Overview
This presentation is the property of its rightful owner.
Sponsored Links
1 / 25

Overview PowerPoint PPT Presentation


  • 114 Views
  • Uploaded on
  • Presentation posted in: General

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

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.While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server.


- - - - - - - - - - - - - - - - - - - - - - - - - - E N D - - - - - - - - - - - - - - - - - - - - - - - - - -

Presentation Transcript


Overview

1


Overview

Overview

1 : Legal Framework

2 : Review of applications received until now

3 : Points of concern

2


1 legal framework

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


1 legal framework1

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


1 2 chmp guideline on cu of medicinal products pursuant to ec 726 2004 article 83

- 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


Overview

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


Overview

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


Overview

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


Overview

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


Overview

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


1 4 rd executive measures law 01 05 06

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


1 4 rd executive measures law 01 05 061

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


1 4 rd executive measures law 01 05 062

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


1 4 rd executive measures law 01 05 063

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


1 4 rd executive measures law 01 05 064

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


1 4 rd executive measures law 01 05 065

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


1 4 rd executive measures law 01 05 066

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


1 4 rd executive measures law 01 05 067

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


1 4 rd executive measures law 01 05 068

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


2 applications received until now

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


2 applications received until now1

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


2 applications received until now2

2.2 : Medical Need Program :

 legal framework too limitative ( data from the literature not retained as “ 4th criterium for MNP “ current position FAMHP : stimulate applicationfor MA orphan drug / off-label use

to be rediscussed with new government ?

2: Applications received until now

22


3 other points of concern

* 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 [email protected]

* 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


4 measures for improvement

* Suggestions :

 All applications / advices introduced via EC to be reported to CA via [email protected]

 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


Overview

Many thanks for your attention

25


  • Login