The IMPACT Survey – how we identified knowledge gaps and created a meaningful survey for the OI-community

The IMPACT Survey was an international research project exploring the real impact OI has on people’s lives. The survey was run by Sam and Maria from the Wickenstones Company – an experienced scientific agency who, OIFE, OIF and Mereo Biopharma have engaged to do the work. Five articles will be written with the support of Wickenstones and the first one about the initial literature search and scoping review has already been published in Orphanet journal.

Who are you & what is your relationship to OI?

Maria:
I am an employee at a market access agency – so I came to OI completely by accident. A few years ago, I started a new job and the IMPACT Survey was one of the first projects I worked on. Before working on IMPACT and with the patient community I knew very little about the condition OI, but now this work has become my favourite task at work.

Sam:
I am a market access consultant, working with pharmaceutical companies to help bring new medicines and interventions to patients with all types of conditions. Over the last 3 years, I have been heading up the IMPACT Survey, and had the great opportunity to learn a lot about OI and to working closely with OI clinical experts and community members.

What is your professional background?

Maria: I have a Master’s degree in Immunology. After finishing my degree, I wanted to work outside of academia but stay close to scientific work, so I started to work as a scientific writer. It felt like the best of both worlds – reading lots of scientific papers, while having more opportunities than academia might offer.

Sam: I have a PhD in the field of medical and molecular genetics from the University of Birmingham where I was conducting focussed research on a very rare inherited condition called Wolfram syndrome. I am fascinated by biology and medicine, particularly rare genetic conditions. Back in the lab, I got to work on the basics of understanding how diseases come about. These days, I now work to apply my background knowledge and interests in a field where I can contribute to people getting access to new and innovative treatments, which is very rewarding.

What does a company like Wickenstones do?

Wickenstones is a global ‘market access’ consultancy, which means we help clients from all around the globe bring new medicines to patients who need them. Our work involves a whole spectrum of things, including planning strategies for rolling out new treatments, gathering data and evidence to understand whether new drugs will meet an existing need, helping to communicate the value of medicines to the people who make decisions about their availability, and looking at ways of getting around specific barriers to patient access in different countries, whether those be economic, clinical or policy-related challenges.

Tell us about the background for the scoping review!

When we started to plan the IMPACT Survey it very quickly became clear that we needed to understand what was known about the condition OI and the OI community. To make IMPACT as useful as possible we wanted to know where the gaps were in the published scientific literature. IMPACT was designed based on the findings of the review and includes many questions we would have never thought to ask if we had not done the review first. The review was systematic – which means that we set out stringent criteria for the search and selection of relevant publications to make sure to capture everything.

Did you search in grey literature – what is it?

Grey literature is any literature that isn’t published in peer reviewed scientific journals. Peer review means when scientists submit their research for publication, other experts in the field will review their submission to make sure that it is sufficiently high quality. So grey literature is basically everything else – for example information from patient organisations, healthcare providers and charities. While such information cannot replace scientific publications, it can help scientists to understand what patients are actually concerned about and inform future research, as it did in this project.

How was it financed?

The review and the survey were both financed by Mereo BioPharma, a pharmaceutical company developing a new treatment for OI.

How did you use the scoping review results when creating the survey?

When we finished the review, we made a list of all the gaps we had found in the research. For some topics we had only found very few datapoints, or only insights from interviews with patients. For others, only information on some patient groups but not others, was available (for example only research in children but not adults). We discussed the gaps with everyone involved in the project, OI researchers and patients, to understand which ones were most relevant. Then we considered which topics were most suitable to research in an online survey. Not every question can be answered through survey data, but many can be. For example, questions about the experience of people with OI and their families can be answered in this way.

What are the challenges of doing a good literature search? How did you choose the right keywords?

The searches are actually more straight forward than one would think. Scientific literature is organised in databases that have good tools for searching and with some experience and trial and error, one can be pretty certain to find the right information. The real challenge with this work was to make sense of our search results. We had over 200 reports to read and sort into clear categories to make sense of what information they contained. This can be quite tricky, because a lot of research in the field is based on interviews with patients, and while those interviews brought up tons of interesting information it was difficult to pull out information from such research and get an overview.

What were your most interesting findings?

Identifying the biggest knowledge gaps was the most interesting aspect of the review, particularly when we talked to experts and community members about the things they felt were most important to the medical and OI communities. This was invaluable for shaping the survey questions.

What are the most important knowledge gaps?

There’s a larger volume of research into bone health and the clinical aspects of OI compared to other topics. Significant data gaps exist around the socio-economic impact of OI on people with OI, their families, and wider society. There is also a lack of data on wider concerns beyond bone health, access and standards of OI healthcare and how health-related quality of life changes throughout life stages. There is also an underrepresentation of males with OI, young adults, and siblings of children with OI. Here you can read the open access article called “The patient clinical journey and socioeconomic impact of osteogenesis imperfecta: a systematic scoping review”.

Were there any surprising findings?

The lack of data on some of the things people say are very important, such as pain.

Why do you think we need socioeconomic data on OI?

Socioeconomic data serves to highlight the unmet needs of the OI community in terms of expenses incurred, access to services and care, and other financial burdens that people and their families may experience. Gathering this information enables decision-makers to better understand the value of interventions, policies, and healthcare access for the future.

What will come out of IMPACT in 2023 and 2024?

The high volume of responses means there are a lot of data to process. Over the coming year or two, the key findings of IMPACT from a global perspective will be submitted for publication in scientific journals and at some relevant congresses and meetings. The topics will cover the methods and demographics, economic impact, clinical and quality of life impact, and healthcare journey (care pathways) and experiences. Once the main findings have been shared, there will be many opportunities to look at the data from a country-specific perspective or focus down on certain questions or topics.

Did you expect as much as 2300 responses and has it created any challenges?

No, not at all! When we were conducting the literature review the biggest survey projects had a few hundred participants, so this is what we hoped we could achieve as well. When we hit 150 after a few days, I was stoked, even more so when we hit 500. But I did not expect the numbers to keep climbing the way they did. This has really only been possible because of the contribution of the OIFE and OIF, specifically Ingunn Westerheim, and Tracy Hart, who put so much effort into recruiting community members. The incredible response we got is of course a huge success – it means that the findings coming out for the survey will be more robust. It also allows us to analyse the data in more complex ways. However, it is also challenging. The survey is written to accommodate many different ages and respondent types which means that to be able to get an answer to a question, you first have find the relevant respondents within the database. Also the survey was available in 8 different languages, which is what enabled us to get this many respondents, but it of course also adds another layer of complexity, in both designing the survey and interpreting responses. For example, the same medicine may be called different things in different countries, and healthcare is organised in different ways. This means that when writing the survey (and now interpreting the results) we need to be mindful of that to make sure that our respondents understand the questions and we correctly interpret their answers.

In which ways did you have input from people with OI in the process?

We had input from people with OI throughout the entire process. There are members of the OI community on our project Steering Committee, who have provided invaluable insights and guidance to help us develop questions that are important and meaningful to people with OI and their families. We also had volunteers with OI from around the globe, who kindly gave up their time to review the survey in all 8 translations, help us to understand more about country-specific elements we may wish to consider, and to pilot test the electronic survey platform to make sure it worked. A whole network of community volunteers was also involved in survey roll out and recruitment, spreading the word and encouraging people to take part and have their say.

Any messages for the readers of OIFE Magazine?

Thank you! This project has been ground-breaking – the success of this initiative is down to the input, time and efforts of the community members who got involved.

Follow our dedicated project page for more news and updates from the IMPACT survey!

This interview (except the Stockholm photos) was first published in OIFE Magazine 2-2023.