Clinigen_debbie AingeEarlier this month, the International Society for Pharmaceutical Engineering (ISPE) launched a white paper offering guidance on the development and implementation of regulatory-compliant patient access programmes. Dr Debra Ainge, Head of Project Management at Clinigen Global Access Programs (Clinigen GAP), whose colleague  Mark Corbett chaired the interdisciplinary team behind the paper, outlines the rapidly evolving access programme space and the key groups involved in the supply of unlicensed medicine to patients with unmet medical needs.

Providing access to investigational products can be a challenging business for Pharma and Biotech companies.

The treatment of two Ebola victims with an experimental medicine in the US this month and the huge social media campaign surrounding a well-publicised plea for access to an anti-viral medication on behalf of a young patient with Adenovirus infection in the US has also raised awareness of the need for better understanding of access programmes across both industry and wider society.

An access programme is often needed most when a company is busy focussing on clinical trials and the approval, launch and commercialisation of a product when time, resources and the ability to scale up supply of the product is limited.  These programmes can also be extremely complex to set up, and many companies both large and small lack the infrastructure and experience to deliver the medicine.

Planning early is key, but companies can sometimes struggle to gain internal consensus on how a programme should be run. Education and the development of clear policies and procedures to ensure readiness to respond to requests in an ethical and compliant manner can help overcome this, and is an area where Clinigen GAP is frequently asked to provide support.

Early access – strategies for a successful programme

The white paper Improving Access to Patients with Unmet Medical Needs – Overview and Best Practices for Success, describes these regulatory-compliant mechanisms that enable Pharma and Biotech companies to provide access to investigational or unlicensed medicines to patients who have exhausted all other treatment options.

The paper looks at country-specific regulatory guidelines on access to medicines outside the clinical and commercial framework, patient eligibility guidance, stakeholder involvement, timing and logistics. The majority of countries have mechanisms in place to allow access to these medicines for those with serious of life-threatening conditions where no licensed alternatives are available; it is the navigation of these regulatory systems that can be the challenge.

Mark Corbett, who heads Clinigen GAP speaks regularly on these mechanisms and strategies for delivering successful early access programmes. He will present the findings of the white paper at the ISPE Annual Meeting and will speak at the BIA’s UK Bioscience Forum on early access to unlicensed drugs in October.

In November, Clinigen GAP will host a panel at BIO-Europe in Frankfurt, chaired by the BIA’s Steve Bates and featuring Luc Dochez, Chief Business Officer at Prosensa and Pfizer’s Lead Oncology Europe, Africa and the Middle East, Dr Markus Kosch, entitled ‘Mind the GAP? Meeting an emerging demand for access to medicines in late-stage clinical trials, prior to commercial launch’. The panel will explore the industry environment, regulatory environment with a focus on the new UK Early Access to Medicines Scheme (EAMS).

Chris Watkins, Director of Translational Research and Industry, MRC

Chris Watkins, Director of Translational Research and Industry, MRC

Last month the Medical Research Council (MRC) unveiled a new partnership with seven pharmaceutical companies to share deprioritised compounds for research in new disease areas. Here Chris Watkins, the MRC’s Director of Translational Research and Industry, explains how the collaboration came about and the role it plays in the MRC’s strategy for collaborating with industry.

One of the big challenges in medical research is understanding the biology of human disease. But there’s a problem with that. The best way of achieving this goal is to do experimental medicine studies in man, using molecules where you know the probable target and which you know are safe to give to study participants.

But these molecules are mainly developed by industry, once they’re pretty confident of the importance of the target. So how do you gain the evidence for the importance of the target? You need access to the molecules. That’s exactly what the current drug discovery landscape needs, and what the partnership we announced is aiming to do.

If we are to discover safer and more effective treatments, we need to bring together the strengths of academic researchers — with their study of the intricate mechanisms of diseases — with the experience of pharmaceutical companies in developing, testing and producing drugs.

In 2012 we announced a scheme to fund researchers to use 22 deprioritised compounds from AstraZeneca. Last month we built on this with a new initiative in which UK researchers will be granted access to a ‘virtual library’ of deprioritised pharmaceutical compounds offered up by AstraZeneca, GlaxoSmithKline, Janssen Research & Development LLC, Lilly, Pfizer, Takeda and UCB. We hope more will come on board, both large multinational biopharmaceutical companies and smaller biotechs.

The aim is to use these molecules in experimental medicine studies to improve our understanding of a range of diseases, revealing new therapeutic opportunities with a view to developing more effective treatments, either through repurposing or new drug development. The compounds will have undergone some degree of industry development, but have all stalled at some point in early testing for a particular disease. However, they may still be useful for other diseases with shared biological pathways. And because the compounds have already undergone some preliminary development, such as safety testing, any new treatments arising from the research could reach patients much faster.

The initial MRC/AstraZeneca scheme is already demonstrating success. More than 100 ideas came out of the academic research base in the UK, many novel and unprecedented in nature. It’s a great example of “crowdsourcing innovation”. The first human trials to see whether a drug designed to treat gastro-oesophageal reflux disease can be ‘repurposed’ to treat chronic cough are already underway in Manchester.

The rights to intellectual property generated using the compounds will vary from project to project, but will be equitable and similar to those currently used in academically led research. This kind of open innovation model is about ensuring that everyone involved receives appropriate recognition for their contribution.

I’m really excited by the opportunities that will undoubtedly emerge from this initiative. We’ve planned this to be a “rolling” activity over a number of years, allowing the best ideas to come forward at the right time. It also means that more companies can participate as they see the benefits of such an approach, not only in terms of the molecules included in the virtual library, but also the greater understanding of disease mechanisms which may be of relevance to their own R&D programmes.

We anticipate this approach will become an integral part of the world-leading science supported by the MRC, enabling ground-breaking discovery and clinical science to be undertaken by the best researchers in the UK. This will only be achieved through the partnership we have with the biopharmaceutical industry, who clearly see the benefits and opportunities in working in this collaborative way.

These recent collaborations are just some of the many ways that we fund academic researchers to work with industry to accelerate the development of treatments. I’ve written before about why the MRC collaborates with industry. These recent collaborations are just some of the many ways that we bring academic researchers and companies together to accelerate the development of treatments.

I hope that we will continue to drive forward innovation for patient benefit using inventive ways to enable researchers from academia and industry to collaborate together, breaking down barriers and playing to their respective strengths.

Chris Watkins

The full list of compounds will be published later this year when researchers will have the opportunity to apply to use them under our normal response-mode funding.

The BIA has eight established advisory committees who deal with a range of topics, from regenerative medicine to finance and tax. Meeting once a quarter, the committees are crucial mechanisms to highlight and explore key issues facing bioscience companies. Committee members include influential experts from across the sector. Their work informs and guides BIA policy and priorities, ensuring that member needs are met. 

This week we have updates on the activities of the Synthetic Biology Advisory Committee and the Communications  Advisory Committee.

Synthetic Biology Advisory Committee (SBAC)

This year SBAC has remained front and centre within the evolving UK and international synthetic biology ecosystem. In April the committee partnered with SynBioBeta to bring their conference to London, which was a huge success, and BIA with UKTI will be running a trade mission to the SynBioBeta in San Francisco this November. This is the premier global conference focused on translational synthetic biology. Although targeted principally at start-ups and SMEs, it also draws in larger companies, investors and academics in the field.

The committee has heard presentations from a number of key leaders in the field including those from the new Synthetic Biology Research Centres, the Innovation and Knowledge Centre in Synthetic Biology, government investors and the British Standards Institute. Bringing all these parties together in the same room with 13 of the UK’s leading synbio companies represented by our committee was widely acknowledged as an extremely useful thing to do and the committee looks forward to continuing to promote the UK synthetic biology industry through more activities of this kind.

Communications Advisory Committee (CAC)

The CAC continues to provide a platform for discussions on communications within the sector. At each meeting committee members share best practice and intelligence on the latest developments in communications – the August meeting included some discussion on the FDA’s draft guidance on social media and the FCA’s approach to financial promotions.

Earlier this year the committee was asked to review and provide feedback on BIA communications, including its social media presence on Twitter, LinkedIn and YouTube. Other topics covered this year included the Concordat on openness on animal research and, following an update from the BIA, the committee also contributed feedback on the content for this year’s State of the Nation Report with Ernst & Young – you can see last year’s report here.

So far in 2014, the CAC has heard presentations from a variety of interesting speakers including national journalists new to the beat at the Financial Times and the Wall Street Journal, a digital media expert and communications professionals from across the sector on topics such as charity communications and responsibility reporting.

Earlier this week we launched our competition for financial assistance to attend SynBioBeta SF 2014, as part of our trade mission with UKTI. For more information on the competition click here.

To help get you in the mood for SynBioBeta 2014, this week’s member video features a TED talk from Sean Ward, founder of BIA member Synthace, an applied synthetic biology company which bioengineers micro-organisms for the production of high value chemical products. In his presentation, Sean discusses how synthetic biology is exploring biological complexity.

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

The BIA has eight established advisory committees who deal with a range of topics, from regenerative medicine to finance and tax. Meeting once a quarter, the committees are crucial mechanisms to highlight and explore key issues facing bioscience companies. Committee members include influential experts from across the sector. Their work informs and guides BIA policy and priorities, ensuring that member needs are met. 

This week we have updates on the activities of the Regulatory Affairs Advisory Committee and the Intellectual Property  Advisory Committee.

Regulatory Affairs Advisory Committee (RAAC)

Throughout this year, RAAC has continued to build upon a positive working relationship with the Medicines and Healthcare Regulatory Agency (MHRA) and the European Medicines Authority (EMA). This has included collaborating on a number of successful events, for example the popular joint conference with MHRA in June on the opportunities and challenges in the regulation of healthcare innovation to stimulate innovation and accelerate patient access, which attracted over 130 delegates.

One upcoming event to note is the BIA Regulatory Dinner Discussion – Healthcare innovation and regulatory decision-making in the biocentury – with guest speaker Dr Ian Hudson on 22 October 2014. Dr Hudson will reflect on his first year as MHRA’s Chief Executive, supporting innovation as well as the challenges of regulation in the current environment.

The committee continues to advocate for a supportive environment for clinical research and innovation. Earlier in the year BIA produced a briefing paper on the new EU Clinical Trials Regulation, which explains the major changes brought in by the new legislation and provides some practical considerations until the Regulation is implemented. The BIA, together with EuropaBio, is taking an active role in influencing the development and design of the EU clinical trial portal and database. The committee has also been closely involved with the clinical trials transparency debate and contributed in May to the EMA’s targeted discussions with key stakeholders.

Collaboration with sister organisations has also been an important area in 2014. Back in June, BIA teamed up with the Association of the British Pharmaceutical Industry (ABPI) and the Centre for the Advancement of Sustainable Medical Innovation (CASMI) to host a one-stop-shop event on the EMA’s adaptive licensing pilot project – you can read a write-up of the event here. In another joint effort with ABPI, BIA also developed communication materials on biological medicines, including biosimilars, to enhance prescribers’ and other stakeholders’ understanding of these medicines.

Intellectual Property Advisory Committee (IPAC)

This year IPAC has continued its focus on issues relating to intellectual property in the biotech sector. Ongoing discussion topics have included the implementation of the European regulation arising from the Nagoya Protocol on Access and Benefit Sharing, and the question of grace periods (the period of time within which, if an invention is disclosed to the public, it can still be the subject of a patent application). Grace periods exist in the US and Japan and there appears to be a move in various quarters, particularly arising from discussion on harmonisation, to consider bringing in grace periods in Europe where they do not currently exist.

IPAC provided comments and draft wording in relation to the Bolar Exemption in the Legislative Reform (Patents) Order 2014. Following on from last year’s introductory and update blogs on Supplementary Protection Certificates (SPCs), IPAC has continued its regular review of SPC legal cases and reviewed the Law Commission report regarding groundless threats.

Through regular bilateral meetings with the government’s Intellectual Property Office (IPO), IPAC remain updated on various issues including progress on the establishment of the Unitary Patent Court. Earlier this year IPAC members also met with the Minister for IP for a discussion to highlight the relevance of IP to the biotech sector.

For the remainder of 2014, IPAC’s priorities will continue to reflect some of these major issues. The Unitary Patent and the Unified Patent Court will remain a significant area for monitoring and input, and a close watch will be kept on the possibility of grace periods being introduced in Europe so that appropriate advice can be given to BIA members on how it might affect them. The Committee will also be keeping a watching brief on developments relating to recent US Supreme Court decisions regarding what is considered patentable subject matter – and subsequent USPTO guidance. The implementation of the Nagoya Protocol will be kept under review, taking action as and when appropriate to strive for proportionate enforcement and keeping adverse impact on patenting to a minimum

IPAC and RAAC are also considering areas of common interest where they might work together, such as exploring the regulatory and intellectual property aspects of personalised medicines.

Earlier this month a survey was launched into the UK’s medicines manufacturing skills. The findings of the survey will help to inform the work of the Medicines Manufacturing Industry Partnership (MMIP), a group set up to promote the benefits of the UK as a location of choice for medicines manufacturing.

This week’s member video tackles the topic of biologics manufacturing. The video is the first in a series of five, developed by BIA member Amgen, who discover, develop, manufacture and deliver innovative human therapeutics. The series is a good example of an educational resource – click here to watch the remaining four.

Through the MMIP survey we are seeking to capture the most urgent skills needs within process development and manufacturing of small molecule, biological and cell and gene therapies. Any responses will help to identify skills gaps and to inform ministers about the challenges involved in manufacturing medicines. Please take the time to complete this important survey at: http://www.smartsurvey.co.uk/s/119096XSESP

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

Steve Bates, BIA CEO: Back in November 2013, I announced that Fight for Sight – the UK’s leading charity dedicated to funding eye research to prevent sight loss and treat eye disease – had been chosen as BIA’s official charity for 2014. Recently they have been involved in a project with the National Institute for Health Research (NIHR) Horizon Scanning Centre (HSC), helping to map the landscape in inherited eye disease. This collaboration has led to the identification of 40 potential new treatments – including technologies, drug and stem cell treatments and gene therapies – around the world, which are currently being developed for inherited retinal diseases. This is vital information that could provide the backbone of a value story for anyone developing a product in this area.

Once you’ve read Carol’s blog below, do take a look at the publication. By including patient perspectives in this analysis for the first time, the report has been able to identify the treatments likely to have the biggest impact and this approach is at the heart of putting the patient at the centre of developing a treatment. It also ensures from a company perspective, that what is being developed is what patients actually want, and what payors should be willing to pay for.

Best,

Steve

 

Carol Bewick, Director of Policy and Communications at Fight for Sight: Knowing what your beneficiaries want and understanding the landscape in which you operate are important to any charity.  But for a medical research charity that has to turn away five out of every six research grant applications, knowledge and understanding are vital to inform priorities.

Fight for Sight is the UK’s leading charity dedicated to funding pioneering research to prevent sight loss and treat eye disease.  Money is very limited and we have to prioritise.

In 2013 we led the Sight Loss and Vision Priority Setting Partnership which has now established the priorities for eye research based on the views of 2,220 patients, relatives, carers and eye health professionals.

But that was just the start.  With limited funds we need to understand what research is already being undertaken and how we can ensure all funders co-ordinate activities for maximum impact.

Artistic EyeA great example of this relates to work around the priorities for people with inherited eye diseases, now the most common cause of blindness in working age adults in England and Wales and the second most common in childhood.  With no cure or treatment available, the highest priority was: “Can a treatment to slow down progression or reverse sight loss in inherited retinal diseases be developed?”

Being told that you are going blind and nothing can be done is devastating.  Many patients, their friends and family want to know what research is being undertaken and how far we are away from a treatment.

To help answer these questions, the National Institute for Health Research (NIHR) Horizon Scanning Centre (HSC), working with Fight for Sight, has identified 40 potential new treatments around the world which are currently being developed for inherited retinal diseases in their latest exercise published on 16 July. These include technologies, drug and stem cell treatments and gene therapies. For the first time, NIHR HRC asked patients how useful they felt the treatments would be, whether they would try the proposed treatment and what looked exciting.

Professor Tara Moore and Sarah Atkinson

Professor Tara Moore and Sarah Atkinson

The report is excellent news.  It helps inform patients and gives hope that treatments are in the pipeline.  But we can’t be complacent.  Nine out of every ten trials still fail and therapies that may help address one particular inherited eye disease may not work for others.

We have taken important steps in making the landscape less foggy. We know what patients want and we know what is currently happening.  We now need funders to work together to build on current research and ensure that new funding is maximised and allocated in a co-ordinated way.

Fight for Sight is the BIA 2014 charitable partner. They are the main UK funder of research to prevent sight loss and treat eye disease. For further information about Fight for Sight go to www.fightforsight.org.uk. Click here to view the horizon scanning report.

AMRC_MartinTurner_12629-49Dr Martin Turner, Senior Policy Adviser at the Association of Medical Research Charities (AMRC), tells us about their work to promote collaboration between charities and industry, and how BIA members can get involved.

The UK’s medical research environment is evolving before our eyes. It’s now well-acknowledged that traditional approaches to drug development aren’t going to get us the life-saving new treatments we need in the 21st century. Innovative approaches are required, and it’s increasingly clear that collaboration is key.

Charities and biotechnology companies share a common purpose – to develop new treatments and get them to the patients that need them as quickly as possible. So they are natural partners.

Charities have unique insights into the needs of patients and a single-minded focus on investing their money to improve care. And more than ever they are looking to invest that money in smart new ways and to take a more hands-on approach to getting new treatments from bench to bedside.

By partnering with biotechnology companies, charities gain valuable expertise in drug development and the regulatory requirements for bringing new treatments to market. In turn, the company benefits from the charity’s unique relationship with patients, access to the fruits of basic research and, in some cases, direct financial investment from the charity.

Some of our members have been doing this for many years already whereas others are interacting with the private sector for the first time. But what we are certainly seeing is that charities are becoming more entrepreneurial.

At AMRC we are working to support our members who want to take this collaborative approach. We have established an Industry-Charity Advisory Group with representatives from our membership and industry (including BIA) to guide this work. In 2014 and ’15 will be delivering networking and training events, producing new support materials, and using what we learn to demonstrate the benefits of our collaborative approach to the government. The combined voice of charities and industry is hard to ignore!

Deborah O'Neil, NovaBiotics and Ed Owen, Cystic Fibrosis Trust at the AMRC/BIA lunch

Deborah O’Neil, NovaBiotics and Ed Owen, Cystic Fibrosis Trust at the AMRC/BIA networking lunch

We have already held a networking lunch in Parliament with BIA to showcase successful collaborations and provide opportunities for BIA and AMRC members to meet. There will be more of these types of events to come.

We are also re-writing our 2008 guide An essential partnership, to bring it up-to-date with new case studies, advice and support for charities looking to collaborate with companies. It covers everything from contracts to managing conflicts of interest.

To help us develop these support materials for our members, it’s important that we know about the needs of the industry partners too. BIA members can help us by answering our short online survey about how you collaborate with charities or how you would like to. By getting involved you will be helping to make sure both charities and their industry partners get the most out of future collaborations.

The UK is blessed with a dynamic and diverse medical research community made up of public, private and charity research funders. There are very few countries in the world that are so lucky. It’s vital that we harness our combined expertise and work together to drive forward medical innovation to benefit patients and the UK economy.

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