Presentation of the GMC

Dieter Deforce

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- Ensuring, the quality, safety and efficacy of medicines for

human use

- Help in a timely access to new therapies (Unmet Medical Need),

with best interest of patient in mind

- Advisory commission

Mission

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The new commission: legislative framework

Advices given by Commission:

Law on medicines adapted:

Royal Decree of 14th december 2006 on medicinal products for

human and veterinary use

Change regarding to advices given: optional advice for variations

focus on critical dossiers only, to allow scientific discussion

where needed

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The new commission: competences

Changes: Extension of competences:

Previous competences:

• Marketing authorisations (MA)

• Compassionate use and Medical need programs (Unmet Need) (UMN)

• Safety reports

• Risk management plans, risk minimisation activities

• Scientific Advice on general matters

New Competences:

• Clinical Trial applications (CTA, informal)

• Scientific technical advice (STA, informal)

• Advanced Therapeutic Medicinal Products (ATMP’s) - Hospitalexemption

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Not the remit of the commission

- pricing/reimbursement

- Marketing- “commissie van toezicht op de reclame”

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Commission – FAGG/FAMPH

- FAGG/FAMPH staff prepares reports and presents to

commission

- For each report a “coodinator” within the commission is

appointed

- Discussion @commission:

- Members and FAGG staff

- Report is accepted/amended by the commission

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The new commission: composition

8 effective members– right to vote

Each member has an alternate

Additional members can be co-opted

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Prof. Dr. Yves Horsmans

UCL

Vice-president

Gastro-entérologie, Hépatologie, Tumeurs hépato-bilio-pancréatiques,

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Prof. Dr. Joeri Aerts

VUB

Member

immunotherapeutic approaches for treatment of cancer and HIV infection.

10

Prof. Isabel Spriet

UZ Leuven

Member

Hospital Pharmacist special interest in antimicrobial pharmacokinetics/pharmacodynamics.

11

Prof. Dr. Karel Allegaert

KULeuven

Member

clinical pharmacology in pregnancy and in paediatrics; neonatal and pediatric intensive care

12

Prof. Jutte van der Werff ten Bosch

UZ VUB

Member

pediatric hemato-oncology and immunology

13

Prof. Dr. Jean-Marie Colet

Université de Mons

Member

preclinical and clinical toxicology

14

PharmD. Manon Gillet

CHU Liège

Member

Pharmacien hospitalier clinicien

15

Prof. Dr. An De Sutter

UZGent

Co-opted member

family doctor with special interest in evidence based prescribing of medicines in primary care.

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Prof. Dr. Bert Bravenboer

UZ Brussel

Alternate Member

all-round general internist with special expertise in endocrinology and clinical pharmacology.

17

Prof. Kristien De Paepe

VUB

Alternate Member

Pharmacist, Technology and Compounding, dosage forms, product formulation and excipients, patient communication

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Prof. Gert Matthijs

KULeuven

Alternate Member

Human genetics, molecular testing and diagnosis

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Prof. Paul Declerck

KULeuven

Alternate Member

Pharmaceutical quality, biotechnological drugs, monoclonal antibodies, biosimilars

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Prof. Patrice Forget

VUB, UZBrussel

Alternate Member

Anesthesiology, chronic pain management, clinical research

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Prof. Christian Rolfo

UA

Alternate Member

(Phase I- Early) Clinical Trials, Thoracic Oncology, Translational Oncology. New Biomarkers and resistance mechanisms in oncology. Liquid Biopsies.

22

Prof. Isabelle Vande Broek

UA, AZ Nikolaas

Alternate Member

Diagnosis and treatment of hematological disorders

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Stéphanie Bauduin

Alternate Member

General practice

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The new commission: composition

Additional experts can be invited ad hoc, without right to vote:

- eg. geriatrics

Consultative members – advisory vote:

Patient representation

4 members:

LUSS (Ligue des Usagers des Services de Santé)

NIC (Nationaal Intermutualistisch College)

VPP (Vlaams Patiëntenplatform)

Test-Aankoop

Chief Executive Officer of the FAMHP

Director General PRE-Authorization

Director General POST Authorization

Director General INSPECTION

RIZIV/INAMI

Ethics Committee representation

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DOI and COI

The DOI of all members has been assessed by the famhp.

In certain dossiers, a member can participate in the discussion concerning the

medicine in question or a competitor, but the member is not allowed to take

part in the decision process (voting).

At the start of each session, each member will be asked by the (vice)-

president to declare any conflict of interest to the dossiers presented on the

agenda.

DOI of the members will be made publically available on the website of the

famhp

Kickoff meeting famhp/DG Pre

17/12/2015

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• Weekly meetings

• Meetings cancelled due to small number of dossiers: 26%

• Quorum was established in all the meetings

Number of meetings

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Number of dossiers treated by the commision 1st year

active discussion

VHP (voluntary harmonization procedure) 2CTA (clinical trial application) 1UMN 39MA CP new 8MA MRP new 4MA DCP new 6MA NP new 18Variation CP 2Variation MRP 3Variation NP 12referral 6vigilance 4other 19total 124

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Focus on critical files

decision based on internaldecision matrix

Active discussions

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Dossiers treated by the commision: active discussion

VHP/CTA

UMN

MA

Variations

Referral

Vigilance

other

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Royal decree 25/04/2014

• amending RD 14/12/2006

• provides legal context for UMN programs

Subsequent to member state implementation of EC regulation726/2004, Art. 83

Compassionate use programs are limited to medicinal products that have no market authorisation, in any therapeutic indication, in Belgium.

Medical need programs relate by shear nature to products that are authorised in Belgium for which:

• the sought indication has been obtained but the product is not yet commercially available in Belgium, or

• a demand to obtain the sought indication is ongoing, or

• clinical trials are ongoing in the sought indication.

Legal context UMN

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MNP/CUP programs are restricted to patients with

• a life threatening disease, or

• a disease with a serious impact, or

• a chronic disease

that cannot be treated satisfactorily by a product licensed for this indication and commercially available in Belgium.

Also applicable when B/R of a novel product isconsidered significantly better than commerciallyavailable treatment options.

Legal context

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GMC must establish (legally foreseen criteria)

• whether alternative therapies are available to treat the condition,

• the justification why the proposed patient population cannot be adequately treated with available alternatives,

• the justification why the proposed patient population cannot enter a clinical trial [investigating the concerned product],

• the quality of the product,

• the pertinence of the non-clinical data of the product,

• the relevance of the clinical data of the product addressing the unmet medical need in the concerned indication.

Legal context

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Assessors’s recommendation vs GMC decision

Some key figures – October 2016

Program

N

Comparison advice

N

N

N

N

%

identical

modified

different

N

%

N

%

N

%

Medical Need

46

36

78

9

20

1

2

Compassionate Use

20

18

90

2

10

0

0

Total

66

54

82

11

17

1

1.5

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GMC vs ANSM (ATU) decisions

Some key figures – October 2016

Agency Total

Identical

indication Approved Rejected Invalid Retracted

In

assessment

N N % N % N % N % N % N %

FAMHP

68 53 78

40 76 7 10 2 3 4 8 0 0

ANSM 50 94 0 0 0 0 0 0 3 6

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• 3 hearings with industry

• 2 ad hoc working groups

“other”

federal agency for medicines and health products

QUESTIONS???

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Contact

Federal Agency for Medicines and Health Products –FAMHP

Place Victor Horta 40/40

1060 BRUXELLES

tel. + 32 2 528 40 00

fax + 32 2 528 40 01

e-mail welcome@fagg-afmps.be

www.afmps.be