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Overview

Overview. 1 : Legal Framework 2 : Review of applications received until now 3 : Points of concern. 1 :Legal Framework. 1.1 : Regulation ( EC ) 726/2004 Art. 83 : Compassionate Use

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Overview

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  1. 1

  2. Overview 1 : Legal Framework 2 : Review of applications received until now 3 : Points of concern 2

  3. 1 :Legal Framework • 1.1 : Regulation ( EC ) 726/2004 • Art. 83 : Compassionate Use • «  making a medicinal product available for CU reasons to a group of patients with a chronically or seriously delibilating disease or whose disease is considered to be life threatening and who cannot be treated satisfactorily by an authorised medicinal product . » • -> The medicinal product must be subject of a MA application or must be ondergoing a Clinical Trial 3

  4. 1 :Legal Framework 1.2 : CHMP Guideline on CU of medicinal products pursuant to (EC) 726/ 2004 Article 83 ( adopted 19 July 2007 ) • Compassionate use implementation remains a MS’s competenceArt.83 is complementary to national legislations and provide an option to MS who wish to receive a CHMP opinion • The medicinal product is either the subject of an application for a centralised marketing autorisation or is undergoing clinical trials in the EU and/or elsewhere • - Patients should always be considered for inclusion in clinical trials before being offered compassionate use programs 4

  5. - In this guideline compassionate use does not refer to the use of an authorised medicinal product for an indication different from the one mentioned in the SPC - Other Principles and definitions - Initiation and request of CHMP opinion - CHMP opinion itself : grounds for assessing a request ; documentation to be supplied ; Pharmacovigilance - Link with MA 1.2 : CHMP Guideline on CU of medicinal products pursuant to (EC) 726/ 2004 Article 83 5

  6. 1.3 : National Law on medicinal products :1 May 2006 • Compassionate Use : • For medicinal products without a MA • Medical Need Program : • For medicinal products with a MA for a certain indication 6

  7. 3 : National Law on medicinal products :1 May 2006 • Compassionate Use : • Art.6 quater point 2 • Articles 106 and 107 in the RD executive measures of the Law 1 May 2006 • Medical Need Program : • Art.6 quater point 3 • Articles 108 and 109 in the RD executive measures of the Law 1 May 2006 7

  8. 3 : National Law on medicinal products :1 May 2006 • Medical Need Program : Art.6 quater point 3 : • Application for MA for the indication envisaged is ongoing • or • MA for the indication envisaged has been granted but is not yet available on the market for the indication envisaged • or • Clinical trials arestill runningor clinical trials have been performed prooving the feasability of the MP for the indication envisaged 8

  9. 1.4: RD executive measures (Law 01.05.06) • Compassionate Use : • Art. 106 : Establishing a general program : • Positive advice of the EC mandatory • - Inclusion criteria for acceptance of patients • - Indication • - Timespan in which the program will be running • - Distribution ( modalities , costs , accountability ) • - Informed Consent • - data as required by the EU NfG 9

  10. 1.4: RD executive measures (Law 01.05.06) • Compassionate Use : • Art. 106 : Establishing a general program : • Application to the CA ( DGMP : Department R&D ) • - same documentation including the positive advice of EC • - data and formulars according to EU NfG • CA evaluates whether a CHMP opinion is to be requested or not •  If yes : CHMP advice to be followed •  If not : tacit approval after 2 weeks period • - Archiving : copy of documents for a 10 years period 10

  11. Compassionate Use : Art. 106 : Establishing a general program : In case of emergency :  Application to CA and EC justifying the urgency of the case 1.4: RD executive measures (Law 01.05.06) 11

  12. 1.4: RD executive measures (Law 01.05.06) • Compassionate Use : • Art. 107 : Acceptance of Patiënts • Treating physician sends a request per patiënt , declaring that : • - he takes the responsability for the use of this MP ( not yet authorised • - chronic , life threatening disease , no alternative MP available on the Belgian market to treat the disease ; he describes the disease • - he will inform the patiënt completely of all the modalities of the program • - he will asap ask the patiënt for informed consent 12

  13. Compassionate Use : Art. 107 : Acceptance of Patiënts - The company verifies the conformity to the program for each request and informs the applicant asap . - negative outcome : rationale is explained - positive outcome : the MP is provided to the treating physician according the established modalities and he labels the MP «  compassionate use / not for sale ) - documents prooving that the treating physician has respected his duties are to be archived for 10 years. 1.4: RD executive measures (Law 01.05.06) 13

  14. 1.4: RD executive measures (Law 01.05.06) • Medical Need Program : • Art.108 : Establishing a general program : • - Notification to EC : • - Inclusion criteria for acceptance of patients • - Indication • - Timespan in which the program will be running • - Distribution ( modalities , costs , accountability ) • - Informed Consent 14

  15. 1.4: RD executive measures (Law 01.05.06) • Medical Need Program : • Art.108 : Establishing a general program : • - Notification to CA ( FAMHP: Department R&D ) : • including the name of the EC concerned • - Tacit approval aswell for EC as CA after a period of time of 2 weeks 15

  16. 1.4: RD executive measures (Law 01.05.06) • Medical Need Program : • Art.108 : Establishing a general program : • MAH informs the treating physician of the existence of the Medical Need Program and its conditions for application • - Copy of documents to be archived by MAH to proove conformity to the Law/RD for a period of 10 years 16

  17. 1.4: RD executive measures (Law 01.05.06) • Medical Need Program : • Art.108 : Establishing a general program : • In case of emergency : •  Application to CA and EC justifying the urgency of the case 17

  18. 1.4: RD executive measures (Law 01.05.06) • Medical Need Program : • Art. 109 : Acceptance of Patiënts • Treating physician sends a request per patiënt , declaring that: • - he takes the responsability for the use of this MP • ( not yet authorised ) • - chronic , life threatening disease , no alternative MP available on the Belgian market to treat the disease ; he describes the disease • - he will inform the patiënt completely of all the modalities of the program • - he will asap ask the patiënt for informed consent 18

  19. 1.4: RD executive measures (Law 01.05.06) • Medical Need Program : • Art. 109 : Acceptance of Patiënts • - The company verifies the conformity to the program for each request and informs the applicant asap . • - negative outcome : rationale is explained • - positive outcome : the MP is provided to the treating physician according the established modalities and he labels the MP «  compassionate use / not for sale ) - documents prooving that the treating physician has respected his duties are to be archived for 10 years. 19

  20. 2.1 :Compassionate use : * Number of applications received :only 8  urgent cases ( one patient )  one program with positive advice of EC ( orphan drug ) * Questions raised :  one patient programs  CU programs requested for registered drugs ( incorrect comprehension of the definition ; is improving with time … but still a concern ) 2: Applications received until now 20

  21. 2.2 : Medical Need Program : * Number of applications received : 17  Only 7 out of 17 with notified EC approval  Data obtained from the “ Literature “ : considered as “invalid“ Questions raised :  patients included in the original clinical trial but not fulfilling the re-imbursement criteria of RIZIV/INAMI 2: Applications received until now 21

  22. 2.2 : Medical Need Program :  legal framework too limitative ( data from the literature not retained as “ 4th criterium for MNP “ current position FAMHP : stimulate application for MA orphan drug / off-label use to be rediscussed with new government ? 2: Applications received until now 22

  23. * Need for information / communication towards EC’s and health care professionals * Request to EC’s to notify the FAMHP for outcome decision related to approval CU-MNP via ct.ec@fagg-afmps.be * Enhanced scientific interaction between EC-CA about the justification of existing alternative therapies for instance or in case of negative opinion of EMEA * Notification to CHMP and role of CHMP ( need for harmonisation for CU programs in patients view perspective ) * Publication of register of approved CU ? * Follow-up of inclusion of patients in a CU/MNP program? * Amendments of CU/MNP programs ? * Safety reporting 3 : Other points of concern 23

  24. * Suggestions :  All applications / advices introduced via EC to be reported to CA via ct.ec@fagg.be  Inventory of practical cases by EC’s  Specific attention for 3th criterium for MNP: feasability of enrigestered drug to be prooved by CT in the new indication envisaged …  Template for CU/MNP to be provided by sponsors ? ( Informed Consent ; MNP ; Commitment of the treating physician )  Prospectives ..?  Inventory of problems as experienced by sponsors … 4:Measures for improvement..? 24

  25. Many thanks for your attention 25

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