Vertex Pharma’s vision for reimbursement innovation

As regional vice president for Northern Europe & Australia, Simon Lem led the digital launch of Vertex Pharma’s novel cystic fibrosis drug Kaftrio. He tells pharmaphorum about embracing digital change and the need for innovative reimbursement solutions.

For Vertex, COVID-19 accelerated a digital transformation much needed for the patients they serve.

As the leading developer of novel drugs to treat cystic fibrosis (CF) the Boston-based company has always taken a very focused approach.

“For CF, you have a proportion of the population predominantly quite young and tech-savvy. They do not want to be coming into the hospital,” Lem tells pharmaphorum. “There was already a direction of the clinical community to be having these interactions or helping patients manage their disease virtually. COVID-19 sped up all of that.”

With COVID-19 posing an added risk to people with CF, Vertex was “even more motivated” to secure its supply chain and access to drugs. In June 2020, NHS England swiftly approved a reimbursement deal for Vertex’s fourth CF drug Kaftrio meaning the medicine would be accessible for doctors and eligible patients in England from the day (in August 2020) it received licensing from the European Commission, with the medicine being accessible by eligible patients in Scotland, Wales and Northern Ireland soon after. The drug has since launched digitally.

“I never thought that in my career, I would see a launch of a significant product without having a face to face interaction with a customer, but it has worked. Early results are positive, it hasn't changed the mission,” says Lem.

Like other pharmaceutical companies, Vertex still faces challenges with reimbursement and market access. The agreement for Kaftrio followed a long battle with NHS England to make the company’s three other cystic fibrosis medicines available.

“The challenge we face is universal to any biotech company. You are operating in multiple different countries with multiple different healthcare systems, multiple different cultures and all the different ways they approach, reimbursement, or engagement,” explains Lem.

“It’s one thing if you can develop an innovative medicine, but then you've got to get it to patients for it to be very useful and I think that is challenging for many healthcare systems, particularly ex-US where you have finite and limited budgets. You also have very well-established long-term health technology, appraisal systems.”

The speedy nature of Kaftrio’s approval is encouraging for how reimbursement could look in the future, says Lem. Working flexibly with NHS England and NICE, Vertex managed to ensure patients got access to the drug on the day of licensing.

“I don't know of many examples where that's happened before if any, but primarily it was about taking a step back and asking what we had learned from our ongoing discussions and how we could bring those forward into the next step of the negotiations.”

Engaging with the health system as early as possible and working flexibly was key to the success, he adds. “It has to be more collaborative. Especially if you move into things like gene therapies.”

UK’s opportunity for change

The UK government also has an opportunity now to speed up the process, Lem says. “I think sometimes other healthcare systems have looked at the UK as the ones that are playing catch up, and there is a less flexibility to change, but I think the UK could lead the way with this. We saw with our CF medicines; they can do it if all parties are motivated to do it.”

A more flexible approval process would also benefit patients within the UK. “Sometimes patients in the UK only get access to medicines much later than other European countries.  I think there needs to be innovation, flexibility, and open communication with all parties involved in in the process including the HTA system and the health authorities."

A bigger challenge for healthcare systems in future will be deciding reimbursement for curative medicines. “We're at a very embryonic stage across the pharmaceutical industry. There's going to have to be even more collaboration, even more flexibility and even more talking at a far earlier stage than we've done previously.”

Starting his pharma career as a sales rep, Lem has worked across the industry in management and marketing roles. He notes how business practice has changed significantly over the years. “Now we are far more focused on individual patients and individual prescribers and that has changed how you go about doing the businesses. It is far more personalised, and that's down from a key account manager's perspective to even reimbursement.”

Diversifying focus

Vertex's focus for now is on being less of “a one-franchise organisation” and diversifying its portfolio. The company is investigating new medicines for serious diseases with key unmet needs.

“We want to treat the underlying cause of the disease," says Lem. "It will be interesting seeing how that pipeline pans out and the impact that that will have on the business.”

Expanding into new diseases areas will require a different approach from the organisation, says Lem, but presents an exciting new challenge. Having worked at Vertex for nine years, he says that no day is the same as the day before.

“I can tell you what I am doing today is not the same as what I was doing this time last year. I think it's a really exciting journey because we're doing stuff so rapidly and having such a big impact that and we go into new disease areas.”

However, the goal to find a cure for CF remains core to the company.

“People say to me, 'Are you happy? Patients have got the medicines. It's over and done with.' I say, no, it is not - there is still a lot of work to do for CF. We brought four medicines to the market. Yet there's still work to be done.

“We’ve got medicines with the potential to treat up to 90% of people with CF, but the other 10% still don't have a medicine. Until that 10% have got a medicine, the game's not over.”

About the interviewee

Simon Lem is regional vice president of Northern Europe and Australia at Vertex and has 23 years’ experience in the Pharma industry, including previous roles at Novartis and Biogen. He joined Vertex in 2012 as a country manager, setting up the UK and Irish business.

He is now responsible for the leadership of Northern Europe (UK, Ireland, Sweden, Denmark, Norway, Finland) and Australian commercial organisations and has proven success in launching medicines within rare diseases including achievement of unique and ground breaking reimbursement agreements.

About the author

Dr Paul Tunnah founded pharmaphorum in 2009, which combines industry leading publications (pharmaphorum) with a specialist strategy and content marketing/communications consultancy (pharmaphorum connect). He is a recognised author, speaker and industry advisor on content marketing, communications and digital innovation, having worked with many of the world’s leading pharmaceutical companies and the broader ecosystem of healthcare organisations.

In June 2020, he became chief content officer for Healthware Group, a next-generation integrated consulting group that operates at the intersection of the transformation of commercial operations and digital health, offering a unique range of services combining design, strategy, communication and innovation with technology and corporate venturing. Connect with Dr Tunnah at LinkedIn or Twitter