Archives for category: Regulatory Affairs

q-report-cover-oct-2016-to-jan-2017Our blog today, taken from our latest quarterly policy and regulatory affairs update, takes a look at clinical trials hot topics.

Revision of first-in-human clinical trials guideline

Following on from our comments in our last quarterly updates regarding the tragic incident which occurred during a Phase I clinical trial in France in January 2016, the BIA has kept a watching brief on the investigations and any implications for the conduct of first-in-human trials in the EU.

The European Medicines Agency (EMA), in cooperation with the European Commission and EU Member States, has proposed a revision of its guideline on strategies to identify and mitigate risks for first-in-human clinical trials with investigational medicinal products to further improve the safety of trial participants.

The EMA published the draft revised guideline for public consultation on 15 November. This follows an initial consultation in the summer on a concept paper that outlined the key areas of the current guideline (adopted in 2007) that need to be amended, taking into account the lessons learnt from the tragic incident, as well as the evolution of practices in the last 10 years.

While the 2007 guideline focused on the single-ascending-dose design used at that time, the practice for conducting first-in-human clinical trials has evolved towards a more integrated approach, with sponsors conducting several steps of clinical development within a single clinical trial protocol. For example, these trials may also include collection of data on food interaction, in different age groups as well as early proof of concept / proof of principle parts and bioequivalence of different formulations.

The BIA intends to respond to the consultation in collaboration with EuropaBio (the European Association for Bioindustries). If members wish to contribute to the consultation response please contact Christiane Abouzeid at


BIA attends EMA meeting on EU Clinical Trials portal and database

In November the BIA participated upon invitation in an EMA stakeholders meeting on the development of the EU clinical trials and Union database as part of the implementation of the EU Clinical Trials Regulation. Discussions focused on user management – a set of features and capabilities that manage user access to the clinical trials system functions according to their role, so the system will display the appropriate data and also the appropriate activities for them.

We will continue our ongoing dialogue with the Agency in order to address industry concerns and ensure that the clinical trials system is fit for purpose.

BIA responds to EMA consultation on clinical trial guideline

In December, the BIA inputted, together with EuropaBio, to a consultation by EMA on a draft guideline on the requirements for quality documentation concerning biological investigational medicinal products in clinical trials in connection with the implementation of the EU Clinical Trials Regulation. This guideline is of particular interest to members involved in clinical development of biological and biotechnology derived medicinal products in the EU.

Voluntary Harmonisation Procedure (VHP) for clinical trials – 1000th procedure

The Heads of Medicines Agencies’ Clinical Trials Facilitation Group has recently announced receipt of the 1000th application for the evaluation of a multinational clinical trial by the VHP procedure. Established in March 2009, the VHP is a useful tool for the initiation of the authorisation procedure for clinical trials in more than one EU member state by submitting a single application. It is worth noting the VHP served as a model for the new procedure in the EU Clinical Trials Regulation, which will come in to force in October 2018.

As we head towards Christmas, we take a look at BIA’s year in numbers…



q3-2016-updateSince our last update in July there have been a number of major developments in life sciences policy and in politics more widely. In this edition we have provided a key section focusing on Theresa May’s Government, Industrial Strategy and Brexit, and we also celebrate the Chancellor’s commitment to re-fill the Biomedical Catalyst scheme – a very welcome result after years of advocacy by BIA and our members!

Download the report in full here, and read on for a summary of the key topics covered in this edition.

The May Government, industrial strategy and Brexit – The last few months have seen significant change in the political landscape following the outcome of the EU Referendum. We now have a new Prime Minister, new Government, new Whitehall department and a new policy focus. Our latest update examines the work of the UK EU Life Sciences Transition Programme including next steps, BIA engagement with the newly re-structured Government, the renewed focus on industrial strategy and how it links to Brexit, and medicines regulation in the context of the UK leaving the EU.

Commitment to refill the Biomedical Catalyst – The Government has committed to fund the Biomedical Catalyst for the rest of this Parliament following a long-fought campaign by the BIA. The Chancellor, Philip Hammond MP, used his speech at the Conservative Party conference on 3 October to formally announce the £100 million investment in life science companies. The Government said in a statement that the BMC will provide grant support to ensure that “emerging disruptive healthcare technologies in disease prevention, earlier diagnosis and tailored treatments are delivered to market”. The programme will offer funding in 2017, 2018, 2019 and 2020 to support varying stages of technical and commercial development from feasibility studies through to proof of concept and early clinical trials.

Party conference insights – The BIA had a presence at four political party conferences this year. We hosted private roundtable meetings with the Conservatives and Labour and attended the Liberal Democrats and Scottish National Party conferences. The focus of the roundtable meetings was on maintaining the UK’s world-leading position in the life sciences after Brexit. As in previous years, the BIA partnered with the AMRC, BIVDA and ABPI to deliver the events and invited a range of stakeholders from academia, the NHS and our memberships to meet with MPs and policy makers. Our update also covers the key announcements from the conferences for the life science sector.


UN report on global access to medicines and landmark declaration on AMR – The United Nations (UN) published a report on access to medicines written by a high-level panel of experts. The International Council of Biotech Associations, of which BIA is a member, published a response to the report which you can read in full in our update. It was also announced at the United Nations General Assembly that 193 countries had signed a landmark declaration agreeing to combat antimicrobial resistance as a matter of priority.

PRIME scheme six months on – The BIA has played an active role in influencing the development of PRIME, representing the views of member companies. As of 6 October 2016, 64 applications to the EMA’s PRIME scheme had been received. We have seen high levels of interest in PRIME from BIA member companies and we are pleased that a number of our members’ products have been accepted onto it.

Adaptive pathways: where are we today? – On 3 August the EMA published a final report on the experience gained during its pilot project on adaptive pathways. The EMA received 62 applications with oncology accounting for a third of the total submissions; 18 were selected for in-depth face-to-face meetings with the participation of other stakeholders. At the end of the pilot, six applications had progressed to parallel regulatory HTA scientific advice and one to scientific advice with EMA. The EMA will now explore the adaptive pathways approach further in the context of parallel scientific advice with HTA bodies – more details in our update.

Download Influencing and shaping our sector: BIA update July – October 2016 for details on the above topics and more.

lobbyingTo learn more about BIA’s policy, public affairs and regulatory affairs work, see our previous quarterly policy updates, our consultation responses, or email Martin with any questions.

In May the BIA launched a briefing paper on Orphan Medicinal Products that looked into the 15-year application of the Orphan Regulation in Europe.

The paper coincided with the European Conference on Rare Diseases & Orphan Products, which was held in the UK for the first time and brought together stakeholders from across the rare disease community.

The briefing paper explores the EU regulatory framework and the setting up of the Committee for Orphan Medicinal Products within the European Medicines Agency (EMA) and raises awareness around the latest regulatory policy developments in this field. The European Commission is currently in the process of considering a review of its Communication (2003/C 178/02) to streamline and adapt the existing guidance to technical progress. This revision is of great interest to BIA members as a large number of biotech medicines are authorised with an orphan indication.

To download the briefing paper go to the BIA website.

omp table

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.

To date, the European Commission has authorised 121 orphan medicines for the benefit of patients suffering from rare diseases. Some 25 orphan marketing authorisations are from SMEs as of January 2016. Furthermore, the European Commission has designated 1,349 products as orphan medicinal products.

The European Commission has launched a public consultation on the concept of ‘similar medicinal product’ in the context of the orphan legislation until 4 November 2016. If members wish to comment on the proposed changes to Article 3 of Commission Regulation (EC) 847/2000 please contact Christiane Abouzeid.

For more information on the latest policy and regulatory affairs work at the BIA, download our quarterly report covering April to July 2016.

Q2 2016 reportOngoing BIA engagement enables our members’ voices to be heard at the highest levels. Our latest quarterly update gives an overview of key policy developments and the BIA’s continued engagement with policymakers, regulatory authorities and wider stakeholders on behalf of the UK life sciences industry, from April to July 2016.

In the update, you can read up on key issues for the life sciences industry such as:

The EU Referendum, the new Government and the life sciences industry – Following the EU Referendum result, the BIA has acted swiftly to position our sector at the heart of Government thinking as events and Brexit discussions continue to unfold. Find out more about the formation of the UK EU Life Sciences Steering Group, set up to oversee and manage the transition for the sector.

The political fallout from the EU referendum result ushered in a new Prime Minister sooner than many expected. The former Home Secretary, Theresa May, entered Downing Street on 13 July and quickly set about establishing a new Government with a ministerial reshuffle and rearrangement of Whitehall departments. Published alongside the quarterly update, the BIA has produced a quick briefing for members on the new Government and key ministerial appointments. It will be updated as information emerges. You can download the guide here.

May Cabinet

Changes to innovation support not slowed by Brexit – The Government is pressing ahead with the reorganisation of the UK’s research and innovation system, including bringing the operation of Innovate UK and the seven Research Councils closer together. In addition, the announcement of a £10m Biomedical Catalyst competition for 2016 is welcome news that Innovate UK and the Government are listening to the BIA’s calls for an effective innovation policy for the life sciences.

28144680336_8a00f60fc5_oBIA Parliament Day – On 7 July 2016, 35 senior representatives from the UK’s life science industry convened for the BIA’s sixteenth annual Parliament Day – a key day of engagement between BIA members and policymakers in Whitehall and Westminster.

BIA briefing paper on orphan medicinal products – In May the BIA launched a briefing paper on Orphan Medicinal Products that looked into the 15 year application of the Orphan Regulation in Europe. The paper coincided with the European Conference on Rare Diseases and Orphan Products, which was held in the UK for the first time.

BIA-MHRA joint regulatory conference 2016 – The BIA and MHRA have published a report following their sixth annual joint conference, Accelerated Development and Access to Innovative Medicines for Patients, which took place on 4 May 2016. BIA and MHRA brought together experts from across the sector to assess the state of play, discuss how the various schemes have performed, and how the NHS might respond to current innovation challenges.

Ian Hudson quote

BIA welcomes new NHS report on access to biosimilar medicines – The BIA has welcomed the recent publication of the report of the biosimilar medicines workshops hosted by NHS England in coordination with a range of national partners. The BIA has partnered with NHS England in developing this document and is a member of the National Biosimilar Medicines Programme Board, which will be considering how to prioritise and advance the proposed next steps arising from the workshops.

For details on the above topics and more, download our latest quarterly update, Influencing and Shaping Our Sector: BIA update April – July 2016.

To learn more about BIA’s policy, public affairs and regulatory affairs work, see our previous quarterly policy updates, our consultation responses, or email Zoë with any questions.


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.

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.


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.


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.


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.


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

Q1 2016 reportOngoing BIA engagement enables our members’ voices to be heard at the highest levels. Our latest quarterly update gives an overview of key policy developments and the BIA’s continued engagement with policymakers, regulatory authorities and wider stakeholders on behalf of the UK life sciences industry, from January to April 2016.

In the update, you can read up on key issues for the life sciences industry such as:

The EU referendum and how BIA has engaged in the debate – With the EU referendum set for 23 June, the BIA has been working to ensure that the voice of the UK life sciences industry is heard loud and clear. BIA members have said they believe the industry will be stronger if we remain in Europe and we have conveyed this message through policy and media routes.

The new MMIP_logoministerial and industry taskforce securing the UK’s manufacturing environment for advanced therapies – A new Advanced Therapies Manufacturing Taskforce was launched by the Medicines Manufacturing Industry Partnership (MMIP) to secure the future of manufacturing advanced therapies in the UK. The taskforce will be co-chaired by George Freeman MP and Ian McCubbin, GlaxoSmithKline.

The latest developments from the EMA on the PRIME priority medicines access scheme and clinical trial data transparency – The new PRIME (PRIority MEdicines) scheme, launched by the European Medicines Agency on 7 March, has got off to a good start with 18 applications as of 21 April. We understand that 60% of the applications were from SMEs, an important target audience of the scheme. Also in March, the EMA published detailed guidance on the implementation of its policy on the publication of clinical trial data. This detailed guidance has been finalised following an extensive consultation with stakeholders concerned throughout 2015, including the BIA.


An update on BIA Advisory Committee 2016 priority policy areas – Our annual Committee Summit took place on 25 February, during which each of BIA’s eight advisory committees held their Q1 meeting. In a plenary session with fellow BIA members, each committee’s focus areas for 2016 were set out. The quarterly report details the specific policy priorities of each committee for the year.

BIA Committee Chairs 2016

While November’s Comprehensive Spending Review may now seem like a distant memory, details are now starting to emerge about some of the spending policy changes it led to. We’ve updated our Spending Review outcomes table to summarise the current state of play in areas including the BIS budget, the research and innovation public funding landscape, regional devolution deals and more.

To learn more about BIA’s policy, public affairs and regulatory affairs work, see our previous quarterly policy updates, our consultation responses, or email Zoë with any questions.

On Tuesday, BIA CEO Steve Bates appeared before the House of Commons Science and Technology Select Committee to give evidence on the impact of European regulation on UK life sciences, building on the BIA response we submitted to the inquiry earlier this month.

Watch the evidence session by clicking on the image below. You can find out more about the BIA’s position on the EU referendum and our recent advocacy work in this area here.

Steve Commons STC

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

Throughout the year the BIA advocates for our members in the UK life science industry, engaging with decision makers and influencers in Westminster, Whitehall and across the science, health and business sectors, as well as with regulators in the UK and Europe.

80meetingsIn 2015, we met with over 80 parliamentarians, civil servants, ministers, shadow ministers and regulators at national and European level.

Our latest quarterly report, ‘Influencing and shaping our sector: BIA update October 2015 – January 2016’, sets out what we’ve been up to in the last few months. You can download the report in full here. Highlights include:

  • Our summary of outcomes of the Government’s 2015 Spending Review and the Nurse Review’s recommendations for the UK’s research landscape, covering the key implications for the sector. For more a more comprehensive analysis of the 2015 Spending Review announcements, our detailed table is available to download here.osborne-csr-2015
  • Further information on the European Medicines Agency’s (EMA) priority medicines (PRIME) scheme. The BIA inputted, together with EuropaBio, to the EMA consultation on the key elements of the PRIME scheme, which is due to launch by March 2016.
  • An update on the Accelerated Access Review, which has continued apace since the publication of the interim report in October 2015. The BIA and members have continued to input to the review over the past quarter, including via surveys and workshop events with the Government’s Office for Life Sciences (OLS).
  • Our Advanced Therapy Medicinal Products (ATMP) briefing paper, launched in November to coincide with the Alliance for Regenerative Medicine European Investor Day. The paper revealed a significant new level of interest in cell and gene therapies and showcased the BIA’s work in this area to raise awareness around regulatory changes for 2016 that will impact on the development of ATMPs.ATMPpaper
  • Details on the implementation of the EU Clinical Trials Regulation, which will come into effect by October 2018 at the latest, following the endorsement of the timeframe for the implementation of the EU clinical trial portal and database by the EMA Management Board.

We’re always keen to hear from our members. Which policy areas do you care about; what’s impacting your business and R&D? Do you have a story that would help us to make the case for continuity in what works and to change what doesn’t?

To learn more about BIA’s policy, public affairs and regulatory affairs work, see our previous quarterly policy updates, our consultation responses, or email Zoë with any questions.