Tracy Hart (CEO OIF) and Ingunn Westerheim (OIFE president) in 2017.

The IMPACT Survey is a joint initiative between OIFE, OIF & Mereo BioPharma. The survey will be launched in summer 2021 and we will need the help of all our member organizations and the OI-community to spread the word and encourage as many people with OI (and parents) to fill in the survey. 

Written by Ingunn Westerheim (OIFE)

It is an exciting time in the field of research into potential new treatments for OI. For many years, bisphosphonates have been our only therapy option but, recently, several different companies have started looking at potential new treatments specifically designed for OI.

But can we be sure that these new potential therapies will be accessible for people living with OI? No, certainly not. There are many potential obstacles. And to make sure we have strategies in place to meet those future obstacles and challenges, we need to start our preparations now. And we need to work together. This is one of the reasons we have launched the IMPACT Survey.

What is needed and why?

Developing a new treatment for a rare condition is a long process, where every step of the way is scrutinised by regulatory authorities (EMA and FDA) to make sure that any new potential medicine is safe, as well as proven to work. Only once the regulatory authorities have reviewed the scientific data and are convinced about this, will they give the OK for a new medicine to be sold, granting what is called a “Marketing Authorisation” or a “License”.  When granted, the company in question has permission to offer the new treatment in their country or region – like the USA or the EU.

But while those steps are important, it is only the first of several, because each individual country provides their own healthcare system with their own priorities and processes. To decide what they will pay for, each country have their own special national systems to evaluate each and every new treatment before they decide if they want to pay for it or not.

The countries review additional scientific data on top of the Marketing Authorisation/ License, to understand about the condition; and how good the new treatment is at addressing the impact of that disease or condition. They look at what the condition costs their healthcare system each year (ex: fractures, hospital visits, surgeries etc); and they compare the cost for the new medication with potential savings and/or healthcare benefits of the new treatment. The different national authorities might ask for completely different information than the EMA and FDA. It can even differ between regions within a country! This means we need to develop as good data as possible both internationally and nationally.

Infographic describing vaccine development process

How can the OI-community get involved?

The first thing that we can do, is to volunteer to participate in clinical trials, to have bigger numbers and better documentation. But not everyone can do this of course. We also have a role to play when the regulatory authorities ask our opinion. Representatives from OIFE have for instance participated in formal scientific review meetings with the European Medicines Agency on several occasions.

Last but not least, we have a role to play when our national healthcare systems will evaluate the impact that a condition has on our lives as well as the healthcare systems.  And this is where the IMPACT Survey comes in as a first step. The scientific name being “Living with Osteogenesis Imperfecta: understanding experiences based on community Insight & evidence.”

Since bisphosphonates have always been given off label, and have not been specifically approved for OI (except in Italy and Japan) they have never been through the process that new medicines go through. As a consequence, both the EMA/FDA as well as national agencies do not really know a lot about OI.

With the IMPACT Survey, we are aiming to get a scientifically validated and published set of data that will show to the healthcare systems the impact that OI has, on people and also on the economics of the healthcare system. This will be important because when they will evaluate any new treatment, they will need to have a “baseline” to compare the new treatment against. The more people who answer the one time survey, the stronger documentation and data we will have.

Who is doing what? 

The survey will be run by Wickenstones – an experienced scientific agency who Mereo 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 treating children and adults.

The data will be 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 will then be analysed according to the plan developed by the Steering Committee, to prepare the first publication. Once the first analysis is finished, 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, the results 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.

So what will happen, when?

The survey will be launched in summer 2021 and we will need the help of all our member organizations and the OI-community to spread the word and encourage as many people with OI (and parents) to fill in the survey. A communication package will be put together in several different languages and shared with the national OI-organizations to assist in this process.

Since IMPACT will be a global project, the survey and communication materials will be available not only in English but also in Dutch, French, German, Italian, Portuguese, Spanish and possibly some more languages. The survey will be open for 2-3 months and once it’s completed, the results will be collected on an anonymous basis and put forward for publication in a peer-reviewed, scientific journal. This means that once the data is published, it becomes “official”, and becomes something that the national healthcare systems can formally refer to.

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. This means, that the more people who answer the survey in a local country, the stronger the data that can be used for later policy work, will be.

A joint project with mutual benefit

Even if the survey was originally initiated by Mereo Biopharma, the OIFE and OIF fully supports the project, because we regard 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 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. This is why we strongly encourage you to support the project – and make it the biggest and best OI-survey possible. Bigger and better data matters – and with your help we can develop it. The IMPACT survey will be one of the main topics at the OIFE AGM on June 19th. But don’t hesitate to ask if you should have any questions about the project before that time.