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Kerstin Westermark Prof., Senior expert, Medical Products Agency, Sweden Chair, Committee for Orphan Medicinal Products,

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Kerstin Westermark Prof., Senior expert, Medical Products Agency, Sweden Chair, Committee for Orphan Medicinal Products,

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    1. 1 Kerstin Westermark Prof., Senior expert, Medical Products Agency, Sweden Chair, Committee for Orphan Medicinal Products, EMEA Treatment of Rare Diseases and EU Legislation Prag, May 21, 2009 "Role of COMP and support of research and development of drugs with orphan medicinal product designation in the EU"

    2. Who wants to be an Orphan?

    3. Why an Orphan Regulation? Regulation (EC) No 141/2000 of the European Parliament and of the Council on Orphan Medicinal Products of 16 December 1999 “Persons suffering from rare conditions should be entitled to the same quality of treatment as other patients” But… “ the pharmaceutical industry would be unwilling to develop the medicinal product under normal market conditions” As… “some conditions occur so infrequently that the cost of developing and bringing to the market a medicinal product (…) would not be recovered by the expected sales”

    4. Which Incentives in EU for Orphan designated Medicinal Products (OMP)? Market Exclusivity in the EU - 10 years for all orphan medicines (from marketing authorisation) + 2 years if Paediatrics Investigational Plan (PIP) results included in the MA and reflected in the SPC Fee reductions for OMP development Application for OMP Designation: free of charge Protocol assistance and follow up from the EMEA: free of charge Application for Marketing Authorisation: free of charge for Small and Medium-sized Enterprises (from 2009) + Extended incentives for SMEs in post authorisation EU marketing authorisation through unique centralised procedure Priority to EU Research - Framework programs Access to Member States Incentives (EC inventory)

    5. EMEA European Medicines Agency (EMEA) Coordinates evaluation and supervision of medicinal products throughout the EU Brings together scientific resources of the 27 EU Member States – network of the national competent authorities Has around 100 working groups/parties and access to a network of c:a 4000 European experts Works through 6 Committees: - CHMP Committee for Medicinal Products for Human Use - COMP Committee for Orphan Medicinal Products - PDCO Paediatric Committee - CAT Committee for Advanced Therapies - HMPC Herbal Medicinal products Committee - CVMP Committee for Veterinary Medicinal Products The committees give Opinions – The EU Commission gives Decisions

    6. Committee for Orphan MPs (COMP)

    7. Tasks of the COMP Examine applications for designation of a medicinal product as an orphan medicinal product (OMP) Assist the Commission in drawing up guidelines Assist the Commission in liaising internationally and with patient support groups Advise the Commission on a policy on orphan medicinal products for the EU

    8. Examine applications for designation of a medicinal product as an orphan medicinal product (OMP) What was expected in year 2000? C:a 15 applications Four-five1 day meetings/year What was the result? More than 100 applications/year Eleven 2-day meetings/year

    9. Criteria for Orphan Designation SERIOUS CONDITIONS (life threatening, chronically disabling) RARE < 5/10.000 individuals in the EU or LACK OF RETURN ON INVESTMENT NO SATISFACTORY TREATMENT or "SIGNIFICANT BENEFIT" (A clinically relevant advantage or a major contribution to patient care)

    10. Protocol assistance Protocol assistance ? scientific advice for companies developing OMPs Questions on quality-efficacy-safety Questions on significant benefit Company position required SAWP (Scientific Advice Working Party) provides answers; COMP represented by 3 members CHMP adopts answers COMP involved/adopts answers on significant benefit Follow-up Protocol Assistance possible

    11. Protocol assistance - improved chances for a successful marketing authorisation application! Orphan Drug development requires: Flexibility at Regulatory level Potential for alternative approaches Strive for efficiency But - No new methods for rare disorders

    13. Guidance Documents (cont'd) Points to consider on the calculation and reporting of the prevalence of a condition for orphan designation (EMEA/COMP/436/01) Guideline on clinical trials in small populations (CHMP/EWP/83561/2005) + several CHMP – guidelines (general and disease/drug specific)

    15. EMEA Marketing Authorisation – Centralised procedure Mandatory for orphan designated products One single marketing authorisation application to the EMEA A single evaluation by the Committee for medicinal products for Human Use (CHMP) If quality, safety and efficacy sufficiently proven – positive opinion Opinion sent to EU Commission – transformation into a single marketing authorisation valid for the whole of EU

    16. 10 (+2) years of Market exclusivity Protection against similar products (structure/mechanism of action) for same indication Three derogations Sponsor’s consent Lack of supply Clinical superiority

    17. International Co-operation (Confidentiality arrangements existing) Facilitating Cooperative Efforts on the Orphan Regulatory Process EU (EC and EMEA) and the US FDA/DHHS (actions ongoing) EU (EC and EMEA) and Japan MHLW/PMDA (actions planned) EU (EC and EMEA) and Health Canada (actions started)

    18. Since November 2007 Common Application Form EMEA/COMP – FDA/OOPD for Orphan Medicinal Product designation A common form to simplify administrative process First step in harmonising administrative practices BUT Different regulations and procedures: - Different criteria/requirements (prevalence threshold, significant benefit) - Different approaches (definition of condition) - No single opinion EU and US

    19. EMEA/COMP-FDA/OOPD (currently ongoing/planned) Regular contacts – monthly teleconferences EMEA-FDA: Exchange of information on ongoing orphan designation application procedures between the two agencies Annual reports on development: Timeline and structure harmonisation Guidelines on orphan designation: Existing EMEA/COMP guidelines Proposal for future discussion and harmonisation of common terms

    20. TOMORROW: Facilitating Cooperative Efforts on the Orphan Regulatory Process – The Global Approach Creating an international forum for discussion on the harmonisation of terminology and concepts related to designation Sharing international expertise and discussing divergent opinions Creating an international network of experts to be used for designation Discussing a global approach for stimulation of research linked to orphan designation Global administrative harmonisation

    21. EU Orphan regulation 141/2000 – A SUCCESS STORY Before the EU - 27 different marketing approval procedures for every new drug Before the EU Orphan Regulation – almost no development of drugs for rare diseases/conditions Today, after more than 9 years of the Orphan Regulation – 937 applications for orphan designation/ 648 positive opinions/ 231 withdrawals/ 13 negative opinions Today, after more than 9 years of the Orphan Regulation - 53 marketing authorisations for orphan drugs - via centralised procedure = 1 procedure for 27 Member States Update May 6, 2009

    22. EU Orphan Regulation 141/2000 - Opportunities for patients with rare conditions Potential benefits for 30 milj. European patients with rare conditions Potential benefits for neglected diseases, e.g. tbc, malaria, leishmaniasis – rare in the EU – prevalent in the rest of the world To date, 53 orphan medicinal products have been given marketing authorisation, a main part innovative drugs from micro-, small and medium sized enterprises (e.g. gene therapy for SCID) Potential benefit for the understanding of and drug development for common diseases "Learning from the Rare" (William Harvey) but Challenges Profitability – Costs - Avalibility

    23. How can COMP contribute- What's on/What's next? COMP members in Scientific Advice Working Party (SAWP) "full advice" – not only significant benefit questions Increased collaboration/harmonisation between EMEA committees (COMP- CHMP-PDCO-CAT) Increased transparency in COMP opinions - COMP role in scientific assessment of "clinical added value" of OMPs Increasing role of COMP visavi stakeholders (patient organisations, industry, health care professionals/academia) – regular meetings/focus groups/conferences COMP members in EU Commission Rare Disease Task Force (RDTF) – future Committee of Experts on Rare Diseases (CERD) COMP as advisors to the EU Commission (DG Enterprise/Sanco/Research (Rare diseases FP7 priority: Support fro clincial trials in phase I-II in designated OMPs – 2009? COMP members as Member States "ambassadeurs" for Rare Diseases/Orphan Drugs (National plans; National registries etc.)

    24. From challenge to solution? 4th Eurordis' survey on OMPs availability in the EU (F.Bignami, MEDEV meeting, Brussels, Jan. 2008)

    25. The way forward Conclusions and Recommendations from the High Level Pharmaceutical Forum (Oct. 2, 2008) http://europa.eu/pharmaforum/docs/final_conclusions_en.pdf - Commission Communication and Recommendation on Rare Diseases; "Rare diseases - Europe's challenges" (Nov. 11, 2008) http://ec.europa.eu/health/ph_threats/non_com/rare_10_en.htm Proposal for a COUNCIL RECOMMENDATION on a European action in the field of rare diseases http://ec.europa.eu/health/ph_threats/non_com/docs/rare_rec_en.pdf

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