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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
Federal Agency for Medicines and Health Products
(FAMHP)
Compassionate Use and Medical Need Program
Pharma.be-BeApp , Brussels
16 November 2007
Greet MUSCHHead of R&D
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
Overview
1 : Legal Framework
2 : Review of applications received until now
3 : Points of concern
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
1 :Legal Framework1.2 : 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
competence Art.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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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 ongoingor
• MA for the indication envisaged has been granted but is not yet available on the market for the indication envisagedor
• Clinical trials are still running or clinical trials have been performed prooving the feasability of the MP for the indication envisaged
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
1.4: RD executive measures (Law 01.05.06)
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.
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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.
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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 )
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
2: Applications received until now
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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
2: Applications received until now
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 ?
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
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 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
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FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS
4: Measures for improvement..?
* 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 …
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