Meet the Rare Bone Disease Research Study team!
![[image description: collage of the rare bone disease study team's headshots]](/wp-content/uploads/2021/04/Meet-the-Rare-Bone-Disease-Study-Team.png "Meet-the-Rare-Bone-Disease-Study-Team!")
Get to know the people behind a new research study that’s looking to learn about life with a rare bone disease!
As part of the MCDS-Therapy consortium project, Newcastle University research fellows are looking to explore the views and preferences of young people and their parents or guardians about available treatments for rare bone conditions known as skeletal dysplasias, such as MCDS.
You can learn more about their research study at our Get Involved page, but for now meet the people who are working tirelessly on the study to make sure your voice is heard!
How did you end up working in Newcastle, UK as a health economist?
Naomi: I was interested in the preference elicitation work for MCDS. I applied for the position at the University of Bologna, Italy and got it! Part of this project is working closely with the team at Newcastle University, so that’s how I got here.
Raj: I came to Newcastle University as a Health Foundation funded Doctoral Research Fellow. I liked the health economics group and the North East so much that I am here even after completing the fellowship a couple of years ago.
Luke V: I came to Newcastle to lead the health economics group in 2011. At that time, the group contained just the six of us. Since then, it has grown to be much larger.
Francesco: I was interested in looking at the world through an economic lens and the synthesis of life has a lot do with health.
Marwa: I studied my master’s in health economics and management at the University of Bologna in 2016. From then on, I have worked as a health economist on different projects and in pharmaceutical companies as well.
![[image description: Naomi headshot]](/wp-content/uploads/2021/04/Naomi-1-300x300.jpg "Naomi (1)")
![[Image description: headshot of Nawaraj B]](/wp-content/uploads/2021/04/Nawaraj-B.jpeg)
What sparked your interest in rare bone diseases? Did you always want to be a health economist?
Naomi: My research interests are in behavioural economics and the study on patient preferences is what drew me to this project. I am now on the path of trying to bring patient preferences into policies. And for the second part, no – but I’m just a health economist by proxy.
Raj: It is something new in terms of research and health economics. My interest in health economics developed while I was doing my undergrad in Public Health back in Nepal and I always focussed on that since then.
Luke V: I was first introduced to health economics as an undergraduate. As a topic area, it seemed interesting, more so than alternatives in other areas in economics. I don’t have a special interest in rare bone diseases, but I do have an interest in focusing on problems that are complex – rare bone diseases is one as there are many causes for a common set of symptoms and the symptoms themselves can have life-long effects. All this makes it challenging to work out which treatments work and for who and whether they are worthwhile from the perspective of the child and society in general.
Francesco: Rare diseases present one of the most challenging and complex areas to study due to the prognostics and that those with a rare disease have a very difficult, sporadic, and challenging (emotionally and physically) journey alongside their families. It is important to serve the needs of these patients in their journey to find an optimal management for their condition and demonstrate the value of treatments for rare diseases. Part of this is what makes the economic analysis for rare diseases so interesting, challenging and valuable.
For the second part, no I’m still considering it, and that fact is what keeps it as a career and profession that I love.
Marwa: My core belief is that access to care and medicine is important to everyone. That is why I was intrigued by the idea of medicine for rare disease.
No, my first degree was a master’s in pharmacy. I was fascinated by the new field of health economics and pharmacoeconomic, which is why I took this career turn.
![[Image description: black and white photo of Luke Vale]](/wp-content/uploads/2021/04/Luke-VALE-300x300.jpg "Luke VALE")
![[image description: headshot of Francesco]](/wp-content/uploads/2021/04/Francesco.jpeg "Francesco")
What’s a typical day like in the life of a research fellow/health economist?
Naomi: Typically, I have meetings in the morning and late at night because of the time zones of my colleagues. During the systematic review process, I was doing a lot of reading and extracting information from the articles. And now, I am focusing on writing and preparing the information for the work we’re doing with the interviews and future survey.
Raj: Each day is different as each project or parts of a project are different to each other- Grant proposals, peer reviews, manuscripts, economic evaluation models, preference elicitations, feasibility trials and tool development, coding, data cleaning, teaching, supervision etc.
Luke V: My day is typically a long series of meetings. Each meeting is on a completely different topic and it would not be unusual to have to think about 10 very different disease areas, population groups and methods over these meetings.
Francesco: It would have been better if you asked what is a typical night.
Marwa: If I am not responding to an email or attending video conferences, I would be writing proposals, searching for some evidence in literature, extracting data for our project or studying a new methodology to handle data.
What other research studies have you been involved in and what did you learn through them?
Naomi: Despite being early in my career, I’ve worked on projects related to behaviour in taxation, crowdfunding, insurance and risk. What I’ve learnt is that information that seems insignificant can change behaviour and that we can use this positively in our policy recommendations.
Raj: I have worked on a number of projects as a health economist; there was/is something new to learn each time. I was/am mostly into health economics methodology, though.
Luke V: I’ve worked in health economics for over 25 years and for many years have expected to work on between 20-30 projects of the size of MCDS at the same time. What I have learned from these projects is that each one is unique and that there is no one-size-fits-all set of methods that should be used.
Francesco: I’ve been involved in projects evaluating insurance practices, preference work for patients and decision makers, health impacts on communities and well-being etc. What I know is that every issue is complex and patient insight is invaluable to policy.
Marwa: I have participated in projects for operation room management, which aim to lean management through increasing efficiency and efficacy in hospital operating rooms. I have also conducted research on market access and authorization of cancer medicine in the Italian market.
What goes into designing, planning and running a research study like the MCDS research project for skeletal dysplasias?
Meetings, lots and lots of meetings 😊
Working on the MCDS project has involved a fantastic team of health researchers from backgrounds in health economics, psychology and behavioural economics. With so many team members and the intricacy of designing the economic and preference work for MCDS, we end up meeting, emailing and reading a lot! This is amongst us and with the broader MCDS network, so we can incorporate clinical and stakeholder input into our economic analysis and preference elicitation design.
How can enrolling in the MCDS research study help MCDS families, especially those who weren’t eligible for the clinical trial?
Something that often gets overlooked until far too late is that healthcare should improve the health (quality of life and length of life) of the people treated. This part of the research seeks to understand what is important to people and how improvements in one aspect of health might be preferred over improvements of other aspects.
Enrolling in our study means that we can use the information families provide us to shape our policy recommendations to clinicians and governing bodies who decide how treatments will be applied. They will also help us ensure that patients and families opinions are integrated into our policy proposals.
![[image description: headshot of Marwa]](/wp-content/uploads/2021/04/Marwa-226x300.png "Marwa")
Say hello to the rest of the Rare Bone Disease Research Study team!
![[image description: collage of the rare bone disease study team's headshots]](/wp-content/uploads/2021/04/Meet-the-Rare-Bone-Disease-Study-Team-36x36.png){kind=small}
