myRIS DRUGS & BIOLOGICS

myRIS DRUGS & BIOLOGICS
Friday November 8, 2013 (SW45 2013)
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NEWS
WORLDWIDE
EUROPEAN UNION
FRANCE
EUROPE
RUSSIA & RELATED COUNTRIES
NORTH AMERICA
LATIN AMERICA
AFRICA
AUSTRALIA & NEW ZELAND
INDIA, PAKISTAN & ASIA
DISCOVERY AND BUSINESS DEVELOPMENT
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Parmi les sujets sélectionnés dans ce numéro :
Johnson & Johnson débourse 2,2 milliards de dollars pour éviter un
procès
p.2
Teva says it may have violated bribery laws around the world
p.2
WorldMedical Association Declaration of Helsinski Ethical Principles for
Medical Research involving human subjects (64th WMA General
Assembly, Fortaleza, Brazil, October 2013)
p.4
FDA: Summary of ICH Q3D – Guideline for elemental impurities draft
consensus guideline
p.5
New EMA guidance on development of antibacterials to help in the
fight against multidrug-resistant pathogens
p.8
EMA: Questions and answers on design space verification
p.8
EMA: Amendments to the pharmacovigilance legislation: new
notification requirements for marketing-authorization holders and
changes to scope of European safety referrals
p.9
EMA : First qualification opinion on a statistical methodology for dose
finding released for public consultation
p.9
EMA launches public catalogue of medicine shortages assessed by the
Agency
p.11
CMDh : Regulation (EC) N° 1234/2008 on variations
p.12
Décret N° 2013-935 du 18/10/2013 relatif aux modalités de déclaration
de certaines contributions pharmaceutiques
p.14
Décret N° 2013-923 du 16/10/2013 pris pour la transposition de la
directive 2012/26/UE du 25/10/2012 modifiant en ce qui concerne la
pharmacovigilance la directive 2001/83/CE instituant un code
communautaire relatif aux medicaments à usage humain
p.14
Antimicrobial prescribing and stewardship competencies
p.19
Following simple steps can help reduce surgical-site infections, saving
lives and money, says NICE in new standards
p.20
FDA: Guidance for Industry: Chronic hepatitis C virus infection:
developing direct-acting antiviral drugs for treatment – Revision 1
p.23
UEMOA – Union Economique et Monétaire Ouest Africaine
p.28
TGA: Biosimilar medicines – information for health professionals p.30
The PMDA publishes a list of new drugs which are unapproved in Japan
but approved in the USA and the EU
p.32
China issues revised GSP for cold chain quality control
p.32
India’s health ministry directed to re-evaluate 157 clinical trials
p.34
«Beyond words, the world »
1
© Adrien Tillet
NEWS
 Johnson & Johnson débourse 2,2 milliards de dollars pour éviter un
procès
Le groupe pharmaceutique américain était accusé d’avoir versé des pots-de-vin à des pharmaciens. Un
accord a été conclu avec le gouvernement américain pour mettre fin aux accusations.
Les accusations concernent « la promotion pour des usages non approuvés comme sûrs et efficaces par
l’autorité américaine des médicaments (FDA) et le paiement de pots-de-vin à des pharmaciens » et des
médecins pour la vente pour ces indications des médicaments Risperdal, Invega et Natrecor, détaille le
communiqué.
« Le comportement incriminé dans cette affaire a mis en péril la santé et la sécurité des patients », fait
valoir le ministre américain de la Justice Eric Holder. … (Source : LesEchos)
 Teva says it may have violated bribery laws around the world
While other countries are scrambling to understand if they have exposure to a Chinese probe into bribery,
Teva Pharmaceutical Industries ($TEVA) says it has found it has similar issues elsewhere. The Israeli
2
drugmaker said in a securities filing that its own investigation has turned up suspect practices in Latin
America, Eastern Europe and Russia.
Teva reported in its quarterly report Thursday that it may have violated the U.S. Foreign Corrupt Practices
Act (FCPA) and perhaps local laws and could face fines, lawsuits and criminal actions. It said it turned all of
the information over to the Securities and Exchange Commission (SEC) and the Department of Justice in the
U.S. … (Source: FiercePharma)
 Scientists voice fears over ethics of drug trials remaining unpublished
Drug companies and other organisations that carry out clinical trials are violating their ethical obligation to
the people who take part by failing to publish the results, scientists will argue on Wednesday.
Almost one in three (29%) large clinical trials in the United States remain unpublished five years after they
are finished, according to scientists writing in the British Medical Journal. Of those, 78% have no results at
all in the public domain.
The scientists calculate about 250,000 people took part in the unpublished trials and have therefore been
exposed to all the risks involved in research without the benefits to society they were led to believe would
ensue. This "violates an ethical obligation that investigators have towards study participants", say
Christopher Jones from the Department of Emergency Medicine, Cooper Medical School of Rowan
University, New Jersey, and colleagues. They call for additional safeguards "to ensure timely public
dissemination of trial data." … (Source: TheGuardian)
 Merck's Experimental HPV Vaccine Shows Promise in Late-Stage Trial
(Source: WSJ)
 AstraZeneca's asthma drug benralizumab advances in Phase III
clinical trial
AstraZeneca has started the Phase III Windward program of its biotech drug benralizumab for the
treatment of patients with severe asthma.
3
MedImmune, the company's biologics research and development (R&D) unit, is responsible for developing
benralizumab, a monoclonal antibody binding to the interleukin-5 receptor (IL-5Ra) that reduces
eosinophils.
Eosinophils are a type of white blood cells which play a major role in the cause and extremity of asthma
and its side-effects.
According to the company, in patients with elevated eosinophil counts, treatment with an IL-5 inhibitor in
addition to guideline-based strategies might improve their asthma control and decrease the frequency of
asthma attacks. … (Source: PBR)
WORLDWIDE
Government and Regulatory Bodies
http://www.pharmweb.net/pwmirror/pwk/pharmwebk.html
http://www.who.int/en/
 WorldMedical Association Declaration of Helsinki Ethical Principles
for Medical Research Involving Human Subjects (64th WMA General
Assembly, Fortaleza, Brazil, October 2013)
World Medical Association Declaration of Helsinki 2013.pdf
 Pharma's Transparency Troubles
It seems like open season on the pharmaceutical industry. Academics and consumer activists charge
pharma companies with hiding clinical trial information on medical product safety, fueling the campaign to
expand public access to confidential research information. The federal government’s “Sunshine” program
for disclosing financial ties between industry and physicians reflects a lengthy campaign to curb marketing
tactics perceived to boost inappropriate prescribing. Media reports regularly attack high drug prices, both
for life-saving specialty drugs and for widely used treatments such as asthma inhalers. And recent
disclosures raise questions about too-close ties between pharma companies and Food and Drug
Administration officials. … (Source: PharmExec)
 Biotech research requires strong protections
Recent biopharmaceutical advancements are changing the way we think about medicine. “Biologics” are
being manufactured from living cells to treat sickness at the most basic biological level. These drugs are far
more complex and effective than traditional chemical-based medicines.
Texas-based research centers are testing, for example, a virus-based biologic that may someday treat
melanoma, as well as a sophisticated antibody that will target cancers and lupus.
4
Developing such groundbreaking treatments is neither cheap nor easy. A mere five out of every 5,000
compounds devised will ever make it to clinical testing. The testing process itself takes, on average, at least
seven years. And after all that, only one in five drugs tested on humans will ever make it to pharmacy
shelves.
The biologic development process is, unsurprisingly, extremely expensive. Including the cost of drugs that
fail, the average cost to bring a new drug to market is $1.2 billion. In 2011 alone, the biotechnology sector
spent $49.5 billion in research and development. … (Source: MySA)
 Risk-based supply chain management means more audits for
excipient firms
Regulatory pressure for risk-based supply chain management is increasing excipient suppliers’ audit burden
according to the team preparing to launch the 'excipact' certification scheme as an independent
association.
The comments came from European Fine Chemicals Group (EFCG) vice chairman Frithjof Holtz, head of
advocacy at Merck Millipore, who told attendees at CPhI in Frankfurt, Germany last week the economic
burden extra audits places on suppliers and excipient users “should not be underestimated.”
“There are increasing expectations from regulators on ensuring the safety of the supply chain means more
physical audits of manufacturers, suppliers and also distributors, warehouses, forwarders and so on.”
He cited –in Europe - the FMD which requires risk assessment for appropriate good manufacturing
practices for excipients and moves to revise GMPs and – in the US – FDASIA’s supplier registration
requirement as examples of regulatory moves that have increased the audit burden. … (Source: In-Pharma)
 Vaccines Do Not Cover Most Common HPV Types in Black Women
The HPV subtypes that are most common in black women in the United States are not targeted by the
currently available vaccines Gardasil and Cervarix, according to new research.
The findings suggest that current HPV vaccination will be less beneficial for black women in the US than for
their white counterparts, said study coauthor Catherine Hoyo, PhD, MPH, of Duke University, in Durham,
North Carolina. … (Source : Medscape)
http://www.ich.org/
http://www.picscheme.org/
http://www.ipec-europe.org
 Summary of ICH Q3D – Guideline for Elemental Impurities Draft
Consensus Guideline
Within scope:
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–New finished drug products (as defined in ICH Q6A and Q6B) and new drug products
employing existing drug substances
–Drug products containing proteins and polypeptides (produced from recombinant or nonrecombinant cell-culture expression systems), their derivatives, and products of which they are
components (e.g. conjugates)
–Drug products containing synthetically produced polypeptides, polynucleotides, and
oligosaccharides
Summary_FDA Draft Guidance (Q3D) (October 2013).pdf
EUROPEAN UNION
 Submission of comments on Revision 4 of the Guideline on the
format and content of application for designation as orphan medicinal
and on its transfer from one sponsor to another (ENTR/6283/00 Rev 4)
and Revised Application form for orphan medicinal product designation
(Source: EC)
 Submission of comments on “Guideline on the format and content of
applications for designation as orphan medicinal products and on the
transfer of designations from one sponsor to another” (ENTR/6283/00
Rev 4; 12 June 2013) and Annex (Application Form for Orphan Medicinal
Product Designation) (Source: EC) (Hoffman-La Roche)
6
 Submission of comments on ' Guideline on the format and content of
applications for designation as orphan medicinal products and on the
transfer of designations from one sponsor to another ' (ENTR/6283/00
Rev 4) (Source: EC, Novartis) (EC, SHIRE)
 Commission Européenne: Que signifie le triangle noir?
L’Union européenne (UE) a mis en place un nouveau moyen d’identifier les médicaments faisant l’objet
d’une surveillance particulièrement étroite (Source: EC)
 Use of '-omics' technologies in the development of personalised
medicine
Under the heading ‘Towards more personalised medicines’ in its Communication1 of 10 December 2008 on
a Renewed Vision for the Pharmaceutical Sector, the Commission announced a report on the use of ‘omics’ technologies2 in pharmaceutical research and development.
This report, presented in the form of a staff working document, focuses on:
- the potential and issues with the use of -omics technologies in the research and development of
personalised medicine and current EU research funding in the area;
- recent developments in EU legislation for placing medicinal products and medical devices on the market;
- factors affecting the uptake of personalised medicine in health care systems. … (Source : EC)
 Janssen files EMA marketing authorisation application for blood
cancer medicine Ibrutinib
Janssen-Cilag (Janssen), a subsidiary of Johnson & Johnson pharmaceutical firm, has submitted marketing
authorization application (MAA) to the European Medicines Agency (EMA) for the approval of once daily
oral drug ibrutinib for the treatment of two forms of blood cancer.
The approval is intended to treat adult patients with relapsed or refractory chronic lymphocytic leukemia
(CLL)/small lymphocytic leukemia (SLL) and or relapsed or refractory mantle cell lymphoma (MCL).
The company claims that ibrutinib is the first in a class of medicines called Bruton's tyrosine kinase (BTK)
inhibitors.
According to the company, ibrutinib covalently bonds to BTK in malignant B cells, shutting down major
proliferation and survival pathways. … (Source: PBR)
 Baxter files application to market hemophilia B vaccine Rixubis in
Europe
Baxter has submitted a marketing authorization application (MAA) to the European Medicines Agency
(EMA) for approval of Rixubis, recombinant factor IX for the treatment and prophylaxis of bleeding in
patients of all ages with hemophilia B.
7
Hemophilia B is the second most common type of haemophilia and results from insufficient amounts of
clotting factor IX, a naturally occurring protein in blood that helps to control bleeding.
The submission is based on results of research with hemophilia B patients previously treated with other
therapies and is further supported by data secured from a study of 23 hemophilia B patients less than 12
years old. … (Source: PBR)
 EUnetHTA strengthens its position as the scientific and
technical cooperation on HTA in Europe (Source: EUnetHTA)
European Medicines Agency
http://www.ema.europa.eu/ema/
Human medicines: Regulatory
http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/landing/human_medicines_regulatory.js
p&mid=WC0b01ac058001ff89
 New EMA guidance on development of antibacterials to help in the
fight against multidrug-resistant pathogens
The European Medicines Agency (EMA) today released an Addendum to the guideline on the evaluation of
medicinal products indicated for the treatment of bacterial infections. One of the most important aspects
of the addendum is that it outlines a new approach facilitating the development of antibacterial agents
targeted against multidrug-resistant (MDR) pathogens where patients have very limited or no remaining
treatment options. It also gives guidance on data-gathering strategies to support the benefit-risk evaluation
as part of the marketing-authorisation process for different indications.
This addendum complements the Guideline on evaluation of medicinal products indicated for treatment of
bacterial infections. … (Source: EMA)
 Questions and Answers on Design Space Verification
Q&A - Design Space Verification - October 2013.pdf
8
 Amendments to the pharmacovigilance legislation: new notification
requirements for marketing-authorisation holders and changes to scope
of European safety referrals
Amendments of the European Union (EU) pharmacovigilance legislation that were adopted in October 2012
came into force on Monday 28 October 2013. These changes, which cover various aspects of the legislation,
aim to further strengthen the protection of patient health by increasing the ability of the European
medicines network to take prompt and appropriate regulatory action. … (Source: EMA)
 Meeting highlights from the Committee for Medicinal Products for
Human Use (CHMP) 21-24 October 2013
This page provides an overview of the opinions adopted at the October 2013 meeting of the Committee for
Medicinal Products for Human Use (CHMP) and other important outcomes.
Three new medicines recommended for approval
This month, the CHMP recommended the approval of Opsumit, an orphan medicine intended for the
treatment of pulmonary arterial hypertension in adults.
The Committee gave a positive recommendation for Brintellix in the treatment of major depressive
episodes in adults.
The generic medicine Levetiracetam Hospira also received a positive opinion for use in epilepsy. … (Source:
EMA)
 EMA: How access to full clinical-trial data sets will benefit medicines
developers (Source: EMA)
 European Medicines Agency advises on compassionate use of
sofosbuvir (Source: EMA)
 First qualification opinion on a statistical methodology for dose
finding released for public consultation (Source: EMA)
 EMA: Revised reflection paper on injection site residues:
considerations for risk assessment and residue surveillance - Draft (Source:
EMA)
 Concept paper on the revision of the Note for guidance on the
approach towards harmonisation of withdrawal periods (Source: EMA)
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 Comments invited on concept paper on the need to revise the
guideline on medicines to treat Alzheimer's disease
The European Medicines Agency has released a concept paper on the need to revise the guideline on
medicines for the treatment of Alzheimer's disease and other dementias for public consultation.
Comments should be sent by 31 January 2014 to cnswpsecretariat@ema.europa.eu, using the template
provided. … (Source: EMA)
 The patient's voice in the evaluation of medicines
How patients can contribute to assessment of benefit and risk
The ultimate raison d’être of any medicine is to benefit patients. However, older and more paternalistic
models of medicine often treated patients as a passive group who were to be given instructions but who
should not be confused or worried by too much information. Although the best healthcare professionals
have always recognised the importance of listening carefully to their patients, the idea that patients’
knowledge, views and preferences were as significant as those of any other stakeholder in the healthcare
process was not a standard part of this model. Over the past decades this has changed, as it has become
increasingly clear that such a model is neither appropriate nor useful in a better connected and less
deferential world.
The Workshop on the patient’s voice in the evaluation of medicines brought together representatives of
patients, consumers and healthcare professionals as well as the pharmaceutical industry and members of
the Agency’s scientific committees and staff.
Action points identified included the need to:
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identify where quantitative versus qualitative input is needed, and develop and validate new tools
for eliciting values and preferences and representing benefit and risk; these tools need to take
account of the way values vary between patients and can change during the patient journey;
develop means for identifying and managing differences of view among patients, and between
patients and other stakeholders;
consider what training and support is needed to maximise patient involvement at all stages of the
process for the full range of disease states, and who should be supplying these;
further develop the role and timing of patient involvement in later, post-marketing stages of the
product lifecycle (e.g. in signal assessment);
identify and try to minimise legal, regulatory, financial and procedural barriers to patient
involvement.
These action points will be considered as part of the on-going review of the existing formal framework of
interaction between the Agency and patient and consumer organisations. This framework has been in place
since 2005 and defines how the Agency interacts with patients and consumers.
EMA - Report - The patient's voice in the evaluation of medicines - 18Oct2013.pdf
10
 European Medicines Agency launches public catalogue of medicine
shortages assessed by the Agency
The European Medicines Agency has launched a public catalogue that contains information on supply
shortages of medicines.
The catalogue includes information on medicine shortages affecting more than one Member State and that
have been assessed by the Agency.
The aim of the catalogue is to offer a reference point for stakeholders for up-to-date information on
shortages that have been assessed by the Agency.
The catalogue provides:
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information on the reason of the shortage and the current status of the shortage (ongoing or
resolved)
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information on the extent of the shortage;
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specific information for patients and healthcare professionals;
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links to relevant related documents.
Over the past few years, there have been a number of public-health crises caused by supply shortages of
medicines as a result of manufacturing issues and good-manufacturing-practice (GMP) compliance
problems.
Shortages can have important consequences for patients and healthcare professionals, such as the need to
switch to an alternative treatment and the setting-up of priority-access programs.
The creation of this catalogue is part of an implementation plan developed by the Agency in 2012 to help
the European medicines regulatory network deal with medicine supply shortages following manufacturing
problems. This short- and medium-term plan includes a number of actions that aim to prevent, mitigate
and manage shortages of medicines.
As part of these planned actions, the Agency held a workshop on the prevention of medicine shortages due
to manufacturing and quality problems in October 2013. The workshop aimed to discuss how to improve
existing risk-management strategies and how to mitigate the impact of shortages. A workshop report will
be published shortly.
 European Medicines Agency meeting and holiday dates 2013
(Source:
EMA)
 European Medicines Agency's Management Board supports plan to
publish agendas and minutes of all committees (Source: EMA)
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 Nomination au Comité des médicaments à usage humain de l’Agence
européenne des médicaments
Le Dr Pierre Demolis, directeur de la direction des médicaments en oncologie, hématologie, immunologie,
néphrologie de l’ANSM, membre du CHMP depuis 2007, a été élu à la vice-présidence du Comité des
médicaments à usage humain de l’Agence européenne des médicaments (EMA) pour une durée de 3 ans.
HERBAL MEDICINES FOR HUMAN USE
http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/herbal_search.jsp&mid=WC0b01ac058001fa1d
http://www.hma.eu/cmdh.html
WHAT’S NEW: http://www.hma.eu/186.html
 Regulation (EC) No 1234/2008 on variations
The CMDh has adopted a new version of Chapter 7 (work-sharing) of the CMDh Best Practice Guide for the
submission and processing of variations in the mutual recognition procedure. The document was updated
to clarify that all Member States as proposed by the MAH for work-sharing procedures for purely national
marketing authorisations should accept the submission of the variation application if the work-sharing
fulfils the requirements for a work-sharing procedure. The revised Chapter 7 will be published on the CMDh
website under “Procedural Guidance, Variation”.
The CMDh has revised the Questions & Answers on variation. Questions No. 3.10 on deletion of a
pharmaceutical form or strength and No. 4.15 on the introduction or update of the summary of the
pharmacovigilance system and change in the QPPV, have been amended for clarification purpose. revised
Questions & Answers document will be published on the CMDh website under “Questions and Answers,
Variations”.
CMDh - Q&A - Submission of variations - Oct 2013.pdf
 CMDh BEST PRACTICE GUIDE ON WORKSHARING
This guidance covers worksharing procedures for:
a group of products from the same marketing authorisation holder1 where none of the
marketing authorisations is a centralised marketing authorisation. It may include marketing authorisations
granted via MRP/DCP as well as purely national marketing authorisations or a mixture of MRP/DCP and
purely national marketing authorisations.
a purely national marketing authorisation held by the same marketing authorisation holder in
more than one Member State.
In these cases, the competent authority of a Member State concerned chosen by the Coordination Group
shall be the ‘reference authority’. In case the worksharing procedure only contains products with the same
RMS there is no need for the CMDh to choose the reference authority (see section 4 “Worksharing of
MR/DC procedures with the same RMS”).
12
CMDh - BEST PRACTICE GUIDE ON WORKSHARING Chapter 7 - October 2013 - track changes.pdf
 Pilot on work-sharing procedure for the assessment of Active
Substance Master Files (ASMF)
The working group on Active Substance Master File procedures is actively preparing for the start of the
pilot on work-sharing procedure for the assessment of ASMF. A meeting with representatives of Interested
Parties has been organised in the margin of the CMDh meeting in order to discuss the pilot. The objective
of the meeting was to present to Industry the procedures for request of an EU ASMF reference number and
submissions of a new ASMF and to answer questions from Interested Parties.
Companies wishing to participate in the pilot will have to fill-in the EU AMSF number request form that will
be published on the CMDh website under “CMD Working Parties/Working Groups, Working Group on
Active Substance Master File Procedures”. In order to facilitate the request of EU ASMF numbers, a
dedicated list of contact persons has been set up that will be added to the published “Contact Points”.
CMDh - EU_ASMF_number_request_form.docx
 “BLUE – BOX” REQUIREMENTS
CMDh - Blue Box requirements - October 2013.pdf
http://www.edqm.eu/en/edqm-homepage-628.html
 Council of Europe’s European Directorate for the Quality of
Medicines and Healthcare (EDQM) and European Medicines Agency
joint meeting on raw materials used for the production of cell-based
and gene-therapy products
REPORT symposium - raw materials - cell-based and gene therapy products.pdf
 Safety Data Sheets subscriptions and translations (Source: EDQM)
 Counterfeiting of medical products: the Council of Europe steps up
action worldwide with the Medicrime Convention (Source: EDQM)
 Certification Monthly Report of Activities (Source: EDQM)
FRANCE
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 Décret no 2013-935 du 18 octobre
2013 relatif aux modalités de déclaration de certaines contributions
pharmaceutiques (Source: LegiFrance)
 Décret no 2013-923 du 16 octobre 2013 pris pour la transposition de
la directive 2012/26/UE du 25 octobre 2012 modifiant en ce qui
concerne la pharmacovigilance la directive 2001/83/CE instituant un
code communautaire relatif aux médicaments à usage humain (Source:
LegiFrance)
 Vente à l’unité des antibiotiques
L’Assemblée nationale a autorisé, dans le cadre du projet de loi de financement de la Sécurité sociale
2014, des expérimentations de vente à l’unité des antibiotiques, « lorsque leur forme le permet ».
Un décret fixera les conditions d’expérimentations qui dureront trois ans. (Source : Assemblee-nationale)
 André Choulika, chef du plan industriel sur les biotechs, mise tout sur
la thérapie cellulaire
Pour ce pragmatique, pas question de multiplier les réunionites. Il a profité du dernier conseil
d’administration de France Biotech pour arbitrer. "Sachant qu’il n’y a pas beaucoup d’argent mis sur la
table et que cela doit être un projet industriel, pas une usine à gaz, nous nous sommes concentrés sur un
seule thématique", confie André Choulika. Les traitements issus de la biologie de synthèse ont été écartés,
au profit de la thérapie cellulaire. Un domaine émergent des biotechnologies qui constitue "un vrai saut
quantique : pouvoir corriger d’emblée la cellule du patient plutôt que lui donner un traitement palliatif",
estime Patrick Biecheler, associé en charge de la santé au cabinet Roland Berger. Dans lequel la France voit
se structurer un centre d’excellence, avec la plateforme de production unique en Europe inaugurée en
septembre par CellforCure, filiale du laboratoire pharmaceutique public LFB. … (Source : UsineNouvelle)
14
 Les investissements pharma s’effondrent en France
Voici une volée de chiffres qui va apporter de l’eau au moulin des industriels français de la filière
pharmaceutique française, très remontés contre le PLFSS 2014 et la politique fiscale menée par le
gouvernement qui, selon eux, compromettent gravement l’attractivité du territoire.
Ainsi, selon
l’Observatoire des investissements productifs créé par le Leem et le cluster Polepharma qui présentait hier
ces données en avant-première, les investissements corporels bruts engagés en 2012 se sont élevés à 590,7
M€, soit une baisse… de 44 % en quatre ans. Environ 70 % de ce montant peut être attribué à cinq groupes
seulement.
« Il est temps de sonner le tocsin », a souligné Pascal Le Guyader, directeur des affaires
générales, industrielles et sociales du Leem à l’issue de cette présentation. Il a par ailleurs estimé que le
solde net des emplois créés par la filière pourrait, en 2013, « être négatif, probablement de l’ordre de – 1 %
». … (Source: ActuLabo)
 Valorisation et modélisation médico-économique du couple
Test diagnostique compagnon et Thérapie ciblée
Cette contribution a pour objectif de discuter de la démarche à suivre dans le cadre d’une évaluation
médico-économique d’un couple test compagnon/thérapie ciblée en abordant les points méthodologiques
clés à considérer dans ce type d’analyse.
Ce travail s’est structuré autour d’une analyse des données de la littérature et de la présentation d’un
modèle conceptuel envisageable dans le cas d’une thérapie ciblée anti-cancéreuse et de son test
compagnon prédictif de la réponse au traitement.
Bien que la cancérologie constitue le principal champ de réflexion de ce travail au travers des exemples
cités et du cas d’étude, les couple(s) test(s) compagnon(s)/thérapie(s) ciblée(s) et plus largement la
médecine personnalisée représentent un enjeu commun à de nombreuses aires thérapeutiques. …
(Source : Leem)
 LEEM: Fiscalité des entreprises du médicament - La France accroît son
décrochage avec les autres pays européens (Source: LEEM)
 Les contrats commerciaux liant les industriels du générique et les
officines, plus transparents
Un article du projet de loi de financement de la Sécurité sociale (PLFSS) pour 2014 prévoit d’imposer aux
industriels de déclarer les avantages commerciaux et financiers accordés aux pharmaciens. ... (Source :
Daso Santé)
Quand les applications électroniques (e-apps) sont-elles des Dispositifs Médicaux ?
Le Cabinet WHITE-TILLET est spécialiste de la question et peut vous aider à y répondre ; le cas échéant,
nous pouvons vous assister pour le marquage CE de votre application.
15
http://ansm.sante.fr/
 ANSM: Vigilances
Mise en place de la nouvelle législation en pharmacovigilance, quel bilan un an après? (Source: ANSM)
 Textes publiés en 2013 concernant les médicaments
Médicaments, médicaments combinés de thérapie innovante, préparations magistrales, hospitalières,
officinales, APSI (allergènes préparés spécialement pour un individu) substances stupéfiantes, psychotropes
ou autres substances vénéneuses, insecticides, acaricides et antiparasitaires à usage humain, huiles
essentielles, plantes médicinales, matières premières à usage pharmaceutiques, pharmacopée. … (Source :
ANSM)
 L’ANSM a réuni tous ses agents pour préparer l’avenir - Point
d'information
L’Agence nationale de sécurité du médicament et des produits de santé a rassemblé, le 15 octobre 2013,
l’ensemble de ses agents pour une journée de partage et d’échanges, plus d’un an après sa mise en place et
20 ans après la création de la première agence du médicament. Après une matinée réservée aux
collaborateurs internes qui a permis de dresser un premier point d’étape sur les nouveaux modes de
fonctionnement de l'agence et sur sa stratégie d’action pour répondre aux enjeux qui lui ont été assignés,
l’Agence a accueilli l’après-midi des acteurs de la santé qui ont fait part de leur analyse et de leur vision sur
les évolutions en cours en matière de transparence, d’indépendance, d’information et de surveillance des
médicaments. … (Source: ANSM)
Le Cabinet WHITE-TILLET s’est entouré de juristes de haut niveau pour vous aider
à valider la publicité et la promotion de vos médicaments. Leur expertise se
combine à notre expérience réglementaire et à notre maîtrise de la
méthodologie clinique. Contactez-nous pour le reviewing de vos documents
publicitaires ou promotionnels!
 Valdoxan (agomélatine): Ajout d’une nouvelle contre-indication et
rappel sur l’importance de la surveillance de la fonction hépatique Point d'information (Source: ANSM)
 Caustinerf Arsenical et Yranicid Arsenical, pâtes pour usage dentaire
: un signal de génotoxicité est susceptible de remettre en cause le
rapport bénéfice/risque de ces spécialités - Point d'information (Source:
16
ANSM)
 Ce qu’il faut savoir avant de commencer un traitement par
isotrétinoïne orale (Source: ANSM)
 Spécialités à base de fer pour injection intraveineuse: une utilisation
réservée aux établissements de santé en raison du risque de réactions
graves d’hypersensibilité - Point d'information (Source: ANSM)
 Déclarer un effet indésirable: quoi de neuf pour les professionnels
de santé et les patients? - Point d’Information (Source: ANSM)
Le Cabinet WHITE-TILLET a plus de 20 ans d’expérience et d’expertise dans le
domaine des dossiers d’AMM. Nous maîtrisons l’eCTD. Par notre structure et
notre réseau (cabinet associé au UK, réseau international), nous maîtrisons les
procédures et assurons un suivi efficace. Confiez-nous vos dossiers d’AMM ou de
variations!
http://www.has-sante.fr/portail/jcms/j_5/accueil
 FICHES DE BON USAGE (Source : HAS)
 SYNTHÈSES D'AVIS SUR LES MÉDICAMENTS (Source : HAS)
 Label de la HAS - Troubles du Comportement chez les Traumatisés
Crâniens: Quelles options thérapeutiques?
Les troubles du comportement des victimes de traumatisme crânien constituent la séquelle majeure chez
ces blessés. Les troubles du comportement ont des conséquences néfastes multiples impliquant les
familles, les soignants, et nombre d’autres professionnels (magistrats, avocats, assureurs etc.). Le présent
travail a pour objectifs :

d’organiser la démarche de soins dont une meilleure information des intervenants occasionnels,

de fournir un guide pratique de prise en charge pour le praticien,
17

d’améliorer l’efficience des différentes modalités thérapeutiques : traitements médicamenteux et
non médicamenteux, stratégie thérapeutique, hospitalisation, suivi ambulatoire, insertion
professionnelle y compris en milieu adapté.
… (Source: HAS)
 Maladie d'Alzheimer et maladies apparentées: diagnostic et prise en
charge de l'apathie - Note de cadrage
Cette note de cadrage présente le projet de recommandations de bonne pratique sur le thème « diagnostic
et prise en charge de l'apathie chez les patients atteints de maladie d'Alzheimer ou de maladies
apparentées » qui est en cours de réalisation par la HAS.
Les recommandations sur la prise en charge des troubles du comportement perturbateurs chez les patients
atteints de maladie d’Alzheimer ou de maladies apparentées publiées par la HAS en 2009 ont exclu les
troubles du comportement déficitaires ou de retrait telle que l’apathie, également fréquente au cours de
ces maladies. L’élaboration de ces recommandations professionnelles entre dans le cadre du Plan
Alzheimer 2008-2012. … (Source: HAS)
 HAS: RAPPORT D’EVALUATION TECHNOLOGIQUE - Utilité clinique du
dosage de la vitamine D (Source: HAS)
CEPS
http://www.sante.gouv.fr/comite-economique-des-produits-de-sante-ceps.html
Le Cabinet WHITE-TILLET a près de 30 ans d’expérience et d’expertise dans le
domaine du remboursement des médicaments, incluant la maîtrise de la
méthodologie clinique. La rédaction de ces dossiers est un art complexe. Les
conséquences pour les firmes peuvent être cruciales. Confiez-nous vos dossiers de
remboursement !
EUROPE
http://www.mhra.gov.uk/
 MHRA: Department of Health (DoH) appoints new Chair of the British
Pharmacopoeia Commission (Source: MHRA)
18
 Antimicrobial prescribing and stewardship competencies
The goal is to improve the quality of antimicrobial treatment and stewardship and so reduce the risks of
inadequate, inappropriate and ill-effects of treatment. This will improve the safety and quality of patient
care, and make a significant contribution to the reduction in the emergence and spread of antimicrobial
resistance. Antimicrobial stewardship is an important element of the UK five-year antimicrobial resistance
strategy (1) and the Chief Medical Officer’s annual report (2).
Antimicrobial resistance is a global public health issue driven by the overuse of antimicrobials and
inappropriate prescribing. The increase in resistance is making antimicrobial agents less effective and
contributing to infections that are hard to treat. The number of infections due to multi-drug resistant
organisms is growing, however, the number of new antibiotics in the pieline is extremely limited.
Antimicrobial stewardship initiatives aim to improve the prescribing of all agents, whether they target
bacterial, viral, fungal, mycobacterial or protozal infections.
Antibiotic resistance is of particular threat to children, older people and those with weakened immune
systems. Effective antibiotics have revolutionised many treatments, such as those for cancer, allowing more
aggressive therapy to be used and consequently leading to higher survival rates. Nevertheless, an increase
in infections that are more difficult to treat with antibiotics affects everyone, not just vulnerable groups.
Bacterial resistance potentially complicates the management of every infection, no matter how mild they
may be at the time of first presentation.
Educating the public and clinicians in the prudent use of antimicrobials as part of an antimicrobial
stewardship programme is of paramount importance to preserve these crucial treatments and to help
control resistance. Improving surveillance, and infection prevention and control are other key strategies.
UK - DH - Antimicrobial prescribing and stewardship compentencies - October 2013.pdf
 U.K. Drug Regulator Recalls 5 Drugs Made by India's Wockhardt (Source:
WSJ)
 UK health research body backs trial data disclosure & access to
patient data (Source: Clinica)
 Information sent to healthcare professionals in October about the
safety of medicines
Copies of letters sent to healthcare professionals in October 2013, to inform of new safety information and
advice. … (Source: MHRA)
 Ten years and beyond
In the last ten years, the Agency expanded its offerings including:

licensing decisions on whether to grant authorisations to place medicines on the market

authorisation and inspection of clinical trials of medicines

new procedures for authorising medicines intended to be marketed across Europe
19
Recently, National Institute for Biological Standards and Control (NIBSC) join the Agency. NIBSC is
responsible for producing over 90% of the International Standards used to assure the quality of biological
medicines. … (Source: MHRA)
http://www.nice.org.uk/
 Following simple steps can help reduce surgical-site infections, saving
lives and money, says NICE in new standards
Surgical site infection is a type of healthcare-associated infection in which a surgical incision site becomes
infected after a surgical procedure. Surgical site infections have been shown to account for up to 16% of all
healthcare-associated infections. The rate of surgical site infection varies depending on the type of
procedure, with rates of over 10% for large bowel surgery, and less than 1% for orthopaedic procedures.
Surgical site infections can often be prevented with appropriate care before, during and after surgery. If an
infection does develop, the correct treatment will minimise complications resulting from the infection.
The NICE quality standard is based on the NICE guidance on both surgical site infection and healthcareassociated infections. … (Source: NICE)
 NICE draft "yes" for Astellas' prostate cancer drug
The National Institute for Health and Care Excellence (NICE) has issued draft guidance recommending
Astellas Pharma’s Xtandi (enzalutamide) for treating hormone relapsed metastatic prostate cancer.
Xtandi is an effective treatment and, as it can be taken orally, it allows patients to be treated at home, said
Professor Carole Longson, director of NICE’s Centre for Health Technology Evaluation.
Xtandi works in a different way to other drugs currently available for prostate cancer treatment. NICE’s
draft guidance recommends it as an option for treating hormone relapsed prostate cancer in adults, only if
their disease has progressed during or after one docetaxel-containing chemotherapy regimen and if the
manufacturer provides it with the discount agreed in the patient access scheme (PAS).
NICE says that its independent appraisal committee had heard that, although both Xtandi and Johnson &
Johnson’s prostate cancer drug Zytiga (abiraterone) are oral treatments, only Xtandi can be taken on a full
stomach, making it more convenient to take. The panel was also told that Xtandi can help control the
cancer longer because there is no need to reduce the dose to prevent liver toxicity, as with Zytiga.
The draft guidance has been welcomed by Prostate Cancer UK, which points out that Xtandi is one of the
few treatments shown to extend the lives of men with advanced prostate cancer. … (Source :
PharmaTimes) (NICE)
 NICE says yes to eye condition treatment in draft guidance
NICE has published draft guidance recommending ocriplasmin (Jetrea, ThromboGenics) as an option for
treating some adults with the rare eye condition, vitreomacular traction.
Vitreomacular traction occurs when the vitreous, the gel-like substance in the eye, pulls abnormally on the
retina, the light-sensitive layer of tissue at the back of the inner eye, which is responsible for processing
20
visual images. The pulling of the gel disturbs the retina, causing swelling and distorted vision, and
sometimes a hole in the macular area. It can occur as a result of ageing.
NICE has recommended ocriplasmin as an option for treating vitreomacular traction in adults, including
when associated with a macular hole with a diameter of 400 micrometres or less, only if they have severe
symptoms and an epiretinal membrane is not present. … (Source: Nice)
 NICE: Choroidal neovascularisation (pathological
ranibizumab: final appraisal determination (Source: NICE)
myopia)
-
 NICE: Non Hodgkin's lymphoma (relapsed refractory) - pixantrone
monotherapy: appraisal consultation 2 (Source: NICE)
 NICE rejects J&J's Zytiga as too expensive
The National Institute for Health and Clinical Excellence issued draft guidance recommending
against Zytiga use, saying that the drug doesn't work well enough to justify its price. And that's after J&J
offered a discount to help sway the agency in its favor. … (Source : FiercePharma)
http://www.imb.ie/
 IRISH MEDICINES BOARD - GUIDE TO AMENDMENT AND RENEWAL
APPLICATIONS FOR PROJECTS REGULATED BY DIRECTIVE 2010/63/EU
AND S.I. NO. 543 OF 2012 (Source: IMB)
 IMB: Guide to Interchangeable Medicines (Source: IMB)
http://www.fagg-afmps.be/fr/
 Pharmacovigilance : Nouvelle réglementation en matière de
pharmacovigilance pour les médicaments à usage humain (Source : AFMPS)
http://www.bfarm.de/DE/Home/home_node.html
http://www.agenziafarmaco.gov.it/en
21
http://www.aemps.gob.es/en/home.htm
http://www.legemiddelverket.no/English/Sider/default.aspx
http://www.lakemedelsverket.se/english/
 Health system review
Health system review - Austria.pdf
http://www.eum.hu/about-us/the-ministry/ministry-of-health
http://www.swissmedic.ch/index.html?lang=fr
 Fake drugs intercepted by Swiss customs (Source: SwissInfo)
 DHPC – Erivedge® (vismodegib): prolongation de 7 mois à 24 mois de
la période pendant laquelle une contraception est recommandée après
la fin du traitement par Erivedge chez les femmes en état de procréer
(Source: SWISSMEDIC)
 Toxine botulique de type A: médicaments autorisés et indications,
mode d’emploi correct, risques et precautions (Source: SWISSMEDIC)
Les Bonnes Pratiques de Fabrication (GMP) et les Bonnes Pratiques
Cliniques (GCP) sont au cœur de nos préoccupations et dans notre
champ de compétence. N’hésitez pas à venir vers nous pour réaliser vos
audits. Nous avons un réseau de des correspondants locaux en Asie et
aux Etats-Unis.
22
RUSSIA & RELATED COUNTRIES
Ministry of Healthcare of the Russian Federation
http://government.ru/eng/power/23/
Ministry of Health of Ukraine
http://www.kmu.gov.ua/control/en/publish/article?art_id=88456
 Health system review
Health system review - Belarus.pdf
NORTH AMERICA
http://www.fda.gov/
 FDA: Guidance for Industry - Chronic Hepatitis C Virus Infection:
Developing Direct-Acting Antiviral Drugs for Treatment - Revision 1
(Source: FDA)
 FDA: Guidance for Industry - Acute Bacterial Skin and Skin Structure
Infections: Developing Drugs for Treatment (Source: FDA)
 FDA: Guidance for Industry - Q4B Evaluation and Recommendation
of Pharmacopoeial Texts for Use in the ICH Regions Annex 14 Bacterial Endotoxins Test General Chapter (Source: FDA)
23
 FDA: DRAFT CONSENSUS GUIDELINE GUIDELINE FOR ELEMENTAL
IMPURITIES Q3D (Source: FDA)
 Draft Guidance on Iron Sucrose
FDA - Draft Guidance on Iron Sucrose.pdf
 FDA approves extended-release, single-entity hydrocodone product
(Source: FDA)
 Statement on Proposed Hydrocodone Reclassification from Janet
Woodcock, M.D., Director, Center for Drug Evaluation and Research
(Source: FDA)
 FDA to complete phase-out of chlorofluorocarbon inhalers (Source: FDA)
 FDA awards 15 grants to stimulate drug, device development for rare
diseases (Source: FDA)
 FDA: Draft Guidance for Industry: Use of Nucleic Acid Tests to Reduce
the Risk of Transmission of West Nile Virus from Donors of Human Cells,
Tissues, and Cellular and Tissue-Based Products (HCT/Ps) (Source: FDA)
 FDA Report Outlines Approach to Personalized Medicine
The report, Paving the Way for Personalized Medicine: FDA's Role in a New Era of Medical Product
Development, outlines the fundamental ways in which the FDA has modified its traditional approaches to
drug and device regulation in the new era of products that are tailored toward specific patient subtypes,
rather than broad diagnostic groups.
"We're very, very excited about this report because I think it captures the broad context of what's
happening in science and medicine today and the role of the FDA as we enter the era of personalized
medicine, and for us, personalized medical product development," FDA commissioner Margaret A.
Hamburg, MD, said in a press briefing held at the FDA headquarters Monday. … (Source: Medscape)
 Study questions FDA's shorter drug approval times
Study authors Thomas Moore of the Institute for Safe Medication Practices and Dr Curt Furberg, a
professor at Wake Forest School of Medicine, examined the development times, clinical testing and risks
associated with 20 new drugs approved in 2008. Eight were given expedited review and 12 standard
review.
24
It found that expedited drugs underwent a median of 5.1 years of clinical testing before being approved,
compared with 7.5 years for those that underwent a standard review. But in many cases safety monitoring
trials that were supposed to be conducted after the products were approved were either not conducted,
not completed, or not submitted to the FDA. … (Source: Reuters)
 FDA helps generic drugmakers get copies of Glaxo's Advair to market
GlaxoSmithKline's asthma drug Advair is hard to copy. The British pharma ($GSK) has said it time and again,
and recently, generics giant Teva backed up that claim, saying producing a generic would be too difficult
before 2018. Some companies have given up on generic Advair, while others have shied away altogether
despite the substantial sales up for grabs. But what if generics makers had some help--and an expedited
review process--from the FDA?
According to Leerink Swann analyst Jason Gerberry, that's exactly what's in the works. At this week's
Generic Pharmaceutical Association (GPhA) conference, the agency announced plans to aim for a 10-month
review on copies of complex drugs like Advair. It also said it's working with those companies looking to
produce a rival to make sure they're on the right track. … (Source: FiercePharma)
 Novartis Group calls on FDA to maintain the well-established naming
policy for all biologics to help ensure patient safety
Novartis announced today it has filed a Citizen Petition with the US Food and Drug Administration (FDA),
urging the Agency to require that a biosimilar share the same INN as the reference product.
Novartis files Citizen Petition with FDA, urging uniform approach to use of international nonproprietary
names (INN) for all biologic medicines
A modified INN for biosimilars would impede their ability to compete fairly in the marketplace, create
confusion and limit patient access to these critical medicines
Novartis is confident in the FDA's ability to ensure safety and efficacy of all biologics, including biosimilars,
through its well-established review process
Novartis argues that changing the well-established convention that the INN describe the active ingredient
would undermine the safe and rational use of all biologics. In its petition, the company also highlights that
'assigning different INNs to biosimilars would introduce unnecessary confusion into the healthcare system
and could unintentionally communicate increased caution, unfounded risk, or other regulatory reservations
that are purely hypothetical.' … (Source: PMPNews)
J&J's Zytiga gets expanded approval that could double sales
The FDA on Monday said it approved Zytiga for treatment in men with late-stage, prostate cancer even
before they receive chemotherapy. A study found that it reduced tumor growth and could extend life
expectancy about 5 months.
… (Source : FiercePharma)
 FDA approves Roche leukemia drug Gazyva
U.S. regulators said on Friday they approved a new treatment from Roche Holding AG for patients with
chronic lymphocytic leukemia (CLL) who have not previously been treated for one of the most common
forms of blood cancer. … (Source: Reuters)
25
USA
 BIO calls for different names for biosimilars, branded biotech drugs
Like their pharmaceutical counterparts, branded biotech drugs carry both a brand name and a generic
chemical name, such as the autoimmune drug Enbrel, made by Amgen and Pfizer, known generically as
etanercept. By law, generic pharmaceutical drugs are chemically identical to branded drugs and use the
same generic names, and the companies wishing to make follow-on biologics, or biosimilars, want the same
policy for their products.
But biotech companies say that because biosimilars are made from different cell lines from branded
biologics, they are only similar rather than identical, and thus their generic names should be different, such
as carrying a prefix. … (Source : DrugStoreNews)
http://www.pcori.org/
 PCORI Launches Community-Building “Pipeline to Proposal” Awards
(Source: PCORI)
 Harold Sox, MD, to Serve as Senior Adviser to Patient-Centered
Outcomes Research Institute (Source: PCORI)
 PCORI and AHRQ Partner on Request for Applications to Study
Treatments for Uterine Fibroids (Source: PCORI)
http://www.ahrq.gov/
 AHRQ: Letters of Intent Due November 15 for Uterine Fibroid Project
(Source: AHRQ)
http://www.iom.edu/
26
SANTE/HEALTH CANADA
http://www.hc-sc.gc.ca/index-fra.php#tabs1_3
 TPD: Guidance Document - Acetylsalicylic Acid Labelling Standard
(Source: HC-SC)
 Health Canada is pleased to announce the release of the revised
Post-Notice of Compliance (NOC) Changes - Quality Guidance (Source: HC-SC)
 CADTH : CDR Update — Issue 95 (Source: CADTH)
 Health Canada: Therapeutic Products Directorate Statistical Report
2012/2013 for the Patented Medicines (Notice of Compliance)
Regulations and Data Protection (Source: HC-SC)
 CADTH: High-Sensitivity Cardiac Troponin for the Rapid Diagnosis of
Acute Coronary Syndrome in the Emergency Department (Source: CADTH)
LATIN AMERICA
SSA http://www.salud.gob.mx/
 Faes Farma sets up JV in Mexico
Spain's Faes Farma is looking to expand in Latin America by setting up a joint venture with Mexico's
Laboratorios Senosiain.
The 50:50 JV will be called MIT Farma and is an extension of the companies' 15-year relationship which has
seen Senosiain sell the Bilbao-based firm's vasoproective product hidrosmine.
Faes says "we are convinced that Senosiain "is the best possible partner", in Mexico where it is the leading
domestic pharmaceutical company and the fifth biggest drugmaker, including multinationals. … (Source:
PharmaTimes)
http://portal.anvisa.gov.br/wps/portal/anvisa/home
27
 Anvisa Sees Sectoral Agreements as New Form of Regulation
(Source:
ANVISA)
 RPT-Brazil to produce measles/rubella vaccine for poor countries
Brazil’s top biomedical research and development center announced plans on Monday to produce a
combined measles and rubella vaccine for developing countries, mainly in Africa.
The first Brazilian vaccine developed specifically for export will be made by Bio-Manguinhos, a unit of the
Oswaldo Cruz Foundation (Fiocruz), in partnership with the Bill & Melinda Gates Foundation. … (Source:
Reuters)
AFRICA
 UEMOA - UNION ECONOMIQUE ET MONETAIRE OUEST
AFRICAINE
http://www.uemoa.int/Pages/Home.aspx
 REGLEMENT N°02/2005/CM/UEMOA RELATIF A L'HARMONISATION
DE LA REGLEMENTATION PHARMACEUTIQUE DANS LES ETATS
MEMBRES DE L'UEMOA (Source: UEMOA)
28
 REGLEMENT N° 06/2010/ CM/ UEMOA RELATIF AUX PROCEDURES
D’HOMOLOGATION DES PRODUITS PHARMACEUTIQUES A USAGE
HUMAIN DANS LES ETATS MEMBRES DE L’UEMOA (Source: UEMOA)
 Fiscalité des médicaments, consommables et équipements médicaux
dans les pays membres de l’UEMOA (Source: Cerdi)
 L’Afrique de l’Ouest: des opportunités d’affaires à découvrir
(Source:
SlideShare)
https://www.dphm.ci/fr/dpm-c%C3%B4te-divoire-3
L'arrêté N° 297 MSP/ CAB/ DGS/ DPM du 13 DEC 2006 fixe l’organisation, les attributions et le
fonctionnement de la Direction de la Pharmacie et du Médicament en abrégé (DPM), conformément
au decrét N° 2006-33 du 08 mars 2006, portant organisation du Ministère de la Santé et de l’Hygiène
Publique.
La Direction de la Pharmacie et du Médicament , en abrégé, DPM, exerçant sous l’autorité de la Direction
Générale de la santé, a pour missions d’élaborer, de mettre en œuvre et de veiller à l’application de la
politique pharmaceutique nationale ;
A ce titre, elle est chargée :
· De l’élaboration des projets de textes législatifs et réglementaires en matière d’enregistrement
de médicaments y compris des substances vénéneuses,des produits diététiques, cosmétiques
et d’hygiène dans les secteurs publics et privés ;
· De l’application des Conventions et Traités internationaux relatifs aux stupéfiants et aux
substances psychotropes ;
· De la réglementation des professions de pharmacie et de laboratoires d’analyses médicales ;
· De l’établissement et de l’entretien des relations avec les organisations professionnelles de
pharmacie et de laboratoires d’analyses médicales ;
· De l’organisation de la pharmacovigilance ;
· De l’organisation de la lutte contre le trafic illicite de médicaments, stupéfiants et substances
psychotropes ainsi que de la participation à la lutte contre la toxicomanie ;
· De la promotion et de la mise en œuvre du plan de développement de l’industrie
pharmaceutique ;
· Du secrétariat de divers commissions notamment la commission de programmation des
créations, des transferts, des gérances et des ventes d’officine de pharmacie et de laboratoires
d’analyses médicales ; la commission nationale de thérapeutique ; la commission nationale de
pharmacovigilance ; la commission d’enregistrement des médicaments ; la coordination des
pays de la zone franc et les pays associés sur la politique du médicament.
29
 Promotion de l'Industrie Pharmaceutique (Source : DPHM)
 Pharmacovigilance et Lutte contre les Médicaments Illicites
(Source:
DPHM)
AUSTRALIA – NEW ZELAND
http://tga.gov.au/
 Biosimilar medicines - information for health professionals (Source: TGA)
 Consultation: Invitation for public comment - ACCS and joint
ACCS/ACMS meetings, November 2013 (Source: TGA)
 Macutec - Stiltec Pty Ltd - Complaint No. 2011-11-017
Background: Regulation 9 of the Therapeutic Goods Regulations 1990 (the Regulations) permits the
Secretary of the Department of Health (the Secretary) or her delegate to order a person, such as the
advertiser of therapeutic goods, to undertake certain actions related to the advertising of therapeutic
goods. Such an order can only be made on the recommendation of the Complaints Resolution Panel (the
Panel), which is a statutory committee that examines complaints about advertisements for therapeutic
goods.
On 21 August 2013, the TGA delegate of the Secretary (the Delegate) made an order in relation to Stiltec
Pty Ltd about an internet advertisement at www.macutec.com.au regarding the product, Macutec. The
order was made following a recommendation by the Panel on 15 August 2012 in relation to a complaint
(No. 2011-11-017) made on 22 November 2011. The recommendation was made because Stiltec Pty Ltd
had not fully complied with the Panel’s determination issued on 15 June 2012. … (Source: TGA)
 Fifty new drugs listed on PBS
Fifty new and amended medicines have been added to the Pharmaceutical Benefits Scheme (PBS),
including cancer drugs.
Health Minister Peter Dutton says it means about 230,000 people can receive treatments they might not
otherwise have been able to afford.
The new listings include a ground-breaking treatment for melanoma skin cancer.
Dabrafenib, sold as Rafinlar, is the first melanoma medicine that targets a genetic mutation present in
about half of all melanoma cases.
Mr Dutton says the genetic testing needed to determine eligibility for the medicine will also be subsidised,
benefiting more than 800 Australians. … (Source: SMH)
30
 Role of the Advisory Committee on Prescription Medicines (ACPM) in
the TGA's regulatory decision making process (Source: TGA)
 TGA presentations given at the complementary medicines road
shows, July - August 2013 (Source: TGA)
 DSM opens Australia biologics plant
The contract manufacturing arm of the Dutch-based Royal DSM officially opened the $65 million facility
Wednesday. The first custom mammalian-based biopharmaceutical manufacturing facility in Australia, it is
a pet project of Australia's $345 million Translational Research Institute, which is being built to attract
medical research to Australia. The Dutch company has gotten support from the governments of
Queensland and the Commonwealth to build the plant. … (Source: FiercePharmaManufacturing)
ANZTPA http://www.anztpa.org/
INDIA - PAKISTAN & ASIA
China SFDA | Hong Kong MDCO & PSDH | India CDSCO | Japan MHLW | Korea KFDA | Malaysia MOH |
Philippines DOH | Singapore HSA | Taiwan TFDA | Thailand FDA | Vietnam MOH
http://www.pmda.go.jp/english/
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 The PMDA publishes a list of new drugs which are unapproved in
Japan but approved in the USA and the EU
The following drug types are in scope of this list; USA: NDA Chemical Types 1(New molecular entity (NME)
and Biologics license application (BLA); EU: New Active Substance (NAS)
Medicines not approved in Japan but approved in EU or US.xls
 B-MS files first all-oral, interferon-free HCV drug in Japan
Bristol-Myers Squibb Co has filed an all-oral combination in Japan for hepatitis C, seeking "the world’s first
interferon- and ribavirin-free treatment regimen" for the disease.
The submission with Japan's Pharmaceutical and Medical Devices Agency is for daclatasvir, a NS5A inhibitor
and the protease inhibitor asunaprevir. It is based on results from a Phase III study demonstrating that the
combo achieved an overall sustained virologic response 24 weeks after the end of treatment of 84.7% in
Japanese patients. … (Source: PharmaTimes)
 Bristol seeks Japan approval of all-oral hepatitis C treatment
The submission with Japan's Pharmaceutical and Medical Devices Agency marks the first time that any
drugmaker has filed for approval of a hepatitis C treatment regimen that does not include either of the
standard older treatments - the injected, difficult-to-tolerate interferon, or ribavirin, a pill. … (Source:
Reuters)
http://eng.sfda.gov.cn/WS03/CL0755/
 China Issues Revised GSP for Cold Chain Quality Control (Source: SFDA)
 CFDA Releases New Rules Governing Regulatory Process (Source: SFDA)
 GSK's Chinese executives, but not company, likely to face charges in
China - sources
Chinese police investigating allegations of widespread corrupt practices at GlaxoSmithKline Plc (GSK) are
likely to charge some of its Chinese executives but not the British drugmaker itself, legal and industry
sources said. … (Source: Reuters)
Food and Drug Administration - Department of Health
http://www.taiwan.gov.tw/ct.asp?xItem=25613&ctNode=1970&mp=1001
32
KFDA
http://www.kfda.go.kr/eng/index.do;jsessionid=8WaGRaioTcYSanehB1hMj6KOHjYV58zMGvaefbMlRWUQqdgxsIWA05GwBX1zZoJG
 Samsung BioLogics signe un partenariat de fabrication stratégique
avec Roche
Samsung BioLogics a annoncé aujourd'hui la signature d'un accord de fabrication stratégique à long terme
entre Samsung BioLogics et F. Hoffmann-La Roche. Les termes de cet accord prévoient la fabrication par
Samsung des médicaments biologiques commerciaux et exclusifs de Roche dans ses deux installations de
fabrication situées à Incheon, en Corée du Sud, dont l'une est en construction. Les conditions de l'entente,
y compris les modalités financières et les produits qui seront fabriqués, ne seront pas dévoilées. … (Source :
PRnewswire)
 WuXi adds two 2000-L bioreactors to Shanghai facility
WuXi PharmaTech (NYSE: WX), a leading pharmaceutical, biotechnology and medical device research and
development outsourcing company with operations in China and the United States, announced today that
it had completed a cell culture capacity expansion, including two 2000L disposable bioreactors that are
ready for cGMP manufacturing. Previously WuXi AppTec had built a state-of-the-art cGMP biologics
manufacturing facility with two cell culture suites containing a 500L and a 1000L disposable bioreactor,
respectively. As a result of this new expansion, WuXi AppTec operates the largest biologics facility with
disposable bioreactors in China and the largest disposable bioreactor in the world. The facility has passed
GMP audits from global clients and an audit by former U.S. FDA biologics inspectors. … (Source:
FiercePharmaManufacturing)
HSA
http://www.hsa.gov.sg/publish/hsaportal/en/home.html#page=tab1
33
CDSCO
http://www.cdsco.nic.in/
 India’s health ministry directed to re-evaluate 157 clinical trials (Source:
MedCityNews)
 After price cuts, big pharma companies focussing on brands,
distribution
Big pharmaceutical companies' profit margins may be under pressure because of a government-induced fall
in prices of dozens of medicines, but they could end up with a larger share of the market at the expense of
small-and-mediumsized drug firms.

Given the narrowed-down price differential between the prices of their drugs and those of small and medium
firms, big pharmaceutical companies are now focussing on their brands and strengthening their
distribution setup. … (Source: TheEconomicTimes)
 Department of Pharmaceuticals rejects drug price review pleas of
DRL, Abbott
The Department of Pharmaceuticals has rejected a review petition filed by leading drug makers, including
Dr Reddy's Laboratories (DRL) and Abbott Healthcare, on ceiling of prices on their products.
While Abbott Healthcare filed the petition against the fixing of ceiling price of its Phenobabitone tablets-60mg and 30 mg--and Promethazine Injection, 25mg/ml, DRL opposed fixing of the ceiling price of
Omeprazole 20 mg capsules. … (Source: TheEconomicsTimes)
 India slaps $787 per tn anti-dumping duty on Chinese paracetamol
India has imposed anti-dumping duty of $787 per tonne on import of Paracetamol, a widely used medicine,
from China for five years to protect interest of domestic players from the cheap shipments.
"The anti-dumping duty imposed (on Paracetamol) under this notification shall be effective for a period of
five years...," said the Central Board of Excise and Customs ( CBEC) in the Revenue Department. … (Source:
EconomicTimes)
34
 Patent office rejects BDR Pharma’s compulsory licensing application
The Patent Office rejected the compulsory licensing application of Mumbai-based BDR Pharmaceuticals to
make a generic version of US drug maker Bristol-Myers Squibb's anticancer drug Dasatinib, sold under the
brand name Sprycel.
The ruling comes after India last year issued its first compulsory licence to Natco Pharma to manufacture
the generic version of German drug maker Bayer's kidney cancer drug Nexavar. … (Source: EconomicTimes)
RESEARCH & DISCOVERY / BUSINESS DEVELOPMENT
 Drug Development Lags for Neglected Diseases
A new study published in The Lancet Global Health highlights what its authors call a 'fatal imbalance' in
research and development of treatments for the world's poorest patients.
The authors acknowledge that pharmaceutical and biotechnology companies have little financial incentive
to invest R & D funds in finding treatments or cures for neglected diseases, and point to a failure of public
policy to encourage them. They conclude, "Despite substantial political attention towards the burden of
neglected diseases, we detected no evidence of a substantial improvement in research and development
activity compared with previous decades." … (Source: VOA)
 UC Develops Unique Nano Carrier to Target Drug Delivery to Cancer
Cells
A unique nanostructure developed by a team of international researchers, including those at the University
of Cincinnati, promises improved all-in-one detection, diagnoses and drug-delivery treatment of cancer
cells.
The first-of-its-kind nanostructure is unusual because it can carry a variety of cancer-fighting
materials on its double-sided (Janus) surface and within its porous interior. Because of its unique structure,
the nano carrier can do all of the following:

Transport cancer-specific detection nanoparticles and biomarkers to a site within the body, e.g.,
the breast or the prostate. This promises earlier diagnosis than is possible with today’s tools.

Attach fluorescent marker materials to illuminate specific cancer cells, so that they are easier to
locate and find for treatment, whether drug delivery or surgery.
35

Deliver anti-cancer drugs for pinpoint targeted treatment of cancer cells, which should result in
few drug side effects. Currently, a cancer treatment like chemotherapy affects not only cancer cells
but healthy cells as well, leading to serious and often debilitating side effects.
… (Source: University of Cincinnati)
 Integrated DNA Technologies Launches its First Cancer Panel
Integrated DNA Technologies (IDT) is enabling cancer research with its latest next generation sequencing
(NGS) product, the xGen Acute Myeloid Leukemia Cancer Panel v1.0. Consisting of 11,743 xGen Lockdown
Probes, this cancer panel targets more than 260 clinically relevant genes that were found to be mutated in
a study of 200 patients with acute myeloid leukemia (AML), and published by The Cancer Genome Atlas
consortium. The AML Cancer Panel is used for enriching the genome for regions of interest before
performing NGS. It can, therefore, be used to study disease occurrence and progression, and help with the
development of better targeted therapies. … (Source: CPhI)
 Using Fluorescence Lifetime Imaging Microscopy to Monitor
Theranostic Nanoparticle Uptake and Intracellular Doxorubicin Release
Basuki J et Al. ACS Nano, Article ASAP DOI: 10.1021/nn404407g - Publication Date (Web): October
16, 2013
We describe the synthesis of iron oxide nanoparticles (IONPs) with excellent colloidal stability in both water
and serum, imparted by carefully designed grafted polymer shells. The polymer shells were built with
attached aldehyde functionality to enable the reversible attachment of doxorubicin (DOX) via imine bonds,
providing a controlled release mechanism for DOX in acidic environments. The IONPs were shown to be
readily taken up by cell lines (MCF-7 breast cancer cells and H1299 lung cancer cells), and intracellular
release of DOX was proven using in vitro fluorescence lifetime imaging microscopy (FLIM) measurements.
Using the fluorescence lifetime difference exhibited by native DOX ( 1 ns) compared to conjugated DOX (
4.6 ns), the intracellular release of conjugated DOX was in situ monitored in H1299 and was estimated
using phasor plot representation, showing a clear increase of native DOX with time. The results obtained
from FLIM were corroborated using confocal microscopy, clearly showing DOX accumulation in the nuclei.
The IONPs were also assessed as MRI negative contrast agents. We observed a significant change in the
transverse relaxivity properties of the IONPs, going from 220 to 390 mM–1 s–1, in the presence or absence of
conjugated DOX. This dependence of MRI signal on IONP-DOX/water interactions may be exploited in
future theranostic applications. The in vitro studies were then extended to monitor cell uptake of the DOX
loaded IONPs (IONP@P(HBA)-b-P(OEGA) + DOX) into two 3D multicellular tumor spheroids (MCS) grown
36
from two independent cell lines (MCF-7 and H1299) using multiphoton excitation microscopy. … (Source:
ACS)
 Cryo-EM Structure of a Fully Glycosylated Soluble Cleaved HIV-1
Envelope Trimer
Lyumkis D et Al. Science DOI: 10.1126/science.1245627
The HIV-1 envelope glycoprotein (Env) trimer contains the receptor binding sites and membrane fusion
machinery that introduce the viral genome into the host cell. As the only target for broadly neutralizing
antibodies (bnAbs), Env is a focus for rational vaccine design. We present a cryo-electron microscopy
reconstruction and structural model of a cleaved, soluble SOSIP gp140 trimer in complex with a CD4
binding site (CD4bs) bnAb, PGV04, at 5.8 Å resolution. The structure reveals the spatial arrangement of Env
components, including the V1/V2, V3, HR1 and HR2 domains, and shielding glycans. The structure also
provides insights into trimer assembly, gp120-gp41 interactions, and the CD4bs epitope cluster for bnAbs,
which covers a more extensive area and defines a more complex site of vulnerability than previously
described. … (Source: ScienceJournals)
 The Major Brain Cholesterol Metabolite 24(S)-Hydroxycholesterol Is a
Potent Allosteric Modulator of N-Methyl-d-Aspartate Receptors
Steven P. et Al. The Journal of Neuroscience, 30 October 2013, 33(44): 17290-17300; doi:
10.1523/JNEUROSCI.2619-13.2013
N-methyl-d-aspartate receptors (NMDARs) are glutamate-gated ion channels that are critical to the
regulation of excitatory synaptic function in the CNS. NMDARs govern experience-dependent synaptic
plasticity and have been implicated in the pathophysiology of various neuropsychiatric disorders including
the cognitive deficits of schizophrenia and certain forms of autism. Certain neurosteroids modulate
NMDARs experimentally but their low potency, poor selectivity, and very low brain concentrations make
them poor candidates as endogenous ligands or therapeutic agents. Here we show that the major brainderived cholesterol metabolite 24(S)-hydroxycholesterol (24(S)-HC) is a very potent, direct, and selective
positive allosteric modulator of NMDARs with a mechanism that does not overlap that of other allosteric
modulators. At submicromolar concentrations 24(S)-HC potentiates NMDAR-mediated EPSCs in rat
hippocampal neurons but fails to affect AMPAR or GABAA receptors (GABAARs)-mediated responses.
Cholesterol itself and other naturally occurring oxysterols present in brain do not modulate NMDARs at
concentrations ≤10 μm. In hippocampal slices, 24(S)-HC enhances the ability of subthreshold stimuli to
induce long-term potentiation (LTP). 24(S)-HC also reverses hippocampal LTP deficits induced by the
NMDAR channel blocker ketamine. Finally, we show that synthetic drug-like derivatives of 24(S)-HC, which
potently enhance NMDAR-mediated EPSCs and LTP, restore behavioral and cognitive deficits in rodents
treated with NMDAR channel blockers. Thus, 24(S)-HC may function as an endogenous modulator of
NMDARs acting at a novel oxysterol modulatory site that also represents a target for therapeutic drug
development. … (Source: TheJournalOfNeuroscience)
37
 Genetic basis of neurocognitive decline and reduced white-matter
integrity in normal human brain aging
Glahn D. et Al. doi: 10.1073/pnas.1313735110 PNAS November 4, 2013
Identification of genes associated with brain aging should markedly improve our understanding of the
biological processes that govern normal age-related decline. However, challenges to identifying genes that
facilitate successful brain aging are considerable, including a lack of established phenotypes and difficulties
in modeling the effects of aging per se, rather than genes that influence the underlying trait. In a large
cohort of randomly selected pedigrees (n = 1,129 subjects), we documented profound aging effects from
young adulthood to old age (18–83 y) on neurocognitive ability and diffusion-based white-matter
measures. Despite significant phenotypic correlation between white-matter integrity and tests of
processing speed, working memory, declarative memory, and intelligence, no evidence for pleiotropy
between these classes of phenotypes was observed. Applying an advanced quantitative gene-byenvironment interaction analysis where age is treated as an environmental factor, we demonstrate a
heritable basis for neurocognitive deterioration as a function of age. Furthermore, by decomposing geneby-aging (G × A) interactions, we infer that different genes influence some neurocognitive traits as a
function of age, whereas other neurocognitive traits are influenced by the same genes, but to differential
levels, from young adulthood to old age. In contrast, increasing white-matter incoherence with age appears
to be nongenetic. These results clearly demonstrate that traits sensitive to the genetic influences on brain
aging can be identified, a critical first step in delineating the biological mechanisms of successful aging. …
(Source: PNAS)
 BI HCV drug faldaprevir shows promise
Boehringer Ingelheim has presented new data from a Phase III trial programme which shows that
faldaprevir was highly effective in a broad range of patients with genotype-1 hepatitis C.
The study, the data from which were presented at the Liver Meeting in Washington DC, evaluated
faldaprevir in combination with pegylated interferon and ribavirin in treatment-naive and experienced
patients as well as in HIV co-infected participants.
Among the highlights of the data, in the STARTVerso 1 and 2 trials, 84% of treatment-naïve patients
receiving faldaprevir were able to shorten the total time on treatment from 48 to 24 weeks and 83% of
these patients achieved viral cure (SVR12). Interim results from STARTVerso 4 showed that 74% of patients
with HCV/HIV co-infection had undetectable HCV four weeks after the conclusion of treatment. … (Source:
PharmaTimes)
38
Le Cabinet WHITE-TILLET est enregistré pour la formation et agréé
pour le Crédit Impôt Recherche (lorsque les prestations le justifient)
39
Experience & Expertise
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