Joint Rare Diseases / Orphan Medicinal Products Task Force

JOINT RARE DISEASES / ORPHAN

MEDICINAL PRODUCTS TASK FORCE

A collaborative initiative with national trade associations and policy developments at EU level.

Lugdivine Le Dez (Alexion)

Laura Gutierrez (Celgene)

Agenda

I. Presentation of the joint taskforce

II. Cooperation with national OD groups:

◦ Objectives, rationale, proposal, expected

outcomes

III. Highlight of EU policy developments

I. Presentation of the joint

taskforce

European member companies of all sizes that have either developed or intend to develop orphan drugs

Members of EBE and/or EuropaBio,

4 Meetings per year

Name of active member companies: Actelion, Alexion, Astra Zeneca, Biomarin, Celgene, CSL Berhing, Dompé, Genesis Pharma, Genzyme, GSK, Novartis, Novo Nordisk, Merk Serono, Orphan Europe, Pharma Mar, Pfizer, Shire.

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companies

What is the Joint Task Force?

Objectives of the

Joint Task Force

Contribute to the development of EU rare diseases-related policies

and regulatory frameworks

Work towards a harmonised implementation of EU legislation and

regulatory guidelines for OMPs

Improve timely access to therapies for all rare disease patients

across the EU

Raise awareness about the economic and social value of OMPs

Why the EU matters?

• Member States’ individual responsibilities:

Health care financing

Pricing and reimbursement of drugs

• Influences the political agenda of the individual EU

countries:

Laboratory of ideas

Meeting point of national authorities: Exchange of

best /worst practices among Member States

• Regulation of the pharmaceutical sector:

Marketing Authorizations

Clinical Trials rules

Orphan Drugs Regulation (ME)

Intellectual Property (Data protection, SPC)

Joint taskforce 2011-2012 priorities

• Industry input in the CAVOD process and Mechanism of

coordinated access to ODs • Exceptions for OMPs both at EU and national level and establish links with national trade association.

• National Plans for Rare Diseases

ACCESS TO OMPs FOR PATIENTS

• Build tools to communicate the values and specificities of the OMP business models

• Implementing a tailored outreach (educational) programme mainly targetted at EU audiences

VALUE AND REPUTATION OF

THE RARE DISEASE

BUSINESS MODEL

• Ensure that the EU legislative and regulatory frameworks continue to be appropriate and predictable for OMP developers

• Ensure that a consistent framework for registries is implemented at national level

LEGISLATIVE AND

REGULATORY ASPECTS OF

OMPs

II. Cooperation with national

OD groups

A collaborative approach with national trade

associations

Creating interaction

EU Orphan Drug

Taskforce

National trade

associations

Main objectives

- Enhancing cross-communication

- Information sharing / best practice sharing

- Supporting local efforts/crisis when

needed

• European Commission

• European Parliament

• EU Council

EU Regulators • EMEA/COMP

Other Stakeholders

• Patient group

• Healthcare professionals

• Academia

National trade

associations

An new partner

EU institutions

Joint EBE-EuropaBio

Task Force

Rationale

- Relevance of MS policy work on EU policy and vice

versa

- Dominoes effect of local policy/cost containment

measures on other MS

- Common objective to orphan drug policy

- Unstable EU economic/financial environments needing

sustained policies for ODs

Interaction process

EU Orphan Drug Taskforce National trade associations

• Share with you with tools we

developed to promote the value of

ODs, and punctual EU policy

updates

• Organise a yearly “get together” in

Brussels with all national relevant

leaders to share best practices and

align on common goals.

• Share information about major

national political and legislative

decisions that can have an impact

on orphan medicines in your

country but also in the rest of

Europe

• Share tools/concepts • Send your group representative to

the yearly meeting in Brussels

• Ensure an industry unified approach to OD policy

developments

• Ensure EU support is provided when requested to

reinforce local lobby

• Ensure all parties (EU/Local) are ready to react in time

Tools to update you on ongoing EU

work and to promote national plans

Tools to communicate OD specificities

The budget impact is

estimated to have grown from 0% in 2000 to 3.3% by 2010

From 2010 to 2016, the rate

of growth is likely to slow

and will reach a plateau of

approximately 4.6%

3.3%

4.6%

Orphan Drug Budget Impact

The budget impact of orphan medicines in Europe has grown steadily over the 10 years

since the introduction of the orphan drug legislation in 2000, but growth is predicted to

slow and plateau over the next decade

III. Key highlight of EU policy

developments

Highlights of EU ongoing policy

developments

EU policy

Follow up to implementation

of National Plans

CAVOD

MOCA Clinical Trials

Directive

Transparency Directive (P&R)

Directive on patients’ rights in cross-border

healthcare

EUCERD

Council Directive 89/105/EEC on P&R

processes– “Transparency” Directive

Does not affect national decisions regarding prices

Sets rules for decision regarding P&R (time-limits, transparency, and right to appeal)

Timelines:

◦ December 2011/January 2012: New legislative proposal expected end.

◦ Debate in the EP and Council is foreseen in 2012;

◦ Implementation by Member States in 2013/2014.

Expected content:

- Shortening of timelines for both generics (15/30 days) and innovative medicinal

products (60/120 days).

- HTA in the scope

- Results of the marketing authorization (quality, safety, efficacy, bioequivalence) shall

not be reassessed in HTA decisions

Concerns

Instrumentalisation by Member States to facilitate cost-containment

measures and increased international reference pricing

Demands by European Parliament for harmonisation of prices

Council Directive 89/105/EEC on P&R

processes– “Transparency” Directive


developments

EU policy


of National Plans

CAVOD


Directive



healthcare

EUCERD

Background: Clinical Added-Value of Orphan

Drugs (CAVOD)

Initiated by Eurordis, included in the recommendations of High Level Pharmaceutical Forum ◦ Possible exhange of knowledge on the scientific assessment of

the clinical added value of orphan drugs (CAVOD)

Original objectives – speed patients access ◦ Make the most of evidence on Clinical Added Value of Orphan

Drugs gathered by EMA

◦ Coordinate payers requirements for additional data

European Commission commissioned E&Y to identify implementation options

Main purpose of the

CAVOD study

PURPOSE OF THE STUDY

• Identify and assess the possible options for the creation of a mechanism for the exchange of knowledge between Member States and European authorities on the scientific assessment of the relative effectiveness of orphan medicines (to be implemented, if possible, from 2011)

PURPOSE OF THE MECHANISM • To facilitate MS informed decision on the scientific

assessment of the clinical effectiveness of an orphan drug

• To contribute to the development of a continuum between pre-market authorization practices (clinical development) at EU level and post-marketing authorizations practices at member state level

• SOURCE: European Commission

CAVOD: a potential tool for better

access to orphan drugs

Based on the initial opinion of the COMP and CHMP at the time of

orphan designation, which

includes an evaluation of the significant clinical benefit of an OMP –

which is confirmed at time of MA

CAVOD: The E&Y report

A state of play of regulatory processess

and HTAs for ODs in Europe.

An identification of the data collection

which could be accepable for assesing

relative effectiveness of ODs.

A proposal for a collaboration mechanism

between regulatory and HTA bodies for

OD assessment.

Sources: Draft Backgroung review, EUnetHTA JA WP5: Relative Effectiveness Assessment (REA) of Pharmaceuticals; April 2011 EUnetHTA WP4 – HTA Core Model for Medical and Surgical Interventions, Dec ember 2008

(1) Approach aligned with the EUnetHTA JA WP5: REA of Pharmaceuticals, Draft Background review, April 2011:. WP5 model

to be particularly considered as being part of

rare diseases’ specificity

► The type of evidences (1) covered by the clinical added value are based on the EUnetHTA JA WP4 & WP5 & HTA core model and adapted to the specificity of orphan drug

► 1. Health problem and current use of the technology

► 2. Description and technical characteristics of technology

► 3. Safety

► 4. Effectiveness

► 5. Costs, economic evaluation

► 6. Ethical aspects

► 7. Organizational aspects

► 8. Social aspects

► 9. Legal aspects

Data to measure the CAVOD

already considered

as part of EMA scope

E&Y: Interaction process

EUnetHTA-CAVOD/EMA ► The objective of the interaction between EUnetHTA-CAVOD and EMA is to build a bridge and

develop a continuum between pre-market authorization practices (clinical development) at EU level and post-marketing authorizations practices at member state level.

► Through this process, the National HTA bodies through EUnetHTA are engaged collectively and bring their experience together to give inputs and valuable requirement to the EMA prior to the Risk management plan.

► Member states will be able to contribute to the Risk management plan.

Evidence requirement

gathering from HTA bodies

Writing of recommendation

paper

Identification of the project manager PM

EUn

etH

TA-C

AV

OD

Exchange of information with the rapporteur of OD at the CHMP

Marketing Autorisation review process, prior to CHMP opinion

Exchange of information with EUnetHTA-CAVOD PM *

One EUnetHTA-CAVOD PM for 4 days (FTE)

EMA

•Focused on HTA expectations for evidence generation • Based on national HTA bodies experience of the disease

&/or drug

Recommendation paper for EMA

* Discussion to consider at the CHMP committee

including EUnetHTA-CAVOD PM ?

CAVOD: The E&Y Report

Time

CHMP opinion, T0 EC marketing authorisation T0 + 90 days

T0+ΔT (after 3 to 5 years, flexible depending of the disease)

Period 1: for EMA / EUnetHTA

coordination

Period 2: for simple

Compilation report & evidence

generation plan

Period 3: for follow up of the

evidence generation plan

Period 4: relative

effectiveness assessment

Protocol assistance

Significant Benefit, COMP

E&Y report: Concerns

Involvement of EUnetHTA, namely:

◦ Involvement of HTA bodies in the regulatory

process -> impact on MA.

◦ Interactions between EMA and HTA not well

defined additional hurdles for access?

◦ Lack of expertise in OD of HTA bodies.

CAVOD: Next steps

Rare Diseases stakeholder community

• Industry comments under development Dec

2012

• EUCERD drafting group

• Workshop tentatively scheduled for March 2nd

2012

• EUCERD opinion document to be adopted

June 2012


developments

EU policy


of National Plans

CAVOD


Directive



healthcare

EUCERD

Mechanism of coordinated access to ODs-

Background

Sept 2010: Launch of “Tajani initiative” including all

relevant stakeholders in pharmaceutical sector.

3 platforms aimed at developing recommendations

focusing on specific issues:

◦ Ethics and Transparency

◦ Access to medicines in the least developed countries

◦ Access to medicines in Europe, including a platform on

Mechanism of coordinated access to orphan medicinal

products.

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Mechanism of cooordinated access

to ODs Project vision: The coordination between stakeholders and

Member States at EU level to provide real access to a real solutions (orphan medicinal products) for real patients with real unmet medical needs, for which these solutions would otherwise be out of reach – in an affordable and sustainable way (-> “real life access”).

Project objective: Designing a concrete operational manual of coordinated real access for stakeholders and Member States to achieve the vision irrespective of the local conditions starting with the identification of the unmet medical need and the possible answers (phase zero).

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Mechanism of coordinated acces to ODs -

Project Methodology

Mechanism of coordinated access to orphan medicinal products

WP1 Identifying and

assessing a relevant orphan drug

(Assessment/evaluative)

WP2 Selection of target

population and mechanism of funding

(Structural access)

WP3 Treatment

(Individual access)

#1 operationa

l step

#2 operationa

l step

#1 operationa

l step

#2 operationa

l step

#2 operationa

l step

#1 operationa

l step

#1 impl. activit

y

#2 impl. activit

y

Reminder

Objective: an industry unified approach

when dealing with Orphan Drug Policy

Proposed next steps:

◦ Sharing of messages/tools

◦ Regular updates

◦ Yearly meeting (date tbd)

Your feedback on cooperation?

Thank you for your attention!

Back-up slides

Directive on patients’ rights in

cross-border healthcare

Directive on patients’ rights in cross-border healthcare Adopted by the European Parliament

(EP) on 19 January and by the Council on 28 February. Member States have until 25 October

2013 to implement it

The EP managed to include rare diseases in the scope of the directive, which had been opposed

for long by MS

Key elements of the adopted text:

◦ Prior authorisation system (could only be refused on obvious grounds)

◦ Reimbursement (a patient being treated in another Member State should be reimbursed up to the amount

of the reimbursement anticipated by his own national health system for similar treatment)

◦ Diagnosis and treatment of rare diseases (support to EU Member States in cooperating in the development

of their diagnosis and treatment capacity )

◦ Information to patients (obligation to set up a kind of one-stop shop)

◦ European reference networks (objectives: facilitate mobility of expertise (virtually or physically); develop,

share and spread information, knowledge and best practice; foster developments of the diagnosis and

treatment of rare diseases)

◦ HTA cooperation (new legal basis for an improved HTA-related cooperation between EU Member States)

◦ Prescription recognition (a prescription issued in another EU country will be recognised in a patient's

country of residence and vice versa)

WP 1 WP 2 WP 3

BELGIUM Ri De Ridder 2

Francis Arickx 1 2 3

Catherine Adriaens 1 2

Marleen Mortier 2

Céline Hermans

Mireille Pierlet Diane Kleinermans 3

AUSTRIA Christine Leopold 3

Florian Bachner ESTONIA Dagmar Ruutel 1

FINLAND Sari Ekholm

Jyrki Vanakoski 1

FRANCE Danielle Golinelli Pierre Pribile

Patrick Cayer Barrioz 1 2 3

Arlette Meyer 1 2

Catherine Choma

Nadine David

Renaud Morin

GREECE Lena Katsomiti

HUNGARY M Szabados

Mi Palosi Claudia Habl 1 2 3

Sabine Vogler

ITALY Pietro Folino Gallo

Paolo Siviero 2

MALTA Jennifer Farrugia 1

Isabelle Zahra-Pulis

NETHERLA

NDS

Hugo Hurts

Huub Kooijman

HJJ Seeverens

POLAND Jakub Adamski 2

PORTUGAL Ines Ramos 2

SPAIN Mercedes Vallejo

Martinez

2

SWEDEN Anna-Marta Stenberg

Maria Storey 1

EFPIA Katia Finck 2 3

Wills Hughes-Wilson 1

Ana Palma 1

Kevin Loth 1

Adam Heathfield 2 3

Laura Gutierrez 1

Enda Scott 2 3

Thomas Cueni

François Bouvy

EGA Greg Perry

Ilina Markova

EuropaBio Ludovic Lacaine

GIRP Monica Derecque-Pois

Martin Fitzgerald 2 3

EPF Yann LeCam 1 3

Ariane Weinman

Flaminia Macchia 1 3

AIM Heidi Goethals 2

ESIP Anna Bucsics 2

Hans Seyfried 2

CPME Oscar Arias 3

Birgit Beger

Synnove Lindemalm 1 3

COMP Elodie Carmona

Dominik Schnichels

SANCO Jerome Boehm

Anders Lamark Tysse

Nathalie Chaze

Patricia Brunko

Mirjam Soderholm

MARKET Jean Bergevin

Jan Willem Verheijden

ENTERPRISE 1 2 3

Thomas Heynisch

Christophe Roeland

Valerie Vanhoeck

Giulia DelBrenna

Laura Nistor

Aurelie Vandeputte

Eminet 1 2 3

Joint Rare Diseases / Orphan Medicinal Products Task Force

Health & Medicine

Transcript of Joint Rare Diseases / Orphan Medicinal Products Task Force