Marketing Authorisation Routes in the EU The EU medicines regulatory - - PowerPoint PPT Presentation

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Marketing Authorisation Routes in the EU The EU medicines regulatory - - PowerPoint PPT Presentation

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Marketing Authorisation Routes in the EU The EU medicines regulatory system and the European Medicines Agency: an introduction for international regulators and non-governmental organisations 18 September 2017 Presented by Maria Nieto-Gutierrez

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An agency of the European Union

Marketing Authorisation Routes in the EU

The EU medicines regulatory system and the European Medicines Agency: an introduction for international regulators and non-governmental organisations 18 September 2017

Presented by Maria Nieto-Gutierrez Procedure Management Department

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International Regulators – Awareness Session 1

Contents

Who grants MA in the EU National procedures Mutual recognition and decentralised procedures Centralised procedures Pricing and reimbursement

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International Regulators – Awareness Session 2

Contents

Who grants MA in the EU National procedures Mutual recognition and decentralised procedures Centralised procedures Pricing and reimbursement

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3

Who grants a MA in the EU?

  • To protect public health and ensure the availability of high quality, safe and

effective medicines for European citizens, all m edicines m ust be authorised before they can be placed on the market in the EU.

  • 50 years of EU pharm aceutical legislation

 In 1965 the first EU law on medicinal products was adopted  Progressive harmonisation of requirements for the granting of marketing authorisations and post-marketing monitoring has been implemented across the entire EU

  • The data requirem ents and standards governing the authorisation of

medicines are the same in the EU.

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4 4 International Regulators – Awareness Session

Who grants a MA in the EU?

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Authorisation by a single MS Authorisation by several MSs, based on assessment by Reference MS Authorisation by European Commission, based

  • n assessment by

EMA, valid in all MSs

Who grants a MA in the EU?

International Regulators – Awareness Session

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Who grants a MA in the EU?

Depending on:

  • Type of product
  • Authorisation history in EU
  • Regulatory & marketing strategy

Different authorisation routes: one set of com m on rules

International Regulators – Awareness Session

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International Regulators – Awareness Session 7

Contents

Who grants MA in the EU National procedures Mutual recognition and decentralised procedures Centralised procedures Pricing and reimbursement

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  • The majority of medicines available in the

EU are authorised at national level:

  • Authorised before EMA creation
  • Not in the scope of the centralised

procedure

  • Each EU Member State has

its own national authorisation procedures

National procedures

International Regulators – Awareness Session

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International Regulators – Awareness Session 9

Contents

Who grants MA in the EU National procedures Mutual recognition and decentralised procedures Centralised procedures Pricing and reimbursement

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Mutual recognition procedures (MRP)

  • Principle of recognition of an

already existing national MA by

  • ne or more EU Member States

Allow s Mem ber States to rely on each other’s scientific assessm ents.

1 2 2

International Regulators – Awareness Session

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  • No existing MA in the EU

Decentralised procedure (DCP)

Sim ultaneous authorisation in m ore that one Mem ber State

1 1 1

International Regulators – Awareness Session

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International Regulators – Awareness Session 12

Contents

Who grants MA in the EU National procedures Mutual recognition and decentralised procedures Centralised procedures Pricing and reimbursement

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  • One single MA application to EMA
  • Compulsory for most innovative medicines,

including rare diseases.

  • One assessm ent procedure

(scientific committee’s opinion) based on individual assessments by Member States

  • Common decision making process (one European

Commission decision)

  • One MA valid in all EU m em ber states and EEA
  • Transparent evaluation

Centralised procedures

International Regulators – Awareness Session

1

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What is the benefit of the centralised procedure for EU citizens?

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  • Wider/ quicker access to medicines for

patients

  • Safer medicines for humans and animals
  • Better and equal information for EU citizens
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International Regulators – Awareness Session 15

Contents

Who grants MA in the EU National procedures Mutual recognition and decentralised procedures Centralised procedures Pricing and reimbursement

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Pricing and reimbursement

  • Health technology assessm ent ( HTA) bodies provide recommendations on medicines that can

be financed or reimbursed by the healthcare system in a particular EU Member State or region.

  • The assessment criteria used by HTA bodies differ between EU Member States, in accordance with

regional and national legislation.

  • The EMA has been w orking closely w ith HTA bodies since 2 0 0 8 .

EMA offers consultations in parallel with European Network for Health Technology Assessment (EUnetHTA) as of July 2017. The procedure is a single gateway for parallel consultations with EMA, EUnetHTA and HTA bodies on their evidence-generation plans.

  • A close interaction between regulators, HTA bodies and other relevant bodies is critical to enable

patients’ access to important new medicines.

International Regulators – Awareness Session

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Any questions?

Maria.NietoGutierrez@ema.europa.eu

European Medicines Agency

30 Churchill Place • Canary Wharf • London E14 5EU • United Kingdom

Telephone + 44 (0)20 3660 6000 Facsim ile + 44 (0)20 3660 5555 Send a question via our w ebsite www.ema.europa.eu/ contact

Further information

Follow us on @EMA_ New s

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An agency of the European Union

The role of EMA

The EU medicines regulatory system and the European Medicines Agency: an introduction for international regulators and non-governmental organisations 18 September 2017

Presented by Maria Nieto-Gutierrez and Helena Matos Procedure Management Department

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International Regulators – Awareness Session 1

Contents

The role of EMA Evaluation of medicines Safety monitoring of medicines Development and access to medicines Transparency

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International Regulators – Awareness Session 2

Contents

The role of EMA Evaluation of Medicines Safety Monitoring of Medicines Development and Access to Medicines Transparency

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  • Decentralised agency of the EU
  • Founded in 1 9 9 5
  • Located in London until March 2019
  • Reg. ( EC) No 7 2 6 / 2 0 0 4

[ previously Reg. (EEC) No 2309/ 93/ EC]

  • Scientific secretariat responsible for

coordinating the existing scientific resources put at its disposal by Member States for the evaluation, supervision and pharmacovigilance

  • f medicinal products*

3

* Art. 55 of Reg. (EC) No 726/ 2004

The role of EMA

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International Regulators – Awareness Session

The role of EMA

Who do we work for?

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  • Centralised procedure, via EMA;
  • National licence, Mutual recognition procedure, Decentralised procedure,

via NCAs.

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Who do we work with?

The role of EMA

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Evaluate m arketing authorisation applications

Monitor the safety

  • f m edicines

throughout their life cycle

Provide inform ation

  • n m edicines to

healthcare professionals and patients Facilitate developm ent and access to m edicines

International Regulators – Awareness Session

The role of EMA

What do we do?

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International Regulators – Awareness Session 7

Contents

The role of EMA Evaluation of medicines Safety monitoring of medicines Development and access to medicines Transparency

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  • The EMA’s scientific com m ittees provide independent recommendations on

medicines for human and veterinary use, based on a comprehensive scientific evaluation of data.

  • EU experts participate in the work of EMA as members of its scientific

committees, working parties, scientific advisory groups, or as members of the national assessments teams that evaluate medicines.

  • Increasingly, patients and healthcare professionals (HCP) are involved in

the work of the Agency including evaluation of medicines.

  • By w orking together, Member States reduce duplication, share the workload

and ensure an efficient and effective regulation of medicines across the EU.

8 International Regulators – Awareness Session

Evaluation of medicines

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EMA

Committee for Veterinary Medicinal Products CVMP Committee for Advanced Therapies CAT Committee for Human Medicinal Products CHMP Committee for Herbal HMPC Paediatric Committee PDCO Committee for Orphan Medicinal Products COMP PhV Risk Assessment Committee PRAC Industry Organisations Patients and HCP Representatives Working Parties PCW P & HCPW P

9

The EMA’s scientific committees

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Composition of scientific committees:

  • 1 member + 1 alternate nominated by each of the 28 EU MS
  • 1 member + 1 alternate from NO and IS (observers)
  • Co-opted members for specific expertise on scientific or technical matters
  • Elected Chair and Vice-Chair
  • 3 years renewable mandate

 Diversity: Exchange of knowledge, ideas and best practise striving the highest scientific standards.  I m partiality: EMA policy on Handling Conflicts of

  • Interest. Public Declaration of Interest.

10 International Regulators – Awareness Session

The EU experts

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International Regulators – Awareness Session 11

Contents

The role of EMA Evaluation of medicines Safety monitoring of medicines Development and access to medicines Transparency

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  • EMA continuously monitors and supervises the safety of medicines that have

been authorised in the EU, to ensure that their benefits outweigh their risks.

  • The Pharm acovigilance Risk Assessm ent Com m ittee ( PRAC) of EMA is

dedicated to the safety of medicines.

  • EMA coordinates the EU pharm acovigilance system and operates services

and processes to support pharmacovigilance in the EU.

  • EMA operates Eudravigilance, an EU web-based information system that

collects, manages and analyses report of suspected side effects of medicines.

  • Public hearings are now a tool available during EU safety reviews of

medicines.

International Regulators – Awareness Session

Safety monitoring of medicines

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International Regulators – Awareness Session 13

Contents

The role of EMA Evaluation of medicines Safety monitoring of medicines Development and access to medicines Transparency

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  • EMA is committed to enabling tim ely patient access to new medicines, and

plays a vital role in supporting medicine development for the benefit of patients.

  • EMA prepares scientific guidelines on requirements for the quality, safety

and efficacy testing of medicines, reflecting the latest thinking on developments in biomedical science.

  • EMA provides product-specific scientific advice through the Scientific Advice

Working Party (SAWP).

  • EMA supports research and innovation in the pharmaceutical sector, and

promotes innovation and development of new medicines by European micro-, small- and medium-sized-enterprises.

International Regulators – Awareness Session

Development and access to medicines

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International Regulators – Awareness Session 15

Contents

The role of EMA Evaluation of medicines Safety monitoring of medicines Development and access to medicines Transparency

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  • EMA strives towards being as open as possible about how it works and how it

comes to its decisions.

  • EMA publishes clear and im partial inform ation about medicines and their

approved uses. This includes transparency of agenda and minutes of committees, public versions of scientific assessment reports and summaries written in lay language.

  • The public has the right to request information and documents from EMA in

accordance with its rules on access to documents and on access to information.

  • Since October 2016, EMA publishes clinical data submitted by pharmaceutical

companies to support their regulatory applications for human medicines under the centralised procedure. International Regulators – Awareness Session

Transparency

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Any questions?

Maria.NietoGutierrez@ema.europa.eu; Helena.Matos@ema.europa.eu

European Medicines Agency

30 Churchill Place • Canary Wharf • London E14 5EU • United Kingdom

Telephone + 44 (0)20 3660 6000 Facsim ile + 44 (0)20 3660 5555 Send a question via our w ebsite www.ema.europa.eu/ contact

Further information

Follow us on @EMA_ New s

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An agency of the European Union

The EU Marketing Authorisation

The EU medicines regulatory system and the European Medicines Agency: an introduction for international regulators and non-governmental organisations 18 September 2017

Presented by Rocio Gonzalo Ruiz Procedure Management Department

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International Regulators – Awareness Session 1

Contents

The Marketing Authorisation The Centralized Procedure Pre-Submission Evaluation Post Opinion

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International Regulators – Awareness Session 2

Contents

The Marketing Authorisation The Centralized Procedure Pre-Submission Evaluation Post Opinion

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Marketing authorisation

EU law requires all medicinal products to obtain a Marketing Authorisation (MA) before they can be placed on the market.

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  • Directive 2 0 0 1 / 8 3 / EC - The core

legislation governing the regulation of drugs in EU

  • National Law , implementing Directive

2001/ 83/ EC at national level

  • Regulation ( EC) No 7 2 6 / 2 0 0 4 – sets out

the centralised procedure

International Regulators – Awareness Session

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Choice of the authorisation route

Eligibility for centralised procedure has to be confirmed in advance of submission of the MA application

International Regulators – Awareness Session 4

Some products can now be authorised

  • nly via centralised

procedure (“m andatory CP scope”) For some the applicant can choose either centralised procedure or national/ MRP/ DCP procedure (“optional CP scope”) Some products are excluded from centralised procedure (outside

  • f any CP scope)
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http: / / www.ich.org/ products/ ctd.html

Requirem ents for Marketing Authorisation

Dossier based on an internationally agreed CTD format.

International Regulators – Awareness Session 5

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International Regulators – Awareness Session 6

Contents

The EU Marketing Authorisation The Centralized Procedure Pre-Submission Evaluation Post Authorisation

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International Regulators – Awareness Session 7

A system based on Trust & Reliance

Benefit/ Risk Evaluation

I ndividual Evaluation Com m on Review Connected Expertise Transparent

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International Regulators – Awareness Session 8

Contents

The EU Marketing Authorisation The Centralized Procedure Pre-Submission Evaluation Post Authorisation

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Scientific Advice / protocol assistance Pipeline information Request Eligibility for Centralised Procedure Request Invented name review Letter of intent to submit Appointment of Rapporteurs Innovation task force Orphan designation ATMP classification

Pre-submission for centralised procedure

Request for accelerated assessment SME designation Presubmission meeting

Paediatric requirements

Mandatory steps

ATMP certification CHMP Opinion on compassionate use (triggered by MSs)

Priority medicines (PRIME) scheme launched in March 2016

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International Regulators – Awareness Session 10

Contents

The EU Marketing Authorisation The Centralized Procedure Pre-Submission Evaluation Post Authorisation

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Assessm ent process in centralised procedure

International Regulators – Awareness Session 11

Primary Evaluation Clock Stop Secondary Evaluation

Post Authorisation D.1 D.120 D.121 D.210 D.277

List of Questions

Responses

  • Pharmacovigilance
  • Variations
  • Extensions
  • Renewal

Validation

D.180

  • D. 180

Hearing?

Clock Stop

List of Outstanding Issues

Final Ev.

D.181

Responses

Opinion EC decision

The system is collaborative, connected, and transparent

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Adoption of CHMP Opinion

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  • The CHMP assessment and Opinion:
  • based on scientific criteria
  • determine whether or not the products meet the

quality, safety and efficacy requirements

  • ensure that medicinal products have a positive risk-

benefit balance in favour of patients/ users

  • CHMP Opinion adopted by consensus or m ajority vote
  • In case of majority vote – divergent opinions of minority

included in the final documents.

Benefit Risk

International Regulators – Awareness Session

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Types of m arketing authorisations

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Am ount of Evidence Norm al or Full Conditional Exceptional Circum stances Type of Marketing Authorisation

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International Regulators – Awareness Session 14

Contents

The Marketing Authorisation The Centralized Procedure Pre-Submission Evaluation Post Opinion

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Centralised procedure ( post-opinion)

  • European Commission (EC) prepares draft decision
  • Standing Com m ittee on Medicinal Products for Human Use (representatives from

Member States) gives Opinion on the draft decision

  • EC adopts the decision on granting (or refusing) Marketing Authorisation, which takes

effect from the data of notification

  • Outcome is published by EC and EMA (including assessment reports and clinical data

The outcom e of the decision is published in a transparent w ay!

International Regulators – Awareness Session 15

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Any questions?

Rocio.Gonzalo@ema.europa.eu

European Medicines Agency

30 Churchill Place • Canary Wharf • London E14 5EU • United Kingdom

Telephone + 44 (0)20 3660 6000 Facsim ile + 44 (0)20 3660 5555 Send a question via our w ebsite www.ema.europa.eu/ contact

Further information

Follow us on @EMA_ New s

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An agency of the European Union

Lifecycle Product Management

The EU medicines regulatory system and the European Medicines Agency: an introduction for international regulators and non-governmental organisations 18 September 2017

Presented by Helena Matos Procedure Management Department

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International Regulators – Awareness Session 1

Contents

Post-Authorisation Activities Renewal Variations PSUSA & RMP PASS, PAES & PAMs Other activities post authorisation

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International Regulators – Awareness Session 2

Contents

Post Authorisation Activities Renewal Variations PSUSA & RMP PASS, PAES & PAMs Other activities post authorisation

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EU law requires the holder of the marketing authorisation (MA) to take account of scientific and technical progress and to keep the MA up to date with regards to its Quality, Safety and Efficacy

PSUR/ PSUSA

Variations

Sunset clause

Referral Urgent Safety Restrictions Withdrawal, Suspension

International Regulators – Awareness Session

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International Regulators – Awareness Session 4

Contents

Post Authorisation Activities Renewal Variations PSUSA & RMP PASS, PAES & PAMs Other activities Post Authorisation

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Renewal of MA

Marketing Authorisation Validity Norm al or Full 5 years 1 year Conditional Exceptional Circum stances Type of Marketing Authorisation 1 year Re-evaluation of risk/ benefit

International Regulators – Awareness Session

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International Regulators – Awareness Session 6

Contents

Post Authorisation Activities Renewal Variations PSUSA & RMP PASS, PAES & PAMs Other activities Post Authorisation

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An evaluation procedure adapted to the level of risk Variations

Do and tell

Changes requiring prior approval Extension Type II Type IB Type IA Design space Changes not requiring prior approval

Variations

International Regulators – Awareness Session

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International Regulators – Awareness Session 8

Contents

Post Authorisation Activities Renewal Variations PSUSA & RMP PASS, PAES & PAMs Other activities Post Authorisation

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Period Safety Update Reports (PSURs)

9 International Regulators – Awareness Session

Periodic benefit risk evaluation 6 m 1 2 m 3 years Cumulative safety data

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Risk Management Plan (RMPs)

International Regulators – Awareness Session

  • Prospective planning based on the

knowledge on the product

  • Risk minimisation measures
  • Ensuring effectiveness of RMM
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International Regulators – Awareness Session 11

Contents

Post Authorisation Activities Renewal Variations PSUSA & RMP PASS, PAES & PAMs Other activities Post Authorisation

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Post-authorisation studies (PASS / PAES) & Post Authorisation Measures (PAMs)

  • Regulators can require studies to be

conducted at first authorisation / post- authorisation

  • Study is a condition of the authorisation

and is legally binding

  • Protocol and final study results are

submitted to the EMA (e.g. PAM)

International Regulators – Awareness Session

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International Regulators – Awareness Session 13

Contents

Post Authorisation Activities Renewal Variations PSUSA & PAMs RMP , PASS & PAES Other activities Post Authorisation

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Other activities Post Authorisation

Overview of the European Regulatory System for Medicinal Products 14

Sunset clause Urgent Safety Restriction Referrals

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Referrals

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  • Triggered by issues on Q, S or E
  • EU assessment of the impact
  • n the benefit risk
  • Harmonised outcome across the EU

International Regulators – Awareness Session

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Any questions?

Helena.Matos@ema.europa.eu

European Medicines Agency

30 Churchill Place • Canary Wharf • London E14 5EU • United Kingdom

Telephone + 44 (0)20 3660 6000 Facsim ile + 44 (0)20 3660 5555 Send a question via our w ebsite www.ema.europa.eu/ contact

Further information

Follow us on @EMA_ New s