What is your current role and background?
I’m a commercial director for spinal muscular atrophy (SMA), which means that I head up the SMA business unit. I get involved in all aspects of running the business, such as working with patient advocacy groups, clinicians, and most recently I have been working a lot with healthcare organisations like the National Institute for Health and Care Excellence (NICE) and NHS England. I have been at Biogen for 15 years, and have worked right across our broad portfolio, so I’m very privileged to now be working on SMA. For a while I worked across key functions like market access and supply chain as well as marketing, so I have a real breadth of knowledge from across the industry, which helps me head-up the business unit.
What does your day-to-day job involve?
Every day is slightly different, but what gets me out of bed is that ability to make a difference. Most recently I’ve been working with the likes of NICE and NHS England on the reimbursement of an SMA product – so, now my concentration moves to making sure that the patients get access to the treatment as quickly as possible. So, I’m working together with patient advocacy groups and clinicians, so that we can get closer to what’s actually going on in the SMA community.
What drove your passion for working in the SMA region?
Thinking back, around seven years ago I was shown a video of an SMA patient that detailed the impact it had on them and their family around them. I remember thinking then: This is an area I would really like to work in. I wasn’t in the area at all at the time, so I developed my experience to be able to move into it. SMA is a rare and debilitating disease, and I think to know that and to know that there’s potential to change these patients’ lives, you can’t really help but be drawn to that. So, for me the passion lies in making a difference in this area and I continue to be inspired by the work that we’re doing, and also the work that the clinical community and patient advocacy groups are doing. That’s really what gets me out of bed and drives the passion, and will continue to – because I know there’s still a lot of work to be done in this area.
What do you see as the biggest challenges currently facing rare disease drug reimbursement in the UK?
There’s always high unmet need, but for a very small patient population. With a small patient population it’s hard to achieve the same level of cost-effectiveness for drugs when appraising through the NICE Single Technology Appraisal (STA) process, which is the standard process for products in England. It’s unfair for patients with rare diseases as it takes a long time for treatments to get to them, and so the learning that we’ve taken away from the process is that the financial impact of having a rare disease is often borne by the patient and the patient’s family, often impacting on family employment, among other things. So, it really does have a significant impact on the patient’s life. Loss of productivity costs such as these aren’t considered by NICE when making a decision, which is something we believe needs to change.
What are some key recent advancements in the field?
When I look back there have been some significant changes, it’s a really exciting area to be in. The earlier you treat, the better the outcome is for the patient, and I really see that there’s a lot going on around genetic testing and newborn screening, which is great and needs to continue. Ultimately this can achieve a better outcome for patients, which then has a knock on effect on the families. I also think that being able to collect data beyond the clinical trial programme, for example through registries, and being able to assess the true impact of a product post-clinical trial and post-reimbursement is important.
What do you see as the next steps towards patient access for all people affected by a rare disease?
It’s fascinating, despite being rare it’s a large and heterogeneous area to work in – there are 300 million people worldwide living with a disease, and around 6,000 different rare diseases. So, within that there’s obviously a much smaller patient population for each disease, and for that there needs to be a lot more understanding around the area. I believe that to overcome these issues we need collaborative working between patient groups and clinicians from very early stages, and that bringing everybody together will really help drive things forward, and achieve access quicker than we are seeing today.
So, how can that collaborative working help bring the right treatments to market?
Nobody can do this on their own, we can only do it by working closely with other parties. Understanding the requirements of healthcare bodies, and the impact from a patient and a clinical point of view has to happen at a really early stage. Bringing people together helps enable them to develop what the outcomes will look like, and input these into trial design. It takes a long time to get a product to market, but there’s definitely a drive to bring people into the journey at a much earlier stage.
What more can organisations do, both internally and externally, to expedite access to rare disease drugs?
I believe that the reimbursement process needs to evolve and change, there’s obviously the upcoming NICE methods review as well as the ABPI’s work on rare disease, which Biogen is inputting to. We have a responsibility as an industry to come together and use our voice to drive the change that needs to be seen, to ensure that patients get access as quickly as possible. There’s a number of different industry bodies that could have a collective say, which in turn would result in a stronger voice.
How can rare disease trial design be optimised to counteract low patient enrolment?
It comes back to collaboration – bringing people in at the early stages and ensuring that the patient has the voice. Whether it’s through an advocacy group or through clinical input, we need to ensure that at the early stages more people get in on the journey and actually enrol on the clinical trials.
What are your goals for the future?
This is both personal and work related, because working in this field gets under your skin – we’re lucky to be working in this area because of the potential with the high unmet medical need, and everyone you meet genuinely wants to make a difference. I’d say my goal for the future is to ensure that we all work together within both Biogen and the industry to keep driving change to help increase patient access to rare disease medicine.
What keeps you awake at night?
Apart from my children, it’s the fact that the job is not yet done. It’s been a fascinating few years and we’ve learnt a lot, but by no means do I feel the job is done. We still have an awful lot to do to ensure that we play our part so that patients get access to treatment as quickly as possible. That’s the centre of everything we do at Biogen – our mission is to become pioneers in neuroscience. That’s what keeps me here – that drive to ensure that we’re doing the right thing for patients.
If you could invite anyone (alive or dead) over for dinner, who would it be?
For me it’s a tough call between Ayrton Senna and Jackie Stewart – I love cars and I would love to sit down and talk to some world champions!
