European Biotechnology News 6-7/2013 – Free Excerpt: Rules unklock access to Orphan drugs

Nº 6-7 | Volume 12 | 2013 | 10.00 € | ISSN 1618-8276 | A 60711 |

European Biotechnology News Science & Industry

CENTRAL EUROPE

Billionaires buy Merck Serono site for biotech campus NORTHERN EUROPE

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AstraZeneca enters fish-oil drug arena with a340m deal Special

Single use equipment – disposables for life sciences WESTERN EUROPE

GSK enters partnership with US agency for dual use pathogens SOUTHERN EUROPE

Development in Italy’s biotech sector hits the skids Eastern EUROPE

Rules unlock access to Orphan drugs

Hungarian and Polish biotech associations join forces science & technology

New protein transistor set to revolutionise sequencing

Peace of mind with triple-A polishing technologies

UVivatec 速 virus inactivation, Sartobind速 membrane chromatography and Virosart速 filtration represent today's spearheading technologies for quantitative virus and contaminant clearance. Together they form a unique orthogonal platform for state of the art polishing of biopharmaceuticals.

www.sartorius-stedim.com/polishing-technologies

N –º 6-7 | Volume 12 | 2013

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Intro

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Editorial

Another step towards increasing patient access to orphan drugs?

Wills Hughes-Wilson, Chair of the European Industry Task Force on Orphan Drugs & Rare Diseases, Brussels

A

Wills Hughes-Wilson is Vice President External Relations & Chief Patient Access Officer at Swedish Orphan Biovitrum (Sobi).

She is also Chair of European Industry Task Force on Orphan Drugs & Rare Diseases – a joint industry trade association group between EBE-EFPIA and EuropaBio, and is also an Industry Member of the European Commission’s EU Committee of Experts on Rare Diseases (EUCERD).

s part of the outcomes of the European Commission’s Process on Corporate Responsibility in the Field of Pharma­ ceuticals from 17 April 2013, Member States formally endorsed recommendations on a po­ tential coordinated and collaborative approach to address access to orphan medicinal prod­ ucts: the Mechanism of Coordinated Access to Orphan Medicinal Products (MOCA). These recommendations – which have been developed collaboratively by a group of EU Member States, patient organisations, industry representatives and other stakeholder representatives – are aimed at exploring ways to increase patient access to Orphan Medicinal Products (OMPs) through cross-border cooperation.

The key conclusions and deliverables include a proposal for a “Transparent Value Frame­ work” (TVF), which would form the basis for a structured discussion between all stakeholders around the value of an individual OMP. This would create a shared understanding between stakeholders, laying the foundations for differ­ ent pricing and reimbursement discussions in individual countries.

A variety of aspects should be taken into ac­ count during this process, including the sever­ ity of the disease, the availability of other treat­ ments as well as the impact of the new treatment on disease progression. Rarity is also a key as­ pect, because it increases complexity through­ out the process. In many cases, an OMP might be the only available treatment for the rare dis­ ease in question. Therefore, the ability to assess its value and to make it available within a na­ tional healthcare system in a timely way is vi­ tal. The TVF attempts to capture these points and to provide the beginning of a framework for discussions around the value of an individual OMP in national healthcare systems.

The multi-stakeholder dialogue surrounding the shared challenges in ensuring that OMPs get to patients in a timely and sustainable way is also a key deliverable of the two-year MOCA process. Improved cooperation between stakeholders and across geographical bounda­ ries is vital to giving European patients with rare diseases access to innovative and effec­ tive therapies.

The MOCA recommendations will, how­ ever, only create meaningful change for pa­ tients if there is a concrete strategy and ac­ tion plan to develop the next steps that must be taken to have the necessary multi-criteria and multi-stakeholder discussions involving OMPs. Many issues still have to be resolved, and a number of steps still need to be taken to improve access to OMPs in Europe – includ­ ing the need to ensure that Health Technology Assessments are able to effectively capture the value of an OMP for patients.

This sector is affected by a multitude of dif­ ferent factors, systems and actors. Each stake­ holder within the process has a part to play. But only through a collaborative and connected ap­ proach involving all stakeholders – industry, patients, national HTA agencies, clinicians, payers and national healthcare authorities – will we be able to ensure that patients have ac­ cess to the treatments they need. The joint work on the shared challenges over the past two years should now lead to the next steps as we move to increase access to rare disease treatments across Europe. Industry is looking forward to being part of that ongoing process.

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EuroBiotechNews covers the biotechnology sector of the current 27 EU member states, Norway and Switzerland. If you would like to subscribe, please refer to Portugal: www.apbio.pt

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N –º 6-7 | Volume 12 | 2013

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Euro|Biotech|News

contents

FREE EXCERPT

Insight

Cover Story

IMI reports progress; MEPs won’t talk about GMOs with US; Heard in Brussels

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EU sets rules for risk assessment of GM animals; Bad bugs on the rise

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Biofuel producers lobby against ILUC 12 ERA-Net Synbio launches call; Stricter controls on GM rice; IP Flash

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Regulatory Affairs Update on clinical trials

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Breaking news from the EMA

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Economic Focus on newsflow and dividends

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Stock markets

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Perspectives EU companies head for Asia

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After more than two years of negotiations, a collaboration involving Member States, the European Commission, patient representative organisations, national insurers and industry has finally published the first non-binding guidelines for evaluating and reimbursing orphan drugs. The framework is a first tentative step forward in addressing a thorny and divisive topic. How much should pharma companies be allowed to earn for treating rare diseases? And how much can insurers afford to pay?

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Fujifim takes single-use plant online 44 Sequencing breakthrough

Putting a fair price-tag on orphan drugs

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Regional News

Services

Special

Partners & Associations

Single-use equipment Growing with disposables

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Interview: Uwe Gottschalk, Sartorius Stedim

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Corning: Systematic expansion

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Northern Europe

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Central Europe

22

Western Europe

24

Multifaceted solution

38

Southern Europe

26

Continous mAb processing

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Eastern Europe

28

Effective cell removal

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Biopeople News from Endocyte, AiCuris, Stratified Medicine Scotland Innovation Centre, Merck Millipore, European Bioplastics, and BioInvent International AB 45 Company index

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Events What’s on in June–July 2013

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Encore

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Imprint European Biotechnology News is published monthly by: BIOCOM AG, Lützowstr. 33–36, D-10785 Berlin, Germany, Tel.: +49-30-264921-0, Fax: +49-30-264921-11, E-Mail: service@eurobiotechnews. eu; Internet: www.eurobiotechnews.eu, Publisher: Andreas Mietzsch, Editorial Team: Thomas Gabrielczyk (Managing Editor), Derrick Williams (Co-editor), Dr. Patrick Dieckhoff, Dr. Bernd Kaltwaßer, Dr. Martin Laqua; Advertising: Oliver Schnell, +49-30-2649-2145, Advertising USA: Avani Media, Inc. , Leslie Hallanan, Tel.: +1-415-331-2150 , Fax: +1-415-289-0402, E-Mail: leslie@avanimedia.com; Distribution: Marcus Laschke, +49-30-2649-2148; Printed at: Druckhaus Humburg, Bremen; Graphic Design: Michaela Reblin. European Biotechnology Science & Industry News is only regularly available through subscription at BIOCOM AG. Annual subscription fees: € 100.00, Students € 50.00 (subject to proof of enrolment). Prices include VAT, postage & packaging. Ordered subscriptions can be cancelled within 2 weeks directly at BIOCOM AG. The subscription is initially valid for one year. Subscriptions will be renewed automatically for one more year, respectively, unless they are cancelled at least 6 weeks before the date of expiry. Failures of delivery which BIOCOM AG is not responsible for do not entitle the subscriber to delivery or reimbursement of pre-paid fees. Seat of court is Berlin, Germany. As regards contents: individually named articles are published within the sole responsibility of their respective authors. All material published is protected by copyright. No article or part thereof may be reproduced in any way or processed, copied and proliferated by electronic means without the prior written consent of the publisher. Cover Photo: © Mopic / Fotolia.com ® BIOCOM is a registered trademark of BIOCOM AG, Berlin, Germany.

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INSIGHT EUROPE IMI

Progress report

Heard in Brussels M

A Brussels – The Innovative Medicines Initiative (IMI) has provided an update of what’s going on in its current funding projects. At the beginning of May, the public-private partnership of the EU pharma association EFPIA with the European Commission reported that its researchers had identified neuroligin-3 as a marker for congenital autism in a mouse model in the course of the a36m project EU-AIMS, which kicked off in April 2012. The initiative also reported that clinical trials in which drugs against depression or schizo­ phrenia are compared to placebo have been shortened by 30% within the a22m project NEWMEDS, which started in September 2009. According to the IMI, the finding will cut the time for identification of patients who respond to the therapies.

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US/Eu trade Agreement

No-go zones A Strasbourg – The European Parliament (EP) has defined topics within the Trans­ atlantic Trade and Investment Partnership (TTIP) that it wants to exclude from the EU-US free trade agreement. At the end of May, MEPs passed a non-binding resolution urging the European Commission not to change existing EU rules on GMOs and cloning in negotiations with the US expected to start in July. MEPs emphasised “the sensitivity of certain fields of negotiation, such as the agricultural sector, where perceptions of Genetically Modified Organisms, cloning and consumer health tend to diverge between the US and the EU…” and stressed that “the agreement must not undermine the fundamental values of either side...(like) the precautionary principle in the EU.” EU Trade Commissioner Karel De Gucht rejected the idea of excluding discussions on the topics. The Commission needs EP approval for the agreement to come into force.

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Genetic testing: should consumers have the right to decide?

Brussels – Europe’s consumers have an often turbulent relationship with science.

Advice from scientists that clashes with lifestyle preferences finds governments reluctant to appear as over­-zealous legislators, especially if it makes them unpopular with either groups of voters or the large companies that are often associated with the particular bit of lifestyle under threat. Things like high fat foods, alcohol and tobacco all have their proponents – some very vocal.

Ironically, in a situation where scientists have generally said “it’s OK” – in the case of food from GM crops – people didn’t believe them. Of course, hysterical media reports and poor company management of the situation didn’t help. Now we have an interesting scenario where it could be the other way round: the question of accessibility in direct-to-consumer (DTC) genetic testing without face-to-face counselling.

Caution from clinicians A survey conducted by INSERM and the KU Leuven has revealed that the vast majority of clinicians would prefer such testing to be linked to face-to-face genetic counselling (see p. 12). Where the associated condition was untreatable or unpreventable, almost 100% of clinicians wanted this direct patient contact. Indeed, the German Ethics Council recently recommended to its government to lobby for international rules on DTC testing.

Of course, DTC is a growing business in Europe – a shift towards US healthcare models where patients receive direct marketing on all aspects of healthcare (OMG, have you seen the adverts?). The quandary for European governments is around their favourite catch-phrase, “consumer choice”. Should consumers be free to take genetic tests without clinical advice, and possibly make poor lifestyle or treatment

Claire Skentelbery, Secretary General of the European Biotechnology Network

decisions based on a meaningless result?

We all know people who have had food intolerance testing, and subsequently decided to stop eating nine out of ten food groups because a nice man off the Internet said they would probably blow up and die if they continued to eat bread.

However, legislating on precautionary advice from clinicians risks annoying consumers who might like to take a test to see if they are special, as well as of course those nice (hopefully) tax-paying companies that provide such tests. I suspect that national governments will fail under pressure from them, so this is something that Brussels can step up to do. Because it is the right thing to do.

Being the bad guy One thing Brussels is very good at doing is taking the blame for implementing legislation at the European level on nationally unpopular issues like pollution, water quality or human rights. So it might as well do the right thing on genetic counselling, and add it to the list for beleaguered national politicians to rant about.

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INSIGHT EUROPE

News M

Biosimilar comparators

London – The EMA has published a draft guideline on biosimilar medicines pointing out the conditions for comparators authorised outside of the EEA. According to the EMA, which has requested input from the industry before 31 October, analytical data on in vivo non-clinical studies with the comparator must demonstrate comparability between the biosimilar, the non-EU reference product and the EU­-approved originator reference product. With the new rules, the EMA wants to prevent double work for the developers of biosimilars.

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Banning DTC testing

Brussels – A survey published at the end of May has revealed that the vast majority of European clinical geneticists reject direct-to-consumer (DTC) genetic testing offered over the Internet without prior faceto-face counselling (Genome Medicine, doi:10.1186/gm449). A poll taken among 131 geneticists across the EU shows that DTC testing for monogenetic and complex diseases is already widespread. 44% of the patients who had asked their doctor for genetic counselling had previously already taken a DTC test. 84% of the responders were not in favour of replacing face-to-face supervision by a physician with supervision via telephone, which is offered by some DTC testing firms. Most clinical geneticists also found it unacceptable to provide non-face-to-face medical supervision for pre-symptomatic tests in conditions with very high penetrance, predictive tests for a condition that has “medium” penetrance, for carrier testing, or for conditions that cannot be prevented or treated.

FREE EXCERPT

EUropean union

Biofuel producers take on ILUC A Brussels – This summer the European Commission is expected to translate its new biofuel legislation into law, and the bio­fuel industry is already protesting plans to establish a new certificate for biofuels that also takes into account indirect effects in their life-cycle assessments. According to the Commission, the demand for crop-based fuel is driving deforestation and food insecurity in other parts of the world – and that has to be taken into account when assessing the sustainability of biofuels.

Based on shaky science In mid-May, the two German biodiesel and biofuel industry lobby groups OVID and VDB made another attempt to stop the Commission’s plans, attacking the incorporation of the so-called ILUC factors in the EU’s Renewable Energy Directive.

That’s unsurprising, since if established, ILUC would significantly corrupt the eco­footprint of most first-generation bio­fuels, such as bioethanol made from maize. If the new rules came into force, admixtures of rapeseed bio­diesel to conventional fuel would be prohibited beginning in 2017.

Presented by the well-known expert Matthias Finkenbeiner (TU Berlin), the new study commissioned by OVID and VDB says that ILUC effects cannot be measured exactly, and therefore do not provide a suitable foundation for policy decisions. “There

is hardly any fact-based support for a scientifically robust and consistent inclusion of ILUC factors into life-cycle assessment and carbon footprints,” said Finkenbeiner.

The study argues that ILUC effects cannot be measured and quantified, and insists economic land-use models cannot separate direct from indirect land use. According to Finkenbeiner “there is full agreement in the scientific community that ILUC factors are highly uncertain. The level of cruelty for characterising the uncertainties stretches from significant to enormous.” Additionally, the study identifies significant variations in the ILUC factors attributed to specific biofuels. “ILUC factors are a hasty reaction in method development, and an arbitrary choice for decisionmaking,” concludes Finkenbeiner.

Industry associations from Poland, Portugal, Spain and the UK back the study’s conclusion that ILUC is based on unsound science. In April, they published a joint position paper together with the Germans prompting the Commission to adhere to its former target – to have 10% of all transport energy come from renewable sources. According to the industry, billions of euros have been invested and hundreds of thousands jobs will be affected by the decision. In contrast, the Commission has cited 15 studies that it says confirm that its current biofuel policy has had an indirect impact on an area of land around the size of Denmark. 

© David Büttner - Fotolia.com

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N –º 6-7 | Volume 12 | 2013 FREE EXCERPT

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Company And Advertiser Inde x

4SC AG (GER)… ………………………………… 14

A AB Science SA (F)………………………………… 15 AbbVie Inc. (USA)………………………………… 16 Ablynx (B)… ……………………………………… 25 Addex Pharma (CH)… …………………………… 22 Aegerion Pharmaceuticals, Inc. (USA)…………… 16 Agennix AG (GER/USA)… ……………………… 19 AiCuris GmbH & Co KG (GER)…………………… 45 Algeta ASA (N)… ………………………………… 21 Alpharma (USA)…………………………………… 20 Amarin Corp plc (IRL)… ………………………… 20 Aradigm Corp. (USA)……………………………… 27 AstraZeneca AB (S/UK)…………… 15, 20, 21, 25 Austrian Orphan Pharma (A)… ………………… 25 Axis-Shield (UK)…………………………………… 26

B BASF SE (GER)… ……………………………… 20, 45 Bavarian Nordic A/S (DK)……………………… 16, 21 Bayer AG (GER)…………………………………… 21 Berlin Partner GmbH (GER)………………………… 9 BIOCOM AG (GER)… ………………………… 11, 17 Bioinnovation & ScanBalt Forum 2013 (PL)… … CP3 BioInvent International AB (S)…………………… 45 BioMedInvest AG (CH)…………………………… 23 BioMotiv LLC (USA)… …………………………… 45 BioPartners GmbH (GER)………………………… 16 BioPlan Associates, Inc. (USA)…………………… 31 Biosignal (AUS)… ………………………………… 45 Blueberry Therapeutics (UK)… ………………… 25 Boehringer Ingelheim (GER)… ………………… 23 Boehringer Ingelheim Venture Funds (GER)… … 26 Boston Consulting Group (USA)… ……………… 31 Braskem SA (BR)… ……………………………… 45 BTG plc (UK)……………………………………… 18

C Caixa Capital Risc (E)… …………………………

Calchan Ltd. (UK)… ………………………………

Cardio3 Biosciences (B)… ………………………

Celgene Europe Ltd. (UK)…………………………

Celon Pharma Ltd. (PL)……………………………

Cenix BioScience GmbH (GER)… ………………

CLS Behring GmbH(GER)…………………………

Cobra Biologics (UK)………………………………

Convergence Pharma (UK)… ……………………

Corning Life Sciences (USA)………………………

Cosmo Pharmaceuticals (I)… ……………………

CSM NV (NL)………………………………………

Cyclacel Limited (USA)……………………………

26 14 24 16 28 23 16 37 14 31 26 45 45

D DASGIP GmbH (GER)… …………………………

Debiopharm S.A. (CH)……………………………

Deutsche Messe AG (GER)… ……………………

DNA Dieta (PL)… …………………………………

Druid Consulting LLC (USA)………………………

31 23 15 28 45

E

O

Elan plc (IRL)……………………………………… 18 Endocyte Inc. (USA)… …………………………… 45 Eppendorf AG Bioprocess Center Europe (GER)… 35 Eppendorf AG (GER)…………………………… 31, 38 ERNST & YOUNG (GER)… ……………………… 26 EryDel SpA (I)… ………………………………… 27 European Biotechnology Network (B)…………… 30

Okairos AG (CH)… ………………………………

Omthera Pharmaceuticals (USA)…………………

Oncos Therapeutics Ltd. (FI)… …………………

Ophtotech Inc. (CH)………………………………

F Ferring BV (NL)… ………………………………… 26 FGK Clinical Research GmbH (GER)… ………… 23 Filtrox AG (CH)… ………………………………… 31 Fujifilm Diosynth Biotechnologies (UK)… ……33, 44

P Pharmacia Corp. (USA)……………………………

PharmaMar (E)… …………………………………

Pharming Group N.V. (NL)… ……………………

Probi AB (S)… ……………………………………

Prosensa BV (NL)… ………………………………

Prosonix plc (UK)… ………………………………

45 27 15 45 18 14

Q/R

G GE Healthcare (UK)… …………………………… 31 Genmab A/S (DK)……………………………… 14, 19 Genovive Inc. (USA)……………………………… 28 GlaxoSmithKline (UK)… …………… 15, 20, 23, 24 Grifols S.A. (E)… ………………………………… 27

H Helsinn Healthcare S.A. (CH)… ………………… 23 Horizon Discovery Ltd. (UK)……………………… 21 Hospira One 2 One Global Pharmaceutical (USA)… 7

I ID Invest Partners (F)… …………………………

Illumina Inc. (USA)…………………………………

Imagen Biotech (UK)………………………………

Integromics S.L. (E)… ……………………………

InterMune Inc. (USA)……………………………

Ion Beam Application S.A. (B)……………………

Iroko Pharmaceuticals LLC (USA)…………………

23 20 16 23

26 46 25 27 16 25 45

J/K Janssen Biotech Inc. (USA)… …………………… 14 Janssen Pharmaceutica N.V. (B)… ……………… 22 Kurma Life Sciences Partners (E)… …………… 26

L/M Life Science Partners (GER)……………………… 23 Life Technologies Corp. (USA)…………………… 45 Merck Millipore Corp. (GER/USA)… …… 31, 40, 45 Merck-Serono (GER)……………………………… 22 Merieux Developpement SAS (F)… …………… 25 Monsanto (USA)… ……………………………… 13 MorphoSys AG (GER)… ………………………… 14 Myriad Genetics (USA)…………………………… 15

N Neurophage Ph. Inc. (USA)… …………………… 25 Nordion Inc. (UK)… ……………………………… 18 Novartis AG (CH)… …………………………… 15, 25 Novartis Europharm Ltd. (UK)…………………… 16 Novartis Venture Fund (CH)……………………… 23 Novo Nordisk A/S (DK)…………………… 14, 21, 23 Nutripharma (N)… ……………………………… 45

Qiagen NV (NL/GER)… ………………………… 46 Redx Pharma (UK)………………………………… 25 Reed Exhibitions Japan Ltd (JP)… ……………… 43 Regeneron Pharmaceuticals Inc. (USA)… ……… 24 ReNeuron Group plc (UK)… …………………… 16 Roche AG (CH)… ………………… 14, 15, 25, 43, 46 Roche Diagnostics GmbH (GER)………………… CP4 Roquette Group (F)… …………………………… 45

S Sanofi SA (F)…………………………………… 24, 43 Santarus Inc. (SA)………………………………… 26 Sartorius Stedim Biotech GmbH (GER)… CP, 2, 31, 32 STAT Diagnostica & Innovation SL (E)…………… 26 Swedish Orphan Biovitrum AB (S)… ……………… 3 Symphogen A/S (DK)… ………………………… 20 Synageva BioPharma Ltd. (USA)… ……………… 16

T TauRx Therapeutics (UK)………………………… 15 Thermo Fisher Scientific (UK)… ………… 31, 45, 47

U UBS ETF (CH)… ………………………………… 17 Ugichem GmbH (A)… …………………………… 23 uniQure BV (NL)… ……………………………… 25

V Veloxis Pharmaceuticals A/S (S)… ……………… 15 Versant Ventures (USA)………………………… 23 Vital Therapies Ltd. (USA)………………………… 16

X Xcellerex (USA)… ……………………………… 31, 39 Xellia Pharmaceuticals ApS (DK)………………… 20

Y Yakult (JP)… ……………………………………… 14 Ysios Capital Partners (E)………………………… 26

Z Zealand Pharmaceuticals A/S (DK)……………… 23 Zeltia Group (E)…………………………………… 27

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