Looking ahead to what 2017 will bring for the pharma industry from the vantage point of January and the only thing that seems certain is change.
The fall-out from the UK's vote to leave the European Union has so far favoured uncertainty over stability - Brexit may mean Brexit to the UK's prime minister, but for the rest of us it's nigh on impossible to know what it will really mean.
Meanwhile, in the UK the health service seems ever-more cash-strapped and its health technology assessment processes appear to be much in need of an update, with a consultation on the latter underway.
Change has also been the order of the day at Shire, with the Dublin-headquartered company taking strides towards future growth over the last couple of years, with rare diseases and orphan drugs very much in its sights.
The biggest of these moves was its $32bn acquisition of Baxalta, which was officially completed in June last year and elevated its position in orphan drugs, which it expects to account for around 65% of its revenue by 2020.
“The combination of Shire and Baxalta was extremely transformative,” says Sebastian Stachowiak, the general manager of Shire UK and Ireland. “It allowed us to solidify our position as the world's largest rare disease company and we now are the leaders in haematology, lysosomal storage disorders with strong capabilities in ADHD and HAE.”
The enlarged firm has its sights set on quickly ramping up sales - its combined portfolio of products generated over $12bn in revenue last year and Shire has an annual revenue target of $20bn by 2020. Underpinning this, and its longer-term growth, is a pipeline that encompasses over 35 products in development, over ten of which are in phase III clinical trials - 75% of them geared towards rare diseases. Taken together Sebastian says the firm's pipeline is “one of the richest in industry”.
But it's not so long ago that Shire itself was an acquisition target, at least until AbbVie stepped away from a $55bn deal to combine the firms after the US took steps to prevent 'tax inversion' mergers. Since then the Baxalta deal has been Shire's biggest expansion move, but it's also taken several other steps to build up operations, acquiring rare disease firm Dyax in a $6bn deal that was completed in January 2016. Before that the firm had a busy 2015, buying US rare disease-focused biotech NPS Pharma for $5.2bn and rare disease and gastrointestinal company Meritage Pharma for at least $70m in two February deals, and then in August picking up Foresight Biotherapeutics, an ophthalmology company for $300m.
For Shire's Sebastian Stachowiak, having only joined the firm in August 2015, the changes have come in quick succession. “Since I've been with the company it has changed tremendously, so the Shire of today is certainly different than the company that I joined in 2015, but more in terms of the pipeline and the scale of it. Our underlying culture is still to be a leading global biotech, to be agile and to be transformative. And I don't think that's changed, we just have a broader scale now than we had before.” To accommodate that scale the company also starts 2017 with a new UK base, having moved its operations from Basingstoke to more central premises in Paddington in west London in December.
Our underlying culture is still to be a leading global biotech, to be agile and to be transformative
Sebastian joined Shire from Cubist, whose commercial operations for the UK, Ireland and Nordics he established to launch two novel antibiotics. This was a real 'from the ground-up' role in which he was tasked with concurrently establishing a beachhead for the company and bringing new products to market. “There is a difference when you work in companies where the infrastructure is already in place and you have to launch new therapies versus where you're setting up the infrastructure and launching a new therapy at the same time.”
Thinking back to that time Sebastian says it provided lessons that will stay with him for the rest of his career.
“It's an environment where, as an individual, you have to really roll up your sleeves, and you're much more personally accountable for a broader set of functional expertise. To set up an office from scratch, to set up the legal entities, to have employees on board while at the same time developing the market access, health technology assessment (HTA) strategy and going through regulatory approvals can be challenging for individuals and the organisation.
“But at the same time, you learn so much more about the way companies operate, and about the regulatory and market access environment. I was closer to every single detail of what was happening than I could be in a bigger company. It really made me realise that when you have pivotal projects and timelines, as an individual, as a manager, you really have to be at the centre of what's happening and on top of things.”
Prior to this he held a number of roles at Astellas, most recently as specialty care director for the UK business, and country manager, for Finland, where he established the company's first offices. “Finland was an amazing experience. On a personal level, I enjoyed my two years there and made a lot of good friends. The Finnish people are extremely hardworking and it takes a while to really understand the culture, and the language is extremely difficult, but once you're a friend of a Finn, you're a lifelong friend. So I really appreciated the personal aspect of my experience there.”
However, such senior roles were not achieved in a conventional manner. Sebastian entered pharma in 2003 having studied for an MBA, focusing on pharmaceutical sales management and marketing, at the University of Wollongong, Australia.
“When I was studying the industry, the one thing that amazed me was its complexity. It's so multidimensional that in order to have a full appreciation of the industry, you need a lot of years of experience and a real breadth of positions and viewpoints.” It's something that Sebastian's early roles certainly provided.After an academic start, he had a year-long stint as a sales rep in London for Schering-Plough moving quickly moving through market research, strategic planning and on to commercial effectiveness and market planning.
“One of the things that's helped is to not have a traditional career path within the industry, but to take unorthodox moves as my next steps that broadened my knowledge of the industry.”
A rare challenge
That knowledge is currently focused on bringing new treatments to market for rare diseases and working to ensure the environment for orphan drugs is fit-for-purpose, twin aims Sebastian says form his biggest long-term project.
At the forefront of his mind is the position of access to treatments for rare diseases in England. The country lags behind the rest of Europe, with only around 70 of the 120 approved orphan drugs able to be routinely used on the NHS.
As part of his efforts in this area Sebastian chairs the Rare Disease Strategy Group with UK industry body the ABPI. It involves companies who either already specialise in orphan drugs or have some in their R&D pipeline, and it's ultimate remit is to ensure the environment is 'fit-for-purpose'. However, it hasn't yet started formally engaging with NHS England or NICE, waiting instead for individual companies, and the ABPI itself, to respond to the joint NHS England/NICE consultation on changes to the way drugs are evaluated and funded through the STA and HST programmes. That consultation is due to close on 13 January and until then the focus has been on increasing levels of awareness.
“One of the group's aims is around communicating the fact that rare diseases aren't rare, but as a movement it's not centralised or very cohesive. Unlike, for example, oncology, where there's a certain awareness of it and it resonates with people.
“The second aspect for the group is that, while the market access environment has been positive, it is also challenging by its very nature. The UK is the slowest in terms of uptake as well when we benchmark versus the top five EU countries.
“So if you benchmark versus Germany, Italy and France, not only do we have one of the lowest rates of uptake in terms of the rare disease space, we also have the slowest rate of uptake. Is that really a position that we want to be in as a society moving forward? And so can we work with NICE and NHS in England to have an environment that's fit-for-purpose?”
Currently NICE will only assess drugs through its Standard Technology Appraisal (STA) process or via its Highly Specialised Therapies (HST) one. The latter, says Sebastian, is “a very good first step, in terms of recognising that there is a difference when it comes to ultra-orphan medicines”.
But, he notes, there's also a capacity issue in that NICE can only put around three of the available orphan drugs through the HST process in any given year. “By its very nature there's going to be a backlog, and some real kind of trade-offs will need to be made.”
Added to which he says “there's a really stringent criterion in terms of which orphan drugs go through the NICE HST process”. This is, he adds, “extremely restrictive”, and he points to Revestive (teduglutide), Shire's treatment for the rare and potentially fatal gastrointestinal disorder short bowel syndrome. Despite a patient population of 500 the drug has been put into NICE's standard STA process, “which isn't geared towards evaluating ultra-rare disease products, so the chances of you getting a positive in that environment are slim to none, to be honest”.
“There is a body of work that needs to happen between industry, patient associations, the clinical community, NICE and NHS England around setting up a more holistic NICE process where we can look at orphan drugs in their entirety as opposed to having one system for ultra-orphans and then everything else defers to a general system, that's not geared towards rare diseases.”
He says there is a need for stakeholders to work together to set up a system that's “fit-for-purpose for this wave of innovation in rare diseases that's coming through industry and making sure that ultimately patients don't suffer by not having access to it”.
There's a body of work that needs to happen… around setting up a more holistic NICE process
The affordability question
Winter, with its flu crises and bed shortages, brings the UK's healthcare spending into sharp relief, but the affordability question facing the NHS isn't a seasonal dilemma. With the National Audit Office expressing concerns in November about the financial sustainability of the NHS, pressures are expected to peak in 2018/19 and 2019/20, begging the question, can the affordability question ever really be answered?
“For me it's ultimately what does the population of the UK want from the NHS,” says Sebastian. “We are working with our NHS and NICE partners to address affordability and we do deliver value with the products that are coming through. Ultimately, long term, the question for the nation is, what is the appropriate level of investment in the NHS?
“To me that is more of a societal question that needs to be addressed in terms of how much and what importance is there from a societal perspective on the NHS moving forward, and do we want this to be a best-in-class world health economy? And if we do, then we have to question the amount of investment that's going in right now.”
Making sense of change
From a newly enlarged company to new premises, to - perhaps - new HTA arrangements for rare disease treatments to the meaning of Brexit.
For pharma as a whole it presents a huge challenge, and Sebastian was among the industry leaders expressing their support prior to the EU vote for the UK to remain in Europe.
Now, with Brexit a reality, he says the referendum result hasn't caused Shire to put any of its plans on hold or stopped any investment in the UK. “In the immediate aftermath of Brexit, it hasn't changed the way that we operate in the UK.”
But the question for Shire, and the wider industry, is about the long-term impact of the move. “We are very dependent on the regulatory authorities, and therefore the ultimate decision of the regulatory pathway that the UK will operate under will be critical to our industry. As an industry, we exported about £30bn worth of goods in 2015, therefore any restrictions on the exportation of goods out of the country will have an impact on us,” he explains.
“And as an industry, we rely on great scientific research to advance therapies, and that requires a collaborative way of thinking. And that collaboration can be achieved if you have people from different cultures, different backgrounds, coming together to try to address one goal. Therefore, for us, the freedom of movement of scientific capital and human capital is extremely important.
“From a corporate perspective, we're taking a cautious approach, and we're trying to see prospectively where Brexit is going. We're looking at all the scenarios that are in front of us and, ultimately, depending on what the environment looks like, we will make the appropriate decisions. But right now, it's just too early.”
He concludes: “I can't really speak to what other companies are doing. For us, it hasn't stopped any decisions that were already in process. For any major decisions moving forward, we will have to evaluate what Brexit actually means.”