Revising the Pharmaceutical Landscape in the EU

Transcription

1 Revising the Pharmaceutical Landscape in the EU The Industry Perspective Dr. Christine-Lise Julou June

2 Revising the pharmaceutical landscape in Europe Preamble New Legislation Other regulatory initiatives including harmonisation initiatives (harmonisation) Considerations in relation to other legislation 2

3 EFPIA s mission and priorities Improving the competitiveness of the pharmaceutical industry in Europe by setting up a regulatory and political environment, which above all stimulates R&D and rewards innovation. This will guarantee industry s continuous quest for better therapies and enable it to meet the growing healthcare expectations of today s and tomorrow s patients. 3

4 Pharmaceutical R&D expenditure (million national currency units*) Europe USA Japan 12,0 10,0 8,0 R&D expenditure Annual growth rate (%) 8,5 10,7 7,5 10,7 6, % 6,0 4,0 5,1 Europe USA * National currency units: Europe: million; USA: $ million; Japan: million x 100 Data 2007: estimate EFPIA & PhRMA Source: EFPIA member associations, PhRMA, JPMA Changes in research sites: data relate to 22 global companies Source: IMI (EFPIA Research Directors Group & IFPMA) 2,0 0,

5 New Molecular Entities Europe USA Japan Others Source: SCRIP - EFPIA calculations (according to nationality of mother company) 5

6 Estimated full cost of bringing a new chemical or biological entity to market ($ million year 2005 $) $ m illion Year Source: J.A. Di Masi and H.G. Grabowski, The Cost of Biopharmaceutical R&D: Is biotech Different? Managerial and Decision Economics 28 (2007):

7 Allocation of R&D investments by function 100% 90% 80% 70% 60% 50% 40% 30% 20% 10% 0% 27,2% 6,7% 13,1% 28,0% 6,1% 12,9% 6,0% 1 Pre-human/Pre-clinical Clinical trials (Phase I) Clinical trials (Phase II) Clinical trials (Phase III) Approval Pharmacovigilance (IV) Uncategorized Source: PhRMA, Annual Membership Survey 2008 (percentages calculated from 2006 data) 7

8 Years R&D: Scientific Risk Discovery and Development of a Successful NCE Introduction/Registration Post-Marketing Surveillance Clinical Tests (Humans) Development 5-10 Preclinical Tests (Animals) Basic Research 3,000 10,000 Quantity of Substances 1 Phases IV III II I Source: Based on PhRMA analysis, updated for data per Tufts Center for the Study of Drug Development (CSDD) database. 8

9 ROUTE OF A NEW SUBSTANCE FROM DISCOVERY TO PATIENT S ACCESS 9 Patent application Pharmacology Phase I clinical trials Chronic toxicity Acute toxicity Phase III Registration and transparency Phase II Price Pharmacovigilance Reimbursement 1 medicinal product 10 years of research 5 years 10 years 15 years 20 years Patent expiry 2 to 3 years of administrative procedures

10 Better Regulation Initiative and Revision of the Legislation Regulatory framework on changes to medicinal products Legislative proposals to strengthen and rationalise the EU system of pharmacovigilance 10

11 EU System of Pharmacovigilance Overall EFPIA considers the draft proposals to be a valuable and important step forward in rationalising/simplifying and strengthening pharmacovigilance. Particularly welcome a number of initiatives such as single point reporting to Eudravigilance, implementation of a Pharmacovigilance System Master File electronic submission of PSURs. 11

12 EU System of Pharmacovigilance Legislative proposals also raised several issues in particular in relation to Proposal to establish a Committee on Pharmacovigilance (Role & responsibilities of this Committee) Applicability of the requirements regarding the submission of PSURs (EFPIA does not believe this should depend on whether a marketing authorisation was granted based on a certain application type (e.g. generic, herbals, well-established use, biosimilar, etc.) Addition of a new key safety information section to the SmPC and Patient Information leaflet. 12

13 EU System of Pharmacovigilance Other issues raised by the proposals Management and presentation of the proposed European list of medicines under intensive monitoring; addition of specific text on outer box of medicines/package leaflet in this regards Publication of list of QPs Inclusion of ADR form within packages (EFPIA suggests that more efficient and possibly more effective alternatives are considered) 13

14 EU System of Pharmacovigilance Need to dissipate concerns/clarify misunderstanding in relation to: Suggestion that non-serious ADRs that occur in the Community might have to be subject to expedited reporting Suggestion that adverse reactions that lack causality assessments might have to be reported Scope of definition of Post-Authorisation Safety Studies 14

15 EU System of Pharmacovigilance It will be crucial to ensure enforcement of submission to Eudravigilance alone throughout all Member States and a single Pharmacovigilance System Master File (No imposition of additional national requirements); also acceptability of a Single Risk Management Plan. Striving for consistency through a single piece of legislation to allow industry to proceed with greater clarity, save time and deploy resources in a more efficient manner. Safety Communication and transparency. Consistency of the info made available in different databases Info assessed and made available in an appropriate manner (i.e. can be readily and accurately interpreted by the public including its limitations and constraints.) 15

16 Variations Regulation Regulation has the potential to significantly lower the current regulatory burden for introducing changes and/or updating information. EFPIA particularly welcomes: Principle of consistency in requirement applicable throughout the EU Grouping & worksharing Concept of definition of principles based on which variations categories are laid down and development of guidelines on the details of the various categories to bring further predictability while making regular updates in the light of scientific and technical progress, taking in particular account of developments regarding international harmonisation easier. 16

17 Variations Regulation Will need some reassurance in relation to implementation of Regulation with respect to timelines (e.g. acknowledgement of receipt of a valid notification, information as to whether the variation is accepted or rejected, amendments to the decision granting the marketing authorisation, completion of the procedure provided for in Article 10) Acceptance of minor variations of type IB Use of the clause that a decision or approval cannot be recognised on grounds of a potential serious risk to public health 17

18 Variations Regulation Provisions on grouping and worksharing appear to be applicable only in cases where all concerned marketing authorisations are held by the same Marketing Authorisation Holder: this restriction will have an impact in cases of licensing agreement for same product or MA is held by different affiliates from same group of companies 18

19 Variations Regulation Guidelines on the details of the various categories of variations and on the operation of the procedures laid down in the new Commission Regulation will be of critical importance 19

20 Transatlantic Administrative Simplification Harmonisation between US and EU risk management plans for innovative medicines (single global RMP; harmonisation of the documentation to be submitted) Surrogate endpoints/biomarkers Aligning and sharing results from the Critical Path and Innovative Medicines initiatives. Parallel Scientific Advice Facilitating and streamlining global development of new paediatric indications (exchange of information and explore possible harmonisation of template requirements and timings in relation to PIPs and Paediatric Study Requests 20

21 Transatlantic Administrative Simplification Collaboration on inspections and acceptance of results Acceptance and planning of 3 rd country inspections Possibility to avoid re-testing of medicinal products imported from the US (seek appropriate arrangement between EU and USA pursuant to Art 51.2 of Directive 2001/83/EC) 21

22 Transatlantic Administrative Simplification Certificate of Pharmaceutical Products Increase exchange of information on counterfeit medicines; harmonised approach to anticounterfeiting initiatives, including e-pedigree systems and technologies Common application format to facilitate parallel submission for orphan designation in EU and USA 22

23 ICH Adoption of important new guidance documents in the quality, efficacy/pharmacovigilance and safety area. 23

24 Upcoming legislation EFPIA submitted responses to public consultation on preparation of legal proposal to combat counterfeit medicines for human use (key ideas for better protection of patients against the risk of counterfeit medicines) and the key elements of a legal proposal on information to patients (detailed responses on Pharmacos ) 24

25 Information to Patients A few guiding principles Overcoming inequalities in accessing high quality information; improving public health Pharmaceutical companies have unique disease expertise and know their medicines better than anyone else The doctor/patient interaction remains crucial Information which is provided unsolicited to the public should be limited to general health information (e.g. prevention, awareness) but not mentioning specific medicines However when citizens/patients actively seek information companies should be able to provide highquality medicines information 25

26 Counterfeit medicines EFPIA overall supports proposed principles Tightening requirements for manufacture, placing on the market of medicinal products and inspections Tightening requirement for the import /export/transit of medicinal products Tightening requirements for manufacture, placing on the market of active substances and inspections EFPIA considers it is critical to ensure that the integrity of the original package is guaranteed throughout the entire supply chain, from manufacturer to end user. 26

27 Revising the Pharmaceutical Landscape Other legislation Regulation (EC) No 1901/2006 on medicinal products for paediatric use Regulation (EC) No 1394/2007 on advanced medicinal products Directive 2001/20/EC «Clinical Trial Directive» 27

28 Implementation of recently adopted legislation Paediatric Regulation: ongoing Concerns in relation to the following matters: Interpretation of the deadline end of pharmacokinetic studies in adults provided for in the Regulation It has been suggested that obligations in relation to paediatric indications/pip had no linkage with adult indications. Pragmatism and clarity are required in relation to linkage to adult indication Compliance checks and linkage with national patent offices. 28

29 Implementation of recently adopted legislation Regulation on advanced therapy medicinal products: Revision of Annex I to Directive 2001/83/EC as modified: overall welcomed however new definition of Gene Therapy product raises concerns (broadened scope) 29

30 Regulatory framework for clinical trial research Key issues pertain to difference in interpretation of legislation in different Member States, national requirements, inconsistency of amendment notification, repeated assessment of the science and methodology by competent authorities, central and local institutional ethics committees Recital 10 of Directive 2001/20/EC stipulated that delays and complications were detrimental to effective conduct of clinical trials in the Community Europe needs a regulatory framework which is conducive of effective conduct of clinical trials and trusted by all stakeholders 30

31 Conclusions Revising the pharmaceutical landscapes is of critical importance; very encouraging steps have been taken; need to continuously improve and adapt to foster a favourable environment in Europe which encourages innovation and makes Europe attractive to invest in pharmaceutical R&D and production and serve public health. 31

32 Additional slides 32

33 Sector R&D investment as % of all sectors 2006 Rest of 22 Sectors Food producers Household goods Oil & gas producers Health care equipment & services Fixed line teleco mmunicatio ns General industrials Industrial engineering Leisure goods Aerospace & Defence Chemicals Software & Computer Services Electro nic & electrical equipment Automobiles & parts Techno lo gy hardware & equipment Pharmaceuticals & Biotechnology 1,1 1,1 1,3 1,8 2,0 2,4 2,5 3,9 4,4 4,7 6,6 7,3 7,4 16,6 17,6 19, Note: Industrial Classification Benchmark set up by Financial Times Stock Exchange (FTSE) & Dow Jones Data relate to the top 1,400 companies with registered offices in the EU, Japan, the USA and the Rest of the World, ranked by the size of their R&D investment (over 23 million) (over million) Source: The 2007 EU industrial R&D investment scoreboard, Joint Research Centre, Directorate General Research, European Commission 33

34 R&D Expenditure as a percentage of GDP (2006) Austria Belgium Bulgaria Cyprus Czech Rep. Denmark Estonia Finland France Germany Greece Hungary Ireland Italy Latvia Lithuania Luxembourg M alta Netherlands Norway Poland Portugal Romania Slovakia Slovenia Spain Sweden Switzerland U.K. EU-27 Croatia Iceland Turkey China Japan United States 0,48 0,42 0,57 1,00 1,32 1,10 0,69 0,80 1,47 0,55 1,72 1,51 0,56 0,81 0,46 0,49 1,59 1,16 0,87 0,79 1,14 1,34 1,54 1,83 1,76 1,84 2,12 2,45 2,43 2,51 2,62 2,93 2,83 3,45 3,33 3,82 0,00 0,50 1,00 1,50 2,00 2,50 3,00 3,50 4,00 4,50 Note: Iceland, Switzerland: 2004 data; China, Italy, Japan, Portugal, Turkey, United Kingdom, USA: 2005 data; EU-27: estimate Austria, Cyprus, Denmark, estonia, France, Germany, Greece, Luxembourg, Malta, Netherlands, Slovenia, Spain: provisional data Source: EUROSTAT new release , 10 March 2008; Science, technology and innovation in Europe, 2008 edition, EUROSTAT 34