La réglementation européenne pour les médicaments orphelins. De Europese regelgeving voor weesgeneesmiddelen.

Transcription

1 (AFMPS) La réglementation européenne pour les médicaments orphelins. De Europese regelgeving voor weesgeneesmiddelen. Dr. André LHOIR AFMPS - COMP 22 février /2000: Rare conditions Orphan drugs «RARE and EQUAL» patients entitled to same quality of treatment need for incentives (see other regions) cost of developing versus expected sales EU level - EMA patients representatives patients deserve same quality, safety & efficacy in MPs OMPs submitted to normal evaluation process fee reduction or waiver; exclusivity 2 1

2 Permission asked to Philippe Geluck. 3 Orphan Medicinal Products (1/2) Regulation 141/2000; Jan 2000, Avril 2000 For human use Prevalence: not more than 5 in in EU Life-threatening or chronically debilitating Significant benefit if other treatments exist Cave: sub-setting Condition: as broad as possible Several designations for the same product: OK 4 2

3 Orphan Medicinal Products (2/2) EMA procedure mandatory Protocol assistance free of charge Fee reduction 10 years market exclusivity years exclusivity if PIP finalised!!! For non-orphan: patent + 6 months if PIP finalised!!! Currently: > 800 designations and > 60 MA Nationaly: INAMI/RIZIV: Specific rules 5 Orphan Condition versus Therapeutic Indication (1/2) Condition: any deviation(s) from the natural structure or function of the body, as manifested by a characteristic set of signs and symptoms (typically a recognised distinct disease or a syndrome). Orphan Condition: the condition as defined above that meets the criteria defined in Art. 3 of Reg (EC) 141/200; prevalence, severity, chronicity Orphan Indication: specifies if the medicinal product which is the subject of the designation application is intended for diagnosis, prevention or treatment of the Orphan Condition. 6 3

4 Orphan Condition versus Therapeutic Indication (2/2) Therapeutic Indication The granted therapeutic indication at the time of marketing authorisation will be the result of the assessment of the quality, safety and efficacy data submitted with the marketing application and may be different to, but within, the one granted at the time of designation. 7 Prevalence 12% 36% less than 1 in 10,000 52% between 1 and 3 in 10,000 more than 3 in 10,000 EMEA, update 1 october

5 Therapeutic domains (designation) musculoskeletal and nervous system 12% other 9% immunology 10% metabolism 11% antiinfectious 4% oncology 44% cardiovascular and respiratory 10% immunology cardiovascular and respiratory metabolism other oncology antiinfectious musculoskeletal and nervous system EMEA, update 1 october Marketing authorisations 12% 4% 9% 38% 14% 23% antineoplastic and immunomodulating agents metabolism blood musculoskeletal and nervous system cardiovascular others EMEA, update 1 october

6 Paediatrics versus Adults Both 45% Adult 46% Paediatric 9% EMEA, update 1 october Marketing authorisation (MA) Centralised procedure mandatory Same criteria as non-orphan Quality, Safety, Efficacy 210 days or accelerated procedure Small populations Exceptional circumstances Conditional approvals Revue of orphan criteria (COMP) 12 6

7 Post-marketing obligations Risk minimisation plan View on safety, communication, distribution Registries: linked to MA Post marketing studies 13 Community Register for OMPs Commission obligation Published on Commission website Policy of transparency Parallels the register for authorisations and FDA approach Link with MA when granted 14 7

8 Removal from the Register Only under specific conditions: At the request of the sponsor Criteria no longer met (at the time of MA) At the end of period of market exclusivity 15 Merci pour votre attention Bedankt voor u aandacht 16 8