18th Workshop EURORDIS Round Table of Companies February 27th, 2013 Brussels, Belgium Le Plaza Hotel Brussels
Corporate Responsibility in Improving Access to Orphan Medicinal Products y Introduction y Concept Paper y Programme y Participants y Presentations & Speakers y Report Breakout Sessions
19th Workshop of the Eurordis Round Table of Companies - February 27th, 2013 - Brussels
Introduction
The 18th Workshop of the EURORDIS Round Table of Companies (ERTC), entitled "Corporate Responsibility in Improving Access to Orphan Medicinal Products" took place on the 27th February 2013 in Brussels, Belgium, and gathered 82 participants from 15 countries representing a wide variety of stakeholders. Yann Le Cam opened the workshop with an overview of all key concepts of the day and introduced proposals aimed at achieving quick access to safe, efficient and affordable medicines with a real therapeutic added-value for rare disease patients in Europe. Amongst these proposals, the importance of a continuum of data collection on medicinal products from clinical trials or compassionate use, real-life studies after marketing authorisation and even data from off-label use was emphasised. The role of regulators and regulatory flexibility have been documented to play an important role as does seeking and complying with scientific advice in the form of protocol assistance or scientific advisory groups, prior to and after application for marketing authorisation, respectively. As part of the evolution of the European Medicines Agency, the involvement of health technology assessment agencies in the above-mentioned procedures has also been a step forward for ensuring faster access to medicines with the additional objective of fewer conflicting messages between marketing and reimbursement obligations. The additional important consequence would be a potential reduction in the number of patients used in clinical trials. The recent proposal for adaptive licensing was also discussed and its applicability in particular to rare diseases. Early dialogue between all stakeholders is a frequently repeated message and a positive step forward has been taken between the EMA and HTA agencies to achieve this. Adapted clinical designs and the modifications to the Clinical Trials Directive are welcomed. Strengthening of the ongoing collaboration between EMA and FDA is encouraged. Finally, initiatives such as the Clinical Added Value of Orphan Medicinal Products (CAVOMP) and the Mechanism of Coordinated Access (MoCA), Differential Pricing and implementation via national measures, as well as the EUROPLAN project were outlined. This introductory ‘setting the scene’ was followed by a presentation by Dr. Thomas Heynisch, from the European Commission, on Corporate Responsibility where he introduced the three platforms launched in 2010 by Commissioner Antonio Tajani in the field of pharmaceuticals: i) Access to Medicines in Europe (6 sub-groups), ii) Access to Medicines in Africa and iii) Ethics and Transparency. The results are expected to be finalised by mid-2013. Of particular relevance to this audience is the Access to Medicines in Europe platform, within which there are five projects: 1. Capacity building on managed entry agreements for innovative medicines 2. Facilitating supply in small countries 3. Promoting a good governance for non-prescription drugs
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4. Market access for biosimilars 5. Mechanism of coordinated access to orphan medicinal products (MoCA), in which EURORDIS has been participating on behalf of EPF which is the official member of the Platform. The objectives for the MoCA working group are to find innovative ways to provide real access to orphan drugs for patients with unmet medical needs, for whom these solutions would otherwise be out of reach, in an affordable and sustainable way. The project comprised three work packages i) Assessing orphan medicinal products (coordinated by Italy and EFPIA); ii) Organising a structural access (coordinated by Spain and ESIP) and iii) Organising the individual access (coordinated by Belgium and EURORDIS). Dr. Heynisch concluded by saying that it is the role of patients’ organisations to convince their Member States to become proactive in order for this to be considered a way forward. A panel discussion, including Dr. Adam Heathfield (Pfizer) and Dr. Paolo Siviero (Italian Medicines Agency), focused on the work performed by the task force established to work on MoCA. The main messages to emerge were a reflection that involving more representatives from Eastern European member states would have been beneficial to the project, as well as the inclusion of more clinicians. There is an understanding that the national competent authorities are reluctant to lose their control over the decision-making for the price of medicinal products however this project concerns more than simply pricing. Adaptive licensing was discussed as it is becoming more accepted as a possible alternative to the current system of evaluation and approval for medicines. A clear need exists, however, less certainty is associated with this type of licensing that would also be reflected in a certain difficulty in deciding on a price. Once again, the absolute need for early dialogue was raised as it is not just a matter of paying less but more so of paying ‘right’ as reimbursement decisions cannot be made based on personal choice, but on the benefit brought by the medicinal product. Building upon the responsibility of Member States, and in particular under the recommendations of the Council of the European Union in 2009, the national measures in the context of National Plans were discussed by Wills HughesWilson. While National Plans have been discussed at previous EURORDIS meetings, the main message here was the importance of building national plans that have ‘teeth’, that is those that will deliver what they promise, that will translate ideas and principles into actions. To assist in this action, two proposals developed separately, the CAVOMP and the MoCA, propose parallel approaches and suggestions for tackling any bottlenecks. These include the development of the appropriate methodological tools and involvement of the right stakeholders, registries, development of a transparent value framework and, of course, national plans, which have a deadline of end of 2013. Under the Belgian Presidency in EU, the recommendation to make valuable innovative medicines accessible in the EU was made a priority. Emphasis was placed on equity between Member States and hence between European citizens. Once again, the CAVOMP and the MoCA were described as two examples of cross-country European initiatives. An informal high-level stakeholder working group chaired by John Bowis was been formed and is composed of individual members of the European Commission, Patients’ organisations, Permanent representatives – Health attaches, payer organisations, health economists and members of industry. The objective of this working group, presented by Johan van Calster, former Head of the Belgian Medicines Agency, is to address unmet medical needs and to focus on differential pricing. The basic aim of price differentiation is to
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improve equality of access to innovative pharmaceuticals for unmet medical needs across Europe. The price reflects ability/willingness of a country to pay. Aspects such as transparent timelines, stability of currency, external verification bodies must all be considered. Pricing and Reimbursement remain within the individual Member State enabling countries to negotiate prices and to decide whether to reimburse based on their own criteria. External Reference Pricing and Parallel Trade are not compatible with differential pricing and would reduce willingness of companies to participate. What is required is a transparent market entry plan with a commitment of participating Member States to apply differential pricing principles and a commitment of companies to launch the medicinal products within two years of receiving the marketing authorisation. A panel session with Richard Bergström (EFPIA), Francis Arickx (RIZIV/INAMI), Johan van Calster and Flaminia Macchia (EURORDIS) discussed the main points of differential pricing as a way to improve access to medicinal products by differentiating prices according to the countries’ ability to pay and the size of the target populations. This instrument is currently being analysed for its application in Europe by a group of experts gathered at EU level. Differential Pricing is often perceived by the public as a socially responsible behaviour, thus enhancing the ethical reputation and the social responsibility of the provider. Richard Bergström introduced the Differential Pricing panel discussion by initiating key questions raised around the idea of development towards differential pricing concept, and which type of processes would be needed to ensure common European political and legal framework keeping the pricing negotiations at the member state or, in certain situations, even at the regional level. The questions and issues such as pricing transparency, parallel import, the European Union single market principal, consequences of referential pricing and single pricing models or how to overcome risk of shortages were presented. The idea of clustering the European Market by grouping the Member States with similar economic conditions together - with or without volume-based pricing - might be an option for further discussions when a developing framework for a differential pricing model. The orphan drugs with patient-tracking system might be a good option to start with as a pilot. Thus, an intense day of discussions and brainstorming concluded another successful ERTC workshop with great speakers and excellent input from the participants. We are certain to be hearing more about these topics as the deadline for the national plans approaches and both the Differential Pricing and the Mechanism of Coordinated Access documents are finalised. No doubt another ERTC workshop will focus on pushing forward measures to improve access to medicinal products for rare disease patients.
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Concept Paper
Access to medications in a timely and affordable manner is a long-standing issue for patients globally whether affected by cancers, infectious or rare diseases. This problem is more acute in current conditions of economic crises or health budget austerity measures as we observe in the EU since 2008. There is no unique or simple solution. Key stakeholders working in partnership include patients, industry, regulators, national competent authorities and payers who are forging new concepts and practical measures to address these challenges. The aim is for faster, fair access for patients to treatments, improvement of medical practices based on these treatments and optimisation or reduction of the healthcare budget allocated to innovative treatments for chronic diseases. There are several solutions, some already consensual, and others emerging, but all well identified. These solutions can be implemented separately or in combination towards a new viable economic model for industry rewarding value, a sustainable public health budget model and for equitable approach ensuring that the most vulnerable (the patients) are at the centre of any proposal. The European Commission published a new policy on Corporate Social Responsibility in October 2011 that states that to fully meet their social responsibility, enterprises “should have in place a process to integrate social, environmental, ethical and human rights concerns into their business operations and core strategy in close collaboration with their stakeholders”1. The term "corporate social responsibility or CSR" came into common use in the late 1960s and early 1970s after many multinational corporations formed the term stakeholder, meaning those on whom an organisation's activities have an impact2. The economic crisis and its social consequences have to some extent damaged consumer confidence and levels of trust in business. They have focused public attention on the social and ethical performance of enterprises. While there are opinions for and against the concept of corporate social responsibility, it cannot be denied that in these times of economic crisis expectations towards the private sector increase. In a study by Giannarakis and Theotokas in 20113, it was observed that at the beginning of the economic crisis, companies moved away from socially responsible behaviour due to the cost involved. This move away was higher in the US than in Europe, however participation in CSR was resumed as companies changed their perception of the idea from a ‘threat to an opportunity’. By renewing efforts to promote CSR, the Commission aims to create conditions favourable to sustainable growth, responsible business behaviour and durable employment generation in the medium and long term, while addressing consumers’ and patients’ needs. An important challenge – and opportunity - is particularly present this year, which is achieving the goals of the EUROPLAN II project within EUCERD Joint Action. As a community we are faced with the looming deadline of this project that aims to increase the capacity of participants, support EU member states with different geographic and economic concerns and different states of readiness to deliver their National Plan on Rare Diseases via conferences and concrete guidelines. Many different stakeholders play a role in the international and interactive network of stakeholders (including patients, policy makers, academics and industry members) to speed up the elaboration and the implementation of Rare Diseases National Plans/Strategies, through scientific and technical assistance, and workshops. Two countries have already held their national conferences as part of the EUROPLAN II project, Sweden and Greece, and both reported back on the challenges faced during their respective meetings. Greece in particular felt that no real improvement had been seen since 2010 and that none of the recommendations of the first
1
Communication from the commission to the European Parliament, the council, the European Economic and Social Committee and the committee of the regions: A renewed EU strategy 2011‐14 for Corporate Social Responsibility http://eur‐lex.europa.eu/LexUriServ/LexUriServ.do?uri=COM:2011:0681:FIN:EN:PDF 2 R Freeman, Strategic management :a stakeholder approach (Pitman 1984) ISBN 978‐0‐273‐01913‐8 3 Giannarakis, G and Theotokas, I (2011) The Effect of the Financial Crisis in Corporate Social Responsibility Performance. Int. J. Marketing Studies. 3 (1): 2‐10
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Conference has been implemented. In addition, the economic crisis has deteriorated the already precarious situation for rare disease patients: lack of registries, no official identification criteria for centres of expertise, no policy for access to orphan medicinal products and increasingly difficult access to those already on the market. Pharmacies in Greece had been reporting shortages of medicines as some distributors have re-exported comparatively cheap drugs from Greece to other European markets, achieving monetary gains of as much as 600%. Currently, there are fears of drug shortages in certain hospitals as a result of unpaid bills. And concerns that the problem could grow have led to a call for the country to re-evaluate the cost-effectiveness of medicinal products covered by its public health system. Companies members of the European Federation of Pharmaceutical Industries and Associations (EFPIA) offered to cap the total amount that Greece has to pay for its medications in a letter to the Greek ministers of health and finance. A request was made to pharmaceutical companies, by the representative of the ministry for health, not to reexport the medicinal products thereby leaving citizens without treatment. It seems that this is due to the pricing policy that obliges companies to adopt the lowest price compared to the other EU countries leading to a shortage of medicinal products in Greece, the so-called external reference pricing. Differential pricing in the context of pharmaceutical products is the strategy of selling the same medicine to different markets at different prices, based on the national GDP and sometimes also on the size of the population. It is an instrument used to improve access to medicines in developing countries, although shortcomings may persist. It seems interesting to launch this debate also inside the EU. In the past, some initiatives were already taken (e.g. Bremen declaration). There are also signals that the pharmaceutical industry is considering to make more use of differential pricing (BMJ 2011, 343, d8049). The industry has always been a bit nervous about differential pricing, mainly because companies fear that lowly priced products will flow back to high income countries and also because of the practice of external reference pricing, whereby countries calculate a price for a drug on the basis of lower prices elsewhere. A key point will be to develop an acceptable method to achieve the goal, while respecting the right of the Member States to operate their national health care systems. The 18th workshop on “Corporate Responsibility in Improving Access to Orphan Medicinal Products” will be divided into three distinct sessions. The morning session will focus on ‘Corporate Responsibility and National Measures’ and ‘Differential Pricing’. The first part of the morning session, will be opened with an overview by EURORDIS of all consensual or emerging concepts and measures. The Commission will present the outcomes of its policy on Corporate Responsibility and will include speakers from the Commission as well as Industry members involved with the project on a Mechanism of Coordinated Access (MoCA) to Orphan Medicinal Products. This first part will be concluded with a presentation by an EBE-EuropaBio Task Force representative speaking about measures expected by industry in National Plans to improve access to orphan medicinal products. The second part of the morning will focus on Differential Pricing, with a presentation followed by a panel discussion on the topic. Finally, we will close the morning session with three parallel breakout sessions enabling open and in-depth discussion on i) National Measures, ii) Differential Pricing and iii) Mechanism of Coordinated Access to OMPs. The afternoon session will begin with feedback from the breakout sessions and then discuss the important issue of how to address the challenges of access to orphan medicinal products in countries most affected by the crisis in Europe. We will hear the industry perspective and follow this with a panel discussion involving the Chair of the Committee for Orphan Medicinal Products, a patient advocate and two representatives of industry. The workshop will conclude with a wrap-up of the main discussions of the day.
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19th Workshop of the Eurordis Round Table of Companies - February 27th, 2013 - Brussels Agenda Chairpersons: Mr. Yann Le Cam (CEO, EURORDIS) & Dr. Jaroslaw Waligóra (European Commission, DG Health & Consumers) 9:00 – 9:30
Welcome address: Yann Le Cam
9:30 – 11:20
Corporate Responsibility & National Measures
9:30-9:50
Corporate Responsibility: Dr. Thomas Heynisch (European Commission, DG Enterprise)
9:50-10:20
Panel discussion involving Dr. Adam Heathfield (Pfizer) and Dr. Paolo D. Siviero (Italian Medicines Agency (AIFA))
10:20-10:50
National Measures to Improve Access to Orphan Medicinal Products in the context of National Plans: Ms. Wills Hughes-Wilson (SOBI/EBE-EuropaBio)
10:50 – 11:20
COFFEE BREAK
11:20 – 13:10
Differential Pricing
11:20 - 11:45
Differential Pricing: Mr. Johan Van Calster (former Head of the Belgian Medicines Agency and special advisor to Fipra for the Differential Pricing project)
11:45 – 12:10
Panel discussion involving Mr. Richard Bergström (EFPIA), Ms. Flaminia Macchia (EURORDIS) and Mr. Francis Arickx (RIZIV / INAMI)
12:10 – 13:10
Parallel breakout sessions • National Measures: What are the measures included in the National Plans for Rare Diseases that should be promoted as priorities in order to improve access to OMPs? • Differential Pricing: Is the approach proposed in the Non-Paper on Differential Pricing sufficient for improving access to OMPs and under which conditions? • Mechanism of Coordinated Access to OMPs: What are the key success factors necessary for making the Common Assessment of the Value of OMP based on the European Transparent Value Framework work?
13:10 – 14:30
LUNCH
Chairpersons: Mr. Patrick Deboyser (European Commission, DG Health & Consumers)& Professor Bruno Sepodes (Chairperson COMP, EMA) 14:40 – 16:30
Feedback from breakout sessions - How to address the challenges of access to orphan medicinal products in the countries most affected by the crisis
14:40 – 15:10
Feedback from each of the 3 breakout sessions 10’ each
15:10 – 15:35
How to address the challenges of access in countries most affected by the crisis: Mr. Richard Bergström (EFPIA)
15:35 – 16:15
Panel discussion involving Professor Bruno Sepodes (Chairperson, COMP, EMA); Mr. Dimitrios Synodinos (BoD EURORDIS/Greek Alliance for Rare Diseases); Drs. Mohit Jain (BioMarin Europe) and Kevin Loth (Celgene Europe)
16h15 – 16:30
Conclusions
16:30
Meeting ends
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Participants
Name
Affiliation
Catherine ADRIAENS Francis ARICKX Laura BACHELOR Catherine BERENS Richard BERGSTRÖM Anne-Mary BODIN Jill BONJEAN Carole BOYER Riccardo BRAGLIA Germano CARGANICO Claudia CARRAVETTA Emmanuel CHANTELOT François CORNU Virginia CUERVO Jurgen DE BEULE Cathy DE GROVE Patrick DEBOYSER Annamarie DILLON Pierre DODION Henk DOUDE van TROOSTWIJK Monica ENSINI Marie-Hélène FANDEL Maria FAGERQUIST Marie-Christine FORTUN Philippe GARD Rüdiger GATERMANN Eric GERVAIS Ruben GIORGINO Christophe GIOT Diane GOETZ Minna GRANGER Lesley GREENE Adam HEATHFIELD Gerald HECKEN-MEISSNER Isabel HENKEL Thomas HEYNISCH Birthe HOLM Wills HUGHES-WILSON Gillian IVERS-READ Dennis JACKMAN Mohit JAIN Kim JOHNSON Diane KLEINERMANS Stephan KORTE
RIZIV / INAMI RIZIV / INAMI Fipra International European Commission - DG Entreprise & Industry EFPIA EURORDIS EURORDIS Medunik Canada, Inc. Helsinn Healthcare SA Rare Partners Srl Pfizer Shire Aegerion Pharmaceuticals Pharma Mar, S.A. Actelion Alexion Pharma European Commission - DG Health & Consumers Genzyme ARIAD Pharmaceuticals, Inc. Raptor Pharmaceuticals Europe B.V. EURORDIS Amgen Novartis Orphan Europe EUSA Pharma CSL Behring Medunik Canada, Inc. Helsinn Healthcare SA InterMune International AG PTC Therapeutics, Inc. EURORDIS EURORDIS Rep. at COMP, EMA/ CLIMB Pfizer Raptor Pharmaceuticals Europe Grifols Deutschland GmbH European Commission – DG Entreprise & Industry EURORDIS/Rare Disorders Denmark EBE-EuropaBio/SOBI Clovis Oncology CSL Behring BioMarin Europe Celgene Europe Ltd. RIZIV / INAMI Novartis Pharma AG
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Round Table of Companies - February 27th, 2013 - Brussels 19th Workshop of the Eurordis Mark KRUEGER Yann LE CAM Lugdivine LE DEZ Kerry LEESON-BEEVERS James LENNERTZ Roberto LORE Kevin LOTH Flaminia MACCHIA Jamie MANGANELLO-RING Frédéric MARIN Maria MAVRIS Thomas MICKENAUTSCH Marleen MORTIER Patricio MURIAS Christel NOURISSIER Thomas OGORKA Ken O'REILLY Ana PALMA Samantha PARKER Kay PARKINSON Julien PATRIS Chris PICKARD Vinciane PIRARD Andrea PONTIGGIA Tamsin RAWLE Oli RAYNER Claude SCHMITT Bruno SEPODES Paolo SIVIERO Dimitrios SYNODINOS Johan VAN CALSTER Peter VANOVERTVELD Jaroslaw WALIGÓRA Geske WEHR
Mark Krueger & Associates, Inc. EURORDIS Alexion Pharma Alström Syndrome UK BioMarin Europe BioMarin Europe EBE-EuropaBio/Celgene EURORDIS Genzyme GMP-Orphan SAS EURORDIS Raptor Pharmaceuticals Europe B.V. RIZIV / INAMI Merck Serono BoD EURORDIS/ Alliance Maladies Rares OrphanReach Biogen Idec International GmbH Shire HGT Orphan Europe Alström Syndrome UK Celgene Pfizer Genzyme Helsinn Healthcare EUSA Pharma Spit It Out! CF blog GSK Rare Diseases COMP, EMA/Lisbon University Italian Medicines Agency (AIFA) BoD EURORDIS/Greek Alliance for Rare Diseases Former Head, Belgian Medicines Agency - advisor Fipra Baxter European Commission - DG Health & Consumers BoD EURORDIS/European Network for Ichthyosis - eni
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Presentations & Speakers
• Yann Le Cam, EURORDIS • Thomas Heynisch, European Commission, DG Enterprise • Wills Hughes-Wilson, Swedish Orphan-Biovitrum • Johan Van Calster, Clivan Bvba • Richard Bergström, EFPIA (oral presentation, please see Introduction)
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Yann LE E CAM Chief Ex xecutive Offficer of EUR RORDIS
Yann Y Le Cam m is a patien nts’ associattion advocatee who has deedicated 25 years of o professio onal and perrsonal comm mitment to hhealth and m medical research non-governm n mental organ nisations in France, Eurrope and thee United Stattes in the t fields of cancer, HIV V/AIDS and rare diseases. He H has threee daughters, the eldest oof whom hass cystic fibroosis. Yann is one of o the found ders of EURO ORDIS in 19996-1997 annd its Chief E Executive Officer since s 2002. He is the Vice V Chairm man of the E EU Committtee of Experrts on Rare Diseases since 2011. 2 He was one o of the firrst patient reepresentativees appointedd to the Com mmittee for O Orphan Mediicinal Products (COMP) att the Europeean Drug Ag gency (EMA A) where hee served 9 yyears and was its he Managem ment Board aand Executive Committtee of Vice Chaairman for 6 years. He served on th the Frencch HTA agency ANAES S now called d HAS for 5 years, on thhe DIA Advvisory Comm mittee Europe for fo 3 years, on o the EPPOSI Board forr 6 years. Yann is one of the founding members m of the t Internatiional Alliannce of Patiennts Organisaations (IAPO). Email: yann.lecam@ y @eurordis.org g
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www.eurordis.org
EURORDIS ROUND TABLE OF COMPANIES Corporate Responsibility in Improving Access to Orphan Medicinal Products
Brussels, 28 February 2013
www.eurordis.org
Improving Access to Orphan Medicinal Products: Gathering the Concepts & Proposals into a Comprehensive Approach Yann Le Cam Chief Executive Officer, EURORDIS Vice-Chair EU Committee of Experts on Rare Diseases, EUCERD
Brussels, 28 February 2013
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EURORDIS’ Objectives
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 3
ERTC Worskhop, 27 February 2013, Brussels
CONCEPTS
• RD Treatments Evidence Generation is a Continuum • Flexibility of Regulators 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
• Enhancing the Dialogue Between all Stakeholders all Along the Product Development & Life Cycle
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Rare Disease Treatments Evidence Generation is a Continuum ! ¾
Marketing Authorisation is not anymore an on/off switch
¾
Better and broader collection of relevant data
Data collected all along the life cycle of the medicine on risks as well as on benefits: ¾ Clinical trials ¾ Compassionate use ¾ Real life studies (actual heterogeneous population and real life constraints beyond clinical trials) ¾ Off label use
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Flexibility of Regulators Should Become an Official Policy ¾
Regulators are flexible (Based on last years experience of EMA and FDA) but need to say it clearly, so have better visibility, predictability, attractivity and better consistency of scientific opinions
¾
Regulators need to have a supportive approach: Being a Gate Keeper is not good enough + Regulators should be Partners for Successful developments
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¾
Conditional Approval
¾
Need for an intense roll-over process of Scientific Advice & Protocol Assistance before and after MA
¾
Adaptive clinical trial design
¾
Next: Progressive / Adaptive Licencing
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Focus on Effectiveness Beyond Quality, Safety and Efficacy • Dialogue between regulators (EMA), sponsors, medical experts, patient representatives to adapt Clinical Trial designs, as early as possible (ex: methodology in small population, de-link efficacy trials and safety trials, historical control)
• Anticipate more the therapeutic value demonstration (ex: registries, natural history, Good Clinical Practice Guideline on Diagnostic & Care) in Protocol Assistance (EMA)
• Early dialogue between regulators (EMA) and HTA (EUnetHTA) to adapt Post MA Research Plan
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Bridging the Gap Between EU Centralised Regulatory Decision and National Decisions on Pricing & Reimbursement
• One way or another, HTA and Payers need to be involved in all procedural aspects at the EMA to be well informed about the reality of medical needs, the potential and reality of the product, the uncertainties and the pathway to generate additional evidence for well targeted patients and good medical practices
• An approach on pricing based on Value, means a common understanding of what is Value and ealier marketing authorisation also means an understanding of this value as well as what the uncertainties are
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Enhancing the Dialogue Between all Stakeholders all Along the Product Development & Life Cycle
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¾
Shared-Value
¾
Corporate Responsibility
¾
Sustainability
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PROPOSALS
• Early Dialogue • Clinical Trials • Progressive / Adaptive Licensing • Call for a Stronger FDA – EMA Collaboration • CAVOMP • MOCA • Differential Pricing • National Measures in RD National Plans/ Strategies 10 ERTC Worskhop, 27 February 2013, Brussels
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Early Dialogue / Scoping / De-Risking • EU Pharmaceutical Forum's Guiding Principles on OMPs recommends early dialogue" • Corporate Responsibility's Mechanism of Coordinated Access to OMPs recommend early dialogue“ • Early dialogue = a dialogue, at a very early stage of development between 1 or more companies and all relevant stakeholders - EMA, HTA, Payers, Medical Experts, Patients -on a specific product, or on a specific disease, to discuss the potential to address an unmet medical need and the optimal research, regulatory, and health policy approach"
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Clinical Trials
• Clinical Trials Æ Ongoing: EU Regulation: European Expert Opinion Æ EMA: Adaptive clinical trial design
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Progressive / Adaptive Licensing
• Progressive / Adaptive Licensing Æ For diseases which are severe, with no alternative therapies or non-satisfactory therapies Æ Within current regulatory framework: • Conditional Approval • Progressive enlargement of targeted population treated based on hospital prescription & inclusion criteria •
Collection of data within post-MA research activities (safety, efficacy, effectiveness) including new pharmacovigilance legislation, risk management plan...
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Call for a Stronger FDA – EMA Collaboration: Beyond Orphan Drugs Designation ¾
Parallel Scientific Advice & Protocol Assistance
¾
Guidelines for methodology of clinical trials per disease or relevant group of diseases
¾
Sharing of File and Assessment at time of MA
¾
Mutual acceptance of data
¾
Coordination of Post-MA research plans
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CAVOMP: Four Time Points
1.
Early dialogue / Protocol Assistance
2.
Compilation Report & evidence definition / Evidence Generation Plan
3.
Follow-up of the evidence generation plan
4.
Assessment of Relative Effectiveness
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Assessment of Significant Benefit
Criterion of Significant Benefit Orphan Designation COMP
Significant Benefit COMP Protocol Assistance
Timepoint 1: Scientific advice through EMA / EUnetHTA coordination
CHMP Opinion T0
EC Marketing Authorisation T0 + 90 days
Timepoint 2: Compilation report & evidence generation plan
T0+∆T (time depending on the evidence generation plan)
Timepoint 4: Updated core HTA Timepoint 3: information For follow-up of the for the (relative) evidence generation plan effectiveness assessment
Time Information exchange and defining the evidence generation plan
Early Dialogue • • • • •
EMA EUnetHTA / payers Sponsor Patients Experts
• Could be implemented already
www.eucerd.eu
• • • •
EMA EUnetHTA / payers Sponsor Patients & treating physicians
• Could be implemented already
Evidence generation • • • •
EMA EUnetHTA / payers MAH Centres of Expertise (CE) & European Reference Networks (ERNs)
• Could be implemented already
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Corporate Responsibility in Improving Access to OMPs
Assessment • • • •
EUnetHTA / payers EMA MAH Patients & CEs/ERNs
• Adapted methodological tools for OMPs to be developed
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MOCA
• A Mechanism for Coordinated Access to Orphan Medicinal Products – Corporate Responsibility Pharmaceuticals
• Consensus: A European Transparent Value Framework • Opportunity: Price negotiation at European level based on Value (Common Assessment) + Volume (prevalence of therapeutic indication as defined in MA) + agreed Post-MA Research Activities
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Differential Pricing
• Differential pricing is already a reality in today's OMP European market with variations of costs actually paid by MSs varying +/- 10%
• Differential pricing for OMP can become a reality if associated with the negotiated / agreed price at European level
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19th Workshop of the Eurordis Round Table of Companies - February 27th, 2013 - Brussels
National Measures in RD National Plans /Strategies
•
EU legislation & European Volunteering collaborative approaches can work only if well "appropriated" by Member States and "translated" into national measures"
• National Plans / Strategies on Rare Diseases are an opportunity to embed these measures in Member States’ policy and organisation
19 ERTC Worskhop, 27 February 2013, Brussels
CONCLUSIONS
20 ERTC Worskhop, 27 February 2013, Brussels
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Lack of Common Understanding and Clarity across NCAs in MSs and Companies in Industry ¾
Price based on value vs price based on cost construction vs certain degree of cost transparency
¾
Condition Approval and Conditional Pricing means Conditional – conditions attached and revisions
¾
Gathering of expertise at EU level for discussion / opinion and national well-informed decision
¾
The opportunities vs the risks of a more European coordinated approach
¾
The volunteering process vs the legislative process
21 ERTC Worskhop, 27 February 2013, Brussels
THANK YOU
22 ERTC Worskhop, 27 February 2013, Brussels
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19th Workshop of the Eurordis Round Table of Companies - February 27th, 2013 - Brussels
Dr. Thom mas HEYNIISCH Deputy Head H of Uniit at the Eurropean Com mmission, D Directorate-G General En nterprise
Th homas Heynnisch is Deeputy Headd of Unit inn the Eu uropean Commission, Dirrectorate-Geeneral En nterprise sinnce 1 Septtember 2011. After haaving stu udied in G Germany, hee obtained a Doctoratte in Medicine M in 1992, folllowed up by studiees in Geeorgetown U University, Washingtonn DC until May 19 994 with the diplom ma “Masteer of Bussiness Ad dministratioon”. He then woorked in the Ph harmaceuticaal Industry uuntil Februarry 2000. ynisch joined the Eu uropean Co ommission, Directoratee-General E Environmentt, as Mr Hey Administtrator in Feb bruary 2000.. Since 2002 2 he works ffor the Direcctorate-Geneeral Enterpriise in the fieldss of Pharmaaceutical Ind dustry and Cosmetics C (22002-2005), Pharmaceuttical Industryy and Biotechn nology (2005 5-2010), and d Food and Healthcare H Inndustries, Biootechnologyy (as of 20100).
T
[email protected] uropa.eu Email: Thomas.Heyn
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19th Workshop of the Eurordis Round Table of Companies - February 27th, 2013 - Brussels
18th ERTC Workshop "Corporate Responsibility in Improving Access to OMPs" Brussels, 27 February 2013
Thomas Heynisch Deputy Head of Unit Food and Healthcare Industries, Biotechnology DG Enterprise and Industry
Process on Corporate Responsibility in the Field of Pharmaceuticals • It consists of three Platforms: 1. 2. 3.
Access to Medicines in Europe (6 sub‐groups) Access to Medicines in Africa and Ethics and Transparency
• Process launched by VP Tajani in September 2010 • All activities should be finalised by mid 2013 • Results to identify areas for further cooperation in our future industrial policy initiative (Commission Communication on Industrial Policy ‐ October 2012) 2
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Access to Medicines in Europe 6 Working Groups 1. 2. 3. 4. 5.
Access to biosimilars Co‐ordinated access to orphan medicinal products Managed entry agreements Facilitating supply in small markets Good governance for non‐prescription medicinal products 6. Priority medicines 3
Working group Mechanism of coordinated access to orphan medicinal products (MoCA‐OMP) •
Aim: find innovative ways to provide real access to orphan drugs for patients with unmet medical needs, for whom these solutions would otherwise be out of reach, in an affordable and sustainable way
•
Three workpackages: ‐ Assessing orphan medicinal products (Italy and EFPIA) ‐ Organising a structural access (Spain and ESIP) ‐ Organising the individual access (Belgium and Eurordis) 4
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19th Workshop of the Eurordis Round Table of Companies - February 27th, 2013 - Brussels
Working group Mechanism of coordinated access to orphan medicinal products (MoCA‐OMP) – state of play Report of the group being finalised: • the group agreed on the added value of a coordinated mechanism involving all stakeholders and using a European TVF, in view of delivering a report on the value of an OMP; • the group intends to propose an ad hoc taskforce with voluntary collaboration of EU/EEA countries and stakeholders. 5
Working group Mechanism of coordinated access to orphan medicinal products (MoCA‐OMP) – state of play Such a task force could help competent authorities to determine access conditions by: •
evaluating the unmet medical needs;
•
coordinating the preparatory steps (incl. horizon scanning, early involvement in the development of clinical development plans and patient registries);
•
testing the mechanism through a concrete pilot, applying a European TVF. 6
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19th Workshop of the Eurordis Round Table of Companies - February 27th, 2013 - Brussels
Future Future industrial policy in the field of pharmaceuticals will take into account the results of all working groups, to address in a comprehensive approach the current challenges in order to strengthen the competitiveness of the European pharmaceutical sector. 7
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Ms. Willls HUGHES S-WILSON Vice Preesident Exteernal Affairss & Chief Patient P Acceess Officer, Swedish Orphan Biiovitrum (So obi) ghes-Wilson is i Vice President Externa al Affairs & Chief Patien nt Access Offficer at Sobi and a Wills Hug member of o the globall Executive Management M Team. She jjoined Sobi inn February 20012. Hughes-W Wilson is resp ponsible for th he company’ss external liai son with all sstakeholders w working to suupport the availaability of treattments for raare diseases on o behalf of S Sobi; and for supporting thhe developmeent of sustainablle healthcare systems for raare disease diiagnosis, treattment and carre. Hughes-W Wilson is a member m of the t European Commissiion’s EU Coommittee of Experts on Rare Diseasees (EUCER RD), the EU U-level com mmittee offiicially responsiible for advissing the Europpean Commisssion on all asspects of implementing efffective rare ddisease policiies across Euurope. This inccludes key a spects aroundd reimbursedd access to oorphan drugs. She S is the spookesperson foor the industryy representatiion on this Com mmittee. She is an a industry representatiive on the w working grou up of European n pricing & reimburseme r ent authorities looking att access to orrphan drugs (“MOCA”). Hughes-W Wilson is also Chair of the Europea an Industry Task Forcee on Orphan n Drugs & Rare Diseases, a joint work king group th hat brings together at a Euuropean level the companiies involved in the research and a developm ment of orphan n drugs and trreatments for rare diseasess. Prior to joining Sobi, Hughes-Willson was the head of Heaalth- & Markket Access P Policy at Gennzyme or more than n 6 years. She S has led the Emerginng Biopharmaaceutical Entterprises (EB BE), a EMEA fo specialiseed group of the t European n Federation of Pharmaceeutical Indusstries & Assoociations (EF FPIA), representiing the interests of biotech hnology comp panies in Eurrope, as well as holding kkey positions in the animal heealth / veterinaary mediciness industry and d Ernst & Youung Consultinng. Hughes-W Wilson is an Honours H gradu uate in Law.
Email: Wills.Hughes W
[email protected]
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Corporate Responsibility in Improving Access to Orphan Medicinal Products National Measures to improve access to Orphan Medicinal Products in the context of National Plans
Wills Hughes‐Wilson, VP External Affairs & Chief Patient Access Officer, Sobi Chair, Joint European Industry Task Force on Orphan Medicinal Products & Rare Diseases EURORDIS Round‐Table of Companies (ERTC) Brussels, 27 February 2013
CONFIDENTIAL
Effective diagnosis, treatment & care is many‐faceted Diagnosis + Treatment
Optimising Care + Advancing the Field
Disease Management + Outcomes Development
Health System Access
Ongoing Mgt.
Revised Treatment Guidelines
Centres of Expertise Symptoms Awareness
Physicians Publications Monitoring
Outcomes Reporting
Testing Diagnosis + Staging Treatment
Label Updates Identification of Unmet Medical Need
Data Capture
2
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“…in order to aim to ensure that patients with rare disease have access to high‐quality care, including: • Diagnostic treatments; • Habilitation for those living with the disease; and, • If possible, effective orphan drugs”
• European countries committed to implement a National Plan for Rare Diseases by 2013 • Limited number of key actions • Sustainable, measurable, funded • Idea is that National Plans in the countries are similar – link up to each other
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Key elements of national plans to support access to Orphan Medicinal Products Key starting points of a National Plan to facilitate access to treatment & care 1. Multi‐stakeholder committee 2. Centralised National funding 3. Timely access to treatments 4. Early diagnosis 5. Centres of Expertise 5
Key starting points of a National Plan to facilitate access to treatment & care 1. Multi‐stakeholder committee
• All stakeholders, including industry, to bring expertise & guide decisions • Reflect national / societal wishes • Continuity & repository of information / expertise, even when government changes • Netherlands and Belgium steering groups on orphan drugs = successful examples 6
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Key starting points of a National Plan to facilitate access to treatment & care 2. Centralised National Funding
• Hospitals do not have to pay from individual budgets • Needs regular review – take account of new patients & treatments • In conjunction with multi‐stakeholder committees • Supports Centres of Excellence concept • If no Centre of Excellence, can be used to allow patients to travel to one where it does exist
7
Key starting points of a National Plan to facilitate access to treatment & care 3. Timely access to treatments
• Timely & equitable access for patients where treatment exists • Reimbursed, timely access before products officially authorised, where high unmet medical need • Budget impact – limited numbers of patients – can be predicted • Rule of rescue – solidarity • Societal aspect – not treating also has a cost 8
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Key starting points of a National Plan to facilitate access to treatment & care 4. Early Diagnosis
• Important element – diagnosis can take years; treatment outcomes are better where diagnosis is early • Building awareness with physicians • Early screening for diseases can be undertaken • Centres of Expertise to play a role
9
Key starting points of a National Plan to facilitate access to treatment & care 5. Centres of Expertise
• Underpin & support the successful creation of all other elements
10
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Overview of development of National Plans for Rare Diseases (1) Country
National Plan Status
Austria
In progress
Belgium
Adopted
Bulgaria
Adopted
Croatia
In progress
Cyprus
In progress
Czech Rep.
Adopted
Denmark
In progress
Estonia
In progress
Finland
In progress
Multi‐ stakeholder committee
Centralised national funding
Timely access to treatment
Overview of development of National Plans for Rare Diseases (2) Country
National Plan Status
France
Adopted
Germany
In progress
Greece
Adopted
Hungary
In progress
Ireland
In progress
Italy
In progress
Latvia
In progress
Lithuania
In progress
Luxemburg
In progress
Malta
In progress
Multi‐ stakeholder committee
Centralised national funding
Timely access to treatment
!! Indicative Snapshot !! Early diagnosis
Centres of expertise
!! Indicative Snapshot !! Early diagnosis
Corporate Responsibility in Improving Access to OMPs
Centres of expertise
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Overview of development of National Plans for Rare Diseases (3) Country
National Plan Status
Norway
In progress
Netherlands
In progress
Poland
In progress
Portugal
Adopted
Romania
In progress
Slovakia
In progress
Slovenia
Adopted
Spain
Adopted
Sweden
In progress
UK
In progress
Multi‐ stakeholder committee
Centralised national funding
!! Indicative Snapshot !!
Timely access to treatment
Early diagnosis
Centres of expertise
Development of National Plans for Rare Diseases – February 2013 FI RU NO SE
NP Adopted
EE LV
NP In progress
DK
LT
IE UK
No national plan
NL BE
Non-EU countries with NP in progress
PL DE CZ
LU FR
SK AT
HU
CH
RO
SL IT
HR BG
PT
ES EL CY
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OMPs are being developed & approved 120 100 80 60 40 20 0
OMPs removed from EU Community Register OMPs with EU Positive Opinion for Market Authorisation 15
* January 2013
The value of cooperation & collaboration • Where do bottlenecks still exist? • Existing acknowledgement that problems are shared / common • Collaborative approaches to identify pathways forward • “The specificities of rare diseases – limited number of patients and scarcity of relevant knowledge and expertise – single them out as a distinctive domain of very high European added‐value” 16
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Two key collaborative proposed approaches already • Mechanism of Coordinated Access to Orphan Medicinal Products (MOCA) • Pricing & reimbursement elements
• Clinical Added Value of Orphan Medicinal Products (CAVOMP) Information Flow • Up to and including Health Technology Assessment
17
Assessment of Significant Benefit
Criterion of Significant Benefit Orphan Designation COMP
Significant Benefit COMP Protocol Assistance
Timepoint 1: Scientific advice through EMA / EUnetHTA coordination
EC Marketing Authorisation T0 + 90 days
CHMP Opinion T0
Timepoint 2: Compilation report & evidence generation plan
T0+∆T (time depending on the evidence generation plan)
Timepoint 3: For follow-up of the evidence generation plan
Timepoint 4: Updated core HTA information for the (relative) effectiveness assessment
Evidence generation
Assessment
Time Information exchange and defining the evidence generation plan
Early Dialogue • • • • •
EMA EUnetHTA / payers Sponsor Patients Experts
• Could be implemented already
• • • •
EMA EUnetHTA / payers Sponsor Patients & treating physicians
• Could be implemented already
• • • •
EMA EUnetHTA / payers MAH Centres of Expertise (CE) & European Reference Networks (ERNs)
• Could be implemented already
www.eucerd.eu
Corporate Responsibility in Improving Access to OMPs
• • • •
EUnetHTA / payers EMA MAH Patients & CEs/ERNs
• Adapted methodological tools for OMPs to be developed
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Challenges are well‐documented; collaborative proposals are complementary / aligned
CAVOMP Early Dialogue
MOCA RD Classification
Horizon Scanning
Early Dialogue
Report
Evalu ‐ation
Evidence Generation
Advice included in Dev’t
Early Access Progs
P&R
Patient Access
Two different processes, two different reflections, have identified remarkably similar challenges; and have proposed approaches & suggestions for tackling them 19
Key actions needed; requiring concerted, collaborative & consistent stakeholder engagement EUnetHTA Appropriate Methodological Tools
CAVOMP Early Dialogue
MOCA RD Classification
Horizon Scanning
Early Dialogue
Report
Evidence Generation
Advice included in Dev’t
Evalu ‐ation
Early Access Progs
Transparent Value Framework
P&R
Patient Access
National Plans / Strategies for RD
20
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And implementation of other elements… • • • •
Registries European Reference Networks Cross‐Border Healthcare Directive …
EUnetHTA Appropriate Methodological Tools
CAVOMP Early Dialogue
MOCA RD Classification
Horizon Scanning
Early Dialogue
Report
Evalu ‐ation
Evidence Generation
Advice included in Dev’t
Early Access Progs
P&R
Transparent Value Framework
Patient Access
National Plans / Strategies for RD
21
This system would offer Member States a sustainable way to understand & handle OMPs New OMP
What we know – Dosing – Pricing – Patient Population – Label • • • •
Adjustment
Conditional Access
Centres of Expertise (Regulatory) tools exist Need new tools? Conditional reimbursement?
Feedback
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CONFIDENTIAL What do we need in National Plans? Effective diagnosis, treatment & care is many‐faceted
Collaborative efforts Collaborative efforts on Disease Management + on Optimising Care + Pricing Approaches value‐identification Advancing the Field
Diagnosis + Treatment Guidelines on Screening?
Outcomes Development
Formal Ongoing guidelines on Mgt. Centres of Expertise
Collaborative efforts on HTA / Assessments
Health System Access
Revised Treatment Guidelines
Centres of Expertise Symptoms Awareness
Physicians Publications Monitoring
Outcomes Reporting
Testing Diagnosis + Staging Treatment
23
Registries guidelines & collaboration
Label Updates Identification of Unmet Medical Need
Data Capture
Collaborative projects around data‐ capture
National measures to improve access to Orphan Medicinal Products in the context of National Plans • Effective diagnosis • Effective treatment and care • On the foundation of continued European cooperation in rare diseases AND… • Continue in the collaborative dialogue around the real challenges that remain for OMP access • Build the elements that are needed to address these remaining challenges into the National Plans (collaboratively)
24
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A renewed EU strategy 2011‐14 for Corporate Social Responsibility • Enterprises should “have in place a process to integrate societal, environmental, ethical and human rights concerns into their business operations and core strategy in close collaboration with their stakeholders”
25
A need to work together in rare diseases since the beginning
Joint European Industry Task Force on Orphan Drugs & Rare Diseases
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Let’s keep it up also when it comes to improving the situation around access questions… …to create National Plans that will truly deliver Thank you! 27
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Johan VAN V CALST TER t Belgian n Mediciness Agency aand special advisor too Fipra forr the Former Head of the Differential Pricing project
ohan Van Calster, C MScc. Pharm., is the form mer Head oof the Jo Beelgian Mediicines Agenncy and speccial advisor to Fipra foor the Diifferential Pricing P projject. He is currently A Administrator at Cllivan Bvba in the Poliicy and Govvernment A Affairs Officce for Medicinal M Pro oducts.
Cllivan Bvba focuses f on iinnovative pproducts, bioopharmaceutticals, orphan drugs, d medicinal productts from human blood an d plasma, ceells and tissuues, implantts and invasive medical dev vices. ohan@clivan n.be Email: jo
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Differential pricing of medicines for unmet medical needs
Johan Van Calster 27-02-2013 ERTC Workshop (With courtesy to Jo De Cock)
BELGIAN PRESIDENCY 2010
ERTC February 27, 2013
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COUNCIL CONCLUSIONS ON INNOVATION AND SOLIDARITY IN PHARMACEUITCALS
3 ERTC February 27, 2013
COUNCIL CONCLUSIONS 6.12.2010 (NR.24) • Examine, based on the principles of solidarity, economically viable and efficient approaches to facilitate availability and access to valuable innovative medicinal products throughout the EU, while respecting the principle of subsidiarity and the competencies of Member States, e,g, on affordability and sustainability of health systems,
ERTC February 27, 2013
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ANALYSIS OF 2011 SALES FOR 11 VALUABLE MEDICINES LAUNCHED IN THE EU IN THE PERIOD 2005- 2007
ERTC February 27, 2013 5
A QUESTION OF SOLIDARITY
• The sense of fairness and equity between Member States is being eroded. And without equity between Member States, how can there be equity between European citizens – State of the union, 11 sept 2012, by Jose Manuel Barroso, President of the European Commission
ERTC February 27, 2013
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CROSS-COUNTRY EUROPEAN INITIATIVES 1. CAVOD – Clinical Added Value of Orphan Drugs • •
Information needed to make (informed) decisions Approaches to understand what that information means
2. MOCA – Mechanism of Coordinated Access to Orphan Drugs • Way to understand and make value judgments at time of pricing & reimbursement • Project managed by Belgium (NIHDI) • Final report published I/2013 ERTC February 27, 2013
7
ERTC February 27, 2013
8
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INFORMAL HIGH LEVEL STAKEHOLDER WORKING GROUP
- Chair : Mr. John BOWIS – OBE - Composed of individual members from : * European Commission * High Level Patient Organisations * Permanent Representatives–Health Attachés * Member States Payer Organisations * Health Economists * Industry ERTC February 27, 2013
9
REFLECTION ON DIFFERENTIAL PRICING • Scope: unmet medical needs • Problem statement: – Equal prices in all countries imply unequal access • Differential pricing: – The basic objective of price differentiation is to improve equality of access to innovative pharmaceuticals for unmet medical needs across Europe – Price reflects ability/willingness to pay of a country ERTC February 27, 2013
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19th Workshop of the Eurordis Round Table of Companies - February 27th, 2013 - Brussels
CODE OF CONDUCT – SOFT LAW APPROACH FOR ACCESS TO INNOVATIVE DRUGS • VOLUNTARY BASIS, protocol agreement between INDUSTRY and MEMBER STATES • SCOPE for the application of the principle : “VALUABLE MEDICINES” – High added value – Unmet medical need – Market Authorization granted
ERTC February 27, 2013
11
CODE OF CONDUCT – SOFT LAW APPROACH FOR ACCESS TO INNOVATIVE DRUGS • “LIKE FOR LIKE” Principle – PRICING • Transparent Ex factory price • Confidentiality of net prices • Price differentiation (no IRP nor ERP) • According to ability and willingness to pay (incremental and marginal value) – WEIGHING • Clustering of countries with similar GDP, average income per capita • No linking to % spent on healthcare • Prevalence of disease • Elasticity of the demand ERTC February 27, 2013
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CODE OF CONDUCT – SOFT LAW APPROACH FOR ACCESS TO INNOVATIVE DRUGS • TRANSPARENCY OF TIMELINES : European Market Entry Plan (max. 2 years from MA) • CURRENCY STABILITY : prices in EURO - 24 months fixed exchange rates • EXTERNAL VERIFICATION BODY : monitoring of differential pricing system by Trusted Third Party with at least one representative of each cluster ERTC February 27, 2013
13
PRICING AND REIMBURSEMENT
• Pricing and reimbursement remains a matter of individual countries – Countries can still negotiate the price with companies – Practice of some countries to award reimbursement based on cost-effectiveness (and other) criteria may inform establishment of a pricing system that reflects value for money – Member States and industry partners agree on the principle of confidentiality of net prices
ERTC February 27, 2013
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EXTERNAL REFERENCE PRICING (ERP)
• ERP is not compatible with differential pricing – Prices determined according to prices in other countries rather than ability/willingness to pay of a country • Participating countries need to agree not to implement ERP as a method of setting prices
ERTC February 27, 2013
15
PARALLEL TRADE
• Would lead to unwillingness of companies to step into the process, as ROI seriously reduced by parallel trade • Introduction of possibility for public health exceptions for parallel trade : supply allocation schemes
ERTC February 27, 2013
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TRANSPARENT MARKET ENTRY PLAN
• Commitment of participating countries to apply differential pricing principles • Commitment of companies to launch medicines within 2 years of Market Authorization (MA)
ERTC February 27, 2013
17
THANK YOU!
ERTC February 27, 2013
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Report of the Breakout Sessions
y National Measures y Differential Pricing y Mechanism of Coordinated Access to OMPs
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Report of the Breakout Sessions
Break-out sessions to discuss the three main topics of the morning followed with feedback from each group. Feedback from each of these groups is summarised below:
1/ National Measures: What are the measures included in the National Plans for Rare Diseases that should be promoted as priorities in order to improve access to OMPs? 1. What should be requested? a. A National Champion needs to be appointed at the Ministry made fully & transparently accountable – best type of person possible b. Sufficient resource allocation – no reference to any of the costs of the recommendations c. Could exists already – this is the set of resources that are identified as the responsibility / accountability of this person, how they are allocated d. Indicators from EUROPLAN should be included in the National Plans 2. How to Make it Happen a. EUROPLAN: need to have a benchmark sharing between the different countries b. Find a mechanism on how to give feedback about the status of the National Plans • e.g., where does the UK National Plan stand? “Black hole” c. Need to have a committed & effective national / local champion & coordinator •
e.g., the difference a person can make
3. What happens from 2014? a. EUCERD •
Benchmarking regularly with the EUCERD – successfully moving forward
• Initiate moves now to ensure continuity as of beginning of 2014 – status & action; make a formal report of status?
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b. European Commission will do a formal follow-up to the Member States, asking them the status •
Should be included with the Patient groups with informal commentary to supplement
•
Use EURORDISCare Survey
4. Recommendations – how to make it happen a. European Parliament to follow up? Ask them to do a follow up on the political support that they gave to the National Plans b. “Get it on the Presidency Agenda” – upcoming strategic Presidencies c. OVERALL OBJECTIVE – to keep it high on political agenda, to maintain priority and momentum
2/ Differential Pricing: Is the approach proposed in the Non Paper on Differential Pricing sufficient for improving access to OMPs and under which conditions? 1. Differentiated pricing is an important long-term objective to enable access 2. The approach should remain voluntary; it remains a 'soft law' initiative which requires no legislative change. The following stakeholder positions were identified as important: •
Commission: there is moral support from DG Enterprise and DG Sanco The position of the Guardian of the Treaty ( DG Comp) remains uncertain
•
Member States agreement and political endorsement is vital, given national competence on pricing
•
There must be an appetite from industry
• Payers must be engaged early in the process to get them on board; 3. An agreed entry point should be decided; a pilot scheme could be either with the bail-out countries or for orphan drugs It was proposed that a differentiated approach could be piloted for orphan drugs by introducing it into MoCA, under the Tajani initiative 4. It is important to consider the alternatives which provide access - these are rebates and contractual agreements
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It was pointed out that rebates are now subject to being used in reference pricing systems. The confidentiality of net prices would be a central element of a differentiated approach to pricing 5. Conclusions: It was proposed that these key points be communicated Jo de Cock's group and an advocacy plan be developed, incorporating a pilot.
3/ Mechanism of Coordinated Access to OMPs: What are the key success factors necessary for making the Common Assessment of the Value of OMP based on the European Transparent Value Framework work? •
Discussion focused on Transparent Value Framework
•
Criteria presented on the certainty of evidence, relative effectiveness, relative response and value
•
Should the degree of rarity be used as a criterion – no consensus has been reached on this question.
•
When no comparator exists, quality of life becomes an even more important factor
•
Is the TVF used for the price of the medicinal product or the value?
•
The decision remains with the member state
The afternoon session began with a discussion from Richard Bergström on the challenges of access in countries most affected by the crisis, which was followed by a panel discussion including a patients’ representative from the Greek Rare Disease Alliance, Dimitrios Synodinos, and two members from the pharmaceutical industry, Mohit Jain (BioMarin) and Kevin Loth (Celgene). Dimitrios highlighted the issues in Greece and the potential causes for these problems, some of which are highlighted in the concept paper for this meeting. Despite expenditure for health still being at 9.5% of the current budget, patients are still suffering from lack of access to care and medication. They are choosing either to forego treatment or pay for their own treatment, if they have the means. Thus, an intense day of discussions and brainstorming concluded another successful ERTC workshop with great speakers and excellent input from the participants. We are certain to be hearing more about these topics as the deadline for the national plans approaches and both the Differential Pricing and the Mechanism of Coordinated Access documents are finalised. No doubt another ERTC workshop will focus on pushing forward measures to improve access to medicinal products for rare disease patients.
Corporate Responsibility in Improving Access to OMPs
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