ORPHAN DESIGNATION BY THE EMA. Paillard Juliette M2 AREIPS 15/11/2016

Transcription

1 ORPHAN DESIGNATION BY THE EMA Paillard Juliette M2 AREIPS 15/11/2016

2 Legal basis 1. Legal background 2. Criteria SUMMARY Procedure 1. Prior to the submission 2. Submission and validation 3. Evaluation and opinion 4. Follow-up Incentives offered by the status 1. Protocole assistance 2. Market exclusivity 3. Other incentives

3 Introduction

4 Rare disease = Life-threatening or chronically debilitating condition affecting no more than 5/10000 people in the EU Between 5000 and 8000 different rare diseases affecting 6 to 8% of the population 5 new ones described every week 80% with a genetic origin

5 Legal basis

6 1. Legal background Regulation No 141/2000: -Community procedure -Incentives for development and marketing authorisation -COMP Regulation No 726/2004: -Centralised authorisation procedure mandatory for orphan medicine Regulation No 507/2006: -Conditional marketing authorisation

7 2. Criteria -Life-threatening/debilitating nature of the condition -Prevalence < 5/10000 (US définition: < people) or -Unlikely that marketing the product, without incentives, would generate sufficient return to justify the necessary investment -No satisfactory method of diagnosis, prevention,treatment exists or if it does, the medicinal product should be of a significant benefit to those affected by the condition

8 Procedure

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10 1. Prior to the submission Pre-submission meeting: Review of the documents provided by the sponsor: draft of the EMA application form (or common FDA/EMA form) draft of the scientific sections A to E short powerpoint about the application EMA encourages parallel applications for orphan designation with its international partners, for a global development of medicines for rare diseases. Appointment of coordinators: 1 COMP member + 1 EMA scientific officer

11 2. Submission and validation Submission: deadlines on the EMA website Commission guideline (ENTR/6283/00) for the format and content of applications Cover letter EMA application form (or common FDA/EMA AF) Scientific sections A-E Proof of establishment of the sponsor in the EU Translation of the name of the product and the proposed orphan indication Bibliography Validation: EMA checks whether : Indication acceptable for the designation Medical plausibility justified with relevant data in a non-clinical model/in patients Methodology/conclusion for the prevalence is provided Significant benefit demonstrated

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13 3. Evaluation and opinion Evaluation: -EMA coordinator + COMP coordinator + appointed experts -Summary report about the application sent to the COMP members for comments -Discussion of the COMP (D60): positive opinion/list of questions -Sponsor respond in writing/ oral explanation at the next COMP meeting Opinion: -Within 90 days: consensus or by majority of 2/3 of COMP members -COMP meeting

14 4. Follow-up Follow-up: -Opinion published in the COMP monthly reports (EMA website) within 1 week -COMP opinion forwarded to the EC for designation process and to the sponsor Appeal: -Grounds must be forwarded within 90 days at receipt of the opinion Decision: -Adopted by the EC within 30 days of the COMP opinion -Public summary of the decision published on the EMA website Publication: in the Community Register of Orphan Medicinal Products once the marketing exclusivity is approved for the product

15 Incentives offered by the status

16 1. Protocol assistance -Scientific advice specific for orphan drugs -Available at reduced charge for designated orphan medicines (free for SMEs) -May be requested at any time of the development of the medicine -No restriction on the number of times a sponsor can request protocol assistance -Given by the CHMP on the recommendation of the SAWP -Parallel scientific advice with the FDA is available

17 2. Market exclusivity (1/3) = Protection against the market competition with similar product for the same indication -Orphan medicine: from 10 years of marketing exclusivity after the MA approval Awarded by the EC Specifically linked to an orphan designation for a specific condition -If PIP: 2 years more of market exclusivity After a compliance check from the Paediatric Committee and opinion from the CHMP.

18 2. Market exclusivity (2/3) Orphan Regulation (EC) No 141/2000: Where a MA for an orphan medicinal product is granted, the Agency and the member state shall not, for 10 years : accept another application for MA, grant a MA, accept an application to extend an existing MA for the same indication in respect of a similar product. Commission Regulation (EC) No 847/2000: Similar medicinal product Similar active substance(s) for the same therapeutic indication Similar active substance -Identical active substance -Substance with the same principal molecular structural features, acting via the same MOA

19 2. Market exclusivity (3/3) The MA may be granted, for the same indication, to a similar product only if: The holder of the MA for the original orphan medicinal product has given his consent to the second applicant The holder is unable to supply a sufficient quantities of the medicinal product The 2nd applicant established in the application that his product is safer, more effective or clinically superior

20 3. Other incentives Access to the centralised authorisation procedure Possible conditional approval Additional incentives for SMEs Administrative and procedural assistance from the Agency's SME office Incentives in member states Fundings From the European Commission and other sources

21 Conclusion

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23 Thanks for your attention! Any question?