1 / 25

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

gitel
Download Presentation

Overview

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript


  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

More Related