Joint Rare Diseases / Orphan Medicinal Products Task Force


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Orphan Café, 8 december 2011

Presentation by Lugdivine Le Dez (Alexion) & Laura Gutierrez (Celgene)

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Joint Rare Diseases / Orphan Medicinal Products Task Force

  1. 1. JOINT RARE DISEASES / ORPHANMEDICINAL PRODUCTSTASK FORCEA collaborative initiative with national trade associations andpolicy developments at EU level.Lugdivine Le Dez (Alexion)Laura Gutierrez (Celgene)
  2. 2. AgendaI. Presentation of the joint taskforceII. Cooperation with national OD groups:◦ Objectives, rationale, proposal, expectedoutcomesIII. Highlight of EU policy developments
  3. 3. I. Presentation of the jointtaskforce
  4. 4. European member companies of all sizes thathave either developed or intend to developorphan drugsMembers of EBE and/or EuropaBio,4 Meetings per yearName of active member companies: Actelion, Alexion,Astra Zeneca,Biomarin, Celgene, CSL Berhing, Dompé, Genesis Pharma, Genzyme,GSK, Novartis, Novo Nordisk, Merk Serono, Orphan Europe, PharmaMar, Pfizer, Shire.47companiesWhat is the Joint Task Force?
  5. 5. Objectives of theJoint Task Force Contribute to the development of EU rare diseases-related policiesand regulatory frameworks Work towards a harmonised implementation of EU legislation andregulatory guidelines for OMPs Improve timely access to therapies for all rare disease patientsacross the EU Raise awareness about the economic and social value of OMPs
  6. 6. Why the EU matters?• Member States’ individual responsibilities: Health care financing Pricing and reimbursement of drugs• Influences the political agenda of the individual EUcountries: Laboratory of ideas Meeting point of national authorities: Exchange ofbest /worst practices among Member States• Regulation of the pharmaceutical sector: Marketing Authorizations Clinical Trials rules Orphan Drugs Regulation (ME) Intellectual Property (Data protection, SPC)
  7. 7. Joint taskforce 2011-2012 priorities• Industry input in the CAVOD process and Mechanism ofcoordinated access to ODs• Exceptions for OMPs both at EU and national level and establishlinks with national trade association.• National Plans for Rare DiseasesACCESSTOOMPs FORPATIENTS• Build tools to communicate the values and specificities of theOMP business models• Implementing a tailored outreach (educational) programme mainlytargetted at EU audiencesVALUE ANDREPUTATION OFTHE RAREDISEASEBUSINESSMODEL• Ensure that the EU legislative and regulatory frameworks continueto be appropriate and predictable for OMP developers• Ensure that a consistent framework for registries is implementedat national levelLEGISLATIVEANDREGULATORYASPECTS OFOMPs
  8. 8. II. Cooperation with nationalOD groupsA collaborative approach with national tradeassociations
  9. 9. Creating interactionEU OrphanDrugTaskforceNationaltradeassociations
  10. 10. Main objectives- Enhancing cross-communication- Information sharing / best practice sharing- Supporting local efforts/crisis whenneeded
  11. 11. • EuropeanCommission• EuropeanParliament• EU CouncilEURegulators • EMEA/COMPOtherStakeholders• Patient group• Healthcareprofessionals• AcademiaNationaltradeassociationsAn new partnerEUinstitutionsJoint EBE-EuropaBioTask Force
  12. 12. Rationale- Relevance of MS policy work on EU policy and viceversa- Dominoes effect of local policy/cost containmentmeasures on other MS- Common objective to orphan drug policy- Unstable EU economic/financial environments needingsustained policies for ODs
  13. 13. Interaction processEU Orphan DrugTaskforce National trade associations• Share with you with tools wedeveloped to promote the value ofODs, and punctual EU policyupdates• Organise a yearly “get together” inBrussels with all national relevantleaders to share best practices andalign on common goals.• Share information about majornational political and legislativedecisions that can have an impacton orphan medicines in yourcountry but also in the rest ofEurope• Share tools/concepts• Send your group representative tothe yearly meeting in Brussels• Ensure an industry unified approach to OD policydevelopments• Ensure EU support is provided when requested toreinforce local lobby• Ensure all parties (EU/Local) are ready to react in time
  14. 14. Tools to update you on ongoing EUwork and to promote national plans
  15. 15. Tools to communicate OD specificities
  16. 16. The budget impact isestimated to have grown from0% in 2000 to 3.3% by 2010From 2010 to 2016, the rateof growth is likely to slowand will reach a plateau ofapproximately 4.6%3.3%4.6%Orphan Drug Budget ImpactThe budget impact of orphan medicines in Europe has grown steadily over the 10 yearssince the introduction of the orphan drug legislation in 2000, but growth is predicted toslow and plateau over the next decade
  17. 17. III. Key highlight of EU policydevelopments
  18. 18. Highlights of EU ongoing policydevelopmentsEUpolicyFollow up toimplementationof NationalPlansCAVODMOCAClinicalTrialsDirectiveTransparencyDirective (P&R)Directive onpatients’ rightsin cross-borderhealthcareEUCERD
  19. 19. Highlights of EU ongoing policydevelopmentsEUpolicyFollow up toimplementationof NationalPlansCAVODMOCAClinicalTrialsDirectiveTransparencyDirective (P&R)Directive onpatients’ rightsin cross-borderhealthcareEUCERD
  20. 20. Council Directive 89/105/EEC on P&Rprocesses– “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 medicinalproducts (60/120 days).- HTA in the scope- Results of the marketing authorization (quality, safety, efficacy, bioequivalence) shallnot be reassessed in HTA decisions
  21. 21.  Concerns Instrumentalisation by Member States to facilitate cost-containmentmeasures and increased international reference pricing Demands by European Parliament for harmonisation of pricesCouncil Directive 89/105/EEC on P&Rprocesses– “Transparency” Directive
  22. 22. Highlights of EU ongoing policydevelopmentsEUpolicyFollow up toimplementationof NationalPlansCAVODMOCAClinicalTrialsDirectiveTransparencyDirective (P&R)Directive onpatients’ rightsin cross-borderhealthcareEUCERD
  23. 23. Background: Clinical Added-Value of OrphanDrugs (CAVOD)Initiated by Eurordis, included in the recommendations ofHigh Level Pharmaceutical Forum◦ Possible exhange of knowledge on the scientific assessment ofthe clinical added value of orphan drugs (CAVOD) Original objectives – speed patients access◦ Make the most of evidence on Clinical AddedValue of OrphanDrugs gathered by EMA◦ Coordinate payers requirements for additional data European Commission commissioned E&Y to identifyimplementation options
  24. 24. Main purpose of theCAVOD study PURPOSE OF THE STUDY• Identify and assess the possible options for the creationof a mechanism for the exchange of knowledge betweenMember States and European authorities on the scientificassessment of the relative effectiveness of orphan medicines (tobe implemented, if possible, from 2011) PURPOSE OF THE MECHANISM• To facilitate MS informed decision on the scientificassessment of the clinical effectiveness of an orphan drug• To contribute to the development of a continuum between pre-market authorization practices (clinical development) at EUlevel and post-marketing authorizations practices at memberstate level• SOURCE: European Commission
  25. 25. CAVOD: a potential tool for betteraccess to orphan drugs Based on the initial opinion of the COMP and CHMP at the time oforphan designation, which includes an evaluation of the significant clinical benefit of an OMP –which is confirmed at time of MA
  26. 26. CAVOD:The E&Y report A state of play of regulatory processessand HTAs for ODs in Europe. An identification of the data collectionwhich could be accepable for assesingrelative effectiveness of ODs. A proposal for a collaboration mechanismbetween regulatory and HTA bodies forOD assessment.
  27. 27. Sources: Draft Backgroung review, EUnetHTA JA WP5: Relative Effectiveness Assessment (REA) of Pharmaceuticals; April 2011EUnetHTA 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 modelto be particularly considered as being part ofrare diseases’ specificity► The type of evidences (1) covered by the clinical added value are based onthe EUnetHTA JA WP4 & WP5 & HTA core model and adapted to thespecificity 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 aspectsData to measure the CAVODalready consideredas part of EMA scope
  28. 28. E&Y: Interaction processEUnetHTA-CAVOD/EMA► The objective of the interaction between EUnetHTA-CAVOD and EMA is to build a bridge anddevelop a continuum between pre-market authorization practices (clinical development) at EUlevel and post-marketing authorizations practices at member state level.► Through this process, the National HTA bodies through EUnetHTA are engaged collectively andbring their experience together to give inputs and valuable requirement to the EMA prior to theRisk management plan.► Member states will be able to contribute to the Risk management plan.Evidencerequirementgathering fromHTA bodiesWriting ofrecommendationpaperIdentificationof the projectmanager PMEUnetHTA-CAVODExchange ofinformation withthe rapporteur ofOD at the CHMPMarketing Autorisation review process, prior to CHMP opinionExchange of information withEUnetHTA-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 drugRecommendationpaper for EMA* Discussion to considerat the CHMP committeeincluding EUnetHTA-CAVOD PM ?
  29. 29. CAVOD:The E&Y ReportTimeCHMP opinion, T0EC marketingauthorisationT0 + 90 daysT0+ΔT(after 3 to 5 years, flexibledepending of the disease)Period 1:for EMA / EUnetHTAcoordinationPeriod 2:for simpleCompilationreport &evidencegeneration planPeriod 3:for follow up of theevidence generation planPeriod 4:relativeeffectivenessassessmentProtocolassistanceSignificant Benefit, COMP
  30. 30. E&Y report: Concerns Involvement of EUnetHTA, namely:◦ Involvement of HTA bodies in the regulatoryprocess -> impact on MA.◦ Interactions between EMA and HTA not welldefined  additional hurdles for access?◦ Lack of expertise in OD of HTA bodies.
  31. 31. CAVOD: Next steps Rare Diseases stakeholder community• Industry comments under development Dec2012• EUCERD drafting group• Workshop tentatively scheduled for March 2nd2012• EUCERD opinion document to be adoptedJune 2012
  32. 32. Highlights of EU ongoing policydevelopmentsEUpolicyFollow up toimplementationof NationalPlansCAVODMOCAClinicalTrialsDirectiveTransparencyDirective (P&R)Directive onpatients’ rightsin cross-borderhealthcareEUCERD
  33. 33. Mechanism of coordinated access to ODs-Background Sept 2010: Launch of “Tajani initiative” including allrelevant stakeholders in pharmaceutical sector. 3 platforms aimed at developing recommendationsfocusing on specific issues:◦ Ethics andTransparency◦ Access to medicines in the least developed countries◦ Access to medicines in Europe, including a platform onMechanism of coordinated access to orphan medicinalproducts.
  34. 34. 34Mechanism of cooordinated accessto ODs Project vision: The coordination between stakeholders andMember States at EU level to provide real access to a realsolutions (orphan medicinal products) for realpatients with real unmet medical needs, for which thesesolutions would otherwise be out of reach – in an affordableand sustainable way (-> “real life access”). Project objective: Designing a concrete operational manualof coordinated real access for stakeholders and MemberStates to achieve the vision irrespective of the localconditions starting with the identification of the unmetmedical need and the possible answers (phase zero).
  35. 35. 35Mechanism of coordinated acces to ODs -Project MethodologyMechanism of coordinated access toorphan medicinal productsWP1Identifying andassessing a relevantorphan drug(Assessment/evaluative)WP2Selection of targetpopulation andmechanism of funding(Structural access)WP3Treatment(Individual access)#1operational step#2operational step#1operational step#2operational step#2operational step#1operational step#1impl.activity#2impl.activity
  36. 36. Reminder Objective: an industry unified approachwhen dealing with Orphan Drug Policy Proposed next steps:◦ Sharing of messages/tools◦ Regular updates◦ Yearly meeting (date tbd) Your feedback on cooperation?
  37. 37. Thank you for your attention!
  38. 38. Back-up slides
  39. 39. Directive on patients’ rights incross-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 October2013 to implement it The EP managed to include rare diseases in the scope of the directive, which had been opposedfor 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 amountof 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 developmentof 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 andtreatment 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 patientscountry of residence and vice versa)
  40. 40. WP 1 WP 2 WP 3BELGIUM Ri De Ridder 2Francis Arickx 1 2 3Catherine Adriaens 1 2Marleen Mortier 2Céline HermansMireille PierletDiane Kleinermans 3AUSTRIA Christine Leopold 3Florian BachnerESTONIA Dagmar Ruutel 1FINLAND Sari EkholmJyrki Vanakoski 1FRANCE Danielle GolinelliPierre PribilePatrick Cayer Barrioz 1 2 3Arlette Meyer 1 2Catherine ChomaNadine DavidRenaud MorinGREECE Lena KatsomitiHUNGARY M SzabadosMi PalosiClaudia Habl 1 2 3Sabine Vogler
  41. 41. ITALY Pietro Folino GalloPaolo Siviero 2MALTA Jennifer Farrugia 1Isabelle Zahra-PulisNETHERLANDSHugo HurtsHuub KooijmanHJJ SeeverensPOLAND Jakub Adamski 2PORTUGAL Ines Ramos 2SPAIN Mercedes VallejoMartinez2SWEDEN Anna-Marta StenbergMaria Storey 1
  42. 42. EFPIA Katia Finck 2 3Wills Hughes-Wilson 1Ana Palma 1Kevin Loth 1Adam Heathfield 2 3Laura Gutierrez 1Enda Scott 2 3Thomas CueniFrançois BouvyEGA Greg PerryIlina MarkovaEuropaBio Ludovic LacaineGIRP Monica Derecque-PoisMartin Fitzgerald 2 3EPF Yann LeCam 1 3Ariane WeinmanFlaminia Macchia 1 3AIM Heidi Goethals 2ESIP Anna Bucsics 2Hans Seyfried 2CPME Oscar Arias 3Birgit BegerSynnove Lindemalm 1 3
  43. 43. COMP Elodie CarmonaDominik SchnichelsSANCO Jerome BoehmAnders Lamark TysseNathalie ChazePatricia BrunkoMirjam SoderholmMARKET Jean BergevinJan Willem VerheijdenENTERPRISE 1 2 3Thomas HeynischChristophe RoelandValerie VanhoeckGiulia DelBrennaLaura NistorAurelie VandeputteEminet 1 2 3