Archives for the month of: May, 2016

SanFranBIO

There’s now less than a week to go until the biggest biotech event of the year, as BIO2016 heads to San Francisco. This year myself and Jane Thurgood will be there representing the BIA, joining a host of UK companies attending the conference – traditionally the second largest international delegation, second only to Canada.

If you’re one of the 15,000 delegates attending and missed our BIO webinar explainer last week, you can catch up here. It’s well worth a watch and details all the “need to knows” in a 30 minute round-up from myself, Diana DeJoy of BIO, John Lownds from UKTI and One Nucleus’ Harriet Fear. If you can’t spare the time, here are some quick-fire tips to help you out in San Francisco:

  • Transport: When you arrive into San Francisco International airport, you can travel directly to downtown San Francisco by Bay Area Rapid Transit (BART). If you’re arriving in rush hour avoid a taxi! Those of you who plan to travel more widely around the Bay Area should consider a Clippercard – San Francisco’s answer to the Oyster card.
  • The UK stand: UKTI’s stand is located in the North building, number 5744. Visit the UK stand for meetings and partnering. The stand reception will take place on Tuesday from 17.00 PST.
  • Partnering: For the BIO veterans amongst you, the main difference to note this year is that partnering will start from 13.00 PST on Monday. Partnering is located in both the West and South Buildings.

A couple of additional highlights in terms of activities: the One Nucleus Breakfast Seminar takes place on Monday morning from 08.00 PST; also on Monday morning, the London Stock Exchange host a breakfast seminar and panel discussion on ‘Raising Long-Term Growth Capital in London’; and on Tuesday evening head to the UKTI pavilion for the Life Sciences in the Northern Powerhouse Hospitality Reception, an informal drinks reception organised by Bionow.

There are some great UK speakers and talks at this year’s BIO, so do check them out if you’re able. And don’t forget to talk up the UK biotech industry yourselves while you’re out and about! I look forward to catching up with many of you out there next week.

Last week the UK was host to the European Conference on Rare Diseases & Orphan Products for the first time, as stakeholders from across the rare disease community gathered in Edinburgh. To coincide with the conference, the BIA launched a new briefing paper on Orphan Medicinal Products that looks into the 15 year application of the Orphan Regulation in Europe. The briefing paper explores the regulatory framework and the setting up of the Committee for Orphan Medicinal Products within the European Medicines Agency and raises awareness around the latest developments in this field.

The BIA continues to play an active role at EU level in influencing the regulations that have an impact on how UK bioscience tackles rare diseases. We have seen that the incentives of the Orphan Regulation have been essential in stimulating research and development of orphan medicines for rare diseases. It is therefore important that the requirements to demonstrate significant benefit of orphan medicines are appropriate, given the complexities of dealing with small patient populations and rare conditions to be treated. Read the press release here and download the briefing paper here.

I started last week in Oxford, meeting with member company CEOs, local academics and clinicians and the two local MPs Nicola Blackwood and Andrew Smith for a private roundtable discussion with Life Sciences Minister George Freeman. It was a really useful discussion on challenges and opportunities facing the sector and I was delighted to have a similar discussion with London based members and stakeholders with the Minister on Thursday.

The BIA is convening a series of these roundtables over the next month as part of a regional tour the Minister is undertaking to gain feedback from life sciences stakeholders on the issues facing the sector. Alongside these visits, George Freeman is making the case that the UK life sciences sector is stronger in the EU than outside it ahead of the 23 June vote and it’s been good to see individuals from the sector lending their voice to this message.

In other travels last week, BIA hosted another successful dinner – this time at the Red Lion in Hinxton, Cambridgeshire – giving our members the opportunity to network, enjoy some good food, and discuss matters of local and national importance to the sector.  It was good to see some familiar, and some new faces, and a very enjoyable evening was had by all.  We look forward to the next one.

Finally, it was great to see the launch of infocus from Innovate UK last week, the first women only Innovate UK competition and part of a new ‘infocus’ initiative to encourage diversity in innovation. There’s £200,000 on offer and a package of tailored support to award to businesswomen who have exciting ideas and the potential to become leaders in innovation and deliver significant economic growth – more information can be found on the website here.

Best

Steve

With only 10 days left to prepare for the biggest biotech event of the year, catch up on yesterday’s BIA webinar to find out how to make the most of your time at BIO 2016.

A must watch for BIO veterans and newbies, with speakers Steve Bates, BIA, Diana DeJoy, BIO, John Lownds, UK Trade & Investment (UKTI) and Harriet Fear, One Nucleus.

Do you have a video you would like the sector to see? Contact us.

Jordi_MHRAconf_audience

Following our joint conference with the MHRA, and continuing our regulatory theme on the BIA blog this month, today’s post takes a closer look at how the European Medicines Agency’s PRIME scheme is comparing to US and Japanese initiatives.

The last session of the day took an in-depth look at a recent area of innovation in regulation for the EMA: the Priority Medicines scheme (PRIME), which was launched in March 2016.

Like the adaptive pathway approach, PRIME works within the existing regulatory framework to accelerate the delivery of drugs that address high unmet need.

Jordi Llinares, Head of the EMA’s Product Development Scientific Support Department, explained that the three pillars of PRIME are patients, support for R&D, and a pan-European approach. The aim of PRIME is to facilitate earlier scientific and regulatory advice, develop a robust data generation strategy, and thus enable accelerated assessment.

Jordi_MHRAconfOnce a potential therapy receives PRIME designation, a Rapporteur is appointed for products at proof of concept to guide the company through every stage of the process, including meetings with relevant committees to discuss development options and regulatory strategy. Iterative scientific advice is also given throughout and there are fee incentives for SMEs.

Jordi said that the scheme had been well-received, with 18 eligibility submissions in its first applications round, 11 of which were from SMEs. All were in scope and of sufficient detail for assessment, Jordi said, but there was a problem with too few submitting paediatric investigation plans (PIPs) or waivers. Although it is very early days for PRIME, its initial popularity shows industry’s need for such a scheme.

PRIME is modelled on the US Federal Drug Agency’s (FDA) breakthrough therapy designation, launched in 2012, and follows in the footsteps of the Japanese SAKIGAKE strategy launched in 2014. All three schemes aim to facilitate discussions between developers and regulators earlier in the process and provide a more hands-on approach from regulators throughout.

Rhian_AmgenAs PRIME is very much in its infancy, the BIA and MHRA invited Rhian Thomas, Executive Director, Global Regulatory Affairs, Amgen, to speak of her company’s experience gaining approval of Blincyto. Rhian told the conference that the breakthrough therapy designation was valuable but developers shouldn’t under-estimate how labour-intensive the process is.

In the panel Q&A session, Jordi said that the EMA will be exchanging notes with the FDA to compare their schemes and working practices to see what they could learn from each other. Robert Hemmings, member of the EMA’s Committee for Medicinal Products for Human Use (CHMP), who was also on the panel, noted that the FDA has already been stretched by the popularity of the breakthrough designation but that the EMA was confident it could meet demand. But he also sounded a word of caution; developers should not expect Europe to achieve the same acceleration as the US.

MMIP banner

On the blog today, Ian McCubbin, Chair of the Medicines Manufacturing Industry Partnership (MMIP) introduces the Ministerial Advanced Therapies and Medicinal Products (ATMP) Manufacturing Taskforce. The Taskforce has been launched and held its first meeting, with a further meeting planned for June (see press release).

Introduction to the Taskforce

The Advanced Therapies Manufacturing Taskforce originated from the work of the Medicines Manufacturing Industry Partership (MMIP), it is co-chaired by George Freeman MP, Minister for Life Sciences, and by me, the Chair of the MMIP. It is a short-term group which will meet three times over 2016 with the aim of anchoring advanced therapy manufacturing and its associated supply chain in the UK. It will make a small number of targeted recommendations through an action and deliver plan to achieve this aim.

Taskforce membership

The group’s members include senior representatives from:

  • Office for Life Sciences
  • GSK
  • GE Healthcare
  • Pfizer
  • Astrazeneca
  • Cell and Gene Therapy Catapult
  • Centre for Process Innovation
  • Innovate UK
  • MRC
  • Institute for Manufacturing
  • MHRA
  • NHS Blood and Transplant
  • University College London
  • Oxford BioMedica
  • ReNeuron
  • Autolus
  • Cobra Biologics

Taskforce workstreams

The Taskforce has created three workstreams and appointed three leaders:

  • Technology and manufacturing (Keith Thompson, Cell and Gene Therapy Catapult)
    This workstream is looking at the current UK manufacturing position and considering a range of ideas from flexible grant and loan schemes to greater support infrastructure for companies.
  • People, skills & training (Jim Faulkner, Autolus)
    This is the most narrowly focused of the workstreams, it is looking at both training new people to enter the industry and repurposing existing skilled individuals to work specifically on ATMPs.
  • International competitiveness (Roger Connor, GSK)
    This workstream is looking at where intervention is required to support the UK’s ability to compete globally for manufacturing investment in advanced therapies. It is identifying where the UK is already strong and considering the UK’s process development capability, fiscal environment and the route to market for advanced therapies.

Next steps

The next meeting of the Ministerial Taskforce will be on Tuesday 28 June. At this meeting workstreams will submit initial proposals for recommendations to anchor advanced therapy manufacturing in the UK. After this meeting, details of a survey will be shared to ensure that the suggested actions reflect the views of the wider community. A final action and delivery plan will then be drafted ahead of the Taskforce’s third and final meeting on 2 November, with a release date for the plan and follow-up steps yet to be announced.

Get in touch

If you have any colleagues who would be interested in receiving updates on the work of the Taskforce or you would like to engage, then please get in touch with us by clicking this link.

 

AMR NCast header

Across the media last week, after two years of work the Review on Antimicrobial Resistance published its final report. Building on eight interim papers, this is the final report from Lord O’Neill’s Review, established by the UK Prime Minister David Cameron in 2014 to avoid the world being “cast back into the dark ages of medicine”. The report explores the mounting problem of antimicrobial resistance (AMR), why action is needed to combat it and then provides an overview of the solutions that the Review thinks should be implemented to curtail unnecessary use and increase the supply of new antimicrobials.

The BIA welcomes this comprehensive and thorough Review. The UK has a vibrant life science ecosystem of companies with the capabilities and ambition to make a real difference in tackling the global challenge of antimicrobial resistance. The new market incentives identified by the Review are key for life science entrepreneurs to deliver the next generation of antibiotics we all need. Read the reactions to the report from BIA members who are already playing an active role in tackling AMR here.

As predicted, the Queen’s Speech announced a Higher Education and Research Bill that will bring the seven Research Councils, Innovate UK, and a new body called Research England under one umbrella called UK Research and Innovation (UKRI). It was also separately announced that the outgoing Second Private Secretary to the Treasury, John Kingman, will lead UKRI as an interim Chair while it is being established. His appointment means someone who has shown a deep interest and support for UK bioscience translation is the new boss. The inclusion of the term ‘Innovation’ in the name of this new body is also a positive signal at the importance of translation. You can read more about the contents of the Queen’s Speech on our blog.

Celebrating innovation in the sector, last week the winners of both OneStart Europe and the BBSRC’s Innovator of the Year were revealed. It was great to be at the OneStart Europe Finals dinner to see Germany-based Captain T Cell crowned 2016 winners last Monday – mentored by BIA Board member Will West. Many congratulations to the team and to all the finalists who presented some fantastic pitches on the evening.

On the BBSRC’s Innovator of the Year, well done to Dr Martin Pule who was named BBSRC Most Promising Innovator 2016 for frontier innovations in T-cell medicine, technology used to found BIA member Autolus. It was also great to see technology from another BIA member, C4X Discovery, amongst the finalists with co-founder Dr Andrew Almond recognised for his work optimising the design and development of medicines. Some great examples of the high quality science being developed in the UK.

Look forward to seeing many of you at events in Oxford, London and Cambridge this week.

Best

Steve

Queens speech 2016

On Wednesday the Government set out its legislative agenda for the 2016-17 Parliament in the Queen’s Speech. It announced that bills will be introduced to reorganise the UK’s research and innovation funding system and to reduce unjustified patent challenges.

A reorganisation of research and innovation funding bodies

The UK’s research and innovation funding system is set to be reorganised by a new Higher Education and Research Bill this Parliament. It was the first bill introduced to Parliament following the Queen’s Speech.

The Department of Business, Innovation, and Skills also published a white paper last Monday setting out its intentions to bring the seven Research Councils, Innovate UK, and a new body called Research England under one umbrella called UK Research and Innovation (UKRI). The move follows a green paper and Sir Paul Nurse’s recommendations to improve coordination between funders in a similar way late last year. You can read the BIA’s submissions to both the green paper consultation and Nurse Review consultation here and here.

White paperThe nine Councils, as they will officially be called, will keep their branding, discipline-focus and separate budgets and leadership but many backroom functions will be merged, including grant administration. Research England will take over the university block grant funding, which includes support for technology transfer, from the Higher Education Funding Council for England (HEFCE), which is to be dissolved.

The nine Councils will be led by Executive Chairs – “high profile positions appointed by Ministers on the advice of UKRI’s Board and reporting to UKRI’s CEO”. The Chair of UKRI has already been announced as John Kingman, who is stepping down as Second Permanent Secretary to the Treasury in July. He will lead UKRI on an interim basis while it is being established. His appointment means someone who has shown a deep interest and support for UK bioscience translation is the new boss. The inclusion of the term ‘Innovation’ in the name of this new body is also a positive signal at the importance of translation.

The white paper is keen to highlight the importance and distinction of support for innovation in the new architecture. It states that Innovate UK’s business-facing focus will be enshrined in future legislation, which would replicate the functions in its current charter (this will also be done for the Research Councils). As now, the Secretary of State will allocate a hypothecated budget to Innovate UK and will also “consider the option” of nominating a member of the UKRI Board to lead in promoting and championing innovation and business interests and providing strategic support to the Innovate UK Executive Chair. The post holder would sit on the council of Innovate UK.

It’s positive to see that the Government has listened to BIA calls for the Research Councils and Innovate UK to keep their brands, autonomy, and distinct funding streams. We believe that such autonomy is essential to ensure they are led by and employ experts to manage the strategic and efficient use of public funds. However, the devil will be in the detail to assess whether this proposed structure is better placed than what we currently have to underpin the UK’s global competitiveness and attractiveness as a research destination.

From a business perspective, it is essential that Innovate UK retains its ability to support businesses to translate research and to innovate and scale up in order to generate wider economic benefit and is not seen as just a technology transfer office for the other Councils.

As these proposals are taken forward the BIA looks forward to working with Government, Parliament and wider stakeholders to ensure such optimal outcomes are delivered by these proposals.

Higher education reform

As the name suggests, the Higher Education and Research Bill will also make changes to the higher education system. These reforms make up the majority of the white paper in fact and are wide-ranging.

In brief, the Government’s intention is to create greater competition in the higher education sector by making it easier for new universities to be established and unsuccessful ones to close. It also intends to raise institutions’ focus on delivering high-quality teaching by linking performance to tuition fee rises, widen access to students from disadvantaged and under-represented backgrounds, and give students more power as customers.

Clamping down on unjustified patent challenges

An Intellectual Property (Unjustified Threats) Bill was announced in the Queen’s Speech and published the following day to reform the law around unjustified threats of infringement proceedings for patents, trademarks, and design rights. It is intended to provide greater clarity for right holders and third parties that become involved in IP disputes. The Government says that the bill will help create an IP environment that favours negotiation and settlement rather than litigation.

Threats can be made by organisations or individuals that believe their IP has been unlawfully used by another. However, threats can also be used to inappropriately intimidate others to gain an unfair competitive advantage. This can be a particular problem for smaller biotechnology companies that do not have the resources to fight large claims.

The legislation follows a detailed report and draft bill from the Law Commission. The bill will define what a threat is, create a “safe harbour” to discuss disputes, and provide protection to lawyers intended to allow them to better resolve disputes. It will also make a clearer distinction between approaches made to different parts of the supply chain, forcing the right holder to focus their allegations on the source of the alleged infringement.

The BIA’s Intellectual Property Advisory Committee will be assessing the proposed legislation and directing our engagement with the reforms.

After two years, yesterday the Review on Antimicrobial Resistance published its final report.

The report explores the mounting problem of antimicrobial resistance (AMR), why action is needed to combat it and then provides an overview of the solutions that the Review thinks should be implemented to curtail unnecessary use and increase the supply of new antimicrobials.

Read the reactions to the report from BIA members who are already playing an active role in tackling AMR here.

Our video of the week comes from the BBC, and examines just how big a threat superbugs are to modern life. Click on the image below to watch.

BBC AMR May 2016

Do you have a video you would like the sector to see? Contact us.

Following our joint conference with the MHRA, and continuing our regulatory theme on the BIA blog this month, today’s post takes a closer look at some of the European initiatives to increase early access to innovative medicines, including the adaptive pathways pilot.

The afternoon session started with an overview of recent activity at a European level, provided by Robert Hemmings, MHRA, who is a member of the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) and Chair of CHMP’s Scientific Advice Working Party.

RHemmingsRobert set out the challenges for regulators in this area, including the many stakeholders and competing demands agencies must balance. He said that early access must be sustainable as the cost of failure is high and it must be provided in a way that allows the collection of robust data after initial marketing authorisation.

Recognising that both national and EU-wide efforts are required, Robert highlighted the EU Medicines Agencies Network Strategy to 2020. This sets out joint key priorities and a high-level roadmap for how the regulatory agencies can work within their own country and with the EMA to achieve these and foster early access. Concluding his presentation, Robert said that there is a lot of activity ongoing and the challenge is putting all of the available tools for early access into a cohesive structure to help drug developers.

Adapting to adaptive pathways

Next, Francesca Cerreta, Senior Scientific Officer at the EMA, shared some learnings from her agency’s adaptive pathways pilot. The aim of the pilot was to engage in early dialogue with the relevant stakeholders to design a smart development programme that generates the relevant evidence base, using all data sources, so that decisions can be made quickly and seamlessly. A key challenge is bringing regulators, payers and health technology assessment (HTA) bodies together and aligning their different requirements in the programme design. The EMA also had a mixed experience with companies, with the information provided in applications often being “sketchy”, although SMEs have so far been the most creative when proposing solutions. Francesca said that although the adaptive pathways approach is valuable, it is applicable only in a limited number of cases where there is promise to fulfil unmet need and there are clear-cut, actionable endpoints.

FCerretta

Eliot Forster, CEO of Immunocore, provided an industry perspective on the EMA’s pilot. Immunocore have been engaged with the pilot for about a year and Eliot said he was very impressed so far. He believed it was the fastest way to get truly innovative therapies to patients. Eliot praised the practical approach taken by the EMA, adding that the conversations were timely and collegiate, with both sides learning throughout the process. He hoped this was a watershed moment for how regulators and developers could work together.

EForster

NICE’s Sarah Garner echoed Eliot’s view that the conversations as part of the adaptive pathways process had to be honest and focussed on coming up with solutions together. The adaptive approach provides NICE with the opportunity to inform the process so that the right real-world data can be collected, hopefully in the UK. She said there was political ambition in the UK for this to happen but sadly this is not true in all EU countries. The UK therefore has a real opportunity to capitalise on real-world data, she said, especially with the large number of registries we have and data-focussed institutes.

SGarner

The speakers from the session were joined on stage by Joy Duffen of the Cure Parkinson’s Trust and Christian Schneider from the National Institute for Biological Standards and Control (NIBSC) for a Q&A. Both Joy and Christian welcomed the adaptive pilot approach as being critically important for patients. In questions from the audience, the panel was asked whether there had been opportunities for treatment prices to be increased following successful data collection from the pilot. This hadn’t happened yet said Robert Hemmings, but a potentially-curative gene therapy might be the first chance for this in the coming year. Further discussion focussed on harmonising HTA processes across Europe but, as Sarah pointed out, healthcare systems vary massively across Member States making harmonisation very difficult. There will always be country-specific issues she thought.

Panel

For more information on upcoming BIA events go to the BIA website.

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In a new format to our usual Annual Lecture, last Thursday’s “An Evening With Clive Dix” was a fantastic opportunity to hear from one of our sector’s great entrepreneurs. Interviewed by his long term colleague and friend Brenda Reynolds, our very own Piers Morgan for the evening, it was great to hear Clive give a candid review and insight into his career in the industry – it’s one occasion where I have to say you needed to be there to get the insight, as a closed door event with no reporting led to a frank and personal perspective being openly shared. We think it worked as a format and look out for further events like this in future.

Two key policy events to look out for this week. The Queen’s Speech this Wednesday will announce the key pieces of legislation we can expect over the next couple of years. A higher education bill has been widely-trailed in the newspapers this morning and will include provisions to allow the creation of a new body to oversee the Research Councils and Innovate UK. The new body, expected to be called UK Research and Innovation (perhaps a clue about whether the plan is to roll Innovate UK into the new body), is intended to provide greater coordination between research funders but a Government statement says that they will maintain their identities and delegated budgets. A white paper setting out more detail has been published today and we will engage these developments over the coming months on the sectors behalf.

Jim O’Neill’s Review on Antimicrobial Resistance is expected to be published on Thursday. BIA members from large to small have been actively engaged in the policy making process here and I expect to see continued comment from all sides on the final report. It’s good to see UK policymakers and companies playing a leading role in shaping a globally vital discussion that will move swiftly to the World Health Assembly and Organisation and G20. We are particularly interested in developing new incentive models for drug discovery and development and it seems likely the review will suggest delinked incentives, pay or play provisions and priority review vouchers as models to be explored.

Information on the Early Access to Medicines Scheme including its principles and operational guidance developed by the stakeholder task group was published last week. You can download the document, ‘Early access to medicines scheme (EAMS): how the scheme works’, here. EAMS was one of the key topics discussed at our recent BIA MHRA joint conference – pictures from the day are now up on the blog along with a brief summary of the highlights, which you can visit here.

The European Medicines Agency (EMA) has published a report summarising the experience with its small and medium-sized enterprise (SME) initiative over the last ten years. It’s great to see that in terms of geographical distribution the UK remains the top country in Europe for SMEs from 2011 to 2015. It’s a key reason why it is important the Agency remains in London and continued to engage with the BIA and its members.  The initiative started in December 2005 when the SME Regulation came into force and the SME Office was established to address the needs of the smaller businesses in the pharmaceutical development ecosystem. To support SMEs throughout all stages of medicines’ development, EMA’s SME Office provides active regulatory, financial and administrative support to registered SMEs. The new report emphasizes the importance of SMEs in pharmaceutical innovation and draws attention to the trends observed in the past ten years.

On events, there’s now just over a week left to register for the 8th European Conference on Rare Diseases & Orphan Products (ECRD 2016), taking place in Edinburgh from 26 – 28 May 2016. The ECRD, organised by the European Organisation for Rare Diseases (EURORDIS) and co-organised by the Drug Information Association (DIA), unites all stakeholders of the rare disease community across all rare diseases and countries: patients, healthcare professionals and researchers, industry, payers, regulators and policy makers. ECRD 2016 will bring together over 80 speakers and more than 800 participants, covering six themes from the latest research, to developments in new treatments, to innovations in healthcare, social care and support at the European, national and regional levels. Find out more here.

Finally, last Friday marked the 2nd anniversary of the launch of the Concordat on Openness on Animal Research. I’d recommend taking a look though #ConcordatOpenness for some fantastic examples of the work being done by the UK bioscience community to maximise openness and communication on this important issue. A great result and effort over the past two years.

Best

Steve

Today marks the 2nd anniversary of the launch of the Concordat on Openness on Animal Research.

Over 100 signatories from across the UK bioscience sector, including the BIA, have now signed up to the Concordat, pledging to maximise openness and communication on this important issue.

Watch today’s video showcase from Understanding Animal Research, originally produced for the Politics.co.uk website to brief politicians and policy makers about animal research.

Do you have a video you would like the sector to see? Contact us.