Tracking progress of the Rare Disease Strategy

by Claudia Rubin 19. March 2018 09:18





February’s magazine focused on rare diseases, but how can implementation plans for the Rare Disease Strategy be properly monitored?


The Rare Disease Strategy implementation plans for England were jointly published by the Department of Health and Social Care (DHSC) and NHS England. Four years after the Strategy was announced, this is welcome news for the 3.5 million people in the UK who suffer from a rare disease and the countless other people who take an interest in their welfare.


One advantage of publishing these plans so long after the strategy, and for that matter, after the plans of the Devolved Nations, is that simply making it to publication was a cause of some celebration, and presumably relief. The plans do contribute to the greater level of transparency that DHSC and NHSE seek in this sector and demonstrate a commitment to maintaining improvement in the support, care and services that rare disease patients receive.

In an unprecedented move, the 51 commitments have been divided up between NHSE and DHSC, which makes deciphering the nuances of the proposals that little bit harder, and holding someone accountable for the deliverables trickier.


Official oversight is provided by the Rare Diseases Advisory Group (RDAG) which makes recommendations to NHS England on developing and implementing the strategy; and also by the Rare Disease Policy Board (RDPB), a UK-wide committee, managed by DHSC, with responsibility for facilitating the coordination of policy development and meeting the commitments set out in the strategy.


As DHSC puts it: ‘The role of the RDPB is to monitor what is being implemented in respect of the strategy and the role of RDAG is to make recommendations to NHS England on how the Strategy is implemented.’

Considering the potential conflict of interest of the RDPB, given that its membership is comprised of people from the organisations tasked with delivering the Strategy, one must question how effective this oversight and accountability can be. It is certainly crying out for a robust political role, for example, from the Health Select Committee or another cross party group to ensure progress is monitored and shortfalls are exposed.


Among the highlights of the DHSC’s commitments are;


•          The Rare Disease Policy Forum and a new digital platform for the Forum.

•          A new annual conference by the UK Rare Diseases Policy Board

•          The establishment of a task and finish group to improve the ‘diagnostic  odyssey’ of rare diseases patients. 

•          An emphasis on the importance and potential of genomics.



Agreed targets: NHS England’s three main objectives


1. Facilitating earlier diagnosis


NHS England will implement wider genomic testing and additional genetic tests. It also plans to work with GP and nurse groups to increase awareness and improve knowledge of rare diseases.

On DHSC’s part, there is a welcome focus on the so-called ‘diagnostic odyssey’, with the aim of ‘understanding in detail the diagnostic pathway’ as the ‘first step in finding ways to improve diagnosis and subsequent early intervention'. Last year a diagnostic odyssey task and finish group was established to identify and describe the diagnostic journey of three disease case studies. It will report its initial findings later in 2018.



2. Improving care coordination


NHS England is to develop ‘rare disease inserts’, a set of criteria to sit alongside service specifications that will enable patients to hold providers to account for their care. It will insist for example that everyone has an assigned care coordinator. The intention of these inserts, and the related idea of ‘alert cards’ seem positive, and perhaps patient groups should suggest they are best placed to lead this project, ensuring they have the most useful information and that the most uncommon rare diseases are not forgotten.



3. Promoting research


 Though lacking information, the development and implementation of Rare Disease Collaborative Networks (RDCNs) could realise sizeable benefits for patients and the NHS. Anything that can be done to further direct the groundbreaking research from the 100,000 Genomes Project directly into frontline services and care will be of value.



Magic numbers...


The plans take steps for progress evaluation, with a set of measures, through regular stakeholder meetings and reporting to the RDPB. Critics have noted, however, that measures hold no specifics on appropriate standards or targets.

On care coordination, the measure against which NHSE claims it can be held accountable, in terms of delivering recommendations 8, 27, 33 ,47 and 51 of the strategy, are the number of rare disease collaborative networks endorsed and the number of rare disease collaborative centres endorsed.


But what number counts as good? There is no detail here or standard to reach for, which may not encourage much ambition.


Then there is the elephant in the room – is there any new money attached to these plans? Well yes, some. We know that the 100,000 Genomes Project is funded, then there is the £20 million Genomics Education Programme, and specialised services continue to receive a healthy share of the overall NHS budget, but none of these represent new money for new commitments. Significantly the number of conditions screened for at birth would most certainly make a big difference to rare disease diagnosis – but with no new money for the national screening programme, how many new conditions will pass the committee’s current criteria for screening cost effectiveness?


In this brave new world of innovation – stem cell treatments, multiple indication therapies and advanced technologies for complex diseases - the assessment system that evaluates costs and benefits of treatments available on the NHS is absolutely crucial. The only measure that NHS England is holding itself to account for delivering is whether a process is published. The current one, still intentionally referred to as ‘interim’ by Sir Andrew Dillon, is not yet fit for purpose and many of us are keen to help improve it quickly.


A progress report on the UK Strategy for Rare Disease is due and while we all want credit to be given where it is due, we will also be eagerly looking for some robust analysis of the milestones needed to meet the strategy’s ambition.


Claudia is a Director at Decideum. Go to









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Are young people excited by a career in industry?

by Amy Schofield 14. March 2018 11:52



Breakthroughs, tech and innovation are abundant, but are young people excited by a career in industry?


Ask children what they want to do with the rest of their lives and you'll get a range of responses, such as “train driver”, “YouTuber” or “Ronaldo”. How often will you get the answer, “I want to work in the pharmaceutical industry”? But it’s a career with a clear career framework and opportunities to use myriad skills in numerous roles. It also provides the support to succeed and the chance to make a real difference to patients’ lives. What’s not to love? Are young people seeing the potential in taking the pharma path, or is it a struggle to attract the best young talent to the industry? We ask our experts.



Joanna Paish

Recruitment Manager at Apodi Ltd

An acknowledged skills shortage in young people with science qualifications led to a new Sector Deal for Life Sciences being announced by the Government late last year. The perception of top pharmaceutical companies is improving year-on-year according to the 2017 Reputation Institute Report, however, opportunities in pharma are not always well known by young people, despite the industry offering competitive packages and excellent career prospects.

At Apodi we think it is great to hear about new initiatives within the industry that provide graduate programmes, apprenticeships, internships and other training initiatives that will facilitate opportunities to enter the industry in the future.



Caroline Wilcher

Director of Recruitment and Talent Aquisition at Ashfield, part of UDG Healthcare plc

When I graduated 25 years ago, I knew I wanted to be a pharma rep.

At the time, my tutors were mystified as to why I would want a career in sales and tried to push me into R&D, which at that time was a very viable option where many companies were investing heavily. Nevertheless, I was determined to follow my own path.

Now, R&D roles are few and far between, but sales roles still have a negative stigma within the academic world. Sadly, there is still very little awareness of the incredible career paths that this route can offer.

In my opinion, the industry needs to work harder to communicate effectively with universities and spot future talent – we need to build relationships and support their requirements.

We spend a lot of time liaising with employability managers and graduates across the country and have our very own graduate ambassadors attending workshops and seminars.

Combining a passion for science with great influencing skills and business acumen isn’t easy – but for the right kind of person, I couldn’t recommend a career in pharma sales more.



Michele White

HR Director UK & Ireland, UCB

Our applicant rates demonstrate that a career in pharma is an attractive prospect to those currently in education. Many individuals, however, do not appear to have a ‘target’ pharma company in mind when starting their search, or an awareness of the breadth of organisations operating in this sector.

Some graduates and postgraduates joining UCB from university or an academic role said they were attracted by the opportunity to impact drug-discovery and make a difference to the lives of patients.  In the pharmaceutical industry, they interact with colleagues responsible for discovering, developing, testing and launching drugs.

We would like to continue attracting high-quality candidates and make them aware of the wide variety of long-term career opportunities available to them. In particular, we need to attract medics, geneticists and those with skills and qualifications in chemistry, translational biology, early pipeline development and bioinformatics.



Andrew Croydon

Head of Education and Academic Liaison, ABPI

The ABPI is always encouraging more young people to consider a career in an industry, pushing back the boundaries of science. We provide interactive materials aimed at young people and their career advisors, take part in outreach programmes and support specific careers events such as university Bioscience Careers Days.

Our industry has a lot to offer, but we know there are major skills gaps that need to be addressed if the UK is to be ready to research and develop the medicines and vaccines of the future.

In our most recent skills report, we identified gaps in areas such as maths, bioinformatics, statistics, data and informatics, computational skills and translational medicine or clinical pharmacology. All these areas need addressing for the industry to thrive.

We know that young people are showing an interest, however, and are actively pursuing a career in the pharmaceutical industry. University careers advisors tell us that young people are expressing an interest in regulatory affairs, promotion of medicines and, overwhelmingly, R&D roles.

When we surveyed our member companies, they told us that apprenticeships are increasing in popularity as a career entry route, but these aren’t the only solution to bridging the skills gap.

If our industry is to recruit the talented individuals we need, we must get even better at demonstrating the globally competitive benefits of working in the industry, the diversity of roles and continually evolving career paths, while explaining how potential employees will be at the cutting-edge of science, helping create the next generation of medicines for patients around the world.



Getting into pharma

Words by Rachel Cresswell, Sales and Marketing Manager, PharmaJobs


Pharmaceutical companies in the UK currently employ around 73,000 people and this number continues to grow. But with fierce competition among graduates, where’s the best place to start?


1. Why do you want to join the industry?

Why do you want to join pharma, and what is it that you enjoy the most? Graduate roles cover a range of functions, from medical sales to marketing and human resources, so be sure of exactly what you want before telling the company why you can do it.


2. Do you have a related degree?

For many roles, you’ll need a Life Sciences degree, with some requiring specific qualifications such as a PhD. For commercial roles, employers may accept a degree from a different discipline, as long as you can demonstrate the required skills and characteristics. 


3. What work experience do you have?

Having relevant work experience is a great way to stand out from other candidates. Some of the largest pharma companies offer internship schemes, ranging from three months to a year. Alternatively, you can try applying to local hospitals or universities to find out about opportunities. 


Discover available graduate roles at 






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To succeed companies must change their tired sales strategies

by J Pinching 5. March 2018 15:29

Words by Stewart Adkins


Paying lip service to modern sales and marketing isn’t enough, new techniques must be taken seriously and applied.


With the advent of Key Account Management and now digital channels of marketing, it is more important than ever for promotional resources to be analysed and quantified as appropriate.

Even if the assumption were true, that traditional levels of promotional spending were justified by subsequent sales and profits growth that can no longer be an acceptable justification for a promotional budget tweaked from previous years.

Every Commercial Director must start with a zero budget and justify each and every spending item in terms of its impact on sales and its contribution to profit. To do that requires a full understanding of how promotional components interact with every other component. Can you write on a single sheet of paper how your marketing strategy works and then test that strategy?


Testing sales and marketing strategies

Modern methods of data analysis, developed at Cranfield and Aston Universities, have been peer-reviewed and tested in the field, time and again. At least five of the Top 10 pharma companies have used this analysis somewhere within their organisation, as have many medium-sized companies.

These methods are based in theory, but have enormous practical applications and can use a company’s own data, from its CRM system for example, to boost sales and decrease promotional spending, much of which is wasted.

Methods based on regression analysis are unhelpful, as they make too many unsupported assumptions about the relationships between input variables and sales output. Given that only five of the Top 10 pharma companies have outperformed the S&P 500 since Jan 2000 there is a good case to argue that continuing underperformers will face calls for a break up sale – like GSK – to a third party or drastic reorganisation, such as has occurred with Sanofi. Sometimes all three outcomes seem to be on the cards at the same time, damaging employee morale within and shareholder support from without.



Don’t forget the promotional spend

Remember, the doom-mongers’ concerns about reduced returns on R&D use traditional promotional spending levels within the numerator of their ROI calculation. Typically, they will be assuming 25-30% of sales is spent on promotional activities.

A reduction of that spend by five percentage points could almost double returns on R&D. Indeed, Deloitte’s latest research calculates that the top pharma companies have an aggregate return on R&D of just 4.2%. Such a reduction in spending, with no loss of sales momentum, is entirely feasible with modern methods of data analysis and subsequent implementation of conclusions.

The big puzzle today is why so many commercial organisations have sales effectiveness teams and so few have adopted modern statistical tools. The solution to this conundrum may lie in the power of the affiliate, seeing the strength of their commercial organisation as a proxy for their own power and influence.

The application of modern data analysis to CRM and other promotional data could free the country manager from having to make enforced and sometimes arbitrary budgetary cuts. It could also provide the decision-making tools to make rational resource allocations that boost sales and reduce waste.

If used regularly as part of a promotional resource audit – perhaps every promotional cycle – not only should sales and margins rise, but the reduction in strategic drift should make the boom and bust of commercial organisations a less frequent occurrence.


Reality check

For several reasons the pharmaceutical industry is under pressure to deliver better sales and profits growth, and better shareholder returns.

The commercial model that evolved during the supremacy of primary care products, and which served the industry well enough for 20 years, has been slashed and remodelled, supposedly to fit a focus on secondary care.

Despite some reduction in promotional resource overall what remains is a legacy mindset that still considers 25-30% of sales being spent on sales and marketing as appropriate. This is not surprising if budgets are simply tinkered versions of the previous year.

Return to zero budgeting and a thorough analysis of each and every component of promotional spend would be an excellent start. Removal of waste and a focus on activities that actually work would be even better. This would drive sales growth, improve margins and show reasonable returns even on today’s crop of new product offerings.

Such rigorous analysis using modern statistical methodologies might be anathema to affiliate managers, yet the results would become quickly apparent in better commercial key performance indicators. When aggregated they would show up as above expected growth.

Surely an approach that can relieve some of the pressure on beleaguered industry managers, but also begin to reverse the share price underperformance that causes so much soul-searching, must be worth considering.   


Stewart Adkins was a Pharmaceutical Analyst at Lehman Brothers for 23 years and is now a Director of Pharmaforensic Limited. Go to





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Can eating your greens prevent dementia?

by Amy Schofield 26. February 2018 09:52


Don’t believe the hype: health headlines dissected




Research appears to show that eating one or two servings of leafy green vegetables per day could slow the memory decline associated with ageing. The newspapers greeted the study with headlines suggesting that eating your greens could actually stave off dementia. So, does eating a salad a day really keep the doctor away?



Researchers at Rush University Medical Center in Chicago, led by study author Martha Clare Morris, aimed to increase the understanding of biological mechanisms underlying the association between nutrients in green leafy vegetables and cognitive decline.

The primary nutrients and bioactives in greens such as spinach, rocket and kale include vitamin K, lutein, nitrate, folate and kaempferol.

The participants were divided into five equal groups based on how often they ate leafy green vegetables.

For those who ate the most, the rate of decline in scores on thinking and memory tests was slower by 0.05 standardised units per year than the rate for those who ate the least leafy greens – an equivalent difference to being 11 years younger.



This was a prospective study of 960 participants of the Memory and Aging Project. Participants aged 58–99 years – without an existing dementia diagnosis – completed a food frequency questionnaire and had less than two cognitive assessments over a mean period of 4.7 years.

The study results, published in the online issue of Neurology, appeared to show a link between consumption of leafy greens and a slowing of cognitive decline.

The researchers concluded: ‘Consumption of approximately one serving per day of green leafy vegetables and foods rich in phylloquinone, lutein, nitrate, folate, α-Tocopherol, and kaempferol may help to slow cognitive decline with aging.’



The study does build on the body of research that shows a healthy diet could slow memory loss, and proves a fragile link between eating leafy green vegetables and a reduction in cognitive decline and memory loss.

As dementia itself wasn’t measured in the study, at this stage it can’t be concluded that the diet could prevent dementia. In addition, some participants were followed-up for the relatively short period of two years, and it can take longer for memory loss and dementia to develop.

Morris herself pointed out that the study merely showed an association. Dr James Pickett, Head of Research at Alzheimer’s Society, agreed, but added: “What’s good for the heart is good for the head. A healthy diet rich in essential nutrients, combined with regular exercise and avoiding smoking, can help to reduce your risk of developing dementia.”


What the press said:

“A salad a day keeps dementia away, researchers say”

“Eating salad could help stave off dementia, new research reveals”

“A salad a day keeps brains 11 YEARS younger” Mail Online.   



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How can pharma navigate the complex marketing landscape?

by J Pinching 21. February 2018 11:15

Words by Stewart Adkins


The first chapter of pharma’s commercial evolution takes us from the insatiable sales-drive of the 1980s to the present, highly complex marketing landscape.


It is easy to forget that our competitive industry still has 80-90% gross margins and, as a consequence, its traditional commercial model is driven by sales growth, rather than worrying about costs.

Under most circumstances, incremental sales drive incremental profit. Within the affiliates this is obvious, and country managers have often resisted attempts by corporate counterparts to take a centralised approach to sales and marketing, claiming their country’s commercial ecosystem is unique and not amenable to meddling.

Of course, the modern pharma company will also have to conduct market access, medical education and phase IV studies within its affiliates, but the reality is that most affiliate activity is focused on sales. For large pharma companies the sales and marketing budget usually beats R&D budgets by 1.7 times, and this is becoming increasingly difficult to justify.


Rise of primary care dominance

Throughout the 1980s and 90s the focus on sales-driven growth led to the evolution of some very different ways of working within primary care, from co-promotion and co-marketing with embedded local players, to the ‘petal’ system of multiple salesforces detailing overlapping product ranges.

The purpose of these techniques, together with employment of contract sales teams, was a sort of ‘shock and awe’ strategy which swamped the physician with frequent visits about particular products. The competitive response was usually swift and commensurate, resulting in a commercial arms race between players within a hotly contested therapeutic area.

This was known as the ‘share of voice’ model, and when applied to large primary care categories, it drove top line growth so successfully that governments and institutional payers were forced to find a response to escalating drug bills around the world.


Backlash from health technology

This response varied from country to country, but has taken two main forms; the Health Technology Assessment response and the consolidated payer response. Throughout the 1990s and 2000s, in the UK (NICE), much of Europe, Australia and parts of Asia, there has been systematic developing of a process that assesses whether a product represents value for society.

Much of the health economics work is shared among countries, and pricing comparisons made between the same product in different countries are routine. The benchmarks for the monetary value of a healthy human being are the subject of debate, but are necessary to make budgetary choices in a system without unlimited resources.

The consolidated payer model, operating in the US through pharmacy benefit managers such as Express Scripts, relies on large payers exerting pressure on manufacturers for rebates, with some undifferentiated product portfolios having to rebate as much as 50% of their gross price.

The impact of health technology assessments can be seen today, manifesting itself in pricing pressure, therapeutic substitution, a diminution of decision-making by physicians and a conscious shift towards products with a confirmed medical need. A decline in R&D productivity, however, has not made this process easy.


Dead end: Primary care hits a wall

Many commentators blame the decline in R&D productivity for the steep fall in product approvals through the 2000s but, in reality, there have been several forces at work.

The rise in genomics, together with high-speed screening techniques, led to a belief that chemical libraries could be screened against unprecedented targets and that optimised drug candidates would flood through the discovery phase into phase I trials.

The sharp product rise in the early clinical phases then came to a shuddering halt during phase II ‘proof of concept’ studies, when large numbers of clinical failures unveiled the reality – there is no short cut to understanding disease biology.

As research cul de sacs were explored, a squeeze on primary care products began in the form of price pressure from above and greater safety demands from below. As a consequence, and aided by the rise in technology, a rapid increase in the proportion of newly-approved, biological in origin drugs commenced.

Monoclonal antibodies, vaccines, enzyme replacement therapy and other therapeutic peptides, aided by insatiable demand for insulin, developed strong sales and completely changed the nature of commercial interfacing with physicians.


Biologicals change the commercial dynamic

The pressure on primary care products, together with the impact of the patent cliff in 2012/13, have combined to drive sales of primary care products into stagnation. Much of industry downsizing, particularly within commercial operations, has been in response to this.

Perhaps most merger and acquisition activity within pharma also has its origins in this relentless pressure on primary care sales and the need to reload the pipeline quickly with biologicals and specialties.

The success of biologicals and other specialties, such as oral cancer drugs, in terms of both approval and sales, has required the industry to change its commercial emphasis. The huge traditional focus on primary care or family doctors has changed to specialists, and their support workers within a secondary care or hospital environment.

The increased complexity of the specialty sell, sometimes involving multiple decision-makers, formulary approval, health economic arguments, companion diagnostics and performance-related reimbursement, has required a much smaller, but more skilled group of people to interface with the healthcare network.

Many companies have yet to find the necessary mix of skills within their workforce and are still working under the old assumptions that spending on promotional activities can remain as high as it used to be under traditional models. They do so at their peril. Check out Part 2in the next issue, as promotional resources and modern data come under intense scrutiny…   


Stewart Adkins was a Pharmaceutical Analyst at Lehman Brothers for 23 years and is now a Director of Pharmaforensic Limited. Go to





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Mark Samuels' mission to accelerate access to medicine

by J Pinching 20. February 2018 13:26


Mark Samuels is on a mission to get medicines to patients and start dynamic friendships.


There’s a lot of talk in this business about ‘putting the patient first’, but my guest today backs it up by repeatedly putting his neck on the line, to prove he’s doing just that. Chief Business and Strategy Officer at Medicines Discovery Catapult, Mark Samuels, doesn’t like a simple existence and prefers climbing seemingly insurmountable obstacles – metaphorically and literally. Let’s find out why.


What is the brilliantly-named Medicines Discovery Catapult?


The purpose of the Catapult is to support the life sciences sector and, in particular, small to medium-sized enterprises (SMEs) at the early stage of research. Many of these will be biotech companies, diagnostic companies or spin-outs from university. People throw the word ‘exciting’ about a lot in healthcare, but this really is. From a blank sheet of paper, we are now rapidly building and delivering the Catapult’s programmes of work.


In what ways can you help these guys?


We’ve got some very rare scientific kit which, if you were a small company with a highly promising molecule, you wouldn’t otherwise have access to. For instance, we’ve got the UK’s only solid state nuclear magnetic resonance facility, which is vital for drug target validation. Furthermore, we’ve got a vast breadth of expertise, and often this know-how is as important as lab capabilities. By the end of this year, we aim to have over 100 staff, made up of world-class people with commercial, scientific and informatics experience. They don’t just talk about it, they’ve done it for real.


What other ways are there of supercharging the med pathway?


We’re driving collaborations, which combine capabilities from all the different organisations involved. Drug discovery in the UK can seem very fragmented – lots of brilliant teaching hospitals, universities and patient charities doing great work, but it isn’t always obvious to an SME how to bring them together. 


What do patient charities bring to the mix?


Many of them are huge powerhouses in their own right, and they know precisely what a patient needs. This is an area where drug discovery can be strengthened, because eventually people in industry will need to submit patient reported outcomes, so it’s better to trigger that process from the start.


How do you get people who are protective of their discoveries to share information?


I pioneered some of the most complex collaborations in clinical research, when I led the establishment of the Government’s translational research collaborations. These are very large-scale consortia, each involving up to 36 different universities and NHS Trusts, working as one team with industry. So, you can imagine the challenges involved in getting all of them to work together. 


What great things happen when they break bread?


Those partnerships have been running for years now and been responsible for many successful projects, including a trial of the first new drug in 50 years for chronic cough and the world’s largest clinical trial for preventing rheumatoid arthritis. I instigated the ongoing HIV collaboration between Oxford, Cambridge and London biomedical research centres, which is currently undertaking a first in-human trial for a cure. Even against all odds, collaboration can happen as a force for good.


In the vast healthcare landscape, where does the Catapult sit among NICE, the NHS and so on?


As a broker and neutral convener. We’re funded by Innovate UK and, therefore, not-for-profit and here to benefit the UK. I probably have an unusual breadth of insight into UK life sciences; for example, I represented the UK’s diagnostic industry in co-chairing the establishment of NICE’s programme to evaluate diagnostics. I also know what the industry’s position on submissions is, from my time on the strategy board of the NHS’ research arm. At the Catapult, we’ve certainly got a wide range of perspectives we can bring together.


Did you always plan for a career in life sciences?


I studied molecular biology for my first degree, then went to work for Unilever and spent many years in fast-moving consumer goods in the UK, Holland and France. I had a great time and at one point in my career, worked on marketing for Star Wars – a dream job!


That’s an usual career path, Mark. 


I definitely come from a different background – I was a serious mountaineer in my 20s and 30s, and took six months off to climb the Alps and Andes. I was on a mountain called Aconcagua, when I ran out of money and realised it was time to get a job. It was sheer serendipity that, while I was there, I bumped into another couple of climbers who happened to be recruitment consultants for the life sciences sector.

On top of a mountain? Come on, Mark, I wasn’t born yesterday! It’s absolutely true. They introduced me to Roche, and that’s how I came into the industry in 2001. I started in a marketing role and eventually become head of business development for Roche’s diagnostics division. After a decade, I left Roche for a role in Government, where I set up the National Institute for Health Research’s Office for Clinical Research Infrastructure, driving collaboration in the research centres it funded. It was such a big task that many of my respected colleagues told me not to take the job.


How did it pan out?


It had all the challenges of a start-up. At the beginning, we had four members of staff and were operating out of a coffee shop, and yet we were representing the Government, so had all the bureaucracy that comes with that. Within five minutes of starting, Pfizer had announced it was closing Sandwich – but I actually enjoy difficult situations, and things soon improved. We started forming a lot of allies very quickly and built partnerships with many of the foremost scientific leaders in the country. When I started in 2009-10, £30m was coming in through industry investment, and by 2014-15 it was up to £130m.


What are your major ambitions for the Catapult in the future?


Patient-centred collaborations, and starting with the patient need. After all, we are all patients, and we all need this to work.


What record would you choose for the soundtrack of your life?


God is a DJ.


Naturally. It’s your last supper, what are you having?


Pizza and wine, please.


Coming up! Goodbye, Mark.


Bye John.   


Go to





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What is being done to help those suffering from rare conditions?

by Amy Schofield 12. February 2018 15:45


The last day of February every year is Rare Disease Day, but what is being done to help people suffering from these rare conditions?


In the UK, one in 17 people will be affected by a rare disease at some point in their life. Collectively, rare diseases are actually not uncommon.

A rare disease is defined by the European Union as one that affects less than 5 in 10,000 of the general population.

According to the charity Rare Disease UK, there are between 6000 and 8000 known rare diseases and around five new rare diseases are described in medical literature every week.

In the UK, a single rare disease may affect up to 30,000 people. Some diseases will affect only a handful of people, or a single person in an entire country.

Rare diseases are often chronic and life-threatening, and can be single gene, multifactorial, chromosomal or non-genetic. They include rare cancers such as childhood cancers, and conditions such as cystic fibrosis, Huntington’s disease, Duchenne muscular dystrophy and acute myeloid leukaemia.

Rare diseases represent such a large and complex group of conditions, and affect a small proportion of the population, therefore substantial investment by pharma in developing new treatments has always been a barrier to innovation in this area.

Rare diseases actually affect 3.5 million in the UK, and a total of 27-36 million people in the EU have a rare disease. In the UK, one in 17 people will be affected by a rare disease at some point in their life. 

This represents a big opportunity for pharma to meet considerable unmet clinical needs in a market with very limited treatment options, and demonstrates a need to support integration and networking among EU research, patient and healthcare organisations. 

Research from the University of Liverpool in 2016 found that companies which market ‘orphan’ drugs for rare diseases are five times more profitable and have up to 15% higher market value than other drug companies.


What is an orphan drug designation?

Regulatory authorities such as the European Medicines Agency can grant ‘orphan drug status’ to a potential rare disease treatment.

In the EU, this is applied where the potential medicine is for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition that affects not more than five in 10,000 people in the European Union, or where the medicine is unlikely to generate sufficient profit to justify research and development costs. Once authorised, the medicine is given 10 years of market exclusivity.



Accessing treatment

A 2016 report from Rare Disease UK, ‘The Rare Reality – an insight into the patient and family experience of rare disease’ surveyed 1203 UK people affected by rare diseases, representing over 450 different rare conditions. The report found that among those surveyed:

•           For many patients the day-to-day challenges of managing their condition are made worse by the absence of an effective treatment

•           In the absence of effective licensed medicines, unlicensed or off label medicines are an important route of access to treatment for rare disease patients

•           Where a treatment has been licensed for a rare condition, patients may struggle to access it due to the complexities of appraisal and commissioning processes

•           Most patients would participate in research if given the opportunity.



Reality check

The UK Strategy for Rare Diseases sets out ‘a shared six-year vision for improving the lives of all those with rare diseases in the UK by 2020’, incorporating 51 commitments that must be achieved by that year. Each UK country is tasked with taking action and developing plans to implement the strategy that best meet their own health and care systems, but will work together where required. There are discrepancies from country to country however.

For example, Ehlers Danlos Syndrome (EDS), a multi-systemic inherited connective tissue disorder, has no specialists in Scotland, but a number in England. Professor Rodney Grahame, one of the world’s leading experts in EDS, said: “No other condition in the history of modern medicine has been neglected in such a way as Ehlers-Danlos Syndrome”.

Rebecca Holmes has EDS and campaigns on behalf of sufferers. “There are several different types of EDS and, irrespective of the type, symptoms range from being life-changing, to life-limiting, to-life threatening,” she explains.

Rebecca is the Edinburgh Area Coordinator for Ehlers Danlos Support UK, and runs local support groups for those affected by the disease. She also works as part of an action group to lobby for greater awareness of EDS and increased care provision for those who suffer from EDS.

“There is a huge lack of awareness of EDS in Scotland, and perhaps even worse, a massive amount of misinformation. There are currently no specialists in the condition in Scotland. Parents whose children are awaiting diagnosis are being accused of abuse or of having Munchausen’s by proxy syndrome,” she says. “Patients are being misdiagnosed or dismissed entirely; and many are made to believe they are hypochondriacs, or that they suffer from a mental health disorder.”

Rebecca has called for MSPs to take action by getting involved in the Cross Party Group for Rare, Genetic and Undiagnosed Conditions. “We urgently need a patient care pathway for EDS. This would give medical staff the knowledge to put adequate management in place, and improve quality of life and prevent complications. Those with rare diseases are not the majority, but we do exist. And we’re really, really hurting.”



Personal story:

Zoe Gale, 24, on living with Cushing’s Disease.


Considerable weight gain, disruptive menstrual cycle, hair loss, a ‘buffalo hump’, ‘moon face’, stretch marks, a body covered in bruising, loss of muscle mass, insomnia. All of these come together to form the pretty collection of symptoms caused by the rare disease, Cushing’s disease.

At 20 I was relatively healthy and finishing my study abroad year in Madrid. Then symptoms started to surface. As I returned to university for my final year I made an effort to work out, however, this didn’t deter the weight gain. When I went to the doctor I was told, “You’re just stressed out from your final year of university”. I knew this wasn’t the case.

During my final year, trips to the doctor increased with blood tests after blood tests. Still no answers. I returned home where things didn’t get much better. The doctors had no idea what was going on.

A friend mentioned Cushing’s disease, something I’d never heard of, and told me to investigate this with my doctor. I Googled it straight away. It was a rare disease caused by a brain tumour. Surely this wasn’t happening; I was only 22.

My doctor told me I didn’t look like someone who had this disease. I showed him a picture of me before my symptoms started. That was when he referred me to a specialist in endocrinology. On my first appointment he was 99% sure I had Cushing’s disease and that this was caused by a brain tumour. There were more blood tests and an MRI scan to confirm it.

After two years of numerous trips to see five different doctors I finally had a diagnosis. I should have been scared. But just knowing what was actually wrong made me feel much better, and the fact that it was treatable – even if it was through brain surgery – was a huge relief.

It can take decades for an accurate rare disease diagnosis, so what seemed like a long road was maybe a short and lucky street compared to what other people have to go through.

Once diagnosed, I was treated within three months, at St George’s Hospital in London. The surgeon, nurses and staff looked after me with great care on the HDU – they really were incredible.

After two months my life returned to some normality. I went back to work, went out with my friends and started to feel like my life was back to how it should be.

18 months after my operation my health has improved dramatically. I still have to take steroids every day, which could be a lifelong thing, but that’s a small price to pay.

You don’t know how strong you are until you’re faced with a challenge. This would not have been as easy without my family and friends and for that I am truly grateful.

I have moved jobs and now work in the marketing department of an events company that focuses on highlighting the importance of genomics to help patients with cancer and rare diseases. I probably would not have gone for the job interview if I had not been through my experience.    

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Communication challenges & opportunities in pharma

by J Pinching 12. February 2018 15:06



Like anyone in pharma, comms departments rely on a vast knowledge of regulation.


All pharma employees are constantly weighing up what they are doing with the welter of regulations that determine what we can and can’t do. Many will have undergone specific ABPI training – I know, because everyone at the company I work for has just gone through it! Indeed, it is a serpent’s nest, but if you know where the danger points are, you can still communicate messages in a way that stimulates and excites.

PR and comms departments representing pharma are often the spark that ignites the flame of knowledge about a new medicine discovery, an approval, a breakthrough or how lives could be changed forever.

These tenacious information machines also toe a notoriously stringent line; always under the watchful eye of regulators. In many ways, what they produce holds a mirror up to the whole of industry.

Let’s hear from two people at pharma’s information highway.



Claire Martin

Director at GCI Health



We are inspired by a desire to improve the health and lives of people worldwide. Whether it’s raising awareness of health issues or new evidence for a drug’s efficacy, we aim to break down what can be an overwhelming amount of information into accessible stories that resonate with audiences.



We’re well versed in industry regulations and try to avoid seeing them as a barrier to impactful communications. Just as every cloud has a silver lining, with every challenge there’s the opportunity to do something different, to find fresh and thought-provoking ways of engaging our audiences. Ultimately, it’s about looking
for innovative solutions within the boundaries set by regulations.



It isn’t every day you find yourself launching a new therapy that’s set to change the path for generations to come, and it isn’t every day you open the papers to find the story you’ve been working on for months splashed across the front pages. Those are the real highlights that allow us, as communicators, to be part of a collective history that is so much bigger than any one function alone.



As an industry we’ve been telling the value story of pharma more explicitly in recent times, but we can still be more transparent about what it takes to deliver pharmaceutical innovation. We need to continue to be responsible as an industry, always ensuring the patient is at the centre of every decision and demonstrating our commitment beyond the provision of a single product.



In this industry it’s vital to hold on to expertise and keep people with knowledge of pharma’s unique terrain. Team consistency enables us to build relationships with our clients, allowing both sides to get the best out of the arrangement.



I think the industry is starting to come round to the fact that more authentic communications, with greater scope for character and originality, resonate with audiences far better than the heavily-scripted, polished lines with which healthcare communications has previously been synonymous. Granted, we work in a highly-regulated environment, but communications professionals shouldn’t feel shackled by it.

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Gwenan White

Director of Comms and PR at AbbVie



We aim to safeguard our company reputation and advocate for a healthcare system where everyone who falls ill gets the best possible outcomes. This means aiming for partnerships with patient organisations and the NHS in all we do, as everyone is working toward the same goal.



Agility can prove a challenge. In the digital age of ever-shorter news cycles it is especially challenging sometimes to manage our obligations in assuring and auditing our external communication, and responding in a timely fashion.



Healthcare is consistently near the top of public priority and debate in the media. This is because it touches all of us. We are privileged to work in an industry that has the potential to have a transformative impact on people’s lives and there are not many communications professionals who are able to say that.



We aim to be positive and directly engage with the real problems facing the NHS. There is no escaping that an increasingly elderly population – largely driven by past success at tackling disease – and long-term funding constraints, are putting major pressures on the health service. Through our Sustainable Healthcare Initiative we aim to be practical. More than 85% of healthcare spend is not on medicines, so we try to think holistically about the opportunities the NHS has to maximise what it does.



2017 saw us awarded company of the year by multiple organisations, including the Pf People Survey company of choice. We were also the second highest pharma company on the list of UK Great Places to Work and ranked #1 in the UK Pharma RepTrak survey for the second year running. Our way of working positively and ethically is being recognised as important and we saw these as validations of the culture we have built here.



Tone is where I think the industry has the biggest opportunity to make a change. We are science companies, while the NHS is full of jargon. As a result, we have become used to a language that alienates. Companies need to be clearer in how we communicate. There is no regulatory barrier that stops us doing that – it is entirely cultural.

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Healing partnerships: Meds & mindfulness

by Amy Schofield 11. February 2018 12:18


As society increasingly embraces a holistic approach to health, industry, HCPs and pharmacists are moving away from treating the symptoms, focussing instead on the whole person.


The AbbVie habit

AbbVie has created a series of mindfulness podcasts for blood cancer patients to support people living with these diseases and their partners or carers.

Together with mindfulness expert Dr Caroline Hoffman, AbbVie developed a series of podcast recordings with support from the UK’s leading blood cancer charities, including Leukaemia CARE, the Lymphoma Association, CLL Support Association and Bloodwise.

They were created in response to the growing body of research which demonstrates the negative psychological impact of blood cancers on patients, including depression, concerns about body image, relationships with others, loss of self-esteem, isolation and financial stress. These mindfulness podcasts aim to help alleviate the emotional stress felt by those living with blood cancers and improve their overall well-being.

Head of Campaigns and Advocacy at Leukaemia CARE, Zack Pemberton-Whiteley, said: “The emotional and psychological impact of a leukaemia diagnosis is often overlooked. It’s easy to fixate on an issue and lose sight of the bigger picture of how it’s affecting you, so it is important that anybody affected by a leukaemia diagnosis takes time to reflect on their wider thoughts. Mindfulness offers a way of doing this.”

There are three modules to the podcasts: mindfulness of the breath; mindfulness of the soles of the feet, and mindfulness of the body.

“You may have recently been diagnosed with a blood cancer or be waiting to see if or how your condition progresses. You may be undergoing tests or treatments, or perhaps you are in remission,” explains Dr Hoffman. “When you practice mindfulness, you are training your brain into positive habits of coping, and responding to the many stressful events that may be happening in your life.”



Social prescribing

Social prescribing is an innovative, personalised approach to health that enables GPs and other frontline HCPs to work with patients taking into account their social, emotional and practical needs to find solutions to their health problems, often using services provided by the voluntary and community sector. The growing social prescription movement has clear benefits for individual patients, potentially reducing demand on the NHS, particularly in primary care services.

Dr William Bird MBE set up Intelligent Health in February 2010 with the vision of transforming the health of millions across the UK through innovative initiatives to get people moving. As a GP, he set up the Health Walk scheme in April 1996, followed by the Green Gym one year later, then the Beat the Street programme, which has encouraged half-a-million people across the world to get moving and improve their health.

“Our current healthcare system tackles the rise in long-term conditions such as diabetes, obesity, depression and dementia. We need to move to the cause of these illnesses and tackle the underlying problems of loneliness, disconnection with the natural environment and a lack of purpose,” he says.
“In order to transform health and inequalities we are moving to a system where social prescribing becomes a norm. This can save our NHS and help build happier, healthier, more connected communities where we would all want to live.”



Going digital

The so-called ‘social network for health’, HealthUnlocked, has created a ‘social prescribing tool’ which harnesses the power of technology to improve patient outcomes beyond the pill. This digital social prescription tool, integrated into the clinical system EMIS Web, allows GPs to prescribe information, services and online support.

“Social prescriptions are a new way of approaching the management of chronic health conditions in primary care. By signposting non-medical services and resources we can empower people to engage themselves in their health and lifestyle,” explains HealthUnlocked’s Chief Medical Officer, Dr Matt Jameson Evans. “Using our technology we have been able to incorporate the peer support already improving outcomes in online HealthUnlocked communities, and bring it together with voluntary and CCG services. By doing so we can offer a digital version of social prescription to GPs that they find easy to use during the consultation, and their patients find beneficial.”

Initially live in two GP practices, the technology will be rolled out across practices in North East London as part of a partnership between Innovation Test Bed, Care City and HealthUnlocked.

“Over the past year we have seen a significant rise in practices looking to implement this and people actively looking for these type of resources,” says Dr Jameson Evans. “Social prescription in this form may be a relatively new concept, but by further enabling and embracing the technology that does it, we have the potential to give people the tools for a better quality of life, reduce reliance on medications, lessen use of GPs and A&E and radically rethink how we manage chronic health conditions.”



Here & Now

Novartis’ Here & Now campaign is a pan-European advanced breast cancer (ABC) awareness initiative from Novartis Oncology. The project aims to raise awareness of ABC, uncover new insights into its impact and support those living with the disease across Europe, through ‘a series of creative and thought-provoking activities’. This includes a ‘Mindfulness patient resource’, a guide for ABC patients who suffer from depression and emotional stress resulting from their illness.

Here & Now signed up international mindfulness coach, Greg Burdulis, to develop a mindfulness resource that could psychologically and emotionally support women living with ABC. The mindfulness guide consists of five modules that use mindfulness and motivational messages to develop skills: calm, self-compassion, gratitude, pain management and active imagination.

Each exercise along with its introduction lasts 15 to 20 minutes and can be downloaded onto a smartphone or tablet.



Community minded

Jane Devenish, NHS Standards and Services Pharmacist, Well Pharmacy


Community pharmacy also plays a vital role in this more balanced approach to healthcare. Jane Devenish, at Well, says that community pharmacists have the opportunity to have a different relationship with their patients, one that supports the patient to make small changes that can make a big difference to their health.

“Person-centred care is one of the GPhC standards, recognising that improving what matters to the patient is the most important health outcome, and this usually can’t be done by medication alone,” says Jane.

It’s the accessible ‘local touch’ that enables community pharmacy to offer more than a prescription service. “Pharmacists and technicians who work in the community have great links with other organisations in their locality, directing people through the complex network of health and social care,” explains Jane. “Increasingly, this includes charity and social care groups, such as choirs, to improve breathing, or groups for people with Parkinson’s disease or dementia.”



Completely healthy

Pfizer’s ‘Wellness’ section of its website also advocates the power of the ‘whole person’ approach in improving patient wellbeing, featuring tips and advice on social connectedness.

The company offers patients a wealth of information on how to promote complete wellness by adopting a wide-ranging approach to health, including connecting with others, trying new experiences, staying active, tackling insomnia, cultivating healthy sleep habits, eating well and mindfulness techniques.    



Needle work

Ian Rubenstein, GP at Eagle House Surgery in Enfield since 1984, he uses complementary techniques in addition to modern medicine when treating his patients.


I taught myself hypnosis at school and my GP trainer taught me acupuncture in 1982. I am trained in counselling and I’ve written about using placebo in medicine.

In the 1980s I used hypnosis during my regular consultations and I was also clinical hypnotist for the Whittington Hospital cognitive behavioural therapy pain management programme.

As general practice became increasingly busy I found I had less time for hypnosis, so I decided to concentrate on using acupuncture for the relief of pain. This is popular with my patients and colleagues, and forms an important part of my current work.

My conventional practice has always been informed by my alternative interests. Sometimes these are extremely unconventional, such as when I sat in with a group of trainee psychic mediums! It all adds to the mix and keeps me engaged with my patients.

Modern medicine is very effective, but it has become more technical, less personal and many people have become disenchanted with it.

I want to encourage doctors to bring the enchantment back by marrying modern medicine with traditional practices.



Go to Go to For further reference, check out Dr Rubenstein’s book, ‘Consulting Spirit: A Doctor's Experience with Practical Mediumship’, available on Amazon.






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Should the NHS be privatised?

by J Pinching 29. January 2018 12:23



An all-new opinion battle where one divisive subject erupts, giving way to diametrically opposing views from two senior healthcare commentators.



Jane DeVille-Almond, Independent Nurse Consultant and journalist


Having worked in the NHS for almost two thirds of its existence, I love it and its values. Anyone who dedicates a life to the NHS is committed to Bevan’s principles – healthcare provision based on clinical need, not on ability to pay.

But healthcare is not free. Healthcare costs the NHS £116 billion per year and the money comes from us.

The NHS was developed at a time when children were still dying of polio, before kidney transplants, heart bypasses, breast screening and fertility treatment.
Who could have foreseen the change in UK demographics and the impact this would have?

For fear of losing popularity, no one in Government seems prepared to admit that the NHS is simply too expensive to run without making difficult changes.

Already we are seeing community services for the elderly stretched, mental health services almost non-existent in many areas, GPs so overwhelmed they are leaving and nurses so overworked that hospitals are unable to recruit them. The big fear is, if we refuse to look at other models, the NHS could fold completely.

The NHS loses millions every year. Partly because many have no respect for it, partly because people have little idea of the real costs, and partly because no one is in control.

For those who can afford it, we should charge a fee for visiting a GP. Where people can’t, then we subsidise, as they do in Jersey. Charge people for missed appointments, make sure people know the cost of medicine waste and encourage people to look after their own health. We need to stop living in the past and move forward with innovations brought by involvement with the private sector.




Dr Guy Pilkington, Assistant Chair of NHS Newcastle Gateshead Clinical Commissioning Group


The challenges facing healthcare systems across the world are as great now as they have ever been. Why should we stick to the founding principles of the NHS, when the pressures upon it grows inexorably? Let me explain.

The NHS is designed to be comprehensive and accessible to all regardless of ability to pay. The welfare state was created as a universal service and remains one of the few foundations on which our sense of shared community is built. Margaret Thatcher was wrong, there is such a thing as society and the NHS remains the clearest articulation of that.

Remind yourself of these values and read the NHS Constitution when you get the chance. The most cost-effective way to fund a universal system is through general taxation. We have an ageing population, pressures grow year on year, so let us commit to the best way of doing that.

The NHS is far from perfect. I believe it has yet to invest in prevention, mental health and caring for the most marginalised at the levels required to tackle stark inequalities we see across the nation. Part of that requires us to stop over-medicalising many of the things we deal with on a daily basis – unhappiness, loneliness, the effects of poverty and traumatic events in childhood.

A privatised system is incapable of rising to these challenges, and will worsen the inequalities of access and outcome we have today, costing the nation far more. 

Remember, the UK was judged as the top rated health service in the Commonwealth Fund think tank 2017 comparison, despite nearly 10 years of funding squeezes and a much lower proportion of GDP spend on healthcare than more privatised systems.    




Mark Loughridge from M&F Health Communications


The ‘privatisation’ debate has always struck me as characteristic of the problem that has dogged meaningful discussion of NHS policy, and wider public policy to boot. 

Put simply, the problem is one in which a complex series of questions are posed - ‘how can we manage the demographic pressures facing our health service? And, can the NHS keep up with revolutions in medical science? Or, is there still a role for local commissioning in an increasingly collaborative system?’

These questions normally receive a knee-jerk, one-dimensional answer. It is much like the Bank of England looking to discuss how best to manage an uplift in the rate of CPI inflation, only to trigger a spirited debate about whether we should abolish currency altogether.

In a system as clear as mud, where formidable challenges seem to lurk behind every bedpan, privatisation is a soothing medicament for the overwhelmed. But it provides no real answers to the intricate problems facing the health service, instead offering a reheated brand of 80s economics when far more nuance is required. 

Ultimately, an insurance-based system answers none of the questions, and offers no solution to perhaps the greatest problem it would inevitably create: extraordinary operational and political upheaval in an uncertain and febrile climate.

So, let’s ignore the false friend of privatisation and push for real reform in our health service. Let’s challenge ourselves to create innovative change, firmly rooted in the fundamental principle of an NHS free at the point of use.   






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