HTA discussions to drug developers in terms of providing relevant and robust guidance on drug development.

•

To identify opportunities and needs for engagement with other decision makers in multistakeholder consultations.

•

To engage in other relevant programmes, particularly EUnetHTA Joint Action 3.

•

To provide a framework for discussion of proposals for developments using the adaptive pathways concept and contribute through experience to conceptual discussions (particularly IMI ADAPT SMART)

Activities in 2017 CHMP activities (through SAWP) to achieve the objectives set for this area: •

To collaborate with EUnetHTA in the further development of frameworks for parallel regulatory/HTA scientific advice / early dialogue

•

To monitor MAA submissions and compliance within a clinical trial, which has received parallel SAWP/HTA scientific advice

•

Explore the concept of “late dialogues” for post-licensing evidence generation plans

•

Establish the modalities to provide multi-stakeholder consultation on OTC switch applications involving “switch bodies”

•

Subject to proposals for developments using the adaptive pathways concept, facilitate engagement with other decision makers (HTAs/payers) as part of the consultation.

Other contributors:

CHMP work plan 2017 EMA/CHMP/153123/2017

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Member/alternate

Name

MS

Member

Andrea Laslop

AT

Chair

Tomas Salmonson

Member

Daniela Melchiorri

IT

Member Member

Bruno Sepodes Johann Lodewijk Hillege

PT NL

Member Member

Katarina Vučić Karsten Bruins Slot

HR NO

1.1.2. Accelerated Assessment Activity areas Ensuring a robust and continuously enhanced framework for accelerated assessment tool to facilitate early access for the benefit of patients. CHMP topic leader: Tomas Salmonson

Key objectives •

To ensure consistency and robustness of evaluations of reviews for accelerated assessment.

•

To implement the optimised procedural framework for accelerated assessment.

•

To monitor the use of the regulatory tool “Accelerated Assessment” in terms of the assessment approach to such requests as well as the actual use in MAA reviews.

•

To contribute to the activities regarding the scheme to facilitate development and accelerated assessment of innovative medicines of major public health interest and in particular from the viewpoint of therapeutic innovation to address unmet needs.

Activities in 2017 CHMP activities to achieve the objectives set for this area: •

Monitoring of the scientific evaluation under the new timetable, including cross-committee evaluations (e.g. RMPs).

•

Monitoring of reasons for changing from accelerated to standard review timetables.

•

Following up use of PRIME and monitoring the success rate of accelerated assessment requests.

Other contributors: Member/alternate

Name

MS

Member Member

Karsten Bruins Slot Concepcion Prieto Yerro

NO ES


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1.2. Initial-evaluation activities 1.2.1. Benefit/Risk methodology

1

Activity areas Benefit/risk of a medicine requires continuous evaluation throughout the life-cycle. The objective is to balance benefits and risks in a way that is as robust, consistent and transparent as possible. CHMP topic leader: Johann Lodewijk Hillege

Key objectives • •

Continued overview of developments in assessing and communicating benefit/risk. Continue to work on EPARs to improve the structure and information on benefit/risk, including the effects table and implementation of new templates/guidance.


Monitoring implementation of benefit-risk template structure and guidance;

•

Further explore application of benefit-risk methodologies for decision making and communication (eg output of IMI-PROTECT), including visual displays and value elicitation, in co-ordination with relevant EMA scientific committees (linked to Patients involvement in assessment work topic).


Name

MS

Alternate

Aranzazu Sancho-Lopez

ES

Chair

Tomas Salmonson

Co-opted member

Jan Mueller-Berghaus

Vice-Chair

Harald Enzmann

DE

Member

Katarina Vučić

HR

Member

Sinan B. Sarac

DK

Member

Kristina Dunder

SE

1

Linked to Documenting medicines evaluation topic


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1.2.2. Patients involvement in assessment work Activity areas The objective is to facilitate participation of patients and consumers in benefit/risk evaluation and related activities, to capture patient’s values and preferences and obtain information on the current use of medicines and their therapeutic environment, all along the lifecycle of the medicines, from early development throughout evaluation and post-marketing surveillance. CHMP topic leader: Harald Enzmann

Key objectives •

Incorporate additional and regular processes to capture and include patient views and preferences within CHMP benefit / risk evaluations.


Analysis of pilot on patients involvement at CHMP oral explanations (6 cases of involvement during pilot). Discussions should be held and agreed on way forward; i.e. continuation of regular involvement of patients in oral explanations, on a case-by-case basis, as needed;

•

Explore other methodologies for gathering patient input (e.g. written consultations, elicitation of patient preferences, focus groups), collaboration with IMI PREFER project (linked to Benefit/Risk methodology topic).

•

Explore how to report patient input and values in the assessment report.


Name

MS

Chair

Tomas Salmonson

SE

Alternate

Patrick Salmon

IE

Member Member Member

Concepcion Prieto Yerro Bruno Sepodes Sinan B. Sarac

ES PT DK

Member

Katarina Vučić

HR


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1.2.3. Concepts of significant benefit

2

Activity areas The concept of significant benefit is reflected in different pieces of the Pharmaceutical legislation. This relates to the concept of ‘significant benefit’ and ‘clinical superiority’ in the orphan drug regulation, ‘significant clinical benefit’ for obtaining 1 additional year of market protection, ‘significant therapeutic benefit’ in the paediatric legislation and ‘major therapeutic advantage’ for conditional marketing authorisation. Understanding the concepts and purposes of the various provisions and the scientific standards to be applied for each piece of legislation will help understanding how these should be applied and to apply criteria consistently for each legislative provision. CHMP topic leader: Jan Mueller-Berghaus

Key objectives •

Understanding the different concepts and purposes of ‘significant benefit’ provisions.

•

Review how assessment of “significant benefit” is applied across different legislative provisions.


To develop regulatory analysis on the various provisions on significant benefit in the context of +1 year, paediatrics and orphans. Other contributors:

Member/alternate

Name

CA

Co-opted member Co-opted member Member

Robert Hemmings Koenraad Norga Daniela Melchiorri

IT

Member

Outi Maki-Ikola

FI

Member

Bruno Sepodes

PT

Vice-Chair

Harald Enzmann

DE

2

Cross Committee topic with COMP and PDCO involvement


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1.2.4. Documenting medicines evaluation 3 Activity areas Review and optimisation of assessment report preparation process. CHMP topic leader: Kristina Dunder

Key objectives •

Review ways to improve the robustness and soundness of outputs throughout the initial MAA evaluation process.


Optimise process flow for generation of output documents during MAA review including identification of contributors and check points;

•

Optimise the related assessment report templates to best support the process flow;

•

Monitoring the activities of Classification of Post-Authorisation Studies (CPAS) for the definition of conditions;

•

Preparation of training materials, where required.


Name

MS

Chair Alternate Member Alternate Member

Tomas Salmonson Martina Weise Greg Markey Aranzazu Sancho-Lopez Daniela Melchiorri

SE DE UK ES IT

Co-opted member Member Member

Jan Mueller-Berghaus Outi Maki-Ikola Johann Lodewijk Hillege

FI NL

3

Linked to Benefit/Risk methodology


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1.2.5. PRIME (Enhanced early dialogue to foster development and facilitate accelerated assessment) Activity areas PRIME is a scheme that has been developed to reinforce early dialogue and regulatory support to stimulate innovation, optimise development and enable accelerated assessment of PRIority MEdicines. Further to public consultation and adoption of a reflection paper by the CHMP, the scheme was launched in March 2016. The scheme remains closely monitored in the beginning of its implementation and entry into force. CHMP topic leader: Tomas Salmonson Key objectives: •

Implementation of the scheme.

•

Consider whether modifications or enhancements to the scheme should be proposed

Activities in 2017 •

Oversight of scheme performance and reporting of activities.

•

Support for preparation of stakeholder meeting to review 1 year of the PRIME scheme (May).


Name

Co-opted Member

Rob Hemmings

Member

Bart Van der Schueren

BE

Member

Alar Irs

EE

Co-opted Member

Jan Mueller-Berghaus

Member

Bruno Sepodes

PT

Member

Andrea Laslop

AT


MS

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1.2.6. Liaison with non-EU regulators Activity areas Confidentiality arrangements, reliance on CHMP assessments. Article 58 was introduced in the 2004 revision of the Agency’s founding regulation, and allows the Agency to give scientific opinions – in collaboration with the World Health Organization – on medicines for use outside of the European Union. The intention of the process is to increase access by low- and middle-income countries (LMICs) to medicines and improve public health. CHMP topic leader: Tomas Salmonson

Key objectives •

Ensure best use of resources through promoting mutual reliance and work-sharing.

Activities in 2017 •

Explore mechanisms to enhance involvement of and strengthen liaison with non-EU regulators in EMA scientific reviews, in order to facilitate work-sharing and promote capacity building.

•

CHMP involvement in action plan to achieve objectives, improve perception and use of the Article 58 procedure.

•

Exploring different ways to interact with other regulators (meeting related to art 58 taking place in Malta). Other contributors:

Member/alternate

Name

MS

Vice-Chair Co-opted member

Harald Enzmann Sol Ruiz

DE

Member

Fátima Ventura

PT


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1.3. Pharmacovigilance activities 1.3.1. Registries – PRAC/CHMP Cross Committee liaison 4 Activity areas Maximising the potential for use/best practice in relation to drug and/or disease registries for collecting and analysing safety and efficacy data. The project should consider current and potential future use of registries, for example in PAES and adaptive licensing. CHMP topic leader: Milena Stain

Key objectives •

Co-ordinate with any existing EMA initiatives relating to registries; cross-committee collaboration (including PRAC) will be important.

•

Understand the pros and cons of using patient registries in different settings and regulatory questions in Europe.

•

Test elements of the proposed approach for registries through a Pilot phase.

•

Provide a framework for optimising regulatory requests for registries, so that requestare feasible, and study designs are fit for purpose (i.e. capable of answering a specific safety/efficacy question).


Prepare a report on the patient registries workshop held;

•

Engagement with SAWP on the need of specific registries on product level;

•

To identify or further develop guideline/guidance on best use of registries (taking into account existing guidance, engagement with patient registries);

•

Ongoing work with the Pilot phase, (4 candidates selected, 17 pending consideration), evaluation of relevant registries and desired characteristics of a modified/new registry as applicable.


Name

MS

Co-opted member

Robert Hemmings

UK

Member Member Member Chair of the registries task force

Kristina Dunder Daniela Melchiorri Johann Lodewijk Hillege Peter Mol

SE IT NL NL

4

Cross-Committee topic with PRAC involvement


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1.4. Other specialised areas and activities 1.4.1. Extrapolation Activity areas The extrapolation regulatory framework is important for informed and efficient drug development and hence relevant to CHMP/SAWP and PDCO. Use of structured approaches to evidence synthesis and inference will increase in importance for decision making at CHMP. CHMP topic leader: Robert Hemmings Key objectives •

To ensure that ‘best practices’ are captured in existing or developing CHMP guidance documents, specifically CHMP decision making for which extrapolation approaches are commonly used or are seen as being particularly useful.

•

To ensure that the PDCO / CHMP paper on extrapolation between population subsets describes a framework and a set of standards that can be used by CHMP at the time of marketing authorisation.

Activities in 2017 CHMP activities to achieve the objectives set for this area. •

Setting a framework across the Committees and WPs with defined criteria for referrals of extrapolation cases and further setting up a repository.

•

To update extrapolation reflection paper based on the workshop held in 2016 and following the 2017 public consultation.

•

To raise health technology assessment awareness in the extrapolation framework through the EUnetHTA platform.

•

To provide trainings to the network (via EuNTC)


Name

MS

Chair Vice-Chair Member Member Co-opted member

Tomas Salmonson Harald Enzmann Nela Vilceanu Romaldas Mačiulaitis Jan Mueller-Berghaus

SE DE RO LT

Member Co-opted member

Kristina Dunder Koenraad Norga

SE


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1.4.2. Quality assurance of SmPCs Activity areas Quality assurance of summary of product characteristics. CHMP topic leader: Patrick Salmon, Kristina Dunder

Key objectives •

Compliance of SmPCs with the SmPC guideline in accordance with the needs of healthcare professionals, and taking into account the needs of health technology assessment.

•

Scientific consistency and accuracy of SmPC within and across therapeutic classes.

•

Monitoring and further enhancement of labelling review early and all along evaluation procedures, toward consistency and complementarity of SmPC with assessment report, risk management plan and package leaflet.

•

To provide guidance on the wording of the therapeutic indication in section 4.1 that can be applied across therapeutic areas in order to limit discrepancies.

•

To facilitate stakeholders’ understanding of therapeutic indication when making therapeutic decision or recommendation.


Maintenance and development of guidance and other tools (training material, checklist, metrics or labelling review guide) supporting SmPC review such as those published on the EudraSmPC webpage and its public interface.

•

Generate the reports of revised processes to ensure scientific committees' labelling review based on the scientific assessment and to the development of metrics.

•

Provide support and training on any matters related to SmPC.

•

Prepare an annual report on the related activities.

•

Consideration of the recommendations of the upcoming Commission report on "Shortcomings in the SmPCs and the package leaflet".

•

Monitor the need of stakeholder involvement with regard to the product information.

•

To support a consistent approach in the process of defining therapeutic indications to ensure their clarity for stakeholders.

•

Continuation and monitoring of pilot on the wording of the therapeutic indication. Other contributors:

Member/alternate

Name

MS

Alternate Alternate Alternate Co-opted member Member Co-opted Member Member

Martina Weise Aranzazu Sancho-Lopez Fatima Ventura Koenraad Norga Daniela Melchiorri Jan Mueller-Berghaus Ágnes Gyurasics

DE ES PT


IT HU

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Member/alternate

Name

Members of the SmPC Advisory Group

representing EMA, its scientific committees, QRD Group and CMDh

MS

1.4.3. Geriatric medicines strategy Activity areas The rapid aging of the population worldwide means that over 80 are the fastest growing subpopulation group. The EMA geriatric medicines strategy aims to ensure that the benefit/risk balance of medicines is evaluated with respect to the epidemiology of the disease, and that findings are adequately reflected in the CHMP assessment. CHMP topic leader: Aranzazu Sancho-Lopez, Katarina Vučić.

Key objectives •

Investigation of strategies (e.g. registries, PAES, PASS) to acquire data in the older population, particularly with respect to collection of post-approval data in frail/comorbid/very old people.

•

Coordination of geriatric activities across CHMP/PRAC/SAWP/PCWP.


Development of geriatric GVP module (PRAC involvement).

•

Continuation of pilot on 10 products with revised geriatric AR (5 products have been finalised).

•

Adopt final Points to consider on frailty (public consultation ended).

•

Complete QWP geriatric packaging and formulation guideline.

•

Monitoring of geriatric medicines strategy.


Name

MS

Member SAWP SAWP

Bart Van der Schueren Susan Morgan Mario Miguel Rosa

BE UK PT


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1.4.4. The interface of precision medicines and diagnostics Activity areas The indication of precision medicines depends to a great extent on the result of a single crucial in vitro test. Usually, the indication (SmPC section 4.1) of a medicine is not restricted to a specific diagnostic method or product (Dx) used for the selection of eligible patients in the pivotal trials submitted in the MAA. Rather, the SmPC will describe the crucial characteristic finding the Dx is supposed to detect (e.g. a receptor or mutation). Replacing the “original” Dx used in the pivotal trials with an “alternate” Dx with different performance may result in a different patient population. The benefit risk balance for a precision medicine may change (might even turn negative) depending on the “alternate” Dx used. The importance of the Dx for the selection of eligible patients and its possible impact on the benefit risk balance should be made transparent in assessment reports or SmPC. CHMP topic leader: Harald Enzmann Key objectives: •

Develop a (consensus) CHMP position how the importance of diagnostics that are essential for the use of a medicine is reflected in SmPC and assessment reports.

Activities in 2017: CHMP activities to achieve the objectives set for this area: •

Define the scope of diagnostics or diagnostic methods to be included;

•

Explore how characteristics/features of diagnostics used in pivotal trials can be made transparent;

•

Explore how the dependence of a medicine’s benefit-risk balance from the accuracy of the crucial Dx can be described;


Name

Co-opted member

Sol Ruiz

Co-opted member Co-opted member Member

Jan Mueller-Berghaus Koenraad Norga Ondřej Slanař

CZ

Alternate

Filip Josephson

SE


MS

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2. Horizontal activities and other areas 2.1. Working parties 2.1.1. Monitoring of Working Parties (self-standing/temporary) and drafting groups taking into consideration recommendations from their review Activity areas Initiatives ongoing related to the renewal process of working parties, coherent with agency’s evolving needs. CHMP topic leader: Tomas Salmonson

Key objectives •

Review the procedures on how to best utilise Working Parties for the benefit of network.


To establish and implement consistent procedures for the CHMP to input into working parties’ work plans and mechanisms to report back to CHMP.

•

Timely production of work plans in line with Committee work plan preparation.

•

Review of Working Parties support activities to the Committee through 2017.

•

Review of the role of the Guideline Consistency Group.


Name

Co-opted member

Jean-Louis Robert

Co-opted member Co-opted member

Jan Mueller-Berghaus Sol Ruiz


MS

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2.2. Collaboration with PDCO to address the needs of the paediatric population Activity areas Ensure that the needs of the paediatric population are systematically considered in the medicinal products development and assessment of their use. CHMP topic leader: Koenraad Norga, Agnes Gyurasics Key objectives Reinforce cooperation with the PDCO with a view to supporting the continuity of the paediatric safety and efficacy assessment throughout the lifecycle of medicines. Activities in 2017 CHMP activities to achieve the objectives set for this area: •

Further define and implement pilot coordination process with PDCO, with a view to utilising the best available scientific expertise in the network and ensure that paediatric information related to the clinical trial programme are included in the medicine SmPC and labelling.

•

At the end of 2017, report on experience gained with revised PDCO-CHMP coordination process for scientific evaluation of medicines of paediatric interest, in order to assess impact and identify opportunities for improvement.


Name

MS

Vice-Chair

Harald Enzmann

DE


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2.3. CHMP preparedness and capacity building EMA and its scientific committees assess medicines through a network of over 3,700 scientific experts from across the EU from around 50 medicines regulatory authorities from the European Economic Area Member States, the European Commission and EMA. This network is what makes the EU regulatory system unique. The diversity of the experts from across Europe involved in the regulation of medicines in the EU encourages the exchange of knowledge, ideas and best practices between scientists striving for the highest standards for medicines regulation. CHMP topic leaders: Tomas Salmonson Key objectives:

•

Monitor activity level and discuss mechanisms to address changes in workload pressure and assignment of CHMP roles;

Activities in 2017:

CHMP activities to achieve the objectives set for this area: •

Monitor activity and CHMP role appointment and establish regular feedback to CHMP;

•

Explore mechanisms to improve workload distribution;

•

Discuss mechanisms to facilitate work-sharing and promote capacity building;

•

Draft report with findings and proposals for improvement.


Name

MS

Vice-Chair

Harald Enzmann

DE

Co-opted member Member

Sol Ruiz Outi Maki-Ikola

FI


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