These are exciting times to be operating in the rare diseases space in the UK. Two and a half years after the launch of the country’s first Rare Disease Strategy, good progress has been made – although there is more to do.
Meanwhile, the 100,000 Genomes Project is making strong progress in the creation of a new genomic medicine service for the NHS and, more recently, political momentum has been building in the area, with the formation in February of the All Party Parliamentary Group on Rare, Genetic and Undiagnosed Conditions.
One direction all of these initiatives are pointing towards is a greater awareness that rare diseases are not all that ‘rare’. With between 5,000 and 8,000 identified so far, each rare disease affects less than 0.1% of the UK’s population, but together they affect the lives of three million people.
One of the people acutely aware of the scale of the issue – and the potential for pharma to transform it – is Neil Dugdale, UK and Ireland general manager for Sobi (Swedish Orphan Biovitrum) a company that, as its name suggests, is entirely dedicated to rare conditions.
“There is an opportunity for the UK to lead Europe and the world in trying to identify more effective treatments, or new treatments, for currently untreated rare diseases,” he tells PME.
“The UK’s 100,000 Genome Project is a world-leading push to map the genomes of 100,000 patients that have a rare disease, to try to understand what is causing their disease, to understand the mechanism behind that, and to look at new – but also current – treatments that perhaps could help people who are in that position.”
The UK, in common with many developed markets, has large numbers of products that, while they do have a licence and are currently available, are rarely developed as orphan drugs. “There are a lot of products that are used off-licence very effectively, but it’s extremely difficult in small patient numbers to ever get a licence for a product to be used in a rare disease condition.”
Neil says his hope is for continued government investment in the 100,000 Genome Project and that its outputs can be used “to try to get effective treatments for patients”. He also points to the government’s Accelerated Access Review as another area offering hope.
The Review aims to speed up NHS use of transformative drugs, devices, diagnostics and digital health technologies and its final report is now due to be published later in the summer. “One of our responses to the Review as a company was to try to make sure that we don’t just concentrate on transformation, but also focus on simpler innovations. If you’re looking for a really great innovation, it may take a long time to get there, but if you’re open to small innovations that can still really improve healthcare in the UK then that’s simpler to achieve.”
There is an opportunity for the UK to lead Europe and the world in trying to identify new treatments for currently untreated rare diseases
An exciting time
For Neil’s company Sobi it’s certainly been a busy time this year. Sobi UK and RoI launched Elocta at the beginning of the year and in March the EC approved the transfer of the marketing authorisation for Elocta (efmoroctocog alfa) from Biogen to Sobi, handing Sobi commercialisation control over the haemophilia A treatment just four months after it received EU approval. Last month Alprolix was approved by the European Commission, becoming the first licenced long-acting treatment for haemophilia B in the region.
“The haemophilia portfolio is extremely exciting for Sobi,” Neil notes. But he explains that, while the UK was one of the first countries to ‘launch’ Elocta, the UK environment for haemophilia is a very regimented one, with tender processes for the UK and Ireland to be completed before healthcare professionals can actually prescribe the treatment. “The process is never guaranteed, but we are hoping Elocta will be accessible to patients from 1 July,” he says. The decision on that will be taken by NHS England following consultation with the commercial medicines unit, the Haemophilia CRG and input from the United Kingdom Haemophilia Centre Doctors’ Organisation.
As with any tender process there’s likely to be a heavy focus on price and volume, but Neil is hopeful that clinical effectiveness will also feature strongly. “Elocta is an excellent product – it’s the first extended half-life treatment for haemophilia A and the first real advance in the area for patients for over 20 years.”
He adds: “There is a key difference in the UK compared to many other competitive neighbouring countries. If you look at the cost of haemophilia treatments in the UK compared to the rest of Europe, the UK is now one of the lowest-priced countries.”
Compared to normal timelines, the requirements for haemophilia could be quite frustrating, but Neil is sanguine about the need to wait, despite Elocta being approved in Europe and ‘launched’. “You get used to the processes that we have to operate within, and we need to respect the commissioners and budget holders, because they haven’t got an easy job.”
At a time when NHS hospitals in England are running a record deficit of £2.45bn – with suggestions the true scale of overspend could be even higher – Neil is very aware of the difficult job Trusts face.
“The Sobi way is to try and make sure that we’re adding value to patients, healthcare professionals and budget holders, which means we’re showing them what our treatments will offer and we’re pricing them accordingly. We do not want a product which is at a price where few patients can access it and it puts too much pressure on the healthcare system. That’s not good for any of us.”
Neil adds: “At Sobi we talk about ‘patient access’ instead of ‘market access’ – it’s a very clear distinction.”
Making a difference
Neil’s path to the industry and Sobi began in a fairly unusual way, with a stint as a scuba diving instructor around the Great Barrier Reef providing a stepping-stone to pharma and diverting Neil from plans to enter the Royal Marines. Travelling after university he landed up in Cairns, working in a backpacker’s hostel selling dives, getting a free trip for every 10 sold. Three months later he’d become a dive master and, while leading a dive trip, met some people working in pharma who explained what a fantastic job it was.
He subsequently joined Merck in 1994 and went from rep to product manager, group product manager to business unit director. Selected for its leadership development programme he was all set to move to Johannesburg to lead Merck generics in South Africa, when the corporate winds changed and the firm decided to sell its generics business to Mylan. The disappearance of that opportunity combined with changes at Merck following its €10.6bn acquisition of Serono gave Neil the momentum to set up his own consultancy business in 2007. He worked for a number of pharma clients, including Eisai and Merck Serono, the latter of which he began working for as an interim oncology director, before rejoining the company on a permanent basis.
This was followed last year by the chance to join Sobi as its UK and Ireland general manager. That brings the story up to the present, but Neil concludes by looking to the future of rare diseases in the UK. “I would hope that the 100,000 Genome Project, the Accelerated Access Review and the changes that we’re seeing will eventually help people with rare diseases get the treatments that they deserve,” he concludes.