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New Year, new goals

Published on 08/02/18 at 11:35am

With the New Year begun in earnest, Pharmafocus speaks to a range of players in pharma and the life sciences to find out what 2017 meant for them, and what they have in store for 2018.

Spokesperson, EMA

It’s been a busy year for the EMA, on all fronts – what were the key achievements for the agency during this time?

Most of 2017 was a year of uncertainty for the agency. The decision to relocate EMA to Amsterdam, the Netherlands was only taken on 20 November by the EU 27 Member States. Although, the relocation and the preparation of the regulatory network for the departure of the UK from the EU continues to represent a major challenge the agency has been preparing operationally and has re-allocated internal resources to these tasks in 2017. Despite these challenges we have still succeeded to break some new ground launching projects and fulfil our mission to promote and protect public and animal health.

Among the agency’s major successes of the past year was the launch of the new and improved Eudravigilance system. This information system is the backbone for the reporting of suspected side-effects to medicines in the EU. This new version makes it easier to submit reports and analyse information. This will improve patient safety in Europe.

In September we held the first public hearing at EMA. The hearing gave EU citizens an opportunity to make their voices heard. Their views complement the available scientific evidence in the evaluation of Valproate, a medicine that treats epilepsy, bipolar disorder and migraine.

How much focus will the move to your new headquarters require in 2018?

Moving a large organisation such as EMA to a new location is a challenging undertaking under any circumstances. It is made even more complex by the ambitious timetable determined by the withdrawal of the United Kingdom from the European Union, whereby the relocation has to be completed by 30 March 2019.    

The preparation for the relocation to the new headquarters will be a main focus of 2018. EMA’s number one priority for 2018 will be to keep its core activities running to avoid any kind of disruption that might affect the health of citizens across Europe.

Outside of the HQ change, what are the agency’s aims for 2018?

In January 2018 the agency entered Phase 2 of its business continuity plan in order to free up further resources needed to prepare for the withdrawal of the UK from the EU. In these circumstances EMA’s priority is to ensure that the assessment and supervision of medicines continues to be delivered on time and to the same high level of quality the agency's stakeholders have come to expect, and that patients in Europe continue to have access to high quality, safe and effective medicines. For EMA’s other objectives for 2018, I am afraid you will have to wait for the publication of the 2018 work programme that is scheduled later in the first quarter of 2018.

Harmonisation took several steps forward this year, with alignment in certain areas with the FDA and the PMDA – can we expect to see more of such cooperation during 2018?

The new confidentiality commitment signed by the European Commission and the FDA in August represents a milestone in the relations between EMA and FDA. It allows the two regulators to rely on each other’s inspection reports and determine whether similar problems are observed with certain manufacturing processes in the US and the EU. It will avoid duplication, lower costs and enable EU and US regulators to focus on manufacturing sites in parts of the world where there is greater risk.

Another important milestone was the agreement between the FDA, the Japanese Pharmaceutical and Medical Devices agency and EMA on the alignment of data requirements for certain aspects of the clinical development of new antibiotics. This initiative aims to stimulate the development of new treatments to fight antimicrobial resistance and protect public health.                                                                                                                   

These two examples were the result of years of hard work and confidence building between EMA and other international regulators; in 2018 the agency will continue to work with its partners in Europe and beyond to contribute to the health of people at home and globally.

Dr Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research

What do you think were some of the key achievements for the FDA in 2017?

In 2017, the US FDA’s Center for Drug Evaluation and Research (CDER) focused on issues that impact multiple sectors of the pharmaceutical industry. The FDA Reauthorization Act was signed into law, reauthorising the Prescription Drug User Fee Act, the Generic Drug User Fee Amendments (GDUFA), the Biosimilar User Fee Act, and the Medical Device User Fee Amendments until fiscal year 2022. These Acts ensure continuity for these regulatory programmes by authorising the agency to collect user fees from industry. These fees and their corresponding performance goals have helped to move forward the drug and device review process in the United States. Moreover, the second GDUFA reauthorisation is an important milestone, providing long-needed resources to increase consumer access to safe and affordable generic drugs – a top priority for FDA. In 2018, we will continue to implement and expand the user fee programs, striving to meet or exceed our established goals.  

Another key piece of legislation that we are now in the process of implementing is the 21st Century Cures Act. The Cures Act, signed into law in December 2016, gave the FDA new hiring authorities, which will enable us to more speedily bring on scientific experts in specialised areas so necessary to our review processes. We are also using our expedited programmes, such as fast track, priority review, accelerated approval and breakthrough therapy, to bring safe and effective treatments to market in a timely manner. Among its directives, the legislation requires the agency to provide a framework of principles for advanced manufacturing processes, including continuous manufacturing, which promises to produce high-quality drug products more efficiently than ever before. Additionally, the Cures Act contains provisions to advance patient-focused drug development and qualification of drug development tools. In 2018, we will continue to implement these and other key components of the Act.

To meet the anticipated challenges of an evolving biopharmaceutical industry, the agency began a major effort in 2017 to modernise our New Drugs Regulatory Program. In 2018, we will implement our modernisation plan including meaningful incorporation of the needs of patients in drug development.  Moreover, CDER will continue to identify and integrate improved technology into the review process.

One of the biggest threats to public health in the US has been the ongoing opioid crisis. What moves has the FDA made to tackle this?

The FDA continues to address the American opioid crisis by using all the tools available to us. We are encouraging the safe use of opioids through careful and appropriate regulatory activities and policy development, by using better science, and through collaborative partnerships. For example, opioid manufacturers are now subject to more stringent requirements under a Risk Evaluation and Mitigation Strategy (REMS). We are also taking into consideration the impact on population health by carefully weighing risk-benefit ratios when making regulatory decisions about opioids. For instance, we sought withdrawal of the opioid Opana ER when postmarket data showed that the risks associated with its abuse were outweighing its benefits. We are collecting data on the impact of opioids with abuse deterrent properties, and have issued guidance for generic abuse-deterrent formulations. We are also collecting data on prescriber beliefs and attitudes towards opioids and are working to expand education in pain management. In 2018, we will continue to work with agency, state, patient advocacy and health care partners to confront the crisis.

What other challenges did the organisation face throughout 2017?

Pharmacy compounding continues to be an issue of high importance and concern. Working collaboratively with our state partners, we are building a framework for regulating compounding pharmacies that includes conducting inspections of compounding facilities, and issuing timely recalls, risk alerts and warning letters to compounders who are found to be producing contaminated products. In 2018, we plan to issue new guidances aimed at enhancing the safety of compounded drugs.

These are highlights of just some of the agency’s activities, even as we continue to receive historically high numbers of drug applications. We look forward to another productive year collaborating with industry and with our regulatory counterparts worldwide to ensure safe access to effective drugs all over the world.

Tom Delahoyde, Managing Director, Chiesi UK

What have been the highlights of 2017 for Chiesi?

2017 has been a great year for Chiesi UK as we continued to focus on bringing innovation to those that need it the most, the patients. We’ve had a number of important milestones in 2017 including a positive NICE recommendation for the first stem cell therapy, Holoclar (ex vivo expanded autologous human corneal epithelial cells containing stem cells) for the treatment of moderate to severe limbal stem cell deficiency after eye burns. We also saw the successful UK launch of Trimbow (beclometasone/formoterol/glycopyrronium) - the first fixed-dose 3-in-1 combination inhaler therapy for Chronic Obstructive Pulmonary Disease (COPD).

What trends have you noticed within the pharma industry developing this year?

There continues to be a focus on how the industry can work with government to bring breakthrough medicines to patients, particularly within the context of the potential impact of Brexit and the ongoing financial pressures on the NHS.

We have also seen an increase in focus on the digitalisation of healthcare, particularly when it comes to using digital technology such as Apps and Virtual Reality to improve treatment compliance in those in long-term conditions such as asthma and COPD.

Looking ahead, what do you expect will be the major themes for 2018?

In 2018, we can expect high-tech medication and digital health solutions to have a major impact on treatment, compliance and disease management in the long-term. Additionally, the focus will continue on how we can prove the cost-effectiveness of treatments in the real-world at a national and local level – this will be particularly prominent as discussion around access to innovation in a post-Brexit Britain ramp up.

Brexit obviously requires a lot of consideration from pharma companies; how do you view Chiesi’s position within the UK and do you believe there can be a successful outcome for all parties?

It is impossible to predict what Brexit will bring to the industry, but we remain optimistic that Chiesi’s role in bringing innovative medicines to market in the UK will continue.

What should patients expect from the industry in 2018 – are there good reasons for optimism regarding patients’ access to medicines and for further medical innovations?

Patients can expect to have a far greater role in determining their own healthcare choices and be better informed about their own health as the digital world gives patients greater access to information and data about healthcare. As the approval, prescribing and marketing of medicines become more closely linked to patient needs, to effectively collaborate with patients, the industry should be looking at ways to best communicate with them (within our code of practice guidelines) using multichannel and digital media. The industry of late has highlighted that a risk-averse approach to drug regulation has impacted on pharmaceutical innovation, I predict that over the course of 2018 and beyond, this will need to change if we are to broaden access to innovative medicines.

Dr Jayne Spink, CEO, Genetic Alliance

You joined the Genetic Alliance UK as CEO early in 2017 – how has your time with the organisation been so far?

I joined in April 2017, as the new CEO, after having been a member and a supporter of Genetic Alliance UK for many years. As a supporter, I’ve recognised the important and influential work the organisation carries out. Since I’ve joined the team, I’ve found that I’m working with fantastic colleagues and that are really dedicated to the work. I’ve also really valued getting to know other member and supporters of the vital work Genetic Alliance UK does. Everyone has been so giving and generous with their time, it’s been a very warm welcome to the role.

What have been the major events of the year for the organisation?

The surprising event of the year, for us, was the snap general election. We lost our chair, Ben Howlett [former MP for Bath] as a consequence of the election and the APPG met again post-election for the first time in early October. Steven Twigg MP and Vicky Ford MP were elected as co-chairs of the group – the group has 45 members and goes from strength to strength. It’s an important body to raise awareness and bringing about change in rare genetic and undiagnosed conditions.

Has there been any particular research or studies that have changed the rare disease area?

We’ve recently launched a survey component of a mental health project, and we’ve heard responses from over 1,500 individuals in less than a month – the findings from which will be published this year. It’s clear from responses that mental health can be particularly challenging for people who have a diagnosis or their carers and families. What the survey has shown is how important addressing issues around anxiety or low mood, and a lot of those issues seems to be arising from coordination of care and access to care.

Are there are fundamental issues that those with rare diseases have to face that will continue in 2018?

I think there are significant challenges in relation to access to and speed of diagnosis; access to specialist care, coordination of care and social care – all of the things that are outlined in the UK strategy. What has a struck is that there has been a significant gap between knowledge generation and its implementation and uptake, particularly in healthcare. This underlines that that’s a real lack of resources and the inflexibilities in the system.

We also have challenges with a lack of access to medicine and there’s been several initiative and consultations that have really underlined those challenges. Interesting the APPG looked at access to medicines at their autumn meeting and tasked with finding a solution to the barriers preventing access to medicines for rare diseases – something which is a substantial, multi-stakeholder piece of work for this year.

What are you plans for this year?

This year, we celebrate the 10th birthday of Rare Disease UK and we’ll be looking forward to Rare Disease Day, as always. We’ll be focused on reaching more people and raise more awareness, especially around the challenges faced by patients and carers. Hopefully our 10th year will be a significant one for us.

I think there are things to be optimistic about, for instance building on the 100,000 genomes project and how that’s going to be implemented – I think that’s a very positive thing to look forward to.

Dr Justine Alford, Senior Science Officer, Cancer Research UK

Did CRUK hit any notable milestones throughout 2017 that you are particularly proud of?

2017 marked the 25th anniversary of our drug development facility, the Centre for Drug Development (CDD). Throughout its quarter-century lifespan, the CDD has helped bring six drugs to market, including the breakthrough prostate cancer drug abiraterone (Zytiga).

Back in 2012 this drug was first made available on the NHS for some men with advanced disease who had become resistant to standard hormone therapy. But last year, our STAMPEDE trial showed that it could benefit even more men.

The large trial showed that adding the drug earlier, before the disease has become resistant to standard hormone therapy, could substantially boost survival for some men. Excitement of the results rippled through the media and scientific community. These encouraging results make a strong case for further research to see if opening the drug up to more people could bring even more benefits in the future.

Of all your ongoing projects, which stand out to you and why?

Taking big risks can lead to big rewards. That’s why our ‘Dragon’s Den-style’ Pioneer Awards funds innovative science that could transform our understanding of cancer. Last year we funded a string of exceptional scientists to bring their bright ideas to life, including Dr Harry Bulstrode, who wants to harness the power of the Zika virus to help tackle brain tumours.

The Zika virus can cause a serious brain condition called microcephaly in babies born to infected mothers. This happens because the virus infects fast-growing stem cells in the developing brain, whereas in adults, infection usually only leads to mild flu-like symptoms that they can recover from.

In glioblastoma, the tumour’s growth is fuelled by cancer stem cells that share features with normal stem cells in a developing brain. Bulstrode is testing out whether the Zika virus could target these cancer stem cells in glioblastoma, in cell lines and mice, in the hope that this could be translated into a treatment for people.

What else is on the horizon for CRUK in 2018?

With 2017 proving to be another fruitful year for our research, we’re excited to see how 2018’s science unfolds. Excitedly, the second round of Grand Challenge awards – our most ambitious cancer grant which offers up £20 million to teams willing to tackle the greatest challenges in cancer – will be announced in the autumn of 2018.

From distinguishing lethal cancers from those that don’t need treating, to digging into the causes of cancer, these challenges could bring about life-changing discoveries. And with science well underway from last year’s winners, that gives us something more to look forward to this coming year.


Great to see you coming up with new ideas, the future is bright and we are still going strong. 2017 might have been a little uncertain but, nevertheless, was very successful. Looking forward to seeing more of you in 2018!

Best regards,

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