The IMPACT Survey is a joint initiative between the umbrella organization Osteogenesis Imperfecta Federation Europe (OIFE), the OI Foundation & the pharma company Mereo BioPharma. The Survey was developed based on the findings from a systematic literature review which was published in February 2023.

The survey was launched the first week of July 2021 and closed on September 30th 2021. It was available in eight languages: English, Spanish (incl. separate Latin American version), French, German, Italian, Russian, Dutch and Portugese.

Thanks to the impressive help of all our member organizations and the entire OI-community we managed to get more than 2000 unique responses to the survey from more than 65 countries worldwide! Responses came from adults and adolescents with OI as well as parents/caregivers of children with OI. See press release about the completion of the survey enrollment. 

What is the IMPACT survey?

The IMPACT Survey is an international research project exploring the real impact OI has on people’s lives. The IMPACT Survey is aimed at capturing and quantifying the true challenges of life with OI to enable better diagnosis, treatment and care, and to support availability of potential future treatments for OI.

Where can I find the IMPACT survey?

The IMPACT survey is now closed. But a test version can be found here.

 

The deadline to provide answers was expanded from September 10th to September 30th.

 

What were the target groups?

The survey was developed for the following primary target groups:

  • Adults with OI (over 17 years old)
  • Parents of children with OI – who don’t have OI themselves
  • Parents of children with OI who have OI themselves
  • Adolescents with OI (12 – 17 years old)

In addition to the target groups above, there were also a substantial number of responses from

  • Parents of adults with OI
  • Close relatives of people with OI

There were different questions based on which target group you belong to. Adults with OI who had children with OI, could answer both on behalf of themselves and their children.

Who does what?

The survey was run by Wickenstones – an experienced scientific agency who, OIFE, OIF and Mereo Biopharma have engaged to do the work. The overall process is governed by a Steering Committee with representatives of OIFE and OIF as well as expert OI physicians from Europe and the US/Canada treating children and adults.

What will the data be used for and how will it be stored?

The data was gathered on a totally confidential and anonymous basis, with no personal identifiable information. It will be securely stored by Wickenstones according to Data Protection Regulations. The data is now being analyzed according to the plan developed by the Steering Committee, to prepare 5-6 proposed central publications on the impact of OI. in peer-reviewed, scientific journals.

Systematic scoping review published February 2023 shows knowledge gaps

In February 2023 an open access article called “The patient clinical journey and socioeconomic impact of osteogenesis imperfecta: a systematic scoping review” was published in Orphanet Journal of Rare Diseases. The purpose of this review was to capture and quantify the published evidence relating specifically to the clinical, humanistic, and economic impact of OI on individuals, their families, and wider society. Representatives from OIFE were included as co-authors.

The review showed that bone conditions, anthropometric measurements, oral conditions, diagnostic techniques, use of pharmacotherapy, and physical functioning of adults and children with OI were well described.

However, few records included current care practice, diagnosis and monitoring, interactions with the healthcare system, or transition of care across life stages. Limited data on wider health concerns beyond bone health, how these concerns may impact health-related quality of life, in particular that of adult men and other family members, were identified.

The patient organizations will own the data

Once the first analysis is finished in 2024/2025, the anonymous data will be transferred to a Data Management Committee, comprised of OIFE and OIF, and Dr. Frank Rauch (chair). In agreement with OIFE and OIF, data will also be shared with Mereo and its partners for planning potential pathways for OI-treatments being evaluated by national payer authorities. Under no circumstances will the information be used for anything without the permission of the Data Management Committee. This basically means that the organizations will decide what the data can and should be used for after the project.

Countries with enough respondents will receive national reports

The collected aggregated and consolidated results for each country will also be given to the national OI member associations in the countries to use in local policy work, for instance when lobbying for multidisciplinary care or adult clinics. The more people who answered the survey in a local country, the stronger the data that can be used for later policy work is. A minimum of 20 participants per country was needed to fill in the survey before a national report or some kind of summary per country level could be generated.

Why is lack of data a problem in rare conditions like OI?

Rare conditions like OI have few people and we are spread around the world. There is also a great variety within the condition. The reviewers from the European Medicines Agency (EMA) and U.S. Food & Drug Administration (FDA) may have to make their decision based on relatively small studies and with a limited understanding of the natural history or progression of the condition. The reliability and quality of the data are critical for the decision making of the EMA and FDA. The same goes for the medicinal (health technology assessment) authorities in each country. This is why bigger and better data is needed.

We think this educational video from the rare disease umbrella NORD explains it in a good way. The video only refers to the FDA (USA), but the system is quite similar to the European one regarding this stage of the process.

Better data for access to treatment and policy work

Headshot of dr. Frank RauchEven if the survey was originally initiated by Mereo Biopharma, the OIFE and OIF fully supported and took ownership of the project, because we regarded it as mutual beneficial. Not only can the data be used in processes connected to Mereo’s work, but it will also be helpful for other future potential treatments being developed by other companies and projects and for the policy work in the national organizations. Several rare disease federations are doing similar projects, simply because they see that access to orphan drugs relies on bigger and better data.

“The importance of these data cannot be overstated. The learnings directly from the OI community will help shape and improve care in a truly meaningful manner.” commented Dr Frank Rauch, Professor of Pediatrics at McGill University, and Chair of the IMPACT Survey Steering Committee.

What are the findings?

The article about the scoping review was published in February 2023 and the remaining articles will be published in 2024 and 2025. We have permission to share this graphic, which show the most common health conditions in adults with OI (in a 12 month period prior to answering).  Pain, fatigue and soft tissue problems are the most common problems, while fractures are nr. 13 for the adult population.

Hashtag #IMPACTsurveyOI

Are you posting about the IMPACT survey on social media? Please use the hashtag #IMPACTsurveyOI

Presentations and videos about the IMPACT survey

 

Questions?

Don’t hesitate to send us an email to office@oife.org if you should have any questions about the project!

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